SCID WAS CURED IN A PATIENT IN 1990 VIA FULL
HSCT aims to give stable donor stem cell engraftment after partial or full ablation of the recipient’s marrow and immune system using a combination of chemotherapy, antibody therapy, and a graft-versus-marrow effect. There are now nearly 200 molecularly defined PIDs. Indications for HSCT increase as advances in molecular immunology better define PIDs while parallel studies of the natural history of PIDs reveal which will benefit most from early HSCT before organ damage is present. Greater awareness of primary immunodeficiency (PID) amongst general paediatricians, highlighted by campaigns promoting warning signs has lead to earlier diagnosis and referral to specialist centres. Supportive care has also improved, with molecular detection of viral infection enabling pre-emptive antiviral treatment before organ damage supervenes. Since then, HSCT techniques have advanced enormously due to refined HLA-tissue typing, the increased use of alternative donors, the availability of new stem cell sources including umbilical cord blood as well as less toxic chemotherapeutic conditioning, and graft-versus host disease (GVHD) prophylaxis. In 1968, hematopoietic stem cell transplantation (HSCT) was first performed for patients with inherited cellular immunodeficiencies: a child with severe combined immune deficiency (SCID) and another with Wiskott–Aldrich syndrome (WAS) transplanted from matched siblings. You can contact NPR science correspondent Richard Harris at. Now, with continual advances in treating SCID, "it's just nice to see another success for gene therapy," Kohn says.
SCID WAS CURED IN A PATIENT IN 1990 VIA MOVIE
Kohn at UCLA says that for more than a decade, it seemed that the field of gene therapy was a dead end.Ĭlearly it has made a comeback and has been used to treat other rare diseases, including adrenoleukodystrophy, a neurological condition better known as Lorenzo's Oil disease, after a 1992 movie that highlighted a boy with the condition and his parents' hunt for a cure. It works in the laboratory, "and this really sets the stage then for a clinical trial, hopefully in the next 12 to 18 months," he says.Īll this makes the leukemia setback from the 1990s feel like a fading memory.
"Sometimes their families don't understand just how profoundly affected their immune system is."įor example, instead of inserting a healthy gene into blood cells, Porteus, the Stanford pediatrician, has used a precision gene-editing technique called CRISPR to correct the genetic error in blood cells drawn from SCID patients.
Screening for SCID is now done throughout the U.S., though its introduction was gradual and state by state.īefore screening was instituted, these children used to show up in the hospital with life-threatening infections, "and now we're seeing happy, bouncy little newborns who just look perfectly normal and they're never sick," Puck says. In her view, the key to treatment is finding these children early - through newborn screening - before they start to get life-threatening infections. Of course, she adds, they'll be watched carefully for signs of leukemia and to see if the effect of the therapy is wearing off. These children are "growing normally, they're getting colds like everyone else and they're getting over infections - so I would say that is a cure," Puck says. Jennifer Puck, a pediatrician at UC San Francisco and a collaborator in the latest study, says infants who got the gene therapy don't need that medication. As a result, patients require monthly infusions of antibodies called immunoglobulins. But that procedure often restores only part of a child's immune system. Standard treatment for SCID is a bone marrow transplant. They aren't any different from my daughters." Two more infants have been treated since the paper was prepared for publication, the team says. "To be able to see these babies in my clinic now as toddlers is very rewarding," she says. "I am thrilled to see these outstanding results," says Ewelina Mamcarz, a transplant physician and first author on the new paper.
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