AMD is a long term and unpredictable disease. It progresses differently in each individual. Finding effective cures may involve hundreds of patients followed for 3, 5, or even 10 years. It is frustrating to wait years for a new drug to become available, but only rigorous science and continued follow-up can give us the answers we need.
- Because of the number of vulnerable seniors affected by AMD, we hear about a lot of treatments that may or may not be proven to help. Most research is performed by well-respected scientists monitored by their Institutional Review Board, or the National Eye Institute or Food and Drug Administration (FDA) to assure that work is done in an unbiased manner. Other studies do not meet these standards and results are anecdotal.
- If a practitioner claims a ‘secret’ or exclusive treatment effective against multiple medical conditions including AMD, proceed with caution. Members of the scientific and medical community share successful therapies with one another in order to advance science and improve patient care. If only one practitioner performs the treatment, this would be unusual.
- It is just this kind of situation that has encouraged the emergence of useless and ‘magic’ medicines and cures. Be alert to exaggerated claims and expensive therapies that sound too good to be true.
- All medical research, including AMD research, involves well-established steps. Research starts in the laboratory where scientists work on molecules, cells, or animal models to test theories and predict outcomes. These are called Pre-Clinical studies and there are no human participants.
- Clinical trials begin with Phase I trials, which test a treatment or drug on a small number of people for safety and correct dosing. Phase II involves more subjects who test the treatment for effectiveness and continued safety and dose investigation. Phase III expands the study to more subjects, comparing effectiveness to treatments already on the market and to monitor safety and side effects. The results of these studies, which may include several years of follow-up data are presented to the FDA to decide whether or not to approve the treatment for use by the general public. Bringing a new drug or treatment to the point of FDA approval averages 12 years, and the estimated cost of taking a new drug from concept to market exceeds $1 billion.
- To prevent bias in most Phase II/III clinical trials, neither the investigators nor the patients are aware if they are receiving the treatment or if they are a ‘control’ subject receiving a pretend or ‘sham’ treatment. The medication is coded and subjects are selected at random. These are called double masked studies and yield the most reliable results. Once the study is concluded, the code is broken and everyone can tell who got the placebo and who got the real drug. If a treatment is still in the research phase, and you are part of an organized clinical trial, you should not be paying for the therapy.
- Hundreds of clinical trials are underway at any time in this country. Some people are interested in doing their part for the advancement of science by participating in clinical trials. It is necessary in order to test new drugs and treatments, but participation comes with a obligation that may mean long follow-up and multiple visits to the doctor in order to gather data. Talk to your doctor if you are interested. He or she may be able to direct you to a study that will welcome your enrollment.
- There are multiple areas of active research underway for AMD. Many involve novel treatments to treat both wet and dry AMD, including novel drug delivery systems that avoid the use of needles, or drugs that will continue to be effective for several months instead of a few weeks.
- Stem cell research is an active area of research for AMD. Scientists are exploring ways to replace dead or diseased cells in the retinal pigment epithelium (RPE) with stem cells which would slow down or stop the progression of AMD. Stem cells are harvested from two different sources. One is called induced pluripotent stem cells which are collected from skin or other tissue cells and are reprogrammed in the lab to become RPE cells. The other cells are embryonic stem cells. As the name describes, these are collected from human embryos and can also be programmed to grow as RPE cells. Small clinical trials utilizing stem cells are just beginning at a handful of research centers.
- Gene therapy is another promising area of research in which a defective gene is replaced. Several genes that appear to be related to AMD have been identified as potential targets. It has been shown that a clean version of the missing or damaged gene can be introduced via an intravitreal injection. The hope is that the genetic mutation would be corrected and the progression of disease stopped. There are currently no clinical trials using gene therapy for AMD. In late 2017, the FDA approved the first gene therapy for a rare disease causing childhood blindness. Unlike AMD, this disease is caused by a single gene mutation. Over time, the knowledge gained from clinical trials on rare, single gene diseases can be applied to conditions like AMD.
- Other current research focuses on understanding the mechanisms of AMD, including links to other diseases of aging like Alzheimer’s, the role of nutrition, light exposure, and other environmental issues. Other researchers are focused on new technology as a way to improve or restore sight, including artificial vision and wearable and implantable devices.
- Visit CENTERWATCH if you are interested in reading more about current AMD clinical trials and to receive notifications about nearby clinical trials.
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