The cover of the February 22-29, 2016, edition of Time magazine announces: “The Alzheimer’s Pill: A radical new drug could change old age.” The article delves into the groundbreaking drug research being conducted by Dr. Frank Longo, a longtime Alzheimer’s Drug Discovery Foundation (ADDF) grantee and recipient of our inaugural Melvin R. Goodes Prize in 2015.
Dr. Longo is a professor and chairman of the Department of Neurology and Neurological Sciences at Stanford University School of Medicine and founder and chairman of biopharmaceutical firm PharmatrophiX. His drug, LM11A-31, targets the p75 receptor on the surface of neurons. Dr. Longo explains that in targeting this receptor, the drug “counteracts much of the abnormal signaling that occurs in Alzheimer’s disease.” Such abnormal signals lead neurons to degenerate and die, which in turns leads to the memory loss and other cognitive symptoms of Alzheimer’s dementia. LM11A-31 is particularly promising for treating Alzheimer’s because it targets more than one disease mechanism and could be the first drug approved for Alzheimer’s to work in this way.
The ADDF first invested in Dr. Longo’s pioneering research in 2000, when it was in its very early stages. He needed funding to screen 10s of thousands of compounds that might affect the p75 receptor in the way he predicted. Since then, the ADDF has supported the development of LM11A-31 every step of the way, with seven grants totaling over $2 million. The most recent grant—$500,000 awarded in late 2015—is supporting the phase 2a clinical trial of LM11A-31 to test its effectiveness in patients with Alzheimer’s. Upon winning the Goodes Prize last year, Dr. Longo said: “Our approach was a highly risky one, with no good precedent in the field of neuroscience. Our success in obtaining NIH funding for this work required that we first demonstrate success in early stages. ADDF support has been critical to our progress, both at the beginning and at several key points.”
In 2000, we also began funding a project that developed into Amyvid™, the first FDA-approved test for Alzheimer’s. The Time article notes that this PET scan, which was approved in 2012, is critical to researchers like Dr. Longo because it allows them, for this first time, to conclusively diagnose Alzheimer’s in living patients and track its progression.
ADDF Executive Director Dr. Howard Fillit says: "Dr. Longo's research demonstrates our committment to finding and funding the most promising ideas to treat Alzheimer’s disease. Today, we’re seeing the dramatic results of some of our earliest investments and are hopeful that new, effective drugs to treat Alzheimer’s will be in the hands of patients in just a few years."