Foundation Awards $2.3 Million to Develop Treatments for Dopamine-Non-Responsive PD Symptoms
This program was funded with a lead gift from the Edmond J. Safra Philanthropic Foundation in memory of its founder, Mr. Edmond J. Safra. The Edmond J. Safra Philanthropic Foundation has been one of the most steadfast supporters of The Michael J. Fox Foundation since its inception.
Parkinson’s is best known by the general public as a movement disorder whose cardinal symptoms are resting tremor, slowness of movement and rigidity. PD patients also experience a wide range of lesser-known symptoms, however, many of which are non-motor or autonomic in nature, and which are not responsive to existing dopamine replacement therapies. These include depression, constipation, postural instability and gait, cognitive dysfunction, sleep disorders, fatigue and pain, among others. Many patients report that they are among the most debilitating aspects of the disease.
“The clinical features of Parkinson’s that do not respond to dopamine replacement are poorly understood and frequently go untreated — yet little work is currently being done to address them,” said Deborah W. Brooks, president and CEO of the Foundation. “We launched our Dopamine-Non-Responsive initiative to focus researchers on the pursuit of therapies for these unmet symptomatic needs that rob patients of their quality of life.”
Studies on postural instability and gait disorders
Three of the funded studies address postural instability and gait disorders, whose causes are not known and for which there is currently no effective treatment.
Pierre Pollak, PhD, of INSERM –
Chantal François, PhD, of INSERM – Hôpital de la Salpêtrière, Paris, also will focus on the PPN in an effort to increase understanding of how it contributes to posture and gait dysfunction. Dr. François will conduct behavioral tests and molecular analysis of cholinergic degeneration, a pathological feature of Parkinson’s disease, in the PPN of primate PD models. Results of Dr. François’s work will provide direct evidence for a role of the PPN in postural instability and gait disorders, as well as potentially furnish a preclinical model that can be used to test therapies to treat them.
Lisa Shulman, MD, of the University of Maryland School of Medicine will conduct a clinical trial to evaluate the effectiveness of treadmill training with significant aerobic exercise on gait and mobility. Dr. Shulman’s team will also evaluate the effects of these exercise therapies on other non-dopamine-responsive symptoms of Parkinson’s, including depression, apathy and fatigue. Positive results from this trial would provide physicians with an optimal exercise regimen to prescribe patients suffering from postural instability and gait disorders, something that does not currently exist.
Studies on depression/apathy, constipation and non-dopaminergic degeneration
Three other studies will examine apathy, constipation and potential PD-related degeneration outside the dopaminergic system.
Hubert Fernandez, MD, of the
James Greene, MD, PhD, a neurologist at
Kenneth Marek, MD, of the Institute for Neurodegenerative Disorders is a world leader in the development and validation of neuroimaging markers. Dr. Marek and colleagues will test two novel imaging markers in PD patients — one for the serotonin transporter and one for the norepinephrine transporter. The team seeks to identify and validate critical research tools that could be used to test the emerging hypothesis that PD-related degeneration may be occurring outside the dopaminergic system, and that this degeneration may contribute to the development of dopamine-non-responsive symptoms.
Like all Foundation funding initiatives, Dopamine-Non-Responsive Symptoms of PD requires the designation of time-dependent milestones. Continued funding will be dependent upon successful completion of these milestones.
Researcher bios and grant abstracts are available on the Foundation’s Web site at www.michaeljfox.org.