What if choosing the very best treatment for any given cancer were as routine as choosing the right kind of antibiotic to treat a common infection?
Kathy Giusti, founder of the Multiple Myeloma Research Foundation (MMRF), and a cancer patient herself, has made it her personal mission. She foresees the day when an individual’s medical care will be tailored in part based on each person’s genetic make-up and other unique characteristics of his or her disease.
“The days of a one-size fits all approach to cancer treatment are over. We can do better than that,” Giusti said.
The kind of precision oncology for which Giusti advocates has already proven lifesaving for some. Patients with many forms of cancer, for instance, undergo molecular testing as a standard part of their care, enabling physicians to select treatments based on this information.
“Armed with focused genetic and molecular information about their patient’s distinct disease, physicians can develop personalized treatment strategies designed for maximum effectiveness,” she said.
But making this routine, Giusti says, requires innovative research models that prize and data-sharing and collaboration to make unprecedented advancements, an approach for which the MMRF has become widely recognized. The MMRF, by working closely with partners in the biotech and pharmaceutical industries, academia, and the cancer community, injects speed and efficiency into the process of discovering and developing new drugs for the treatment of multiple myeloma. Through this collective approach, myeloma patients now have 10 new treatments – 4 treatments FDA approved in 2015 alone – that have almost tripled their survival and given thousands of others living with this incurable disease a fighting chance.
Now, Giusti believes this same collaborative approach can be used to usher in a new era of precision medicine and its promise of a cure: the MMRF has pioneered an end-to-end solution specifically focused on precision medicine in oncology.
“We generate the data, we share and aggregate the data, we analyze the data, and draw insights from these data to develop optimal treatment strategies for patients,” she said.
At the core of the model is a robust data ecosystem, including the first multicenter multiple myeloma tissue bank. This endeavor allowed the MMRF, along with its best-in-class partners, to sequence the myeloma genome. The MMRF also launched the expansive, comprehensive MMRF CoMMpass™ (Relating Clinical Outcomes in Multiple Myeloma to Personal Assessment of Genetic Profile) study, which has performed whole genome sequencing on over 1,000 patients and generated the largest longitudinal genomic dataset in myeloma in the public domain. The MMRF then collaborated with partners like the Translational Genomics Research Institute (TGen) and the Broad Institute of MIT and Harvard, to uncover deeper insights into the disease’s biology, such as BRAF, a target previously never linked to myeloma but for which treatments have already been approved for melanoma or are now in clinical trials.
“Studying a large number of people – deeply and over time – allowed us to ask and answer new questions that were never before possible,” Giusti explained.
Today, through CoMMpass study, the MMRF is tracking the progress of these same 1,000+ patients for at least 8 years, analyzing their genetic and clinical data to better understand how certain factors influence response to treatment and disease progression and to generate hypotheses for genomically informed clinical trials. Most importantly, these data are then openly and freely shared through a global learning network, so that brilliant minds from all corners of the scientific community—including experts at the big data analytics company GNS Healthcare—can collaboratively decipher patterns for further exploration. This network was built to democratize data, allowing researchers and clinicians worldwide to share and contribute to a growing knowledge base that can be used to generate hypotheses for clinical trials and rapidly explore new and potentially more effective ways of treating myeloma.
Any findings can be immediately advanced into the MMRF’s worldwide network of research institutions and cancer centers that are working collectively to test an arsenal of new drugs and combination therapies. More than 30 new therapies have been in clinical trials in the network, including genomically-informed treatments – that is, those that target specific alterations in an individual that give rise to cancer or promote its spread – and immunotherapies, like checkpoint inhibitors and immunostimulatory antibodies, which are drugs that make use of a patient’s own immune system to fight cancer.
The MMRF’s model also includes a community of patients, who Giusti says are “every bit a part of curing the disease as scientists”.
These patients are stepping up to share knowledge they have gained on their cancer journeys with fellow patients—including sequencing data, when available—so they can learn which type of myeloma they have and share their data to advance research.
“We consider these patients as partners in our search for better, more targeted treatments,” said Giusti.
“As these patients, eventually, have their genomes sequenced and learn which, if any, mutations they have, can connect with other patients, engage with researchers who specialize in that particular area of genomics, and sign up for clinical trials that might be appropriate for them,” said Giusti.
Giusti will share more details of the MMRF’s innovative end-to-end solution for precision medicine as she sits down on April 26th for a fireside chat with Dr. Nancy Snyderman to discuss “Patients Driving Innovation” at the 2016 World Medical Innovation Forum, hosted by Partners Healthcare in Boston.
The World Medical Innovation Forum is a global gathering of more than 1,100 senior health care leaders hosted by Partners HealthCare in the heart of Boston. It was established to respond to the intensifying transformation of health care and its impact on innovation. The Forum is rooted in the belief that no matter the magnitude of that change, the center of health care needs to be a shared, fundamental commitment to collaborative innovation – industry and academia working together and its ability to improve patient lives.