NEW YORK, NY — The Michael J. Fox Foundation for Parkinson’s Research (MJFF) announced $1.2 million in total awards to four research teams working to advance potentially disease-modifying therapeutic targets for PD along the drug development pipeline. The funding was awarded under the Novel Approaches to Drug Discovery for Parkinson’s Disease program, made possible by generous leadership funding from Elan Corporation, plc (NYSE: ELN), a neuroscience-based biotechnology company.
"For successful translation into real-world PD therapies, early stage therapeutic approaches must be chaperoned through the necessary preclinical and clinical stages of drug development," said Sarah Orsay, MJFF's CEO. "The Foundation uses creative strategies to provide the opportunity for researchers working on novel therapeutic strategies to partner with first class, large industry organizations. If projects funded under Novel Approaches to Drug Discovery for Parkinson's Disease warrant further development, Elan has the option to participate more actively and further the progress made by the awardees - taking us that much closer to our shared goal of delivering new treatments to patients."
Novel Approaches complements the Foundation’s annual Target Validation initiative, whose awardees were announced last month. Whereas the latter program supports initial work to determine the validity of cellular proteins and pathways as potentially promising drug targets, Novel Approaches seeks to push work forward in developing therapies against targets that already have some promising initial data. Both programs provide critical resources for underfunded stages of the drug development process and reflect the
Foundation’s emphasis on bridging early discovery work and late-stage translational research to reduce industry’s risk around investment in new PD therapeutics.
The Novel Approaches program is also an important element of the Foundation’s increasing engagement with pharmaceutical and biotech company partners. This engagement is multi-pronged, including not only funding industry researchers under any Foundation initiative, but also working with companies as strategic partners to accelerate the rate at which new PD therapeutics are brought to market. Awardees under Novel Approaches include both academic and industry scientists.
“We are very pleased to work side by side with The Michael J. Fox Foundation in the pursuit of effective treatment options for patients suffering from Parkinson’s disease,” said Dale Schenk, PhD, Elan’s Chief Scientific Officer. “We congratulate the awardees and are impressed that the Novel Approaches program has attracted such high-quality proposals for potential treatment of Parkinson’s disease. We look forward to working together to move us closer to a therapy that can slow or stop progression of this terrible disease.”
Awardee Asa Abeliovich, PhD, of ColumbiaUniversity is targeting the autophagy pathway, a cellular pathway involved in clearing away protein such as alpha-synuclein, whose clumping is a hallmark of PD pathology. Disruption of this pathway in animals leads to loss of dopamine neurons. Dr. Abeliovich will test small-molecule drugs that can enhance the autophagy pathway to see if they can reduce deficits seen in animal models.
Neil Howell, PhD, of San Diego-based MIGENIX Corporation will work in rodent models to optimize administration of the compound MX-4565, a non-feminizing estrogen analog that has been shown to be effective in protecting nerve cells from toxic stresses.
Peter Jenner, PhD, of Proximagen Neuroscience in the United Kingdom will investigate the potential of the protein osteopontin (OPN) as a PD treatment. Expression of OPN is decreased in PD, and Dr. Jenner’s team has previously shown that it is neuroprotective for dopaminergic neurons. The researchers will now investigate a gene therapy approach to deliver the protein to the brains of rodent models of Parkinson’s using viral vectors.
Pamela Maher, PhD, of the Salk Institute in La Jolla has identified a neuroprotective small molecule called fisetin. At least part of fisetin’s neuroprotective effect is due to its ability to maintain levels of glutathione (GSH), a molecule cells produce naturally as a defense against oxidative stress. There is good evidence that GSH levels decrease in PD, and this decrease may contribute to disease progression. Dr. Maher’s team will use medicinal chemistry to create fisetin derivatives with improved potency and efficacy, and then will test these derivatives in cell-based and rodent models of PD.
Grant abstracts and researcher bios are available on the Foundation’s Web site, www.michaeljfox.org. The selection of awardees was made exclusively by the Foundation via its standard peer-review process.