NIH to Start Enrollment for Precision Medicine Research

Patient ResearchIn his most recent State of the Union address, President Obama announced a bold new research effort to revolutionize how we improve health and treat disease. According the White House website, the Precision Medicine Initiative, as it’s called, will pioneer a new model of patient-powered research that promises to accelerate biomedical discoveries and provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patients.

Last week, we learned that the one million patient research cohort at the National Institutes of Health (NIH) could begin enrolling patients in the next fiscal year.

As noted by the NIH, “Precision medicine is an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person. Many efforts are underway to help make precision medicine the norm rather than the exception.”

The NIH is currently finalizing a plan for the cohort that will be reviewed by their research advisers and then must receive a sign-off from NIH Director Francis Collins.

Kathy Hudson, NIH’s director for science, outreach, and policy, believes the funding for the cohort will be approved by Congress, noting that Senate and House appropriators will invest $200 million for the Precision Medicine Initiative.

In addition to necessary funding, the cohort initiative will require policy updates, including those for protection of human research participants. Also, patient access to fully interoperable electronic health records will need to be improved.

As the original Politico article notes, “Hudson said NIH is working with the Senate to include any necessary policy updates for the initiative into its biomedical reform legislation. The chamber’s work is expected to ultimately synch with the House’s recently passed 21st Century Cures bill.”

For more information on precision medicine and the Precision Medicine Initiative, visit nih.gov/precisionmedicine.

House Passes ’21st Century Cures’ Bill Today

Capitol HillToday, a bi-partisan bill called the “21st Century Cures,” which would add $8.5 billion of funding to the NIH’s budget, passed by a 344-77 vote in the House.

The bill seeks to “accelerate the discovery, development and delivery of life saving and life improving therapies, and transforms the quest for faster cures by:

  1. Removing barriers to increased research collaboration;
  2. Incorporating the patient perspective into the drug development and regulatory review process.
  3. Measuring success and identifying diseases earlier through personalized medicine.
  4. Modernizing clinical trials.
  5. Removing regulatory uncertainty for the development of new medical apps
  6. Providing new incentives for the development of drugs for rare diseases.
  7. Helping the entire biomedical ecosystem coordinate more efficiently to find faster cures.
  8. Investing in 21st century science and next generation investigators.”

More information about the bill is available at http://energycommerce.house.gov/cures.

What does the Government Shutdown mean for Mesothelioma?

Each week that the government is shutdown, the National Institutes of Health (NIH) will be turning away 200 patients. According to a piece in the Wall Street Journal, NIH director Francis Collins said, “About 200 patients who otherwise would be admitted to the NIH Clinical Center into clinical trials each week will be turned away.”1 An estimated 1000 patients have already been turned away in the past year due to the sequester.2 The Mesothelioma Applied Research Foundation estimates that the NIH sees 125 mesothelioma patients per year, which means that 2.4 mesothelioma patients are being turned away from clinical trials each week that the government is shutdown.

Lisa Gonneville

Lisa Gonneville speaks at the Meso Foundation’s Congressional Briefing on mesothelioma

With only one FDA approved treatment for mesothelioma, patients often turn to clinical trials conducted at NIH after they have exhausted all of their other options. Mesothelioma warrior Lisa Gonneville, who is currently participating in a clinical trial at the NIH, shared her experience with mesothelioma to Capitol Hill staffers on Mesothelioma Awareness Day. “I’ve endured all of the treatment options available for mesothelioma, which are very limited,” said Mrs. Gonneville, “my only hope at this point is clinical trials.”

This shutdown hurts researchers as well. The NIH has already suspended intramural (in-house) research projects, and will stop accepting new patients and enrolling patients in any of the clinical trials it is conducting. If Congress remains at a stalemate, NIH-funded research at universities will continue although researchers could face funding delays. Also, some government-run databases may have problems, as support staff is furloughed. Approval of new NIH extramural grants to researchers in a university setting may be delayed as well.

The NCI Office of Advocacy Relations sent an email out yesterday confirming that no new research would begin, stating “In terms of intramural research, doctors will continue to see patients at the NIH Clinical Center; however, no new research may begin.”

Some other NIH Activities that will cease under a shutdown:

  • Initiation of new protocols at the NIH Clinical Center
  • Basic research conducted by NIH scientists
  • Translational research conducted by NIH scientists that develops clinical applications of scientific knowledge
  • Training of graduate students and postdoctoral fellows at NIH facilities
  • Scientific meetings at NIH facilities
  • Travel of NIH scientists to scientific meetings
  • NIH scientific equipment services
  • Almost all NIH administrative functions
  • NIH mail, cafeterias, and most visitor services

The Mesothelioma Applied Research Foundation will continue to follow the government shutdown and update the community.

View the Washington Post blog here.


Footnotes

[1] The shutdown could prevent kids with cancer from getting treatment
[2] According to the American Cancer Society