This Parkinson’s Awareness Month was a busy one—at The Michael J. Fox Foundation and throughout the Parkinson’s community. April was filled with promising research news, innovative partnerships, surprise appearances, great media hits, key milestones, new friends and more.
The results: More people are aware of the urgent need to speed better treatments—and a cure—to patients. And they’re ready to take action.
A few of April’s highlights: Continue reading “Recapping Parkinson’s Awareness Month” »
This week, research published in Neurology, the medical journal of the American Academy of Neurology, found that a nicotine patch may improve the memory loss common in mild cognitive impairment (MCI), a condition that is often a precursor to Alzheimer’s disease. Sixty-seven people completed the study which took place over six months, and according to lead investigator Paul Newhouse, MD, of Vanderbilt University, improvements were seen in attention, memory, and cognitive impairment.
This positive result could have implications for people with various neurological diseases, including Parkinson’s. To date, there has been widespread epidemiological data to suggest a protective effect in nicotine for PD: the disease is less prevalent among smokers as compared to non-smokers, and early results from pre-clinical studies have shown potential therapeutic benefits.
MJFF is funding a clinical study set to begin this spring called NIC-PD that will begin to look into the disease modifying potential of a nicotine patch, as well as its potential effects on the non-motor and in particular, cognitive features of PD. The study will engage more than 150 people across more than 20 sites in the U.S. and Germany. Clinicians will assign either nicotine or placebo patches to the participants over a 12 month period to determine efficacy against the disease.
While the recent study results on MCI are encouraging, it is important to note the relatively small scale nature of the study, and that the use of nicotine patches as a treatment for any neurological disease is still in the early stages of investigation.
This past week, the Boston Globe profiled MJFF awardee Civitas Therapeutics, a Chelsea, Massachusetts based pharmaceutical company that is working to develop a therapy to address the challenges in how levodopa, the gold standard therapy for Parkinson’s disease (PD), is delivered into the body. Many patients who take the drug experience “off” periods, when the effects of the medication wear off before they are ready to take their next dose, leaving them in a state where symptoms like lack of mobility may return. Finding a more consistent method of delivering levodopa could help to minimize these periods, and is therefore one of the critical unmet needs for those living with PD.
To date, the practical way that the drug has been absorbed is in pill form. Civitas, however, is studying an inhaled formulation of levodopa for its potential to produce more rapid and continual relief from the debilitating motor fluctuations that are associated with Parkinson’s.
The Globe’s story highlights how, in January of 2011, Civitas was launched as an independent spin-out from a company called Alkermes that was already working to develop a new way to deliver drugs into the lungs through an inhaler. Insulin, human growth hormone, and osteoporosis drugs were among Alkermes’ early targets. Civitas founders Glenn Batchelder and Martin Freed believed that the inhaler might have a positive impact on PD, in that it could deliver levodopa more quickly than pills, which could in turn help to limit these off periods that are associated with taking the drug.
Civitas is in the initial stages of clinical assessment of their drug candidate, called CVT-301. Today, they announced early results showing that a sufficient amount of the compound was found in the blood plasma of patients when inhaled into the lungs. They also reported that the doses administered to trial participants were safe.
This past October, Austrian biotech AFFiRiS announced $1.5 million in funding from MJFF for an early stage clinical trial of a first-of-its-kind vaccine approach to treating Parkinson’s disease (PD), called PD01. Then, in November, the company announced 30 million Euros in additional funding from two investors to develop its portfolio of PD and Alzheimer’s vaccines.
In this podcast, Dave Iverson speaks with AFFiRiS’ Chief Medical Officer Achim Schneeberger about PD01, and how it could work to break up the clumps of the protein alpha-synuclein that occur in the brains of those with PD. The hope is that, in so doing, the treatment could modify the course of the disease itself.
A PD Vaccine Approach
MJFF Co-Founder and Executive Vice Chairman Debi Brooks has not only been instrumental in conceiving, structuring and fundraising for the Parkinson’s Progression Markers Initiative (PPMI); she also is participating in the study as a control.
This is the first time Debi has volunteered for clinical research, and she is blogging about what she learns along the way. In her first post (below), she reflects on her decision to enroll in the study.
Early in our planning for PPMI, our goal was to recruit 30 people per site — 20 newly diagnosed Parkinson’s patients, and 10 controls — by the end of 2012. That meant each site needed to recruit roughly one PD patient per month and one control every two months, which seemed doable to me. But the immediate reaction from the scientists in the room was that this was an incredibly lofty goal, maybe even impossible, due to the routine challenges they face in finding volunteers for studies. It was a striking exchange, and it inspired me to enroll.
Continue reading “Debi’s Blog: Why I Raised My Hand to Participate in PPMI” »
In this first edition of “The Sherer Report,” which will become an ongoing series, Todd Sherer, PhD, MJFF CEO, highlights recent developments in three of the Foundation’s high-priority research areas, which hold clear implications for those living with PD today.
