ASCI Perspectives: Kirsten Bibbins-Domingo, MD, PhD, MAS – video clip

At the 2024 AAP/ASCI/APSA Joint Meeting in Chicago, Dr. Kirsten Bibbins-Domingo (view profile) sat down with DEIC member Vijay Sankaran, MD, PhD, to discuss her route to becoming a scientist and clinician; expanding public trust in medical communication; and the value of a mentorship “board of directors.” Dr. Bibbins is the Lee Goldman, MD Endowed Professor of Medicine and Professor of Epidemiology and Biostatistics at the University of California, San Francisco, and Editor in Chief of JAMA and the JAMA network. Her research in cardiovascular disease epidemiology combines observational studies, trials, and simulation modeling to examine clinical, public health, and policy interventions aimed at prevention. — Posted April 2024

Click the image below for a video clip of the interview. (For the full interview, click here; 23 minutes.)

ASCI Perspectives: Kirsten Bibbins-Domingo, MD, PhD, MAS – full video

At the 2024 AAP/ASCI/APSA Joint Meeting in Chicago, Dr. Kirsten Bibbins-Domingo (view profile) sat down with DEIC member Vijay Sankaran, MD, PhD, to discuss her route to becoming a scientist and clinician; expanding public trust in medical communication; and the value of a mentorship “board of directors.” Dr. Bibbins is the Lee Goldman, MD Endowed Professor of Medicine and Professor of Epidemiology and Biostatistics at the University of California, San Francisco, and Editor in Chief of JAMA and the JAMA network. Her research in cardiovascular disease epidemiology combines observational studies, trials, and simulation modeling to examine clinical, public health, and policy interventions aimed at prevention. — Posted April 2024

Click the image below for the full interview. (For a video clip, click here; 4 minutes.)

ASCI Perspectives: Courtney D. Fitzhugh, MD – video clip

In this Perspectives video, DEIC member Vijay Sankaran, MD, PhD, interviewed Dr. Courtney D. Fitzhugh, Chief of the Laboratory of Early Sickle Mortality Prevention and Lasker Clinical Research Scholar at the National Heart, Lung, and Blood Institute, NIH. Dr. Fitzhugh studies how stem cell transplantation can be applied using allogeneic and autologous sources to cure sickle cell disease, as well as the long-term health effects of SCD therapies. In this interview, she discusses her research on SCD along with her passion for patient advocacy. — Posted March 2024

Click the image below for a video clip of the interview. (For the full interview, click here; 13 minutes.)

ASCI Perspectives: Courtney D. Fitzhugh, MD – full video

In this Perspectives video, DEIC member Vijay Sankaran, MD, PhD, interviewed Dr. Courtney D. Fitzhugh, Chief of the Laboratory of Early Sickle Mortality Prevention and Lasker Clinical Research Scholar at the National Heart, Lung, and Blood Institute, NIH. Dr. Fitzhugh studies how stem cell transplantation can be applied using allogeneic and autologous sources to cure sickle cell disease, as well as the long-term health effects of SCD therapies. In this interview, she discusses her research on SCD along with her passion for patient advocacy. — Posted March 2024

Click the image below for the full interview. (For a video clip, click here; 3 minutes.)

Transcript of ASCI Perspectives – Bibbins-Domingo interview

Interview with Kirsten Bibbins-Doming MD, PhD, MAS, University of California, San Francisco (elected 2010), April 6, 2024, at the ASCI Annual Meeting, Chicago
Interviewed by Vijay Sankaran, MD-PhD, (elected 2018)
; member, ASCI Diversity, Equity, and Inclusion Committee

Note: The text has been edited for readability by ASCI staff.

