Combination immunotherapy shrank melanoma that has spread to the brain in more than half of the patients in a clinical trial reported online on August 23, 2018 in the New England Journal of Medicine led by an investigator at The University of Texas MD Anderson Cancer Center. The article is titled “Combined Nivolumab and Ipilimumab in Melanoma Metastatic to the Brain.” Of 94 patients in the single-arm study combining checkpoint inhibitors ipilimumab and nivolumab, at a minimum follow-up of nine months and a median of 14 months, 24 (26 percent) had a complete response, 28 (30 percent) had a partial response, and 2 (2 percent) had stable disease. "As treatment for stage 4 melanoma has improved greatly in recent years, our patients with metastases to the brain have remained the group most in need, they've had the worst prognosis, so we are very excited about these results," said the national study's principal investigator and lead author Hussein Tawbi, MD, PhD, Associate Professor of Melanoma Medical Oncology at MD Anderson. "This practice-changing study proved that you can start with immunotherapy first with these patients, tackling both brain and extracranial disease at the same time," Dr. Tawbi said. "And it opens up new opportunities for development of systemic therapies for metastatic melanoma." About 40 percent of patients with stage 4 melanoma have brain metastases at diagnosis, and 75 percent eventually develop the condition, which previously was so intractable to treatment that these patients were routinely excluded from clinical trials of new drugs. Median overall survival of patients with brain metastases has been four to five months. In the reported study, at nine months, 59.5 percent of patients with brain tumors had not progressed.
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