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Genetic Treatments On British Children Is A Success

British doctors have cured youngsters of a deadly inherited disorder using a ground-breaking stem cell treatment which heralds a new dawn for genetic therapies.

Patients with the most severe form of the rare blood condition Wiskott-Aldrich Syndrome have now been free of the disease for four years. Although the disorder affects fewer than one hundred children in Britain, the pioneering therapy offers hope to the hundreds thousands of people suffering from other genetic blood conditions such as sickle cell anaemia.

“We are entering a new era where genetic treatments are entering mainstream medicine and offering hope to patients for whom conventional treatments don’t work well or are simply unavailable,” said Adrian Thrasher, Professor in Paediatric Immunology, at Great Ormond Street Hospital in London.

“The work shows that this method is successful in patients who, in the past would have very little chance of survival without a well match bone marrow donor,” he said. Seven children between the ages of eight months and 15 years were chosen because a bone marrow match could not be found. Without bone marrow transplantation, patients usually do not survive their teens.

All the children had eczema and associated

recurrent infections and most experienced severe bleeding and autoimmune disease that, in one case, confined the child to a wheelchair.
The children went from spending an average of 25 days in hospital to no days in the hospital in the two years after the treatment. And the child using the wheelchair was able to walk again.

Fulvio Mavilio, Chief Scientific Officer at Genethon, the biotech company which developed the treatment said: “It is the first time that a gene therapy based on genetically modified stem cells is tested in an international clinical trial that shows a reproducible and robust therapeutic effect in different centres and different countries.”

The research was published in the Journal of the American Medical Association.

Peace-lover, creative, smart and intelligent. Prapti is a foodie, music buff and a travelholic. After leaving a top-notch full time corporate job, she now works as an Online Editor for Biotecnika. Keen on making a mark in the scientific publishing industry, she strives to find a work-life balance. Follow her for more updates!