PharmExec Blog

There's New Hustle in Myeloma

Kathy Giusti, CEO of MMRF, Eric Lander, PhD, Founding Director Broad Institute of Massachusetts Institute of Technology and Harvard University, and Margaret A. Hamburg, MD, FDA Commissioner

Kathy Giusti, CEO of MMRF, Eric Lander, PhD, Founding Director Broad Institute of Massachusetts Institute of Technology and Harvard University, and Margaret A. Hamburg, MD, FDA Commissioner

The Multiple Myeloma Research Foundation (MMRF) has launched a new initiative to increase the pace of innovation in myeloma research through faster cures and more efficient, timely trials for patients. Founded in 1998, MMRF is the leading non-profit organization in the myeloma space, dedicating 90 per cent of its funding to medical research, disease awareness and patient advocacy. But the new focus is on forging closer partnerships with pharma and biotech firms as well as major academic research institutions.

The big news is that MMRF has unveiled two new online engagement platforms, or ‘open access gateways,’ to pool vital information that researchers and patients require to speed advances in myeloma research.  The first, MMRF’s Researcher Gateway, will collect comprehensive genomic data and make it accessible to more scientists, with the aim of developed targeted treatments in personalized medicine. The second, The MMRF CoMMunity Gateway, will compile different sub-types of myeloma patients and organize and guide them to trials and experimental treatments that address their particular condition.  The essence here is collaboration, with the MMRF serving as facilitator to get the right information, to the right people, at the right time.

MMRF unveiled the gateways project last week at a sponsored symposium, ‘The Revolution Starts Now’, with 200 doctors, researchers, scientists and philanthropists at the Alexandria Life Sciences Center in New York. The founder and CEO of MMRF, Kathy Giusti, presented these initiatives along with an inside look at the organization’s CoMMpass? study, started in 2011, which tracks the progress of 1,000 patients from diagnosis through treatment.  The study will serve as a data reserve and resource to drive the agenda of the Researcher Gateway project.   Margaret Hamburg, Commissioner of the FDA, showed up to endorse MMRF’s work and stressed that innovation is a team sport; smart, creative partnerships with the public sector are also essential in making sure new breakthrough therapies are approved and brought to market, she said.

The Researcher and CoMMunity gateways have already gained core partnership support from MillenniumTakeda;  Onyx Pharmaceuticals; BMS; and Janssen Pharmaceuticals, as well as the following academic research centers:  Washington University in St. Louis; Hackensack University Medical Center; and Mount Sinai School of Medicine. MMRF Chief Operations Officer Walter Capone explained to Pharm Exec that forging this alliance involved all parties agreeing to work toward a common objective.  “Getting all  parties involved to give up intellectual property and patent rights on this data required a consensus around the fundamental premise that no one entity was going to own or control it. Everyone had to accept that the data would be put it out into the public domain for everyone to be able to access.” The Researcher Gateway thus creates a space for companies and academics notorious for shying away from collaboration with each other to share data much more quickly and creatively than they would have on their own.

“The intent was to make the gateway accessible not just to biomathematicians and data-analytical scientists, but also to clinicians who are trying to treat patients and who are looking to see how other centers and other regions might be treating patients and what their results have been,” said Capone. The comprehensive database also allows for the data to be shared readily across different platforms; it can be downloaded and sent to outside parties that need or request it.  “With more traditional platforms, data sharing is not the premise; it’s constructing a database around a particular therapeutic study,” he said. The MMRF is taking a much more inclusive approach that will allow many ideas to seed and take root simultaneously.

The hope is that with such a free flow exchange of ideas and data, new insights and breakthroughs will quickly follow. MMRF has already discovered new targets in mapping genomic data in the myeloma space.

With high hopes come some significant challenges. Coordination between trial sites and academic organizations to rapidly assemble patients may prove tricky due to their decentralized nature. “We’re going to need a good network of information about these patients and where they reside to get them into trials quickly, and to complete these trials on a quicker time scale than any individual company could do on their own,” explained Capone. In addition to patient recruitment, there are issues of sustaining the program with the best possible partners, especially those with deep pockets.

The ultimate goal of the MMRF and its Researcher and CoMMunity Gateways is to facilitate and promote breakthrough research in a disease marked by biological complexity and many different mutations, while developing targeted therapies and companion diagnostic tools to expand the range of treatment options for patients along the way. The basic message here?  The quicker, the better.

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