Sickle Cell Disease: Progress

In a New England Journal of Medicine online editorial, a professor/hematologist commented on a study about sickle cell disease and gene therapy. The study, which also appeared in NEJM, successfully prevented complications from the disease by adding a gene to a patient’s blood stem cells. “Gene therapy with autologous stem cells extends the possibility of a cure to all patients without the need for immunosuppression,” commented Martin Steinberg, MD, Boston University School of Medicine and Boston Medical Center. Study participants no longer had symptoms, but their red blood cells had a shorter than normal lifespan.

The original study concluded, “One-time treatment with LentiGlobin resulted in sustained production of HbA^T87Q [an antisickling hemoglobin] in most red cells, leading to reduced hemolysis and complete resolution of severe vaso-occlusive events.” Although the results are promising, most of the people who suffer from sickle cell disease might not have access to treatment, or be able to afford it if they did.

 

Access the editorial.

Access the journal article.

Read the press release.

Other Categories:

• inflammation

• pain management

• research/study

• sickle cell disease (SCD)