MJFF Awards $2 Million to Advance Nine Potential Drug Targets for PD
“Through Target Validation, the Foundation strategically allocates funding to demonstrate the relevance of putative drug targets early in the PD therapeutics discovery process,” said
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
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
The following is a complete list of researchers who were awarded grants under Target Validation 2007. For grant abstracts and researcher bios, please visit www.michaeljfox.org.
“Functional Inhibition of Ras GRF1 in the MPTP-lesioned
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,
“Inhibition of c-Abl Tyrosine Kinase as a Novel Therapy to Prevent PD progression”
Syed Imam, MS, PhD, University of
“Validation of Sirtuin 2 Deacetylase as a Therapeutic Target in Parkinson’s Disease”
Aleksey Kazantsev, PhD,
“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,
“PGC-1 Alpha as a Neuroprotective Target in Parkinson’s Disease”
David Simon, MD, PhD,
David Standaert, MD, PhD,
“Validation of Cathepsin D as a Target for Parkinson’s Disease Therapy”
Jianhua Zhang, PhD,