November 25, 2016
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Missed research opportunities, real-time data sharing remain key obstacles for cancer moonshot

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The cancer research and clinical communities owe a great deal of gratitude to Vice President Joe Biden and his national cancer moonshot initiative.

This has been a very exciting and inspiring development in the fight against cancer, most notably because of the attention the issues are getting on the national stage through the White House, and with Biden as champion.

Key strategies and solutions are emerging. We held a successful HHS–designated regional cancer moonshot summit at Fox Chase Cancer Center on June 29 that covered important topics, including basic research, big data and precision oncology, as well as patient issues like clinical trials.

Wafik S. El-Deiry, MD, PhD, FACP
Wafik S. El-Deiry

After Biden’s telecast comments, Bert Vogelstein, MD, of Johns Hopkins Medicine in Baltimore, spoke about the importance of cancer prevention. Panelists discussed the uncertain state of support for basic research, while the need for collaboration and data sharing was unquestioned in the era of precision medicine.

Improved clinical trial availability and enrollment remain high priorities. Reports from the NCI blue ribbon panel and the report from Biden highlight strategies for accelerating progress, and they have been met with widespread enthusiasm.

‘Support the soldiers’

Key challenges remain that must be addressed if the community is to achieve the goals of the cancer moonshot effort.

How research is supported in the United States has been approaching the state of a lottery, with numerous missed opportunities in unfunded grants. The NCI, American Cancer Society, American Association for Cancer Research, ASCO, Stand Up To Cancer and many other private foundations have processes in place to support basic and clinical research, but these are greatly limited by resources.

With less than a 10% success rate in funding, most scientists have been spending much of their time writing grant proposals. This incredible waste of talent and effort does not translate into an opportunity to push the field forward due to lack of resources.

Many advances have impacted patients’ lives, and they have come from existing pathways for discovery and translation. However, numerous great ideas are not being pursued due to limited funds. There is a danger with reinventing the wheel or developing too many special interests.

As I learned in an NIH study section meeting nearly 20 years ago, we need to support the soldiers in the war — those principal investigators whose data and ideas are at the forefront of innovation and discovery. If we do not, certain lines of investigation come to a halt often for many years.

It is important to remember to support the soldiers, not just the superstars. The grassroots innovative ideas emerge from the creative scientific process and from clinical experience that allow the national effort to cover the waterfront in basic and translational/clinical research. Clinicians and scientists are by their very nature collaborative and keep up with the latest advances and technologies.

We need to pay attention to the underlying systemic problems if we are to have a successful shot at accelerating progress in the war on cancer. We need the best and brightest to go into science and to thrive. We need to empower the scientific community broadly within the moonshot effort, and we need to support the institutions that are in place — such as NCI, among others — with more meaningful and sustained resources so they can do what they do well.

Data sharing

Another comment worth making is that data sharing — especially of clinical outcomes from clinical trials with new agents — is very challenging to accomplish in real time.

The promise of precision medicine is that a molecular profile of a new patient’s tumor shows up as a match — as a potential responder — for an ongoing trial with novel therapy, and that patient gets directed to that clinical trial.

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However, beyond some known cancer drivers and available inhibitors — and trials like MATCH and TAPUR — we are still very far from that, and very far from developing a national database that has molecular profiles and clinical outcomes tied to specific therapeutics. This is a major challenge for any one institution, let alone the variety of testing platforms.

The plethora and diversity of therapeutics — including immunotherapy — only add to the complexity, as do privacy issues and informed consent. There must be a willingness to share openly with privacy protections, and perhaps more incentives other than punitive measures. The value of “big data” needs to be demonstrated and used in the context of specific patients to address the use — and cost — of promising therapeutics.

I look forward to a day when molecular profiling, whether from tumor or blood, can inform the best therapy option with expected precisely calculated evidence-based outcomes for a specific patient, and with a much greater armamentarium of therapies, including for resistant disease.

For more information:

Wafik S. El-Deiry, MD, PhD, FACP, is deputy director for translational research at Fox Chase Cancer Center. He also is a HemOnc Today Editorial Board member. He can be reached at wafik.eldeiry@gmail.com.

Disclosure: El-Deiry reports no relevant financial disclosures.