Looking to the future with Dr. Francis Collins

What gets the leader of the NIH jazzed?

Speaking to a packed West Pavilion auditorium March 6, Francis Collins, M.D., Ph.D., director of the National Institutes of Health, shared his picks of 10 “areas of particular excitement and promise” in biomedical research. (Watch the full talk here.)

In nearly every area, UAB scientists are helping to lead the way — as Collins himself noted in several cases. At the conclusion of his talk, Collins added his advice for young scientists. Here is Collins’ top 10 list, annotated with some of the UAB work ongoing in each area and ways that faculty, staff and students can get involved.

1. Single-cell sequencing 

[see this section of the talk here]  

“I am so jazzed with what has become possible with the ability to study single cells and see what they are doing,” Collins said. “They have been out of our reach — now we have reached in. Whether you are studying rheumatoid arthritis, diabetes or the brain, you have the chance to ask each cell what it is doing.”

Single-cell sequencing and UAB: Collins noted that Robert Carter, M.D., the acting director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, was a longtime faculty member at UAB (serving as director of the Division of Clinical Immunology and Rheumatology). For the past several years, UAB researchers have been studying gene expression in subpopulations of immune cells in patients with rheumatoid arthritis.

Join in: Researchers can take advantage of the single-cell sequencing core facility in UAB’s Comprehensive Flow Cytometry Core, directed by John Mountz, M.D., Ph.D., Goodwin-Blackburn Research Chair in Immunology and professor in the Department of Medicine Division of Clinical Immunology and Rheumatology.

Learn more: Mountz and other heavy users of single-cell sequencing explain how the techniques let them travel back in time and more in this UAB Reporter story.

2. New ways to see the brain 

[See this section of the talk here] 

The NIH’s BRAIN Initiative is making this “the era where we are going to figure out how the brain works — all 86 billion neurons between your ears,” Collins said. The linchpin of this advance will be the development of tools to identify new brain cell types and circuits that will improve diagnosis, treatment and prevention of autism, schizophrenia, Parkinson’s and other neurological conditions, he said.

Brain tech and UAB: Collins highlighted the work of BRAIN Initiative grantee Harrison Walker, M.D., an associate professor in the Department of Neurology, whose lab “has been developing a more sophisticated way to understand the benefits of deep brain stimulation for people with Parkinson’s and maybe other conditions,” Collins said.

Join in: UAB’s planned new doctoral program in neuroengineering would be the first of its kind in the country.

Learn more: Find out why neuroengineering is a smart career choice in this UAB Reporter story.

3. Induced pluripotent stem (iPS) cells 

[See this section of the talk here] 

Researchers can now take a blood cell or skin cell and, by adding “four magic genes,” Collins explained, induce the cells to become stem cells. These induced pluripotent stem (iPS) cells can then in turn be differentiated into any number of different cell types, including nerve cells, heart muscle cells or pancreatic beta cells. The NIH has invested in technology to put iPS-derived cells on specialized tissue chips. “You’ve got you on a chip,” Collins explained. “Some of us dream of a day where this might be the best way to figure out whether a drug intervention is going to work for you or you’re going to be one of those people that has a bad consequence.”

iPS cells at UAB: Collins displayed images of the cutting-edge cardiac tissue chip developed by a UAB team led by Palaniappan Sethu, Ph.D., an associate professor in the Department of Biomedical Engineering and the Division of Cardiovascular Disease. The work “allows the development of cardiomyocytes that can be used to study heart failure” and other conditions, Collins said.

Join in: UAB’s biomedical engineering department, one of the leading recipients of NIH funding nationally, is a joint department of the School of Engineering and School of Medicine. Learn more about UAB’s undergraduate and graduate programs in biomedical engineering, and potential careers, here.

Learn more: See how this novel bioprinter developed by UAB biomedical researchers is speeding up tissue engineering in this story from UAB News.

