UCLA receives funding for a new trial for gene therapy in X-linked chronic granulomatous disease

Scientists from the University of California, Los Angeles (CA, USA) have been awarded a US$7.4 million grant to lead a trial utilizing a stem cell gene therapy for chronic granulomatous disease

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Jul 31, 2015
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A team of researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, CA, USA, has been awarded funding by the California Institute for Regenerative Medicine (CIRM) to conduct a Phase I/II clinical trial for X-linked chronic granulomatous disease (CGD), utilizing stem cell gene therapy. The trial is designed to assess treatment safety over the course of 2 to 3 years.

X-linked CGD is an inherited immunodeficiency disorder that prevents white blood cells from effectively killing foreign invaders. It currently affects approximate 1 in 200,000 in the USA and is usually diagnosed at an early age, causing severe, persistent and untreatable tissue infections that lead to the formation of granulomas (a mass of tissue produced in response to infection). Without treatment, sufferers often die before 10 years of age. X-linked CGD accounts for 65% of CGD patients.

The trial will be led by Donald Kohn, professor of pediatrics in the UCLA David Geffen School of Medicine, and will enroll ten patients across three sites, Mattel Children’s Hospital UCLA (USA), Dana-Farber/Boston Children’s Cancer and Blood Disorders Center (MA, USA) and the National Institute of Allergy and Infectious Diseases (MD, USA). It is being run in partnership with Genethon (Evry, France), which engineered the virus delivery system designed to transport the gene therapy into the patients' blood-forming stem cells. It is hoped that the modified blood stem cells will produce white blood cells without the mutation, leaving the immune system able to fight infection.

Commenting on the trial, Kohn stated: “CIRM has provided us with a very exciting opportunity to assess the viability of a potential cure for this devastating disease using a novel therapy that, if successful, could possibly be utilized in other diseases in the future.” The team hopes the treatment will provide a lasting cure for X-linked CGD, and hope to expand the treatment to cover all forms of CGD.

– Written by Francesca Lake

Source: https://www.stemcell.ucla.edu/news/new-clinical-trial-funded

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