Vijay Sankaran: Welcome to this ASCI Perspectives interview. I’m Vijay Sankaran from Boston Children’s Hospital and Harvard Medical School. It’s my distinct pleasure to have as today’s guest Dr. Kirsten Bibbins-Domingo. Dr. Bibbins-Domingo is the Lee Goldman, MD Endowed Professor of Medicine and Professor of Epidemiology and Biostatistics at the University of California, San Francisco. She’s currently the 17th editor in chief of the Journal of the American Medical Association (JAMA) and the JAMA Network. Dr. Bibbins-Domingo was previously the inaugural Vice Dean for Population Health and Health Equity at UCSF, as well as the chair of the Department of Epidemiology and Biostatistics from 2017 to 2022. She’s a leader at identifying preventive strategies and interventions to address health disparities. Dr. Bibbins-Domingo is an elected member of the American Society for Clinical Investigation, the Association of American Physicians, the National Academy of Medicine, and the American Academy of Arts and Sciences. Dr. Bibbins-Domingo, welcome to this Perspectives interview.

Kirsten Bibbins-Domingo: Thank you so much for having me.

VS: To begin with, I was wondering if you could talk a little bit about your career path, the experiences you’ve had during your training, and also what got you interested in science and medicine.

KBD: Yeah, sure. Well, I love being part of this interview, because I have a PhD and an MD. Being a physician-scientist is very much part of my identity and what my career has been prior to joining JAMA. But I did it a little bit out of order. In fact, when I came to medical school, I didn’t know much about what, well . . . when I was an undergraduate, I didn’t know much about medicine, and I didn’t know much when people were asking me if I was premed. I didn’t exactly know what that was. I knew I love science, and frankly, people seemed a little bit weird to me who were asking me if I was premed. So I said, “No, for sure I’m not going to medical school. I’m doing science.” So I did my PhD first. Actually, I worked with Harold Varmus before he went to become the head of the NIH. But I was always interested in the application of the science. I loved doing the science, but I was interested more in the application. And so I went to medical school at UCSF after finishing my PhD and then switched the type of research that I was doing from being a bench scientist, a basic scientist, to doing more population and clinical research. And so the path for me has really been very much about being a scientist, loving the asking and answering questions that comes with science, but also then always trying to think about the applications to human health. And it’s the thing that I spent most of my career at UCSF doing is thinking along those dimensions.

VS: And I was wondering if you could talk a little bit more to the role that different mentors have had at different stages in your career as you’ve made these various transitions and as you’ve kind of taken this path as a physician-scientist.

KBD: Yeah. I have to say, I’ve benefited — as many of us do who get to at different points in their career — from really outstanding mentors. I just talked about Harold Varmus, who was my graduate advisor and has really been there at different points in my career. When I became the editor in chief of JAMA — he has a lot of ideas about scientific publication, about open access. He did the first interview that I did as an editor in chief with me, and he’s been really terrific. Lee Goldman was the head of medicine at UCSF when I was in training, became the dean at Columbia, and is one of those people who gave me advice and then was a research mentor as I switched to cardiovascular disease modeling. I worked with him closely there. And [he] has continued to be a really important person in how I think, not just about research, but also about the career that we all have in science and medicine.

I’m fortunate to have his name now, as I’m the Lee Goldman Professor of Medicine, as you said at the outset. And then I’ve had other people along the way in epidemiology, people who’ve helped me understand what it’s like to be a woman as a leader in an academic institution. And I like to say that not all of my mentors necessarily agree all the time. And I don’t need people to agree all the time. I need people to be interested in me and really aware of what’s going on and being generous in giving advice and generous in opening doors. And I’ve been really fortunate to benefit from those. But sometimes you have to still make your own decision when people disagree on what it is they think is the next step for you.

VS: Absolutely. Yeah, it’s such great advice for, I think, so many of our trainees as you reflect upon your career in research. I know you had done a lot of work to try to develop insights to develop actionable approaches to address health disparities and to increase health equity. I was wondering if you could speak to where you see that field going and what advances you’re excited about in that area.

KBD: Yeah. I think we have hopefully moved beyond the descriptions of the fact that although we hopefully aspire to all patients being treated equally when they enter in our clinical settings, of all communities experience[ing] having an equal chance of achieving a long and healthy life, that really is not the case; that both in general life is full of structural and social inequities: even within our clinical settings, the chance that somebody comes in and has exactly the same outcome — they’re very different based on a lot of other things related to what a patient brings with them when they come into our setting and how we have systems that are also biased. What I’d like to see, and I think where we are now, is we’re starting to look at interventions and how do we think about interventions.

