Fifty years ago, President Nixon signed the National Cancer Act into law to restructure the country’s way of dealing with a leading cause of death and suffering. Cancer cases were increasing, and a new approach to fight it was needed. The Act set forth a National Cancer Program to strengthen the role of the federal government in researching and treating cancer and helped establish the network of cancer centers that still spearheads major research efforts around the country. It was an unparalleled effort to galvanize the medical community, but it would take a sustained commitment to achieve success against the rising cancer burden.
To commemorate the 25th Anniversary of the Act, a non-profit organization named Friends of Cancer Research was formed to re-engage policymakers and scientists and recommit to fighting cancer and other diseases as a national priority — starting with efforts to double the annual federal budget for medical research. While progress was being made, it still usually took well over a decade for new discoveries in research labs to be transformed into treatments for patients, most of whom can’t wait.
Since the initial Act was passed in 1971, scientists have learned that cancer is a collection of hundreds of disease subsets, which are driven and defined by diverse biological processes and genetic alterations. The ability to understand the scientific underpinnings of different cancers has led to marked achievements and new ways to detect and combat various cancers.
New drugs typically go through a series of stepwise — sometimes lengthy — research studies to progressively characterize and review their safety and efficacy. But by the early 2000s, it was increasingly clear that cancer medicines were changing. Several new drugs could be designed to target specific aspects of a tumor, or even stimulate a patient’s immune system to help fight off the cancerous cells. This new approach to drug design was yielding useful results much earlier in the discovery process. As treatments became more effective, it would again take new methods to leverage the opportunities to get transformative medicines to patients in a timely manner.
In 2010, Friends of Cancer Research brought together a team of experts from universities, government, industry, and patient advocacy to develop evidence-based policies to pave the way for future discoveries. Recognizing this emerging shift to cancer drug development, a novel approach aimed to compress traditional research processes was proposed. To rapidly develop these transformative medicines for patients in need, the new model would require a level of collaboration, coordination, and interactions between developers and regulators not previously seen in drug discovery. This work shed light on historic policy impediments in drug development, so Friends of Cancer Research worked with Congressional and federal leaders to develop the Breakthrough Therapy Designation — a 2012 law that identifies the most promising new treatments and sets them on an expedited trajectory to reach patients who need them most.
Since its inception, this policy has reduced the development time for new breakthroughs by approximately three years, representing meaningful access to patients that had no other available treatments. Such game-changing policies came to be through the collaboration and commitment of all sectors to do things differently.
By uniting experts from universities, government, industry, and patient advocacy, we can develop evidence-based policies to pave the way for future discoveries, but we can also accelerate the pace of scientific progress. An example of this is working to rapidly understand the use of biologic measures, such as circulating tumor DNA (ctDNA), which is genetic material from cancer cells naturally found in the bloodstream. Exploratory studies have shown great potential for using ctDNA changes to signal whether a drug is working, but initial studies were conducted independently or using different methods, leaving questions about the applicability until further research is done.
To expedite the process, we gathered diverse scientific leaders to design a comprehensive plan to first leverage prior studies and determine whether ctDNA can better identify potentially effective new treatments and learn whether patients were responding to their therapy — sooner than current tools can. This collaborative approach to research and data sharing is providing more information in a larger dataset, using advanced statistical analyses to optimally characterize the outcomes, and producing results faster than a company or organization could if working independently. The project is called ctMoniTR (ctDNA for Monitoring Treatment Response), and it has now grown to include more than 25 studies representing more than 3,000 cancer patients, 16 different treatments, and several advanced cancer types.
By lowering barriers and working together toward common goals, we have shown that progress can be made much faster. This is a paradigm shift to bring treatments more efficiently to those that need them.