Sustained-release implants on track for retinal vascular disease treatment

by:Elias Reichel

KAANAPALI, Hawaii — Sustained-release corticosteroids will play a significant role in retinal vascular disease, but the current implants still need improvements, one presenter here said.

"It is important to realize [steroids] will have a continued role in our treatment of diseases. They may also serve for adjunctive [therapies] like stem cell treatments and [instances] where transplantation or immunoreactions may occur," Elias Reichel, MD, who discussed outcomes using Ozurdex (dexamethasone intravitreal implant, Allergan) and Iluvien (fluocinolone acetonide intravitreal implant, Alimera), said at Retina 2011. "So, having a better delivery system for corticosteroids is very useful for many of the diseases we are seeing in our offices."

Challenges include needing to achieve and maintain clinically effective concentrations in the vitreous over time while maximizing efficacy and minimizing adverse effects, Dr. Reichel said.

As part of a 6-month study, the dexamethasone implant demonstrated 2- to 3-month peak efficacy, had a low incidence of IOP issues and conferred a cataract progression of approximately 5%. Additionally, 45% of the patients gained three lines of visual acuity, Dr. Reichel said.

The U.S. Food and Drug Administration has approved the device for all retinal venous obstructive disease.

The fluocinolone implant is now part of a 36-month study and has demonstrated efficacy to at least 30 months. Between 30% and 40% of patients gained at least three lines of visual acuity. However, the device has a moderately higher risk of IOP issues and a cataract progression rate of 50%, according to Dr. Reichel.

The device has received a complete response letter from the FDA.

"Current formulations that we have are good but can be better," Dr. Reichel said. "We really have to look at longer duration and this reduced risk trade-off."

* Disclosure: Dr. Reichel is a consultant to Alimera and Allergan.

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.

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)

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/

German company's retinal implant can restore vision

by: Retina Implant

Restoring vision to the blind is the sort of feat reserved for ancient religious texts and modern science fiction novels. But a company in Germany did just that with an eye implant.

Retina Implant AG is in the process of developing a sub-Retina Implant, designed to be inserted into the eye to treat back-of-the-eye disorders. A first clinical trial showed that the device can enable people suffering from a certain type of macular degeneration to see. The patients had retinitus pigmentosa, an inherited and incurable degenerative condition that causes tunnel vision and often, eventually, complete blindness. Retina Implant estimates that the condition affects about 200,000 people in the U.S. and Europe.

In 1995, the company’s founder and current board member Dr. Eberhart Zrenner said that implanting a chip inside the eye was as far-fetched as using the space shuttle to get to an adjacent star in the Milky Way. But Dr. Zrenner, who is also chairman of the University of Tuebingen Eye Hospital in Germany, didn’t give up. Ten years later the company launched its first human trial. At the time, Retina Implant also began talking to venture capital firms in Europe, but the technology was deemed too risky. It wasn’t until a wealthy businessman, who was personally interested because he had relatives who suffered from vision loss, that the company found significant outside investment.

Retina Implant isn’t the only group working on bionic-eye-like projects, but it seems to have a jump on the competition. Scientists at the Mass. Institute of Technology are also conducting clinical trials with their own version of a sub-Retina Implant, but don’t anticipate implanting their device in humans until 2013. Sylmar, Calif.-based Second Site Medical Products Inc. is developing a device for sufferers of retinitus pigmentosa, but that’s an epi-retinal prosthesis used in conjunction with an external camera attached to eyewear.

Retina Implant’s product sits underneath the retina ’directly replacing light receptors lost in macular degeneration,’ according to the company. Its most advanced and studied sub-Retina Implant is a 3mm-by-3mm microchip with an array of approximately 1,500 photosensitive electrodes. Electrical power for the device is provided inductively through transmitter coils attached to the skin.

In November, the company published results (PDF) from the first in-human clinical trial that proved the device can work in the Proceedings of the Royal Society B, a peer-reviewed academic journal. The company’s research with 11 human subjects showed that people who had been completely blind had regained up to two percent of their vision. enough to recognize letters in a newspaper headlines and see utensils before them when seated at a table. That number may seem insignificant ’ but it’s impossible to calculate the value a blind person places on seeing again, no matter how dimly.

In October, Retina Implant announced that it would increase its manufacturing footprint 25-fold (PDF).

