The Hematologist

November-December 2015, Volume 12, Issue 6

Leading the Charge for Precision Medicine

David A. Williams, MD Chief of Hematology/Oncology; Director of Clinical and Translational Research
Boston Children's Hospital, Boston, MA

Published on: October 19, 2015

Nearly nine months ago, in February 2015, I made a statement on behalf of ASH, applauding President Obama’s funding proposal for the National Institutes of Health (NIH) as well as his unveiling of the Precision Medicine Initiative (PMI; www.hematology.org/Newsroom/Press-Releases/2014/3645.aspx). The administration proposed $215 million in investments among the NIH, the National Cancer Institute (NCI), U.S. Food and Drug Administration, and the Office of the National Coordinator for Health Information Technology, supporting projects including research, development of new databases, innovations in data collection and sharing, and creation of new requirements and standards. It was also in 2015 that ASH defined a new set of research priorities, and the areas of genomic profiling and chemical biology are, quite literally, at the top of the agenda (www.hematology.org/ResearchAgenda).

Customized approaches to treatment have been around for some time, and hematologists have made many contributions using patient- and tumor-specific information to prevent, diagnose, and treat diseases. However, ASH’s sense of urgency is reflected in the development of the new ASH Research Agenda; renewed commitment to research funding at the federal level; advances in genome sequencing, bioinformatic analysis in the nascent era of big data; and other areas that will have critical implications for the future of informed, personalized clinical care. This year, ASH took direct action to further define the scope of our role in this crucial area, establishing the Task Force on Precision Medicine, whose chief priorities include exploring ways in which ASH can work directly with the NIH and other entities to initiate and address gaps within genomically defined, precision medicine trials. The ultimate goal of the Task Force is to make sure that the opportunities for both malignant and nonmalignant hematology are fully explored, working in an advisory capacity to help the Society best realize the promise of precision medicine in patient care.

Interest in precision medicine goes well beyond the new task force. The 57th ASH Annual Meeting in Orlando next month will offer many opportunities to tap into some of the most fascinating breakthroughs in genomics and to learn how genomic data translates into information that is clinically meaningful. To spotlight just a few of these opportunities, Dr. Jill Johnsen will chair the Education Session “Guiding Hematologic Care with Genetic Testing,” and Dr. Charles Mullighan will lead the Special Scientific Symposium “Precision Medicine in Cancer Therapy.” Dr. Louis Staudt of NCI will conduct a highly interactive Meet the Scientist session, answering questions on many aspects of precision medicine as well as discussing where the field is headed. Abstracts and talks will describe how specific disease-causing molecular defects in both malignant and nonmalignant conditions are being targeted by genetic approaches and with drugs that have surgical precision rather than using the blunt instruments of the past. It is so exciting to witness what seems like the tipping point in bringing these treatments to patients.

Serving as ASH President during such an eventful and auspicious time for the field has been an honor. In 2015, ASH made major strides in developing new clinical guidelines, in continuing to evolve as a global society, and redefining our leadership in sickle cell disease. And as part of an ongoing effort to support access to care and treatment, and related legislative goals, ASH targeted its advocacy efforts by working with Congress to address Medicare reimbursement and supporting the Cancer Drug Coverage Parity Act, which was introduced in Congress in June. ASH will continue to push for these legislative priorities at both the state and federal levels. As always, we made many many lobbying trips to Congress to advocate for additional NIH research funding.

Looking ahead to 2016, many research and patient care-related agenda items lie before us that require ASH’s advocacy. I look forward to passing the gavel to Dr. Charles Abrams in Orlando, and welcome his leadership in the months ahead.

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