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Gene Therapy Developed at St. Jude Children's Research Hospital is Safe and Effective for Babies with X-linked Severe Combined Immunodeficiency Disorder.

M2 PHARMA-December 12, 2017-Gene Therapy Developed at St. Jude Children's Research Hospital is Safe and Effective for Babies with X-linked Severe Combined Immunodeficiency Disorder


- Preliminary results from an ongoing, multicenter study of X-linked Severe Combined Immunodeficiency Disorder (XSCID) suggest that gene therapy developed at Memphis, Tennessee-based St. Jude Children's Research Hospital will lead to broad protection for infants with the devastating X-linked severe combined immunodeficiency disorder, the company said.

Approximately four months after treatment, five of the seven patients enrolled in the St. Jude clinical trial had functional immune systems, and no longer require protective isolation.

Four patients have started making serum immunoglobulins (antibodies) for the first time. One has stopped monthly immunoglobulin supplementation and recently received his first set of vaccinations.

Immune protection will likely return to the last patient. There is evidence that the seventh patient, who joined the study six weeks ago, now carries the vector in his immune cells.

No serious or unexpected treatment-related complications have been reported.

The results are the first involving infants with XSCID treated with the St. Jude lentiviral vector. The patients in this study were between the ages of 2 and 13 months and born with a disorder caused by a mutation in the IL2RG gene.

St. Jude researchers have re-engineered a lentivirus to function as a vector to ferry a normal copy of IL2RG into the patients' hematopoietic stem cells.

The vector includes novel features to enhance safety and effectiveness, including genetic insulators to block activation of genes adjacent to the IL2RG DNA insertion site. The design aims to ensure gene therapy will not lead to leukemia by inadvertently activating an oncogene in the patient's hematopoietic stem cells.

St. Jude Children's Research Hospital is leading the way the world understands, treats and cures childhood cancer, sickle cell disease, and other life-threatening diseases. It is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children.

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Publication:M2 Pharma
Date:Dec 12, 2017
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