Retina Implant is in the process of commencing a larger, 60-patient clinical trial in several sites across Europe. In this trial, the subjects will have the option to keep the implant permanently. In the first trial, the chips were all removed after four months except for one patient who lobbied the government to allow him to keep the device because his outcomes were so positive. To date, no complications have been reported, according to the company.

Retina Implant believes its device can win CE Mark approval in the European Union as soon as the end of 2011, but it does not have yet have definite plans for entering the U.S. market.

MassDevice recently spoke with Retina Implant CEO Walter Wrobel about the difficulties of developing such a technically advanced medical device and his company’s efforts to prove the device’s effectiveness enough to get it to market.

Drug Therapy To Cure Macular Degeneration

By Administration

Lucentis-is a drug therapy based on the cancer drug Avastin for wet macular degeneration. The treatment involves periodic injection of the drug directly into the eye. A higher percentage of those treated with Lucentis have shown improved vision than with any other treatment. Macugen-is another drug therapy for macular degeneration. It also involves periodic injections directly into the eye. It employs a molecule that attacks a protein involved with the growth of macular degeneration related blood vessels. macular degeneration treatment Vitamins and Minerals-are a treatment that is being used to treat dry macular degeneration. There is evidence to suggest that vitamins and minerals that have antioxidant properties will slow the progression of intermediate dry macular degeneration to the advanced form. The treatment typically involves large doses of Vitamin C, Vitamin E, Beta Carotene and Zinc Oxide.
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Acceleron's ACE-041 Demonstrates Clinical Efficacy

By Acceleron
CAMBRIDGE, Mass – November 19, 2010 – Acceleron Pharma, Inc., a biopharmaceutical company developing novel protein therapeutics that regulate the growth and development of tissues and cells, including muscle, bone, red blood cells, and vasculature, today announced the presentation of interim results from the first-in-human clinical study of ACE-041 in patients with advanced cancer at the 22nd EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics in Berlin, Germany. The presentation was given by Professor Sunil Sharma, the Jon and Karen Huntsman Presidential Professor of Cancer Research at the Huntsman Cancer Institute, University of Utah, Salt Lake City, Utah.

“It has been very encouraging to see so many signals of efficacy in this trial, in particular because these are end-stage cancer patients,” said Dr. Sunil Sharma. “It is also important to note that while we have demonstrated significant activity with ACE-041 monotherapy in this study, we might expect to see even more efficacy in future studies with ACE-041 used in combination with other therapies.”

“The clinical activity and safety profile of ACE-041 confirms our understanding that the ALK1 pathway plays a fundamental and distinct role compared to the VEGF-axis in tumor angiogenesis,” said Dr. Matthew Sherman, M.D., Chief Medical Officer at Acceleron Pharma. “This has significant implications because nearly all patients treated with VEGF inhibitors eventually either fail therapy or develop resistance to these drugs. There is a tremendous need for novel therapies that go beyond VEGF to target the tumor blood supply.”

In a phase 1, first-in-human clinical study, patients with advanced-stage tumors were treated with multiple ascending doses of ACE-041, to evaluate the safety and pharmacokinetics of ACE-041. This study also explored antitumor activity of ACE-041.

Summary of interim findings presented:

· ACE-041 subcutaneously (SC) injected once every three weeks (q3w) is generally well-tolerated · Common side effects include mild or moderate peripheral edema, fatigue, nausea, headache, anorexia, and anemia. A single case of Grade 3 congestive heart failure was reported · Toxicities commonly associated with VEGF inhibition (hypertension, proteinuria, or bleeding) have not been observed · ACE-041 pharmacokinetics support dosing every 3 weeks · One patient with refractory head and neck cancer achieved a partial response and three patients had prolonged disease stabilization · Rapid reduction in tumor metabolic activity observed in several patients, measured by FDG-PET imaging · An expanded cohort study is ongoing at the dose level intended for Phase 2 studies,

ACE-041 is being developed for the treatment of advanced cancer and age-related macular degeneration (AMD).

About ACE-041

ACE-041 is a recombinant receptor fusion protein that inhibits angiogenesis by preventing BMP9 and BMP10, members of the TGFβ protein superfamily, from interacting with activin receptor-like kinase 1 (ALK1), a receptor that is found on proliferating endothelial cells. ACE-041 inhibits ALK1 signaling, which is required for the development of mature, functional capillary networks. In animal studies, treatment with ACE-041 inhibits tumor angiogenesis and growth and ocular neoangiogenesis. In a clinical study of patients with advanced, refractory solid tumors, treatment with ACE-041 was generally well-tolerated and antitumor activity was observed, resulting in tumor shrinkage and stabilization of disease. ACE-041 is being developed for the treatment of advanced-stage cancer and age-related macular degeneration (AMD).

