BBDF is excited to have the international spotlight on Batten disease last week. We have worked with Dr. Pearce in the past and agree that emerging technologies like stem cell therapy may be important in the fight against Batten disease. For this reason we’ve been funding the development of iPS stem cells with the New York Stem Cell Foundation over the past few years, as well as many other tools like it. As Dr. Pearce mentioned, stem cell therapy is probably still several years away. We are also closely following and in some cases supporting other new technologies/research initiatives like gene therapy and gene skipping, which are currently being applied to Batten Disease. Some are showing promising results. That is why the medical breakthrough discovered by our research funded at Texas Children’s Hospital is so important. The treatment slows the disease progression, buying precious time for more research. We will continue executing our 24 month, $6 million plan to validate the treatment option and to enlist the help of regulatory experts to prepare a package to present to the FDA for clinical trial approval. Your help comes at a crucial time and with your support we will continue advancing all treatment options to get it to the kids who need it most.
Read more about it here.