Posted on June 15, 2016
Last August, Douglas Oliver, 52, of Nashville, was legally blind. Today, he can see. His story is just one of many stunning breakthroughs in biomedical research that Congress is seeking to accelerate in a “21st Century Cures” Act.
Early last year Vanderbilt University doctors told Mr. Oliver that there was no cure for the inherited form of Macular Degeneration that had gradually stolen his eyesight. But doctors suggested that he check the internet for promising research. Oliver found a clinical trial in Florida. There, doctors inserted a needle into his hip bone, extracted his own cells, spun them in a FDA-cleared centrifuge, and injected some cells into his retina. Oliver’s left eye vision improved from 20/2000 to 20/40 and in his right eye improved from 20/400 to 20/30. Last December he received his Tennessee driver’s license 11 years after surrendering it because of blindness.
In January of last year, Bill Elder attended a White House symposium. Elder had been cured of cystic fibrosis by medicines discovered as a result of research by Dr. Francis Collins, the scientist who led our government’s successful effort to map the human genome. At that symposium the President launched a “Precision Medicine Initiative” to map the genomes of 1 million volunteers to allow more research that might produce treatments and cures that do not treat every individual as the same.
Now the same Dr. Collins is director of the National Institutes of Health, a federal agency that this year funds $32 billion in biomedical research, mostly at American universities. At a United States Senate hearing earlier this month, Dr. Collins offered “bold predictions” about major advances to expect if there is a sustained commitment to such research.
One prediction is that science will find ways to identify Alzheimer’s before symptoms appear as well as how to slow or even prevent the disease. Today Alzheimer’s causes untold family grief and costs $236 billion a year. Left unchecked, the cost in 2050 would be more than our nation spends on national defense.
Dr. Collins’ other predictions are equally breathtaking. Using pluripotent stem cells—the kind of regenerative medicine treatment that helped Doug Oliver see—doctors could use a patient’s own cells to rebuild his or her heart. This personalized rebuilt heart, Dr. Collins, says, would make transplant waiting lists and anti-rejection drugs obsolete.
He expects development of an artificial pancreas to help diabetes patients by tracking blood glucose levels and creating precise doses of insulin. A Zika vaccine should be widely available by 2018 with a universal flu vaccine and an HIV/Aids vaccine available within the decade. To relieve suffering and deal with the epidemic of opioid addiction that led to 28,000 overdose deaths in America in 2014, there will be new non-addictive medicines to manage pain.
As an example of the advances possible, Collins points to President Jimmy Carter who seven months ago revealed that melanoma had spread through his brain and that he was beginning a course of therapy to boost his immune system’s ability to destroy his cancer cells. Last month President Carter announced he was cancer-free and no longer needs treatment.
Last year the House of Representatives passed its version of “21st Century Cures” by a vote of 344-77. As companion legislation, our Senate health committee has approved 50 bipartisan strategies designed to make predictions like those of Dr. Collins come true. These include faster approval of breakthrough medical devices like the highly successful breakthrough path of medicines enacted in 2012 and making the problem-plagued electronic health records system interoperable, less burdensome for doctors and more available to patients. We approved measures to target rare diseases and runaway superbugs that resist antibiotics. We would make it easier for the National Institutes of Health and Food and Drug Administration to hire experts needed to supervise research and evaluate safety and effectiveness.
The House also has approved an $8.8 billion multi-year surge of funding for the National Institutes of Health. The Senate is working on a similar “NIH Innovation Fund” focused on the President’s Precision Medicine Initiative, the Vice-President’s Cancer Moonshot and the NIH Brain Initiative.
In this year of political turmoil, the “21st Cures” Act offers a welcome opportunity for consensus. We will succeed because we know that there never has been a moment when our actions might so profoundly improve the health of virtually every American. We know also, as Dr. Collins says, that for hundreds of millions the National Institutes of Health is really the “National Institute of Hope.”