About Acceleron Pharma

Acceleron is a privately-held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel biotherapeutics that modulate the growth of red blood cells, bone, muscle, fat and the vasculature to treat musculoskeletal, metabolic and cancer-related diseases. Acceleron’s scientific approach takes advantage of its unique insight into the regenerative powers of the TGF-β superfamily of proteins. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company’s internal GMP manufacturing capability to rapidly and efficiently advance its therapeutic programs. The investors in Acceleron include Advanced Technology Ventures, Alkermes, Bessemer Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For further information on Acceleron Pharma.

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.

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.

Retinal Disease Highlights From the 2010 AAO Meeting

By Dr. Randall Wong

Many of us just returned from the largest ophthalmic “trade show” in the world. The American Academy of Ophthalmology convened in Chicago last week. This meeting was combined with the Middle East Africa Council of Ophthalmology (MEACO). Perhaps 40 K attended the meeting.

I also attended the 2 day retinal subspecialty meeting which preceded the larger AAO meeting. Thus, I had 5 days to expand my knowledge.

Most of the congress was focused on technology, especially electronic medical records. Few discoveries were revealed. This may be for two reasons: there is nothing really new going on right now, and/or, the Internet allows such rapid sharing of information, that it is impossible to “wow” anyone at this meeting. Probably both are true.

With regard to retinal disease, my particular specialty, there is little new, but lots to be excited about.

Avastin and Lucentis continue to be the mainstays of treatment for wet macular degeneration. There is evidence that the two drugs are similar in clinical efficacy…a notion I support. It is likely that these types of drugs will be delivered with a sustained release system, thus, obviating the need for repeated intraocular injections.

Ozurdex is now indicated for the treatment of uveitis. It was originally FDA approved for the treatment of RVO only. By itself, not earth-shattering, but does make sense clinically. Sustained release steroids for chronic intraocular inflammation. Sounds much better.

The highlight of the meeting is the potential for Iluvien to be FDA approved soon. Iluvien is similar to Ozurdex in that both are injectable intraocular sustained release systems. Iluvien will be a sustained release system that delivers intraocular steroids for the treatment of diabetic macular edema. While this is a particularly promising development for patients with diabetic retinopathy, this has larger implications for eye treatment overall.

What Does This Mean? Illuvien is likely to be the second FDA approved intraocular drug delivery system. This will be a significant endorsement of drug delivery to the eye. We are entering a new era of pharmaceutical therapeutics; sustained release inside the eye. For now, we are focused on retinal disease. But soon, very soon, devices will emerge promising better therapeutics for almost any eye condition.

Just think, glaucoma may be treated by such a device.

Novartis therapy Lucentis recommended for approval in EU to treat Diabetic Macular Edema

By Thomas Reuters
- CHMP positive opinion supports Lucentis approval in EU for treatment in patients with visual impairment due to diabetic macular edema (DME)

- Pivotal data shows Lucentis provided rapid, superior and sustained vision gains compared to the current standard of care

- Diabetes-associated eye diseases such as DME are a leading cause of blindness in most developed countries in the working-age population

Basel, October 22, 2010 - Novartis has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for Lucentis(R) (ranibizumab) for the treatment of patients with visual impairment due to diabetic macular edema (DME), a leading cause of blindness in the working-age population in most developed countries.

"Lucentis was designed specifically for use in the eye, and its efficacy and safety have now been demonstrated in patients suffering loss of vision due to diabetic macular edema through a robust program of clinical trials," said David Epstein, Division Head of Novartis Pharmaceuticals.

The submission was supported by data from two Novartis-funded clinical trials, RESTORE and RESOLVE, which showed that Lucentis was superior in providing rapid and sustained visual acuity gain versus sham (dummy therapy) or laser therapy, the current standard of care. The RESTORE study showed patients treated with Lucentis alone or with Lucentis plus laser therapy achieved an average 5.9 letters and 5.5 letters gain in visual acuity at 12 months, respectively, compared to laser-treated patients as measured on a standard ETDRS eye chart.

The RESOLVE study showed that Lucentis-treated patients achieved an average 11.7 letters gain in visual acuity at 12 months compared to sham-treated patients, some of whom received laser treatment.

