The National Institutes of Health (NIH) now stands as the largest single public funder of biomedical research in the world.1 The FY2022 Consolidated Appropriations Act (H.R. 2471), signed into law in March, increases biomedical research funding by nearly 5%, and it provides a total of $45 billion for the NIH in fiscal year 2022—an increase of $2.03 billion over FY2021.2 The additional funding for the NIH provides $6.9 billion for the National Cancer Institute (NCI)—an increase of $353 million over FY2021—as well as $1 billion through September 2024 to establish the Advanced Research Projects Agency Health (ARPA-H).
Ronald DePinho, MD
For more than 50 years, NIH-funded research has fueled a revolution in medicine. Yet, despite its proven benefit, the agency has been underfunded in recent decades. Its current funding is simply inadequate to address the growing medical challenges facing the nation and humanity.
One is reminded of the admonition from Mary Lasker, the tireless mid-20th-century health activist who devoted her life to securing greater research funding for cancer: “If you think research is expensive, try disease.”
Jed Manocherian, Founder and Chairman of ACT for NIH: Advancing Cures Today, a nonprofit advocacy organization committed to making increasing biomedical research funding a national priority, echoed this sentiment when he made the case on the funding inadequacy of the NIH in a memorandum sent to the scientific community and to members of Congress. “Even with the historic NIH funding increases in the past 7 years, funding levels remain inadequate to capitalize on the immense scientific opportunity available to NIH-funded researchers,” he wrote. “There is simply not enough money available to fund all the highly merited research applications. Biomedical research drives our economy, reins in unsustainable health-care costs, and spares millions across the globe from the ravages of disease. The cost is billions, the return is trillions, and it is priceless to patients and their families.”
Reversing the Trend in Inadequate NIH Funding
For many decades, sustained government funding for basic biomedical research has yielded a steady stream of unexpected scientific insights. These insights have led to revolutionary medical breakthroughs, including statins for cardiovascular disease and vaccines that prevent virally induced cancers of the cervix and throat.
Still, funding levels for the NIH fall short of what’s needed to expeditiously unravel the mysteries of the top disease killers in the United States, including heart disease, cancer, COVID-19, Alzheimer’s disease, and diabetes. In the case of cancer, thanks to technological and conceptual advances in biomedical research, which have led to more effective therapies and prevention strategies, cancer mortality rates have declined over the past nearly 3 decades by 32%,3 finally making a dent in dethroning this “emperor of all maladies.”
And yet the very welcome $353 million increase for the NCI will allow the agency to fund only a small fraction of the meritorious research applications it will receive this year—setting up a crisis for researchers and our patients.
This year, the federal government will invest $6.56 billion in the NCI. However, that is a far cry from the $200 billion–plus annual burden of cancer in the United States, a cost projected to increase annually with the aging of America. Ditto for diabetes, heart disease, and Alzheimer’s disease.
Academic research laboratories, which are major generators of scientific breakthroughs, rely heavily on NIH funding and have produced such stunning advances as the Human BioMolecular Atlas Program and mRNA vaccines for the coronavirus. We need consistent increased funding for these programs to continue producing advances in more effective therapies for cancer and other life-threatening diseases.
Long-term government investments in fundamental biomedical research have made the United States an economic powerhouse and the hands-down world leader in science, as reflected by its vibrant biotechnology industry and its dominance in Nobel Prizes. However, billions more are still needed to continue to fund innovative science developments.
The current funding of the National Institutes of Health is simply inadequate to address the growing medical challenges facing the nation and humanity.— Ronald DePinho, MD
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Reigniting the Cancer Moonshot Initiative
Recent White House and congressional attention to this financial need signals an important opportunity to make progress. For example, earlier this year, the White House announced its intention to reignite the U.S. Beau Biden Cancer Moonshot Initiative. Originally launched by then Vice President Joe Biden and then President Barack Obama, in 2017, as a $1.8-billion 7-year cancer research program with the goal of achieving 10 years of cancer research in 5 years, the Cancer Moonshot’s new goals are far more expansive. They include decreasing cancer deaths by at least 50% over the next 25 years and improving the experience of those living with and surviving cancer. But it will take aggressive investments in cancer research to meet these goals and enable Moonshot 2.0 to succeed.
As mentioned previously, included in the NIH’s FY2022 budget is $1 billion to create the ARPA-H, which will be part of the NIH, to make pivotal investments in transformative technologies and platforms and speed biomedical and health research.
