By Jill U. Adams, Special to the Los Angeles Times
The FDA has approved a new study aimed at fighting dry age-related macular degeneration.
About 10 million Americans suffer some degree of vision loss caused by age-related macular degeneration, and that figure is expected to grow as more baby boomers become senior citizens. There is no cure for the disease, but last week the U.S. Food and Drug Administration gave a green light to an unusual clinical trial that seeks to restore patients' sight by employing human embryonic stem cells.
None of the stem cells will be injected into patients; instead, they are grown into another kind of cell that will be delivered to the back of the eye, where the retina is damaged by the disease. The hope is that the cells will help repair the damaged retinal tissue.
The company behind the trial, Santa Monica-based Advanced Cell Technology Inc., developed the therapy to treat Stargardt's macular dystrophy, a rare childhood version of macular degeneration that affects about 1 in 10,000 kids. The FDA gave the company permission to test the therapy in Stargardt's patients in November. However, if they work, the cells would have a much bigger effect as a treatment for age-related macular degeneration.
Here's a closer look at the disease and the new therapy.
What is age-related macular degeneration?
Age-related macular degeneration is the leading cause of vision impairment and blindness among people who are 65 and older, says Dr. Jose Pulido, an ophthalmologist at the Mayo Clinic in Rochester, Minn. The dry version of the disease begins with tiny deposits of fat and protein — called drusen — that appear in the center of the retina, called the macula. As the deposits grow in number and size over the course of years, things begin to look blurry in the center of a person's field of vision. As the disease worsens, the blurriness may progress to a blind spot.
What causes it?
The main problem is that light-sensing cells in the macula, called photoreceptors, slowly break down. This is thought to be caused by the loss of another population of cells, called retinal pigment epithelial (RPE) cells, which support the photoreceptors in a number of ways.
Among other things, the RPE cells release growth factors important for photoreceptors to thrive. "The RPE are also the garbage trucks of the retina," removing toxic byproducts that the retina makes as it performs its light-sensing function, says Stephen Rose, chief research officer for the Foundation Fighting Blindness, a fundraising organization based in Columbia, Md.
Dry age-related macular degeneration can also progress into wet age-related macular degeneration, in which blood vessels grow abnormally and leak fluid into the macula. It's a much more aggressive form of the disease, but it's also more treatable.
How can human embryonic stem cells help?
The stem cells are grown into healthy replacement RPE cells and injected into the retina, says Gary Rabin, Advanced Cell Technology's chief executive. The company hopes the lab-grown cells will replace the dying RPE cells and keep vision intact — or even restore it to some degree.
"We've had incredible success with this in animal studies," Rabin says. A study published in the journal Stem Cells found that the RPE cells restored eye function in sick mice and rats to "near-normal levels," and another study in Cloning and Stem Cells reported that the treatment improved vision in affected rats until it was 70% as good as that of healthy animals.
Of course, success in animal studies doesn't always translate to humans. In addition, the eyes of people in their 50s and 60s likely present "a very different milieu for the RPE cells to try to hook onto," Pulido says.
Rose adds that the treatment, if it works, wouldn't amount to a cure because it doesn't address the reason why RPE cells deteriorate in the first place. But it would buy time for patients, delaying vision loss for perhaps years. "That's huge," he says.
What will the new trial assess?
The FDA granted permission to conduct a Phase I/II clinical trial, which is essentially a safety trial, that will involve a dozen patients. The first patients will get a very low "dose" of cells — 50,000 — and will be monitored for any untoward effects.
"If there are no safety issues after three-ish months, we will increase the dose [to a level where we] hope to see efficacy," Rabin says. "We anticipate that the photoreceptor cells will awaken and that there will be a gradual increase in visual acuity over time." For now, the protocol calls for a one-time treatment of up to 200,000 cells.
Aren't there ethical concerns about using human embryonic stem cells?
Generally speaking, many people are troubled by research involving human embryonic stem cells because they are typically made by dismantling — and thus destroying — embryos that are a few days old.
Advanced Cell Technology uses a proprietary technique to extract a single cell from a young embryo, allowing the rest to remain intact and develop normally, Rabin says. A similar method is routinely used to biopsy embryos for pre-implantation genetic diagnosis, in which embryos created through in vitro fertilization are scanned for genetic disorders before being transferred to a uterus.
Showing posts with label gene therapy. Show all posts
Showing posts with label gene therapy. Show all posts
Saturday, January 15, 2011
Sunday, January 9, 2011
FDA approves embryonic stem cells to reverse macular degeneration
by:Admin
Sacramento is now the hub of stem-cell research focusing on regenerative medicine. See the article, UC Davis: Stem Cell Research. After receiving $62 million for stem cell research last year, the new UC Davis Institute for Regenerative Cures opened. And the center already is testing dozens of therapies in the laboratory. The center will bring 200 scientists and laboratory personnel together under one roof. Check out the UC Davis Stem Cell Institute. See UC Davis Stem Cell Program. And check out the site, UCDMC Stem Cell Research News.
Now that the FDA has approved embryonic stem cells today, to help reverse certain types of macular degeneration, consumers should know that the stem cell taken from an embryo does not destroy the embryo. Just a single stem cell is taken from the embryo. Then the embryo continues to thrive and is not destroyed.
California sites currently under consideration for the trials include the Jules Stein Eye Institute at UCLA and the Ophthalmology Department at Stanford University. Check out the government site listing clinical trials, Clinical Trials.gov.
Also, in the Sacramento and Davis regional area, did you know that the University of California is recruiting for or has completed at least 193 clinical trials on various types of health studies ranging from the health benefits of ground flax seeds to stem cell research? Also see the January 4, 2011 news article, Read: Vitamin Drug Could Stop Dry Macular Degeneration.
Concerning studies at another university on macular degeneration and stem cell research, if you're interested in stem cell research for macular degeneration, the breaking news is that the FDA has just approved the use of stem cells to treat certain types of macular degeneration. According to a January 4, 2011 news article, "FDA Approves Stem Cell Treatment Trial for AMD-Related Vision Loss," the US Food and Drug Administration (FDA) has approved a clinical trial of human embryonic stem-cell treatments on patients who have suffered vision loss related to dry age-related macular degeneration (AMD).
Advanced Cell Technology of Massachusetts will begin a Phase I/II open-label study on twelve patients at multiple clinical sites to determine the safety and tolerability of the treatment. The dry version of macular degeneration is a leading cause of blindness in older adults. Dry age-related macular degeneration is one of two forms of an eye disease that breaks down retinal pigment epithelial (RPE) cells in the macula of the retina, a layer of light-sensitive tissue at the back of the eye. Progressive loss of RPE cells and the accompanying loss of photoreceptors can cause severe vision loss. There are no current treatments available for AMD.
Dry AMD is the leading cause of blindness in individuals over the age of 55, afflicting approximately 10 million people in the US. And as the population ages, according to the article, "FDA Approves Stem Cell Treatment Trial for AMD-Related Vision Loss."
In the clinical trials and approved experiments, patients will receive 50,000 to 2,000,000 RPE cells derived from human embryonic stem cells to replace those lost due to AMD. While human embryonic stem cell use is controversial, ACT maintains that their cells are derived from a single-cell extraction technology that “does not destroy the embryo.” Also read the article, Read: Smoking Raises Risk of Macular Degeneration.
Sacramento is now the hub of stem-cell research focusing on regenerative medicine. See the article, UC Davis: Stem Cell Research. After receiving $62 million for stem cell research last year, the new UC Davis Institute for Regenerative Cures opened. And the center already is testing dozens of therapies in the laboratory. The center will bring 200 scientists and laboratory personnel together under one roof. Check out the UC Davis Stem Cell Institute. See UC Davis Stem Cell Program. And check out the site, UCDMC Stem Cell Research News.
Now that the FDA has approved embryonic stem cells today, to help reverse certain types of macular degeneration, consumers should know that the stem cell taken from an embryo does not destroy the embryo. Just a single stem cell is taken from the embryo. Then the embryo continues to thrive and is not destroyed.
California sites currently under consideration for the trials include the Jules Stein Eye Institute at UCLA and the Ophthalmology Department at Stanford University. Check out the government site listing clinical trials, Clinical Trials.gov.
Also, in the Sacramento and Davis regional area, did you know that the University of California is recruiting for or has completed at least 193 clinical trials on various types of health studies ranging from the health benefits of ground flax seeds to stem cell research? Also see the January 4, 2011 news article, Read: Vitamin Drug Could Stop Dry Macular Degeneration.
Concerning studies at another university on macular degeneration and stem cell research, if you're interested in stem cell research for macular degeneration, the breaking news is that the FDA has just approved the use of stem cells to treat certain types of macular degeneration. According to a January 4, 2011 news article, "FDA Approves Stem Cell Treatment Trial for AMD-Related Vision Loss," the US Food and Drug Administration (FDA) has approved a clinical trial of human embryonic stem-cell treatments on patients who have suffered vision loss related to dry age-related macular degeneration (AMD).
