By Steve Zisson
The Michael J. Fox Foundation (MJFF) for Parkinson's Research gets it. They have figured out just how many bottlenecks there are for getting good research out of academia and to the patient. And Michael J. Fox is willing to put its money where it is needed to move along the drug development process.
It is no small task, but MJFF is headed in the right direction. The foundation has awarded $95 million for Parkinson’s disease (PD) research since its founding in 2000. Last week, MJFF kicked off its 2008 Target Validation effort. Under this annual program, the foundation will award up to $2 million for projects designed to “validate the therapeutic potential of scientific discoveries, pushing them one step closer to possible pharmaceutical industry investment and clinical trials.”
Maybe the most interesting step the foundation took this year is to enlist contract research organizations (CROs) in its efforts for the first time. The foundation and many observers understand that discoveries will be a long time in coming to clinical trials if everything is left up to academics.
MJFF has identified several CROs to help applicants gather critical information essential for ensuring studies are appropriately designed to validate or invalidate targets, such as dosing amounts and brain bioavailability.
Grant submissions must include the necessary pharmacokinetic and toxicology studies required to fully validate their targets, and the Request for Applications (RFA) includes a list of CROs equipped to perform these studies.
For investigators who are not able to carry out the required test in their own labs, MJFF has identified several CROs to help applicants gather this critical information. The expertise and resources provided by the CROs will allow awardees to obtain information critical to fully validating the proposed targets.
"Target Validation is emblematic of our Foundation's aggressive, 'get-it done' approach to driving PD drug development and speeding delivery of treatments that can improve patients' quality of life," said Katie Hood, interim chief executive officer of MJFF.
"Target Validation has a single purpose: to definitively assess the importance of promising targets by proving or disproving their druggability. This is a vital component of the Foundation's strategy to accelerate the pace of drug development by reducing industry's risk around investing in potential PD treatments."
Target Validation 2008 specifically seeks investigator-initiated applications to demonstrate that modulation of a specific cellular target (such as a gene or protein) results in a PD-relevant therapeutic response in an appropriate whole-animal mammalian model of PD. Of particular interest are targets whose manipulation holds the potential to treat the symptoms of PD, including both 'motor' and 'non-motor' symptoms such as cognitive dysfunction, autonomic dysfunction, sleep disorders and depression.
Also of interest are targets that could be used to protect or restore degenerating neurons, as well as those that can treat complications of current PD treatments (e.g., dyskinesias).
Target Validation pre-proposals are due by Dec. 18, 2007 and funding is anticipated by spring 2008.
A conference call with MJFF Research Programs staff to further clarify the aims and goals of this initiative will be held Nov. 27 at noon U.S. Eastern Time. Researchers wishing to participate in the call must RSVP to conferencecalls@michaeljfox.org and will receive an e-mail reply with call-in details.
Steve Zisson, managing editor of Thomson CenterWatch.
