AACR Project GENIE

Precision medicine requires an end-to-end learning health care system, wherein the treatment decisions for patients are powered by the prior experiences of every similar patient who has preceded them. Oncology is currently leading the way in precision medicine because the factors that fuel cancer initiation, progression, and recurrence—namely, the genomic and other molecular characteristics of patients and their tumors—are routinely collected at scale. A major challenge to this approach, however, is that no single institution is able to sequence and treat sufficient numbers of patients to improve clinical decision-making independently. The AACR Project GENIE® (Genomics Evidence Neoplasia Information Exchange) registry was established in 2015 to address this challenge—accelerating the pace of drug discovery, improving clinical trial design, and driving progress for cancer patients.

AACR Project GENIE® is an open-source, international, pancancer registry of real-world data assembled through data sharing between 19 leading international cancer centers. The registry leverages ongoing clinical sequencing efforts at participating cancer centers by pooling their data to create a collective evidence base. The consortium and its activities are driven by openness, transparency, and inclusion to ensure that the project output remains accessible globally and ultimately benefits patients.

In its eighth year, AACR Project GENIE® took several bold steps toward delivering on the promise of precision medicine:

In September, GENIE® shared its fourteenth public data release, which increased the size of the registry to nearly 161,000 patients. With this latest release, genomic and baseline clinical data from more than 183,000 tumors are accessible through the efforts of the project’s strategic and technical partners, Sage Bionetworks and cBioPortal.
During the AACR Annual Meeting 2023 in April, AACR Project GENIE® announced the addition of four new Participating Institutions: Children’s Hospital Los Angeles; Korea University Anam Hospital; Louisiana State University Health Sciences Center New Orleans; and the University of Miami Sylvester Comprehensive Cancer Center. These new partners were selected following an open call in 2022 for sites that regularly treat and sequence patients from underrepresented and underserved backgrounds. These new members of the Consortium will further expand the diversity of the patient data in the AACR Project GENIE Registry.
More than 14,000 registered users now utilize AACR Project GENIE® data in their research, and the registry has been cited more than 1,000 times through 2023. The high level of interest in the data was demonstrated at the AACR Annual Meeting 2023, as 26 proffered papers using GENIE data were presented. The most impactful papers were again featured in a minisymposium titled “Advancing Cancer Research Through an International Cancer Registry: AACR Project GENIE® Use Cases.”

Understanding the genomic complexity of RAS-mutant tumors is key for mapping the therapeutic vulnerabilities of these tumors and designing RAS-targeted clinical trials. In a paper published in the AACR journal Cancer Research, Robert B. Scharpf and colleagues used targeted next-generation sequencing data of 607,863 mutations from 66,372 tumors in 51 cancer types from the AACR Project GENIE® Registry to outline the genomic landscapes and hallmarks of mutant RAS in human cancers, creating what is likely the most comprehensive analysis of the genomic landscape of RAS-mutant tumors to date. This resource can be used to develop clinical strategies for the one-third of all cancers driven by RAS mutations.

The AACR Project GENIE® Biopharma Collective (BPC) is a five-year collaboration with a coalition of ten biopharmaceutical companies launched in 2019 with the goal of obtaining clinical and genomic data from an estimated 50,000 de-identified patients treated at GENIE® participating institutions.

In September, the BPC team published a comprehensive analysis of the BPC public dataset of more than 1,800 non-small cell lung cancer patients in the AACR journal Clinical Cancer Research. The survey revealed that 44% of all patients in the cohort had an actionable mutation within their tumors. Analysis of the genomic data also revealed the prevalence and type of genomic alterations by smoking status. Further, real-world overall survival (rwOS) and progression-free survival (rwPFS) were determined for the cohort, as well as for various treatment regimens. The detailed clinico-genomic data included in the AACR Project GENIE® dataset also enabled the team to analyze genomic drivers of sites of metastasis as well as emergence of treatment-induced resistance mutations.

Illustration credit: Christina Kostandi and Valsamo Anagnostou.

The AACR Project GENIE® Biopharma Collective (BPC) is a five-year collaboration with a coalition of ten biopharmaceutical companies launched in 2019 with the goal of obtaining clinical and genomic data from an estimated 50,000 de-identified patients treated at GENIE® participating institutions.

In September, the BPC team published a comprehensive analysis of the BPC public dataset of more than 1,800 non-small cell lung cancer patients in the AACR journal Clinical Cancer Research. The survey revealed that 44% of all patients in the cohort had an actionable mutation within their tumors. Analysis of the genomic data also revealed the prevalence and type of genomic alterations by smoking status. Further, real-world overall survival (rwOS) and progression-free survival (rwPFS) were determined for the cohort, as well as for various treatment regimens. The detailed clinico-genomic data included in the Project GENIE® dataset also enabled the team to analyze genomic drivers of sites of metastasis as well as emergence of treatment-induced resistance mutations.

AACR Project GENIE®

Accelerating Drug Discovery

AACR Project GENIE® by the Numbers