A brand new cancer drug known as BLU-667 has moved via phase I human trials, and the results are promising. Taken orally, the drug targets what’re known as RET driven cancers, including types of lung and thyroid cancers, that are difficult to treat. RET is a type of receptor tyrosine kinase, and mutation that kicks on its action into overdrive is connected to specific sorts of cancer. Among others, RET plays an important role in about 50 percent of the medullary thyroid gland, 20 percent of the thyroid gland, and up to 2 percentage of non small cell lung cancer. These could be treated with traditional methods, although thus far success has not been leading.

“There is a critical un-met need for effective drugs against cancers that have the RET alteration, as there are no highly potent inhibitors currently approved specifically for these RET-driven cancers,” says Vivek Subbiah, lead researcher on the study. “The current treatments for these cancers are limited to traditional chemotherapy and earlier generations of multiple kinase inhibitors. These options have had limited success with often considerable side effects that significantly impact the patient’s quality of life.”

BLU-667 works by inhibiting the action of RET, and it was chosen for further research after it was discovered to be 100 times more selective for RET, meaning it should not impact the functions of other kinases. It has also been shown to prevent specific genetic mutations that may permit the body to withstand this sort of treatment. For this trial, the team studied 43 patients with advanced tumors which were not able to be worked on, as well as 26 patients with medullary thyroid cancer, 15 with a non-small cells cancer of the lung, and 2 with some other RET related cancers. The results have been promising, with the researchers saying that BLU-667 appears to be safe and effective to use.

“Tumor reductions and durable responses were observed in most patients, especially those patients whose cancer progressed with chemotherapy and multi-kinase inhibitors,” says Subbiah. “Our study reported an overall response rate of 37 percent for RET-driven cancers, with responses of 45 percent for non-small cell lung cancer and 32 percent for medullary thyroid.”

The results of the trial have been published in the journal Cancer Discovery, also presented in the American Association for Cancer Research Annual Meeting 2018 over the weekend.