Blood Builder Offers New Hope for Brain Diseases
Aug. 10, 2001 -- Patients with Alzheimer's and other brain diseases may have something in common with endurance athletes.
According to animal research described in the Aug. 9 issue of Nature, both might benefit from the genetically engineered hormone erythropoietin, known as EPO. This drug mimics a naturally-occurring hormone that normally regulates red blood cell production and is released by the brain when oxygen levels are low or during similar stress.
EPO has already gained popularity among competitive athletes, most controversially among cyclists, because it is an effective way to get more oxygen to working muscles. "Instead of training for weeks at high altitudes, cyclists use EPO to improve stamina and endurance," study author Stuart A. Lipton, MD, PhD, tells WebMD.
But along with increasing the amount of oxygen the blood can carry, Lipton says that EPO "may also protect against stroke and other forms of brain injury" by protecting nerve cells from toxic chemicals released when brain cells are damaged.
"This research paves the way for clinical trials of EPO in stroke patients and for the development of novel drugs that stimulate [similar pathways] in the brain," Mark P. Mattson, PhD, tells WebMD after reviewing the study. He is chief of neurosciences at the National Institute on Aging in Bethesda, Md.
Although the Olympic Committee bans EPO from competition, the drug is safe in humans and is already approved by the FDA to treat anemia, especially in patients with cancer or kidney failure.
"Developing drugs to treat brain disease takes 10-15 years, because 99% turn out to be unsafe," says Lipton, director of the Center for Neuroscience and Aging of the Burnham Institute in La Jolla, Calif. "We're testing drugs that we know are safe, so we're that much more ahead of the game."
While EPO easily gets into the brain, it protects against nerve cell damage without affecting normal brain function. Lipton's research in rat brain nerve cells shows how it works -- by attaching to a specific protein. This triggers a series of chemical signals that turn on several genes, which produce additional proteins that neutralize poisons causing cell death.