Scid cured12/7/2022 ![]() ![]() Only weeks after giving birth to fraternal twins in 2012, Alysia Padilla-Vaccaro quickly felt something was wrong with one of her daughters, Evangelina, now 2 years old. “We now have the potential to take the gene that blocks sickling and get it into enough of a patient’s stem cells to block the disease.” “We’ve been working for the last five years to take the success we’ve had with this stem cell gene therapy for SCID to sickle cell,” Kohn said. Their cutting-edge research also lays the groundwork for the gene therapy to be tested for treatment of sickle cell disease clinical trials are set to begin in 2015. The researchers’ next step is to seek FDA approval for the gene therapy, with the hope that all children with ADA-deficient SCID will be able to benefit from the treatment. “We were very happy that over the course of several clinical trials and after making refinements and improvements to the treatment protocol, we are now able to provide a cure for babies with this devastating disease using the child’s own cells,” Kohn said. With the newly transplanted cells now able to produce the needed enzyme, the research team harnessed the powerful self-renewal potential of stem cells to repopulate the blood stream and the children developed their own new, fully functioning immune systems. ![]() Kohn and his colleagues tested, modified and perfected viral delivery as the best method to put the healthy ADA genes back into the bone marrow cells of the patients. The genetically corrected blood-forming stem cells then produced T cells capable of fighting infection. Using a virus delivery system that he developed in his lab in the 1990s, Kohn inserted the corrected gene that produces the missing enzyme into the blood-forming stem cells in the bone marrow. The children’s blood stem cells were removed from their bone marrow and genetically modified to correct the defect. Since 2009 and over the course of two multiyear clinical trials, Kohn and his team tested two therapy regimens on 18 children with ADA-deficient SCID. Or they can undergo bone marrow transplants from siblings, but bone marrow matches are very rare and can result in the patient’s body rejecting the transplanted cells, which then turn against the child. They can be injected twice a week with ADA - a lifelong process that is very expensive and often doesn’t return the immune system to optimal levels. About 15 percent of all SCID patients are ADA-deficient.Ĭurrently, there are only two treatment options for children with the disease. The disease causes their cells to not create ADA, an enzyme that is critical for producing the healthy white blood cells needed for a normal, fully functioning immune system. “We can now, for the first time, offer these children and their families a cure, and the chance to live a full, healthy life.”Ĭhildren born with SCID, an inherited immunodeficiency, are generally diagnosed when they are about six months old. “Other current options for treating ADA-deficient SCID are not always optimal or feasible for many children,” Kohn said. ![]() “Instead they are all thriving with fully functioning immune systems.”Ĭhildren born with SCID are kept in controlled, isolated environments because without an immune system, ordinary illness and infection could be lethal. “All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy,” said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in the UCLA College. To date, 18 children with SCID have been cured of the disease after receiving the therapy in clinical trials at UCLA and the National Institutes of Health. Kohn’s breakthrough was developed over three decades of research aimed at creating a gene therapy that safely restores the immune systems of children with ADA-deficient SCID using the patients’ own cells with no side effects. ![]() Donald Kohn, a renowned stem cell researcher and member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. The groundbreaking treatment was developed by Dr. Often called Bubble Baby disease, the condition can be fatal within two years if left untreated. UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with a life-threatening condition called adenosine deaminase–deficient severe combined immunodeficiency, or ADA-deficient SCID. ![]()
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