Advanced Cell Technology of Massachusetts will begin a Phase I/II open-label study on twelve patients at multiple clinical sites to determine the safety and tolerability of the treatment. The dry version of macular degeneration is a leading cause of blindness in older adults. Dry age-related macular degeneration is one of two forms of an eye disease that breaks down retinal pigment epithelial (RPE) cells in the macula of the retina, a layer of light-sensitive tissue at the back of the eye. Progressive loss of RPE cells and the accompanying loss of photoreceptors can cause severe vision loss. There are no current treatments available for AMD.
Dry AMD is the leading cause of blindness in individuals over the age of 55, afflicting approximately 10 million people in the US. And as the population ages, according to the article, "FDA Approves Stem Cell Treatment Trial for AMD-Related Vision Loss."
In the clinical trials and approved experiments, patients will receive 50,000 to 2,000,000 RPE cells derived from human embryonic stem cells to replace those lost due to AMD. While human embryonic stem cell use is controversial, ACT maintains that their cells are derived from a single-cell extraction technology that “does not destroy the embryo.” Also read the article, Read: Smoking Raises Risk of Macular Degeneration.
Sunday, January 2, 2011
Genetic Testing for AMD is here Today
By; Diana Shechtman OD FAAO & Steven Ferrucci OD FAAO
Age-Related macular degeneration (AMD) is a progressive disease and the leading cause of vision loss among the elderly, affecting central vision required for daily activities such as driving and reading. There are a number of factors affecting AMD, such as advanced age, smoking, UV exposure, overall health (that contribute to high blood pressure, obesity, diet) and family history. Many factors may be modified and yet others like genetics cannot. Although AMD may seem to be hereditary in some families and not others, genetics have been shown to contribute significantly to the disease. Multiple twin and sibling studies have collaborated to the familial nature of the disease. First-degree relatives of patients with AMD are at a significantly increased risk for the disease. Furthermore, large epidemiological studies have suggested a strong genetic risk factor for AMD. In fact, the risk of developing AMD increases 4 fold among patients with a positive family history. In 2005 a breakthrough occurred in the area of genetic research and AMD; Klein and associates discovered a strong link between AMD and certain genetic variants. Similarly, numerous other genes have been implicated in AMD, which may increase the risk of AMD up to 70%.
Until recently there was no test to help determined patient’s inherited risk for AMD. Today, Macula Risk (ArcticDX, Toronto, Ontario) is a genetic test specifically designed to determine genetic predisposition to AMD and vision loss attributed to the more advanced stage of the disease.
Macula Risk genetic test separates individuals into one of 5 macula risk (MR) categories, with MR 3 through 5 representing an increased risk for the more advance stage of the disease. This accounts for approximately 20% of the general population. MR1 has less than a 5% risk of the advanced stage of the disease, while MR 5 carries greater than 55% risk. These results can aid the doctor in devising a specific management plan and follow-up protocol in order to reassure early intervention to prevent vision loss.
The test only requires a simple in-office cheek swab, which is sent directly to the genetic lab. The report includes test results and written genetic support information (including access to genetic counseling). Macula Risk genetic testing is covered by most insurance providers, including Medicare, as long as the specific diagnosis (ICD-9) are identified by the doctor. The prognostic genetic test is intended for patients who have a diagnosis of early or intermediate AMD. Thus, the “at risk patient,” would have to pay a fee for the test.
Despite our best efforts and new treatment options available today, many people are still losing vision from AMD. New advancements in the area of AMD are becoming an integral part in preventing future visual deterioration. AMD is affected by both environmental and genetic factors that interact with one another to determine prevalence and progression of the disease. Furthermore, at this time we do not know all of the genes linked to AMD. Hence, genetic testing in the area of AMD is only a risk indicator and cannot predict “without a shadow of a doubt,” which patients will and which will not develop the disease. However, this test provides a genetic profile screening to help identify those at risk as well as aids in tailoring a distinct management approach for those with the disease. With various researches devoted to treatment options for AMD, genetic testing in the area of AMD is at the frontier of providing crucial information.
REFERENCES
1. Swaroop, Branham KE, Chen W, Abecasis G. Genetic susceptibility to age-related macular degeneration: a paradigm for dissecting complex disease traits. Hum Mol Genet 2007; 16: 174-82.
2. Klein ML, Mauldin WM, Stoumbos VD. Heredity and age-related macular degeneration. Observations in monozygotic twins. Arch Ophthalmol. 1994; 112: 932-7.
3. Meyers SM, Greene T, Gutman FA. A twin study of age-related macular degeneration. Am J Ophthalmol. 1995; 120: 757-66.
4. Heiba IM, Elston RC, Klein BE, et al. Sibling correlations and segregation analysis of age-related maculopathy: The Beaver Dam Eye Study. Genet Epidemiol. 1994; 11: 51-67.
5. Klaver CC, Wolfs RC, Assink JJ, et al. Genetic risk of age-related maculopathy. Population-based familial aggregation study. Arch Ophthalmol. 1998; 116: 1646-51.
6. Seddon JM, Ajani UA, Mitchell BD. Familial aggregation of age-related maculopathy. Am J Ophthalmol. 1997; 123: 199-206.
7. Klein RJ, Zeiss C, Chew EY, et al. Complement Factor H Polymorphism in Age-Related Macular Degeneration. Science 2005; 308: 385-389.
8. Seddon JM, Reynolds R, Maller J, Fagerness JA, Daly MJ, Rosner B. Prediction model for prevalence and incidence of advanced age-related macular degeneration based on genetic, demographic, and environmental variables. Invest Ophthalmol Vis Sci 2009; 50 (65): 2044-53.
9. www.macularisk.com/en/physicians/order.html (accessed Dec. 14th , 2010)
Age-Related macular degeneration (AMD) is a progressive disease and the leading cause of vision loss among the elderly, affecting central vision required for daily activities such as driving and reading. There are a number of factors affecting AMD, such as advanced age, smoking, UV exposure, overall health (that contribute to high blood pressure, obesity, diet) and family history. Many factors may be modified and yet others like genetics cannot. Although AMD may seem to be hereditary in some families and not others, genetics have been shown to contribute significantly to the disease. Multiple twin and sibling studies have collaborated to the familial nature of the disease. First-degree relatives of patients with AMD are at a significantly increased risk for the disease. Furthermore, large epidemiological studies have suggested a strong genetic risk factor for AMD. In fact, the risk of developing AMD increases 4 fold among patients with a positive family history. In 2005 a breakthrough occurred in the area of genetic research and AMD; Klein and associates discovered a strong link between AMD and certain genetic variants. Similarly, numerous other genes have been implicated in AMD, which may increase the risk of AMD up to 70%.
Until recently there was no test to help determined patient’s inherited risk for AMD. Today, Macula Risk (ArcticDX, Toronto, Ontario) is a genetic test specifically designed to determine genetic predisposition to AMD and vision loss attributed to the more advanced stage of the disease.
Macula Risk genetic test separates individuals into one of 5 macula risk (MR) categories, with MR 3 through 5 representing an increased risk for the more advance stage of the disease. This accounts for approximately 20% of the general population. MR1 has less than a 5% risk of the advanced stage of the disease, while MR 5 carries greater than 55% risk. These results can aid the doctor in devising a specific management plan and follow-up protocol in order to reassure early intervention to prevent vision loss.
The test only requires a simple in-office cheek swab, which is sent directly to the genetic lab. The report includes test results and written genetic support information (including access to genetic counseling). Macula Risk genetic testing is covered by most insurance providers, including Medicare, as long as the specific diagnosis (ICD-9) are identified by the doctor. The prognostic genetic test is intended for patients who have a diagnosis of early or intermediate AMD. Thus, the “at risk patient,” would have to pay a fee for the test.
Despite our best efforts and new treatment options available today, many people are still losing vision from AMD. New advancements in the area of AMD are becoming an integral part in preventing future visual deterioration. AMD is affected by both environmental and genetic factors that interact with one another to determine prevalence and progression of the disease. Furthermore, at this time we do not know all of the genes linked to AMD. Hence, genetic testing in the area of AMD is only a risk indicator and cannot predict “without a shadow of a doubt,” which patients will and which will not develop the disease. However, this test provides a genetic profile screening to help identify those at risk as well as aids in tailoring a distinct management approach for those with the disease. With various researches devoted to treatment options for AMD, genetic testing in the area of AMD is at the frontier of providing crucial information.
REFERENCES
1. Swaroop, Branham KE, Chen W, Abecasis G. Genetic susceptibility to age-related macular degeneration: a paradigm for dissecting complex disease traits. Hum Mol Genet 2007; 16: 174-82.
2. Klein ML, Mauldin WM, Stoumbos VD. Heredity and age-related macular degeneration. Observations in monozygotic twins. Arch Ophthalmol. 1994; 112: 932-7.
3. Meyers SM, Greene T, Gutman FA. A twin study of age-related macular degeneration. Am J Ophthalmol. 1995; 120: 757-66.
4. Heiba IM, Elston RC, Klein BE, et al. Sibling correlations and segregation analysis of age-related maculopathy: The Beaver Dam Eye Study. Genet Epidemiol. 1994; 11: 51-67.
5. Klaver CC, Wolfs RC, Assink JJ, et al. Genetic risk of age-related maculopathy. Population-based familial aggregation study. Arch Ophthalmol. 1998; 116: 1646-51.
6. Seddon JM, Ajani UA, Mitchell BD. Familial aggregation of age-related maculopathy. Am J Ophthalmol. 1997; 123: 199-206.