How do we think about using lots of different information about patients, about communities, but also looking closely at “How do we challenge the systems and the care processes that we have in clinical settings to make sure that we don’t fall into those patterns that lead to inequitable outcomes.” So I’m very interested in interventions. I’m very interested in areas that help us to understand the social and structural inequities that contribute to the different outcomes that we see. And I think it’s an exciting time when you look at different data sources that we can come together. When I started in this field, I would say probably the number of people who thought about disparities or health equity was a small niche. And now pretty much people can speak with some degree of sophistication. They understand that this is an issue that we should try to address. And so I think that bodes well for interventions. If I’d say the one area I would like to see more of: I really appreciate the interest in social and structural inequities in health, but I think as physicians, we should be personally offended that people have a different chance of having good outcomes when they come into clinical settings. And I think we should own more, that we should think about those interventions that make sure that that is not the case in our own clinical settings.

VS: Absolutely. Wow. I was wondering if we could just backtrack a little bit in your own research career, because you’ve done different types of research at different stages of your career. And I think it’s quite valuable as trainees are thinking about maybe making switches. And I was wondering if you could talk a little bit about how you transitioned from some of the work you were doing as a PhD student with Harold Varmus to some of the work that you did to really address epidemiologic questions. And I was wondering sort if you could talk more to that.

KBD: Yeah. The advice I usually give to people who are thinking about these careers — these careers are long, as you know; you’ve trained a long time as well. But I think they’re so satisfying as careers. If you are driven by the questions you want to address using various types of scientific methods, I think early on in your career the most important thing is to be in a place with a good mentor. I think that’s probably as important, if not more important, than the specific question you’re trying to address. Because understanding the how to ask and answer a question, understanding what it is, what the scientific process is, I think that’s the most important thing. I think later on . . . what I think people should do more of is to pay attention to those things that really give you satisfaction and the types of ways of asking and answering a scientific question that are most compatible with the way in which you want to build a career and not to be worried about switching, not to be worried about — you don’t want to switch all the time — but not to see as a barrier.

We go into these careers, we have so many more options available to us than we sometimes perceive at the given point in time. And I think you have a lot of options available to you as a physician. You have a lot of options available to you when you are trained with the rigorous scientific methods. And then thinking about how those things come together to build a career: that’s how I think we have to think. Because ultimately the career is long, and the training is long, and you want to do it in a way that you can combine those that is personally fulfilling to you, that is compatible with having a job and where you want to have a job. And I think that’s how to think of these as sustainable careers is to think more about the options you have within the ways you train, as opposed to just that there’s one path you always have to be down.

VS: Absolutely. Wow. Such a great perspective. And I think it really sort of reflects on the idea of seasons in your career and how you can have those different seasons as well.

KBD: Absolutely. Absolutely.

VS: So, switching gears a little bit. You’ve become the editor in chief of the Journal of the American Medical Association, and I was wondering if you could speak a little bit to what trends you’re excited about as you think about the broader landscape of medical and scientific publishing and where you see the field evolving and going. And I know you talked a little bit about it during your fantastic lecture this morning at the ASCI meeting [2024 AAP/ASCI/APSA Joint Meeting], but I was wondering if you could talk more about that.

KBD: Yeah. I mean, I became the editor in chief, and I hadn’t had editorial experience at all, but I’ve always been interested in how science is communicated. And I think we’re at a time when we are sort of drowning with information right now. We’re drowning with information in all of the ways we train in and the ways we keep up with our specialties and the information from the lay press. And I think this is such an interesting time to think through “How do you communicate science within this noise of everything else that’s going on within an environment where people have opinions in the sort of the general public about. Do we trust science? Do we not trust science?”

