The National Institute of Allergy and Infectious Diseases (NIAID) then chose to combine both low-dose chemotherapy and gene therapy to treat five people with the condition whose ages range from 7 to 24 years old.
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Gene therapy are able to carefully reconstruct the immune systems of older children and young adults with X-linked severe combined immunodeficiency (SCID-X1), a rare hereditary disorder that largely affects males.
SCID-X1, also referred to as “Bubble Boy disease”, prevents infection-battling immune cells from developing properly. According to the background information related to the study, patients with this disease are also at high risk of infections that could also be life threatening. Babies without a matched sibling often inherits stem cells from a parent that rarely restores immunity.
In a new study conducted by U.S. researchers, it can be used to treat people with severe combined immunodeficiency (SCID-X1). The corrected cells were infused back to the patient after a low dose of chemotherapy.
Two of the patients who received the treatment showed significant improvements in their immune system, even three years after.
The study’s findings are to be presented in the annual meeting of the American Society of Hematology from December 5-8 in Florida. However, because these transplant procedures are not always effective, researchers are studying gene transfer treatment as an approach to treating XSCID.
Gene therapy appeared to have benefitted SCID-X1 or X-linked severe combined immunodeficiency, a rare genetic disorder caused by IL2RG gene mutations and mostly affecting men. The findings of the research was displayed by experts from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.
Looking for an alternative treatment pathway, NIAID scientists found that gene therapy holds hope for immunodeficiency disorder patients.
“These patients are alive, but they suffer the long-term consequences of the partial immune reconstitution and continue to face problems”, Suk See De Ravin, MD, PhD, staff clinician at the NIH’s Laboratory of Host Defenses, said during a press conference.
The remaining participants have received their treatments between 3 to 6 months ago, are showing improvement and pose as a promising result.
Previously, therapies for SCID centered on bone marrow transplant to replace the patient’s immune system.
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Researchers removed stem cells from the patients’ bone marrow, and then delivered a normalized IL2RG gene to the cells using a lentiviral vector. By collecting an individual s stem cells and modifying them with a lentivirus, the gene-corrected cells can be returned into the blood to help produce normal healthy immune cells. The researchers realized and emphasized the importance of beginning the new treatment as early as possible.
