News

New gene therapy clinical trial offers hope for sickle cell disease

David Williams, MD, discusses Dana-Farber/Boston Children's new gene therapy clinical trial for sickle cell disease in a live webchat.

One boy’s cure raises hopes and questions about gene therapy for sickle cell disease

Dana-Farber/Boston Children’s researchers, who are developing a gene therapy trial for sickle cell disease, commented in Stat and other outlets on a case report from a different trial.

Sickle cell disease: An unpredictable adversary

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.

Gene therapy offers hope for treatment of sickle cell anemia

Scientists are finalizing plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anemia, the Guardian reports. The technique could begin trials next year, say researchers.

BCL11A-based gene therapy for sickle cell disease passes key preclinical test

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, according to a study in the Journal of Clinical Investigation. The work sets the stage for bringing a decades-old discovery about sickle cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in 2017.

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

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.

Genetic treatments for sickle cell

Researchers at Dana-Farber/Boston Children's and other centers are focusing on controlling the switch that regulates hemoglobin production to prevent the devastation of sickle cell disease, Scientific American reports.

Making a difference in global sickle cell disease care

Venee Tubman, MD, has deep roots in Liberia, where her grandfather was the nation’s longest serving president. The pediatric hematologist/oncologist is bringing her expertise in sickle cell disease to the African country, where she has been instrumental in establishing newborn screening and follow-up care, Penn Medicine reports.

Therapy Directed Against Platelets Does Not Significantly Reduce Painful Crises in Sickle Cell Disease

Treatment with the antiplatelet agent prasugrel does not significantly reduce the rate of pain crises or severe lung complications in children with sickle cell disease, according to a report published in the New England Journal of Medicine describing one of the largest and most geographically diverse international clinical trials on sickle cell disease to date.

Gene Editing Study Reveals Possible "Achilles Heel" of Sickle Cell Disease

Researchers from Dana-Farber/Boston Children's, using CRISPR-based gene editing tools, have found that changes to a small stretch of DNA may circumvent the genetic defect behind sickle cell disease (SCD). The discovery, published in the journal Nature, creates a path for developing gene editing approaches for treating SCD and other hemoglobin disorders, such as thalassemia.