Even us as physician-scientists, how do we keep up with our specialties and with the latest scientific innovation. And so I think in some ways, I think of this time as being — it’s a very interesting, somewhat daunting, definitely exciting time to be in medical publishing. Because in some ways we have to double down on doing the thing that we’ve always done at top medical journals, which is really to do rigorous vetting of science, to think about how science and all of its nuances is communicated to a broad audience, thinking about how to communicate to clinicians as well as to scientists. Because both audiences are important to a journal like JAMA. And to do that in a way that maintains the integrity of and ultimately the trust of people who are reading the work. So in some ways, we have to double down on the same thing we’ve always done. And in some ways, we have to be completely innovative, to think of: you’re reading journals, people are reading journals — I don’t know whether you are — but people are reading journals on their phone. People are reading journals in the clinic. People are reading journals in different places. People are on social platforms, or they might be watching a video summary. I didn’t realize people listen to podcast summaries until I started at JAMA, but people do. And so we have to think about all of those many tools that are available for communicating about science but be clear on our core mission, which is doubling down on making sure we can trust what it is that — when we’re publishing it, when others are publishing it, that we can trust what we’re reading.

VS: Absolutely. And I know this morning during your talk, you also discussed the variation amongst different groups in trusting the science. And I was wondering if you could comment a little bit on how you think scientific communication or medical communication can be improved potentially to try to address some of those issues, particularly the distrust that some groups might have towards medical . . .

KBD: Yeah. So I said that when I think of our audience — our audience is definitely clinicians and definitely scientists, but of course our audience is also the broader public, right? So, if we are publishing a top paper, it’s going to be picked up in the newspaper, it will be in the lay discussion, it will influence policymakers, it will influence health systems leaders. So we have to keep that in mind. And the good thing, as I talked about this morning, is that in general, physicians are trusted people within civic society in the US. Scientists are as well. But we also know there’s a growing mistrust of experts, mistrust of science. And so it’s interesting to think about communicating science in that type of environment. I sort of think . . . I go back to: So I’m a general internist. I believe I can talk to a patient even if they don’t totally believe what I’m offering them right now. That I can come to framing it in a different way, understanding what motivates them, thinking, coming back if we need to come back, to circle back to that advice that I want to give them as a physician. What I’ve been trying to figure out how to do and what we are trying to do at JAMA is figure out how do we do that at scale? Like, how do we have that type of openness at scale to not be: “This is what JAMA says, and so therefore we should do it.” But to say: “Here’s the scientific study”; to recognize there’s always nuances in this.

So we’ve done a lot with producing editorials around complicated scientific information to come at it from different angles. So I want to invite more people into the conversation that we’re having about a particular scientific finding. You’ll see us using not just more editorials, but also more in multimedia to frame different ways of talking about a particular scientific finding — for different platforms, but sometimes framed in slightly different ways. I think there are different ways that people come into a particular disease process or understanding what a recommendation is, and we’re trying to use different ways of making it interesting, not assuming that everyone comes with the same frame that we are coming at it with. And so for me, it’s just about inviting more people into the discussion. And I think that’s what we need. It’s not easy to do. And I don’t think anyone has the game plan on how to do it. But I’m pretty sure that bringing, inviting more people in has got to be part of it. Because I think the finger wagging and saying, “No, no, it’s this way, not this way” is probably not going to help to engender trust.

VS: Absolutely. And it’s really important as we think about communicating these ideas, making sure we have a great conversation with so many people.

KBD: Exactly.

VS: So, many of our viewers of this interview will be trainees at different stages of training to become physician-scientists. And I was wondering: You talked a little bit about some of the advice and some of your own experiences, but what sort of advice do you have based upon your own experiences at different stages of your training that you think could be valuable.

KBD: Find those good mentors and keep them. I think about mentors as people who are interested in you, but I also think you can have multiple people who play some sort of role in helping guide your career. The motif that’s always been helpful for me is to think of having a board of directors of people who are going to help you out along the way. But you yourself have to make decisions ultimately so that you have people who are going to help you and are going to help at different ways, at different stages in your career. But you ultimately have to also know what’s important to you. I will say for me, I started a family when I was in medical school. So for me, I knew that was also important to me. And I made many decisions that involved not moving because of family commitments and things like that.

