December 2007 Stop ALD Foundation Update

Extracted from our December 2007 fund raising letter...

Highlighted in the movie Lorenzo’s Oil, ALD is a terrible, often fatal, degenerative brain disease which affects 1 in 15,000 young boys. Back in 2000, few resources were dedicated towards aggressively finding a treatment for ALD. We started The Stop ALD Foundation to make a difference. Thanks to past donor support, our entrepreneurial drive, and personal passion, The Stop ALD Foundation has helped pioneer cutting-edge medical research responsible for significant advances not only for children with ALD, but also potentially for children and adults suffering from a host of genetic diseases. Exciting breaking scientific news was announced last month in Europe on a novel gene therapy clinical trial which was based on The Stop ALD Foundation’s direct involvement and co-funding. A team of researchers in France used gene therapy to treat two boys deteriorating from ALD who had no other therapeutic options. While still early, the two boys are producing the previously deficient key ALD protein. This treatment could have broad implications for a number of disorders from sickle cell anemia to leukemia. This news was reported in many media outlets, including Nature.

Our major goals at this time are continuing the current European gene therapy trial and launching a parallel collaborative trial in the U.S. Additionally, The Stop ALD Foundation is supporting and facilitating research in mesenchymal stem cell therapy, identifying new therapeutic targets, advancing diagnostics, prognostics, better understanding disease pathology, neonatal testing, and helping to promote collaboration of scientists and ALD researchers in the U.S. and around the world.

As we look towards 2008, we are energized by our recent scientific and clinical developments and remain committed to driving forward the development of ALD therapies on an aggressive timetable. Doing so requires not just our commitment of time and energy, but resources as well. By giving, you can help us drive research forward that will help children suffering from ALD and, hopefully, other diseases as well. “To save one life is as if you saved the world.”