Physicians Study Potential Therapy for ALS Patients

Benjamin Rix Brooks, MD

Benjamin Rix Brooks, MD
Director of Carolinas Neuromuscular/ALS-MDA Center

Benjamin Rix Brooks, MD, director of Carolinas Neuromuscular/ALS-MDA Center at the Neurosciences Institute, and Elena Bravver, MD, also with the Neurosciences Institute, are teaming up with investigators from across the country in a new study. The research team is attempting to prove the safety and effectiveness of a new therapy for the treatment of muscle weakness and muscle fatigue in patients with Amyotrophic Lateral Sclerosis (ALS).

During the study, the drug CK-2017357 will be tested to determine how well it’s tolerated in ALS patients, compared to a placebo pill.  “The study will determine the drug’s effects on the performance of a patient’s elbow, wrist, hand, knee and ankle muscles, their breathing, and how long they can maintain a task,” Dr. Brooks said.

Approximately 680 ALS patients are expected to take part in this research study at an estimated 80 sites throughout the United States, Canada and Europe. The results for this trial will be made available at the end of the study, once all data has been analyzed. The study is no longer recruiting additional patients.

Elena Bravver, MD

Elena Bravver, MD
Neurosciences Institute

This is the second Cytokinetics study the Neurosciences Institute’s ALS clinic has been invited to join. The first trial concluded in 2011 and involved the same investigational compound. Cytokinetics is an emerging biotechnology company that is dedicated to the discovery, development and commercialization of therapeutics. Drs. Brooks and Bravver were asked to participate a second time, as the Neurosciences Institute’s ALS clinic has grown to be well-respected throughout the industry.

“We were also one of the highest enrolling sites for both the previous and current study, and are proud to be able to offer this opportunity to our patients and participate in this very important research,” said Dr. Bravver.

Additionally, Dr. Brooks is currently seeking patients to participate in a research study of resistance and endurance exercise in ALS at the Neurosciences Institute. The trial, in collaboration with John Hopkins University, makes Carolinas HealthCare System just one of eight study sites in the United States participating in this trial. The purpose of this study is to see whether one type of exercise is tolerated better and is safer than another for people with ALS.

During this study, participants with ALS will be asked to exercise in one of three ways:

  • Weightlifting (resistance exercise)
  • Stationary bicycling (endurance exercise)
  • Range of motion stretching exercise (the current “standard of care” for ALS patients).

If resistance, endurance or range of motion exercise are shown to be well-tolerated and safe over 24 weeks, a larger trial will be planned to determine if exercise is beneficial to all ALS patients.

“By participating in these studies, we are providing access to excellent care and clinical expertise for our patients. It’s because of our Center’s continuous efforts to improve and succeed that we are consistently ranked at the top of comparable neurology centers across the country,” said Dr. Brooks.

View a full list of current and upcoming neuroscience clinical trials taking place at Carolinas HealthCare System.

Fox 46 recently covered a patient’s experience as a participant of the study. Watch the video clip.

Learn more about neurology and sign up for The Neuro Report, a physician-focused newsletter.

Physician Participates in Michael J. Fox Foundation Panel for Parkinson’s Patients

Sanjay S. Iyer, MD, director of Carolinas Center for Parkinson’s Disease and Movement Disorders and co- medical director of Carolinas HealthCare System’s Neurosciences Institute, was invited to participate in a research roundtable for Parkinson’s disease (PD) hosted by The Michael J. Fox Foundation for Parkinson’s Research (MJFF).

“Since the audience was mainly composed of patients and their caregivers, this was a perfect opportunity to educate them about what is novel in PD, including what the latest treatments are, how far we’ve come in terms of research and how close we are to finding a cure,” said Dr. Iyer.

Each year, MJFF hosts several educational research roundtables across the United States. These educational forums, comprised of expert panelists, cover topics that matter most to patients and their loved ones including learning about the latest developments in Parkinson’s research and what they can to do to help be a part of finding a cure.

For the Charlotte event, hosted in January, Dr. Iyer, was joined by Mark Stacy, MD, professor of neurology and vice dean of clinical research at Duke University; Deborah W. Brooks, co-founder and executive vice chairman of MJFF; and Mark Frasier, PhD, vice president of research programs, also with MJFF.

Until there is a cure, Dr. Iyer believes physicians have opportunities to help patients with their own self-management. “Patients always ask how they are doing. They want to know how far along their disease has progressed,” said Dr. Iyer. “To help our patients keep track and to help us determine if they are trending in a negative direction, we’ve started using predictive analytics to measure a patient’s status.”

Dr. Iyer and his team are planning a pilot with a test known as the Timed Up and Go Test (TUG), which is used to assess balance and mobility in PD. TUG is a simple exercise that patients and their caregivers can perform on their own. The test measures, in seconds, the time taken by patients to stand up from a chair, walk a marked distance, turn around and sit back down in the chair. The time is then compared to normative values for age, gender and research-based guidelines.

“By asking patients to routinely perform this test and record their results, we will then analyze to predict if they have a high risk of falling, and over time, we think this is will be a good predictor of when there could be a bad outcome and we can do an intervention to help prevent that from happening,” said Dr. Iyer.

Learn more about Parkinson’s research at Carolinas HealthCare System.