7. Klein RJ, Zeiss C, Chew EY, et al. Complement Factor H Polymorphism in Age-Related Macular Degeneration. Science 2005; 308: 385-389.
8. Seddon JM, Reynolds R, Maller J, Fagerness JA, Daly MJ, Rosner B. Prediction model for prevalence and incidence of advanced age-related macular degeneration based on genetic, demographic, and environmental variables. Invest Ophthalmol Vis Sci 2009; 50 (65): 2044-53.
9. www.macularisk.com/en/physicians/order.html (accessed Dec. 14th , 2010)
Monday, December 20, 2010
New Investment to Boost Gene Therapy Development
National Neurovision Research Institute (NNRI), the Foundation Fighting Blindness’ clinical trial support organization, announced today that one of its key partners, biopharmaceutical company Oxford BioMedica, has established a collaboration with sanofi-aventis, a major international pharmaceutical company, to develop and commercialize gene therapy treatments for vision-robbing retinal degenerative diseases that affect tens of millions of people around the world.
The collaboration will significantly bolster the development of the following gene therapy products: StarGen™ for the treatment of Stargardt disease, a form of early-onset macular degeneration; UshStat™ for the treatment of Usher syndrome, the leading cause of deaf-blindness; and RetinoStat® for the treatment of age-related macular degeneration, the leading cause vision loss in people 55 and older in developed countries.
The development of treatments for these and other retinal degenerative diseases is a key goal of the National Neurovision Research Institute and is the basis for its partnership with Oxford BioMedica. In 2006, Paul and Diane Manning, with the National Neurovision Research Institute, established a partnership with Oxford BioMedica to advance gene therapies for these and other related retinal degenerative diseases.
“The investment from sanofi-aventis is a wonderful boost for the development of Oxford BioMedica’s gene therapy products and will greatly enhance our ability to move these emerging treatments into and through the clinical trial process,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “This collaboration affirms the great potential for gene therapy to treat and cure a number of retinal degenerative diseases including Stargardt disease and Usher syndrome that, as rare diseases, often do not receive the attention or investment necessary to bring about promising treatments.”
“An important goal of the Foundation, through NNRI, is to attract investment from large pharmaceutical companies for the development and production of treatments for inherited retinal disease,” says Morton Goldberg, M.D., chairman of the board of NNRI. “The NNRI-Oxford partnership is an excellent model of how NNRI collaborations can accelerate the translation of laboratory-based research into clinical trials, ultimately getting successful treatments for rare diseases like inherited retinal degenerations to the market and out to the people who need them.”
Based on the agreement, Oxford BioMedica will receive an upfront payment of $26 million and a further $24 million from sanofi-aventis over a three-year period.
The treatments will utilize Oxford BioMedica’s LentiVector® gene delivery technology to deliver healthy vision-saving genes to the retina. For more information on this technology, visit www.oxfordbiomedica.co.uk/
The collaboration will significantly bolster the development of the following gene therapy products: StarGen™ for the treatment of Stargardt disease, a form of early-onset macular degeneration; UshStat™ for the treatment of Usher syndrome, the leading cause of deaf-blindness; and RetinoStat® for the treatment of age-related macular degeneration, the leading cause vision loss in people 55 and older in developed countries.
The development of treatments for these and other retinal degenerative diseases is a key goal of the National Neurovision Research Institute and is the basis for its partnership with Oxford BioMedica. In 2006, Paul and Diane Manning, with the National Neurovision Research Institute, established a partnership with Oxford BioMedica to advance gene therapies for these and other related retinal degenerative diseases.
“The investment from sanofi-aventis is a wonderful boost for the development of Oxford BioMedica’s gene therapy products and will greatly enhance our ability to move these emerging treatments into and through the clinical trial process,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “This collaboration affirms the great potential for gene therapy to treat and cure a number of retinal degenerative diseases including Stargardt disease and Usher syndrome that, as rare diseases, often do not receive the attention or investment necessary to bring about promising treatments.”
“An important goal of the Foundation, through NNRI, is to attract investment from large pharmaceutical companies for the development and production of treatments for inherited retinal disease,” says Morton Goldberg, M.D., chairman of the board of NNRI. “The NNRI-Oxford partnership is an excellent model of how NNRI collaborations can accelerate the translation of laboratory-based research into clinical trials, ultimately getting successful treatments for rare diseases like inherited retinal degenerations to the market and out to the people who need them.”
Based on the agreement, Oxford BioMedica will receive an upfront payment of $26 million and a further $24 million from sanofi-aventis over a three-year period.
The treatments will utilize Oxford BioMedica’s LentiVector® gene delivery technology to deliver healthy vision-saving genes to the retina. For more information on this technology, visit www.oxfordbiomedica.co.uk/
Monday, November 15, 2010
Macular Degeneration Treatments/Therapies Breakthroughs
by Isobel Washington
Macular degeneration is an eye condition characterized by the deterioration of the macula, which is the central "lens-like" part of the eye's retina responsible for sharp central vision. Affecting central vision and often leading to vision loss, macular degeneration has had limited treatment options. Recently, there have been some breakthroughs to help patients retain vision.
Significance
Macular degeneration is the leading cause of vision loss and blindness among Americans age 65 and older. Vision loss from this condition is a growing problem, since this age demographic represents an increasingly larger percentage of the U. S. population.
Function
Treatments for macular degeneration work to preserve vision ability at the time of treatment, and slow the progression of the disease (it's a progressive disease that affects vision over time). Treatment cannot restore vision that is already lost through the disease. There is no cure or treatment to stop the progression of macular degeneration, there are ways to preserve and prolong current vision.
Eye Injections
Lucentis and Macugen are FDA-approved ocular injection treatments, and are highly effective for preserving vision and inhibiting macular degeneration symptoms, according to AllAboutVision.com. A 2005 study on Lucentis demonstrated success rate of 95 percent for improving and sustaining vision in macular degeneration patients.
Laser Treatment
Laser technology is now used to destroy the abnormal, leaky blood vessels that cause vision loss in macular degeneration patients. The National Eye Institute points out, however, that while this treatment may be effective for preventing vision loss, it is also comes with the risk of destroying healthy tissue that surrounds the treatment area.
Photodynamic Therapy
This method uses special light treatment to activate an injected drug, verteporfin, in the blood vessels, so that it destroys the new, abnormal blood vessels being hyper-produced in the eye (those that cause vision impairment or loss). The National Eye Institute reports that this light-activation method slows vision loss, but doesn't stop it.
Investigational Treatments
Investigational treatments for macular degeneration, in various stages of research and FDA clinical studies, include Avastin, a cancer treatment drug. As of 2009, the National Eye Institute reports that no available treatment provides a cure for macular degeneration, and that vision loss may result, despite treatment.
About the Author
Isobel Washington has been a freelance journalist since 2007. Washington's work first surfaced in Europe, where she served as a restaurant critic and journalist for "LifeStyles" magazine. Her love of travel and culture inspired her first novel, which is currently underway. Washington has a 10-year career in marketing communication and holds a Bachelor of Science degree.
Macular degeneration is an eye condition characterized by the deterioration of the macula, which is the central "lens-like" part of the eye's retina responsible for sharp central vision. Affecting central vision and often leading to vision loss, macular degeneration has had limited treatment options. Recently, there have been some breakthroughs to help patients retain vision.
Significance
Macular degeneration is the leading cause of vision loss and blindness among Americans age 65 and older. Vision loss from this condition is a growing problem, since this age demographic represents an increasingly larger percentage of the U. S. population.
Function
Treatments for macular degeneration work to preserve vision ability at the time of treatment, and slow the progression of the disease (it's a progressive disease that affects vision over time). Treatment cannot restore vision that is already lost through the disease. There is no cure or treatment to stop the progression of macular degeneration, there are ways to preserve and prolong current vision.
Eye Injections
Lucentis and Macugen are FDA-approved ocular injection treatments, and are highly effective for preserving vision and inhibiting macular degeneration symptoms, according to AllAboutVision.com. A 2005 study on Lucentis demonstrated success rate of 95 percent for improving and sustaining vision in macular degeneration patients.
Laser Treatment
Laser technology is now used to destroy the abnormal, leaky blood vessels that cause vision loss in macular degeneration patients. The National Eye Institute points out, however, that while this treatment may be effective for preventing vision loss, it is also comes with the risk of destroying healthy tissue that surrounds the treatment area.
Photodynamic Therapy
This method uses special light treatment to activate an injected drug, verteporfin, in the blood vessels, so that it destroys the new, abnormal blood vessels being hyper-produced in the eye (those that cause vision impairment or loss). The National Eye Institute reports that this light-activation method slows vision loss, but doesn't stop it.
Investigational Treatments
Investigational treatments for macular degeneration, in various stages of research and FDA clinical studies, include Avastin, a cancer treatment drug. As of 2009, the National Eye Institute reports that no available treatment provides a cure for macular degeneration, and that vision loss may result, despite treatment.
About the Author
Isobel Washington has been a freelance journalist since 2007. Washington's work first surfaced in Europe, where she served as a restaurant critic and journalist for "LifeStyles" magazine. Her love of travel and culture inspired her first novel, which is currently underway. Washington has a 10-year career in marketing communication and holds a Bachelor of Science degree.
