Research Update

Update on Current Research
By Florian Eichler, M.D. Massachusetts General Hospital

HSAN1 Trial Update: The two-year, double-blind, placebo-controlled trial studying the efficacy of L-serine in patients with HSAN1 is well underway. Eighteen individuals enrolled to date. Sixteen subjects were enrolled between mid-September and early December. Two more were enrolled in early April. To date, 16 subjects have completed their 6 month follow-up visit. We need two more subjects to reach our target enrollment of 20. No adverse effects have been attributed to L-serine.

The study is randomized to placebo versus study drug, and the investigators are blinded to who is on placebo and who is on study drug. Therefore we cannot at this point report any conclusions. However, we hope that in the second year, data analysis will allow us to report some insights. A full assessment will not be possible until all patients have completed the two year trial.

As HSAN1 is thought to be to a rare disease, we have been surprised at the rapid enrollment. We credit the Deater Foundation greatly for their support and attribute the successful enrollment to date in large part to their effort. It also shows that trials bring about greater disease awareness

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