#Gene therapy scid full
#Gene therapy scid trial

Clinical Stage Programs Stanford School of Medicine (X-linked SCID) These regimens kill many types of cells beyond those intended and result in numerous toxic side effects. Most of the risk of current BMT procedures comes from the radiation or chemotherapy given to patients before the transplant to wipe out the patient’s own stem cells that form immune cells. They are trying to improve the safety of bone marrow transplant (BMT), which essentially uses the stem cells in bone marrow to give the children a new immune system that works properly. Researchers funded by California’s stem cell agency are looking for a better alternative to help these children. Newer gene therapy techniques appear to be safer but have been tried on fewer than 20 patients.

Gene therapy has been used to correct the defect in certain types of SCID, but early gene modifying techniques resulted in some patients developing cancer. Because these patients already have a compromised immune system, 10 to 20 percent don’t survive the transplant. Left untreated the children die before the age of 2, and the only readily available treatment involves high-risk bone marrow transplants. SCID – also known as ‘bubble boy disease’ - is a rare genetic disorder, effecting one in 30,000 newborns. Severe combined immune deficiency or SCID is an example of a primary immunodeficiency. Typically, these diseases have genetic causes and many of them are rare. Primary immunodeficiencies are disease that compromise or destroy the immune system, leaving patients susceptible to serious infections. Every innovation happens one patient at a time.CIRM funds many projects seeking to better understand Severe Combined Immune Deficiency (SCID) and other primary immunodeficiency diseases to translate those discoveries into new therapies. Researchers are pioneering gene therapy in this very rare disease right now, but we are using techniques that can be exported to other situations and can help many other conditions worldwide. An additional three patients, who were followed for fewer than 24 months, had promising B cell development when compared to previous outcomes for donor-transplanted patients.Īll of the results are better than those previously seen with Artemis-SCID patients who received donor bone marrow transplants.

#Gene therapy scid full

All 10 were growing their T cells and B cells by 12 weeks, and four of nine (excluding a patient who received a second treatment) achieved full T-cell immune reconstitution by 12 months.įour of nine also achieved full B cell immunity by 24 months, allowing them to discontinue immunoglobulin replacement and receive standard childhood vaccinations. The second outcome was T-cell reconstitution at 12 months, a measurement of the strength of the immune system.Īll 10 patients were safely transfused with their gene-corrected stem cells which gave rise to corrected peripheral blood cells within 42 days.

Researchers theorized patients would need less chemotherapy to prepare their marrow for transfusion when their cells were being used thus only 25% of a full dose of busulfan was administered.

#Gene therapy scid trial

The first outcome of Phase I/II trial involved the safe transfusion of gene-corrected cells that would differentiate into white blood cells by 42 days after infusion. Signs of Stronger Immunity in Children Having Artemis-SCID Gene correction has been used before in patients with other genetic forms of SCID, but its use in Artemis-SCID is significant because these patients usually respond more poorly to standard bone marrow transplants.Ĭomplications can include rejecting the marrow graft, graft vs.host disease - in which the donor T cells attack the recipient's tissues - chronic infections leading to organ damage, stunted growth, and premature death.