Specialist biometrics CRO Phastar has supported a Great Ormond Street Hospital-sponsored study into new treatments for children with severe combined immunodeficiency (SCID). The company provided pro bono support from its specialist statisticians, data managers and programmers for the study, testing efficacy and safety of cryopreserved haematopoietic stem cells for transplant.
SCID is the name given to a group of rare, inherited disorders that cause major abnormalities of the immune system. Germs that would be relatively harmless to healthy individuals can cause serious and life-threatening illness in a child with SCID. Adenosine deaminase (ADA)-SCID was identified almost four decades ago and represents approximately 15-20% of all cases of SCID. The majority of ADA-SCID patients are diagnosed in the first year of life. The patients rarely survive beyond 1 to 2 years unless immune function is restored or contact with pathogens is avoided by creating a sterile environment around the patient (the so-called “bubble-children”). In most cases, haematopoietic stem cell transplantation offers the only long-term cure.
Gene therapy offers an alternative treatment for ADA-SCID whereby a correctly functioning copy of the ADA gene is introduced into haematopoietic stem cells (HSCs) that have been harvested from the patients themselves using a gene transfer vector. These transduced cells are then returned to the patient where they initiate immune reconstitution much like HSCs from a healthy donor.
OTL-101 is a cell suspension comprising autologous CD34+ HSCs that are transduced ex vivo with Elongation Factor 1α Short form lentiviral vectors encoding for the human ADA gene. These transduced cells are then cryopreserved until such time of infusion back to the subjects.
The objective of this trial, supported by Phastar, was to determine the efficacy and safety of cryopreserved OTL-101 treatment.
Prof Claire Booth, of Great Ormond Street Hospital, says: “Great Ormond Street Hospital have been engaging with Phastar for a number of years to deliver this trial. They have performed numerous analyses on an ongoing basis and have been professional and knowledgeable throughout. We are grateful for their generous pro bono support that ensured this critical study could be completed. Continuing developments and improvements are transforming the lives of children with ADA-SCID and we are delighted to be making vital contributions to this.”
Andrew MacGarvey, CEO, Phastar commented: “It was brought to our attention that Great Ormond Street Hospital needed additional support to ensure their project reached completion. Phastar therefore agreed to provide their services on a pro-bono basis through the Phastar Cares initiative. At Phastar, we appreciate the significance of this research to the scientific community and understand the benefits it will bring to children with this life-threatening condition. The Phastar Cares pro-bono scheme enables charities to access Phastar’s expertise in biometrics and we are proud to provide support in the form of our industry-leading specialist statisticians, data managers and programmers to this research.”
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