The Stop ALD Foundation is excited to announce that the University of Pennsylvania's Orphan Disease Center has awarded a one-year, $100,000 research pilot grant to Dr. Florian Eichler at Massachusetts General Hospital. Dr. Eichler and his team have already established an Adrenomyeloneuropathy (AMN) animal model proof of concept showing a gene therapy approach could provide benefit where no treatment is currently available. Today when young men with Adrenoleukodystrophy (ALD) begin showing adult-onset signs of AMN, they slowly lose their ability to walk. This pilot grant will help move Dr. Eichler's research program forward towards human studies. However, additional funds will still be necessary to initiate the study.
This $100,000 grant was a result of the Penn Orphan Disease Center's annual Million Dollar Bike Ride. The Million Dollar Bike ride focuses on raising money for rare disease research doubling money raised. The Stop ALD Foundation lead the ALD Team and raised a total of $50,000 of which The Stop ALD Foundation contributed $40,000, amplifying donor money into important research areas. Dr. Eichler's grant will advance much-needed research into a promising treatment for Adrenomyeloneuropathy (AMN). This $100,000 pilot grant is just the beginning and we encourage other donors and foundations to fund this important AMN research further to help it move towards a clinical trial.