Mayo Clinic Florida has inked a sponsored research agreement with SK Biopharmaceuticals of Seoul, South Korea. The goal of this collaboration is to develop new treatments for Lou Gehrig’s disease, amyotrophic lateral sclerosis (ALS). The collaboration marks a new approach to combating a disease that has proved difficult to treat in recent clinical trials.

“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.

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.

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