Improving Patient Outcomes in Rare Cancers
As their name implies, awareness of rare cancers is lower compared to cancers that are more common. In turn, research funding to improve patient outcomes with rare cancers is also lower. Donations to general cancer funds, such as Garding Against Cancer, allow the UW Carbone Cancer Center’s director, Dr. Howard Bailey, to provide funds for areas of greatest need – including underfunded rare diseases.
Neuroendocrine tumors (NETs) are a rare form of cancer, representing only around one-half of one percent of all newly diagnosed cancers, though their incidence is rising. Current therapies mostly help with long-term disease management but are not curative. A promising clinical trial to treat patients with NETs, funded in part by Garding Against Cancer and The Aly Wolff Foundation, is currently underway at the UW Carbone Cancer Center.
In this clinical trial, NET patients are treated with a new combination of two existing anti-cancer drugs. Both drugs have been found to be well tolerated on their own, and the combination approach may result in another line of treatment previously not available to NET patients.
"For patients with metastatic neuroendocrine tumors, current therapies are mostly temporizing measures, and most patients still succumb to their disease," said Dr. Nataliya Uboha, a gastrointestinal oncologist at UW Carbone who is leading the trial. "Patients need systemic therapies, and that is why we are doing this trial."
NET patients whose disease has metastasized from its initial site typically begin treatment with a relatively benign course of drugs to manage their disease; however, those drugs will not work indefinitely. The next steps depend on how much disease is present and how aggressive it is, and often include targeted drug therapies.
"The targeted drugs don't shrink tumors, they just keep them stable for some time," Uboha said. "However, if someone comes in with pain and symptoms from their disease, you want something that will shrink the tumor, and that is where chemotherapy may become useful."
While older chemotherapies were studied for NETs in the past, the drugs had such high toxicity profiles that their use is now limited. Retrospective studies of a combination of two of the newer oral chemotherapy agents, temozolomide and capecitabine, have shown a response in up to 70 percent of patients specifically with the pancreatic form of NET, and showed less toxicity than prior treatments.
This current NET trial at UW Carbone is evaluating the combination of temozolomide and a newer drug, TAS-102, that is related to capecitabine but that has a different mechanism of action.
"TAS-102 was approved for colon cancer patients in 2015 and many of those patients who no longer respond to capecitabine show a response to TAS-102," Uboha said. "If this trial is successful, then we are one step closer to adding another line of therapy in treating neuroendocrine tumors."
To date, the trial has accrued four patients. There is early evidence that the drugs are clinically active and there have been no unexpected toxicities so far.
The trial is sponsored by Taiho Oncology, Inc., with support from the Aly Wolff Foundation, Garding Against Cancer and other philanthropy funds to UW Carbone.
Date Published: 04/30/2018