The agency’s objective is to apply basic research findings to improve our ability to prevent, detect, and treat a range of diseases including cancer, infectious diseases, and Alzheimer’s disease. ARPA-H is modeled after the Pentagon’s highly successful Defense Advanced Research Projects Agency, which funded Moderna’s mRNA technology when other agencies were skeptical of the approach and led to the rapid development of its COVID-19 vaccine.
Why does foundational research matter? Scientific discovery is neither linear nor predictable. Unfettered research can illuminate surprising mechanisms underlying disease causation, mechanisms that can catalyze translational research for possible development of effective new drugs. Examples abound of such serendipitous discoveries. Here are three.
Immune Cancer Therapy: Nobel Prize laureate James Allison, PhD, set out simply to understand how T cells work and instead uncovered an immune regulatory mechanism, CTLA-4, which is found on the surface of T cells and acts as a brake the body uses to avoid an overreactive immune response. He then developed an antibody to block CTLA-4’s braking action, freeing the T cells to attack cancer. His discovery led to the development of ipilimumab, the first in a new class of checkpoint inhibitors, for the treatment of advanced melanoma. Subsequent research focusing on other immune system “brakes,” especially PD-1 and PD-L1, has resulted in effective treatments for additional cancers, including lung, kidney, bladder, gastric, liver, cervical, colorectal, head and neck, and Hodgkin lymphoma. Dr. Allison was not looking for a cancer cure but nevertheless discovered a way to unleash the immune system’s own tumor-attacking T cells to kill cancer.
Mechanism Involved in the Development of Alzheimer’s Disease: Although focusing on the role of the telomerase enzyme, which endows immortal growth to cancer cells, molecular studies ultimately revealed that the enzyme also plays a role in regulating genes related to the development of Alzheimer’s disease.4 Such unexpected findings point to a completely new epigenetic avenue for investigation and a dramatic departure from decades of research and drug development focused largely on reducing the accumulation of amyloid in the brain. Alzheimer’s disease is now the fifth leading cause of death for people aged 65 and older,5 a rate that continues to multiply with profound economic and humanitarian costs.
mRNA Vaccines: The feat of creating mRNA COVID-19 vaccines, which the U.S. Food and Drug Administration approved in August 2021, just a year and a half after the coronavirus was declared a pandemic, was regarded by many as a miracle. It was indeed remarkable. However, the underpinnings of the enabling technology emerged over 30 years before, when it was discovered that mRNA could be delivered safely and effectively into patients, opening the possibility of mRNA therapies. Subsequent decades of research laid the foundation for the rapid development of successful SARS–CoV-2 vaccines.
We owe it to our patients and their families to do everything we can to advance only the best scientific innovations...and turn them into new life-saving medicines.— Ronald DePinho, MD
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Heart disease, cancer, diabetes, and Alzheimer’s disease are consistently among the top 10 causes of death year after year. Making consistent NIH funding for basic research a national priority is critical to nurturing innovative research, which generates maximum benefit for the patients and families suffering from these deadly and costly diseases.
We must always remember that our work is about serving this purpose. We owe it to our patients and their families to do everything we can to advance only the best scientific innovations through the drug discovery and regulatory process and turn them into new life-saving medicines. As Mr. Manocherian said: “There are too many patients to be patient.” We must act now.
Dr. DePinho is Professor, Department of Cancer Biology, Division of Basic Science Research; Harry Graves Burkhart III Distinguished University Chair in Cancer Biology; and Past-President of The University of Texas MD Anderson Cancer Center in Houston.
Disclaimer: This commentary represents the views of the author and may not necessarily reflect the views of ASCO or The ASCO Post.
DISCLOSURE: Dr. DePinho is the Founder and Chairman of Opa Health and Vice Chair of Act for NIH.
1. National Institutes of Health: Our Society. Available at www.nih.gov/about-nih/what-we-do/impact-nih-research/our-society. Accessed May 26, 2022.
2. ASCO in Action: Congress Increases Federal Funding for Cancer Research. March 11, 2022. Available at www.asco.org/news-initiatives/policy-news-analysis/congress-increases-federal-funding-cancer-research. Accessed May 26, 2022.
3. American Cancer Society: Risk of Dying from Cancer Continues to Drop at an Accelerated Pace. Available at www.cancer.org/latest-news/facts-and-figures-2022.html. Accessed May 26, 2022.
4. Shim HS, Horner JW, Wu CJ, et al: Telomerase reverse transcriptase preserves neuron survival and cognition in Alzheimer’s disease models. Nat Aging 1:1162-1174, 2021.
5. Centers for Disease Control and Prevention: Alzheimer’s Disease. Available at www.cdc.gov/dotw/alzheimers/index.html. Accessed May 26, 2022.