Gene therapy treatment reverses sickle cell anemia, early trial results show

Dec. 4 (UPI) — A new gene therapy ickle cell anemia, or SCA, has shown promise in early clinical trials after reversing the condition’s symptoms in two adults, researchers report.

The researchers, whose findings were presented Monday at the American Society of Hematology’s annual meeting, transferred healthy fetal hemoglobin genes into the patients blood stem cells and tracked the effects.

“One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms,” Punam Malik, professor in the department of pediatrics at University of Cincinatti and study’s chief investigator, said in a press release. “This includes near elimination of chronic pain and sickling events and improved anemia.”

When the body has enough HbF in its system, it can stop red blood from sickling.

Fetal hemoglobin, or HbF, normally deactivates shortly after birth. But the researchers also found it present in some adults who never stop producing HbF.

SCA is an inherited disease that develops when blood cells form a sickle shape causing them to get stuck in the person’s vascular system. It can lead to vital organ damage and death.

So, the researchers injected HbF into bone marrow cells they removed from the patient’s body and preconditioned with one low-dose of chemotherapy. They then infused the gene-corrected cells back into the patient.

This process keeps the HbF production from deactivating.

The American Society of Hematology says that between 70,000 and 100,000 people in the U.S. have sickle cell disease.

“Although it’s still early post-treatment, these preliminary results are quite promising,” Malik said. “If sustained this therapy will provide a transportable, safe and feasible gene therapy for all SCA patients.”

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