You have to understand yourself as much as you have to understand the advice that somebody else is giving you. So I think that’s important. I think these careers are long. I would urge people not to think of waiting until the very end — that the journey is in fact the thing we are living, the thing we’re trying to do all the time. We do that even when we’re in training. So being involved in understanding what science means, what clinicians are talking about, it’s one of the reasons this meeting is so great, because I think it immerses those in training within the conversations that people who are at many different stages in their careers are having.

And so I think that’s the way you have to approach this, because the career is long, right? And the training is long. And so to think of yourself as a part of this great biomedical-physician-science ecosystem and journey, even when you’re early in your training, I think is important. And then as we talked about before, there are many different paths, there are many different ways. People are working in so many different types of environments that understanding what’s available and to realize you have many doors open to you, I think, is also important.

VS: Absolutely. And I guess one other thing that you’d mentioned earlier that really resonated, at least in my experiences, is you have a panel of mentors, but you might not always agree with some of your mentors. And I think that’s something valuable to sort of, that I would love to maybe just, dial into a little bit more.

KBD: Absolutely. And, I mean, there is value as many people who are training a long time, you’re at a point if you’re . . . I spoke to some MD-PhD students earlier today. One of them is the age of my son, who’s 28. And there are people who are in their late 20s who’ve started startups and who started businesses. So you have agency even though you are a person in training. You have agency. By the time you’re in your late 20s, you’ve had a variety of experiences. You hopefully know yourself a little bit. And so I don’t think . . . I think mentors, hopefully, they have strong opinions, and they don’t have to always agree. And I think to be a good . . . to get the most out of that mentoring relationship, you have to also know yourself and understand yourself. And that doesn’t mean you have to have a disagreement. It means you have to understand you’re asking a person to advise you, to give you their best advice, to hopefully help you to learn the skills for the things you want to do. But ultimately, it’s: You have to decide how you’re going to take that advice and use it to build the career that you want to have.

And I have to say, I’ve been fortunate. In some ways, I sometimes say that my many mentors could sometimes probably not even be in the same room together. That’s the degree to which they might have a different perspective. But they’ve all been so helpful to me in learning the skills I needed and understanding where opportunities were and helping open doors that I wouldn’t have it any other way. But part was learning along the way: who I was going to . . . whose advice resonated most with where I thought I needed to go. And you have agency at this stage, so you should use that as well.

VS: Wow. Very valuable advice, I think for me and for I’m sure a lot of our listeners. Well, thank you so much. This has been a really insightful interview and I really appreciate your time Dr. Bibbins-Domingo. Thank you very much.

KBD: Wonderful. It’s been a pleasure talking with you.

ASCI Perspectives: Juan Pablo Arroyo, MD, PhD – video clip

DEIC member Jennifer S. Yu interviewed Juan Pablo Arroyo, MD, PhD, Assistant Professor of Medicine at Vanderbilt University Medical Center and recipient of a 2022 ASCI Emerging Generation Award. Dr. Arroyo’s work focuses on how the kidney regulates fluids and electrolytes. His research group has recently shown that kidney epithelial cells make biologically active vasopressin. In this video, Dr. Arroyo discusses his circuitous career path and the importance of forefronting kindness. — Posted February 2024

Click the image below for a video clip of the interview. (For the full interview, click here; 15 minutes.)

ASCI Perspectives: Juan Pablo Arroyo, MD, PhD – full video

DEIC member Jennifer S. Yu interviewed Juan Pablo Arroyo, MD, PhD, Assistant Professor of Medicine at Vanderbilt University Medical Center and recipient of a 2022 ASCI Emerging Generation Award. Dr. Arroyo’s work focuses on how the kidney regulates fluids and electrolytes. His research group has recently shown that kidney epithelial cells make biologically active vasopressin. In this video, Dr, Arroyo discusses his circuitous career path and the importance of forefronting kindness. — Posted February 2024

Click the image below for the full interview. (For a video clip, click here; 4 minutes.)