New Approaches to Treat Symptoms
Many of the motor symptoms of Parkinson’s result from a decrease in dopamine, a brain chemical that helps control movement, balance and walking. For the last 40 years, nearly every treatment for PD, including the currently available medications Sinemet, Mirapex, Azilect and Stalevo, have focused on attempting to replace this lost dopamine.
However, new lines of research are developing PD treatments based on different mechanisms that target brain chemicals other than dopamine. These approaches could replace or supplement existing therapies, limiting side effects such as dyskinesias, the uncontrollable movements that are a common side effect of existing PD drugs, while targeting some of the currently untreated symptoms of PD. Continue reading “The Sherer Report” »
In the fall 2011 issue of our newsletter, Accelerating the Cure, Nate Herpich, MJFF’s associate director of research communications, provided updates on some of our funding, including: drug repositioning, a potential PD vaccine and PPMI. Check out the newsbriefs below or click here to read the rest of the fall issue.
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RESEARCH NEWSBRIEFS
Reducing the Drug Development Timeline: Repositioning RFA
Over the past year, The Michael J. Fox Foundation has ramped up its interest in repositioning. This is the process of testing a drug that is already FDA-approved or that has been proven safe in a clinical trial for a certain indication, to determine whether the drug might be efficacious in the treatment of another therapeutic area, like Parkinson’s disease (PD). “Repositioning can drastically reduce the timeline for getting a drug into clinical testing for PD, making an impact on patients’ lives that much sooner,” says Kuldip Dave, PhD, associate director of research programs at MJFF.
While MJFF has supported repositioning projects before, its first-ever repositioning specific Request for Applications (RFA) launched in fall 2010. MJFF announced the results of this RFA in July: six researchers with awards totaling $2.4 million. Three additional awards were granted in August through other programs, bringing the total funding for repositioning to $3.4 million. Several of the research teams are working with drugs that could offer protection to the brain cells that die in Parkinson’s disease, including a tuberculosis vaccine and the antidepressant duloxetine (marketed as Cymbalta). Others are testing symptomatic therapies, including a compound used to treat Attention Deficit Hyperactivity Disorder (ADHD).
A Potential Vaccine to Slow Parkinson’s Disease Progression
This summer, MJFF awarded funding for a first-of-its-kind therapeutic approach to Parkinson’s disease: a vaccine targeting alpha-synuclein, a protein implicated in Parkinson’s disease. The vaccine aims to slow or stop the progression of Parkinson’s, something no current treatment can do.
Vienna, Austria-based company AFFiRiS AG will conduct a clinical study of the safety and efficacy of its vaccine candidate PD01. The hope is that this agent will simulate the production of antibodies that bind to alpha-synuclein, clearing it from the brain and slowing disease progression. Alpha-synuclein is a high-priority target for MJFF, largely because there is compelling evidence that it may play a role in both genetic and idiopathic cases of PD. It is also the major component in the Lewy bodies that are the pathological hallmark of Parkinson’s.
The trial, led by AFFiRiS chief medical officer Achim Schneeberger, MD, will engage 24 subjects with mild Parkinson’s disease over two years at a single clinical site in Austria. “Cautious optimism is called for,” says Jamie Eberling, PhD, MJFF’s associate director of research programs, “but it’s important to bear in mind that PD01 is still in the early stages of clinical testing.”
On the Path to Finding Biomarkers: PPMI Update
Since MJFF launched the Parkinson’s Progression Markers Initiative (PPMI) more than a year ago, the project has grown in scope, and it continues to expand its presence across the United States and Europe. Nine industry funders, including leading pharmaceutical and biotech companies, have partnered with MJFF to support this work, and researchers and clinicians across the world have begun to analyze the data and biological samples that could unlock clues to the onset and progression of Parkinson’s disease. In a move to make PPMI a truly global study, in August MJFF announced a collaboration with the Shake It Up Foundation Australia to bring PPMI down under, increasing our chances to quickly identify universally relevant biomarkers.
Of 21 planned clinical sites in the United States and Europe, 19 are now active, with two more in the EU set to begin enrolling participants soon. 179 individuals are actively enrolled at PPMI sites, including 101 newly diagnosed PD participants, and 78 control participants who do not have PD. Another 45 volunteers are pending enrollment. Samples and data collected in PPMI are available to the scientific community at large in real time for promising biomarker validation studies. Scientists using the initiative’s resources agree to report back to PPMI with findings and data, enabling future researchers to build on their work to help speed promising PD biomarker validation efforts worldwide.
You, too, can help with these efforts. PPMI continues to recruit and must find at least 400 people who are newly diagnosed with PD, and 200 controls. If you or someone you know might want to participate in PPMI, visit www.michaeljfox.org/PPMI to learn more about our study and find the nearest site.