More than half a million individuals die of cancer and more than 1.5 million new cases are diagnosed in the US each year. Worldwide, trends suggest that death and suffering from cancer will increase sharply over the years to come. Current standards of care typically offer two options to patients with advanced cancer—enrollment in a clinical study—and palliative care, including hospice—neither of which takes maximum advantage of our increased understanding of the disease and the rapid emergence of new technologies. What if there were a third option?: a “last resort clinic” (LaRC) in which clinicians and bench scientists around the world collaborate intensively, in some cases remotely, and on a case-by-case basis to help save terminally ill patients, one at a time. This clinic would use the most advanced therapies, some of which are so experimental that they are based on as-yet-unpublished research and are not yet part of any clinical trial. Such therapies do exist, but the LaRC does not. Not yet.

The goal of this working group is to explore the possibility of creating a LaRC by conducting applied research on the medical, financial, business, and legal challenges involved in launching and sustaining such an endeavor. The end product of this working group will be a business plan and/or supporting research documentation for a LaRC (or why such a business is not feasible). This end product will be placed in the public domain and any LaRC Working Group participant will be free to use this information in any way. There is no obligation or commitment of Working Group members after the completion of this task.

For concreteness, we propose to focus on a clinic specializing in providing beyond standard-of-care cancer treatments. These treatments may include the use of off-label drugs and other therapeutics generally regarded as safe in select patients based on an understanding of the unique biology driving each individual’s tumor, the treatment’s mechanism of action, and available human or animal efficacy data. The clinic will be staffed by a multidisciplinary team of oncologists, other doctors, and biomedical cancer scientists who can bring their collective expertise to each case. In addition, experts from outside the clinic, including those with expertise in drug repurposing, will be engaged on a regular basis. In doing so, the clinic will open new choices for patient care and contribute to medical innovation as treatment data is mined and shared.