Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency
An investigational gene therapy can safely restore the immune systems of infants and children suffering from a rare, life-threatening inherited immunodeficiency disorder, according to a study supported in part by the National Institutes of Health. Researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatment for the condition known as severe combined immunodeficiency due to adenosine deaminase deficiency or ADA-SCID. The findings were published today in the New England Journal of Medicine.
DB Kohn, C Booth etal. Lentiviral gene therapy for autologous ex vivo adenosine deaminase deficiency. New England Journal of Medicine DOI: 10.1056 / NEJMoa2027675 (2021)
Explanation
An experimental gene therapy developed by researchers at the University of California, Los Angeles (UCLA) and Great Ormond Street Hospital (GOSH) in London has been announced that uses a modified lentivirus to deliver the ADA gene into cells.Previous gene therapy relied on a "gamma retrovirus" and some people who received this treatment have reported developing leukemia. This lentiviral vector is said to be designed to avoid the development of leukemia and increase the efficacy of the therapy.
Results from three separate Phase 1/2 clinical trials showed that patients gained and retained strong immune function after gene therapy, to the point of discontinuing other drug therapies. From the results, the new gene therapy appears to be safe, but participants have developed some side effects.
However, the side effects were reported to be mild to moderate.
