27 OncoKB

For creating the first tumor mutation database 

The first seeds of what would eventually become OncoKB were planted in 2001. That was the year the U.S. Food and Drug Administration (FDA) approved the drug imatinib for certain patients with chronic myeloid leukemia, transforming a fatal disease into one that could be managed indefinitely with a once-daily pill. It was also the first drug to successfully target specific genetic alterations associated with tumor initiation or progression. 

In the two decades since, hundreds more targeted therapies have been released. Yet treatment options change and expand rapidly as new research is published, creating a deluge of information too vast for any one physician to track, let alone memorize. 

With the goal of making it easier for clinical oncologists and molecular pathologists to access up-to-date and accurate treatment information, Memorial Sloan Kettering (MSK) Cancer Center began developing a database. Bringing together a team of cancer biologists and software engineers, the U.S. healthcare organization came up with OncoKB, a cancer knowledge base like no other.

“We created OncoKB as a tool to help doctors understand which mutations in a particular gene are important and may predict for sensitivity or resistance to a particular drug,” says Dr. David B. Solit, director of the Marie-Josée and Henry R. Kravis Center for Molecular Oncology at MSK. 

In October 2021, OncoKB became the first and only cancer knowledge base to receive partial recognition from the FDA. That, in turn, gives OncoKB a new audience: researchers looking to streamline the development of next-generation sequencing-based tumor profiling tests. 

The path to this groundbreaking nod from the FDA began in April 2018, when the agency announced a regulatory approach for the use of human genetic variant databases. It’s similar to what a next-generation test developer would go through for a companion diagnostic—and required some new thinking from the team. 

“With the FDA partial recognition of OncoKB, the agency credentials our knowledge base as providing accurate, reliable and clinically meaningful information to the medical and scientific communities,” Dr. Debyani Chakravarty, OncoKB lead scientist and assistant attending physician at MSK’s Department of Pathology, said in a news release.

“Part of the mission of precision oncology is empowering the clinical community to leverage the cancer genome of a patient’s tumor to guide treatment,” Dr. Chakravarty says, giving more patients the opportunity to benefit from the potentially lifesaving information it contains.