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MCL 2608.0: Compounds for the Treatment of Parkinson's Disease

Kwang-Soo Kim, Ph.D., et al.

Background and Description

Parkinson's Disease is a chronic, progressive motor system disorder affecting as many as one million Americans. Current therapy for this disease, which relies primarily on replacing brain dopamine using oral doses of the dopamine precursor L-DOPA, has had only limited effectiveness. This invention features the use of a family of chemical compounds to treat PD, and arises from the identification of a new molecular target for this disease. There have been observations that expression of the transcription factor Nurr1 is down-regulated in midbrain dopamine neurons with increasing age, and so activation of this factor may facilitate the survival of dopamine neurons and increase the production of dopamine by increasing the activity of tyrosine hydroxylase, the enzyme catalyzing the rate-limiting step for dopamine biosynthesis. Using a Nurr1-based screening assay, the inventors have identified as drug candidates for Parkinson's Disease a family of compounds that includes known chemical entities with a history of safe use in humans.

Potential Commercial Uses

This invention features a family of compounds that are strong candidates to become safe, effective new treatments for Parkinson's Disease. These are compounds in the aminoquinoline family, several of these which are currently used to treat other diseases, and therefore have known therapeutic profiles. These compounds have been shown to be effective against the Nurr1 target in vitro and to have beneficial effects in animal models. The invention also features a screening method for identification of additional PD drug candidates, based on Nurr1 gene expression.

Publication and Patent Status

Patent applications claiming this invention have been filed in the United States and several other jurisdictions including Europe and Japan.

Licenses Available

McLean Hospital is offering a worldwide exclusive license to this technology.

For more information, please contact:

Anne Ritter, Licensing Manager
Partners Research Ventures and Licensing
(617) 954-9529