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By Real World Health Care Editorial Staff  |  Aug 5, 2020

Multiple Myeloma Research Foundation: CAR-T Cell Therapy Initiatives

Last year, the Multiple Myeloma Research Foundation (MMRF) launched a new, three-year strategic plan to help transform how multiple myeloma is treated and ultimately cured. One of the plan’s three strategic pillars focuses on immunotherapy, including chimeric antigen receptor T-cell (CAR-T) therapy.

Real World Health Care spoke with the MMRF’s Anne Quinn Young, MPH, Chief Marketing and Development Officer, and Hearn Jay Cho, MD, PhD, Chief Medical Officer, about the potential of CAR-T therapy and how the MMRF is supporting research on individualized, targeted therapies for multiple myeloma patients.

Building Partnerships for CAR-T Therapy Innovation

Real World Health Care: How does the MMRF support CAR-T research?

Anne Quinn Young

Anne Quinn Young, Multiple Myeloma Research Foundation

Anne Quinn Young: The MMRF is a bit different than other groups. From the very beginning, we’ve focused first on our strategic plan and then used that strategic plan to guide our scientific plan.

One of our initial tasks was to bring together all the critical players in the multiple myeloma ecosystem—researchers, clinicians, industry and the FDA—and through a series of topic-focused roundtables and workshops, create solution-driven action plans.

Our goal is to bring new, more precise treatments to patients faster, and we’ve implemented several important foundational elements to help us reach that goal. We established a multi-center tissue bank at the Mayo Clinic in Scottsdale, Arizona, from which samples were used to sequence a myeloma genome. We’ve helped to open more than 80 trials across a clinical network of more than 24 sites. And, we have a longitudinal study called CoMMpass, in which we’ve been collecting genomic and clinical data, and are now expanding to include immune data as well. This data and the tissue bank samples are huge assets.

Filling the ‘White Space’ in CAR-T Therapy Research

RWHC: What are some of the challenges MMRF faces in moving your CAR-T therapy research agenda forward?

Hearn Jay Cho

Hearn Jay Cho, Multiple Myeloma Research Foundation

Hearn Cho: There’s a bit of a paradox in CAR-T therapy research. We’re seeing unprecedented initial response rates, but progression-free survival has fallen short of expectations. Now, if we were talking about any other type of therapy that gave 12 months of progression-free survival, we would celebrate. But because it’s CAR-T therapy, it’s somehow viewed as disappointing.

CAR-T therapy research is unique because the capital costs involved in T cell engineering and trials are much higher than with standard drug or antibody trials. Although CAR-T therapies are born in academia, the transition to clinical trials requires the assets of industry, which is shouldering most of the costs. The MMRF respects that dynamic and strives to not be redundant in the trials we support. Instead, we strive to fill the “white space” in the clinical trial landscape.

One of those white spaces is mechanisms of action and resistance and understanding how an individual’s immune system and tumor state affects their outcomes. Also, because each CAR-T cell is unique in terms of its target, signaling domains and composition, each is a class of drugs unto itself. Understanding how these agents act in a different disease, under different circumstances, with different targets is essential.

Consortium Focuses on Collaborative Research

RWHC: Tell us about how MMRF is bringing the multiple myeloma community together to address some of these issues.

HC: MMRF has established the Multiple Myeloma Research Consortium (MMRC) to bring together academic and community cancer centers with industry, to advance innovative Phase I and Phase II clinical trials.

MMRF also is making an enormous investment in a project called the Immune Atlas, a gold-standard immune profiling platform for myeloma research and clinical studies. We are utilizing the assets from our collective tissue bank and CoMMpass samples and partnering with five leading academic centers from our MMRC, which will employ cutting-edge analytical technologies to comprehensively characterize the immune repertoire and activity of key immune cell populations in myeloma patients. We expect the platform to generate robust immune data that drives future research and eventually enables clinicians to customize treatments and therapies that will work best with individuals’ immune systems.

AQY: We are trying to get to the point where we can identify subgroups of patients and better predict which treatments they will benefit from. Correlative studies and translational research—two areas in which MMRF excels—are important to achieving this end. We know that CAR-T therapy has a place for some patients. Now we need to identify who those patients are. We also need to consider the patients who lie outside of the typical response rates. Do we focus on making the treatment better, or do we just not treat those patients who aren’t likely to benefit?

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