NEWYORK, NY— As part of its ongoing efforts to do whatever it takes to speed delivery of transformative treatments and a cure for Parkinson’s disease, The Michael J. Fox Foundation for Parkinson’s Research has awarded up to $3 million in total funding to four industry teams seeking to push potential new PD treatments closer to the clinic. The awards were granted under MJFF’s Therapeutics Development Initiative (TDI) program. Open exclusively to industry researchers, TDI is the cornerstone of the Foundation’s efforts to expand industry investment in PD drug development. Through TDI, the Foundation shares the risk of drug development, thus helping to speed companies’ abilities to reach critical decision points for Parkinson’s disease projects. Each of the four TDI grant awardees listed below will undertake research aimed at solving critical gaps in the development of new PD treatments.
“While industry plays a vital role in shepherding new therapeutics through the development process toward clinical trials and patients, competitive pressures and tough allocation decisions too often get in the way of making the kinds of ‘big bets’ necessary for breakthrough developments,” said Katie Hood, CEO of The Michael J. Fox Foundation. “By funding industry partners directly, TDI seeks to advance promising treatments that might otherwise get stuck at the pre-clinical stage. Our capital may be comparatively modest, but it can serve as a ‘carrot’ to leverage companies’ expertise and infrastructure and speed the development of therapeutics that could have an immense impact on patients’ quality of life.”
While the Foundation has funded industry researchers since its inception, the Therapeutics Development Initiative was launched in 2006 as part of a larger initiative to capture the attention and imagination of company decision-makers and encourage them to allocate resources to Parkinson’s projects. To date, of the approximately $21 million the Foundation has committed in total funding to industry, almost a third (nearly $8 million) has gone to 14 projects under TDI. The current round of awardees will focus on developing and optimizing new treatments targeting alpha-synuclein toxicity; chronic inflammation; trophic factors; and mitochondrial dysfunction.
- Christine Bulawa, PhD, of FoldRx Pharmaceuticals Inc., will work to develop a disease-modifying drug that could block the toxicity associated with clumping of the protein alpha-synuclein, a hallmark of PD pathology. Dr. Bulawa’s team has identified chemical compounds that protect neurons from alpha-synuclein toxicity and will now work with the compounds in a rodent model of Parkinson’s. The researchers hope to identify promising small molecules that, with further optimization, can be developed into drug candidates to be tested in PD patients in clinical trials.
- Chronic inflammation plays a role in the death of the dopamine-producing neurons that are lost in Parkinson’s disease. Patrick Flood, PhD, of TheraLogics, Inc., and his group will test compounds that specifically target the inflammatory pathway in a PD animal model to determine whether certain drugs can protect against this neuronal loss. The team will assess whether blocking inflammation reverses destruction of dopamine-producing neurons, and actually leads to regeneration of these cells within the brain, to determine the most effective dose and timing for therapeutic intervention.
- The blood-brain barrier is a thin layer of tightly packed cells separating the central nervous system from the body’s bloodstream. This layer is crucial to protecting the brain from foreign substances, but also poses a major challenge in delivering potentially therapeutic treatments via orally administered drugs. Antonia Orsi, PhD, and her team from Phytopharm have developed a small orally active molecule that crosses the blood-brain barrier. In vivo and in vitro, the molecule increases levels of trophic factors, specialized proteins that potently promote survival of neurons. The team has already demonstrated that the compound can increase the number of dopaminergic neurons in a mouse model of PD. The goal now is to gain greater understanding of the neurorestorative properties of this compound in mice and, if successful, test the compound in a primate model of Parkinson’s disease.
- Mitochondria are the ‘energy factories’ of body cells. It is believed that mitochondrial function is decreased in people with Parkinson’s disease and that mitochondrial toxins induce parkinsonian symptoms in animal models. Rebecca Pruss, PhD, and her colleagues at Trophos are developing unique compounds that improve mitochondrial function and that are currently being evaluated in patients for the treatment of ALS and diabetic neuropathy. Dr. Pruss will test whether these compounds are neuroprotective in an animal model of Parkinson’s disease.
Grant abstracts and researcher bios for all projects are available on the Foundation’s Web site, www.michaeljfox.org.