The Foundation Fighting Blindness is committed to advancing the most promising sight-saving research, and has invested over $33 million into cutting-edge science since the organization was founded. Recognizing that science is tied to policy frameworks, the Foundation is also actively involved in health policy activities across Canada.
Many research groups are working to develop treatments and cures for Bardet-Beidl Syndrome. Experimental treatments can be divided into three broad categories:
- Protective Therapies
- Corrective Therapies
- Sight-Restoring Therapies
Protective therapies aim to stop (or at least slow) the damage caused by genetic mutations. Often protective therapies are not specific to one mutation, but may benefit people with different genetic forms of Bardet-Beidl Syndrome. These include treatments to stop the process of photoreceptor death (apoptosis), as well as cell-derived therapies that aim to help photoreceptors survive.
Some protective therapies are being developed to specifically to prevent the death of cone photoreceptor cells and thus, prevent the loss of central vision.
Corrective therapies aim to reverse the underlying genetic mutations that cause vision loss. If these therapies are successful they might prevent a person who is treated when first diagnosed, from ever developing vision loss. Corrective therapies might also help slow the disease in people whose vision has already been affected, especially in the earlier stages. The corrective therapies being developed now are specific to certain genetic forms of Bardet-Beidl Syndrome. Gene therapies, which replace a non-functioning gene, are one type of corrective therapy. Clinical trials of gene therapies for several kinds of inherited retinal diseases are underway, and the results so far are encouraging.
Sight-restoring therapies are also a growing area of research success. These therapies are intended for people who have already lost all, or much, of their vision. Stem cell therapies aim to replace the retina’s lost photoreceptors. There are promising early results with stem cell trials involving other retinal degenerative diseases, such as age-related macular degeneration. Retinal prosthetics, such as the Argus II or “Bionic Eye,” use computer technology to generate vision. The Foundation Fighting Blindness helped to support the first Canadian trial of the Argus II and continues to work closely with health policy experts across Canada to ensure that patients who could benefit from the Argus II device have access to this innovative treatment. Drug and gene therapies are also being developed that may give non-photoreceptor nerve cells in the retina the capacity to sense light.
Thanks to our generous donors, we are funding ground-breaking research in these areas. Click on the button below to review the full list of FFB-funded projects:
At the bottom of this webpage, you will find an updating list of stories that detail new research and health policy developments relevant for individuals affected by Bardet-Beidl Syndrome.