LONDON, (Pakistan Point News - 23rd Jun, 2020) A promising investigational gene therapy to treat Sanfillippo disease type A will be manufactured for clinical trial, currently taking place in Manchester, at the Zayed Centre for Research into Rare Disease in Children in London.
The condition, Sanfillippo disease type A, is a rare, inherited disease caused by a genetic mutation, which stops the body from being able to break down complex sugar molecules properly. These molecules build up in cells within the brain and nervous system and have a devastating and progressive impact on a child’s development with most patients sadly dying by early adulthood.
Gene therapy is designed to work by correcting the genetic mistake: Replacing a ‘faulty’ section of DNA with a healthy one. Cells are taken from the patient, modified in the lab, then returned to the bloodstream, where it’s hoped they will restore production of an enzyme needed to break down complex sugars. The team will closely monitor patients treated as part of the recently launched clinical trial –developed and delivered collaboratively between teams at The University of Manchester, UoM, and Royal Manchester Children’s Hospital, RMCH, to understand whether this enzyme production could ultimately slow or prevent progression of the disease.
The formal clinical trial currently taking place at RMCH aims to treat three to five patients aged under two years and follow each patient over the course of three years. The first patient to join the trial was treated earlier in 2020.
This effort is part of a decades-long collaboration with researchers and medical experts across multiple UK-based entities, including The University of Manchester, UoM, the UCL Great Ormond Street Institute of Child Health, ICH, Great Ormond Street Hospital, GOSH, Royal Manchester Children’s Hospital, RMCH, and Manchester University NHS Foundation Trust, MFT.
"Gene therapy is such a promising approach and we want to make sure as many children as possible have the chance to benefit," said Professor Thrasher, GOSH researcher co-leading this trial. "We’re very lucky at GOSH to have a brand-new facility in the Zayed Centre for Research, where we’ll be able to modify the DNA inside patient cells and prepare gene therapies that can be given back to patients for therapeutic benefit."
Professor Rob Wynn, who leads the clinical trial at RMCH, said: "RMCH has a long-standing expertise in cell therapy of metabolic diseases, and the original ideas and early attempts to use gene therapies in these diseases were three decades ago.
"This advance builds on that clinical experience and those ideas. We hope and expect that this gene therapy approach will make our treatments safer, more effective and, course, most importantly will lead to better lives for our patients."
Professor Brian Bigger, who led more than a decade of development and pre-clinical work, added: "Sanfilippo is an appalling disease which causes misery to these children- so the prospect of a treatment is tantalising – especially when it was thought for years that no treatment would be possible.
"Our early results are promising and provide some hope for these children whose condition was previously thought to be incurable. The trial is the critical next stage, but it’s true to say we are excited."
The Zayed Centre for Research was made possible thanks to a transformative ₤60 million gift from H.H. Sheikha Fatima bint Mubarak, Chairwoman of the General Women's Union, GWU, President of the Supreme Council for Motherhood and Childhood, and Supreme Chairwoman of the Family Development Foundation, FDF, in 2014. It is a partnership between Great Ormond Street Hospital, UCL and Great Ormond Street Hospital Children’s Charity.
The ambitious work of the new state-of-the-art facility brings together pioneering research and clinical care under one roof, combining the best possible patient care and the latest technology and cutting-edge medical research, with an aim to develop more effective and kinder treatments and potential cures for children with rare and complex conditions from across the UK and internationally.
