More than 250 of the brightest minds working on Parkinsonís disease (PD) ó scientists, clinicians and industry leaders from around the world ó convened in New York City on October 29 for the eighth annual Parkinsonís Disease Therapeutics Conference, hosted by The Michael J. Fox Foundation.
The day was filled with updates on the biomarker search and on the status of symptomatic and disease-modifying drugs in development.
Below we summarize a few of the highlights. †
Vaccine Targeting Alpha-Synuclein Deemed Safe
Alpha-synuclein ó the protein that clumps in cells in PD ó is a promising target for disease-modifying therapies. Researchers believe that removing this ďsticky proteinĒ may slow or even stop Parkinsonís progression. Austrian company AFFiRiS created a vaccine to stimulate the body to make antibodies against alpha-synuclein, hopefully clearing it from the brain.
At the conference Achim Schneeberger, MD, of AFFiRiS shared that their MJFF-funded Phase I clinical trial found the vaccine safe and well tolerated. Each of 24 people with early Parkinsonís received a total of four vaccinations over three months. Side effects were mild; the most common was an injection site reaction of pain or redness.
Phase I trials are not designed to evaluate efficacy of a therapy. Still, investigators saw an antibody response in 15 of the 24 who were vaccinated. They also noted an improvement in motor function in the vaccinated group as compared to a control group that did not receive the vaccine.
MJFF is supporting the next step ó the same study participants will receive a fifth vaccination. The goal is to boost the level of antibodies against alpha-synuclein to ensure longer lasting effects.
Expanding the Search for a Tool to Speed Research
The investigation of PD presents many hurdles. The disease process begins long before symptoms appear, no test exists to confirm diagnosis or to track progression, and Parkinsonís shows differently in each individual.
Biomarkers ó objective measures that aid in diagnosis, disease tracking and faster testing of therapies ó would address these challenges. Finding accurate biomarkers is the goal of the Parkinsonís Progression Markers Initiative (PPMI).
MJFF and industry partners established PPMI in 2010, and the international, observational study has evolved over time. PPMI has enrolled more than 400 people with newly diagnosed Parkinsonís and 200 control volunteers without PD. They undergo extensive and standardized motor, memory and other testing and submit blood, urine and spinal fluid samples. All of the original study participants have now completed one year of study visits.
At the PD Therapeutics Conference, principal investigator Ken Marek, MD, spoke about the growth of PPMI. Early this year ó to increase understanding of the role of genetics and Parkinsonís ó PPMI began recruiting people with certain genetic mutations associated with PD. In addition, to find a way to diagnose the disease earlier, people without PD but with symptoms that often predate Parkinsonís (smell loss or REM sleep behavior disorder) are also joining the study.
As its model proves strong, PPMI also has expanded to incorporate additional motor and memory testing, specialized brain imaging and skin biopsies.
Collection of this valuable data over time and collaboration among investigators will lead to biomarkers that will accelerate advancement of disease-modifying therapies.
Continuous Levodopa Lessens Motor Fluctuations
In an MJFF-supported Phase II clinical trial, the first liquid formulation of the popular PD drug combo levodopa/carbidopa decreased motor fluctuations ó ďoffĒ time and dyskinesia ó in people with moderate Parkinsonís disease.
The ďpump-patchĒ delivery system provides a continuous amount of medication under the skin, avoiding the irregular absorption that occurs when the drug is taken orally.
Sheila Oren, MD, of biotech Neuroderm reported that people with moderate PD who used the pump-patch in place of their regular medication experienced a decrease in ďoffĒ time of over two hours and an improvement in time without dyskinesia. Drug levels remained at a consistent steady concentration in the blood.
Dr. Orenís team is now evaluating the same dose and a higher dose in people with more advanced Parkinsonís disease, and preliminary test results are promising. This may be an alternate option for those considering deep brain stimulation.
Investigators say the drug will reach the pharmacy by 2018.
The 2015 Parkinson's Disease Therapeutics Conference will be held November 3. Register today to attend.