MSDC-0160 is an insulin sensitizer being developed by Metabolic Solutions Development Company (MSDC) of Kalamazoo, Michigan, to treat Parkinson’s disease. MSDC-0160 was originally intended to treat type 2 diabetes. However, the Van Andel Research Institute recognized its potential as a treatment for neurodegenerative disorders such as Parkinson’s.

How MSDC-0160 works

Research increasingly supports the idea that mitochondrial dysfunction might be a key feature of Parkinson’s. Mitochondria, an essential component of cells, are responsible for converting food into energy, as well as for signaling. In Parkinson’s patients, a mitochondrial sensor called mTOR is frequently over-activated in the brain. This prevents the cell from reacting appropriately to threats, such as the buildup of the harmful alpha-synuclein protein, which happens in those with Parkinson’s. The mTOR sensor is activated by a protein called mitochondrial pyruvate carrier (MPC), which carries the pyruvate molecule into the mitochondria to be converted into energy.

MSDC-0160 is part of a recently discovered class of insulin sensitizers called mTOR modulators, which are believed to reduce mTOR activity by regulating MPC.

It is anticipated that MSDC-0160 could prevent or reverse the dyskinesia, or involuntary movements, caused by levodopa, which is prescribed to many Parkinson’s patients. MSDC-0160 has the potential to become the first Parkinson’s treatment with neuroprotective properties. Researchers hope it could slow the progression of the disease rather than just mask its symptoms.

MSDC-0160 in clinical trials

MSDC-0160 has been tested in pre-clinical studies using two different mouse models of Parkinson’s. The drug showed significant neuroprotective and anti-inflammatory effects in the mice, while reducing alpha-synuclein clumping. The results appeared in Science Translational Magazine.

Researchers hope to proceed to clinical trials in people with Parkinson’s disease in 2017. MSDC-0160 has already been tested in Phase 2 clinical trials for patients with both type 2 diabetes (NCT01103414) and Alzheimer’s disease (NCT01374438). Neither trial led to severe side effects. Results of these trials were published in the scientific journals Clinical Pharmacology & Therapeutics and Current Alzheimer Research.

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