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The ALS therapeutics program has three components; two are laboratory based. We have an active research program using models of cell death in a petri death both to analyze events that cause neuronal cell death and in ALS and to test drugs that may help. One member of our team, Dr. Piera Pasinelli, has devised a cell-based assay that mimics in cell culture aspects of nerve cell death triggered by mutant but not normal (wild-type) SOD1 protein. We also have a large program to study how certain drugs affect the course of ALS in the mouse model.
The third arm of the ALS therapeutics program involves human drug trials in ALS. This component is directed by Dr. Merit Cudkowicz who also directs the Clinical Trials Unit in the Neurology Department at the Massachusetts General Hospital. This group is currently conducting three clinical ALS trials: brain-dervied neurotrophic factor (BDNF) instilled directly in to the spinal fluid; BDNF delivered subcutaneously; and an anti-excitotoxic compound, topiramate.
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