Get to Know...Raajit K. Rampal, MD, PhD

Science and medicine swirled in every corner of Dr. Raajit Rampal’s upstate New York childhood home, from being quizzed by his scientist father about the chemical processes of cooking on the stove to spending a summer in high school conducting cancer research. From Buffalo to the Manhattan borough, the leukemia specialist shares his journey to becoming a hematologist-oncologist at Memorial Sloan Kettering Cancer Center in this exclusive Get to Know interview.

Where did you grow up, and how did you know that you wanted to become a hematologist-oncologist?

I was born and raised in Buffalo, New York. My dad was a scientist, so science is in my blood. Since childhood, everything in our house [has] had some relation to science. When my mom was cooking eggs on the stove one day, my dad asked me, “Do you know why the eggs are turning white in the pan? Do you know what’s happening here?” That’s the type of conversations we used to have that influenced my thinking from a very early age.

Science led me to medicine. It was that sense of asking simple questions: “Why does this process happen?” During high school, I spent a summer in a chemistry lab at Roswell Park Cancer Institute. That was the first time I got exposed to cancer research. I worked on a very basic synthetic chemistry project, which got me interested in chemistry. From there, I went on to study biochemistry in college.

I was absolutely fascinated by it. I spent my summers doing research, but it wasn’t necessarily all medically applicable. It was more basic science work. During one summer in college, I was working in a hematology research lab at the University of Rochester as an undergraduate, where I really began to understand the application of science to medicine. I worked in a lab that studied a clinically focused project. That was the moment I thought, “I want to do something that has a more practical application, and if I could do it in a way that merged with science, that would be perfect.”

From there, I went into the MD/PhD program at Stony Brook University. I loved my basic science/PhD training and my clinical rotations as a medical student, but I hadn’t figured out what I wanted to do. I went into internal medicine with an open mind. When I was a resident at the University of Chicago, I knew I wanted to pursue malignant hematology. I rotated there on the leukemia service and got to work with some of the giants in the field, like Drs. Richard Larson and Wendy Stock. That’s when I knew I wanted to be a leukemia doctor.

It was very evident that patient outcomes were not good. They’re better now, but they’re still not good when we think about things like leukemia. It was obvious the only way forward is through research. That inspired me to aim my focus on both the basic biology and the clinical application of science to malignant hematology, particularly in the leukemia world.

I’m fortunate to be in a position where I can still carry on cutting-edge bench research and genomic research and take care of patients and run clinical trials.

You are the lead investigator of the MANIFEST-2 trial evaluating pelabresib plus ruxolitinib in first-line myelofibrosis. What are the key takeaways of this study?

This is a key example of something that went from the bench to the bedside, where that inhibition as a therapeutic strategy was developed in the lab and subsequently led through a long process to the MANIFEST-2 trial. The results recapitulate the preclinical observations, which is that the combination of ruxolitinib and pelabresib is better than ruxolitinib for many aspects of myelofibrosis, including shrinking the spleen and not adding toxicities that result in worsening symptoms for patients. The symptom benefits were more or less the same in both arms.

We also saw other improvements in terms of bone marrow fibrosis and hematologic parameters like anemia. All in all, there is absolutely clear evidence that the combination has effects that are beneficial for patients beyond those that we see with ruxolitinib alone. The interesting and important thing is that the data continue to mature. Updates of the initial data were presented subsequently at ASH [American Society of Hematology Annual Meeting & Exposition] and EHA [European Hematology Association Congress], showing that the difference continues to exist and that the durability of that initial response seems to be maintained. All of this is very encouraging, and I hope that pelabresib can find its way to approval.

What do you hope to see in the field over the next 10 years?

I’m very optimistic. We have seen extraordinary development from the time I entered the field when we had one JAK [Janus kinase] inhibitor to where we are now talking about next-generation JAK inhibitors, targeting calreticulin, and drugs that are combination partners for JAK inhibitors. The field has moved forward tremendously.

That being said, there is still a huge unmet need and questions that pertain to developing better drugs that prevent patients from progressing and prevent bad outcomes in general. But, the other part of this is knowing the patients who are at risk. This is an area where we need to do a lot more. The biggest worry for patients and for physicians is whether this disease will progress and turn into AML [acute myeloid leukemia]. We don’t have a lot of great tools to understand who is at risk, because we know there are patients who are going to do well overall. If we can figure out who is at risk, we can potentially intervene earlier with better drugs and prevent those outcomes without exposing patients who are likely going to do fine to additional therapies they don’t need.

I’m proud to be a part of a major project launched by the MPN Research Foundation, which is a progression registry project to understand if we can identify which patients are at risk using large real-world datasets. Aside from advancing new drugs, that is another key thrust in moving the field forward.

What hobbies or activities do you enjoy outside of work?

I’m somebody who very much loves music, photography, and art. From that perspective, living in New York City is absolute paradise. I’m an amateur guitarist and an amateur saxophonist, and my wife is a professional saxophonist. Music is a huge part of our household. For a wedding, most people have a first dance, which we did, but then we played dueling saxophones.

What is a fun fact that most people would be surprised to learn about you?

Some people are surprised to learn that I’m a diehard hockey fan and one of the people yelling in the stands. That’s not my persona in professional meetings and professional settings, but put me into an NHL arena and it’s a different thing!

What’s on your bucket list?

Now that we have gotten to the point where there are commercial companies launching people into space, it’s my hope that within our lifetime we can take trips around the low orbit of the Earth on a regular basis. That would be awesome.