FDA gives 'go ahead' for phase II clinical trials of Bryostatin for the treatment of Alzheimer's disease

Apr 22 2009

The Food and Drug Administration (FDA) has given the Blanchette Rockefeller Neurosciences Institute (BRNI) the go-ahead to conduct Phase II clinical trials of Bryostatin for the treatment of Alzheimer's disease patients.

The drug showed pre-clinical efficacy to not only treat Alzheimer's disease symptoms, but also its underlying causes.

"We are very excited about the FDA's agreement for BRNI to move forward with clinical trials," said Dr. Daniel Alkon, Scientific Director of BRNI. "Bryostatin shows the promise to repair and protect against neurodegeneration caused by Alzheimer's disease, stroke and other brain trauma, as well as enhance the brain's normal memory functions."

Bryostatin was originally created as an anti-cancer chemotherapy. When BRNI scientists extensively tested PKC activators against Alzheimer's disease models, they discovered the drug's hidden potential to stop Alzheimer's disease. Over the past six years, the drug has shown remarkable possibilities. In preclinical testing, BRNI scientists experimented with Bryostatin on three species of Alzheimer's disease transgenic mice, each species based on different human Alzheimer's disease genes. The test results revealed that Bryostatin, and a related class of drugs discovered at BRNI, can reduce the toxic Alzheimer's disease protein A Beta, restore lost synapses, and protect against the loss of memory functions. In related preclinical testing, Bryostatin has been shown to enhance and restore memory by rewiring connections in the brain previously destroyed by stroke, head trauma, or aging itself.

The Phase II trials, slated to begin in approximately two to four months, will test these preclinical findings on human Alzheimer's disease patients as well as controls, along with Bryostation's effects on molecular targets in the human body, such as the signaling enzyme PKC. The drug's side effects will also be carefully monitored using low doses that were previously found to be generally benign in human cancer patients.

"With the potential to not just treat symptoms, but also stop the causes, the Bryostatin trial on Alzheimer's disease patients represents a new direction for the treatment of a disease with no current cure," said Alkon. "And the timing is crucial because as many as 5.3 million people live with Alzheimer's disease in the United States alone, with a new American developing Alzheimer's disease every 70 seconds."