Here are selected highlights of recent MJFF investments toward better treatments and a cure for Parkinson's disease.
Our investments typically fall into one of two categories:
- Research exploring specific therapeutic approaches, where our input is likely to push prospective targets or therapies to the next stage of development.
- Research to develop tools and resources, where our input can provide clarity or information required to speed the creation of new treatments and/or advance the field.
04/30/2007 - Highlights of 2006 MJFF Investments in Specific Therapeutic Approaches
A novel strategy for alleviating levodopa-induced dyskinesias. Angela Cenci-Nilsson, MD, PhD, of Lund University in Sweden received funding under the Foundation’s Target Validation initiative to test a novel compound that may prevent and/or reduce dyskinesias — disruptive, debilitating movements that are a common side effect of long-term levodopa therapy. At the midpoint assessment of her grant, Dr. Cenci-Nilsson confirmed that the compound had prevented dyskinesia-like abnormal movements in a rodent model of Parkinson’s. If the results are validated, this work will advance to the next stages of the drug development pipeline, continuing toward clinical testing.
A partnership to keep promising drug targets moving forward. In 2006 MJFF partnered with biotech firm Elan, Inc., to launch Novel Approaches to Drug Discovery for Parkinson’s Disease, a $2-million initiative that picks up where Target Validation leaves off. Novel Approaches focuses exclusively on therapeutic targets whose potential benefit has already been demonstrated in an animal model of Parkinson’s. The program holds unique potential to rapidly advance promising leads: If therapeutic approaches identified through this program warrant further development, Elan will be poised to work with grant recipients to quickly carry the work forward, one critical step closer to the clinic and patients.
Catalyzing and expanding industry investment in pre-clinical Parkinson’s research. In 2006 MJFF awarded $4.6 million total to 10 industry research teams under the Therapeutics Development Initiative, our first industry-exclusive funding program, designed to catalyze and expand industry investment in preclinical research for Parkinson’s disease. The program adds MJFF resources to companies’ own — making industry investment in PD, specifically, more likely — and thereby helping to advance Parkinson’s research toward the clinic and patients. The majority of awardees are pursuing the “Holy Grail” of Parkinson’s treatments: a disease-modifying therapy that could slow or stop neuron degeneration rather than simply masking symptoms while the underlying disease continues to progress.
Funding for the Phase II clinical trial of a new gene therapy approach. The Foundation provided $1.9 million to support Ceregene Inc.’s Phase II study of CERE-120, a novel gene therapy product that uses a vector to deliver neurturin, a potent nervous system trophic factor. Neurturin is a member of the same family as GDNF, and like GDNF it has shown potential in preclinical studies to slow or stop Parkinson’s disease progression. The Phase II funding followed on the announcement of promising early results from Ceregene’s open-label Phase I trial of CERE-120, which also received Foundation support.
04/29/2007 - Highlights of 2006 MJFF Investments in Tools and Resources
A potential biomarker breakthrough. With over $4 million in biomarker research funded to date, the Foundation continues to marshal the search for a PD biomarker. The field recently received a considerable boost when MJFF-funded researchers found a specific expression pattern of eight genes that might someday be used to spot people at high risk of developing Parkinson’s disease. The work is an early but vital step toward a simple blood test for Parkinson’s disease. Additionally, the results could improve clinical trials by allowing researchers to better track disease progression in the presence of a given therapy.
Maximizing the potential of a unique resource. In fall 2006 the Foundation announced a $2.8-million collaboration with the Arizona Parkinson’s Disease Consortium (APDC). The Prescott Family Initiative at the Arizona Parkinson’s Disease Consortium will expand APDC’s Brain and Body Donation Program, a unique resource that conducts in-depth clinical and post-mortem studies of normal-aging adults as well as PD patients. The MJFF collaboration will maximize the program’s potential to advance PD research by tying clinical evidence to pathological underpinnings of onset and progression — information that could contribute to earlier detection and diagnosis of PD.
A first-of-its-kind PD genetics database. Given the rapid pace of the field of genetics, a centralized information source for genetic discoveries is critical to their translation into treatments. Yet until recently, the field lacked such a tool. Last fall the Foundation, together with collaborators at Harvard Medical School and the Alzheimer Research Forum, announced the launch of PDGene (www.pdgene.org), an online inventory of studies on genes implicated in Parkinson’s disease. This regularly updated Web portal provides scientists with the most current information, in one place, about every gene that previous research has shown may play a role in Parkinson’s.
