Potential Parkinson's-fighting compounds identified

Patient with Parkinson's disease--Courtesy of Obeso JA et al. Nat. Med. 2010

Scientists at the U.K.'s Sheffield Institute for Translational Neuroscience (SITraN) have identified new potential disease-modifying compounds that could stop brain cell death and slow the progression of Parkinson's.

"The best treatments currently available only improve some of the symptoms, rather than tackle the reason why Parkinson's develops in the first place, so there is a desperate need for new drug treatments which could actually slow down the disease progression," Dr. Oliver Bandmann, reader in Neurology at SITraN, said in a news release.

Using skin cells from people with the disease, investigators conducted a large-scale drugs trial in the lab, testing over 2,000 compounds--including 1,040 licensed drugs and 580 naturally occurring compounds--to find out which ones could make faulty mitochondria work normally again. Researchers focused on mitochondria--the powerhouse of cells--because malfunctioning mitochondria are a major cause of brain cell death in Parkinson's. The research is published in the journal BRAIN.

The extensive drug screen, which was funded by the nonprofit Parkinson's UK and carried out in collaboration with University of Trondheim, Norway, took more than 5 years to complete. Scientists initially identified 60 compounds for their Parkinson's-fighting potential, and after toxicity tests and further studies, whittled that pool of candidates down to two compounds--ursocholanic acid and the related compound dehydro(11,12)ursolic acid lactone. A licensed drug--ursodeoxycholic acid, which is close to the naturally occurring compounds ursolic acid--has been in clinical use for several decades to treat certain forms of liver disease.

The scientists hope to begin clinical trials using the drug in Parkinson's patients soon.

- here's the study in BRAIN
- read the press release

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