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For babies born with the serious genetic disease known as "bubble boy disease", a common cold can be deadly.
Born without immune cells essential for disease control, they must remain isolated from the outside world for their own protection. The months spent in the hospital and the treatment of serious infections are the norm. Without treatment, most people born with the disease die at a young age.
Researchers at the St. Jude Children's Research Hospital are calling a new experimental drug a cure for the disease. Ten babies born with the genetic disorder, combined severe immunodeficiency related to X (XSCID), have been successfully treated with no apparent side effects, researchers said Wednesday.
The children are now making their own immune cells. Almost everyone was able to return home with their families and lead a normal life, including daycare. A recently treated child has remained in St. Jude for the time being.
"This is a first for patients with XSCID," said Dr. Ewelina Mamcarz of the St. Jude Department of Bone Marrow Transplantation and Cell Therapy. Mamcarz is the first author of an article on the results, which discusses the first eight children to have received treatment. It is published in the New England Journal of Medicine, which has been peer reviewed.
A serious genetic disease that was successful in the 1970s could now heal
The XSCID, estimated by the US government, probably affects at least one in 100,000 newborns, became famous in the 1970s thanks to a young boy with XSCID, David Vetter.
Vetter lived all his life in a plastic bubble, to protect him from a deadly infection, and became known as a "bubble boy".
His story quickly captured the public's sympathy and imagination and even inspired a TV movie about Vetter starring John Travolta.
These plastic chambers have now disappeared, but those with XSCID still need to be kept in protective isolation protecting them from infection.
One current treatment option is bone marrow transplantation, but not everyone can find a compatible donor and the treatments do not always work. This last case was the case of Vetter, who died at 12 years after an unsuccessful transplant.
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It has long been hoped that gene therapy, a new field of state-of-the-art medicine that modifies the body's genetic material to treat diseases, could be useful. However, at the beginning of treatments, some patients developed leukemia, hindering research efforts.
The new experimental treatment is called MB-107 and is developed by the biotech Mustang Bio, whose market value is about $ 80 million. The researchers worked to minimize the risk of leukemia in patients.
This has so far been successful, no patient from the research trial developing cancer.
The treatment begins with the patient's stem cells, which are collected and treated outside the body with a hollow virus, introducing a gene that normally functions in the cells.
Patients then receive chemotherapy before being infused with their newly modified cells. The whole process takes about 10 days from start to finish.
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The use of low doses of chemotherapy was an innovation borrowed from bone marrow transplants, where it is used to remove an existing immune system from a patient. In the new experimental gene therapy, it seemed to improve the absorption of the treatment and to minimize the safety problems.
Researchers say it's actually a cure for XSCID, but they still do not know how long it will last. They followed the patients for up to two and a half years until now.
In terms of "physiological quality of life – it's a cure," said Dr. James Downing, President and CEO of the St. Jude Children's Research Hospital. "The question is, will it be sustainable and will it last 10, 20 or 50 years for these children?"
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