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Stem-cell-derived cells flag possible new treatment for Diamond-Blackfan anemia

February 08, 2017
Researchers were able, for the first time, to use patients’ own cells to create cells similar to those in bone marrow, and then use them to identify potential treatments for a blood disorder. The team derived the so-called blood progenitor cells from two patients with Diamond-Blackfan anemia. After converting some patient skin cells into induced pluripotent stem cells (iPS), researchers got the iPS cells to make blood progenitor cells.

‘It’s not an even playing field’

January 30, 2017
An emerging area of research explores the impact of poverty and financial hardship on care and outcomes for children with cancer, WBUR reports.

In overhauling Obamacare the details matter

January 27, 2017
As lawmakers consider overhauling the Affordable Care Act, a pediatric palliative care physician writes an op-ed column for Tribune News Service urging them to preserve the law’s access to palliative care for seriously ill and dying children.

Travel dreams fulfilled for 9-year-old with terminal cancer

January 20, 2017
The family of a 9-year-old with the devastating diffuse intrinsic pontine glioma (DIPG) is filling her life with travel and other positive experiences.

Precision medicine advances pediatric brain tumor diagnosis and treatment

January 19, 2017
Precision medicine – in which diagnosis and treatments are keyed to the genetic susceptibilities of individual cancers – has advanced to the point where it can now impact the care of a majority of children with brain tumors, a new study by investigators at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center suggests.

Gene therapy trial gives Coopersville toddler second chance at life

December 07, 2016
A West Michigan toddler born with “bubble boy” disease is active and healthy, thanks to newborn screening and gene therapy, Fox 17 TV in Grand Rapids reports.

Children with Down syndrome and acute lymphoblastic leukemia fare as well as other children treated on Dana-Farber ALL Consortium protocols

December 05, 2016
Despite an elevated risk of toxicity from chemotherapy, children with Down syndrome and acute lymphoblastic leukemia (ALL) did not experience higher rates of relapse or treatment-related mortality compared with other children treated on Dana-Farber Cancer Institute ALL Consortium Protocols, according to research presented at the 58th annual meeting of the American Society of Hematology.

Deaths in cancer trials remain mystery for promising therapy

December 04, 2016
The experimental CAR T-cell immunotherapy has shown dramatic results, including long-term cures, for cancer patients with severe disease, but has also left puzzling questions about deaths that have occurred during some tests, Bloomberg reports in a story published by The Washington Post.

Pioneering doctor reflects on gene therapy’s trajectory

November 23, 2016
Alessandra Biffi, MD, director of gene therapy at Dana-Farber/Boston Children’s answers New Scientist’s questions about the resurgence of gene therapy after early setbacks.

Sickle cell disease: An unpredictable adversary

November 19, 2016
One teenage girl describes the intense pain of a sickle cell disease crisis as the feeling that heavy books are piled on her. Her sister likens it to being stabbed by needles. They don’t know when a debilitating crisis will strike, only that it will and that the sudden bouts of pain can last days, The Boston Globe reports.
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