Grail nets FDA breakthrough designation, turns eye toward DNA methylation for early cancer detection

Grail’s multicancer blood test has taken a helpful step closer to approval, by obtaining a breakthrough device designation from the FDA.

The investigational liquid biopsy is currently being developed to spot the early signs of a range of different cancers in individuals age 50 and older—hopefully at times when treatments can be more effective.

“We’re excited the FDA recognizes the potential of our multicancer early detection blood test,” Grail CEO Jennifer Cook said in a statement. “There are no effective early detection tests for the majority of cancer types, and many deadly cancers are often detected too late.”

The agency’s breakthrough program aims to help speed up development, assessment and review—which could come in handy as Grail sifts through the massive amounts of data from its large-scale clinical studies, including two that now sit with a total enrollment of about 115,000 participants.

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The Menlo Park, California-based company presented initial data from its Circulating Cell-free Genome Atlas (CCGA) project last year at the annual meeting of the American Society of Clinical Oncology. At 98% specificity, the liquid biopsy’s detection rates ranged from 38% to 51% in trial participants with early-stage lung cancer.

While the five-year CCGA study had planned to enroll more than 15,000 people, including about 70% with either newly diagnosed disease or treatment-naive cancer, Grail’s STRIVE study was designed to track more than 100,000 women undergoing mammography to validate the ability of its blood test to detect breast cancer and other malignancies, with data due to report in 2020.

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A third study, SUMMIT, is focused on accruing about 50,000 men and women in London who do not have a cancer diagnosis at the time of enrollment. About half will be people at high risk of cancer due to a significant smoking history. SUMMIT’s goal is to validate Grail’s tests in a second intended-use population and to explore its utility in a high-risk group.

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But since that CCGA presentation last year, Grail has selected measurements of DNA methylation as its preferred approach, targeting the informative regions of the genome through a new methylation sequencing blood test.

The 2016 Fierce 15 company plans to present new results from the CCGA trial at this year’s ASCO meeting in Chicago—including data on the ability of Grail’s methylation technology to identify the tissue of origin when cancer DNA is detected in the bloodstream, as well as a survival analysis among participants who were screened early using that method.

DNA methylation is a natural process used by cells to regulate gene expression, featuring chemical modifications to the epigenome. In cancer, abnormal methylation patterns and changes in expression can contribute to tumor growth, including the inactivation of tumor-suppressing genes, Grail said.