NEW YORK, Jan. 23 /PRNewswire-USNewswire/ -- The Michael J. Fox Foundation for Parkinson’s Research today announced $4.6 million in total funding to 10 industry research teams under its Therapeutics Development Initiative. The Foundation launched the program in 2006 as a key element in its strategy to ‘de-risk’ preclinical Parkinson’s disease research for biotech and pharmaceutical companies, thus expanding and catalyzing industry investments in the development of improved PD treatments and a cure.
“No matter how much capital a pharmaceutical or biotech company may have, every investment of that capital is a bet -- and, by definition, may not pay off,” said Deborah W. Brooks, president and CEO of The Michael J. Fox Foundation. “Our goal with the Therapeutics Development Initiative is to add our resources to companies’ own, making Parkinson’s a more attractive bet and helping to push PD research further out the drug development pipeline toward the clinic and patients.”
While the Foundation encourages the submission of applications from industry teams under all its initiatives, and has previously awarded significant funding to industry researchers and academic-industry collaborations, the Therapeutics Development Initiative is its first program open exclusively to industry researchers.
“Developing disease-modifying therapies for Parkinson’s has been very much one of our long-term goals within the Wyeth Neuroscience group,” said awardee Peter Reinhart, PhD, of Wyeth Research. “The MJFF funding will accelerate our internal timelines as well as our ability to reach critical decision points for this Parkinson’s disease project.” Dr. Reinhart seeks to develop drugs to inhibit the PD-implicated gene LRRK2. His team will also investigate other proteins and cellular signaling pathways on which LRRK2 may act, as these may prove to be additional worthwhile targets for new drugs.
Neuroprotective Approaches
Like Dr. Reinhart, the majority of awardees will work toward the development of neuroprotective treatments that would rescue dopamine neurons from dying, thus slowing or stopping the progression of Parkinson’s disease. Currently available therapies only mask symptoms while the underlying disease continues to progress.
Brandon Wustman, PhD, and Sean Clark, PhD, of Amicus Therapeutics will attempt to develop drugs that take advantage of a possible genetic link between Parkinson’s and Gaucher’s disease. Gaucher’s disease is an inherited deficiency of the enzyme glucocerebrosidase, which results in the buildup of a toxic substance in parts of the body including the spleen, liver and bones; carriers of the mutation for this disorder have a significantly elevated risk of developing PD. Drs. Wustman and Clark will test a compound known to stabilize glucocerebrosidase for its ability to block Parkinson’s and Gaucher’s pathology. They will also look for interactions between glucocerebrosidase and alpha-synuclein, a protein whose clumping is a hallmark of PD pathology.
One theory holds that the cell death seen in Parkinson’s disease results from destabilization of the internal “skeleton” of neurons. Awardee Bruce Moriomoto, PhD, of Allon Therapeutics will test a therapeutic known as AL-108, which may be able to stabilize this skeleton, thus rescuing dopamine neurons from death. AL-108 is already in Phase II clinical trials for cognitive impairment, so if it is determined to hold potential as a neuroprotective agent, it could progress rapidly into clinical trials for PD.
Steve Zhang, PhD, of Sangamo BioSciences will work in rodent models to develop a novel gene therapy approach targeting the trophic factor GDNF, which is of great interest to Parkinson’s researchers because it has been shown in preclinical testing to slow or stop progression of the disease. Clinical trials of direct brain infusion of GDNF in patients have led to inconclusive results, possibly due to inadequate delivery of the factor; thus, novel attempts to target GDNF may still hold promise. Dr. Zhang and his team will seek to deliver a gene for a transcription factor, ZFP-TF, that could increase production of natural GDNF in the brain. If successful, this strategy would allow doctors to enhance GDNF expression in the brain in a potentially more controlled and targeted fashion than standard gene therapy approaches.
“MJFF funding has been key to bringing my project from the idea stage to the action stage,” said Dr. Zhang. “With the Foundation’s enthusiasm and financial support, Sangamo will aggressively pursue this important project.”
