LentiGlobin

Gene therapy cured a test group of patients from sickle cell anemia

Svetlana Maslova, Hi-tech+

The treatment was carried out once and for at least three years (so much time has passed since the beginning of the experiment) helped patients forget about the disease. Clinical studies have shown incredible results, eliminating all the main signs and symptoms in the body of adults and adolescents.

Scientists from Columbia University presented the results of Phase 1/2 clinical trials involving more than 35 adults and adolescents with sickle cell anemia, according to a press release of Experimental Gene Therapy Reverses Sickle Cell Disease for Years. The disease develops due to a mutation in the beta-globin gene, which leads to disturbances in the production of hemoglobin and a change in the shape of red blood cells. The only possibility for a cure today remains the transplantation of donor bone marrow, but it is available only to a very limited group of patients.

Gene therapy called LentiGlobin consists in taking hematopoietic stem cells from a patient and processing them in the laboratory using lentiviruses that deliver modified copies of the beta-globin gene to stem cells.

The repeated introduction of cells back into the body contributes to the restoration of natural processes — settling in the bone marrow, stem cells begin to produce healthy red blood cells.

The therapy completely eliminated pain attacks in volunteers during the entire observation period, which lasted 38 months, and also significantly corrected the shape of red blood cells.

"People with sickle cell anemia live in constant fear (of a new painful episode). Our therapy will be able to return them to a normal life," commented co—author Markus Mapara. Currently, patients with these diagnoses live on average about 40 years.

Since the therapy uses its own stem cells, there is no risk of rejection, scientists explain. Meanwhile, the treatment has an important limitation — it is previously found to undergo a course of high-dose chemotherapy to make room for modified stem cells. Firstly, this is a difficult procedure in itself. Secondly, it is associated with the risk of developing cancer. In the pilot phase of the study, two patients developed leukemia, so now scientists are considering alternative chemotherapy options.

"The ultimate goal is to carry out treatment as early as possible, long before the development of internal injuries and severe complications," the authors said.

If successful, therapy can provide invaluable benefits for young patients at the beginning of life and relieve them of the heavy burden of the disease.

Article by Kanter et al. The Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease is published in the New England Journal of Medicine – VM.

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