4. Microbiome advances

[See this section of the talk here] 

“We have kind of ignored the fact that we have all these microbes living on us and in us until fairly recently,” Collins said. But now it is clear that “we are not an organism — we are a superorganism” formed with the trillions of microbes present in and on our bodies, he said. This microbiome plays a significant role not just in skin and intestinal diseases but “much more broadly.”

Microbiome at UAB: Collins explained that work led by Casey Morrow, Ph.D., and Casey Weaver, M.D., co-directors of the Microbiome/Gnotobiotics Shared Facility, has revealed intriguing information about how antibiotics affect the gut microbiome. Their approach has potential implications for understanding, preserving and improving health, Collins said.

Join in: Several ongoing clinical trials at UAB are studying the microbiome, including a study modifying diet to improve gut microbiota and an investigation of the microbiomes of postmenopausal women looking for outcomes and response to estrogen therapy.  

Learn more: This UAB News story explains the UAB research that Collins highlighted.

5. Influenza vaccines

[See this section of the talk here] 

Another deadly influenza outbreak is likely in the future, Collins said. “What we need is not an influenza vaccine that you have to redesign every year, but something that would actually block influenza viruses,” he said. “Is that even possible? It just might be.”

Influenza research at UAB: We’re probably at least a decade away from a universal influenza vaccine. But work ongoing at UAB in the NIH-funded Antiviral Drug Discovery and Development Center (AD3C), led by Distinguished Professor Richard Whitley, M.D., is focused on such an influenza breakthrough.

Join in: For now, the most important thing you can do to stop the flu is to get a flu vaccination. Employees can schedule a free flu vaccination here.

Learn more: Why get the flu shot? What is it like? How can you disinfect your home after the flu? Get all the information at this comprehensive site from UAB News.

6. Addiction prevention and treatment of pain 

[See this section of the talk here] 

The NIH has a role to play in tackling the crisis of opioid addiction and deaths, Collins said. The NIH’s Helping to End Addiction Long-term (HEAL) initiative is an “all-hands-on-deck” effort, he said, involving almost every NIH institute and center, with the goal of uncovering new targets for preventing addiction and improving pain treatment by developing non-addictive pain medicines.

Addiction prevention at UAB: A big part of this initiative involves education to help professionals and the public understand what to do, Collins said. The NIH Centers of Excellence in Pain Education (CoEPE), including one at UAB, are hubs for the development, evaluation and distribution of pain-management curriculum resources to enhance pain education for health care professionals.

Join in: Find out how to tell if you or a loved one has a substance or alcohol use problem, connect with classes and resources or schedule an individualized assessment and treatment through the UAB Medicine Addiction Recovery Program.

Learn more: Discover some of the many ways that UAB faculty and staff are making an impact on the opioid crisis in this story from UAB News.

7. Cancer Immunotherapy 

[See this section of the talk here] 

“We are all pretty darn jazzed about what’s happened in the past few years in terms of developing a new modality for treating cancer — we had surgery, we had radiation, we had chemotherapy, but now we’ve got immunotherapy,” Collins said.

Educating immune system cells to go after cancer in therapies such as CAR-T cell therapy is “the hottest science in cancer,” he said. “I would argue this is a really exciting moment where the oncologists and the immunologists together are doing amazing things.”

Immunotherapy at UAB: “I had to say something about immunology since I’m at UAB given that Max Cooper, who just got the Lasker Award for [his] B and T cell discoveries, was here,” Collins said. “This is a place I would hope where lots of interesting ideas are going to continue to emerge.”

Join in: The O’Neal Comprehensive Cancer Center at UAB is participating in a number of clinical trials of immunotherapies. Search the latest trials at the Cancer Center here.

Learn more: Luciano Costa, M.D., Ph.D., medical director of clinical trials at the O’Neal Cancer Center, discusses the promise of CAR-T cell therapy in this UAB MedCast podcast.

Assistant Professor Ben Larimer, Ph.D., is pursuing a new kind of PET imaging test that could give clinicians a fast, accurate picture of whether immunotherapy is working for a patient in this UAB Reporter article.

