Personalized Medicine Education and Advocacy

Thought leadership in personalized medicine

Of Moonshots and Precision Medicine: Why the Time is Ripe for a National Cancer Moonshot

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by Kenna R. Mills Shaw, Ph.D., Executive Director, Sheikh Khalifa Bin Zayed Al Nahyan Institute for Personalized Cancer Therapy, MD Anderson Cancer Center

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Kenna R. Mills Shaw, Ph.D.

Vice President Joe Biden’s leadership of a national “cancer moonshot” is well-timed both to take advantage of and to enhance the hard-won capabilities of the nation’s cancer-fighting community.

Its proposed strategic investments in precision medicine, cancer immunotherapy, early detection, prevention, genomic analysis, data-sharing and collaboration can have a life-saving impact.

New approaches, based on decades of research, help patients now. The Wall Street Journal recently profiled the experience of a 23-year-old man struck with a particularly hard-to-treat form of acute myeloid leukemia (AML).

The story relates how genomic analysis of his leukemia led to targeted therapy that corralled his disease enough to permit a blood stem cell transplant. When his leukemia returned, his oncologists tried a series of treatments until another genomic analysis pointed to yet another targeted therapy. The result: remission, another stem cell transplant and now a year free of AML.

Such a relay race, moving from one therapy to the next to stay ahead of the disease, has been a hallmark of the treatment of advanced cancers for years. With the tools we have in hand now, we’re poised to ensure that more of these races than ever end with a victory.

How do we do better?

A national cancer moonshot can provide pivotal support and encouragement for focused efforts underway at comprehensive cancer centers and other institutions.

For precision medicine to flourish will take a coordinated research effort to truly understand the impact of genomic and molecular variations in cancer. About 120 genes out of the 21,000 in the human genome have variations that are actionable for cancer treatment. However, we find many more variations when we analyze tumors and most of the time we don’t know what they do.

One hindrance is the common assumption that all variations in a gene do the same thing — result in activation or inactivation of a molecular pathway. Not all mutations are created equally.

For example, a clinical trial underway at MD Anderson features a drug that targets a specific fusion alteration in a gene. Other variations in that same gene don’t necessarily cause malignancy or can render even therapy targeted at the fusion ineffective.

So it’s critical to combine genomic analysis with research in functional genomics, using patient-derived xenograft animal models and cell lines, and discovery genomics. We need to combine research data with clinical data, including treatment response, and to have this not just in a subset of patients, but in all patients.

It’s critical to use our combined research and clinical information in the context of tumor evolution during treatment. We need to change the paradigm of when we test patients’ tumors so it occurs closer to when they need to switch to a new therapy than is routinely the case now.

Multiple biopsies — be they liquid gathered from circulating biomarkers or through fine-needle or core needle approaches — will be needed to guide treatment, and are generally not covered by insurers now.

When MD Anderson established its Moon Shots Program in 2012, 10 platforms were established to support our effort to accelerate the pace of converting scientific discoveries into life-saving advances in treatment, prevention and early detection.

These platforms systematically and efficiently provide expertise, technological capacity and infrastructure to the “moon shots,” multidisciplinary teams of experts that focus on 12 cancer types.

Our areas of emphasis overlap with the priorities chosen for the national cancer moonshot. We’re connecting our clinicians, basic scientists, and professional drug discovery and development teams for innovative translational research and building a powerful infrastructure to combine, share and learn from clinical and research data.

All of this is coming together in innovative clinical trials and practice-changing advances.

The time is ripe for a national cancer moonshot to accelerate the progress that is underway now in institutions across the country.

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