Regorafenib Effective for Gastrointestinal Stromal Tumor When Other Treatments Stop Working

ASCO Annual Meeting
June 4, 2012

A new study showed that the targeted drug regorafenib is an effective treatment for patients with gastrointestinal stromal tumor (GIST) that has worsened because the other available treatments have stopped working. Targeted therapy is a treatment that targets a tumor's specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. Specifically, regorafenib targets an abnormal enzyme called KIT. The currently available GIST treatments, imatinib (Gleevec) and sunitinib (Sutent), often slow or stop tumor growth at first, but eventually the drugs stop working and the cancer continues to grow. Regorafenib appears to work in a different way, even helping to slow GIST growth when other treatments are no longer working. 

The 199 patients who participated in this study had GIST that had spread to other parts of the body or that could not be removed with surgery. They received either regorafenib or a placebo (inactive treatment). All patients received supportive care, which is treatment to relieve the symptoms of the disease. It is usually the best option for patients with GIST when all the approved treatments no longer work.

For patients taking regorafenib, the disease worsened almost five months after treatment, compared with almost a month for patients taking the placebo. Because regorafenib worked so well, 85% of the patients receiving the placebo and supportive care switched to regorafenib once the GIST worsened.

What this means for patients

“Regorafenib will fulfill an urgent unmet need for patients with GIST who have received all other treatment options,” said George Demetri, MD, Director, Ludwig Center and Sarcoma Center at Dana-Farber Cancer Institute and Harvard Medical School in Boston, Massachusetts. “Targeted therapy has revolutionized treatment for this rare cancer, but we've been on the hunt for additional effective treatments when the only two available therapies stop working.” Currently, regorafenib is only available in clinical trials. It's important to talk with your doctor about the treatment options for GIST, including clinical trials.

Questions to Ask Your Doctor

  • What are my treatment options for GIST?
  • What treatment plan do you recommend? Why?
  • How will the cancer be managed if this treatment stops working?
  • What clinical trials are open to me?

For More Information

Guide to Gastrointestinal Stromal Tumor

Understanding Targeted Treatments

Placebos in Cancer Clinical Trials

Palliative Care

When the First Treatment Doesn't Work