The pivotal data from RESTORE and RESOLVE are further supported by results of an independent US study conducted by the Diabetic Retinopathy Clinical Research Network (DRCR.net), showing that at one year nearly 50% of patients' eyes treated with Lucentis and laser therapy improved their visual acuity by 10 letters or more, compared to 28% with laser alone. In addition, the study demonstrated superior gains in visual acuity among Lucentis-treated patients up to two years.

Lucentis was generally well tolerated in clinical studies, either when given as monotherapy or when combined with laser treatment. Its safety profile was consistent with that previously reported in large controlled clinical trials, and in rigorous monitoring since Lucentis was first approved for wet age-related macular degeneration (AMD). Lucentis is currently licensed in more than 85 countries for the treatment of wet AMD.

Diabetic macular edema (DME) is a consequence of diabetic retinopathy - the most common diabetic eye complication, characterized by changes in the blood vessels of the retina - to the light-sensitive layer at the back of the eye. In patients with DME, leakage from these abnormal blood vessels occurs in the central portion of the retina, called the macula. Because this part of the eye is responsible for sharp central vision, DME can lead to significant visual impairment. Visual impairment due to DME affects approximately 1-3% of patients with diabetes, and DME is a leading cause of blindness in the working-age population in most developed countries.

Lucentis offers an entirely new pharmacological approach to treatment compared to the current standard of care for DME that involves the use of laser burns to stop the capillary leakage and to reduce swelling. Lucentis is an antibody fragment that is injected into the eye and acts by neutralizing vascular endothelial growth factor (VEGF), a protein that is known to increase vascular permeability, resulting in capillary leakage and macular edema in patients with diabetes.

Lucentis was developed by Genentech and Novartis. Genentech has the commercial rights to Lucentis in the United States where Lucentis is also approved for the treatment of macular edema following retinal vein occlusion (RVO). In addition, Genentech is conducting two Phase III studies, RISE and RIDE, in patients with diabetic macular edema. The results are expected in 2011. Novartis has exclusive rights in the rest of the world and plans to file in the European Union for approval of Lucentis for the treatment of visual impairment due to macular edema following RVO.

Disclaimer

The foregoing release contains forward-looking statements that can be identified by terminology such as "recommended," "plans," or similar expressions, or by express or implied discussions regarding potential new indications or labeling for Lucentis or regarding potential future revenues from Lucentis. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results with Lucentis to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Lucentis will be submitted or approved for any additional indications or labeling in any market. Nor can there be any guarantee that Lucentis will achieve any particular levels of revenue in the future. In particular, management's expectations regarding Lucentis could be affected by, among other things, unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; competition in general; government, industry and general public pricing pressures; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; the impact that the foregoing factors could have on the values attributed to the Novartis Group's assets and liabilities as recorded in the Group's consolidated balance sheet, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

Complement inhibition may be the future of dry AMD therapy

CHICAGO — Ongoing clinical trials exploring the use of complement inhibitors for treating nonexudative age-related macular degeneration offer promise for a disease state that has long been considered difficult to treat.

A number of recent studies have implicated the complement immune system in AMD. However, nonexudative AMD remains a "uniquely human disease," and, therefore, no animal model exists in which to prove potential therapeutic strategies, Philip J. Rosenfeld, MD, PhD, said here are Retina Subspecialty day preceding the joint meeting of the American Academy of Ophthalmology and the Middle East Africa Council of Ophthalmology.

As a result, Dr. Rosenfeld said, human trials will prove important in determining whether complement inhibition is of any real clinical benefit.

Several such trials are already underway. Alcon is currently investigating its POT-4 (AL-78898A) anti-C3 cyclic peptide in human trials. There is some evidence that POT-4 may also affect VEGF expression in the retina, Dr. Rosenfeld said.

Separately, Genetech/Roche has an anti-factor D (FCFD4514S) that inhibits the C3 and C5 alternative pathway convertases. Phase 1 studies have been successfully completed with that agent, Dr. Rosenfeld said.

Two separate C5 inhibitors are being studied: Eculizumab/Sollris (Alexion) and ARC1905, an anti-C5 aptamer (Ophthotech).

"One of the advantages is that while you block the downstream activated species, you preserve the more proximal species that are important for the clearance of bacteria, particularly the encapsulated organisms," Dr. Rosenfeld said.
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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.

