Since her Parkinson’s diagnosis in 2005, Nancy Kaminsky and her husband, Rich, have been “putting one foot in front of the other,” she says. But things changed within the past year, when she began experiencing dyskinesia, the debilitating, uncontrollable movements that are a common side effect of the gold-standard treatment for PD. “Now,” she says, “it’s difficult to feel like you’re moving forward.”

Residents of Miami, Florida, Nancy and Rich have tried to manage this troubling side effect by working with her doctor to find the optimal dosage of levodopa-carbidopa. If she stays under a certain threshold, she can more effectively stave off the dyskinesia. But that also limits her relief from the equally debilitating motor symptoms of Parkinson’s. As with many patients, Nancy has turned to exercise, which has been helpful. With her ramped up exercise regimen, she’s even noticed she can better tolerate the lower dosage of levodopa-carbidopa.

While she and Rich maintain a positive outlook—and a good sense of humor—Nancy admits her dyskinesia is both frustrating and embarrassing. “You feel like you’re not in control,” she says. They recently attended a concert with some friends when her dyskinesia began, which is often worse at night. “It’s so unpredictable—you just never know when it will start. Nonetheless, you deal with it as best you can.” Continue reading “Dealing with Dyskinesia as Best You Can: One Couple’s Story” »

This week, Envoy Therapeutics announced a second grant from The Michael J. Fox Foundation (MJFF) for the ongoing development of an oral drug aimed at alleviating the symptoms of Parkinson’s disease (PD).  MJFF has committed more than $1.2 million toward this effort.

Envoy is developing compounds that target an area of the brain called the indirect pathway, the same pathway targeted in Deep Brain Stimulation (DBS).  In the case of DBS, a surgically implanted, battery-operated medical device called a neurostimulator delivers electrical stimulation to the brain to block the abnormal nerve signals that cause tremor and PD symptoms.  Continue reading “Michael J. Fox Foundation Awards Follow-on Grant to Envoy Therapeutics” »

“When I first started taking medication after my diagnosis, I was fine. To the point that I wanted to think I’d get over Parkinson’s,” shares Annette O’Brien, of Orlando, Florida. Almost 10 years later, the disease now exerts much more control over her day—especially through her dyskinesia, the debilitating, uncontrollable movements that are often a side effect of the gold-standard treatment for PD.

Annette says she wasn’t the first to notice her dyskinesia; others realized she was moving before she did. As the side effect became more apparent a few years ago, she says that she didn’t want to go places like she used to. She admits, “I’m embarrassed that I felt embarrassed, but I knew I looked funny.” The dyskinesia also causes her to drop things, and its exaggerated movements make her neck and shoulders tired. While she tries to manage her condition with medication, some aspects are out of her control—like stress.

A former school teacher, she loves being around children—especially her three grandchildren and extended family. But it can be difficult to keep up with their level of activity. She’s learned it helps her to take a “time out” in her room if she feels overwhelmed. “I can hear them laughing from my room,” she says. “Even if I can’t be with them in the moment, it makes me happy.” Continue reading “Hope for a More Dependable Day: One Patient’s Story of Dyskinesia” »

Researchers funded by The Michael J. Fox Foundation (MJFF) have identified the best clinical scale for measuring patient response to therapies to treat dyskinesia, the debilitating, uncontrollable movements that are often a side effect of Parkinson’s disease (PD) drugs.

The study, led by co-coordinating principal investigators Chris Goetz, MD, and Glenn Stebbins, PhD, of Rush University Medical Center, demonstrated that the Unified Dyskinesia Rating Scale (UDysRS) most sensitively tracks treatment effect.

Establishing UDysRS as a validated tool for use in dyskinesia clinical trials could have important implications moving forward. In the past, clinicians used several different scales measuring dyskinesia, without any real evidence that they accurately measured if drugs were working. Now, researchers should be able to better design accurate clinical trials to verify that a dyskinesia drug is — or isn’t — working.

It’s an important step toward bringing dyskinesia-targeted drugs closer to pharmacy shelves.

MJFF spoke with Stebbins and Scientific Advisory Board member David Weiner, MD, to gauge what the study results could mean for the development of new dyskinesia therapies, a major unmet need for those living with Parkinson’s today.  Read the entire interview here.  Excerpts are below. Continue reading “Weighing Different Scales: Unified Dyskinesia Rating Scale Comes out on Top, Could Speed Development of New Dyskinesia Treatments” »

Americans with advanced Parkinson’s disease (PD) who struggle with the levodopa-induced motor complications of wearing off and dyskinesia may soon have another treatment option: A levodopa-carbidopa gel delivered directly into the small intestine is moving closer to market, and test results suggest it may offer the same benefits as deep brain stimulation (DBS) — without the need for brain surgery.

