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Mayo Clinic, Korean Biotech to Collaborate on ALS Research

Illustration of brain (NIDA)

(National Institute of Drug Abuse)

Mayo Clinic’s campus in Jacksonville, Florida and SK Biopharmaceuticals in Seoul, South Korea have agreed to develop new treatments for amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig’s disease. The collaboration will center around the work of Mayo Clinic’s Leonard Petrucelli, who heads the neuroscience research department at the Jacksonville campus.

ALS 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 three to five years of onset. More than 30,000 Americans live with ALS and every year nearly 6,000 people in the U.S. are newly diagnosed with the disease. Worldwide, the disease affects approximately three out of every 100,000 people.

The new agreement is motivated by recent advances in ALS research, which show that the TDP-43 protein plays a prominent role. The protein has been found to go awry in approximately 90 percent of all ALS patients. Petrucelli and his lab colleagues have developed cell and animal models that provide insights into neurotoxic processes that begin the abnormal aggregation of proteins such as TDP-43.

The collaboration is expected to combine Petrucelli’s work with these preclinical models that mimic human disease characteristics with SK Biopharmaceuticals’ experience in medicinal chemistry in generating potential compounds or treatments. SK Biopharmaceuticals has several drugs in its pipeline to treat neurological conditions, including Parkinson’s disease, Alzheimer’s disease, and Epilepsy.

The Mayo Clinic says its ALS Center in Florida is also in a position to conduct clinical trials if the drug discovery effort identifies a lead chemical compound during the preclinical research.

Read more: Trial Shows Drug Effect on ALS Blood Biomarker

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