University of Pennsylvania researchers have announced what they are describing as a major advance in treating thyroid cancer.
New research reported by medical experts at the Perelman School of Medicine has found two novel treatment approaches that build on recent discoveries about the genetics and cell signaling pathways of thyroid tumors that amount to exciting new weapons against the disease.
Two recent clinical trials incorporating the new approaches — using a class of drugs known as "kinase inhibitors" that specially target tumors and the blood vessels that supply them — offer "great promise" in treating thyroid cancer patients, who often face difficult prognoses and few treatment options.
The findings, presented at a meeting of the European Cancer Congress in Amsterdam this week, show the drug sorafenib — now approved to treat kidney and liver cancer — nearly doubled the survival rate of patients taking it in a clinical trial, compared to those taking an inactive placebo.
The first study, led by Marcia Brose, M.D. an assistant professor with the Abramson Cancer Center, found that patients who received sorafenib survived 10.8 months, five months longer than those taking a placebo arm. Most of the 417 patients enrolled in the trial had two common genetic mutations tied to thyroid cancer that are targeted by the drug.
"Our results are important because they show that regardless of the presence of these two common genetic changes, the group that was treated with sorafenib did better than the placebo," Dr. Brose said. "There was no subgroup that didn't appear to benefit from the intervention with the sorafenib."
The Food and Drug Administration is now evaluating sorafenib for use in treating advanced thyroid cancer. If it is approved, it would represent the first new drug for advanced thyroid patients in 40 years.
The second study Dr. Brose presented during the European Cancer Congress evaluated the effects of the drug vemurafenib on patients with papillary thyroid cancer (PTC), the most prevalent form of advanced thyroid cancer, which is also tied to genetic factors. Those taking the drug survived an average of 15.6 months, compared to 6.3 months among those not given the medicine.
"Our results show that we can effectively treat PTC patients that have progressive disease by targeting a common [genetic] mutation, and produce clinically meaningful periods of progression free survival," Dr. Brose said.
She added that the two trials offer substantial new hope for patients with progressive thyroid cancer.
"A few years ago there was nothing to offer these patients," Dr. Brose said. "By understanding similarities across different types of cancers, we have been able to show that therapies previously shown to be effective in other cancers, such as liver, kidney and bone, can be effectively used to treat a rare cancer, providing significant hope to these patients."
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