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Umbilical Cord Blood takes medical – and now administrative – strides forward in treatment of cerebral palsy and autism spectrum disorder

Ongoing trials in the use of autologous and allogeneic umbilical cord blood in the treatment of cerebral palsy and autism spectrum disorder, have produced positive results and have changed the lives of many people who suffer from these and similar neurological conditions. As such, the United States has approved an expanded access clinical trial at Duke University Medical Centre, which will continue to build on this ground-breaking work and offer life-changing solutions to more people who need it.

Now, children who suffer from these disorders are able to receive therapy with their own cord blood or cord blood from a sibling, regardless of whether they qualify for a targeted clinical trial. It is the result of the inspirational work by Dr Joanne Kurtzberg who pioneered the use of umbilical cord blood as an alternative stem cell source for unrelated hematopoietic stem cell transplantation (HSCT). It means that the results produced from autologous and allogeneic umbilical cord blood transplantations – already documented to improve gross motor function, whole brain connectivity and the social lives of the children it impacts – can now filter down to many more who are in need.

Banking cord blood for later use in a child or a sibling, is redefining what was previously thought impossible, thanks to her work. In A History of Cord Blood Banking and Transplantation, Kurtzberg explains that “The use of banked unrelated donor cord blood as a source of donor cells for HSCT, is now emerging as a promising new cell therapy.” Cord blood is not just a ‘‘bag of blood stem cells,’’ she explains, but creates “hope for cord blood-derived cellular therapies as novel treatments for diseases that cause life-long disabilities, which currently have no curative options.”

More excitingly, she predicts, “The use of cord blood cells, in both autologous and allogeneic settings, as cellular therapies in the emerging field of regenerative medicine, currently in its infancy, will emerge as one of the major great advances in novel therapeutics in medicine over the next decade.”

Erna West, General Manager at Cryo-Save, a South African stem cell bank motivated to store umbilical cord for its use in regenerative medicine and life-saving treatments, is eager to see Kurtzberg’s work help as many people as possible: “We are extremely excited about the latest developments coming out of the US, which reinforce the reasons we do this work. We are encouraged knowing that this will improve lives around the world, all thanks to the extraordinary applications of umbilical cord blood and the hard work of doctors and scientists like Kurtzberg, who are slowly but surely, uncapping its potential.”

Providing families with cord blood that can improve and save lives has a worldwide impact. This latest FDA approval for an expanded access clinical trial, is a celebration of medical innovation, and contributes to a global momentum that could ultimately help hundreds of thousands of children who suffer from neurological conditions like this. Being able to take advantage of this new treatment pathway is built on the back of blood banks like Cryo-Save who believe in the vision of this type of emerging medicine, and in saving that brilliant “bag of blood stem cells”.

In the United States alone, there are over a million cord blood units in family storage, which means, according to cell trials.org, “If 2% of the inventory corresponds to children with eligible conditions, that potentially translates into 20,000 patients.”

In South Africa, West looks forward to the ongoing developments still to be uncovered by the expanded access clinical trial, and the burgeoning applications of umbilical cord blood worldwide: “The day-to-day functioning of people’s lives can be improved significantly with umbilical cord blood,” she explains, “Now, cerebral palsy and autism spectrum disorder patients will directly feel the positive effect of what Kurtzberg and her colleague have proven is not just a ‘bag of blood stem cells’, but a  ‘bag of blood’ that could bring much-needed relief from other conditions like this in future. The potential is overwhelming.”