Advancing Research at KU Med Through Project5

Neuroscience at KU Medical Center

In confronting the great medical and scientific challenges of our time, physician scientists at the KU Medical Center respond with innovation. This is particularly true of our neuroscientists. For generations, the Kansas City community has relied on the nationally recognized doctors and researchers at the University of Kansas Medical Center for advanced prevention and treatment strategies to battle neurodegenerative disease. Each day neuroscientists work to answer the most difficult questions in medicine. Richard Barohn, MD, one of the medical center’s most distinguished physician scientists, is searching for the clues that will one- day unravel the mysteries of the brain. With a focused interest in ALS and working in partnership with organizations like Project5  for ALS, Dr. Barohn continues to explore the use of new therapies in treating this devastating disease.

Advancing Research through Philanthropy

Funds contributed by Project 5 for ALS were allocated to support a phase 2 trial of Rasagiline for the treatment of ALS.  Specifically, KU Neurology researchers set up some novel biomarkers to measure oxidative stress in the blood. These tests are performed in Dr. Russell Swerdlow’s lab in the Department of Neurology. The research protocol using Rasagiline in ALS patients was approved by our Internal Review Board (IRB). KU Medical Center was the main coordinating center for this study.  Other sites from the Western ALS Study Group were involved, and we ultimately enrolled 30X patients. All patients were put on Rasagiline 2mg a day. There was no placebo. The sites drew blood for biomarker studies before they started Rasagiline at six months and at the end of the study at 12 months. The goal was to determine if there was any change in the biomarkers on Rasagiline therapy. In addition, we were looking at the ALS Functional Rating Scale (ALSFRS) throughout the course of the study as our main measurement of efficacy. KU Medical Center presented preliminary data of the study at the annual international motor neuron disease meeting in Chicago in December. This interim six-month analysis did show Rasagiline seemed to affect the mitochondria for patients with ALS while they were taking the drug. It is too early to know if the Rasagiline will ultimately change the course of ALS in these patients.  The primary funding for this study came from TEVA, but they did not provide funds to do the biomarker tests. Therefore the Department of Neurology research team, led by Dr. Rick Barohn used Project 5 ALS funds to buy the laboratory equipment and to pay some of the lab technicians’ salary to do the biomarker tests. The blood was shipped in from the sites in the Western ALS Study Group on the patients enrolled in the study, and all the tests were performed in Dr. Swerdlow’s lab here at KU Medical Center.  KU Medical Center is now starting a new second study of Rasagiline in ALS while the first study is completing. This study will involve 80 patients. Sixty will receive Rasagiline, and 20 will receive placebo. The research team plans to continue to do biomarker blood testing in the patients and some new biomarker testing in the urine for oxidative stress. In addition we are going to do novel brain imaging biomarker pilot testing on the patients enrolled at KU Medical Center. Any future Project 5 for ALS funds that we receive will once again help us to fund these cutting edge biomarker research initiatives for ALS.

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