By Kathy Giusti
Published July 25, 2015, Volume 6, Issue 13
We’ve seen how dramatically patients’ lives can change when they are matched with the right treatment at the right time in their disease course. Although this is still an exception and not the rule, we believe collaborative research approaches will make this kind of precision medicine a reality for all patients with cancers.
At the Multiple Myeloma Research Foundation (MMRF), collaboration is not just a priority; it is the only way we do things. We’ve worked shoulder to shoulder with the best partners in the biotech and pharmaceutical industries, academia, and the cancer community to inject speed and efficiency into the process of discovering and developing new drugs for the treatment of multiple myeloma. Through this collective approach, we’ve seen the development of seven new treatments—breakthrough innovations that have almost tripled patients’ lifespan and have given patients such as journalist Tom Brokaw and thousands of others living with this incurable disease a fighting chance. Now, we believe this same collaborative approach can be used to usher in a new era of precision medicine and its promise of a cure.
The Genomics of Myeloma
We began by building a robust data ecosystem, including the first multicenter multiple myeloma tissue bank. This allowed us to sequence the myeloma genome and then launch the MMRF CoMMpass (Relating Clinical Outcomes in Multiple Myeloma to Personal Assessment of Genetic Profile) study, the largest long-term genomic research study ever conducted in myeloma to have data outputs placed in the public domain. Collaborating with key partners like the Translational Genomics Research Institute (TGen) and the Broad Institute of MIT and Harvard, we uncovered deeper insights into the disease’s biology, which revealed important new drug targets, like BRAF, previously never linked to myeloma but for which treatments have already been approved for melanoma or are now in clinical trials.
Today, through the CoMMpass study, our team is tracking the progress of 1,000 patients for at least 5 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. We then openly and freely shared these data through a global learning network, so brilliant minds from all corners of the scientific community—such as 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.
At the same time, we have built a worldwide network of research institutions and cancer centers that are working collectively to test an arsenal of new drugs and combination therapies. So far, 30 new therapies have been in clinical trials within our network, including immunotherapies, targeted therapies, and drugs that work in entirely new ways to stop cancer in its tracks. We are also working closely with partners in the pharmaceutical and biotech industries and regulatory officials to leverage new clinical trial designs to speed these new treatments to patients.
Partnering With Patients
Finally, we are building a massive community of patients, who 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.
Our future hope is that these same patients, as they 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.
Making Precision Medicine a National Priority
This approach is so promising to advancing more effective treatments and cures for diseases like cancer that President Barack Obama has made precision medicine a national priority, and it is an honor to serve on the President’s advisory board for the groundbreaking Precision Medicine Initiative. Launched with a $215 million investment in the President’s 2016 budget, the Precision Medicine Initiative aims to accelerate biomedical discoveries and provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for individual patients. The near-term goal is to create more and better treatments for cancer. In the longer term, the project would provide information on how to individualize treatment for a range of diseases.
At the heart of the program is the national study involving the health records and DNA of 1 million volunteers to determine how peoples’ genes, environment, and lifestyle affect their health. The $130 million study will recruit new volunteers as well as merge data from more than 200 large ongoing American health studies, together involving at least 2 million people. The investment also allocates $70 million for DNA-driven research on cancer and another $10 million for the U.S. Food and Drug Administration to improve the regulation of genome tests.
These programs speak to the incredible power of working collaboratively and adapting and maximizing the use of new technologies to ensure continued progress. Only then will the promise of personalized cancer care become a reality—not years from now but today. We look forward to drawing insights from one another, as we work urgently toward a cure. ■