![]() ![]() “The FDA recognizes that it’s a tough haul for rare diseases that often aren’t working with a lot of funding, and of course, we never have enough patients to do placebo control trials or big trials with statistical significance that many drug trials routinely conduct for more common disorders. ![]() ![]() It’s a leg up, an extra helping hand from the FDA,” said Puck. “This designation invites us to meet with the experts in rare disease at the FDA to help streamline the process of getting to the next steps of a pivotal trial and eventual full acceptance. The Artemis-SCID gene therapy clinical trial is headed up by UCSF pediatric professors and researchers Dr. The RMAT designation means the FDA will help guide the development of a pivotal gene therapy clinical trial leading toward approval and FDA registration as a standard of care for Artemis-SCID. Also, one baby who had cytomegalovirus (CMV) required a second infusion of gene-corrected cells to assist in building T cell reconstitution strong enough to clear the virus.Īll the infants who received the clinical trial gene therapy developed functional T and B cells, offering new hope to families coping with this rare, life-threatening condition.Įncouraged by progress made in a gene therapy clinical trial for severe combined immunodeficiency (SCID), the Food and Drug Administration (FDA) awarded research performed at the University of California San Francisco (UCSF) a Regenerative Medicine Advanced Therapy (RMAT) designation. Of the four that stopped Ig replacement therapy, three displayed normal responses to immunizations, and the fourth is healthy enough to start immunizations, according to the published study.įour babies developed a condition in which the body attacks its own red blood cells, called autoimmune hemolytic anemia, about 4 to 11 months after the transplant, but the condition resolved after T cell reconstitution. Five babies experienced full T cell immune reconstitution when they turned about one year old, and four babies produced sufficient levels of B cells to allow discontinuation of immunoglobulin (Ig) replacement therapy. Researchers detected T cells in all babies within one to four months after they received gene therapy. In the UCSF study, babies newly diagnosed with Artemis-SCID received gene therapy treatment, preceded by low-dose chemotherapy. Researchers remove stem cells from the baby, insert a working copy of the gene into the cells, and transplant the cells back into the baby. Unlike BMT, gene therapy treatment provides the baby with a functioning immune system using the baby’s own corrected cells. But babies with Artemis-SCID who receive BMTs suffer from complications including higher rates of graft-versus-host disease (a condition in which the donor cells attack the recipient), greater rejection of the donated cells, and weaker recovery of their immune systems.īabies with Artemis-SCID are also more vulnerable to side effects caused by the chemotherapy conditioning necessary to suppress the immune system and make room for the new donor cells in BMT. The standard treatment for many types of SCID is BMT, which provides a healthy immune system through donor stem cells. The deficiency in the Artemis enzyme causes SCID. Without functioning Artemis enzyme, these babies have no T and B cells, which are essential to the immune system function. The condition is fatal unless treated within the first year or two of life.īabies with Artemis-SCID have changes in the DCLRE1C gene, which codes for the Artemis protein, a DNA repair enzyme responsible for fixing DNA strand breaks during the re-arrangement of T and B cell receptors. Morton Cowan, a renowned SCID specialist and immunologist at the University of California San Francisco (UCSF), and his research team shows that gene therapy restored the immune systems of babies who have Artemis-SCID.īabies with SCID are born without a functioning immune system and are vulnerable to life-threatening infections. Update: Families of babies born with Artemis-deficient severe combined immunodeficiency (SCID) who don’t respond well to standard bone marrow transplant (BMT) treatment may consider pursuing a more effective treatment for their infants – gene therapy.Ī recently published study by Dr. ![]()
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