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Gene therapy trial wrenches families as one child’s death saves another

July 20, 2016
MIT Technology Review reports on a gene therapy trial in Italy that has reported strong early results for an inherited neurodegenerative disease. The trial, led by Alessandra Biffi, MD – now director of gene therapy at Dana-Farber/Boston Children’s – helped a number of babies identified through newborn screening after an older sibling died of metachromatic leukodystrophy (MLD).

Biden should take a note from the original moonshot: Focus on just one cancer

June 30, 2016
In an opinion piece for Boston Globe Media’s Stat, the author argues for focusing the cancer Moonshot on diffuse intrinsic pontine glioma or DIPG – the universally fatal pediatric brain tumor that claimed the life of original moonshot astronaut Neil Armstrong’s daughter.

Finding a cure wouldn’t mean we’ve defeated cancer

June 29, 2016
While the cancer Moonshot aims to double the rate of progress toward improving cure rates, the Huffington Post quotes Chief Medical Officer Lisa Diller, MD, in a piece asks if enough attention is being paid to survivors, particularly survivors of pediatric cancer.

Dana-Farber/Boston Children’s remains nation’s #1 pediatric cancer program

June 21, 2016
U.S. News & World Report has once again ranked Dana-Farber/Boston Children’s Cancer and Blood Disorders Center the nation’s #1 pediatric cancer program in its new 2016-17 Best Children’s Hospitals rankings.

How drug companies use gifts and internships to reach valuable hemophilia patients

June 14, 2016
In a Washington Post story on new research about pharmaceutical companies’ direct outreach to hemophilia patients, Ellis Neufeld, MD, notes: These tactics can “get in the way of the doctor-patient relationship.”

Dual stem-cell transplant improves outlook for children with high-risk neuroblastoma

June 05, 2016
Children with high-risk neuroblastoma whose treatment included two autologous stem-cell transplants were more likely to be free of cancer three years later than patients who underwent a single transplant, according to results of a Phase 3 clinical trial presented at the annual meeting of the American Society of Clinical Oncology. The tandem transplant technique produced even better results when followed by treatment with immunotherapy agents, investigators found.

Science commentary explores ways to pay for success in gene therapy

May 26, 2016
Noting the potential of gene therapy to be a one-time treatment for rare and serious diseases that otherwise cost hundreds of thousands, if not millions, of dollars in chronic care over a lifetime, the authors suggest several models for valuing, pricing and developing gene therapy.

Q & A: Making sense of new research on possible link between phototherapy for newborn jaundice and cancer

May 23, 2016
Two new studies published in the journal Pediatrics analyze large datasets to see if there is a link between phototherapy (light therapy) for newborn jaundice and pediatric cancer. Lindsay Frazier, MD, a pediatric oncologist at Dana-Farber/Boston Children’s, and Anne Hansen, MD, MPH, medical director of the Neonatal Intensive Care Unit at Boston Children’s Hospital, answer questions about the new research and its implications.

Evidence suggesting link between cancer and light therapy for newborn jaundice is inconclusive but warrants consideration

May 23, 2016
Two new studies raise enough questions about a possible link between childhood cancer and light therapy for newborn jaundice that clinicians should exercise caution in prescribing the treatment for infants whose jaundice is likely to resolve on its own.

We’ve known for 50 years what causes sickle cell disease. Where’s the cure?

May 19, 2016
Boston Globe Media’s Stat examines efforts to use gene therapy or gene editing – or develop a pill – to harness the latest research on sickle cell disease. “This is the right time for a sickle cell moonshot,” says Dana-Farber/Boston Children’s President David Williams, MD.
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