The Obama administration's "cancer moonshot" took a major step forward Wednesday when a committee of top cancer researchers and patient advocates recommended an ambitious set of scientific goals designed to sharply accelerate progress against the disease.
The 10 ideas, which were developed by a blue-ribbon panel, deal with an array of topics, including research, clinical trials, data mining, prevention and patient engagement. The recommendations were sent to a task force headed by Vice President Biden, who is leading the administration's moonshot campaign.
Biden's son, Beau, died of brain cancer in May 2015. In January, President Obama announced the cancer initiative and placed Biden in charge. Since then, the vice president has crisscrossed the country talking to physicians, researchers and patients about ways to accomplish his mission — to make a decade's worth of progress against the disease in five years.
The new recommendations represent areas that the experts concluded hold promise and are poised for relatively rapid progress. One of the most significant initiatives calls for the creation of a national network that would allow patients across the country to have their tumors profiled. Such tests, which include genetic analysis, can provide oncologists with important information about how to treat specific malignancies.
Another recommendation would create a clinical trial network dedicated to immunotherapy, which uses the immune system to fight cancer and is revolutionizing the treatment of some types of the disease. Yet another calls for the increased testing for hereditary cancer syndromes for certain patients and their families. These syndromes can increase the risk for breast, colon and other cancers.
The panel's report, which was accepted by the National Cancer Advisory Board and Douglas Lowy, acting director of the National Cancer Institute, was forwarded to a moonshot task forced headed by Biden and made up of almost two dozen officials from federal agencies and departments. The task force is working on a comprehensive report, due out next month, about what the government can do to make further gains against cancer.
The panel's report comes at a time of uncertainty. Federal officials are well aware that there is still skepticism about the moonshot effort in many parts of the science community. Moreover, many of the ideas highlighted Wednesday would require millions — if not hundreds of millions — of dollars in additional funding, but Congress has not yet approved any extra spending specifically for the moonshot.
Supporters hope the list of recommendations will help win lawmakers' financial backing. "We can now take these 10 recommendations to Capitol Hill to try to inspire support for the cancer moonshot," said Jon Retzlaff, managing director for science policy and government affairs at the American Association for Cancer Research. "It's important to have something of substance we can show, and today we have it."
The 28-member panel set up several working groups that had some 150 experts concentrating on various topics, such as pediatric cancer, enhanced data sharing and clinical trials.
In recommending a new network for tumor profiling, the committee said such a move would allow patients to contribute to researchers' understanding of the disease. It also would allow patients to "preregister" for appropriate studies based on their tumors' characteristics. The report added that if patients were willing to provide serial biopsies at "clinical inflection points," such as when tumors become resistant to treatment, it could give researchers a better understanding of the disease.
George Demetri, co-director of the Ludwig Center at Harvard University and a member of the working group that proposed the tumor profiling, said the network could be "transformational" in cancer treatment. He said the approach could result in patients in community oncology practices, which is where many patients are cared for, getting the kind of cutting-edge treatment provided at big cancer centers.
The report also said that a clinical trials network for immunotherapy could help increase the cure rates, as well as lead to new vaccines to prevent a range of cancers. Immunotherapy is showing stunning success in treating some hard-to-treat cancers, but it only works in a minority of patients. The goal is to understand "why it works when it works, why it fails when it fails and what can be done to increase its efficiency," said one of the chairs of the panel, Tyler Jacks, director of the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology.
In another recommendation, the panel called for a redoubling of efforts to understand why cancer treatments often stop working over time, an effort aimed at reducing recurrent cancer.
The panel also proposed several strategies to prevent cancer or detect cancer in its earliest stages. It called for finding new ways to further reduce tobacco use and increase vaccination against the human papillomavirus, which causes several cancers. It also endorsed greater testing for hereditary cancer, including for patients with colon cancer. A condition called Lynch syndrome, which genetically predisposes people to colon cancer, causes an estimated 7,000 cases a year. But, the report said, few patients are tested for the mutation, and so their family members are not alerted to the potential vulnerabilities.
Other panel recommendations included increased research into rogue proteins, called "fusion proteins," that drive many cancers in children; the creation of 3-D "tumor atlases" to document how various malignancies change over time, and the development of new technologies to treat cancer, including implantable devices that deliver micro-doses of different treatments directly to tumors to see which work best.
Besides Jacks, the other co-chairs of the blue-ribbon panel were Elizabeth Jaffee, deputy director for translational research at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University School of Medicine, and Dinah Singer, NCI's acting deputy director.
Ellen Sigal, a member of the panel who is also the founder and chair of the nonprofit Friends of Cancer Research, said she was proud of the report. "Often these scientific reports don't come to the patient level, and this one does," she said. "I think this is a very patient-centric report, and that’s important to me."
The report said that the panel had forwarded several important policy issues outside its scope to Biden's moonshot task force. Those issues include coverage and reimbursement of cancer treatment; the fragmentation of the health-care delivery system; incentives to encourage the development of drugs for children and barriers to data sharing.
Besides the moonshot task force report, Biden will provide President Obama with an executive report outlining his vision for the future of cancer research, prevention and care, according to Greg Simon, who is executive director of the moonshot task force.