OPT-UP Program and Clinical Study

OPT-UP Program and Clinical Study

 

Are some drugs for treating multiple sclerosis (MS) substantially better than others at slowing, stopping, or even reversing the effects of MS in ways that matter most to people with the disease?

How do the available MS drugs compare in terms of unwanted side effects and safety when administered in the broad population of people with MS?

What information do physicians need to determine which of the drugs available for prescribing to a patient with MS is likely to have the most benefit and fewest adverse effects for that patient?

Why aren’t any drugs approved for slowing, arresting or reversing the relentless decline in abilities that is referred to as progressive MS?

The OPT-UP Program is a unique, US-based, real-world, MS outcomes clinical study and collaborative research program designed to answer these questions and enable an era of optimized MS treatment.

Successfully treating MS today presents promises and challenges. Ten medicines are approved for treating relapsing MS. However, a lack of information about the relative effectiveness of the drugs and a paucity of evidence to support personalized treatment means that MS treatment choices are currently made by trial and error, resulting in suboptimal outcomes for many patients and unnecessary healthcare costs. Furthermore, these medicines have so far not been clearly demonstrated effective for controlling progressive MS, a gradual but unremitting loss of neurological function that eventually occurs in a majority of people with the disease. An inadequate understanding of what drives this progressive slide into disability has substantially impeded the development of therapies to slow, arrest, or reverse it.

The Accelerated Cure Project for MS and a network of MS clinics across the U.S. (the ACP Clinical Effectiveness Study Network) have designed a novel collaborative research program to generate robust evidence that will address these knowledge gaps, enable a transformation of the treatment of relapsing MS and focus R&D efforts on progressive MS. This initiative, the Optimizing Treatment – Understanding Progression (OPT-UP) Program, is built upon the well-established infrastructure and management experience for our unique biobank, the ACP Repository.

A key feature of the OPT-UP program is the role of a Community Advisory Panel, comprised of patients, already established to ensure that patients are engaged in determining the goals of the program and contribute to developing methods for two-way communication of OPT-UP with its patient community.

The OPT-UP Program includes a Clinical Study and a “Path to Impact” vision to ensure that the Study serves to benefit all people with MS. The Clinical Study is unique amongst research endeavors in MS: It is a large-scale, real-world, longitudinal, multi-center, clinical and fundamental research enterprise that encompasses treatment outcome measures that are important to both physicians and people with MS.

The Clinical Study will collect biosamples and data (e.g., clinical measures and patient-reported outcomes) from 3,000 people with MS receiving treatments in real-world clinic practices, rather than in the tightly controlled setting of a randomized clinical trial. Subsequently, in the Path to Impact, samples and data will be rigorously analyzed by Program Partners to (a) compare the effectiveness, side effects and safety of different MS drugs in a real-world setting, b) find predictors and early indicators of response to particular drugs, and (c) detect biomarkers specifically associated with mechanisms of progressive MS.

The time is right to launch this coordinated, large-scale effort to bring about a revolution in the treatment of MS. The cost of not initiating such an effort is enormous for people with MS, for the wider community touched by the disease and for the healthcare system.

ACP Clinical Effectiveness Study Network / OPT-UP Members

R. Phillip Kinkel,* MD, Beth Israel Deaconess Medical Center, Boston, MA
Peter Riskind,* MD, University of Massachusetts Medical School, Worcester, MA
Saud Sadiq, MD, James Stark, MD, Multiple Sclerosis Research Center of New York, New York, NY
Arun Venkatesan, MD, Johns Hopkins School of Medicine, Baltimore, MD
Michael Racke, MD, Aaron Boster, MD, Ohio State University Medical Center, Columbus, OH
Benjamin Thrower, MD, Deborah Backus, PT, PhD, Shepherd Center, Inc., Atlanta, GA
Elliot Frohman, MD, PhD, Benjamin Greenberg,* MD, MHS, University of Texas Southwestern, Dallas, TX
Augusto Miravalle, MD, Timothy Vollmer,* MD, Jonathan Campbell, PhD, Kavita Nair, PhD,
University of Colorado, Aurora, CO
Lawrence Steinman, MD, Jeffrey Dunn, MD, FAAN, Angela Jean Campbell,
Stanford University School of Medicine, Palo Alto, CA
Carolyn E. Schwartz,* Sc.D., DeltaQuest Foundation, Inc.
Robert McBurney, PhD, Hollie Schmidt, MS, Sara Loud, MS, Accelerated Cure Project for MS, Waltham, MA
*OPT-UP Leadership Team