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NIH director says breakthroughs and increased funding make it the “National Institutes of Hope”

OCTOBER 22, 2018
Clinical Congress Daily Highlights, Monday First Edition


Despite the many health challenges our society faces, from cancer to the opioid epidemic, we live in a time of hope, National Institutes of Health Director Francis S. Collins, MD, PhD, said in his Martin Memorial Lecture.

For patients who are out of options, the National Institutes of Health Clinical Center, the world’s largest clinical research clinic, is the “House of Hope,” Collins said. The NIH Clinical Center has about 1,500 studies in progress, said Dr. Collins, who has served as director for 10 years.

There is hope that the work of the NIH will flourish due to five straight years of funding increases, set to hit $39 billion this year. This funding was not always a guarantee, as after doubling from 1990 to 2003, NIH funding underwent significant decline over the next decade when adjusted for inflation.

The achievements of the NIH are impressive, starting with the Human Genome Project, which spent $400 million to complete the first-ever sequence of the human genome in 2003. The price of genomic sequencing has plunged since then, now costing about $850 per individual with $100 not far off, Dr. Collins said. In addition, the time it takes to collect a human genome has shrunk from years to a day or two, he said.

This progress has driven significant advancements in cancer. Since the first sequencing, there have been 11,000 cancers sequenced of 35 different tumor types, leading to the conclusion that it is less important where the cancer occurs than its mutation and the genes present in it.

The Cancer Moonshot, begun in 2016 and now called the Cancer Breakthroughs Initiative 2020, has accelerated the prevention, diagnosis, treatment and care of cancer patients, and offers hope that we will be able to develop optimal treatments for individuals based on the molecular signature of their cancers. An increased understanding of these underlying molecular drivers and the discovery of agents targeting them has resulted in a shift toward clinical trials that specifically evaluate patients whose tumors contain these relevant mutations, such as the National Cancer Institute’s clinical study Molecular Analysis for Therapy Choice Program (MATCH).

Meanwhile, the development of cancer immunotherapy has given physicians another effective tool to treat cancer in addition to surgery, radiation and chemotherapy.

“While we have yet to get this approach to work for solid tumors such as those of the colon and prostate, I predict that day will come,” Dr. Collins said.

Precision medicine for breast cancer patients took a significant step forward with the recent groundbreaking Trial Assigning Individualized Options for Treatment (Rx), or TAILORx trial. The trial showed that there is no benefit from chemotherapy for 70 percent of women with the most common type of breast cancer – hormone receptor (HR)-positive, HER2-negative, axillary lymph node-negative breast cancer. That is, treatment with chemotherapy and hormone therapy after surgery is not more beneficial than treatment with hormone therapy alone. As a result, Dr. Collins said, thousands of women will be able to avoid the risks and rigors of chemotherapy.

Other recent scientific achievements out of the NIH include:

  • Image-guided surgery that will allow surgeons to identify tumor margins or to mark nerves
  • Regenerative medicine that can use individualized stem cells to create an experimental organ or “tissue chip” that can be used to test if an innovative therapy would be beneficial or toxic to a particular patient

But a new health crisis now threatens millions of Americans – the crisis of addiction and pain. More than 2 million people are addicted to opioids, most of them after receiving prescriptions for pain medication. There are more deaths due to opioid overdose than to HIV at its peak or traffic accidents, Dr. Collins said. On the other hand, 100 million Americans are burdened with chronic pain, including 25 million who have pain on a daily basis.

There are a number of initiatives the NIH has taken to address this problem:

  • In April 2018, NIH launched the HEAL (Helping to End Addiction Long-term) Initiative, an aggressive effort to speed scientific solutions to stem the national opioid public health crisis. In addition, there is an effort to optimize effective treatment though the HEALing community study.
  • New treatments are in development for those with opioid addiction.
  • There is an advanced clinical trial for treatment of neonatal opioid withdrawal syndrome.
  • The National Institute on Drug Abuse (NIDA) trial network has been enhanced.
  • A Justice Community Opioid Innovation Network, a network of researchers, has been established to rapidly conduct studies aimed at exploring the effectiveness and adoption of medications, interventions and technologies in the state and local justice systems.
  • Better strategies on pain management are in development, with a focus on how acute pain becomes chronic pain.

To expand the boundaries of precision medicine, the NIH is taking an emerging approach to disease prevention and treatment in its All of Us Research Program. The effort will do for precision medicine what the Framingham study did for heart disease – except that it will be 40 times larger by gathering data over decades from more than 1 million patients, Dr. Collins said. By taking into account individual differences in lifestyle, environment and biology, researchers will uncover paths toward delivering precision medicine. More than 100,000 people have signed up for the program, which launched in May.

“This will be the largest ever biomedical database resource,” Dr. Collins said. “We expect it will catalyze a wide range of research.”

Surgeons should consider what projects they might pursue using the database, Dr. Collins said. He offered a few suggestions:

  • Why do some people with the same exposure to opioids become persistent users while others do not?
  • What distinguishes recurring cancer patients from those whose cancer does not recur?
  • Does surgery for benign prostatic hyperplasia (BPH) improve the patient’s metabolic risk profile?

“This is an amazing time for science,” Dr. Collins said. “I am seeing things I never would have imagined could have happened.”

Hope is a privilege, Dr. Collins said, that comes out of action.

“We have a plan of action,” he said. “No action, no hope.”

Additional Information

The Martin Memorial Lecture, NIH: The National Institutes of Hope, was held October 22 at the 2018 Clinical Congress of the American College of Surgeons in Boston. Program, webcast, and audio information is available online at facs.org/clincon2018.

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