$1.9M Grant Aims to Revolutionize Parkinson’s Therapy Development

A £1.375 ($1.9) million grant from the Edmond J. Safra Foundation will support a pioneering clinical trial aimed at speeding the development of new therapies for Parkinson’s disease.

“This project will revolutionise the way we perform clinical trials of potentially disease-modifying drugs for people with Parkinson’s,” Thomas Foltynie, PhD, MD, a professor of neurology at University College London (UCL), in the U.K., said in a university press release.

The Accelerating Clinical Treatments for Parkinson’s Disease project — to be known as the Edmond J. Safra ACT-PD Initiative — will allow researchers to design an innovative platform in clinical development called a multi-arm multi-stage or MAMS clinical trial.

This type of trial design allows multiple therapies to be tested simultaneously, along with a continuous transition from early to late-stage clinical testing. Prospective therapies that fail at early stages are rapidly set aside if they do not show effectiveness.

This means that in a period of five years, 12 potential therapies can get tested, according to the scientists, who noted that it would take 40 years to accomplish the same thing using a conventional clinical trial design.

“Our existing process of ‘one drug at a time’ is far too inefficient, and it is high time that we had a platform capable of assessing multiple approaches simultaneously,” Foltynie said.

The Safra ACT-PD Initiative, which will run until the end of 2023, will be co-led by Foltynie and Sonia Gandhi, PhD, of the UCL Movement Disorders Centre, together with Camille Carroll, PhD, a neurologist and associate professor at the University of Plymouth.

The trial will run in partnership with the Medical Research Council Clinical Trials Unit at UCL and involve leading Parkinson’s researchers in the U.K. Individuals with Parkinson’s disease and their caretakers also will join in the project, along with major U.K. Parkinson’s charities.

This type of MAMS approach was used to evaluate treatments for prostate cancer in 2005 and has also been used to identify treatments for progressive multiple sclerosis. According to the scientists, such research moved along at a much faster rate — up to three times more quickly — than would normally take place.

“The funding provided by the Edmond J. Safra Foundation will enable a sea change in how we investigate therapies that could slow or stop Parkinson’s progression,” said Carroll. “We are very pleased to be partnering with colleagues at UCL to realise the vision of developing this trial platform for Parkinson’s.”

The project also will be supported by the National Institute of Health Research, which has expertise in developing trials and providing delivery infrastructure across healthcare in the U.K.

“Together with our partners at the University of Plymouth and a community of Parkinson’s expert clinicians and academics throughout the U.K., this project can deliver benefits for Parkinson’s patients worldwide,” added Alan Thompson, dean of the faculty of brain sciences at UCL.

Edmond J. Safra, a banker and philanthropist, created the foundation in his name to support four program areas: education, science and medicine, religion, and humanitarian assistance. Run since his death by his wife, Lily, the foundation has made significant contributions to Parkinson’s disease research and patient care worldwide.

A previous grant helped establish a neurosurgery chair at UCL, and a clinical research team at the movement disorders center.

“I have witnessed first-hand the effects that Parkinson’s disease has on patients and their loved ones, and I feel great hope and excitement at the prospect of the Edmond J. Safra ACT-PD Initiative identifying the treatments which hold the promise of a brighter future,” said Lily Safra, the foundation’s chair.

Thompson said this grant will be “critical … in enabling us to take the next step and build upon more than three decades spent at the frontiers of brain science and neurodegeneration.”