Retinal Degeneration Can be Prevented with Nanotechnology Gene Therapy

Friday, 01 October 2010

Researchers from Tufts University published results from a study showing vision-saving gene therapy to the eyes can be delivered via nanotechnology. A protein called Glial Cell Line-Derived Neurtrophic Factor (GDNF) can protect the eyes from diseases of the retina, such as and retinitis pigmentosa.
The previous method for delivering genetic material with a virus had several problems and side effects. Although the study is in its infancy, and the results are temporary, mice injected with the GDNF carrying nanoparticle had a 3.9 to 7.7-fold reduction in damage to the retina. Seven days after treatment, the GDNF-nanoparticle treated mice had up to 39% better eyesight than mice in the control group.
Two weeks after the treatment, GDNF-nanoparticle-injected mice had nuclear layers of the retina that were almost 24 to over 39 percent thicker than control mice. However, after 14 days, these advantages seem to have vanished. According to Dr. Brett Katzen, "The idea of using gene therapy to help cure - or at least improve - the eyesight of patients with macular degeneration should be exciting to eye doctors everywhere. This is a great first step." The research shows an incredible potential for nanotechnology to develop an effective carrier for delivering gene therapy.
This therapy can then lead to a new generation of treatments for retinal diseases. Age-related macular degeneration is the leading cause of blindness in Americans over the age of 65.

Glaucoma eye drops showing some effect against Macular Degeneration

by Dr. Edward Paul

Eye redness was the clue that something was happening to Celia Ramirez's vision. Although she wasn't having trouble driving or doing other tasks, her children urged her to have a checkup. It turned out she was in the early stages of age-related macular degeneration (AMD), a disease that attacks a person's central field of vision.

Mrs. Ramirez, who lives in the Rio Grande Valley, had surgery that successfully controlled her eye pressure, but her central vision continued to degrade.Eye specialist William Sponsel, Ph.D., associate professor and director of research at the Department of Ophthalmology at the University of Texas Health Science Center at San Antonio (UTHSC).Found a treatment with a new combination of drugs.

The miracle is the carbon dioxide, which is crucial for vision. The healthy eye produces enough carbon dioxide to dilate blood vessels around the retina and maintain proper blood flow. The ailing eye does not produce enough. This holds important implications for the treatment of patients with blinding eye diseases such as AMD and glaucoma, which are marked by diminished circulation of blood in the back of the eye. Eye pressure also is a conventional barometer of eye health.

About 60 of 65 patients have seen their vision improve while receiving treatment at UTHSC's teaching hospital, University Hospital. "The patients are receiving combination drug therapy, including agents that trick the eye into retaining more of its carbon dioxide," Dr. Sponsel said. "We have patients with glaucoma and no AMD, patients with AMD and no glaucoma, and patients with both disorders. All appear to demonstrate benefit in the central visual area, regardless of the cause of that visual loss."

The Health Science Center is the first medical center to initiate this combination therapy for patients with central visual loss, Dr. Sponsel said. Considerable research yielded the conclusions that now help patients. "You don't expect these patients to get better, you expect them to get worse," he said. "We have seen dramatic results that bode well for treatment of these disorders in the future."

The researchers measure patients' sensitivity to light on visual function tests. An increase of 10 points represents more than a million-fold increase in actual visual acuity. Mrs. Ramirez's vision in the macula -- the central visual area that enables perception of letters and colors -- was scored as virtually zero on early tests. After the recent treatment, her score is now 24.

The miracle agents are called "carbonic anhydrase inhibitors" (CAI's). Given as eye drops, CAI's reach the back of the eye rapidly and slow the clearance of carbon dioxide while increasing the supply of nutrients. They help dilate blood vessels within and behind the retina, which is the structure that receives, processes and transmits visual images relayed from the brain.

Dr. Sponsel pursued this line of research in an intriguing way -- after discovering that adults who hyperventilated and rapidly blew off their body's carbon dioxide showed decreased vision, eye pressure and circulation in the back of the eye. He was treating subjects one week with a CAI called dorzolamide and the next week with placebo eye drops. During hyperventilation, the subjects on dorzolamide maintained good light sensitivity in their central field of vision, while the same subjects during placebo treatment showed central visual loss. Dr. Sponsel holds two U.S. patents on this work. One valuable aspect of the research is that it proves increased eye pressure is not necessarily the definitive indicator of eye vessel disease.

