The Foundation Fighting Blindness’ scientists, donors and volunteers made 2015 an outstanding year in our fight against blindness. As I tabulated the year’s top 10 research advances—all made possible through FFB funding—I realized that eight are for clinical trials of emerging therapies that are launching or underway.
Moving a treatment out of the lab and into a clinical trial is very challenging; it requires significant financial resources and drug-development knowledge. But advancing a therapy to this critical stage also brings it much closer to the people with retinal diseases who desperately need it. These trials only happen because of the time, money and expertise invested by the Foundation’s supporters.
You can’t wrap a bow around research progress, but I can’t think of a better holiday gift for those hard-working, well-deserving members of the Foundation’s family. So, without further ado, here’s a summary of the year’s top 10 advances made possible by FFB funding:
1. RetroSense Therapeutics received FDA authorization to launch a clinical trial of its optogenetic gene therapy. By harnessing surviving cells in the retina, the treatment holds promise for restoring some vision to people who are completely blind, regardless of the gene mutation causing their disease.
2. Spark Therapeutics will seek FDA approval for its RPE65 gene replacement for people with certain forms of Leber congenital amaurosis and retinitis pigmentosa (RP), thanks to encouraging results in a late-stage clinical trial. The treatment helped children and adults with severe vision loss better navigate an obstacle course. If approved, it would be the first FDA–approved gene therapy for the eye or an inherited condition.
3. ReNeuron has received FDA authorization to launch a clinical trial of its stem-cell therapy for people with RP. Researchers believe the treatment can save and restore vision by replacing lost photoreceptors and keeping existing ones healthy. This treatment has the potential to treat many retinal degenerative conditions, including Usher syndrome, because it might be used to replace photoreceptors regardless of the gene mutation causing the disease.
4. The University of California, Irvine, in collaboration with the company jCyte, has started a clinical trial of a stem-cell therapy designed to preserve and rescue photoreceptors in people with RP. The treatment might also work for other diseases, including Usher syndrome.
5. Applied Genetic Technologies Corporation has received FDA authorization to launch its gene-therapy clinical trial for retinoschisis, a disease that causes the splitting of retinal layers and severe vision loss in males.
6. Spark Therapeutics has launched a human study of a gene therapy for choroideremia, which leads to blindness in males by causing degeneration of several regions in the retina.
7. Thanks to encouraging results from its Phase I clinical study, StemCells, Inc. is launching a Phase II trial of its stem-cell therapy for saving vision in people with the dry form of age-related macular degeneration (AMD).
8. The Foundation and the biopharmaceutical company Vision Medicines are partnering to develop a drug known as VM200 for preserving vision in people with Stargardt disease. FFB is committing up to $7.5 million to co-fund VM200’s development. Vision Medicines plans to launch a human study of the therapy in 2016.
9. In a Phase I clinical trial, Oxford BioMedica’s RetinoStat® gene therapy for wet AMD stabilized vision in 21 patients over a 48-week period. As opposed to other wet AMD treatments which require monthly injections, a single injection of RetinoStat may last several years.
10. A gene therapy for X-linked retinitis pigmentosa (XLRP) rescued vision in canines with a late stage of the disease. The University of Pennsylvania researchers developing the treatment believe it may work for people with advanced vision loss. Applied Genetic Technologies Corporation is planning a clinical trial of XLRP gene therapy.
In 2015, the Foundation also continued to fund early lab studies for identifying new therapeutic approaches. We currently fund 106 projects at 61 prominent institutions around the world. These efforts include advancement of gene, stem-cell and pharmaceutical therapies as well as oversight of natural history studies and identification of outcome measures, both of which aid in the design and successful execution of clinical trials.
The Foundation’s growing patient registry, My Retina Tracker, is now being used by several companies to identify potential clinical-trial participants.
As we move into the new year, please stay tuned to the Foundation’s website, blog and other publications for the latest news on our research progress.
Most of all, I want to extend to the Foundation’s supporters my sincere gratitude for all they do to drive vision-saving treatments and cures.
Have a safe and happy holiday season.
Photo courtesy of National Eye Institute.