This article is part of a series of interviews with recipients of Clinical Research Forum’s Top 10 Clinical Research Achievement Awards. This interview is with Mignon Loh, MD, Professor of Pediatrics, University of Washington; Director, Ben Towne Center for Childhood Cancer and Blood Disorders Research, Seattle Children’s Research Institute; Deputy Director, Fred Hutch Cancer Consortium support grant, Division Head of Pediatric Hematology, Oncology, Bone Marrow Transplant, and Cellular Therapy, Seattle Children’s Hospital. Dr Loh’s research focuses on discoveries that translate into new and better diagnostics and therapeutics for children, adolescents, and young adults with leukemia. She received a 2026 Top 10 Clinical Research Achievement Award for “Blinatumomab in Standard-Risk B-Cell Acute Lymphoblastic Leukemia in Children.” The interview has been edited for length and clarity.
When did your interest in science and medicine begin?
I am, by nature, a curious person, and I have always hewed towards science, especially biology and chemistry. My school had gifted and talented programs and when I was 12, I wanted to study “genetic engineering,” which meant DNA, RNA, and how you can manipulate genes, and I remember thinking, “This is cool! I want to be a scientist.” But my sister said, “Don’t just be a scientist, be a doctor.” (laughter) So, in high school I did the usual things, like volunteering in the hospital as a candy striper and then ventured onto an ambulance core –thrilled by the excitement of working in that environment, but definitely out of my comfort zone and also very intimidated.
Did you continue on that path as an undergraduate?
I knew I was likely to go into medicine during college and felt if I was going to be a successful physician, I needed to major in a non-science so I could learn how to write, how to communicate. So, I ended up being a History of Art major! I didn’t get much experience in a laboratory until the summer before my senior year, when I did my first stint at NYU in the Department of pathology, studying Alzheimer’s disease. That’s when I began to really understand the importance of asking questions in a laboratory and studying how something can translate to the pathogenesis of a disease. That’s what started to drive me towards that realm.
What first drew you to hematology and pediatrics?
In medical school, I really enjoyed learning about adult hematology. At the time I attended medical school, we were taught more about adult medicine, and I felt that pediatrics was a little bit of an afterthought. But then I really gravitated to pediatric oncology during third year rotations after meeting with an inspirational pediatric oncologist who ignited my interest in both the emerging science as well as the rewarding relationships she had with her young patients. I also learned how complex pediatrics was because it could involve caring for a 500-gram premature baby or a young adult and everything in between. I ended up doing a sub-internship in pediatric oncology and had the most amazing month taking care of children with cancer, learning about their diseases, learning about the science that had been done up until that point, and just thriving on the fact that I was interacting with so many different people in the hospital – pathologists, lab folks, radiologists, surgeons, intensivists, the floor hospitalists… We were all collaborating and putting the pieces of the puzzle together for a 5-year-old who had a lot of complicated needs, and it started feeling to me like this is what I am supposed to be doing.
Is that when you knew you were hooked?
Yes. Sometimes in life you’re lucky to land exactly where you’re supposed to land. After my pediatrics residency, I went on to a pediatric hematology oncology fellowship in Boston and as part of that I did laboratory based translational research because I wanted to learn as much as I could about the science of leukemia. That’s when I was truly hooked. I really enjoyed thinking about how the patients were telling us a story with their leukemias and how we could use that information to inform their therapy and influence their outcomes.
That’s sounds like the definition of translational medicine
But back then, translational medicine was not popular, and so the advice I was given was that I could not do that and expect to have a successful career. It sounds so absurd now. But I kept going. I was really wedded to better understanding leukemia at the lab level and then try to figure out how we use that information to risk stratify patients and treat them with different intensities of therapies. That’s what I have continued to focus on, bringing precision medicine into therapy for acute lymphoblastic leukemia (ALL), the most common childhood cancer.
Which brings us to the award-winning clinic trial. What did you research?
Immunotherapy is currently at an inflection point and potentially provided a new therapy for children with newly diagnosed ALL that was not previously possible. In this study, we wanted to be bold and ask a question different than the typical randomizations comparing this kind of chemotherapy regimen versus that kind of chemotherapy regimen. The drug we studied, blinatumomab, is a bispecific T-cell engager (an anti-CD19 and anti-CD3 single-chain molecule), and at the time we designed the study, it was only being used in relapse B-cell ALL. We also had a lot of data supporting its safety. We conducted a phase 3 trial involving children with newly diagnosed standard-risk B-cell ALL who had an average or higher risk of relapse. Ultimately, 1440 patients across over 200 institutions that participate in the Children’s Oncology Group Consortium (COG) were randomly assigned to receive chemotherapy alone or chemotherapy plus two nonsequential 28-day cycles of blinatumomab.
What did the results show?
At a medium follow-up of 2.5 years, the estimated 3-year disease-free survival was 96% in patients treated with blinatumomab and chemotherapy versus 87.9% for those who received chemotherapy alone. After the Data and Safety Monitoring Committee (DSMC) reviewed the results, they recommended early termination of the trial and that blinatumomab be added as a new standard of care for this population of patients. I should note that only two-thirds of the patients ended up being randomized before these early results were released to us, but the outcomes were so good, the DSMC closed the study early, which was really thrilling.
What challenges did you encounter during this trial?
The biggest challenge was how to deliver the blinatumomab – how to get it to the institution and how to get the institution to give it to patients. Not many of the COG centers had familiarity with delivering this investigational agent to this many patients, which is given using a special IV pump for 28 days continuously. Some patients stayed in hospital for 28 days. Others were able to go home because they had access to home care agencies that could help with the delivery system, which must be carried around by the little kid in their backpack for literally a month. It was a very big burden to ask parents to take on, and we are very grateful for those families who signed up for this randomization. We had a lot of education on how to deliver blinatumomab safely, which is why the NEJM paper includes pharmacists, nurses, and clinical research center specialists, all of whom were so essential to how this trial could be operationalized.
What other types of collaborations were involved?
No large clinical trial can get done without a team of highly passionate, dedicated, underpaid investigators, whether they are physicians, PhDs, pharmacists, nurses…we all had to pull together over years, working through the pandemic, for countless hours. We were able to do it because all had the same mission. We all knew it was an important question, and we were all in. I’m representing a very big team, trying to be a conductor in a large orchestra where every instrument is essential.
Where is this research headed next?
The standard of care has been changed – no question there. But what’s difficult is that blinatumomab is a very expensive drug, and it takes a lot of effort to administer, so many countries outside the United States cannot get access to it. We need to work on that, getting blinatumomab through whatever channels are necessary for all patients. In addition, we want to make the drug better. For example, one of the signals we saw in trial is that patients who received blinatumomab had higher rates of infections in their cycles of chemotherapy. One of the side effects of blinatumomab is it reduces all CD-19 positive B-cells, whether those are leukemia cells or healthy cells. So, in my own laboratory, we’re working on designing another kind of T-cell engager that has to recognize two molecules on a B-cell lymphoblast, sparing a normal CD-19 positive B-cell and reducing the risk of infections.
What advice do you have for people beginning their careers in clinical research?
You must stick with it. You must stick to what you think is right and stay in there, as long as the data support it. It may get tough, but you must hang in there because fighting the good fight means something. When I see the patients who we now treat with blinatumomab and I know how much we’ve improved their lives, and I know in the future we’re going to do even more to maintain those outcomes, it reminds me how important this work is. You must remember those first principles and your goal, which for me is to cure these kids.
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