Easing Dyskinesia: PDF-funded Research from 2007 Leads to Testing of Experimental Drug

Earlier this week, PsychoGenics Inc., announced that an experimental drug for Parkinson’s, eltoprazine, seemed to reduce dyskinesia in people with Parkinson’s in early studies.  Dyskinesias are the twisting and writhing movements that occur as PD progresses – a common side effect of the medication levodopa (Sinemet®).   

Back in 2007, it was PDF-funded researcher Manolo Carta Ph.D., along with Anders Björklund, M.D., who performed the pre-clinical research that led to the identification of this drug.  At the time, while many researchers were looking at dopamine neurons as the culprit behind dyskinesias (through their interaction with levodopa), Dr. Carta’s proposal suggested something different – serotonin neurons.  His proposal led to a one-year research fellowship funded by PDF.

After completing his year of research with PDF funding during which he laid the foundation for this approach, he and his colleagues were able to investigate an experimental compounds that might help with dyskinesias.  With the support of other funders, they have now been able to study the effects of eltoprazine in people in this early stage trial – to see whether it is a viable drug treatment.

The study results announced earlier this week indicate positive news about eltoprazine’s potential to ease dyskinesia and possibly some non-motor symptoms of PD.

However, the drug will have to undergo rigorous testing – in additional people with Parkinson’s in phase II and phase III clinical trials – before we know if it’s safe and effective.  While we don’t yet know the fate of this drug, the results help us learn more about dyskinesias so we can find a solution in the future.

PDF believes it’s important to fund ideas like this so that scientists can have both the freedom to explore novel ideas for Parkinson’s and the time they need to gather data that can prove the promise of their ideas.  In Dr. Carta’s work, this proved to be the case. 

We believe this philosophy – funding creativity early on – will help researchers prove their case to other funders that can help them to further develop their ideas ... ultimately into new treatments and a cure for Parkinson’s. 

What are your thoughts? Do you or a loved one need a drug for dyskinesia?

Please share your thoughts below, and as always, call our HelpLine at (800) 457-6676 with any questions you or a loved one may have about Parkinson's.

NINDS and Translational Research

For years now, many of us at the Parkinson’s Disease Foundation (PDF) and other Parkinson’s disease (PD) organizations have fretted that the brain research funded by the National Institutes of Health (NIH) has been too focused upon the basic processes of neuroscience and too little upon the next stage of research: finding clues to potential new therapies for specific conditions like Parkinson’s.

This next stage of scientific investigation is often called “translational research,” because it is here that the molecules and compounds identified as interesting in the laboratory are “translated” into animal studies to determine their therapeutic potential. Those compounds that make it through this phase may then go on to be tested in human trials, and from there – the hope is – to approved treatments.

So it was with delight that I learned at a recent NIH-sponsored meeting that from now on, university centers that are selected for five-year grants for Parkinson’s research under the prestigious Morris K. Udall program must show a commitment to translational research. The news was announced at a session arranged for representatives of PDF and other PD organizations by Story Landis, Ph.D., Director of the National Institute for Neurological Disorders and Stroke (NINDS), the entity that provides more than $20 million a year to support the 14 Udall centers in the program. She said that the Udall centers would henceforth be moving towards including a more translational focus to their work, and that those academic centers that cannot make the switch will need to seek sources of other support, such as the so-called program project grants that NIH offers.

Specifically, in the language of the request for applications recently issued by NIH, “Responsive applications will demonstrate proven ability or considerable potential to pursue rapid translation of research to clinical practice.” Applicants that are not able to demonstrate this translational component – however strong they may be on the basic science – will simply not be eligible for designation as Udall Centers.

Another encouraging note sounded at our meeting was by Robert Finkelstein, Ph.D., who directs the institute’s extramural grants program that includes the Udall Centers. He reminded us that the new focus on translational research reflects the development of the science of Parkinson’s. He observed that 15 years ago, NIH would not have had so much opportunity to focus on translational proposals because the underlying basic science was simply not there to support them. He also said that he welcomed this new direction because “we at the NINDS have the mission to cure Parkinson’s!”

The reformulation of the Udall Centers is not the only good sign of a stronger commitment to translational science at the NIH these days. Another is the appointment of two dynamic new research administrators to fill the new positions of Director of Translational Research (Bill Matthew, Ph.D. ) and Director of Clinical Research (Petra Kaufman, M.D., M.Sc.).

Dr. Matthew has a strong background in industry -- most recently, as leader of the Partnering and Business Development at UCB, an international biopharmaceutical company based in Brussels. His charge at NINDS will be heading up efforts to translate the results of laboratory research into treatments for neurological disorders.

Dr. Kaufman, who is considered to be among the nation’s leading experts in the design and management of clinical trials for neuromuscular disorders, has served for a decade as an associate professor of neurology at the Neurological Institute at Columbia University.

Another promising development with big implications for translational research at the NIH as a whole is the appointment of the new Director, Francis Collins, M.D., Ph.D.. In his first statement about his dreams for the NIH, he included as one of his five top priorities, “translating basic science into new treatments.”

The times are changing, and in some good directions. At PDF, we will be working with our colleague organizations to make sure that these good times continue to roll.