FDA grants pSivida, Alimera expedited review for macular degeneration therapy

FDA grants pSivida, Alimera expedited review for macular degeneration therapy
August 31, 2010 by MassDevice staff

pSivida Corp. and Alimera Sciences win priority review status for their Iluvien drug/device combination, designed to treat diabetic macular degeneration.
PSDV, ALIM logos

The Food & Drug Administration put pSivida Corp. (NSDQ:PSDV) and Alimera Sciences Inc. (NSDQ:ALIM) on the fast track, granting an expedited review for their drug/device combination designed to treat diabetic macular edema.

Watertown, Mass.-based pSivida and Alpharetta, Ga.-based Alimera filed a new drug application with the FDA in June for Iluvien, a sustained release drug delivery system that delivers flucocinolone acetonide, a steroid, for the treatment of DME.

The federal watchdog agency granted priority review status to the application, moving the goal for finishing the review from 10 months back to six months.

That means a response could drop during the fourth quarter, pSivida president and CEO Dr. Paul Ashton said in prepared remarks, adding that FDA approval would trigger a $25 million milestone payment from Alimera.

The milestone payment wouldn't be the first time Alimera ponied up for pSivida. Its $72 million IPO in April triggered the repayment of a $15 million loan and $225,000 in interest. The companies are seeking European approval for Iluvien. If it makes it to market, pSivida will collect 20 percent of the net profits.

The FDA has already cleared a pair of pSivida products: Retisert, for the treatment of posterior uveitis, and Vitrasert for the treatment of AIDS-related cytomegalovirus retinitis. Both are licensed to Bausch & Lomb Inc. The company is also developing other ophthalmic products with Pfizer Inc. (NYSE:PFE), its largest shareholder, and has products of its own in the pipeline outside of ophthalmology, according to a press release.

*

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.

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

Medicine from Moss to produce human protien

Diabetics use human insulin produced in bacteria in order to treat their metabolic disorder. Many other genetically engineered proteins are also on the advance. They are being used for diagnosis as well as for therapy.

Whereas insulin used to be extracted from slaughterhouse waste, today it is produced genetically in bacteria. However, more complex proteins have to be synthesised in more complex organisms. This takes place mostly in bioreactors using animal cell lines. Biotechnologist Prof. Ralf Reski from Freiburg, Germany, has developed the moss Physcomitrella patens into a safe and inexpensive alternative supplier of medicine.

His group has now, under Dr. Eva Decker, for the first time succeeded in producing a human protein in a moss bioreactor, which has been assigned the "orphan drug" status by the respective EU authorities. This means the development and approval of such medication receive particular support from the authorities. In many people the amount of this protein decreases with old age - with severe consequences. Eva Decker explains: "With the complement factor H we have produced a protein in moss that otherwise occurs only in blood and is important for the immune system. Not enough of this protein in older people is the main cause of blindness for 50 million people worldwide. This age-related macular degeneration (AMD) is a problem, particularly in industrialised countries."

Biochemists from the Freiburg Centre for Systems Biology under Dr. Andreas Schlosser were able to show with the help of high-performance mass spectrometers that the human factor H engineered into and produced by moss was a complete protein. Infection biologists headed by Prof. Peter F. Zipfel from the Hans-Knöll-Institute in Jena, Germany, were able to prove in biological assays that factor H from moss is fully functional. "Currently factor H is not available in pharmacies, so treatment for AMD with this protein is not possible. To date recombinant production of factor H was barely feasible. I am convinced that for the first time the moss bioreactor is a promising option", says Peter Zipfel.

This work was supported by the German Federal Ministry of Education and Research (BMBF), the Freiburg Initiative for Systems Biology and the Cluster of Excellence BIOSS.
Dr. Annette Büttner-Mainik, first author of the publication, was a Kekulé scholarship holder from the endowment fund of the German Chemical Industry (FCI).
The title of the original publication is: Annette Büttner-Mainik, Juliana Parsons, Hanna Jérôme, Andrea Hartmann, Stephanie Lamer, Andreas Schaaf, Andreas Schlosser, Peter F. Zipfel, Ralf Reski, Eva L. Decker (2010): Production of biologically active recombinant human Factor H in Physcomitrella. Plant Biotechnology Journal, doi: 10.1111/j.1467-7652.2010.00552.x.

"It will take a while before medication produced in moss is available in pharmacies", says Ralf Reski, member of the Innovation Think Tank of the governor of Baden-Wuerttemberg. "We are further optimising the moss bioreactor using methods from Systems Biology and Synthetic Biology. However, the implementation of clinical studies and the setting up of industrial production is long-winded and expensive; this is the task of industry and not of university research."

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.