Alzheimer's Matters Blog

Closing in on a Cure: Dr. Frank Longo and C-31

December 15, 2016

Category: Developing Drugs

Dr. Frank Longo


The Alzheimer’s Drug Discovery Foundation has supported over 20% of all the treatments for Alzheimer’s currently in clinical trials. One of the most promising of these is C-31 (also called LM11A-31), which was developed by Frank Longo, MD, PhD. Dr. Longo, a professor at the Stanford School of Medicine and founder of the biotechnology firm PharmatrophiX, received his first grant from the ADDF in 2000 for what was then just an idea. He thought that affecting the p75 receptor on the surface of neurons might keep those neurons alive and slow the progression of Alzheimer’s disease. With seed funding from the ADDF, he set out to find compounds to do just that.

And he succeeded. Sixteen years and six ADDF grants later, he’s testing the effectiveness of C-31 in phase 2 clinical trials in Alzheimer’s patients, which are expected to be completed in 2018. By affecting the p75 receptor, the drug counteracts abnormal signals in the brain. Dr. Longo explains, “When this abnormal signaling occurs, synaptic function begins to suffer and neurons begin to die and degenerate—at this stage, patients begin to suffer the memory loss and behavioral changes that are characteristic of Alzheimer’s.”

Thanks to the ADDF, Dr. Longo is moving closer to achieving his goal of curing Alzheimer’s disease. As a child, frustrated that doctors were unable to help his younger sister with cerebral palsy, he dreamed of becoming a physician who could fix diseases of the brain. He made it to medical school and ultimately found his niche in Alzheimer’s research and patient care. “I was always attracted to diseases that were not curable,” Dr. Longo says. “And Alzheimer’s is an area that’s ripe for significant advances. There are so many exciting opportunities for developing therapeutics.”

When Dr. Longo isn’t in the lab, he’s with the patients who inspire him to continue his work. “I feel such frustration at not being able to provide better treatments than what existed 15 years ago,” he says. “Seeing patients provides so much motivation to work hard on the research front.”

When you support the ADDF, 100% of your contribution funds drug discovery research like Dr. Longo's.