New Drug Study Offers Promise of Brain Cell Regeneration for Alzheimer’s Patients

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A promising first-in-class drug that stimulates the creation of new nerve cells in the brains of "Alzheimer's mice," will soon be tested in the brains of human patients with the promise it can help people in the early stages of the disease, based on research funded by the Cure Alzheimer's Fund.

A promising first-in-class drug that stimulates the creation of new nerve cells in the brains of "Alzheimer's mice," will soon be tested in the brains of human patients with the promise it can help people in the early stages of the disease.

New research by Dr. Sam Gandy of Mount Sinai School of Medicine in New York and recently published in the journal Molecular Psychiatry outlines the extraordinary promise of the drug, known as an "mGluR2/3 blocker."

The learning behavior of Alzheimer's mice being treated with the mGluR2/3 blocker has been sustained at normal levels, Gandy’s study has revealed, in contrast to the steady decline of mice not being treated.

"It's extraordinary that, in such a short time, we have moved from ordinary skin cells to induced pluripotent stem cells in a Petri dish, to lab-generated human nerve cells, and now to a drug that could potentially create those cells inside a human brain," said Gandy.

"We realize that we are unlikely to have much impact in late stage Alzheimer's, but we are cautiously hopeful that this drug might arrest Alzheimer's disease at an early stage so that patients can remain functional for more extended periods."

The drug originally caught the attention of Gandy and his team for its possible ability to inhibit production of the toxic amyloid beta 42, associated with Alzheimer's disease. Created by the Japanese pharmaceutical firm Taisho and originally studied for depression, the drug acts by stimulating stem cells in the hippocampus to divide and form new nerve cells.

With funding from Cure Alzheimer's Fund, Gandy’s team conducted a pilot study of the drug's effects on a particular strain of mice. That study produced such promising results that it has drawn $1 million in funding from the Veterans Administration "MERIT Review" program that supports Gandy's lab at the James J. Peters VA Medical Center in the Bronx. The Louis B. Mayer Foundation and the Sarah and Gideon Gartner Foundation provided additional funding.

The mGluR2/3 blocker has also been administered to healthy young human subjects, and so far has seen to be safe. The next step for Gandy's team will be to treat elderly human subjects with the drug to test safety in this population before gearing up to test the drug in Alzheimer's patients.

The mGluR2/3 blocker is one of the few drugs being researched that holds promise for repairing brains damaged by neurodegenerative disease.

All of these efforts proceed from the international Stem Cell Consortium formed by Gandy in 2012 and funded by Cure Alzheimer's Fund. Gandy's mGluR2/3 blocker is one of five brain cell regenerating agents currently undergoing testing in labs around the world.

"Helping incubate cutting edge science that can gain momentum with federal funding -- this is precisely why Cure Alzheimer's Fund exists," said Cure Alzheimer's chairman Jeffrey Morby.

http://www.nature.com/mp/journal/vaop/ncurrent/full/mp201487a.html (Molecular Psychiatry, 08/12/2014)

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