Mutations predict response to pembrolizumab across multiple tumour types

Two-year study shows efficacy of immunotherapy drug in 12 mismatch-repair deficient cancers that failed to respond to prior treatment.

Genomic sequencing

Several markers have been identified that can predict response to the immunotherapy pembrolizumab in specific tumour types. But a universal marker across all tumour types has not been established.

In research published in Science
(8 June 2017), a team enrolled 86 patients with 12 different cancer types whose disease had progressed despite previous treatment to receive pembrolizumab for up to two years. All the patients had mismatch-repair (MMR) deficiencies, meaning they had mutations in genes involved in repairing DNA errors.

The team found that 66 (77%) of the 86 patients (95% confidence interval, 66–85%) achieved disease control for at least 12 weeks, including 18 (21%) patients who had a complete response.

Given that tests for MMR-deficiency are already available, the researchers predict that in future it will become standard to test for MMR deficiency in patients with refractory cancer, regardless of tumour type.


[1] Le D, Durham J, Smith K et al. Mismatch-repair deficiency predicts response of solid tumors to PD-1 blockade. Science 2017. doi: 10.1126/science.aan6733

Last updated
Clinical Pharmacist, CP, August 2017, Vol 9, No 8;9(8):DOI:10.1211/PJ.2017.20203057

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