MSK: 'This is what the future of precision medicine looks like'

Investigators at Memorial Sloan Kettering Cancer Center have changed the rules of the clinical trial game in the first of what they say will be a new wave of studies that examine a drug's potential based on a broad patient population who share the same genetic mutation rather than cancer type.

In this trial the researchers executed a Phase II trial of Zelboraf for nonmelanoma BRAFV600-mutated cancers (Zelboraf is approved for melanoma) in 122 patients from 23 centers around the world. The patients suffered from an array of cancer types, including lung, colorectal and ovarian cancers, as well as rare diseases such as Erdheim-Chester disease.

Doing a "basket" study like this allows the researchers to get a real world look at a drug's potential in different tumor types, which can be explored later. And it can greatly expand the patient population eligible to get a drug.

MSK Chief Medical Officer José Baselga

"This study is the first deliverable of precision medicine. We have proven that histology-independent, biomarker-selected basket studies are feasible and can serve as a tool for developing molecularly targeted cancer therapy," said MSK Chief Medical Officer José Baselga, the study's senior author. "While we can--and should--be cautiously optimistic, this is what the future of precision medicine looks like."

The investigators got back a mixed batch of responses, with non-small cell lung cancer and Erdheim-Chester disease standing out among the disease types. Response rate and median progression-free survival in non-small cell lung cancer was 42% and 7.3 months, respectively. In Erdheim-Chester disease and Langerhans cell histiocytosis, the response rate was 43%. And despite a median treatment duration of 5.9 months, no patients progressed during therapy. Anecdotal responses were seen in many other tumor types.

"This kind of study is a beneficial way to do rare tumor research because it allows us to open the study to patients with diseases that are completely underrepresented in clinical trials in general, such as anaplastic thyroid cancer and glioblastoma," said David Hyman, MD, the study's first author and Acting Director of Developmental Therapeutics at MSK. "By broadening eligibility, we are translating potential benefits to as large a patient population as possible."

- here's the release