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. …
Ed. note: This morning at 8:15 EDT, Isaac Kohane, MD, PhD, will tell the audience at TEDMED 2013 about his goal of using every clinical visit to advance medical science.
To preview his talk, we’ve updated a past Vector story about SHRINE, a system Kohane helped develop to allow scientists to use clinical data from multiple hospitals for research.
Clinical research really comes down to a numbers game. And those numbers can be the bane of the clinical researcher. If there aren’t enough patients in a study, its results could be statistically meaningless. But getting enough patients for a study, particularly for rare diseases, can be a daunting challenge.
Snippets of tissue, vials of blood and tubes of DNA from hundreds of thousands of people sit in freezers and liquid nitrogen tanks right now in laboratories across the globe. They come from people like you and me, donated in the hope that our genes researchers will be able to glean insights for the next breakthroughs for diseases common and rare.
Whenever we sign a consent form and roll up our sleeve, we don’t just join the community of research. We also become part of a debate that has been raging among researchers, clinicians and ethicists for years: What if our DNA sequence turns up bad news unrelated to the research we signed up for?
This responsibility can quickly turn into a numbers problem – a massive administrative burden. Consider that there are more than 104,000 human genetic variations now cited in the medical literature with links to human disease. …
As we’ve discussed before, clinical research really comes down to a numbers game. But getting enough patients for a study, particularly for rare diseases, can be a daunting challenge. Similarly, it can be hard to tell whether observations made in just two or three patients, say a possible new medication interaction or a new diagnostic presentation, are part of a trend – one that’s worth grant money to study.