Ten Years of AACR Project GENIE®

Launched in 2015, the international cancer registry blends real-word genomic and clinical data for all to access.

Charles L. Sawyers, MD, FAACR, has been a leader in the field of targeted cancer therapies since its earliest stages, including playing a major role in the development of imatinib (Gleevec), a targeted treatment for chronic myeloid leukemia approved by the U.S. Food and Drug Administration (FDA) in 2001. But as he continued to research new targeted therapies and treat patients with this new class of drugs throughout the early 2000s, he came to a realization: There needed to be a better way to identify subsets of cancer patients with specific genetic mutations that could be targeted.

During his tenure as President of the American Association for Cancer Research® (AACR) in 2013 and 2014, Dr. Sawyers turned that epiphany into a major initiative—AACR Project Genomics Evidence Neoplasia Information Exchange (Project GENIE®). The goal was to create a publicly accessible cancer registry with the real-world genomic and clinical data necessary to help improve clinical decision-making and accelerate the pace of advances in cancer precision medicine. But at that time, next-generation sequencing technology was only just emerging, so few institutions could afford to profile tumor genomes—and capture information about genetic alterations—during the patient’s diagnostic workup.

“I was aware of about eight to 10 cancer centers doing this,” he explained. “I could see that each one was going to discover one small thing, perhaps every year or so. But if we pooled our efforts and shared our data, we’d find bigger patterns.” 

So, Dr. Sawyers brought eight of those institutions together, working first with Memorial Sloan Kettering Cancer Center in New York City, and Dana-Farber Cancer Institute in Boston, to lay the initial seeds for Project GENIE. On November 6, 2015, the AACR publicly announced the launch of Project GENIE at the AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics Meeting in Boston. Less than two years later, in January 2017, the consortium announced the first public data release for the Project GENIE registry, which included nearly 19,000 samples. In 2025, Project GENIE celebrated its 10th anniversary, with a registry that has grown to include more than 250,000 sequenced samples from more than 200,000 patients and that includes 20 participating institutions.

“In the 10 years since its inception, AACR Project GENIE has become the largest, fully public registry of cancer genomic datasets and is propelling advances in cancer drug discovery and improved patient care,” said Margaret Foti, PhD, MD (hc), chief executive officer of the AACR. “To conceptualize and launch such a novel and impactful project was sheer brilliance on Dr. Sawyers’ part, and the entire cancer community will be forever grateful.”

Project GENIE has  worked over these past 10 years to add new analytes and expand the data types in the registry. As part of this mission, the Biopharma Collaborative (BPC) was launched in 2019 to collect more clinical data. So far, this coalition of 10 biopharmaceutical companies has added data from more than 19,000 de-identified patients across 10 cancer types, but the goal is to eventually add clinical data from about 50,000 patients. Because of these efforts, the registry is able to capture clinical longitudinal timelines, which allows researchers to correlate genomic alterations with treatment response.

Powering the Future of Precision Oncology 

The insights that clinicians and researchers can glean from the Project GENIE registry has led to advances in precision medicine and the care of patients. 

“Because Project GENIE is now well over 200,000 patients, it is possible to find examples of patients who have a mutation that’s so rare that it was missed by the original cancer sequencing efforts, such as The Cancer Genome Atlas,” Dr. Sawyers said. “In several cases, the mutation is in a protein for which there is a drug that was approved for some other indication, but not the cancer these patients have.” 

It is possible that some of these patients were treated with that drug off label since it targets the same mutation, Dr. Sawyers explained. And through Project GENIE, clinicians can check whether the drug led to a positive or negative outcome for these patients, which could help guide treatment decisions for other patients who are found to have that mutation. 

Dr. Sawyers added that data from Project GENIE can also be used to serve as a natural history cohort in clinical trials. Since it can be hard to find enough patients with a rare mutation to fill a randomized clinical trial, data from patients being treated with the investigational drug can instead be compared with real-world data from patients who were treated with other therapies. This was the case in 2020 when Amgen used real-world data from Project GENIE—as well as the Flatiron Health-Foundation Medicine Clinico-Genomic Database—to apply for accelerated approval for sotorasib (Lumakras). In May 2021, the FDA granted sotorasib accelerated approval for the treatment of patients with KRAS G12C-mutated non-small cell lung cancer, making it the first-ever approved KRAS inhibitor. 

The AACR also plans to make improvements through its artificial intelligence working group to further improve data collection and harmonization, and sees limitless potential in serving the needs of people with rare cancers.

“As AACR Project GENIE continues to grow, it will provide cancer researchers worldwide with actionable data to propel the next wave of discoveries, advancing cancer precision medicine, and transforming cancer patient care,” Dr. Foti said. “The staunch commitment of AACR Project GENIE to provide open, fully accessible data has created a remarkable public utility for the global cancer research community.”