Expertise and Current Research Activity
I work in the field of rare neuromuscular diseases such as amyotrophic lateral sclerosis (ALS), muscular dystrophies, myasthenia gravis, and other muscle and nerve diseases. At any one time we are involved in about 50 cutting edge clinical trials through our neuromuscular division at the University of Kansas Medical Center.
Patients come from throughout the Midwest and Southwest and sometimes from much more distant regions to take part in these clinical trials. In the last year, we had three patients come from New Zealand to be part of a drug trial for a rare metabolic muscle disease called Pompe disease, and they lived in Kansas City for several months while they were receiving the research drug. The trials that we are involved in are a combination of investigator-initiated studies that we initiate; investigator-initiated studies that my collaborators around the country initiate and we are asked to be a site in; and industry-sponsored trials.
We have been very fortunate to receive federal funding to lead three multi-center neuromuscular clinical trials: the first was to study the drug mexiletine for nondystrophic myotonia muscle disorders (the results of this positive trial were published in JAMA in October 2012); a trial of methotrexate for myasthenia gravis involving 20 sites in the U.S. and Canada which we have just completed and are now analyzing the data; and a trial of rasagiline for ALS for which we have just begun to recruit patients around the United States. We also are part of the national NeuroNext consortium which consists of 25 sites that the NIH recognizes for their expertise in neurology clinical trials, and we are one of the co-leaders on the recently funded NeuroNext trial to study the drug rituximab in myasthenia gravis. Finally, we recently received funding from the new Patient-Centered Outcomes Research Institute to do a comparative effectiveness trial of four different drugs for the treatment of painful peripheral neuropathy. We will be the coordinating center for a 400-patient trial with 25 sites which will take place over the next three years.
All of these projects are helped in various ways by infrastructure support from Frontiers: The Heartland Institute for Clinical and Translational Research which is funded by our $20 million CTSA grant on which I am one of the two principal investigators (along with Lauren Aaronson, PhD, RN, in the School of Nursing). The CTSA program at the NIH now falls under NCATS, and I was recently named to be one of the 12 principal investigators to serve on a new steering committee that will develop new directions for the CTSA program.