This new study found that the test medication ceritinib effectively shrank tumors and reduced their number in more than half of the lung cancer patients treated.
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This study was led by Alice Shaw, MD, PhD, of the Massachusetts General Hospital Cancer Center.
The study included 130 participants diagnosed with ALK-rearranged non-small cell lung cancer (NSCLC) who were placed into one of two phases. The goal of this research was to verify the effectiveness of ceritinib in treating ALK-positive NSCLC after it had become resistant to the highly effective medication crizotinib.
Phase one of this research included 59 NSCLC patients and was designed to determine the maximum tolerable dose of ceritinib. Patients received daily doses that increased over time and ranged from 50 to 750 mg.
Phase one revealed generally mild adverse effects that included diarrhea, vomiting and dehydration, which stopped when treatment was lowered or discontinued.
The researchers enrolled an additional 71 participants for phase two, in which they received daily oral doses of ceritinib for as long as the medication effectively stopped or slowed tumor growth. Dosages where adjusted to reduce side effects.
The research team found that ceritinib showed dramatic reduction in the size and number of tumors in about 60 percent of the study's participants. The results were similar for those who had previously been treated with crizotinib as well as those who had not.
Resistance to ceritinib developed for most patients after an average of around seven months.
"Crizotinib has become a standard treatment agent for patients with advanced, ALK-rearranged NSCLC, but patients invariably develop resistance, leaving their treatment options limited," said Dr. Shaw in a press statement. "We found ceritinib to be highly effective in the majority of crizotinib-resistant patients, as well as those who had never received the drug, with mostly mild and manageable side effects."
According to Dr. Shaw and colleagues, this study led to ceritinib being designated as a "breakthrough therapy" by the U.S. Food and Drug Administration (FDA). Breakthrough therapy designation can lead to accelerated approval for use in the United States.
This study was first published March 26 in The New England Journal of Medicine.
This research was supported by Novartis, the maker of ceritinib, and through a grant provided by the National Cancer Institute.