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New grant expands reach of St. Jude gene therapy program for "bubble boy" disease

Funding from the California Institute for Regenerative Medicine will allow physicians at the UCSF Benioff Children’s Hospital San Francisco to treat patients with an inherited immune disease using a St. Jude gene therapy protocol

Memphis, Tennessee, February 23, 2017

Mike Meagher, PhD, Ewelina Mamcarz MD, Jay Lieberman MD, and Brian Sorrentino MD

Pictured from left to right, Mike Meagher, PhD, Ewelina Mamcarz MD, Jay Lieberman MD, and Brian Sorrentino MD.

The California Institute for Regenerative Medicine has awarded a five-year, $11.9 million grant to St. Jude Children’s Research Hospital and collaborators to deliver gene therapy to patients with X-linked severe combined immunodeficiency disease (X-linked SCID), also known as “bubble boy” disease.

The new grant supports the opening of the St. Jude gene therapy protocol for X-linked SCID at the University of California, San Francisco (UCSF) Benioff Children's Hospital San Francisco. The bone marrow transplant program at UCSF is one of the largest SCID transplant centers in North America.

“We have made good progress in demonstrating the safety and efficacy of our lentiviral-based gene therapy for the treatment of X-linked SCID,” said Brian Sorrentino, M.D., principal investigator on the grant and a member of the St. Jude Department of Hematology. “One of our goals with this new collaboration is to widen access to this life-saving gene therapy pioneered at St. Jude. We want to rebuild the immune systems of even more patients who suffer from this rare but deadly immune disease.”

X-linked SCID is an inherited disorder that involves a mutation in one of the genes necessary for a normal immune system. It occurs in one of every 200,000 live births. Babies born with X-SCID lack immune cells known as lymphocytes, which leaves them with little or no immune protection.

The lentiviral gene vector developed at St. Jude delivers a functional gene into the patient’s blood-producing stem cells. After infecting the cells, the virus transports the healthy gene to the patient’s cells.

Over the past decade, St. Jude scientists have developed a rigorous battery of tests to assess the viral vector’s safety.  Working with UCSF investigators, they were granted approval for the first time to use the chemotherapy drug busulfan as part of the SCID-X1 gene therapy protocol to help the gene-corrected cells establish themselves in the bone marrow and get to work after being infused back into SCID-X1 patients. The team went on to build an entirely new process for manufacturing the vector in the Children’s GMP, LLC, an on-site facility that produces biological products and drugs in accordance with federal safety regulations.

“It is rewarding to see the benefits of years of pre-clinical development come to fruition for the team that has worked to develop the protocol for this gene therapy,” said Sorrentino. “We are grateful to the California Institute for Regenerative Medicine for this additional funding, which will help bring our protocol to X-linked SCID patients treated in California. We look forward to working with the team at the UCSF Benioff Children's Hospital San Francisco in the years ahead. ”

Patients will be enrolled in St. Jude clinical trial LVXSCID-ND. Further information and details about the clinical trial are available here:

More details and video assets about the St. Jude gene therapy vector are available at

St. Jude Children's Research Hospital

St. Jude Children's Research Hospital is leading the way the world understands, treats and cures childhood cancer and other life-threatening diseases. It is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children. Treatments developed at St. Jude have helped push the overall childhood cancer survival rate from 20% to 80% since the hospital opened more than 50 years ago. St. Jude shares the discoveries it makes, and every child saved at St. Jude means doctors and scientists worldwide can use that knowledge to save thousands more children. To learn more, visit or follow St. Jude on social media at @stjuderesearch.