Researchers Begin Immunotherapy Study on Rare Cancers

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Researchers from UC San Diego and Northwestern University will collaborate with the Southwest Oncology Group in a study of immunotherapy for rare cancer patients. The study, funded by the National Cancer Institute, began enrolling participants from all over the country on Jan. 24.

Officially called the “Dual Anti-CTLA-4 and Anti-PD-1 Blockade in Rare Tumors” or DART, the trial will investigate the efficacy of treating rare cancers of the nerves, skin, glands and bones with the monoclonal antibodies nivolumab and ipilimumab. The idea of immunotherapy is to introduce these antibodies into the immune systems of cancer patients and promote their bodies’ own abilities to stop the growth of tumor cells.

A combination of the two drugs, Anti-CTLA-4 and Anti-PD-1, will be given in six-week cycles, with changes in tumor size being tracked through computerized tomography scans, an imaging technique that utilizes X-ray technology to visualize the inside of the body. In addition to treatment, a basic science team will study the tumor tissue samples to understand responses to the drug as well as potential biomarkers of treatment response.

For The Rare Cancer Research Foundation, this federally-funded clinical trial represents a hopeful shift toward more awareness and research into rare cancer treatment.

“It’s refreshing to see rare cancer patients have the option to join a significant trial,” the foundation said in an email to the UCSD Guardian. “Rare cancers are often ignored as being a ‘niche’ problem, yet they are responsible for 25 percent of new cancer diagnoses and over 40 percent of cancer deaths in the U.S. Having significant clinical trials made available to the rare cancer patient community is a big step in the right direction.”

Dr. Sandip Patel, one of DART’s primary investigators and an assistant director of the Clinical Trials Office at UCSD’s Moores Cancer Center, finds both the basic research and clinical care aspects of the trial to be very exciting and novel.

“The ability to help those patients that I see in clinic that have historically not had clinical trial options … is the most rewarding thing,” Patel told the Guardian. “At a broader level, our hope is that this allows for more clinical trials to be done in rare tumors and especially in immunotherapeutics — [UCSD is going to be integrated….] on the scientific aspect [of studying the tumors to better]… understand the patient’s cancer and immune response to these therapies … I’m excited for both the bench and bedside components of this protocol.”

Dr. Razelle Kurzrock, the co-principle investigator of the DART study, also runs a rare tumor clinic out of UCSD’s Moores Cancer Center. She gave insight into the struggles faced by patients with rare tumors who often have few resources for treatment.

“For those patients [with rare tumors], there are not a lot of options — no FDA approved therapy or even a clinical trial — because most clinical trials are aimed at common tumors like breast, lung and so forth,” Kurzrock explained to the Guardian. “That is why we started the rare tumor clinic … because cumulatively there are a lot of patients suffering and have nowhere to turn to.”

This lack of basic research into rare tumors is being addressed by the NCI, one of the National Institutes of Health’s many divisions. Dr. Elad Sharon, a senior investigator in the institute’s Investigational Drug Branch that works on immunotherapy drug development, described the role the NCI plays in funding these trials all across the country.

“We give money to groups like [the Southwest Oncology Group] … or sometimes specifically to institutions like UCSD,” Sharon told the Guardian. “The NCI tries to complement what industry does on its own …[which] tends to focus on common cancers. There are different incentives that the government tries to use to get them to work in rarer tumors, but a lot of the work on rare tumors ends up coming from the NCI.”

Sharon also explained how the recruitment process of this study will draw from NCI-MATCH, a clinical trial that analyzes patient tumors for gene abnormalities that have drugs that can target them. Participants with rare cancers are hard to find by virtue of having rare tumors; DART aims to enroll 300 participants in its trial. Participants who were not eligible for NCI-MATCH but have a rare cancer specified in the protocol can also enroll, allowing greater access to clinical trials and potential treatments they did not have before.