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MJFF Awards $2 Million to Advance Nine Potential Drug Targets for PD

NEWYORK, NY — The Michael J. Fox Foundation for Parkinson’s Research has awarded $2 million total to nine research teams working to validate therapeutic targets for Parkinson’s disease. The awards were made under MJFF’s annual Target Validation initiative, which is designed to accelerate the rate at which new Parkinson’s therapies are brought to market.

“Through Target Validation, the Foundation strategically allocates funding to demonstrate the relevance of putative drug targets early in the PD therapeutics discovery process,” said Sarah Orsay, chief executive officer of The Michael J. Fox Foundation. “If MJFF capital can help elevate a drug target from ‘possibly interesting’ to ‘clearly important,’ it reduces the investment risk for biotech and pharmaceutical firms to take it into later phases of drug development — and that much closer to patients.”

Target validation is an essential phase of drug development in which researchers conduct highly specific studies to determine whether a molecule of interest is a true drug target —  that is, whether it is critically involved in a disease and whether manipulating it impacts disease symptoms and progression. While researchers have continued to identify novel targets in recent years through genetic, biochemical,  and epidemiological studies, a dearth of funding for validation studies has historically been one roadblock to the efficient translation of these targets into patient-relevant therapies.

Overall, funded projects under Target Validation 2007 fall into two broad categories: targets for therapies to alleviate dyskinesias, the excessive, uncontrollable movements brought on by long-term dopamine replacement therapy; and new targets that have shown potential to slow or stop progression of Parkinson’s rather than merely mask its symptoms.

Angela Cenci-Nilsson, PhD, of LundUniversity in Sweden will carry forward work originally funded under Target Validation 2005. At that time, working in rodent models of Parkinson’s, she determined that blocking the action of a molecule called the metabotropic type 5 glutamate receptor (mGluR5) could prevent the development of levodopa-induced dyskinesias. The current award will allow her to take this work to the next level, working in non-human primates to correlate molecular events with behavioral outcomes.

Other awardees will further the investigation of novel targets identified only recently. For example, Aleksey Kazantsev, PhD, of Harvard Medical School will continue work on the enzyme SIRT2, whose potential neuroprotective effects his team reported in the journal Science just last month. Working in cellular and animal models, Dr. Kazantsev demonstrated that blocking SIRT2 protected neurons from alpha-synuclein toxicity. He will now develop and test tools to selectively silence SIRT2 in cellular models. And Michael Schwarzchild, PhD, also of HarvardMedicalSchool, will follow up on epidemiological studies indicating that high blood levels of urate, an antioxidant found in blood, slow the progression of PD. The team will work to determine whether increasing urate levels protects against PD onset in rodent models, and whether deletion of the gene responsible for urate degradation prevents degeneration — or overexpression increases susceptibility.

The following is a complete list of researchers who were awarded grants under Target Validation 2007. For grant abstracts and researcher bios, please visit

“Functional Inhibition of Ras GRF1 in the MPTP-lesioned NHP Model for Treating Levodopa-induced Dyskinesia”
Erwan Bezard, PhD, INSERM, France

“Validation of Metabotropic Glutamate-receptor Type 5 as a Target for the Treatment of L-DOPA-induced Dyskinesia in a Macaque Model of Parkinson’s Disease”
Angela Cenci-Nilsson, PhD, Lund University, Sweden

“Inhibition of c-Abl Tyrosine Kinase as a Novel Therapy to Prevent PD progression”
Syed Imam, MS, PhD, University of TexasHealthScienceCenter

“Validation of Sirtuin 2 Deacetylase as a Therapeutic Target in Parkinson’s Disease”
Aleksey Kazantsev, PhD, HarvardMedicalSchool

“Validation of Targets of Small Molecules that Ameliorate Alpha-synuclein Neurotoxicity”
Susan Lindquist, PhD, Whitehead Institute for Biomedical Research

“Targeting Urate: A Molecular Correlate of Both Risk and Progression in PD”
Michael Schwarzschild, MD, PhD, HarvardMedicalSchool

“PGC-1 Alpha as a Neuroprotective Target in Parkinson’s Disease”
David Simon, MD, PhD, BethIsraelDeaconessMedicalCenter

“Validation of VPS41, a Protein Involved in Lysosomal Trafficking, as a Target for Parkinson’s Disease Therapy”
David Standaert, MD, PhD, University of Alabama, Birmingham

“Validation of Cathepsin D as a Target for Parkinson’s Disease Therapy”
Jianhua Zhang, PhD, University of Alabama, Birmingham

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