AMD Research
New research is underway to develop additional treatments for both dry AMD and wet AMD. Several pharmaceutical companies are currently conducting trials of new drugs, while non-profit funders like the FFB and the Canadian Institutes of Health Research are working together to speed the translation of treatments from animal studies to human trials.
Learn more about:
- Research currently being conducted by FFB-funded scientists
- Clinical trials of new treatments for AMD
Research Currrently Being Conducted by FFB-Funded Scientists
The Foundation Fighting Blindness supports scientists who are making incredible advances in understanding and treating age-related macular degeneration. For example in 2009, Dr. Gilbert Bernier identified a gene that helps to control the aging of cells in the eyes and brain. This discovery may one day help us prevent conditions of aging like AMD, Alzheimer’s and Parkinson’s. Learn more about this discovery.
Until that day, research on age-related macular degeneration continues, with the support of donors like you. FFB projects and partnerships help fund pre-clinical studies that make the development of new therapies possible. Here are three ongoing projects that your donations will help to support:
Creating combinations of treatment for AMD
In partnership with the Canadian Institutes of Health Research, the Foundation Fighting Blindness is supporting Dr. Kevin Gregory-Evans and a team of experts at the University of British Columbia. Each member of this team has already been working to create treatments that target one aspect of the process of vision loss. By combining their efforts they hope to create more effective combination therapies that can be delivered in a single dose. Learn more about this research team.
Preventing the Death of Vision Cells in the Eye
The FFB has also partnered with the Canadian Institutes of Health Research to support a team of researchers led by Dr. Catherine Tsilfidis at the Ottawa Health Research Institute. This team is exploring ways to slow or stop the death of photoreceptors (cells that capture light and allow us to see) in the retina of the eye. While such treatment would not correct the underlying problem that leads to vision loss, it might preserve sight for many years. The Tsilfidis team has already shown that this approach works to protect the vision of blinded mice. This team hopes to be ready for human clinical trials testing this treatment within five years. Learn more about therapies designed to slow cell death.
Producing New Cells to Restore Sight in Failing Eyes
Dr. Gilbert Bernier at the Maisonneuve-Rosemont Hospital is receiving FFB funding to explore the use of stem cells as AMD treatment. Stem cells are very simple cells that can become other, more complex, cells to replace photoreceptors damaged by AMD. He recently patented a process that is effective for transforming stem cells into adult eye cells. His current studies are using mice to test whether these newly created cells will be an effective treatment for AMD. He and his partners hope to begin clinical trials in the next few years.
Clinical Trials of New Treatments for AMD
- Phase 1 trials involve very small numbers of people and are used to test the drug’s safety and to help determine a dose that people will tolerate. Such trials may give indications that the treatment is likely to be beneficial, but they are too small to provide solid evidence.
- Phase 2 trials involve 100-300 people and aim to provide the first conclusive evidence that a drug will be a useful treatment for human disease. The dose may also be fine-tuned at this stage.
- Phase 3 trials are large trials usually involving over 1000 people. These trials are designed to gather all of the evidence a company needs to have a drug approved by the government, including information about both positive outcomes and negative side effects.
- Phase 4 trials are done after the drug has received approval to gather additional information about the drug’s optimal use.
Dry AMD
Currently, the only treatment available in Canada for dry AMD is the AREDS formulation of nutritional supplements; however, an array of new therapies is now being tested. (Note: Most of these drugs will be renamed prior to marketing.)
Fenretinide is an oral drug from ReVision therapeutics, which has just completed phase 2 clinical trials. According to findings released this month (Sept 2010) at the Annual Meeting of the American Society of Retinal Specialists [1], it show that people with advanced dry AMD who took this drug once a day reduced the risk of developing wet AMD by 50%. The drug also appeared to reduce the size of the damaged area of the retina. More results of this trial are expected in the next year; ReVision therapeutics is expected to begin a phase 3 clinical trial in 2011.
NT-501 is a new treatment, developed by Neurotech, which has also just completed phase 2 clinical trials. This treatment involves a relatively minor surgery to implant a tiny capsule into the eye that contains genetically modified human cells. These cells produce a growth factor (CNTF), which protects the delicate cells of the macula from dying. Phase 2 results appear to show that this treatment can substantially slow vision loss in almost all treated patients (96%) but that lost vision is not restored. [2]
Photobiomodulation Therapy is is the use of non-thermal, non-laser light of specific wavelengths and energy to promote tissue repair [3]. It is already used and approved in Canada for some dermatology treatments such as wound healing and some cosmetic procedures. A phase 2 clinical trial is now underway in Toronto and in an American site evaluated the use of photomodulation to delay AMD progression – and potentially improve vision. This trial has now finished recruiting participants and the results are being evaluated.
