In the symposium given at SMU in honor of NIH Director Francis S. Collins, our 2017 Commencement Speaker and honorary degree recipient, Dr. Collins discussed 10 exceptional opportunities in biomedical research that might come to fruition within ten years.
Before going over his top 10 list, he pointed out that N.I.H.'s mission is to both "seek fundamental knowledge about the nature and behavior of living systems" as well as "the application of that knowledge to enhance health, lengthen life, and reduce illness and disability, and remarked that Texas received over $1bn in FY16. He also discussed statistics that suggest N.I.H. money has been rather well spent: (a) cancer death rates are now falling more than 1% per year, while each 1% drop saves $500bn. (b) cardiovascular disease death rates have fallen more than 70% in the last 60 years, and (c) HIV therapies now enable people in their 20s to live to age 70+.
On to his list of 10 exceptional opportunities. In ten years, we might have:
- advanced our analysis of individual human cells, which will help understand many disorders like lupus and rheumatoid arthritis,
- developed tools to identify new brain cell types and circuits, to help diagnosis, treatment and prevention of autism,
- identified those at risk for Alzheimer's before signs appear. Dr. Collins showed brain cells of six members of a family in South America known to be at risk of hereditary Alzheimer's, 4 with the amyloid gene carriers and 2 without, the six members are in the 35-39 range and Alzheimer's in their family has tended to have onset when people reached their 40s, and those brain scans showing the amyloid build-up in the 4 people at risk were breathtaking - Dr. Collins also mentioned that a possible reason why Alzheimer's treatments may not have been successful so far is that such treatments may have targeted people whose disease was too far advanced, but there is real hope if we are able to take action before the onset of symptoms. This is critical because the amount of money required to take care of the growing Alzheimer's population by 2050 will be equivalent to two Department of Defense budgets if we don't do anything.
- developed effective treatment for spinal cord injuries.
- optimized and widely distributed an artificial pancreas for diabetes. This is actually not that far in the distance since the FDA approved in September 2016 the first hybrid closed-loop system to track changes in blood glucose levels and provide precise doses of insulin.
- generated new organs from induced pluripotent stem cells.
- created a universal influenza vaccine. The idea is to teach the immune system to attack the invariant stem of the virus (instead of the head, which varies from year to year), in order to provide immunity to all flu viruses. We are overdue for a worldwide flu pandemic, so a universal influenza vaccine would be critical in mitigating such a pandemic's consequences.
- deployed genomics, neuroscience, structural biology to uncover new targets for treatment of pain. The U.S. are in the middle of an opioid overdose epidemic, and 80% of people who ODed got started with a legitimate prescription for pain treatment, and some of those then switched to heroin, which apparently is very cheap, when OxyContin or similar was no longer readily available. (The prescription for the pain medication may be for 30 days, but people might feel very lousy when they stop, so they try to continue a little while longer, and when they really can't get a prescription renewal, those who are addicted switch to heroin.) There is a critical need to develop non-addictive pain medicine.
- accelerated immunotherapy and other advances through the Cancer Moonshot. Dr. Collins discussed the case of Emily Whitehead in PA, who had a type of childhood cancer for whom traditional treatments had not worked, and who was saved thanks to immunotherapy.
- actualized the potential of precision medicine through the "All of Us" research program, which aims at enrolling 1m Americans for a long-term research study combining data science, genomics, Electronic Health Records, technologies and patient partnerships. You can learn more about it at joinallofus.org .
Gene editing, which could have made the top 10 list, was discussed in the Q&A.
Additional topics discussed in the Q&A were (I wish I could provide details on all of them, but this post is already long enough):
- the WaPo op-ed by Eric Lander of the Broad Institute and Eric Schmidt of Google "America's "Miracle Machine" is in desperate need of a miracle"
- Dr. Collins's meeting at the White House two weeks ago with senior WH officials and heads of industry and academia to discuss how to best foster a productive relationship between government agencies, corporations and universities regarding scientific research (following by a short meeting with the President afterward), and especially NIH's Accelerating Medicines Partnership, which was launched in February 2014 with the goal to "transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets for therapeutics" in 3 disease areas: Alzheimer's disease, type 2 diabetes and autoimmune disorders (lupus and rheumatoid arthritis). Dr. Collins also mentioned Parkinson's as a current focus, and said that cancer might get added soon.
- the need for an interdisciplinary approach to many of those problems, and the potential of computational biology.
- the issue that over 60% of drugs now coming on the market first originated from academic research - why should N.I.H. fund this work if big pharma reaps the rewards? Dr. Collins talked about the National Center for Advancing Translational Science established in 2012 and the role of the Bayh-Dole Act in encouraging universities to file for patents, which also means that the N.I.H. holds no claims to those discoveries.
- drug pricing, which the N.I.H. has no role in.
- the issue of drug-resistant bacteria, when a drug can take 12 years to be developed and a bacteria can take 2 years to become drug-resistant, the related need to limit the prescription of antibiotics, and the fact that biopharma can make a lot more money developing, say, cancer drugs.
- Dr. Collins's book on "The Language of God" and his personal faith. He said he still gets emails almost every week, especially from young Christian students, who have been homeschooled and then are presented with the evidence for evolution in their first biology course in college and suddenly put their faith back in question. He said he wasn't a believer in college, only became at 27 in medical school, and insisted that science and faith aren't mutually exclusive.
- diabetes. 80m people in the U.S. are pre-diabetic, but diet and exercise with a coach has been proved to lead to a 58% reduction in transition rates to the diabetic stage. There are data privacy issues because it is generally agreed that you should not have your genome used against you for jobs and health insurance. Interestingly, a bill about wellness programs currently under consideration could threaten that because some of those programs ask for DNA analysis and if you refuse, they're allowed to charge you a premium that is 30% higher than the premium if you had accepted.
- the need to do healthcare more efficiently, since about 30% may be wasted now due to improper testing etc.
- the future of healthcare, about which Dr. Collins mostly said (as a response to an audience question) that his crystal ball was rather cloudy, and that most people agree that kids who get sick should be able to get health care.
- The Q&A ended with the question about science funding, which was the topic of my previous post.
You can read SMU's release about N.I.H. Director Francis S. Collins being chosen as 2017 Commencement Speaker here. It is so exciting for SMU but also speaks volumes of its national reputation that we were able to attract such a high-caliber speaker to Dallas. I feel really blessed to have heard Dr. Collins give such an informative talk at the symposium and am looking forward to his Commencement speech tomorrow.