Gene therapy aims to cure sickle cell disease

Success against sickle-cell would be “the first genetic cure of a common genetic disease” and could free tens of thousands of black people.

A breakthrough gene therapy is offering hope for tens of thousands of people who suffer from sickle cell anemia, after researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center found a way to “flip the switch” in red blood cells.

“We’re following very closely but we feel optimistic about his results so far,” said Dr. Erica Esrick, a co-principal investigator in the clinical trial. “That is our hope for this study — that it is a curative approach.”

Red blood cells with sickle cell disease are stiff and distorted, giving them the identifying sickle shape instead of the smooth, round shape that healthy cells have. The stiff sickle cells can block blood vessels causing a host of acute or chronic sickle cell symptoms.

Doctors and researchers in the trial remove blood stem cells from a patient that undergo months of gene modification to induce the production of healthy fetal hemoglobin, which is found in newborns and does not sickle. Therefore, the sickle cells “flip” back to healthy hemoglobin.

The gene-modified cells are then given back to the patient through an infusion. To accomplish this, patients like Emmanuel “Manny” Johnson of Brockton have to go through a one or two month hospitalization and chemotherapy treatment.

“When Manny received the infusion of his own stem cells back in May, it was an emotional day for the whole team,” Esrick said. “This is an exciting time for patients, physicians and researchers in the sickle cell disease community, with potentially curative approaches such as gene therapy and gene editing.”

The trial was a result of a collaborative effort from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and was led by the center’s president, Dr. David A. Williams.

Once the trial has been tested in more adults with sickle cell disease, it will be rolled out to teens and children as well.

TNT Health

Photo Credit: St Jude Research Hospital

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