Cell Replacement Approaches
Two separate awardees will focus on different means to improve and optimize cell replacement strategies for Parkinson’s disease. Ofer Wiser, PhD, of CellCure NeuroSciences Ltd. will systematically examine the developmental pathway that an embryonic stem cell takes to become a dopamine neuron, in order to determine the stage that will give the best post-transplant survival. The team will also seek cellular markers of these developmental stages that could be used to enrich cell grafts with the cells that will give the greatest results.
Erik Miljan, PhD, of ReNeuron Group PLC will attempt to optimize survival of stem cell-derived dopamine neuron transplants by testing several varieties of biodegradable “scaffolds” in which cells can be placed before transplantation. These three-dimensional structures could potentially increase survival of transplanted cells, as well as these cells’ conversion into dopamine neurons.
Funding for the Therapeutics Development Initiative was made possible by a leadership gift from Anne and Burt Kaplan and their family, longtime friends of The Michael J. Fox Foundation. Additional generous support for the program was provided by the Foundation’s Board of Directors.
Researcher bios and grant abstracts are available on the Foundation’s Web site, http://www.michaeljfox.org. The following is a complete list of Therapeutics Development Initiative awardees:
* Somasunder Prasad Gabbita, PhD P2D, Inc. (Cincinnati, Ohio) Small molecule TNF-alpha inhibitors as neuroprotectant drugs for PD * Erik Miljan, PhD ReNeuron Group PLC (Guildford, Surrey, United Kingdom) Development of a Biodegradable Delivery Matrix Containing Dopaminergic Neurons Derived from Clinical Grade Mesencephalic Stem Cell Lines for the Treatment of Parkinson’s Disease * Bruce Moriomoto, PhD Allon Therapeutics (Vancouver, British Columbia) Evaluation of the Neuroprotective Peptide NAPVSIPQ in Models of Parkinson’s Disease * Peter Reinhart, PhD Wyeth Research (Princeton, New Jersey) LRRK2: Accelerated Pre-Clinical Development of a Novel Disease-modifying Therapeutic for Parkinson’s Disease * Stacy Markison Roth, PhD, and Sandra Lechner, PhD Neurocrine Biosciences (San Diego, California) Neuroprotective Effects of A2A Antagonists in Rodent Models of Motor Function and Dyskinesia * Gretchen Snyder, PhD Intra-Cellular Therapies, Inc. (New York, New York) Preclinical Evaluation of a 5-HT2A Antagonist for Treatment of L-DOPA- Induced Dyskinesias in Parkinson’s Disease * Ofer Wiser, PhD, and Benjamin Reubinoff, MD PhD CellCure Neurosciences Ltd. (Jerusalem, Israel) Dopaminergic Neurons Derived from Human Embryonic Stem Cells for Transplantation Therapy of Parkinson’s Disease * Brandon Wustman, PhD, and Sean Clark, PhD Amicus Therapeutics (Cranbury, New Jersey) Treatment of Parkinson’s Disease with AT2101, a Compound that Increases the Activity of Endogenous Glucocerebrosidase * Eti Yoles, PhD Proneuron Biotechnologies (Ness-Ziona, Israel) PN277: A Neurorestorative, Protective Product for Parkinson’s Disease * Steve Zhang, PhD Sangamo BioSciences Inc. (Richmond, California) Developing Engineered Zinc Finger Protein Transcription Factors as a Potential Therapy for Parkinson’s Disease About The Michael J. Fox Foundation
Founded in 2000, The Michael J. Fox Foundation for Parkinson’s Research is dedicated to ensuring the development of a cure for Parkinson’s disease within this decade through an aggressively funded research agenda. The Foundation has funded over $90 million in research to date, either directly or through partnerships.
Michael J. Fox Foundation
CONTACT: Holly Barkhymer (media contact), +1-212-509-0995 x242, orhbarkhymer@michaeljfox.org, or Brian Fiske, PhD (research contact),+1-212-509-0995 ext. 203, bfiske@michaeljfox.org, both of the Michael J.Fox Foundation
