Post-Hoc Analysis of ALS Treatment Study Looks at Multiple Staging Systems
JERSEY CITY, N.J., November 30, 2020 – Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced results from a post-hoc analysis that examined the use of clinical staging systems to measure disease progression in amyotrophic lateral sclerosis (ALS), based on data from an edaravone Phase 3 trial (Study 19). The analysis, which was published in the Journal of Neurology, Neurosurgery and Psychiatry (JNNP), a subset of British Medical Journal (BMJ), show the King’s and Milano-Torino (MiToS) staging systems both provided potential value in assessing efficacy.
“Clinical staging systems have shown value in a variety of neurological diseases, but in ALS many studies have relied on a validated functional rating scale score,” said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs, MTPA. “While this score is useful to assess change in a patient’s functional status, we also recognize the potential benefit of utilizing clinical staging scales to evaluate other important disease progression milestones and provide complementary information. We are pleased to be able to share these post-hoc findings that support further exploration of clinical staging systems in ALS research.”
Study 19 was measured using the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument that measures progression of disability in patients with ALS.1 The post-hoc analysis retrospectively mapped Study 19 ALSFRS-R scores to both King’s and MiToS stages to determine whether the changes observed in Study 19 would have been recognized with other staging schemes.
“I am honored to have worked with other leading ALS researchers on this analysis,” said Ammar Al-Chalabi, Ph.D., FRCP, a professor at King’s College London. “The two staging systems complement each other and provide different insights. This analysis shows the usefulness of clinical staging in ALS.”
The King’s ALS clinical staging system assesses anatomical spread of the disease, based on the number of affected regions (bulbar, upper limb and lower limb) and includes advanced stages defined by nutritional or respiratory failure.2,3 The MiToS system assesses the number of functional domains (movement [walking/self-care], swallowing, communicating and breathing) in which there is a loss of independent function.4
About Mitsubishi Tanabe Pharma America, Inc.
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 percent owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. It was established by MTPC to commercialize approved pharmaceutical products in North America. For more information, please visit www.mt-pharma-america.com or follow us on Twitter, Facebook and LinkedIn.
About Mitsubishi Tanabe Pharma Development America, Inc.
The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc. (MTDA) is located in Jersey City, New Jersey. MTDA is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation’s 100 percent-owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. MTDA is dedicated to research and develop innovative pharmaceutical products that address the unmet medical needs of patients. For more information, please visit https://mt-pharma-development-america.com/.
Overview of Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma, which was founded in 1678, has its headquarters in Doshomachi, Osaka, which is the birthplace of Japan’s pharmaceutical industry. With business centered on ethical pharmaceuticals, Mitsubishi Tanabe Pharma is a well-established company with one of the longest histories of pharmaceutical companies in Japan.5 In accordance with the corporate philosophy of “contributing to the healthier lives of people around the world through the creation of pharmaceuticals,” the Company formulated the key concept of Open Up the Future under the Medium-Term Management Plan 2016-2020. Through the discovery of drugs that address unmet medical needs, centered on its priority disease areas — immune-inflammation diseases, diabetes and kidney, central nervous system, and vaccines — Mitsubishi Tanabe Pharma will strive to contribute to the health of patients around the world. MTPC is the parent company of MTPA. For more information, go to https://www.mt-pharma.co.jp/e/.
- Cedarbaum JM, Stambler N, Malta E, et al.TheALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. J Neurol Sci.1999;169(1-2):13-21.
- Roche JC, Rojas-Garcia R, Scott KM, et al. A proposed staging system for amyotrophic lateral sclerosis. Brain. 2012;135:847-52.
- Balendra R, Jones A, Jivraj N, et al. Estimating clinical stage of amyotrophic lateral sclerosis from the ALS functional rating scale. Amyotroph Lateral Scler Frontotemporal Degener. 2014;15:279–84.
- Chiò A, Hammond ER, Mora G, et al. Development and evaluation of a clinical staging system for amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2015;86:38–44.
- Research by TOKYO SHOKO RESEARCH, LTD.