Scientists have made a gene therapy using HIV to cure children diagnosed with Severe Combined Immunodeficiency (SCID) or "bubble baby" disease.
Doctors in a very unusual treatment injected 50 babies with HIV (the virus that causes AIDS) in hopes to treat the disease known as ‘Bubble Baby’ disease.
This disease is named after the “Bubble Baby” disease, referring to a case in Texas, where a boy lived in an isolated bubble made out of plastic to protect him from infections because he didn’t have an immune system and the slightest infection could be fatal. It is now called Bubble Baby disease because there are a set of 20 genetic variations that cause the disease in both boys and girls.
It is also known as Severe Combined Immunodeficiency Syndrome, or SCID, which is a genetic disorder and hereditary in nature. The flaw in genetics essentially prevents the bone marrow from producing healthy blood cells that constitute our body’s immune system. Without treatment, the life expectancy of patients with SCID is 1 to 2 years.
But the results of this experimental gene therapy are groundbreaking because it managed to generate an immune system in children born with no immune system. 48 out of the 50 now have infection-fighting capabilities.
The study leader, Dr. Donald Kohn of the UCLA Mattel Children’s hospital, said they managed to heal a potentially fatal disease in a single treatment. He also said that the children are now living normal, perfect lives. They are going to school, playing outside without the risk of an infection becoming life-threatening.
The two kids who didn’t show signs of improvement after the gene therapy later went through successful marrow transplants. It is too early to say if any of the children are permanently cured, but they seem to be doing just fine so far. A sure shot cure for the disease is a bone marrow transplant from a genetically matching sibling. Still, the treatment itself is risky, and the probability of finding a perfect donor is very low.
The current treatment of the disease is a twice-weekly dose of antibiotics and germ-fighting antibodies, but it is not considered sustainable in the long run. That is why doctors think gene therapy could be the answer to it. The way it is done is, some blood cells of the patient are taken out and merged with a disabled AIDS virus to replace the faulty gene in the DNA and then it is pumped back in.
A living testimonial of the therapy’s success is 11 years old Josselyn Kish, a Las Vegas resident. She had SCID when she was three years old and often suffered from rashes, painful shingles and diarrhea. She received gene therapy at UCLA, and after the therapy, she showed very rapid improvement. Now she can prevent and beat infections very easily. She recently caught the Covid-19 virus and has shown very mild symptoms so far.
Many more children have been treated in the US and UK combined, and the treatment appears to be safe across multiple hospitals, which is a very positive sign for the study. People are hoping for the success of this study to finally have a cure for the horrible disease, and up till now, the study has been on the up and up.
The study was led by UCLA and paid for by British and US government health agencies and the California Institute for Regenerative Medicine. The results were published in the New England Journal of Medicine and presented at an online conference of the American Society of Gene and Cell Therapy. The treatment is in development by the London based Orchard Pharmaceuticals with Dr. Kohn as their advisor.