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De-Risking Strategy Leads to Significant Investment in Parkinson’s Drug Development


Proof positive of The Michael J. Fox Foundation (MJFF) strategy of de-risking early research for larger investment from venture capitalists and industry partners, two projects jumpstarted by MJFF funding have secured financial commitments to continue their work.

“This is why our Foundation exists,” said Todd Sherer, PhD, MJFF CEO. “We’re here to take the risks, fund new ideas and help build the data to encourage vital partnerships down the road. Investments from venture capital firms and large companies can help translate these initial findings into medications in the hands of patients.”

Biotech Companies Partner Toward PD Cure

Amicus Therapeutics has announced a partnership with Biogen Idec to collaborate on discovery of a new class of small molecules for the treatment of Parkinson’s disease (PD).

MJFF has provided Amicus nearly $700,000 since 2006, funding the biotech to investigate the effect of increasing activity of the lysosomal enzyme glucocerobrosidase (GCase) in the brain. Loss of GCase enzyme activity, from mutations in the GBA1 gene, has been linked to alpha-synuclein accumulation, the hallmark pathology of Parkinson’s. With MJFF funding, Amicus scientists have studied two pharmaceutical chaperones – small molecules that can be administered orally – to increase GCase activity and thereby improve the cell’s ability to keep the accumulation of alpha-synuclein in check. Further development of drugs that target GCase is an important step toward a cure for PD.

Biogen Idec will now work with Amicus to expand their work on small molecules that target the GCase enzyme with the goal of bringing a new drug to market.

“Our preclinical studies funded by The Michael J. Fox Foundation helped to lay the groundwork for our partnership with Biogen Idec,” said Sean W. Clark, PhD of Amicus Therapeutics, Inc. “We greatly appreciate the MJFF’s ongoing support of our efforts to develop new treatments for Parkinson’s disease.” Read more on the Foxfeed Blog.

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