Thursday, November 4, 2010
Qualifing Theraputic Discovery Project Program
by GEN news
Over the last couple of days, companies have been reporting on awards received under the Qualifying Therapeutic Discovery Project program. The IRS in conjunction with the Department of Health and Human Services, approved applications for projects that showed significant potential to produce new and cost-saving therapies, support jobs, and increase U.S. competitiveness.
Under the program, a total amount of $1 billion was allocated for credits and grants with a $5 million limit per each eligible applicant. The full list of companies receiving these awards can be found at http://bit.ly/b3VxSp.
Here are details for a few more companies:
Acceleron Pharma received five grants totaling $1,222,396.25: ACE-031 for Duchenne muscular dystrophy, ACE-536 for myelodysplastic syndrome, ACE-435, ACE-011 for anemia, and ACE-041 for advanced solid tumors, multiple myeloma, and age-related macular degeneration.
Aestus Therapeutics received one grant of $244,000: chronic neuropathic pain drug development.
BioCryst Pharmaceuticals received five grants totaling $1,064,297.89: four grants of $244,479.25 each were given to peramivir for influenza, BCX4208 for gout, forodesine for CLL and CTCL, and JAK inhibitor programs in psoriasis, ankylosing spondylitis, and multiple sclerosis. One grant of $86,380.89 was awarded to BCX4161 for hereditary angiodema.
Biodel received five grants totaling $1,222,396.25: Linjeta, VIAtab, smart basal, glucagon, and glargine.
Cardium Therapeutics received one grant of $244,479.25: Generx for advanced coronary artery disease.
Celsion received one grant of $244,479.25: Thermodox for primary liver cancer and recurrent chest wall breast cancer.
Cerus received two grants totaling $488,958.50: Intercept Blood System for platelets and red blood cells.
Charleston Laboratories received one grant of $244,479.25: CL-108 for moderate-to-severe pain.
CorMedix received two grants totaling $488,959.50: Neutrolin and Deferiprone.
Curis received two grants totaling $488,958.50: CUDC-101 and pipeline small molecules for cancer treatment.
Derma Sciences received one grant of $244,479.25: DSC127 for tissue repair.
Dicerna Pharmaceuticals received two grants totaling $488,958.50: Dicer Substrate Technology and DsiRNA molecules for the treatment of cancer.
DNA Medicine Institute received a grant of $222,999: Universal Blood Sensor.
Enzon Pharmaceuticals received five grants totaling $1,222,396.25: PEG-SN38, mRNA antagonist for HIF-1 alpha, Survivin, Androgen Receptor, and HER3.
Etubics received one grant of $244,479.25: CEA-expressing adenovirus as a colon cancer therapeutic vaccine.
GTx received five grants totaling $1,222,396.25: ALK inhibition therapy, Toremifene 20 mg, Ostarine, GTx-758, Toremifene 80 mg, which are all being developed for cancer and cancer supportive care.
Health Discovery received a grant of $244,479.25: SVM and RFE-SVM technology.
Icagen received three grants totaling $733,000: Selective sodium channel drugs to treat chronic pain, KCNQ agonists for epilepsy and pain, and TRPA1 drugs for inflammatory pain.
Inviragen received two grants totaling $488,958.50: Vaccines against dengue and chikungunya viruses.
InVivo Therapeutics received a grant of $244,000: biocompatible polymer scaffolding device designed for implantation into a lesion to treat acute open-wound SCI.
Light Sciences Oncology received two grants totaling $488,958.50: Aptocine for treatment of primary and secondary liver tumors and benign prostatic hyperplasia.
Lipocine received four grants totaling $977,917: hormone replacement therapy, high-risk pregnancy support, oral treatment of progressive glioblastoma multiforme, and opioid-resistant cough in advanced cancer patients.
Molecular Detection received a grant of $244,479.25: Detect-Ready panel that detects carriers of MRSA and related pathogens.
Neoprobe received a grant of $244,479.25: Lymphoseek.
Omeros received eight grants of uneven amounts totaling $1,723,086.51: PDE7 for Parkinson disease; addiction treatment; therapies for osteoarthritis and the protection of articular cartilage; drugs to treat pain, inflammation, and spasm of the urinary tract; MASP-2 MoAb for traumatic injury; medications for schizophrenia; intracameral OMS302 to maintain intraoperative mydriasis and reduce pain and inflammation; and products to improve function and reduce pain after arthroscopic surgery.
OncoMed Pharmaceuticals received five grants totaling $1,222,396.25: OMP-21M18, OMP-59R5, novel antibodies, Wnt pathway inhibitors, and drugs targeting the Wnt pathway in cancer stem cells, all for the treatment of cancer.
PolyMedix received two cash grants totaling $488,958.50: PMX-30063 for acute bacterial skin and skin structure infections (ABSSSI) caused by Staph and PMX-60056 in percutaneous coronary intervention patients.
pSivida received two cash grants totaling $488,958.50: research on new generations of the company’s drug delivery technologies targeting ophthalmic diseases.
Quanterix received three grants totaling $733,437.75: Diagnostics developed on the Single Molecule Array (SiMoA) for prostate cancer, Alzheimer disease, and Crohn disease.
Regulus Therapeutics received two cash grants totaling $488,958.50: miRNA therapeutics for HCV and fibrosis.
Sangamo BioSciences received four grants totaling $977,917: SB-509 for diabetic peripheral neuropathy, SB-509 for amyotrophic lateral sclerosis, SB-728-T for human immunodeficiency virus/acquired immunodeficiency syndrome, and SB-313-xTZ for recurrent or refractory glioblastoma multiforme.
Sea Lane Biotechnologies received six grants totaling $1.5 million: Surrobody™ therapeutic candidates and influenza antibodies.
Soligenix received a grant of $244,479.25: orBec in acute gastrointestinal graft vs. host disease.
Somaxon Pharmaceuticals received a grant of $244,479.25: Silenor.
Spectrum Pharmaceuticals received four grants totaling $977,917: RenaZorb for hyperphosphatemia in patients with chronic kidney disease and anticancer agents Zevalin, Apaziquone, and Belinostat.
Spherix received two grants totaling $469,478.50: triglycerides.
StemCells received four grants totaling $977,917: HuCNS-SC for diseases and disorders affecting the brain, the spinal cord, and the eye as well as hLEC human liver engrafting cells.
Synergenz BioScience received one grant of $94,836: Respiragene, a genetic-based test for lung cancer predisposition.
Telik received five grants totaling $1,222,396.25: Telintra in severe chronic neutropenia, Telcyta for refractory lymphoma and multiple myeloma, Telintra in low-to-intermediate-1 risk myelodysplastic syndrome, Telintra and Revlimid in myelodysplastic syndrome, and aurora and VEGFR kinase inhibitors for the treatment of cancer.
Threshold Pharmaceuticals received two grants totaling $488,958.50: TH-302 for the treatment of cancer, and the company’s hypoxia-activated prodrug technology platform for drug discovery.
Xoft received two grants totaling $469,478.50: electronic brachytherapy for the treatment of breast cancer and gynecological cancers.
Zalicus receive a grant of $244,479.25: Synavive for immunoinflammatory diseases.
Ziopharm Oncology received three grants totaling $733,437.75: Indibulin, Palifosfamide, and Darinaparsin, all for the treatment of cancer.
Over the last couple of days, companies have been reporting on awards received under the Qualifying Therapeutic Discovery Project program. The IRS in conjunction with the Department of Health and Human Services, approved applications for projects that showed significant potential to produce new and cost-saving therapies, support jobs, and increase U.S. competitiveness.
Under the program, a total amount of $1 billion was allocated for credits and grants with a $5 million limit per each eligible applicant. The full list of companies receiving these awards can be found at http://bit.ly/b3VxSp.
Here are details for a few more companies:
Acceleron Pharma received five grants totaling $1,222,396.25: ACE-031 for Duchenne muscular dystrophy, ACE-536 for myelodysplastic syndrome, ACE-435, ACE-011 for anemia, and ACE-041 for advanced solid tumors, multiple myeloma, and age-related macular degeneration.
Aestus Therapeutics received one grant of $244,000: chronic neuropathic pain drug development.
BioCryst Pharmaceuticals received five grants totaling $1,064,297.89: four grants of $244,479.25 each were given to peramivir for influenza, BCX4208 for gout, forodesine for CLL and CTCL, and JAK inhibitor programs in psoriasis, ankylosing spondylitis, and multiple sclerosis. One grant of $86,380.89 was awarded to BCX4161 for hereditary angiodema.
Biodel received five grants totaling $1,222,396.25: Linjeta, VIAtab, smart basal, glucagon, and glargine.
Cardium Therapeutics received one grant of $244,479.25: Generx for advanced coronary artery disease.
Celsion received one grant of $244,479.25: Thermodox for primary liver cancer and recurrent chest wall breast cancer.
Cerus received two grants totaling $488,958.50: Intercept Blood System for platelets and red blood cells.
Charleston Laboratories received one grant of $244,479.25: CL-108 for moderate-to-severe pain.
CorMedix received two grants totaling $488,959.50: Neutrolin and Deferiprone.
Curis received two grants totaling $488,958.50: CUDC-101 and pipeline small molecules for cancer treatment.
