Novel Targeted Therapy Effective Against Lung, Thyroid Cancers in Preclinical Trial


BLU-667 is being investigated for the treatment of cancers caused by an alteration in the receptor tyrosine kinase.

An investigational drug has shown promise in a phase 1, first-in-human trial for the treatment of cancers caused by an alteration in the receptor tyrosine kinase (RET), according to a study published in Cancer Discovery.

According to the study, which was led by researchers at The University of Texas MD Anderson Cancer Center, the oral drug BLU-667 could fill an unmet need for effective drugs against RET-driven cancers, such as medullary and papillary thyroid, non-small cell lung, colorectal, and bile duct cancers. Current treatments for these cancers are limited to traditional chemotherapy and earlier generations of multiple kinase inhibitors, which have limited success and considerable adverse effects, according to the researchers.

The study is investigating BLU-667 as a novel precision-targeted drug that targets RET-altered cancers with fewer adverse events affecting non-cancerous organs.

For the study, the researchers evaluated 43 patients with advanced tumors not eligible for surgery, including 26 patients with medullary thyroid cancer, 15 with non-small cell lung cancer, and 2 with other RET-driven cancers. According to the findings, treatment with BLU-667 exhibited an overall response rate of 37% for RET-driven cancers, with responses of 45% for non-small cell lung cancer and 32% for medullary thyroid.

“Tumor reductions and durable responses were observed in most patients, especially those patients whose cancer progressed with chemotherapy and multi-kinase inhibitors,” Vivek Subbiah, MD, assistant professor of Investigational Cancer Therapeutics, said in a press release.

The researchers noted that BLU-667 is 100 times more selective for RET than other kinases tested and has proven effective in stopping genetic mutations known as gatekeepers, which have been tied to resistance to multiple kinase therapy.

“By offering a highly selective medicine tailored for this oncogeneic driver, we hope this new therapy will enable patients to benefit from the recent advances in genomic profiling that have revolutionized treatment options for patients with kinase-driven disease,” Dr Subbiah concluded in the press release.


Subbiah V, Gainor JF, Rahal R, et al. Precision targeted therapy with BLU-667 for RET-driven cancers. Cancer Discovery. 2018. Doi: 10.1158/2159-8290.CD-18-0338

First-in-human clinical trial of new targeted therapy drug reports promising responses for multiple cancers [news release]. MD Anderson’s website. Accessed April 16, 2018.

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