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Collaborative Effort to Apply Innovative Therapy to Fatal Disease

An interdisciplinary team from the Mailman School’s Department of Biostatistics and the Department of Neurology at CUMC is collaborating with eminent European investigators to design a decisive trial of a promising stem cell transplant therapy for mitochondrial neurogastrointestinal encephalomyopathy (MNGIE), a rare and fatal inherited disease.  The therapy that will be employed is Allogeneic Hematopoietic Stem-Cell Transplantation (AHSCT).

The prevalence of MNGIE is unknown; however, over 100 cases, affecting virtually all ethnic groups, have been identified. Optimism for the application of AHSCT therapy to this disease is based on anecdotal biochemical and clinical improvements in 5 MNGIE patients who experienced successful transplants (although transplants failed to engraft in 5 other patients). 

Recently, Dr. Bruce Levin, Dr. Seamus Thompson, and senior staff associate  Richard Buchsbaum collaborated with leading neurologists to develop and implement an influential adaptive clinical trial design that minimized the number of patients required for a clinical trial for ALS (Lou Gehrig’s disease). They now seek to address devastating neurological disorders which affect even fewer patients. Since there are many such diseases, afflicting many patients, a successful design would represent a significant achievement.  International collaboration often offers the only effective means of accumulating enough cases to address these diseases. Dr. Thompson and his colleagues at the Statistical Analysis Center (SAC), which he directs, and their clinical collaborators are in a key strategic position to develop an approach that might be quite widely applied.

International Collaboration

Drs. Michio Hirano (Neurology, CPMC) and Michael Schüpbach (University Hospital, Bern), the Principal Investigators of the project, have invited Dr. Thompson to present the SAC proposals at a meeting of international investigators organized by Dr. Schüpbach in Bern, Switzerland, on February 19.  His presentation is titled “Possible Protocol Design, Organization, and Data Management for a collaborative trial of aHSCT for MNGIE.”  The meeting will include participants from Switzerland, the UK, Italy, Spain, Israel, Australia, the U.S., and possibly Brazil. If the SAC proposals are accepted, Dr. Thompson and his colleagues will collaborate with the two clinical groups to develop an application for funding to conduct a trial as rapidly as possible. 

Background

• Professor Thompson is Director of the SAC (Statistical Analysis Center) in Biostatistics, which implements innovative randomized clinical trials (RCTs) and other projects using state-of-the-art statistical methods and web-based data management systems.
• Professor Levin is Chair of Biostatistics and, as Senior Design Consultant at the SAC, develops innovative trial designs.
• Richard Buchsbaum, Senior Data Manager at the SAC, designs advanced data management and web-based trial management systems, on which advanced RCTs increasingly depend.
• The U.S. members of the MNGIE collaboration are members of the NAMDC (North American Mitochondrial Disease Consortium), Principal Investigator Dr. Salvatore DiMauro of Neurology, which recently received NIH ARRA funding.