An eye-drop medication (AL-8309B produced by Alcon Research) is also undergoing a phase 3 clinical trial, but little information has been published about it.
- ACU-4429 is an oral medication developed by Acucela Inc. A phase 2 study is now recruiting participants in two US centres.
- Eculizumab was first developed to treat a rare form of anemia. Alexion Pharmaceuticals is now testing an intravenous version in a phase 2 clinical trial, now recruiting at the University of Miami
- OT-551, from Othera Pharmaceuticals, is an eye-drop used several times a day to stop vision loss. A phase 2 trials is now underway in several centres in the US and is no longer recruiting new participants.
- POT-4, from Potentia Pharmaceuticals, aims to stop inflammation and tissue damage in the eye. A phase 2 trial for both wet and dry AMD is likely to begin this year.
Wet AMD
Treatments for wet AMD aim to prevent severe vision loss by reducing the growth of abnormal blood vessels and slowing the leakage of blood and fluid from these vessels into the retina. To date, anti-VEGF agents, such as Lucentis (ranibizumab), are the most effective therapies we have against the development abnormal blood vessel growth (choroidal neovascularization or CNV) and wet AMD.
VEGF Trap-Eye is a new anti-VEGF agent being developed by Regeneron Pharmaceuticals and Bayer Healthcare. It is currently being compared to Lucentis, in phase 3 clinical trials being conducted in centres in Canada and the US [7] as well as Europe, Latin America and Asia [8]. (These studies are no longer recruiting new participants). VEGF Trap-Eye is administered as by injection into the eye and will likely be given every two months as opposed to the monthly schedule recommended for Lucentis. See more about recent research on this treatment.
Anti-VEGF agents inhibit the growth of abnormal blood vessels by blocking the action of a one growth factor called VEGF. Additional therapies are being developed to inhibit other growth factors or to suppress inflammation in the eye. Nearly a dozen drugs are currently in early-stage clinical trials [6]. Several of these drugs will be administered daily as eye drops rather than by injection into the eye. A vaccine against abnormal blood vessel growth (anti-VEGFR) is also in phase one studies in Japan.
One of the most important approaches to the improved treatment of wet AMD in the near future is to combine several wet AMD therapies. The combined impact of several drugs acting at once may improve outcomes. Combination therapy studies are beginning or ongoing in many centres.
Steroid anti-inflammatory drugs may slow abnormal blood vessel growth, reduce the leakage of fluids from those vessals into the retina, and so reduce the swelling within the eye which is the major cause of vision loss in wet AMD . Kenalog (triamcinolone acetonide) is a steroid that has been approved for many years for other purposes, while Retaane (anecortave acetonide) is a new steroid drug that was created specifically to avoid some of the side effects that occur when Kenalog is injected into the eye. Not enough clinical evidence has been obtained yet for either of these drugs to be approved for use alone for wet AMD, in either Canada or the USA. However both may be useful in combination with other therapies. (Retaane has been approved in Australia to treat wet AMD).
Updated: Sept 15, 2010. This section has been reviewed by Dr. Peter Kertes, Associate Professor of Ophthalmology and Vision Sciences at the University of Toronto and Dr. William Stell, FFB Director of Research Programs and Professor of Ophthalmology at the University of Calgary.
| Learn more about AMD: | |
| What is AMD? | Risk Factors for AMD |
| Early Detection | Early Detection |
| AMD Treatment | Frequently Asked Questions |
- Findings presented at the Annual Meeting of the American Society of Retinal Specialists, Sept 2010.
- Findings presented at the Annual Meeting of the Association for Research in Vision and Ophthalmology, May 2009.
- Desmet KD, et al. Clinical and experimental applications of NIR-LED photobiomodulation. Photomed Laser Surg. 2006 Apr;24(2):121-8.
- Karmel M. Dry AMD: Under Attack on Four Fronts. EyeNet, Sept. 2009.
- Mata NL, Vogel R. Pharmacologic treatment of atrophic age-related macular degeneration. Current Opinion in Ophthalmology 2010; 21:190-196.
- Ni Z, Hui P. Emerging pharmacologic therapies for wet age-related macular degeneration. Ophthalmologica. 2009;223(6):401-10.
- Clinical Trial Listing: Vascular Endothelial Growth Factor(VEGF)Trap-Eye:Investigation of Efficacy and Safety in Wet Age-Related Macular Degeneration(AMD) (VIEW 1).
- Clinical Trial Listing: Vascular Endothelial Growth Factor (VEGF) Trap-Eye: Investigation of Efficacy and Safety in Wet Age-Related Macular Degeneration (AMD) (VIEW 2)
- Clinical Trial Listing: Efficacy of Ranibizumab in Combination With Photodynamic Therapy for Wet Age-Related Macular Degeneration