Mitsubishi Tanabe Pharma America, Inc. (MTPA) has announced a collaboration with the Massachusetts General Hospital (MGH) Neurological Clinical Research Institute (NCRI) to conduct a study designed to identify and measure specific biomarkers in people with amyotrophic lateral sclerosis (ALS).
Established in 2016, based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation’s (MTPC) 100% owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. Mitsubishi Tanabe Pharma, was founded in 1678, and is headquartered in Doshomachi, Osaka, which is the birthplace of Japan’s pharmaceutical industry. Through the discovery of drugs that address unmet medical needs, centered on its priority disease areas — autoimmune diseases, diabetes and kidney diseases, central nervous system diseases, and vaccines — Mitsubishi Tanabe Pharma will strive to contribute to the health of patients around the world.
The ALS biomarker study, which will be sponsored by MTPA and led by MGH, has been designed to have 200 patients complete six cycles (24 weeks) of treatment, and will be conducted at up to 40 sites across the country. The findings may assist with identifying specific biomarkers as quantifiable, biological, non-clinical measures for the treatment effect of the drug edaravone in people with ALS.
The first biomarker study patient is anticipated to be enrolled late spring of 2019, with early interim analyses planned for later in the year. Patient biomarker data and disease progression assessments will be compared to samples stored at biorepositories and progression models, respectively.
The study will evaluate the following biomarkers:
- Oxidative stress – 4-hydroxynonenal (4-HNE), 8-Isoprostanes, 3-nitrotyrosine (3NT), 8-hydroxy-2′-deoxyguanosine (8OHdG), urate
- Inflammation – matrix metalloproteinase-9 (MMP-9)
- Neuronal injury and death – neurofilament (Nf) heavy and light chain proteins, urinary neurotrophin receptor p75
- Muscle injury – creatinine
“We are honored to be working with the team at MGH on our first clinical trial in the U.S.,” … “This biomarker trial has sparked exponential interest in the ALS community, and with the help of MGH, we hope to further our understanding of the potential role these measures may have in evaluating a treatment response.” – Stephen Apple, M.D., Senior Medical Director, Medical Affairs, MTPA
“ALS is a complex disorder with diverse pathophysiology, and we do not currently have validated biomarkers for diagnosing or following ALS progression,” … “This study will broaden our understanding of numerous biomarkers that may be associated with ALS, including those for oxidative stress, inflammation, muscle and neuronal injury and death. It will increase our understanding of ALS and of the biological effects of edaravone in people with ALS undergoing therapy.” – Primary Investigator James Berry, M.D., M.P.H., MGH NCRI, Boston