There Is Hope

Oct. 4,2008

In August 2008 there was a bi annual conference for choroideremia in Baltimore Maryland. This conference was very exciting because of all the hope that the doctors and researchers shared with the CHM members. Dr. Miguel Seabra ,from the Imperial College in London, told us that because of his research we can look forward to gene therapy trials in 2010. Gene therapy will replace the REP 1 gene that is missing in choroideremia patients. Also, the company Neurotech, which developed the Encapsulated Cell Technology with CNTF, now is in Phase 111 trials. Most likely, if the FDA approves, the ECT should be on the market for Choroideremia patients by 2010. The ECT has shown to not only halt the progression of the disease but also regenerate dying cells. The hope is to get the ECT before all the retinal cells have died, thereby improving the vision in those affected.We heard from Dr. Jean Bennett from the Scheie Eye Institute in Pennsylvania. She was recently seen on the news for her work in LCA, Lebers Congenital Amaurosis, also a blinding singlegene defect like CHM. She and her team of fellow researchers found a gene therapy cure and are restoring sight to those affected with LCA. Dr. Bennett said she is now moving on to choroideremia ,also a disease missing a single protein and defect. All of this information filled all of us with so much HOPE!!! We are so close but not quite there yet. We must continue to raise money to help these researchers to continue their important work finding a cure for choroideremia.