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The preliminary trial of a drug called ulixertinib was conducted with 135 patients who had already failed treatments for one of a variety of advanced, solid tumors.
Researchers led by Dr. Ryan Sullivan, of Massachusetts General Hospital, said ulixertinib did seem to spur at least a "partial response" to the therapy or "disease stabilization," regardless of cancer type.
"It was exciting to see responses in some patients," said Sullivan, an oncologist and member of the Termeer Center for Targeted Therapies at the Boston hospital.
"The results of this study can be built upon to develop better treatment regimens for these patients," he said in a news release from the American Association for Cancer Research (AACR).
One cancer specialist explained how ulixertinib works on the cellular level.
"It inhibits the MAPK/ERK pathway, which is a chain of proteins in the cell that communicates a signal from a receptor on the surface of the cell to the DNA in the nucleus of the cell," said Dr. Maria Nieto.
"When one of the proteins in the pathway is mutated, it can become stuck in the 'on' or 'off' position, which is a necessary step in the development of many cancers," said Nieto, a medical oncologist at Northwell Health's Huntington Hospital in Huntington, N.Y.
Ulixertinib effectively inhibits this broken cellular pathway, and that inhibition "can be therapeutically exploited in multiple different cancers such as melanoma, lung, colon, and low-grade ovarian cancer," she explained.
Sullivan said that because ulixertinib targets the "final regulator" in the MAPK/ERK pathway, it might avoid cancer cells' typical resistance to drug treatment.
"A great number of cancers -- including melanoma and lung cancers -- have mutations in the MAPK/ERK pathway, and while current therapies target proteins in this cascade, many patients develop resistance to current drugs," he explained.
"The common denominator in these failed therapies is that the cancer has found a way to activate ERK. Therefore, the development of ERK inhibitors is a crucial next step to target this aberrant pathway," Sullivan said.
When it came to side effects, ulixertinib appeared to have a "tolerable" profile, with most issues not particularly severe, the researchers said. But this was still a small phase 1 trial, Sullivan noted, so larger trials are needed.
The study was funded by the drug's developer, Biomed Valley Discoveries, and published Dec. 15 in the AACR journal Cancer Discovery.
Dr. Stephanie Bernik is chief of surgical oncology at Lenox Hill Hospital in New York City. She agreed that the new medicine has great potential.
"Ulixertinib halts the message at the last stop before the signal can make it into the nucleus and creates a second roadblock, therefore halting growth of the cancer cell," Bernik explained. "This kind of therapy shows great promise and allows drugs to work synergistically, making it much harder for the cancer cell to figure out a way to continue to multiply and spread."
According to the study team, the U.S. Food and Drug Administration has fast-tracked ulixertinib for development and potential approval.
-- E.J. Mundell
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