5 Questions for Christopher Vakoc, MD, PhD

This question-and-answer series explores the motivations, inspirations and accomplishments of investigators at St. Jude. Christopher Vakoc, MD, PhD, recently joined St. Jude as a Cancer Biology Program co-leader in the Comprehensive Cancer Center and a Department of Oncology member after 18 years at Cold Spring Harbor Laboratory.

1. What led you to pursue a career in cancer research?

It really started during my MD-PhD training at the Children’s Hospital of Philadelphia and the University of Pennsylvania, where I did my dissertation research in hematology. What I saw in the clinic inspired me and pushed me further in that direction. 

At the same time, I knew my strengths were in the laboratory, solving puzzles about how molecules work and control genes. My fascination with genes and gene regulation drew me to cancer. Once I understood how genes worked and how they were controlled, I became fascinated by how faulty gene regulation causes disease and how restoring proper function could potentially treat or cure it. That whole loop, and the puzzle-solving involved, resonated with me from a fairly young age.

For me, that process plays out most powerfully in the laboratory, using models of disease to understand the genetic underpinnings and make discoveries that have led to therapeutic trials. It builds out of a fascination with genes but is inspired by the clinical experience. I always imagined a career directed toward pediatric disease, so that combination of interests led me to childhood cancer research.

2. What has been the major focus of your research?

I work in cancer epigenetics, looking at how gene expression is regulated and disrupted in cancer. My fascination with genes and how they are controlled led me to devote my career to understanding that dysregulation and finding ways to exploit it therapeutically.

My group has contributed to this field through the discovery of new epigenetic therapeutic targets. Some of the work I am proudest of is writing the first paper on a brand-new cancer target. We probed the epigenetics of acute myeloid leukemia (AML), identifying BRD4 as a novel target. There is something special about being at the beginning of a new wavefront — whether that is a new therapeutic idea, a new class of targets or a newly recognized disease subtype.

3. What attracted you to bring your research to St. Jude?

I worked in an environment with maximal scientific freedom, and I thrived in it. My discoveries led to publications and some collaborations with the pharmaceutical industry, but I had a limited ability to take those discoveries into the clinic myself. I was always dependent on outside organizations to take those findings forward.

What I saw at St. Jude was an enormous investment in areas that complement my own work. There is a large, thriving community of faculty and access to technologies in those fields, such as chemical biology. For the first time, I would not have to depend primarily on outside groups. We could work together as the “home team.”

On top of that, the cancer scientists here are very much aligned with my own approach. Some scientists describe themselves the way I did earlier: discovering cancer drug targets and writing the first paper on new cancer targets. When I look around the Department of Oncology, many of my colleagues think in similar ways. It’s nice to have people who really speak the same language, including colleagues in the more disparate fields of structural biology and chemistry, all working together to help cure childhood diseases.

4. Looking toward the future, what do you hope to accomplish as a member of the Department of Oncology?

I have several major goals that I hope to accomplish now that I am here. One is to continue building a thriving discovery laboratory that is also highly effective at training the next generation of scientists. At my previous institution, I trained 39 students and postdoctoral fellows. I hope to train even more in my lab here. Training scientists so they can go out and start their own laboratories may ultimately be one of the biggest ways to have an impact. It’s not just what you do in your own lab; it’s the exponential spread of approaches, ideas and goals. Then those new researchers with fresh ideas come up with new approaches and make the discoveries that drive future cures.

I also plan to expand my research programs in AML and pediatric sarcoma. I have worked in both areas before, but not at the scale I now envision. They’re so closely aligned with the mission of St. Jude; it’s the right place to focus on them.

For rhabdomyosarcoma in particular, my goal is ambitious but straightforward: I want to contribute to its elimination as a human disease. A significant part of my research program is built around that objective.

5. What message would you like to share with our families who are dealing with cancer?

To the families, I would say that they have yet another set of highly committed, dedicated scientists working on their behalf. My wife is also a cancer scientist who moved her lab to the Department of Oncology. We come to you as a family of our own, having devoted most of our professional lives — our adult lives — to efforts aimed at doing something about these diseases.

I want families to understand how motivated we are to help find new cures. We uprooted our family, including our teenage children, and moved from a life in New York to here. You do not make a change like that without a strong sense of purpose.

We are really driven by an immense desire to do something about the natural course of these terrible diseases. We will try to make the wisest investments possible with our time, attention and talent and move with urgency and creativity to make a future possible where no child dies of cancer.