Derma Sciences received one grant of $244,479.25: DSC127 for tissue repair.
Dicerna Pharmaceuticals received two grants totaling $488,958.50: Dicer Substrate Technology and DsiRNA molecules for the treatment of cancer.
DNA Medicine Institute received a grant of $222,999: Universal Blood Sensor.
Enzon Pharmaceuticals received five grants totaling $1,222,396.25: PEG-SN38, mRNA antagonist for HIF-1 alpha, Survivin, Androgen Receptor, and HER3.
Etubics received one grant of $244,479.25: CEA-expressing adenovirus as a colon cancer therapeutic vaccine.
GTx received five grants totaling $1,222,396.25: ALK inhibition therapy, Toremifene 20 mg, Ostarine, GTx-758, Toremifene 80 mg, which are all being developed for cancer and cancer supportive care.
Health Discovery received a grant of $244,479.25: SVM and RFE-SVM technology.
Icagen received three grants totaling $733,000: Selective sodium channel drugs to treat chronic pain, KCNQ agonists for epilepsy and pain, and TRPA1 drugs for inflammatory pain.
Inviragen received two grants totaling $488,958.50: Vaccines against dengue and chikungunya viruses.
InVivo Therapeutics received a grant of $244,000: biocompatible polymer scaffolding device designed for implantation into a lesion to treat acute open-wound SCI.
Light Sciences Oncology received two grants totaling $488,958.50: Aptocine for treatment of primary and secondary liver tumors and benign prostatic hyperplasia.
Lipocine received four grants totaling $977,917: hormone replacement therapy, high-risk pregnancy support, oral treatment of progressive glioblastoma multiforme, and opioid-resistant cough in advanced cancer patients.
Molecular Detection received a grant of $244,479.25: Detect-Ready panel that detects carriers of MRSA and related pathogens.
Neoprobe received a grant of $244,479.25: Lymphoseek.
Omeros received eight grants of uneven amounts totaling $1,723,086.51: PDE7 for Parkinson disease; addiction treatment; therapies for osteoarthritis and the protection of articular cartilage; drugs to treat pain, inflammation, and spasm of the urinary tract; MASP-2 MoAb for traumatic injury; medications for schizophrenia; intracameral OMS302 to maintain intraoperative mydriasis and reduce pain and inflammation; and products to improve function and reduce pain after arthroscopic surgery.
OncoMed Pharmaceuticals received five grants totaling $1,222,396.25: OMP-21M18, OMP-59R5, novel antibodies, Wnt pathway inhibitors, and drugs targeting the Wnt pathway in cancer stem cells, all for the treatment of cancer.
PolyMedix received two cash grants totaling $488,958.50: PMX-30063 for acute bacterial skin and skin structure infections (ABSSSI) caused by Staph and PMX-60056 in percutaneous coronary intervention patients.
pSivida received two cash grants totaling $488,958.50: research on new generations of the company’s drug delivery technologies targeting ophthalmic diseases.
Quanterix received three grants totaling $733,437.75: Diagnostics developed on the Single Molecule Array (SiMoA) for prostate cancer, Alzheimer disease, and Crohn disease.
Regulus Therapeutics received two cash grants totaling $488,958.50: miRNA therapeutics for HCV and fibrosis.
Sangamo BioSciences received four grants totaling $977,917: SB-509 for diabetic peripheral neuropathy, SB-509 for amyotrophic lateral sclerosis, SB-728-T for human immunodeficiency virus/acquired immunodeficiency syndrome, and SB-313-xTZ for recurrent or refractory glioblastoma multiforme.
Sea Lane Biotechnologies received six grants totaling $1.5 million: Surrobody™ therapeutic candidates and influenza antibodies.
Soligenix received a grant of $244,479.25: orBec in acute gastrointestinal graft vs. host disease.
Somaxon Pharmaceuticals received a grant of $244,479.25: Silenor.
Spectrum Pharmaceuticals received four grants totaling $977,917: RenaZorb for hyperphosphatemia in patients with chronic kidney disease and anticancer agents Zevalin, Apaziquone, and Belinostat.
Spherix received two grants totaling $469,478.50: triglycerides.
StemCells received four grants totaling $977,917: HuCNS-SC for diseases and disorders affecting the brain, the spinal cord, and the eye as well as hLEC human liver engrafting cells.
Synergenz BioScience received one grant of $94,836: Respiragene, a genetic-based test for lung cancer predisposition.
Telik received five grants totaling $1,222,396.25: Telintra in severe chronic neutropenia, Telcyta for refractory lymphoma and multiple myeloma, Telintra in low-to-intermediate-1 risk myelodysplastic syndrome, Telintra and Revlimid in myelodysplastic syndrome, and aurora and VEGFR kinase inhibitors for the treatment of cancer.
Threshold Pharmaceuticals received two grants totaling $488,958.50: TH-302 for the treatment of cancer, and the company’s hypoxia-activated prodrug technology platform for drug discovery.
Xoft received two grants totaling $469,478.50: electronic brachytherapy for the treatment of breast cancer and gynecological cancers.
Zalicus receive a grant of $244,479.25: Synavive for immunoinflammatory diseases.
Ziopharm Oncology received three grants totaling $733,437.75: Indibulin, Palifosfamide, and Darinaparsin, all for the treatment of cancer.
Sunday, October 10, 2010
Angiogenesis Discovery Points to Novel Therapy for Multiple Diseases
Tatiana Byzova, PhD, a faculty member in Lerner Research Institute's Department of Molecular Cardiology and Director of the Center for Angiogenesis Research, and colleagues have discovered a fundamental biological pathway in angiogenesis (formation of blood vessels), published online October 3, 2010 in Nature (DOI: 10.1038/nature09421). The significance of this discovery is summarized by Dr. Byzova, in that "it affects many biological processes - from wound healing to aging."
Some pathologies included in that spectrum are age-related macular degeneration, atherosclerosis, and rheumatoid arthritis, while their published data indicate a particularly notable role in cancer. The results of this advanced understanding point to an exciting new approach for novel therapies.
In 1989, a protein called vascular endothelial growth factor (VEGF) was identified as a trigger of angiogenesis. This discovery led Genentech to develop the first effective treatment for macular degeneration; related work contributed to the development of a drug to block VEGF function in cancer, essentially starving the tumor of the nutrients the blood would otherwise bring.
However, although many tumors respond to anti-VEGF treatment, most develop resistance to the therapy and continue to survive over time. One possible explanation is that VEGF is not the only trigger leading to angiogenesis. This is precisely what Dr. Byzova's team has found.
Dr. Byzova's research has identified a class of oxidized lipids that are abundantly present in highly vasculated tumors. Further interrogation parsed out the process through which these products induce new blood vessel formation. It appears that cells use a family of receptors previously known to recognize foreign products such as bacteria to sense and respond to the danger of oxidation. The results point to a novel therapy that may well be an alternate way to starve the tumors that have managed to survive despite VEGF inhibition. Through uncovering a potential way to get around tumor resistance to VEGF, Dr. Byzova's group has opened up new doors for developing treatments for cancer. The fundamental discovery also provides substantial insight for novel treatments for other diseases that involve inflammation, oxidative stress, and angiogenesis.
Overall, this foundational discovery of a novel mechanism of angiogenesis comes just one year after Dr. Byzova headed a study that pioneered the identification of a new genetic-based human disease, published in Nature Medicine.
Some pathologies included in that spectrum are age-related macular degeneration, atherosclerosis, and rheumatoid arthritis, while their published data indicate a particularly notable role in cancer. The results of this advanced understanding point to an exciting new approach for novel therapies.
In 1989, a protein called vascular endothelial growth factor (VEGF) was identified as a trigger of angiogenesis. This discovery led Genentech to develop the first effective treatment for macular degeneration; related work contributed to the development of a drug to block VEGF function in cancer, essentially starving the tumor of the nutrients the blood would otherwise bring.
However, although many tumors respond to anti-VEGF treatment, most develop resistance to the therapy and continue to survive over time. One possible explanation is that VEGF is not the only trigger leading to angiogenesis. This is precisely what Dr. Byzova's team has found.
Dr. Byzova's research has identified a class of oxidized lipids that are abundantly present in highly vasculated tumors. Further interrogation parsed out the process through which these products induce new blood vessel formation. It appears that cells use a family of receptors previously known to recognize foreign products such as bacteria to sense and respond to the danger of oxidation. The results point to a novel therapy that may well be an alternate way to starve the tumors that have managed to survive despite VEGF inhibition. Through uncovering a potential way to get around tumor resistance to VEGF, Dr. Byzova's group has opened up new doors for developing treatments for cancer. The fundamental discovery also provides substantial insight for novel treatments for other diseases that involve inflammation, oxidative stress, and angiogenesis.
Overall, this foundational discovery of a novel mechanism of angiogenesis comes just one year after Dr. Byzova headed a study that pioneered the identification of a new genetic-based human disease, published in Nature Medicine.
Monday, August 23, 2010
Gene therapy can correct inherited retinal eye diseases
Researchers at the American Academy of Ophthalmology have found that Leber’s congenital Amaurosis (LCA), a very severe form of retinal disease
can be improved with the help of gene therapy.
Not only that, but the improvements were also found to be stable for two years.
