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Personnel

David Williams, MD
Internationally renowned researcher David Williams joined Children's Hospital Boston last December as chief of the Division of Hematology/Oncology and director of Translational Research. His appointment comes as the hospital looks to build its clinical and translational research programs, with the goal of taking more of its laboratory research into the preclinical and clinical arenas.

Williams will work to build an infrastructure to facilitate this process and enhance interactions between labs and clinics. "As someone involved with both basic research and patient treatment, I see first-hand the importance of connecting the two," he says.

Williams trained in hematology/oncology at Children's and the Dana-Farber Cancer Institute. He founded the Division of Experimental Hematology at Cincinnati Children's Hospital Medical Center, where he was also associate chair of its Translational Research program. His own award-winning research, continuously funded by the NIH since 1986, focuses on blood stem cell biology, blood formation, leukemia and the treatment of genetic blood disorders with gene therapy.

Erik Halvorsen, PhD
Ask Erik Halvorsen what drew him to become director of Children's new Office of Technology and Business Development, and he'll tell you about going on rounds with Children's Cardiologist-in-Chief, James Lock, MD. Watching multiple heart procedures, he saw many children who were alive because they'd been treated with innovative devices—a number of which were developed at Children's.

The experience gave Halvorsen a sense of urgency in his new role: working with the business sector to get discoveries from Children's labs into its clinics so they can start helping patients. He will oversee Children's Intellectual Property Office, expand its services and help launch a new multi-million dollar fund designed to support technology development and translational research.

A primary focus will be increasing industry partnerships and involving the commercial sector earlier in the discovery process. "We believe this will stimulate research and accelerate product development," he says.

Halvorsen was previously the director of Business Development for the Harvard Stem Cell Institute and the Harvard Office of Technology Development.

Mark Schuster, MD, PhD
Mark Schuster was named chief of General Pediatrics and vice chair for Health Policy Research. Since his pediatrics residency at Children's (1988-1991), Schuster has focused on conducting research in child, family and community issues, most recently as director of Health Promotion and Disease Prevention at the RAND Corporation, a nonprofit policy think tank, and as chief of General Pediatrics and vice chair at the UCLA Department of Pediatrics. He is currently leading NIH-funded studies on childhood obesity prevention, adolescent sexual health, children with HIV-infected parents and the use of paid family leave by families of chronically ill children. He is also leading a longitudinal Centers for Disease Control and Prevention study of 5,000 children, looking at substance use, violence, injuries, physical activity, nutrition and other health measures.

Awards

Two more HHMI investigators named at Children's
Two Children's Hospital Boston physician-researchers were among 15 selected by the Howard Hughes Medical Institute (HHMI) in 2007 as HHMI investigators. Chosen from a pool of 242 applicants, they join 10 other researchers at Children's who have HHMI appointments. More.

George Daley, MD, PhD, associate director of the Stem Cell Program at Children's, is working towards improving therapies for patients with bone marrow disease through his work on blood stem cells. His larger goal is to be able to reprogram a patient's own cells to revert to an embryonic state, from which different types of healthy replacement tissues can be made (see "Things to Watch," page 8). Current government funding bans on human embryonic stem cell research have restricted Daley's work, but HHMI's support will allow him to broaden this line of research. Elizabeth Engle, MD, is seeking better treatments for early developmental errors that impair the coordinated movement and fixation of the eyes. Engle's research has already identified genetic causes for several previously unrecognized eye-movement disorders that stem from errors in cranial-nerve development. She heads a laboratory at Children's that is designated by the National Eye Institute as a strabismus diagnostic center.

Two Children's neuroscientists earn NIH Pioneer Awards
The NIH Director's Pioneer Award supports biomedical scientists exploring bold new ideas. Two of the 12 grants for 2007, each totaling $2.5 million, went to neurobiologists at Children's. More.

Takao Hensch, PhD, focuses on how experience shapes the brain during critical periods in infancy and early childhood, when the brain can readily rewire itself. Wiring defects during these periods are thought to underlie developmental disorders like autism and schizophrenia. Hensch is looking to recreate critical periods by manipulating the brain at the molecular level, in particular examining whether non-coding RNAs—bits of genetic material that influence gene activity—can be targeted to restore brain plasticity in adult mice.

Frances Jensen, MD, researches mechanisms of injury and epilepsy in the developing brain. Her work has led to new treatment approaches, now under development for clinical trials, specifically targeting the brains of newborns (see Stopping Newborn Seizures). Jensen is now exploring how early-life seizures cause learning, memory and behavioral deficits in childhood, hoping to find targets to prevent or reverse these complications. In addition, since many people with autism also have epilepsy, she will investigate whether such treatment might improve outcomes in autism syndromes.

Deals

New anti-angiogenic drugs optioned
Caplostatin and Lodamin, developed in the laboratory of Judah Folkman, MD, were optioned in September 2007 to start-up company SynDevRx (Cambridge, Mass.). Both are based on an active analog of TNP-470, a broad-spectrum anti-angiogenic compound developed from a discovery by Folkman's colleague Donald Ingber, MD, PhD, in 1990. Modifications by Folkman's laboratory have increased the compound's safety while maintaining its unusually broad spectrum of activity against many cancers.

Unique cancer biomarkers licensed to Predictive Biosciences
Children's has entered an exclusive license agreement with Predictive Biosciences Inc. (Lexington, Mass.), a molecular cancer diagnostics company co-founded by Marsha Moses, PhD, and Bruce Zetter, PhD, both of the Vascular Biology Program. Predictive Biosciences was formed around a patent portfolio of about a dozen cancer biomarkers, some detectable in urine, that were discovered in the Moses and Zetter labs. It will initially focus on diagnostic applications in bladder, breast and colorectal cancer.

Start-up to develop devices for spinal cord injury treatment
InVivo Therapeutics (Cambridge, Mass.) was founded based on the research of Yang (Ted) Teng, PhD, of Children's Department of Neurosurgery, and collaborators Robert Langer, ScD, of MIT, and Eric Woodard, MD, of New England Baptist Hospital. The company is focused on developing treatments for spinal cord injury and other central nervous system disorders, combining biomaterials and cell therapies. Several implantable devices are under development.

Syntonix Pharmaceuticals acquired by Biogen IDEC
Syntonix Pharmaceuticals (Waltham, Mass.) was founded in 1997 based on technologies developed by Wayne Lencer, MD, PhD, chief of Gastroenterology/Nutrition at Children's, and Richard Blumberg MD, chief of Gastroenterology at Brigham and Women's Hospital. The technologies enable protein drugs to be delivered across certain cell barriers, such as those in the lungs, rather than having to be injected, as is standard today. This may allow treatments to last longer in the body, even with smaller or less frequent dosing. In February 2007, Syntonix was acquired by publicly held Biogen IDEC, marking the sixth start-up company from Children's to be acquired.