Gene therapy restores immunity in children and young adults with rare

Researchers through a recent study have found out that gene therapy can be used for the treatment of the people who suffer from severe combined immunodeficiency (SCID-X1).

SCID-X1 or X-linked severe combined immunodeficiency is among immunodeficiency-related diseases which could now be treatable, thanks to the new findings.

People suffering with the disorder lack infection-fighting immune cells, making patients highly susceptible to life-threatening infections. All the study participants had received stem cell transplants from their parent, and their immune systems were weakening. Such patients needs lifelong treatment and may endure complex medical problems, including chronic infections for the rest of their life.

The study included data from five patients aged older than 2 years (range, 7-24) who had worsening immune dysfunction and were dependent on immune globulin supplementation despite prior haploidentical HSCT. The corrected cells were then infused back into the patient after a low dose of chemotherapy. Despite improvements in immunity, the second patient died of pre-existing, infection-induced lung damage two years after receiving gene therapy, suggesting the importance of early treatment before organ damage becomes irreversible. Abstract 261. Presented at: ASH Annual Meeting and Exposition; Dec. 5-8, 2015; Orlando, Fla.

The U.S. National Library of Medicine has more about X-linked severe combined immunodeficiency. But, in situations where a matching sibling is unavailable, the patients receive the transplantation from the parent. In the years to come, the surviving patients would continue to be monitored.

The researchers expect to, “determine the safety and effectiveness of lentiviral gene transfer as a treatment for children and adolescents with this immunodeficiency since transplant procedures aren’t always effective”.

NIAID conducts and supports research-at NIH, throughout the United States, and worldwide-to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses.

Basically, lentiviral gene transfer therapy uses genes that are great to replace damaged ones.

Looking for an alternative treatment pathway, NIAID scientists found that gene therapy holds hope for immunodeficiency disorder patients. The researchers said that two of the patients have shown remarkable improvement in immunity and clinical status. The transplanted cells transitioned into the healthy bone marrow and started producing new blood cells.

The study is still going on with the three other patients having received the therapy three to six months ago and are beginning to show improvements in immune function. Despite the small scale of the study, researchers hope that the therapy will prove to be an effective treatment for this rare immune disorder.