Researchers revealed that gene therapy was used to cure ten newborns with bubble boy disease. It is a rare genetic disorder. The treatment completely cured the babies of the immune disorder with no complications or side effects. Scientists have been seeking such a result for decades with no success.
In 2003, a similar attempt to treat severe combined immunodeficiency disease (SCID) using gene therapy was stopped. This was after experts discovered that the therapy caused cancer. The treatment that was unveiled does not have side effects. Experts are hoping that it could help in the advancement of cures for other genetic disorders like sickle cell disease.
What does Jennifer Hill say?
Jennifer Hemall who is a pediatric immunologist at the Children’s Hospital of Philadelphia said it was a game-changer. She was not part of the study though. She said that seeing this kind of treatment become reality is exciting. Infants who have SCID are normally born without a proper immune system. If not treated, they barely make it past their first birthday. Even a common cold can prove deadly to them.
In recent years, the survival rate has increased. This is mostly due to the refinement of life-saving procedures like bone marrow transplants and widespread screening tests. However, such treatments come with complications. They may even lead to the children’s new immune systems attacking their own bodies.
What the new gene therapy does is correct the genetic defects that are in the DNA of babies soon after birth. This prompts their bodies to generate what is missing in their immune system.
The researchers first took the bone marrow of the infants and extracted blood stem cells. A modified HIV virus was used as a means to deliver the correct copy of defective genes into the stem cells of the patients. The corrected cells were reinfused back into the patient and they created healthy immune cells.
The disease has been found to be more common after the screening of newborns for SCID became popular.
There are many forms of SCID. There exists gene therapy in Europe for a variant referred to as ADA-SCIDS. However, a cure for –X- linked SCID that only affects boys is yet to be found.
Up to now, the transplanting of blood-forming stem cells from tissue-matched sibling donors was the most effective therapy for SCID. Most patients, however, lack donors. Also, donors who are half-matched or unrelated are crucial in lifesaving but this often results in complications.
Researchers were given hope by the first successful gene therapy trial by French doctors in 2000. They got to hope that instead of just treating its symptoms, they could be able to treat it.
The gene therapy trials temporarily halted in America after leukemia was found in some of the patients. This sent researchers looking for alternative answers which have led to finding the current cure.
Wan, W. (2019, April 18). Science Alert. Retrieved April 19, 2019, from www.sciencealert.com: https://www.sciencealert.com/gene-therapy-has-cured-ten-infants-with-bubble-boy-immune-disease