David Williams, MD, is Boston Children’s Hospital’s newly appointed Chief Scientific Officer. He is also president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and director of Clinical and Translational Research at Boston Children’s. Vector connected with him to get his forecast on where biomedical research and therapeutic development will go in the year ahead.
What do you see emerging in medical science in 2017?
Genetics/genomics will increasingly be used to tailor therapies, for germline-mutated (inherited) diseases as well as somatic-mutated diseases (cancer and other environment-related diseases). We will see continued testing of therapeutic approaches based on genetics, such as gene therapy and anti-sense technology, in a wide range of disorders. Pharmacogenetics, or the use of genomics to adjust dosages of drugs, will also continue to advance and will include “point of service” testing in the future.
What changes do you predict in the research arena?
I foresee increasing interdependence of academic research and biopharma. Biopharma will increasingly rely on academic institutions, not only for human clinical trials, but also for defining relevant and druggable pathways. Industry can then do what it does best: develop drugs. Research-focused institutions like ours will be increasingly dependent on philanthropy and biopharma for capital, because the NIH research budget is likely to never completely recover, in my view. In fact, new approaches will likely include three-way partnerships: biopharma, academia and disease-specific philanthropic organizations. Research institutions would do well to facilitate these partnerships. In pediatrics, this means a continued focus on the basis of pediatric diseases, but also on developing a more robust infrastructure for pediatric clinical trials. Many advancements in adult medicine come from fundamental discoveries made at leading pediatric research institutions.
Gene therapy is an area close to your heart. Where is it headed this year?
The field is still recovering from the serious side effects seen in the early trials, now more than 15 years old. However, newer gene therapy vectors appear to be safe and effective. Biopharma has increased its capital investment, both in cancer (immunotherapies) and in inherited diseases. Several new therapies will likely be licensed in one to two years. For instance, new clinical trials in cerebral adrenoleukodystrophy, hemophilia and β-hemoglobinopathies (sickle cell disease and thalassemia) appear quite promising in registration trials. Genome editing is also on track for successful introduction in the next one to three years.
Do you have any specific predictions around immunotherapy and cancer?
The major question in the field right now is whether the successes seen in B-cell leukemias will be matched in other leukemias and solid tumors. I believe this year will see the discovery and testing of new tumor antigens as targets for CAR T cells. I also expect to see testing of combination therapies, combining standard chemotherapy with different types of immunotherapy, such as cellular immunotherapy (applying an accelerator to the immune system) and pharmacologic inhibitors (taking the brakes off the immune system).
The Precision Medicine Initiative was just allotted $1.4 billion in funding. What’s your forecast for the role of genomics in medicine?
The field is still in its infancy. Except in cancer, epilepsy and a few very specific areas related to pharmacogenomics, it’s not yet clear how much genomics will impact medicine in the coming year. A challenge for pediatrics is to make sure targeted drugs are developed for inherited diseases. Since many of these diseases are rare or even ultra-rare, the markets are small and biopharma has less financial motivation than for many adult diseases. Boston Children’s Hospital and our peer institutions will need to invest not only in the identification of genomic lesions, but in discovery science to further elucidate and validate pathogenic pathways that can lead to rational drug design.
What other fields do you predict will move ahead this year?
The use of highly manipulated cell products will continue to advance into the clinical testing arena. These may include mesenchymal stem cells, pluripotent cells derived from reprogrammed somatic cells and their cellular progeny. Manipulated immune cells (such as CAR T cells) are one example that has already profoundly impacted cancer therapies. In addition, continued advances in robotics and nanotechnology will impact both devices and drugs in the future.
What types of science do you expect to see funded?
Some at the NIH increasingly feel that “big data” will be the key research investment. Personally, I think solely focusing on big data is a mistake. We need continued investment in basic research to validate causative molecular lesions, clarify complicated inter-dependent pathways and vulnerabilities and then develop robust clinical trials to test drugs that intersect these pathways.
In pediatrics, we almost certainly will need research consortia, such as the one called for in the National Pediatric Research Network Act. This legislation still awaits NIH funding, but the 21st Century Cures Act including language directing the NIH to implement it. If funded, it will foster productive collaborations across basic and translational research. The Cancer Moonshot, which was allocated $1.8 billion by the Cures Act, contains language to address the unique nature of pediatric cancers.
What are the prospects for translational research in 2017?
We will see continued development of in vitro model systems, which will reduce dependence on animal models for drug discovery and testing. I also expect the FDA will continue to evolve new kinds of regulatory pathways that will accelerate the review of new drugs and get them to patients more quickly, especially in rare diseases. Last week’s approval of a new drug for spinal muscular atrophy may be a bellwether.
Learn more about Williams’s agenda as Boston Children’s Chief Scientific Officer.
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