NEW YORK (Reuters Health) Jun 01 - Phase I trial results suggest the new drug cabozantinib may be effective against medullary thyroid cancer (MTC). The drug has moved on to a phase III trial because of this early success.
An editorial published with a report of the trial says "significant antitumor efficacy in a large percentage of patients with a rare, genetically well-characterized disease is noteworthy in a phase I study.... A (partial response) rate of 29% in 37 patients with MTC is remarkable, especially for a disease with essentially no conventional therapeutic options."
MTC accounts for only 4% of all thyroid tumors, or fewer than 2,000 new cases each year in the U.S. Patients with metastatic disease face grim statistics: approximately 25% and 10% are alive at 5 and 10 years, respectively, according to Dr. Razelle Kurzrock of the University of Texas MD Anderson Cancer Center in Houston, who headed the trial, and colleagues.
Altogether the research team enrolled 85 patients in the phase I dose-escalation study. Thirty-seven patients in the study had MTC. Thirty-five of the 37 were available for the analysis, which was reported online May 23 in the Journal of Clinical Oncology.
Ten MTC patients (29%) had partial responses after a median follow-up of 17 months -- most commonly at a dose of 175 mg. Another seven patients also had at least a 30% decrease in tumor size from baseline but a response couldn't be confirmed. Fifteen patients (43%) had stable disease for at least six months.
Seventy-seven patients (90.6%) from the overall cohort reported at least one adverse drug-related event. The most common of these were diarrhea, fatigue, nausea, and decreased appetite. There were no differences between the MTC subset and other patients with regard to safety profile, and the study authors wrote that cabozantinib had "an acceptable spectrum of toxicity."
Cabozantinib inhibits multiple receptor tyrosine kinases, including rearranged during transfection (RET), vascular endothelial growth factor receptor 2 (VEGFR2), and Met, all of which have been suggested to have a role in MTC.
In their editorial, Drs. Yariv Houvras and Lori Wirth from Boston's Massachusetts General Hospital note that the available data makes it difficult to determine if cabozantinib's activity against any one particular pathway was responsible for its efficacy, or if the combined inhibition of multiple pathways improved responses.
Dr. Kurzrock and other study authors reported receiving honoraria, research funding, and other remuneration from Exelixis, the company developing cabozantinib. Two authors are employed by or have leadership positions with the company.
An editorial published with a report of the trial says "significant antitumor efficacy in a large percentage of patients with a rare, genetically well-characterized disease is noteworthy in a phase I study.... A (partial response) rate of 29% in 37 patients with MTC is remarkable, especially for a disease with essentially no conventional therapeutic options."
MTC accounts for only 4% of all thyroid tumors, or fewer than 2,000 new cases each year in the U.S. Patients with metastatic disease face grim statistics: approximately 25% and 10% are alive at 5 and 10 years, respectively, according to Dr. Razelle Kurzrock of the University of Texas MD Anderson Cancer Center in Houston, who headed the trial, and colleagues.
Altogether the research team enrolled 85 patients in the phase I dose-escalation study. Thirty-seven patients in the study had MTC. Thirty-five of the 37 were available for the analysis, which was reported online May 23 in the Journal of Clinical Oncology.
Ten MTC patients (29%) had partial responses after a median follow-up of 17 months -- most commonly at a dose of 175 mg. Another seven patients also had at least a 30% decrease in tumor size from baseline but a response couldn't be confirmed. Fifteen patients (43%) had stable disease for at least six months.
Seventy-seven patients (90.6%) from the overall cohort reported at least one adverse drug-related event. The most common of these were diarrhea, fatigue, nausea, and decreased appetite. There were no differences between the MTC subset and other patients with regard to safety profile, and the study authors wrote that cabozantinib had "an acceptable spectrum of toxicity."
Cabozantinib inhibits multiple receptor tyrosine kinases, including rearranged during transfection (RET), vascular endothelial growth factor receptor 2 (VEGFR2), and Met, all of which have been suggested to have a role in MTC.
In their editorial, Drs. Yariv Houvras and Lori Wirth from Boston's Massachusetts General Hospital note that the available data makes it difficult to determine if cabozantinib's activity against any one particular pathway was responsible for its efficacy, or if the combined inhibition of multiple pathways improved responses.
Dr. Kurzrock and other study authors reported receiving honoraria, research funding, and other remuneration from Exelixis, the company developing cabozantinib. Two authors are employed by or have leadership positions with the company.
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