Every clinical trial has its outliers. Some patients respond far better to the treatment than the rest, but the focus on efficacy across the study population means these results--and their implications--are lost in the shuffle. The National Cancer Institute (NCI) is trying to change this by improving the tracking of data on these "exceptional responders."
NCI began the project last year and updated Bloomberg on its progress and plans this week. By sifting through 10 years of clinical trial data, NCI has found 100 patients who fit its definition of "exceptional responder." These patients stood out in their trials. While less than 10% of their fellow participants responded, the patients picked out by NCI had a complete or partial response lasting at least 6 months. NCI is starting to sequence the DNA of these outliers to look for genetic reasons for their strong responses.
The likelihood of hitting upon a gene variant that leads to the reappraisal of a failed therapy or better use of an existing drug will increase as NCI looks at more exceptional responders. The Broad Institute, Memorial Sloan-Kettering Cancer Center and other academic medical centers have teamed up with NCI to gather data. Barbara Conley, associate director of NCI's Cancer Diagnosis Program, said: "The key is, can you find another patient with the same kind of abnormality and will they respond?"
From June, NCI will begin encouraging doctors and researchers to send data on patients who respond unusually well to treatment. The long-term goal is to build a database of such patients, simplifying the process of spotting common characteristics of exceptional responders. Sequencing and data-handling technology underpins the whole process. "These are mysteries we've always tried to solve, but we didn't have the tools until now to figure out the variation of responses in patients," Memorial Sloan Kettering's David Solit said.
- read Bloomberg's article
- here's FierceBiotech's take