Gene Therapy Can Correct Retinal Eye Diseases: LCA can be diagnosed in children at a very early age, sometimes even at infancy.
The main symptoms
of LCA are severity in vision loss and nystagmus(involuntary eye movement). By the time a person reaches his thirties or forties LCA develops into blindness.
* The impact of gene therapy could mainly be observed in children. The visual acuity and light sensitivity was tremendous in these children.
* Not only in children, researchers were also able to observe improvement in adults.
The Research and Theory: LCA is due to the mutations caused in any of the 13 genes in our body. Researchers were studying a Type 2 LCA which is due to mutation in the RPE65 gene.
* The doctors injected a normal functioning RPE65 gene which was joined with a virus into the sub-retinal space upon performing a surgery.
* The altered virus places the normally functioning RPE65 gene into the diseased cells and modifies the defective enzymes.
* After 2 weeks from the date of surgery, doctors observed that the patients reported an improvement in vision even in dim light.
* There were a few patients who also said that their visual acuity improved.
* A few even experienced massive improvement in nystagmus.
* The most encouraging result was that none of the patients experienced adverse effects.
Gene therapy does not improve sight on a permanent basis. But, gene therapy can correct inherited retinal eye diseases and help people restore sight.
People who undergo gene therapy need not be classified as blind any more. Researchers are trying hard to implement this technique in a safer way in younger children.
can be improved with the help of gene therapy.
Not only that, but the improvements were also found to be stable for two years.
Gene Therapy Can Correct Retinal Eye Diseases: LCA can be diagnosed in children at a very early age, sometimes even at infancy.
The main symptoms
of LCA are severity in vision loss and nystagmus(involuntary eye movement). By the time a person reaches his thirties or forties LCA develops into blindness.
* The impact of gene therapy could mainly be observed in children. The visual acuity and light sensitivity was tremendous in these children.
* Not only in children, researchers were also able to observe improvement in adults.
The Research and Theory: LCA is due to the mutations caused in any of the 13 genes in our body. Researchers were studying a Type 2 LCA which is due to mutation in the RPE65 gene.
* The doctors injected a normal functioning RPE65 gene which was joined with a virus into the sub-retinal space upon performing a surgery.
* The altered virus places the normally functioning RPE65 gene into the diseased cells and modifies the defective enzymes.
* After 2 weeks from the date of surgery, doctors observed that the patients reported an improvement in vision even in dim light.
* There were a few patients who also said that their visual acuity improved.
* A few even experienced massive improvement in nystagmus.
* The most encouraging result was that none of the patients experienced adverse effects.
Gene therapy does not improve sight on a permanent basis. But, gene therapy can correct inherited retinal eye diseases and help people restore sight.
People who undergo gene therapy need not be classified as blind any more. Researchers are trying hard to implement this technique in a safer way in younger children.
Wednesday, August 18, 2010
Drug Research could lead to AMD therapy
By Adrian Galbreth
New drug research may pave the way for more effective treatments of age-related macular degeneration - the leading cause of blindness in the western world.
Those are the claims being made by researchers at Tufts University School of Medicine, who say that a protein known as galectin-3 promotes the growth of new blood vessels, and that targeting the protein can "significantly reduce" angiogenesis.
The findings have been published in the Journal of Experimental Medicine and may lead to treatments for diseases caused by excessive angiogenesis, which include AMD, said Dr Noorjahan Panjwani, who led the project.
She explained: "Our study shows that galectin-3 protein binds to glycans of specific cell-adhesion proteins to activate the signaling pathways that bring about angiogenesis. This improved understanding may provide a more targeted approach to preventing harmful angiogenesis."
Meanwhile, another team of researchers at Tufts have found that that non-viral gene therapy can delay the onset of some forms of eye disease and offer hope to retinal degeneration sufferers.ADNFCR-1853-ID-800028486-ADNFCR
New drug research may pave the way for more effective treatments of age-related macular degeneration - the leading cause of blindness in the western world.
Those are the claims being made by researchers at Tufts University School of Medicine, who say that a protein known as galectin-3 promotes the growth of new blood vessels, and that targeting the protein can "significantly reduce" angiogenesis.
The findings have been published in the Journal of Experimental Medicine and may lead to treatments for diseases caused by excessive angiogenesis, which include AMD, said Dr Noorjahan Panjwani, who led the project.
She explained: "Our study shows that galectin-3 protein binds to glycans of specific cell-adhesion proteins to activate the signaling pathways that bring about angiogenesis. This improved understanding may provide a more targeted approach to preventing harmful angiogenesis."
Meanwhile, another team of researchers at Tufts have found that that non-viral gene therapy can delay the onset of some forms of eye disease and offer hope to retinal degeneration sufferers.ADNFCR-1853-ID-800028486-ADNFCR
Tuesday, July 20, 2010
Stem Cell Transplants Stalled Blindness in Rats
Researchers say putting nerve stem cells from StemCells Inc near the retinas of rats with a form of macular degeneration helped keep the disease from advancing to blindness for several months.Nerve stem cell transplants may help slow the progression of macular degeneration, the most common cause of blindness in the developed world, U.S. researchers said on Monday.
They said putting nerve stem cells from StemCells Inc near the retinas of rats with a form of macular degeneration helped keep the disease from advancing to blindness for several months.
"These cells improve the chemical environment in the back of the eye," said Ray Lund of the Casey Eye Institute at Oregon Health & Science University in Portland, whose findings were presented at the Society for Neuroscience meeting in Chicago.
Lund said the mechanism is not clear, but he suspects that when immature nerve cells are placed near the retina, they produce growth factors that protect the cells from damage by the disease.
"It's basically a chemical pump that is sitting in the right place and producing the right things," Lund said in a telephone interview.
Where normally animals with eye disease lost their vision by three months old, rats that got the transplants kept their vision for at least seven months, he said.
"There is no evidence that they (the transplanted cells) do any damage," Lund said, adding that the animals do not develop tumors, a key worry for stem cell transplants.
The findings raise hope for use of the treatment in humans with a range of diseases in which the retina become damaged, including age-related macular degeneration or AMD, which affects nearly 30 million people worldwide, including 15 million Americans.
People with AMD lose central vision when delicate light-sensing cells of the macula, a region at the center of the retina, become damaged.
In the rats, the researchers transplanted immature nerve cells into the space near the retina. Lund said the same could be done in people with retinal disease.
Dr. Stephen Huhn, head of the Central Nervous System research program at StemCells Inc, said the cells are adult neural stem cells. He said they are multipotent, meaning they can morph into different types of nerve cells.
The company has already tested the treatment in a study of six patients with Batten's disease, a fatal inherited disorder of the nervous system.
"Having a cell that has already entered clinical testing that has been well tolerated at very high doses in the brain gives us a lot of confidence about exploring the same type of strategy in the eye," Huhn said.
Huhn said he thinks the cells may be especially well suited for use in the retina, brain and spinal cord, which are less likely to reject the cells than other parts of the body.
Ultimately, he said the hope is to develop a treatment for the dry form of macular degeneration, which affects around 90 percent of patients diagnosed with AMD. No treatments are available for this form of the disease.
Huhn said treating this form of the disease may prevent some people from developing wet AMD, in which tiny new blood vessels grow between the retina and the back of the eye.
This form of the disease can be treated with modern drugs like Lucentis, from Novartis and Roche's Genentech, and Pfizer's Macugen.
They said putting nerve stem cells from StemCells Inc near the retinas of rats with a form of macular degeneration helped keep the disease from advancing to blindness for several months.
"These cells improve the chemical environment in the back of the eye," said Ray Lund of the Casey Eye Institute at Oregon Health & Science University in Portland, whose findings were presented at the Society for Neuroscience meeting in Chicago.
Lund said the mechanism is not clear, but he suspects that when immature nerve cells are placed near the retina, they produce growth factors that protect the cells from damage by the disease.
"It's basically a chemical pump that is sitting in the right place and producing the right things," Lund said in a telephone interview.
Where normally animals with eye disease lost their vision by three months old, rats that got the transplants kept their vision for at least seven months, he said.
"There is no evidence that they (the transplanted cells) do any damage," Lund said, adding that the animals do not develop tumors, a key worry for stem cell transplants.
The findings raise hope for use of the treatment in humans with a range of diseases in which the retina become damaged, including age-related macular degeneration or AMD, which affects nearly 30 million people worldwide, including 15 million Americans.
People with AMD lose central vision when delicate light-sensing cells of the macula, a region at the center of the retina, become damaged.
In the rats, the researchers transplanted immature nerve cells into the space near the retina. Lund said the same could be done in people with retinal disease.
Dr. Stephen Huhn, head of the Central Nervous System research program at StemCells Inc, said the cells are adult neural stem cells. He said they are multipotent, meaning they can morph into different types of nerve cells.
The company has already tested the treatment in a study of six patients with Batten's disease, a fatal inherited disorder of the nervous system.
"Having a cell that has already entered clinical testing that has been well tolerated at very high doses in the brain gives us a lot of confidence about exploring the same type of strategy in the eye," Huhn said.
Huhn said he thinks the cells may be especially well suited for use in the retina, brain and spinal cord, which are less likely to reject the cells than other parts of the body.
