Now that we’ve shaken off the red-eye fog and caught up on emails, we’re ready to share some of the news and insights we learned at last week’s 19th International Congress of Parkinson’s Disease and Movement Disorders in San Diego.
Physicians, researchers and allied health care professionals from around the globe gathered to discuss the latest trends in understanding and treating people with Parkinson’s disease (PD) and other movement disorders. MJFF staff shared Foundation resources, attended educational sessions and presented a poster on our clinical studies matching tool Fox Trial Finder.
Here’s some of what we learned:
Clinician-Researchers Are Noting Non-motor Patient Needs
Speakers presented on the need for more therapeutic development for anxiety and apathy. Antidepressants are used for anxiety, but outcomes are inconsistent and they can bring side effects. Telemedicine may allow for behavioral therapy to treat anxiety and apathy to reach more people with Parkinson’s. The Foundation is funding a study of this technique for treating depression with PD; success may lead to expansion to other conditions.
Another session covered gastrointestinal issues. James Parkinson noted drooling, swallowing problems and bowel dysfunction as part of his newly profiled disease in the early 1800s, but these symptoms persist. Surveys report 30 to 80 percent of people with PD experience gastrointestinal issues (the wide range may come from how questions are asked). Swallowing issues can occur early in the disease course but are more evident later, while constipation can be traced to up to 20 years before diagnosis. We hosted a webinar on speech and swallowing problems earlier this month and on July 16 we’ll discuss constipation. Watch the archive and register for our upcoming event at michaeljfox.org/webinars.
Pain also is more common in people with Parkinson’s, primarily due to the symptom of dystonia (painful, sustained cramping). Rigidity, slowness of movement and dyskinesia are also associated with pain in Parkinson’s. It may be that Parkinson’s affects the way one feels pain. Speakers at the Congress reported how the part of the brain affected by PD (basal ganglia) plays a role in how the body senses potential harm, and, therefore, people with Parkinson’s may have reduced threshold for pain. There are studies ongoing to treat pain and to understand its impact.
Trials of Levodopa Reformulations Move Closer to Patients’ Hands
Pharmaceutical company Acorda presented a poster on the results of its Phase IIb study: “Inhaled Levodopa (CVT-301) Provides Rapid Improvement of OFF States in Parkinson’s Disease.” MJFF supported early development of CVT-301 by biotech Civitas before it was acquired by Acorda.
CVT-301 is a dry-powder formulation of levodopa taken through an inhaler, like those used for asthma, when traditional oral medication wears off. With longer disease duration, levodopa can lose efficacy and wear off before its time for another dose. Oral medication takes time to have an effect, which is where fast-acting CVT-301 comes in.
Acorda’s poster showed that study participants receiving CVT-301 showed a significant reduction in motor symptoms compared to placebo beginning at 10 and up to 60 minutes after using the inhaler. Both doses of CVT-301 were well tolerated, with no increase in dyskinesia relative to placebo.
Also presenting data on its MJFF-funded reformulation of levodopa was biotech Neuroderm. Its product ND0612 is a liquid formulation of levodopa/carbidopa administered through the skin either by a belt-worn pump or a patch pump. The company presented that both its low-dose and high-dose versions (for moderate to severe PD and severe PD, respectively) showed consistent levels of levodopa in blood plasma. This administration technique should maintain drug levels to avoid the motor fluctuations (“on/off” episodes) often seen with oral medication.
Both Acorda and Neuroderm’s posters were chosen for the meeting’s Blue Ribbon Highlights Session, which highlights relevance, novelty and quality of clinical data and basic research.
A different approach, Foundation-funded Cynapsus presented data from its trials of a thin-film, under-the-tongue strip of apomorphine, a dopamine agonist. The company's drug APL-130277 is another "off" rescue therapy and showed improvement in motor symptoms within 15 minutes of use. Apomorphine is currently available but only in an injectible form, which can be difficult to use in an "off" state.
Listen to a podcast about what causes “off” episodes and how these drugs are hoping to overcome this challenge.
Many Questions Surround GBA’s Role in Parkinson’s
In the disease modification realm, researchers are paying increased attention to the glucosidase, beta, acid (GBA) gene as mutations there are associated with Parkinson’s. At the Congress, scientists discussed that three to four percent of all people with PD may carry a GBA mutation. These variations are more common in people of Ashkenazi Jewish descent (perhaps up to six percent).
The meeting presenters outlined how they are studying the relationship between GBA and alpha-synuclein (the protein that clumps in cells of people with PD) and the types of drugs they’re investigating against this target to stop or slow Parkinson’s progression. They’re also looking at why the majority of GBA mutation carriers do not develop PD — are there additional risk factors or protective factors that they could study toward new therapies? Learn more about how MJFF is supporting research into GBA.
MJFF staff Rachel Dolhun, MD, and Maurizio Facheris, MD, MSc, contributed to this post.