Transcript of ASCI Perspectives – Fitzhugh interview

Interview with Courtney D. Fitzhugh, MD, National Heart, Lung, and Blood Institute, NIH (elected 2024)
Interviewed by Vijay Sankaran, MD-PhD, (elected 2018)
; member, ASCI Diversity, Equity, and Inclusion Committee

Note: The text has been edited for readability by ASCI staff.

Vijay Sankaran: Welcome to this ASCI Perspectives interview. My name is Vijay Sankaran from Boston Children’s Hospital and Harvard Medical School. It is my distinct pleasure to have as today’s guest for our Perspectives interview Dr. Courtney Fitzhugh. Dr. Fitzhugh is a Lasker Clinical Research Scholar and heads the Laboratory of Early Sickle Cell Mortality Prevention at the National Hear t, Lung, and Blood Institute. She’s a pioneer in advancing the use of hematopoietic stem cell transplantation in sickle cell disease. Dr. Fitzhugh earned her medical degree at the University of California, San Francisco. While in medical school, Dr. Fitzhugh participated in the National Institutes of Health Clinical Research Training Program and worked with John Tisdale during this time. After completing her MD, Dr. Fitzhugh did a combined internal medicine and pediatrics residency at Duke University Medical Center, and then did a combined adult and pediatric hematology fellowship at the NIH and Johns Hopkins Hospital. Dr. Fitzhugh is widely recognized for her groundbreaking studies in applying hematopoietic stem cell transplantation in sickle cell. She’s a member of the American Society of Hematology and a newly inducted member of the American Society for Clinical Investigation. Dr. Fitzhugh, welcome to this Perspectives Interview.

Courtney Fitzhugh: Thank you for having me.

VS: To begin with, I was wondering if you could tell us a bit about yourself, your training path, and what initially got you interested in science and medicine.

DF: Sure. I’m originally from California. I grew up in San Jose, California. My father was a family practitioner, and I used to go with him to the office. I was his medical student — I’m sorry: I was his medical assistant when I was in late high school. I just really loved working with him. I loved that he had those great relationships with his patients, the continuity of care. Initially I wanted to do family medicine, until I went to medical school and realized I was much more interested in taking care of sick people. And so I read about sickle cell disease and just was really excited about it, the fact that it affects children and adults, so I can still have that continuity of care. It impacts any organ system in the body, so everybody is different. And just from reading the pain and the disparities associated, I felt like it was a place where I could make a difference.

And so when I went to medical school, after my first year, I did research in the lab over the summer, and I really liked seeing the patients more than I enjoyed being in the lab. So I really was interested in the clinical aspects of it and got interested in clinical research. As you mentioned, I went to the NIH after my third year of medical school and met John Tisdale, and he gave me the opportunity to work on developing a new curative approach for sickle cell disease. So since then I’ve been hooked.

VS: Wow. That’s amazing. Now you talked about the impact that being a medical assistant for your father had upon you. I was wondering if you could talk a little bit about the impact more broadly about mentors that you’ve had in your career and how this has helped shape the paths you’ve taken as a physician scientist.

DF: Yeah. It’s been critical at every single stage of my career. Going back to John — and I just had such a wonderful time with him: seeing patients, the way that he interacted with the patients, just how caring he was, and how much of an advocate he was — or is — and how committed he is to my success. He’s been incredible, and I’ve had so many different mentors in every single aspect. So I’ve been interested in talking to women and how they balance work-life balance, and then people outside of the NIH who can give some advice. And I’ve really  — That’s one of my favorite things now about having a lab, is actually being able to pour into the next generation and mentor others.

VS: Wow, that’s really great. Along the way, and you sort of alluded to this, it sounds like you’ve described the value of having mentors not just at earlier stages of your training or when you were sort of . . . before you started your training, but actually even now as a laboratory head. And I was wondering if you could comment on the importance that you view of mentorship and how you believe mentoring others can also help in this process as well.

