NCI and the Precision Medicine Initiative®

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Precision Medicine: Discovering unique therapies that treat an individual’s cancer based on the specific abnormalities of their tumor.

The Precision Medicine Initiative® (PMI) is a $215 million investment in the National Institutes of Health’s (NIH) fiscal year 2016 budget to accelerate biomedical research and provide clinicians with new tools to select the therapies that can be used in a more individualized approach with patients. NCI is using $70 million of that investment to advance the field of precision oncology. Other disease areas will be considered by NIH over the longer term.

Oncology is a natural choice as the initial focus for this ambitious initiative. Precision medicine uses the genetics of disease to identify effective therapies, and, thanks in large part to NCI-supported research, we know that cancer is a disease of the genome.

NCI has been on the forefront of precision medicine since 2014. The institute has launched a series of precision medicine clinical trials and, as a result, many genetically targeted therapies are currently available to cancer patients and several more therapies are expected to become available in the future.

In addition to NIH, the Food and Drug Administration, and the Office of the National Coordinator for Health Information Technology also have major roles in the PMI®.

NCI is leading the PMI-Oncology component of the initiative, which is focusing on expanding precision medicine clinical trials, overcoming drug resistance, developing new laboratory models for research, and developing a national cancer knowledge system.

Expanding Precision Medicine Clinical Trials

Precision medicine clinical trials assign patients to therapy based on the genetic alterations that are thought to be driving their cancer. Over the past several years, in partnership with both the public and private sectors, NCI has launched several precision medicine trials, including the Lung Cancer Master Protocol (Lung-MAP), Adjuvant Lung Cancer Enrichment Marker Identification and Sequencing Trials (ALCHEMIST), Molecular Profiling-Based Assignment of Cancer Therapy (NCI-MPACT), and the first-of-its-kind Molecular Analysis for Therapy Choice (NCI-MATCH) precision medicine trial.

As part of the effort to support PMI-Oncology, NCI expanded NCI-MATCH. In this trial, adult patients are assigned to targeted treatments based on the genetic abnormalities in their tumors, regardless of the type of cancer they have. The expansion of the trial allowed for an increase in the number of patients screened for participation and for more detailed sequencing analyses of treated patients.

Under the PMI-Oncology, NCI has also launched the NCI-COG Pediatric MATCH trial, which will enroll children and adolescents with advanced cancers that have progressed on standard therapy and who have a genetic abnormality that matches with one of the agents being tested. Pediatric MATCH is cosponsored by the Children’s Oncology Group (COG).

NCI also plans to support new immunotherapy clinical trials, testing new combinations of cell, antibody, small molecule, and radiation therapy studies that include sequencing of tumor specimens. Plans are also underway to develop a consortium of research institutions to conduct deep immunological characterizations of patients enrolled in immunotherapy trials.

Overcoming Drug Resistance

NCI is increasing its support of research into the difficult problem of treatment resistance: when tumors initially respond to treatment but eventually begin to grow again. Under the PMI-Oncology, this involves:

  • Using tumor profiling to better understand how patients’ tumors become resistant to cancer therapy
  • Establishing a repository of molecularly characterized samples of resistant disease from biopsies of patients enrolled in NCI early-phase trials
  • Developing a pilot extramural drug resistance consortium

Developing New Laboratory Models for Research

NCI’s efforts to develop new laboratory models of human cancer includes vastly increasing the number of human cancer cell lines (grown as two-dimensional and three-dimensional cultures) and patient-derived tumor xenografts. These new models will help researchers gain new insights into tumor biology and better predict patients’ responses to cancer treatment.

NCI is also conducting immunotherapy studies to better understand, at the molecular level, what drives the response to immune-based treatments, and to identify opportunities that may permit broader use of this treatment approach.

Under the PMI-Oncology, NCI is:

  • Launching the NCI Patient-Derived Models Repository and distributing patient-derived xenograft tissues (and materials produced from those tissues) for drug discovery efforts
  • Initiating a canine immunotherapy models consortium to allow for development of another model system, which may prove especially helpful in studying lymphomas
  • Piloting the development of a Consortium of Preclinical Models
  • Launching the Human Cancer Models Initiative. This international project establishes three centers to create and characterize next-generation cancer cell line models from primary biopsies and make them widely available to the cancer research community

Developing a National Cancer Knowledge System

NCI is establishing a national database to house and integrate genomic information from tumors with clinical response data (e.g., tumor shrinkage) and outcomes information (e.g., length of survival) as a resource for scientists, health care professionals, and patients.

This is the knowledge system envisioned in a 2011 Institute of Medicine report, Toward Precision Medicine, which called for a comprehensive database that would be useful to researchers as well as clinicians actively treating patients.

Under the PMI-Oncology, NCI has:

  • Developed and launched the Genomic Data Commons, a single, scalable repository for cancer genomics data, patient information and response to treatment, pathologic and radiologic images, and relevant preclinical data
  • Launched the Cancer Genomics Cloud Pilots, three pilot programs that provide a cloud-based data and computational framework for data sharing and enable researchers to access, analyze, and compute on a complete set of data from The Cancer Genome Atlas (TCGA) and additional data sets in commercial clouds, such as those supported by Google and Amazon
  • Been working to define standard interfaces for exchanging cancer genomic data, including the Genomics Application Programming Interface

By expanding on earlier successes in cancer genomics, partnering with patients nationwide who participate in NCI-sponsored clinical trials, and extending precision medicine principles into basic science research and biomedical information technology activities, the PMI-Oncology is enabling NCI to advance precision oncology more quickly and efficiently than originally planned or anticipated.