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2013 Part the Cloud Translational Research Funding for Alzheimer’s Disease (PCTR)

Phase 1 Trial for P75 Receptor Ligand

Frank Longo, M.D.
Pharmatrophix Inc
Menlo Park, CA - United States



P75 is a protein on the surface of nerve cells. Molecules outside the cell bind to P75, leading to activation of signaling pathways inside the cell. Under some conditions, the signaling pathways activated by P75 can lead to cell death similar to that which occurs in people who have Alzheimer's disease. Studies in animal models suggest that drugs which inhibit P75 can reduce the detrimental effects of the disease in the brain.

Dr. Longo and colleagues at Pharmatrophix, Inc. have planned a multipart, phase I trial of a drug known as LM11A-31-BHS, which inhibits P75 signaling. The purposes of the trial are to determine the doses of the drug that can be used safely in humans, and to study how the human body disposes of the drug. Participants in the trial will include young, healthy people as well as older people, so that the differences in possible side effects or drug disposal can be assessed. This study may lead to phase II and phase III trials of the drug, which will measure its ability to prevent or slow decline of brain function in individuals with Alzheimer's disease.

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