DF: Yeah, mentoring . . . I think one of the best stories I have about mentoring now is: Just recently, I went to a Cure Sickle Cell meeting, and I started talking to Michael DeBaun about my interest in long-term health effects of curative therapies. And just from that discussion, we started a collaboration and applied to — successfully awarded — a U01 award. And so working closely with him and helping me with writing my first grant at this stage of my career was really critical. And now being able to do that for others, helping my trainees with their presentations, with abstracts, with manuscripts, it’s just really fulfilling and exciting, being able to see others succeed.

VS: That’s terrific. That’s really amazing. And it’s really just such a testament to both your own mentors, but also giving back and continuing that process and helping support people.

DF: Thank you.

VS: So I want to switch gears a bit. You have and you continue to contribute tremendously to advance our understanding of how we can apply hematopoietic stem cell transplantation using allogeneic and autologous sources to cure sickle cell disease. This has been an exciting area, with lots of recent activity. Could you tell us about where you see this research field going in the coming years and what advances that you’ve been most excited about?

DF: Yeah. This is a real critical time not only just for curative therapies. I remember when I first went to my first American Society of Hematology meeting, you would hardly see anything about sickle cell; it was very few and far between. And now, there’s so much, I can’t even get to it all. So, just in the short period of time that I’ve been in the field, seeing how much transformation, all the new drugs that have been FDA approved or are being developed — but in the curative therapy setting, it’s really exciting. As you mentioned, we have both allogeneic and autologous approaches. Unfortunately, the most successful traditionally has been HLA-matched sibling transplant, but less than 15% of sickle cell patients have an HLA-matched sibling donor. So what are the options that are available for others? So, now we have haploidentical transplant, where you increase the chance they have a donor to 90%, because most patients will have a parent or a child or half-sibling who could serve as a donor, or even you could use cousins and nephews and nieces. So that increases the donor pool, and there’s been some exciting new discoveries and new conditioning of regimens that have been successful — and even in the haploidentical setting with results that are approaching the HLA-matched sibling setting. So that’s been really exciting for me, since that’s my interest. And now what I’m trying to do is apply these exciting results to patients who have compromised organ function, who are very frequently left out of clinical trials. In the gene therapy and gene editing areas, there’s also a lot of excitement there too. The biggest limitation is that they use high-dose chemotherapy for these gene-corrected cells to be able to have the advantage. What is exciting in that field is using antibody-based conditioning in order to clear out the patient’s own autologous cells.

So whenever they get that to work, that’s going to be a really exciting endeavor. And then in vivo gene therapy, where they won’t have to do a lot of the manipulation outside the lab, but just being able to inject the cells and ideally cure them that way. So that would give a lot of patients, hopefully even outside of the US, access to this care. So I just love . . . Because people ask me, “What’s the future for haploidentical transplant in the setting of gene therapy and gene editing?” But I just think it’s so great that the patients have these options to choose from. There’s pros and cons for both, and right now we don’t even know the short-term or even the long-term success and toxicity associated with these approaches. A lot more work is done, and I’m excited to be able to work some to contribute to that field as well as others.

VS: Wow, that’s tremendously exciting. I want to also go back to something you mentioned earlier when you were talking about mentors, and you were talking about your work with Mike DeBaun and this recent U01. And it sounds like this was focused around long-term complications of some of these therapies. And I was wondering if you could comment a little bit, because I know you’ve thought a lot about this issue, about what we also need to do and where more research is needed in that space.

DF: Yeah. Thank you for asking. So we’re really interested in how . . . We’re not just trying to cure the patients in the short term. We’re trying to reverse their disease and help them to have a wonderful quality and quantity of life that’s long-term. Unfortunately, a lot of times, the patients are transplanted, and then they’re referred back to their doctors and sometimes they’re not followed for the long term. We really don’t know what’s happening to these patients. So we’ve tried to get some of the biggest transplant institutions together, which includes Children’s National Hospital in DC, Vanderbilt of course, NIH, Emory, and Hopkins. And to look to see, looking not only at the survival, graft-versus-host disease, which a lot of people look at, but what’s happening to the heart, the lung, the kidneys. These organs when damaged lead to early mortality in adults with sickle cell disease.

