The three-year funding agreement backs the international Linked Clinical Trials (LCT) initiative, established by the two organizations in 2012 to identify treatments that might slow, stop, and reverse Parkinson’s disease (PD) largely by repurposing therapies approved to treat other conditions.
Each year, the LCT’s scientific committee identifies the most promising medicines to advance into clinical trials assessing their efficacy in PD. Choosing therapies that have already undergone safety and toxicology tests can potentially slash the time and funding it normally takes to test a new treatment and, if successful, get it to patients.
Right now, 15 clinical trials of committee-selected treatments are underway, and seven investigations have concluded, the release states. Ten additional studies are being planned. More than 2,500 people have taken part in trials to date.
“We are thrilled to continue our long-standing collaboration with the Cure Parkinson’s Trust on the international Linked Clinical Trials initiative and look forward to expanding our program to evaluate additional promising medications in the coming years,” said Patrik Brundin, chair of the program committee and director of the VAI Center for Neurodegenerative Science, in a press release.
“We are especially grateful to the trial participants, without whom this critical work would not be possible. I am immensely hopeful that, together, we will find a way to slow or stop Parkinson’s progression,” Brundin added.
Will Cook, CEO for Cure Parkinson’s, a U.K. group, lauded the funding agreement that builds upon the organization’s relationship with Michigan-based VAI.
“This will enable the launch of many more clinical trials of potentially disease-modifying, repurposed and novel drugs that have been identified through the diligent iLCT process, and thereby bringing us closer to our goal: a cure for the 10 million people living with Parkinson’s globally,” he said.
The LCT initiative has made progress. Most recently, results of a small single-center and open-label Phase 2 clinical trial (NCT02941822) for ambroxol (brand names are Mucosolvan, Mucobrox, and Mucol, among others) — long used in some countries to treat respiratory conditions — suggest the therapy is safe and well-tolerated in Parkinson’s patients.
The study also showed the compound’s ability to cross the blood brain barrier and raise levels of the protein glucocerebrosidase (GCase) in brain cells of those with Parkinson’s. Such activity could keep cells healthier longer to slow PD progression. GCase allows cells to more effectively remove waste, an ability thought to be deficient in some patients.
Mutations in the GBA gene — which encodes for the GCase protein — are one of the most common genetic risk factors for Parkinson’s
Results of the study, led by the University College London, were published last month in the Journal of the American Medical Association. To help ambroxol reach Phase 3 — needed to determine an optimal dose — the VAI and CPT, along with the John Black Charitable Foundation, will contribute a total of $679,000.
“The massive clinical undertaking involving so many drugs repurposed from other therapeutic areas is unique not only in neurology, but is larger than any drug repositioning program, whether academic or commercial, in any other disease,” said Richard Wyse, MD, CPT director of research and development.
“The partner between VAI and CPT bears testament to the principle of collaboration, and we look forward to continuing and growing this partnership in the years ahead.”
The Cure Parkinson’s Trust funds pioneering research globally aimed at finding treatments that can slow, stop, or reverse PD. The Van Andel Institute seeks to improve health and lives through education and cutting-edge biomedical research.