Mayo Clinic Enters Collaboration That Takes Aim at Lou Gehrig’s Disease

Mayo Clinic’s campus in Florida has entered into a collaborative and sponsored research agreement with SK Biopharmaceuticals of Seoul, South Korea, with the goal of developing new treatments for amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. The collaboration marks a new approach to combating a disease that has proved difficult to treat in recent clinical trials. 

More than 30,000 Americans live with ALS, a disease that causes the nerve cells in the brain and spinal cord to die. As a result, a patient loses voluntary muscle movement. Worldwide, the disease affects approximately three out of every 100,000 people.

“This is the future,” says Leonard Petrucelli, Ph.D., a neuroscientist and chair of the Department of Neuroscience Research at Mayo Clinic in Florida, who is leading Mayo’s effort. “This is about academia and industry forming strong relationships to capitalize on the core strengths of each organization.”

The new agreement is spurred by recent advances in ALS research, which show that the TDP-43 protein plays a prominent role. Dr. Petrucelli’s new aggressive measures aim to target this protein. The implications are far-reaching because the protein has been found to go awry in approximately 90 percent of all ALS patients.

“We still haven’t found a cure, but at least now we’re focusing on a target that would benefit the vast majority of ALS patients,” Dr. Petrucelli says.

Every year, nearly 6,000 people in the U.S. are newly diagnosed with ALS. The disease results in gradual loss of nerve cells in the brain and spinal cord that control essential muscle activity such as speaking, walking, breathing and swallowing, and is typically fatal within 3-5 years of onset.

Mayo Clinic’s drug discovery efforts and experience generating preclinical animal models that mimic human disease characteristics will meld with SK Biopharmaceuticals’ experience in medicinal chemistry, or how to make potential compounds or treatments better and more effective.

Dr. Petrucelli’s laboratory has pioneered research in the neurosciences field to understand the underlying mechanisms of ALS and identify potential drug targets for its treatment. In addition, the ALS Center at Mayo Clinic in Florida, which is led by Kevin Boylan, M.D., is well positioned to conduct clinical trials if the drug discovery effort identifies a lead chemical compound during the first phase of the preclinical research collaboration. The ALS clinic at Mayo Clinic in Florida is the only ALS Association “Center of Excellence” in Florida and one of only four in the Southeast.