A Phase 1 clinical trial on patients with cancers that are the result of a change to the tyrosine kinase receptor, also called RET, has produced encouraging results. Cancers caused by alterations to RET include medullary and papillary thyroid, bile duct, non-small cell lung, and colorectal cancers. The full story can be read here, at MedicalXpress.

The RET receptor is an important ‘messenger’ involved in many different biological processes, and development in particular. Alterations to RET have been linked to several cancers. They are responsible for half of all cases of medullary thyroid cancers, as well as a fifth of papillary thyroid cancers, and 1-2% of non-small cell lung cancers.

Cancers caused by RET alterations tend to be difficult to treat, and patients have limited options. The two main alternatives available at the moment are multiple kinase inhibitors and chemotherapy. Multiple kinase inhibitors work by stopping the actions of particular enzymes. While these options are successful in some patients, others find that they are ineffective and come with serious side effects that affect their quality of life. The potential drug being trialled, called BLU-667, would provide patients with the first powerful inhibitor drug specifically targeted at RET-driven cancers.

The results of the clinical trial of BLU-667 were recently presented on the 15^th April at the annual meeting of the American Association for Cancer Research, and are also published in this month’s issue of Cancer Discovery. The study involved several groups of patients. The first group comprised of 43 cancer patients who had advanced tumors but were ineligible for surgery. The second group was of 26 patients who all had medullary thyroid cancer, the third group involved 15 patients with non-small cell lung cancer, and the final group was two patients with other RET-driven cancers. The researchers found that most patients experienced a lasting reduction to tumors. The drug worked more effectively for some cancers than others; patients with non-small call lung cancer had an overall response rate of 45%, while those with medullary thyroid cancer had a response rate of 32%. However, the study was only carried out on a small group of patients so more research is needed to confirm these results.

However, the Phase 1 clinical trial of the drug BLU-667 does show promising results for its use as an effective treatment for RET-driven cancers. The drug is also thought to have relatively few side effects compared to existing treatment options. This is the first time the drug has been studied in people, and more clinical trials would help researchers to better understand the effects of the drug in patients and how it could be used as a treatment.

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