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Ten children with severe combined immunodeficiency have fully recovered through the modified human immunodeficiency virus. Gene therapy has fully restored their immune function without developing cancer or other side effects. ( St. Jude Children's Research Hospital )
Scientists now have a better approach to treating severe combined immunodeficiency by using gene therapy that allows patients to recover completely without medical side effects.
SCID is a rare and potentially fatal condition caused by several types of immune system deficiency. In the United States, fewer than 100 newborns are affected each year.
People with this condition have a complete loss of immune function, making them very susceptible to infections.
Today, children and parents whose lives have been affected by the DICS have the hope of possible treatment through a revolutionary technique in gene therapy.
In the SCID processing memory
A bone marrow transplant is the standard treatment for SCID. The patient must generally undergo monthly infusion cycles of antibodies called immunoglobulins.
A successful transplant does not mean cure because only part of the child's immune system is restored. Finding an exact match is a major challenge, even when the donor belongs to the immediate family.
In 1990, Drs. William French Anderson and Michael Blaese of the National Institute of Heart, Lung and Blood and the National Cancer Institute have begun the gene therapy of the DICS.
The process involved removing the defective blood cells from the patient, injecting a new gene with the help of a virus, and then handing the modified cells back into the body. Doctors were waiting for the new cells to strengthen the child's immune system.
The 20 children who underwent gene therapy all recovered, but five of them eventually developed leukemia. Scientists have faced the dilemma of how to modify defective SCID cells without causing cancer.
Modern Gene Therapy
In a study recently published in The New England Medical Journal, researchers at the St. Jude Children's Research Hospital said they have developed a way to enable effective gene therapy without the risk of leukemia.
The team healed 10 infants born with SCID-X1, a specific type of DICS requiring identical bone marrow tissues from a sibling donor. The researchers have redesigned the human immunodeficiency virus so that it causes neither HIV / AIDS nor cancer.
All children responded well to the experimental treatment and were vaccinated to further strengthen their immune system.
"These patients are now young children, who respond to vaccines and whose immune system is able to make all the immune cells they need to protect themselves from infections when they explore the world and lead a normal life. This is a first for patients with SCID-X1, "said co-author of Dr. Ewelina Mamcarz of St. Jude's Department of Bone Marrow Transplantation and Cell Therapy.
Patients who had infections before gene therapy recovered completely. Development milestones were noted as normal. None of them has developed leukemia or a life – threatening condition as side effects.
Tribute to Bubble Boy
The term "Bubble Boy Disease" has been commonly used to designate a patient with SCID. It was invented in the 1970s by a documentary starring David Vetter, who lived virtually isolated in plastic bubbles to protect him from infections.
Shortly after Vetter received an incompatible bone marrow transplant, he died in 1984 at the age of 12. Bone marrow samples from her sister contained a dormant virus that caused hundreds of tumors.
Vetter's death provided a wealth of scientific material to study the DICS in the hope of developing powerful treatments. It's also through his autopsy that doctors have confirmed that a virus could cause cancer.
Ⓒ 2018 TECHTIMES.com All rights reserved. Do not reproduce without permission.
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