Ultimately, he said the hope is to develop a treatment for the dry form of macular degeneration, which affects around 90 percent of patients diagnosed with AMD. No treatments are available for this form of the disease.
Huhn said treating this form of the disease may prevent some people from developing wet AMD, in which tiny new blood vessels grow between the retina and the back of the eye.
This form of the disease can be treated with modern drugs like Lucentis, from Novartis and Roche's Genentech, and Pfizer's Macugen.
Wednesday, June 30, 2010
Stem cell Therapy to Benefit Blind
Submitted by Jayden Roberts on Tue, 06/29/2010
Italian researchers have reported that about 12 people have regained their sight in a successful experiment conducted with some partly blind and severe eye damage suffering people. This was revealed in a study published online in the New England Journal of Medicine.
This is a remarkable success that will encourage the cell-therapy, which is done by transplanting cells from one’s own body to other parts. It has been claimed that the treatment has proved winning in 82 of 107 eyes. Also, it was partially complete in 14 others eyes. The benefits of the treatment are expected to last till 10 years after the process.
It is also noticeable that one man, who had been blind for more than five decades, have also completely restored his visual capacity. Appreciating the success, Ophthalmologist Ivan Schwab of the University of California praised and congratulated the team.
If the stem cell transplants become popular and are implemented even more, they can also prove helpful for the people who are affected by chemical burns on their corneas from heavy-duty cleansers and other chemicals. This will be a great help for people who have to suffer eyesight loss due to such mishaps.
However, the stem cell approach is not capable to treat optic nerve or macular degeneration, which is caused due to the damage in retina, as the treatment requires a few healthy tissues that can be transplanted.
Italian researchers have reported that about 12 people have regained their sight in a successful experiment conducted with some partly blind and severe eye damage suffering people. This was revealed in a study published online in the New England Journal of Medicine.
This is a remarkable success that will encourage the cell-therapy, which is done by transplanting cells from one’s own body to other parts. It has been claimed that the treatment has proved winning in 82 of 107 eyes. Also, it was partially complete in 14 others eyes. The benefits of the treatment are expected to last till 10 years after the process.
It is also noticeable that one man, who had been blind for more than five decades, have also completely restored his visual capacity. Appreciating the success, Ophthalmologist Ivan Schwab of the University of California praised and congratulated the team.
If the stem cell transplants become popular and are implemented even more, they can also prove helpful for the people who are affected by chemical burns on their corneas from heavy-duty cleansers and other chemicals. This will be a great help for people who have to suffer eyesight loss due to such mishaps.
However, the stem cell approach is not capable to treat optic nerve or macular degeneration, which is caused due to the damage in retina, as the treatment requires a few healthy tissues that can be transplanted.
Thursday, May 27, 2010
Long-Term Bevacizumab Therapy in Macular Degeneration Patients Appears Safe: Presented at ARVO
By Micheal Casasnovas
Most patients with neovascular age-related macular degeneration treated with bevacizumab achieved visual improvement over the course of 30 months without untoward side effects, according to a study presented here at the 2010 Annual Meeting of the Association for Research in Vision Ophthalmology (ARVO).
"This study suggests that a 30-month as-needed therapy with bevacizumab for choroidal neovascularisation in age-related macular degeneration is safe," said Renan Ferreira Oliveira, MD, Pontifícia Universidade Católica do Paraná, Curitiba, Brazil, on May 2.
For this noncomparative, retrospective study, researchers used a consecutive case series of 20 eyes from 16 patients with choroidal neovascularisation caused by age-related macular degeneration who were treated with at least 1 intravitreal injection of bevacizumab and completed a minimum follow-up of 30 months. Patients were excluded from the analysis if they had received prior verteporfin photodynamic therapy, photocoagulation, and intravitreal or periocular injections of triamcinolone, or other antiangiogenic drugs.
Patients who received bevacizumab 1.50 mg were analysed and then examined every 30 to 90 days. Additional treatments were given if the condition worsened after originally showing a positive functional response.
Patients enrolled in this study had a mean age of 75 years (range 59-87); 62.5% were women. They received 1 to 12 injections, but the average patient was injected 5 times.
"After 30 months from the beginning of therapy, best-corrected visual acuity declined in 8 eyes [40%], improved in 7 eyes [35%], and stabilised in 5 eyes [25%]," reported Dr. Oliveira.
No significant ocular or systemic side effects occurred.
"The functional results show that 60% of patients had improved or stabilised visual acuity," said Dr. Oliveira. "However, a large proportion of patients had a vision loss in spite of close ophthalmologic control and multiple intravitreal injections of bevacizumab."
[Presentation title: Intravitreal Bevacizumab for Choroidal Neovascularization in Age-Related Macular Degeneration: 30-Month Results. Abstract A214]
Most patients with neovascular age-related macular degeneration treated with bevacizumab achieved visual improvement over the course of 30 months without untoward side effects, according to a study presented here at the 2010 Annual Meeting of the Association for Research in Vision Ophthalmology (ARVO).
"This study suggests that a 30-month as-needed therapy with bevacizumab for choroidal neovascularisation in age-related macular degeneration is safe," said Renan Ferreira Oliveira, MD, Pontifícia Universidade Católica do Paraná, Curitiba, Brazil, on May 2.
For this noncomparative, retrospective study, researchers used a consecutive case series of 20 eyes from 16 patients with choroidal neovascularisation caused by age-related macular degeneration who were treated with at least 1 intravitreal injection of bevacizumab and completed a minimum follow-up of 30 months. Patients were excluded from the analysis if they had received prior verteporfin photodynamic therapy, photocoagulation, and intravitreal or periocular injections of triamcinolone, or other antiangiogenic drugs.
Patients who received bevacizumab 1.50 mg were analysed and then examined every 30 to 90 days. Additional treatments were given if the condition worsened after originally showing a positive functional response.
Patients enrolled in this study had a mean age of 75 years (range 59-87); 62.5% were women. They received 1 to 12 injections, but the average patient was injected 5 times.
"After 30 months from the beginning of therapy, best-corrected visual acuity declined in 8 eyes [40%], improved in 7 eyes [35%], and stabilised in 5 eyes [25%]," reported Dr. Oliveira.
No significant ocular or systemic side effects occurred.
"The functional results show that 60% of patients had improved or stabilised visual acuity," said Dr. Oliveira. "However, a large proportion of patients had a vision loss in spite of close ophthalmologic control and multiple intravitreal injections of bevacizumab."
[Presentation title: Intravitreal Bevacizumab for Choroidal Neovascularization in Age-Related Macular Degeneration: 30-Month Results. Abstract A214]
Sunday, July 12, 2009
Tips To Improve Your Vision – Get Rid Of Your Glasses!
Eyesight improvement is achievable. You can learn to see without glasses and be relieved permanently of the pain and distress so frequently associated with defective sight. But you cannot Improve your vision by magic.
CENTRAL FIXATION
The retina is a sensitive film on which the picture falls. But there is one point on the retina where the vision is perfect; that is the Macula Lutae, a point only one-sixteenth of an inch in diameter in the very center of the retina. When we focus at this point we have what is known as central fixation and our vision is perfect.
If you have lost the capacity of central fixation you are seeing with Eccentric fixation which often causes headaches, fatigue, pain or discomfort of some kind, such as twitching of the eyelids or the eyeballs. This twitching, by the way, can be stopped by pressing the sides of the base of the nose as high as the inner canthus with the forefingers of both hands, avoiding any pressure on the eyeballs.
Continue the pressure for several minutes, with the eyes closed, and you will obtain relief.
One way of checking on whether you are seeing by central or eccentric fixation is to look at a word on this page. Do you see it most sharply where you are looking or do you see it better when you look a little away from it? When you look at the top of a printed letter do you see the bottom of the letter more clearly than the top? If so, you have lost central fixation.
If you are to see, you must bring your mind to bear on what you see. Because the eye can focus sharply and is at its maximum power only on a very small area at a time, an attempt to see a larger area results in a blurring of physical vision and a lack of mental focus. Teach yourself to look at what you see, to watch one tiny area at a time. For when the central fixation is perfect, the eye sees perfectly.
THINK ABOUT WHAT YOU SEE
For significant eye sight improvement, give the object you are looking at your mental as well as your visual attention. The more clearly it registers on your mind, the more clearly it will register on the eye.
Test this out for yourself. In the room where you are sitting there are probably a dozen objects which you no longer “see” because you are so accustomed to their presence that you are no longer aware of them. Look at each one in turn, not staring, but with quick, easy glances, thinking about what you are regarding. That doorknob-could you have described it before? Now you know its approximate size, contour, the material of which it is made, its relative position on the door, because your mind and not alone your eyes observed it.
Even such a familiar phenomenon as a moving picture gives us what we believe we see rather than what we actually see. A series of still pictures provides us with an illusion of movement.
SEE A SMALL AREA AT A TIME
Instead of staring, trying to take in a whole picture at one time and thus defeating the object of central fixation, look at one small part of the picture, shift your gaze to another small part, and another, blinking naturally all the time. The smaller the area, the more clearly you will see it.
People who have acquired bad seeing habits always try to increase their area of vision by staring, which defeats its own purpose. Staring not only causes muscular tension but a lowering of vision. You can test this for yourself by staring fixedly at an object or a word on this page. After a few moments of this effort the letters lose their sharp clarity and become blurred.