Abbott Pharmaceuticals this week announced positive results from a phase 3 double-blind clinical study of its levodopa-carbidopa intestinal gel (LCIG), finding that LCIG significantly decreased “off” time (when the medication’s effects wear off) and increased “on” time without dyskinesia.  The Phase 3 results from this study are in line with interim results reported last year from an open-label study of LCIG.  (LCIG treatment is already available in Europe under the brand name Duodopa.) Continue reading “Novel Gel Formulation of Parkinson’s Drug May Limit “On/Off” Roller Coaster Ride” »

In this second edition of “The Sherer Report,” an ongoing series, Todd Sherer, PhD, MJFF CEO, provides an update on progress from the front lines of Parkinson’s research, and the implication this holds for those living with PD today.

Patients living with Parkinson’s today have a clear unmet need for improved treatment options to manage symptoms and increase quality of life. Current dopamine-based treatments are inadequate — they address only a subset of symptoms, lose effectiveness over time, and are marked by significant side effects such as dyskinesia (uncontrollable and debilitating movement). The Foundation continues to see encouraging progress toward new treatments that bypass the dopamine system altogether, as well as new formulations of dopamine replacement therapies that could address shortcomings of current treatments. Continue reading “The Sherer Report: Progress in New Drug Targets, Dopamine-Based Therapies and Clinical Trials” »

Today, good news in the search for a treatment for one of the most pressing needs for those living with Parkinson’s disease today — dyskinesia.   

Swiss biotech Addex Therapeutics announced positive results from a Michael J. Fox Foundation-funded phase 2 clinical study of their drug candidate dipraglurant, which is being tested as a therapy to be taken in conjunction with levodopa (the gold standard for treating PD).  Levodopa works to restore levels of dopamine in the brain, improving movement and other functions that decline in Parkinson’s disease.  But levodopa can also lead to dyskinesia, the uncontrollable movements that are a disabling side effect of the drug. The hope is that dipraglurant would allow patients to experience the benefits of levodopa while limiting dyskinesia. Continue reading “Addex Adds It Up for Parkinson’s Patients” »

While levodopa is the gold standard therapy for Parkinson’s disease (PD), current delivery methods have their limitations. Many patients who take it experience “off” periods, when the medication’s effects wear off before they are ready to take their next dose, leaving them in a state where symptoms like lack of mobility may return.  Those taking levodopa also risk developing dyskinesias, the excessive and uncontrollable movements that are among the most difficult aspects of the disease to manage.

But finding a more consistent method of delivering levodopa could help to minimize both “off” periods and dyskinesias alike. Continue reading “Podcast: Limiting On-Off Periods in Parkinson’s Medications” »

Dyskinesia refers to the excessive and uncontrollable movements that are a side effect of long-term dopamine replacement therapy in patients with Parkinson’s disease (PD). It is a harrowing problem for people with PD, who report that it is one of the most difficult aspects of Parkinson’s to manage.

Because they fear developing dyskinesia, many patients wait as long as possible to begin using the drug levodopa, the gold-standard treatment for relieving the stiffness, tremors, and rigidity that are the cardinal features of PD. Even after starting the medicine, many limit the dosage to reduce the dyskinesias, and therefore settle for a sub-optimal benefit from the best medical therapy available for their disease. There is currently no therapy approved by the U.S. Food and Drug Administration (FDA) to treat this side effect. Continue reading “Dave Iverson & Dr. Todd Sherer: New Approaches to Treating Dyskinesia” »

With MJFF funding, Jeff Conn, PhD, at Vanderbilt University has accelerated a promising new PD treatment toward the clinic. Instead of targeting the dopamine system, his approach focuses on the glutamate system, which could match the benefit of levodopa without triggering dyskinesias.

Appearing in the MJFF Winter 2011 newsletter,  this article was the first in “The Fox Approach”–a recurring series highlighting MJFF’s out-of-the-box approach to speed the cure. Stay tuned for more stories like this one!

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In December 2007, the Wall Street Journal reported on a troubling trend in drug development.

“Over the next few years the pharmaceutical business will hit a wall,” the paper wrote. In the modern era of complex disease, blue-chip drugmakers’ in-house research operations were bringing fewer and fewer therapies to market. Struggling to recoup their research investments in the development of new treatments that can cost over a billion dollars, more and more companies were downsizing these programs as part of major cost-cutting initiatives. For many, patents on the most profitable products in their portfolios were soon to expire.

“In five years,” the Journal predicted, “many [of these companies] may look very different. They will be in new businesses.”

Continue reading “Pioneering Partnership with MJFF Drives New Parkinson’s Treatment Toward Pharmacy Shelves” »