Science to care: Q&A with Boston Children’s Hospital’s new Chief Scientific Officer

David Williams, MD
David Williams, MD

When Boston Children’s Hospital decided to hire its first chief scientific officer (CSO) in eight years, the institution sought an individual who could spotlight the hospital’s robust scientific enterprise and effectively connect it to clinical medicine and industry. David Williams, MD, president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and director of clinical and translational research at Boston Children’s, was the ideal choice.

An award-winning researcher, Williams trained in the clinic but also pursued basic science, developing techniques for introducing genes into mouse and human blood cells. He focused on blood stem cell biology, leukemia and gene therapy to correct genetic blood disorders, becoming a 16-year Howard Hughes Medical Institute Investigator, a Member of the National Academy of Medicine and a Fellow of the American Association for the Advancement of Science. He has secured multiple patents for techniques still in use today.

Williams spoke about his vision as CSO to align basic research and clinical care at Boston Children’s and the challenges ahead.

Why is pediatric research particularly urgent now?

This is a unique time in history. The genomics revolution is allowing us to understand the basis of diseases more than ever. Boston Children’s is in a unique situation because pediatric medicine is made up largely of rare diseases, the majority based on development or genetics.

Rare diseases pose challenges for developing new therapies because the groups of patients for each given treatment approach are small, and the diseases are often complex. At the same time, rare diseases in children present a unique opportunity because higher risks may be acceptable to treat debilitating or otherwise lethal disorders. Children are a “purer” population, lacking many of the co-morbidities and environmental influences of adults that can obscure therapeutic effects. Successful interventions in children can lead to significant years of benefit to society and overall health care cost savings.

As Chief Scientific Officer, how do you see your role?

At Boston Children’s, we have two ends of the spectrum: basic research and clinical care. Outstanding basic science is key because that’s what separates us from many children’s hospitals. On the other hand, we have to have experts who know their diseases and are drawing patients from all over for highly sophisticated care. The fact that I was chosen and that my background is in basic and translational research suggests that our future is investing in and translating our science. That’s what my job will be.

David Williams MD chief scientific officer
Boston Children’s Hospital as seen from the Karp Research Building

How ready is Boston Children’s to seize this moment of opportunity?

We’re located in the richest biotechnology neighborhood in the whole world. We’re number one among children’s hospitals for funding from the National Institutes of Health and third of all hospitals in the country. We have the best disease-specific experts and well described, deeply phenotyped disease populations with which to work. We have world class informatics. So we have a wonderful platform.

But because we are a children’s hospital, we are often not recognized for the quality and depth of our discovery scientific platforms. Biotech and pharma in particular often are surprised at that.

We have an opportunity now to collaborate increasingly at a scientific and institutional level with biotech and pharma, MIT, Harvard Medical School, the Broad Institute, the Whitehead Institute, the Wyss Institute, the Koch Institute. These are treasures. So one of my goals is to make us better known to these institutions and foster better collaborative efforts.

What is your long-range vision for Boston Children’s research enterprise?

Our overall goal is to drive innovation around the prevention, diagnosis and treatment of childhood diseases through translation and biomedical discovery. This requires outstanding new discoveries in science, which Boston Children’s has in spades. And it also requires disease-specific experts, which we have more than anyone. My goal is to better pair those two strengths and focus them around the unique patient populations that drive our science.

Many people don’t understand that just because you make a discovery at the bench, it is not going to magically become a treatment for a patient. The incentive for the person working at the bench is to get a paper and grant. It’s hard and time consuming to move things into the patient. The institution has to be able to facilitate that as well as support our basic research.

What is your strategy to achieve this?

One of my main goals is to get some financial support and organization into our research cores. In my view, this is a wise and efficient investment of our dollars, since well-run cores tend to enhance many investigators’ science, making them more competitive to receive NIH dollars. In addition, I will have a broad look at the financial structure of research in the institution to make sure that we are using the resources that we have in the most appropriate way.

David Williams MD research cores

I want to continue to strengthen our ability to translate what we get from our investment and discoveries in science. That means better engaging biopharma and medical device communities in Boston — enhancing their knowledge of us and opening up opportunities for investing in our research, by license agreements, by participating in startup companies or by direct research agreements.

Next fall I will start an in-depth strategic planning process to understand where we want to be a decade from now so we can plan appropriately for research space (building a new building in 10 years). Meanwhile, we need to handle what is a terrible space crunch right now.

In the last 18 months, we’ve completely revamped the clinical research infrastructure. Those efforts will continue.

What will we see in your “first 100 days” as CSO?

A focus on genomics and our basic research investment, especially in our core facilities. Science is evolving more and more into a collaborative, team endeavor. That affects how people are promoted. It affects how money comes into the institution through NIH. I have the strong conviction, coming from a cancer center background, that investment in cores is an extremely efficient way to float everybody’s boat. You’re investing in technology, services and equipment that can be used by multiple people. Often, these investments are too expensive for a given investigator and yet are required for him or her to compete for NIH dollars.

David Williams MD

What are the challenges you see ahead?

The federal budget for research has lost 20 percent in purchasing power over the last eight to 10 years. At the same time, clinical [revenue] margins are shrinking at alarming rates, because of increasing pressure by third-party payers who are trying to reduce costs. That is difficult for basic scientists because the hospital invests money out of its clinical operations into research.

On a national level, small pediatric programs elsewhere are making the decision that they can’t afford to do research anymore. That has led to a concentration of talent around big research institutions such as Children’s Hospital of Philadelphia, Cincinnati Children’s, Texas Children’s and SickKids in Toronto, which are increasingly competing to recruit the best researchers. And these institutions have made strong commitments to expanding their research space.

I hope to impact at the institutional level the realization that we can’t be complacent about our research superstructure.

Any other top priorities?

To start to get a consensus of how Boston Children’s needs to move forward in the genomics area. We have great genomics and genetics. But they have grown out organically with multiple centers of influence at the hospital. We need an organized approach.

We’re sitting three miles from the Broad Institute — one of the best genomics facilities in the world, so shouldn’t we be using that? We have partners at Dana-Farber and Brigham and Women’s Hospital. I’ve already started to have conversations with two international hospitals — SickKids in Toronto and Great Ormond Street in London — to see if there are areas where we can work more effectively together. We already have some deep collaborations, and they have like-minded approaches to research in pediatrics. So, I think we have a great opportunity to collaborate and leverage our investment in science to treat patients with terrible diseases. We’re all trying to help children.

Check out Williams’s predictions for biomedicine for 2017.