Eyesight improvement can be achieved with consistent time, effort and proper eye health care!
CENTRAL FIXATION
The retina is a sensitive film on which the picture falls. But there is one point on the retina where the vision is perfect; that is the Macula Lutae, a point only one-sixteenth of an inch in diameter in the very center of the retina. When we focus at this point we have what is known as central fixation and our vision is perfect.
If you have lost the capacity of central fixation you are seeing with Eccentric fixation which often causes headaches, fatigue, pain or discomfort of some kind, such as twitching of the eyelids or the eyeballs. This twitching, by the way, can be stopped by pressing the sides of the base of the nose as high as the inner canthus with the forefingers of both hands, avoiding any pressure on the eyeballs.
Continue the pressure for several minutes, with the eyes closed, and you will obtain relief.
One way of checking on whether you are seeing by central or eccentric fixation is to look at a word on this page. Do you see it most sharply where you are looking or do you see it better when you look a little away from it? When you look at the top of a printed letter do you see the bottom of the letter more clearly than the top? If so, you have lost central fixation.
If you are to see, you must bring your mind to bear on what you see. Because the eye can focus sharply and is at its maximum power only on a very small area at a time, an attempt to see a larger area results in a blurring of physical vision and a lack of mental focus. Teach yourself to look at what you see, to watch one tiny area at a time. For when the central fixation is perfect, the eye sees perfectly.
THINK ABOUT WHAT YOU SEE
For significant eye sight improvement, give the object you are looking at your mental as well as your visual attention. The more clearly it registers on your mind, the more clearly it will register on the eye.
Test this out for yourself. In the room where you are sitting there are probably a dozen objects which you no longer “see” because you are so accustomed to their presence that you are no longer aware of them. Look at each one in turn, not staring, but with quick, easy glances, thinking about what you are regarding. That doorknob-could you have described it before? Now you know its approximate size, contour, the material of which it is made, its relative position on the door, because your mind and not alone your eyes observed it.
Even such a familiar phenomenon as a moving picture gives us what we believe we see rather than what we actually see. A series of still pictures provides us with an illusion of movement.
SEE A SMALL AREA AT A TIME
Instead of staring, trying to take in a whole picture at one time and thus defeating the object of central fixation, look at one small part of the picture, shift your gaze to another small part, and another, blinking naturally all the time. The smaller the area, the more clearly you will see it.
People who have acquired bad seeing habits always try to increase their area of vision by staring, which defeats its own purpose. Staring not only causes muscular tension but a lowering of vision. You can test this for yourself by staring fixedly at an object or a word on this page. After a few moments of this effort the letters lose their sharp clarity and become blurred.
Eyesight improvement can be achieved with consistent time, effort and proper eye health care!
Sunday, May 10, 2009
Foundation Fighting Blindness’ National Neurovision Research Institute Heralds Collaboration for Gene Therapy Advancements
Foundation Fighting Blindness’ National Neurovision Research Institute Heralds Collaboration for Gene Therapy Advancements
OWINGS MILLS, Md.--(BUSINESS WIRE)--The National Neurovision Research Institute (NNRI), the Foundation Fighting Blindness’ clinical trial support organization, announced today that one of its key partners, biopharmaceutical company Oxford BioMedica, has established a collaboration with sanofi-aventis, a major international pharmaceutical company, to develop and commercialize gene therapy treatments for vision-robbing retinal degenerative diseases that affect tens of millions of people around the world.
The collaboration will significantly bolster the development of the following gene therapy products: StarGen™ for the treatment of Stargardt disease, a form of early-onset macular degeneration; UshStat™ for the treatment of Usher syndrome, the leading cause of deaf-blindness; RetinoStat® for the treatment of age-related macular degeneration, the leading cause vision loss in people 55 and older in developed countries; and EncorStat™ for corneal graft rejection.
The development of treatments for these and other retinal degenerative diseases is a key goal of the National Neurovision Research Institute and is the basis for its partnership with Oxford BioMedica. In 2006, Paul and Diane Manning, with the National Neurovision Research Institute, established a partnership with Oxford BioMedica to advance gene therapies for these and other related retinal degenerative diseases.
“The investment from sanofi-aventis is a wonderful boost for the development of Oxford BioMedica’s gene therapy products and will greatly enhance our ability to move these emerging treatments into and through the clinical trial process,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “This collaboration affirms the great potential for gene therapy to treat and cure a number of retinal degenerative diseases including Stargardt disease and Usher syndrome that, as rare diseases, often do not receive the attention or investment necessary to bring about promising treatments.”
“An important goal of the Foundation, through NNRI, is to attract investment from large pharmaceutical companies for the development and production of treatments for inherited retinal disease,” says Morton Goldberg, M.D., chairman of the board of NNRI. “The NNRI-Oxford partnership is an excellent model of how NNRI collaborations can accelerate the translation of laboratory-based research into clinical trials, ultimately getting successful treatments for rare diseases like inherited retinal degenerations to the market and out to the people who need them.”
Based on the agreement, Oxford BioMedica will receive approximately €43 million ($56 million) from sanofi-aventis over a three-year period. Oxford BioMedica is eligible to receive additional fees if development efforts are successful.
The treatments will utilize Oxford BioMedica’s LentiVector® gene delivery technology to deliver healthy vision-saving genes to the retina.
About Foundation Fighting Blindness
The Foundation Fighting Blindness is the largest source of non-governmental funding for retinal degenerative disease research in the world. The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases. The Foundation has invested over $140 million to provide seed money for scientific research of diseases of the retina, which cause blindness. Further information is available at www.FightBlindness.org.
About National Neurovision Research Institute (NNRI)
NNRI is a recently-established non-profit support organization of the Foundation Fighting Blindness (FFB), the leading non-government funding source for inherited orphan retinal degeneration research. The mission of NNRI is to accelerate the translation of laboratory based research into clinical trials for treatments and cures of retinal degenerative diseases. It is a medical research institute that obtains support from government agencies, corporations and private foundations. It may also receive royalties or licensing fees from the drug discovery processes and commercialization of new therapies. Further information is available at www.nnri.info.
OWINGS MILLS, Md.--(BUSINESS WIRE)--The National Neurovision Research Institute (NNRI), the Foundation Fighting Blindness’ clinical trial support organization, announced today that one of its key partners, biopharmaceutical company Oxford BioMedica, has established a collaboration with sanofi-aventis, a major international pharmaceutical company, to develop and commercialize gene therapy treatments for vision-robbing retinal degenerative diseases that affect tens of millions of people around the world.
The collaboration will significantly bolster the development of the following gene therapy products: StarGen™ for the treatment of Stargardt disease, a form of early-onset macular degeneration; UshStat™ for the treatment of Usher syndrome, the leading cause of deaf-blindness; RetinoStat® for the treatment of age-related macular degeneration, the leading cause vision loss in people 55 and older in developed countries; and EncorStat™ for corneal graft rejection.
The development of treatments for these and other retinal degenerative diseases is a key goal of the National Neurovision Research Institute and is the basis for its partnership with Oxford BioMedica. In 2006, Paul and Diane Manning, with the National Neurovision Research Institute, established a partnership with Oxford BioMedica to advance gene therapies for these and other related retinal degenerative diseases.
“The investment from sanofi-aventis is a wonderful boost for the development of Oxford BioMedica’s gene therapy products and will greatly enhance our ability to move these emerging treatments into and through the clinical trial process,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “This collaboration affirms the great potential for gene therapy to treat and cure a number of retinal degenerative diseases including Stargardt disease and Usher syndrome that, as rare diseases, often do not receive the attention or investment necessary to bring about promising treatments.”
“An important goal of the Foundation, through NNRI, is to attract investment from large pharmaceutical companies for the development and production of treatments for inherited retinal disease,” says Morton Goldberg, M.D., chairman of the board of NNRI. “The NNRI-Oxford partnership is an excellent model of how NNRI collaborations can accelerate the translation of laboratory-based research into clinical trials, ultimately getting successful treatments for rare diseases like inherited retinal degenerations to the market and out to the people who need them.”
Based on the agreement, Oxford BioMedica will receive approximately €43 million ($56 million) from sanofi-aventis over a three-year period. Oxford BioMedica is eligible to receive additional fees if development efforts are successful.
The treatments will utilize Oxford BioMedica’s LentiVector® gene delivery technology to deliver healthy vision-saving genes to the retina.
About Foundation Fighting Blindness
The Foundation Fighting Blindness is the largest source of non-governmental funding for retinal degenerative disease research in the world. The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases. The Foundation has invested over $140 million to provide seed money for scientific research of diseases of the retina, which cause blindness. Further information is available at www.FightBlindness.org.
About National Neurovision Research Institute (NNRI)
NNRI is a recently-established non-profit support organization of the Foundation Fighting Blindness (FFB), the leading non-government funding source for inherited orphan retinal degeneration research. The mission of NNRI is to accelerate the translation of laboratory based research into clinical trials for treatments and cures of retinal degenerative diseases. It is a medical research institute that obtains support from government agencies, corporations and private foundations. It may also receive royalties or licensing fees from the drug discovery processes and commercialization of new therapies. Further information is available at www.nnri.info.
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