8. Tapping the potential of precision medicine 

[See this section of the talk here] 

The All of Us Research Program from NIH aims to enroll a million Americans — to move away from the one-size-fits-all approach to medicine and “really understand individual differences,” Collins said. The program, which launched in 2018 and is already one-third of the way to its enrollment goal, has a prevention rather than a disease treatment approach; it is collecting information on environmental exposures, health practices, diet, exercise and more, in addition to genetics, from those participants.

All of Us at UAB: “UAB has been doing a fantastic job of enrolling participants,” Collins noted. In fact, the Southern Network of the All of Us Research Program, led by UAB, has consistently been at the top in terms of nationwide enrollment, as School of Medicine Dean Selwyn Vickers, M.D., noted in introducing Collins.

Join in: Sign up for All of Us at UAB today.

Learn more: UAB’s success in enrolling participants has led to a new pilot study aimed at increasing participant retention rates.

9. Rare diseases 

[See this section of the talk here] 

Rare Disease Day, on Feb. 29, brought together hundreds of rare disease research advocates at the NIH, Collins said. “NIH needs to play a special role” because many diseases are so rare that pharmaceutical companies will not focus on them, he said. “We need to find answers that are scalable, so you don’t have to come up with a strategy for all 6,500 rare diseases.”

Rare diseases at UAB: The Undiagnosed Diseases Network, which includes a UAB site led by Chief Genomics Officer Bruce Korf, M.D., Ph.D., is a national network that brings together experts in a wide range of conditions to help patients, Collins said.

Participants in the Alabama Genomic Health Initiative, also led by Korf, donate a small blood sample that is tested for the presence of specific genetic variants. Individuals with indications of genetic disease receive whole-genome sequencing. Collins noted that lessons from the AGHI helped guide development of the All of Us Research Program.

Collins also credited UAB’s Tim Townes, Ph.D., professor emeritus in the Department of Biochemistry and Molecular Genetics, for developing “the most significantly accurate model of sickle cell disease in a mouse which has been a great service to the [research] community.” UAB is now participating in an exciting clinical trial of a gene-editing technique to treat sickle cell along with other new targeted therapies for the devastating blood disease.

Join in: In addition to UAB’s Undiagnosed Diseases Program (which requires a physician referral) and the AGHI, patients and providers can contact the UAB Precision Medicine Institute, led by Director Matt Might, Ph.D. The institute develops precisely targeted treatments based on a patient’s unique genetic makeup.

Learn more: Discover how UAB experts solved medical puzzles for patients by uncovering a never-before-described mutation and cracking a vomiting mystery in these UAB News stories.

10. Diversity in the scientific workforce 

[See this section of the talk here]

“We know that science, like everything else, is more productive when teams are diverse than if they are all looking the same,” Collins said. “My number one priority as NIH director is to be sure we are doing everything we can to nurture and encourage the best and brightest to join this effort.”

Research diversity at UAB: The Neuroscience Roadmap Scholars Program at UAB, supported by an NIH R25 grant, is designed to enhance engagement and retention of under-represented graduate trainees in the neuroscience workforce. This is one of several UAB initiatives to increased under-represented groups and celebrate diversity. These include several programs from the Minority Health and Health Disparities Research Center that support minority students from the undergraduate level to postdocs; the Partnership Research Summer Training Program, which provides undergraduates — and especially minority students — with the opportunity to work in UAB cancer research labs; the Dean’s Excellence Award in Diversity in the School of Medicine; and the newly announced Underrepresented in Medicine Senior Scholarship Program for fourth-year medical students.

Join in: The Roadmap program engages career coaches and peer-to-peer mentors to support scholars. To volunteer your expertise, contact Madison Bamman at [email protected] or visit the program site.

Learn more: Farah Lubin, Ph.D., associate professor in the Department of Neurobiology and co-director of the Roadmap Scholars Program, shares the words and deeds that can save science careers in this Reporter story. In another story, Upender Manne, Ph.D., professor in the Department of Pathology and a senior scientist in the O’Neal Comprehensive Cancer Center, explains how students in the Partnership Research Summer Training Program get hooked on cancer research.

 

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