But we’re also excited that we’re going to be able to compare how nonmyeloablative or the lower-intensity conditioning compares with myeloablative conditioning, transplanting adults versus transplanting children, doing transplant versus standard therapy, not transplanting them — and be able to follow these patients. We’re going to have about 20 years of data between the retrospective and the prospective collection of the data and reporting of the data. So we’re really excited to see — and this is the only way we’re . . . And we’re also including people who have undergone gene therapy and gene editing. So I’m really excited to see what we’re going to find.

VS: Wow, that sounds incredibly important. And also just so needed, because while there’s so much excitement around these emerging approaches, obviously there’s a lot of work to be done to understand, what are the long-term consequences of all of these changes. So that’s tremendously important. So I want to move on to a slightly different area. Many of our viewers of the series are likely physician-scientist trainees, including those from traditionally underrepresented backgrounds in medicine and science. I was wondering if you could comment on lessons you’ve learned in your own training as a physician-scientist and what advice you might give to trainees who are watching this interview.

DF: So I would say, one of the most important things is just to realize what you’re passionate about, what excites you. Because I love going to work every day because I’m so excited about what I do. So it’s not really about making the money or where you’re going to make the most money, but where are you going to be happy? Where are you going to be able to make a difference? And I’d also say just to . . . I used to struggle with imposter syndrome. I came to the NIH because I wanted to work with John Tisdale. I wanted to support his research, and that door was not open to me. He only had one position, and it was already filled. But this other door swung wide open, and if I had had fear and didn’t walk through, I wouldn’t be where I am. I would say look for those doors. Where are those doors opening? So that all you have to do is just walk through. And then look to see what success you’ll have and how you’ll be able to make a difference in the lives of your patients and others.

VS: Wow, that’s such great advice. And I think even today it’s such an important thing that I as I think about different opportunities always, it’s hard to get perspective on that. So this has been such an enlightening and fantastic discussion, Dr. Fitzhugh. I was wondering if you could provide us with some closing thoughts for this audience and any other thoughts that you might have about the work that you’re doing or areas that physician-scientists can think about as they develop.

DF: Yeah. I just love being able to be an advocate for my patients with sickle cell. A lot of them go to the emergency room, and no matter where they are in the US or even outside the US, they’re treated like . . . people don’t believe that they’re having the pain. And it’s just so frustrating and disappointing hearing the same story from the patients over and over. But being able to develop relationships with these patients, develop trust with these patients, and then offer them an opportunity to have a new life that doesn’t involve pain and fatigue. And it’s just  so wonderful. So just look for the way that you’re going to be able to make a difference in the lives of your patients.

VS: Well, thank you so much, Dr. Fitzhugh. It has been truly outstanding to be able to chat with you today.

DF: Thank you for inviting me. I appreciate it.

ASCI Perspectives: Neil A. Hanchard, MBBS, DPhil – video clip

DEIC member Dr. Vijay Sankaran interviewed Neil A. Hanchard, MBBS, DPhil (view profile), Senior Investigator at the Center for Precision Health Research at the National Human Genome Research Institute, NIH. Dr. Hanchard’s work has helped to define the genetic causes of a range of rare diseases, while also identifying factors that cause variation in more common diseases such as sickle-cell disease. In this video, he discusses his physician-scientist career, the importance of community engagement, and how to effectively work with mentors. — Posted December 2023

Click the image below for a video clip of the interview. (For the full interview, click here; 6 minutes).

ASCI Perspectives: Neil A. Hanchard, MBBS, DPhil – full video

DEIC member Dr. Vijay Sankaran interviewed Neil A. Hanchard, MBBS, DPhil (view profile), Senior Investigator at the Center for Precision Health Research at the National Human Genome Research Institute, NIH. Dr. Hanchard’s work has helped to define the genetic causes of a range of rare diseases, while also identifying factors that cause variation in more common diseases such as sickle-cell disease. In this video, he discusses his physician-scientist career, the importance of community engagement, and how to effectively work with mentors. — Posted December 2023

Click the image below for the full interview. (For a video clip, click here; 3 minutes.)