“Look at me!” says 11-year-old Michaela, “I can walk now!” Michaela Sky Rodrigues is an exceptional girl with exceptional parents. Refusing to accept the bleak outlook doctors described for her, Michaela, who suffers from secondary dystonia, travelled with her family from her home in Australia to China for a series of pioneering umbilical cord blood stem cell treatments.
And the result? Michaela’s father emphasises that stem cells are not a cure for dystonia; but they are an effective treatment: “small changes” he says, “lead to big changes.” Michaela can now walk, control her limbs and neck, use the toilet and eat solid foods. The list goes on and on, each a landmark for Mr and Mrs Rodrigues who are now looking forward to a bright future for their daughter: “Look what she’s accomplished,” Mr Rodrigues says, “in such a short time after stem cells… look how far she’s come.”
The clearest evidence of Michaela’s progress is the enormous smile on the face of both father and daughter, and the confidence with which Michaela addresses the video camera after her treatment. ‘Thank you,” she jokes, “and good night!”(1)
Dystonia is a painful and debilitating neurological movement disorder and although it affects around 70,000 people in the UK, many of us have never heard of it. Dystonia Awareness Week (5th – 13th May 2018) aims to raise awareness of the condition, enabling The Dystonia Society to support those affected.(2)
The cause of dystonia varies from patient to patient, though the condition is linked to the part of the brain that controls movement. The disorder may be inherited, or result from a related illness such as Parkinson’s disease, stroke, cerebral palsy or multiple sclerosis.
Dystonia can start at any age and might affect any part of the body. The symptoms vary and may be continuous or sporadic but often include uncontrolled muscle cramps and spasms; unusual twisting of parts of the body and uncontrolled blinking. (3)
Stem cell therapies for dystonia are currently being researched and trialled. One process involves taking a small skin sample from patients with dystonia, growing living fibroblasts from the skin, and then converting the fibroblasts into stem cells for making neurons. Although these cells, collected from the dystonic patient, cannot be used therapeutically, scientists hope that this process will increase understanding of the condition and future therapies.(4,5)
In their 2016 paper, Application of Umbilical Cord Blood-Derived Stem Cells in Diseases of the Nervous System, a team of specialists surveyed the efficacy of cord blood stem cells in treating conditions including stroke, cerebral palsy, sceloris and glioblastoma. They concluded, “It is clear that UCB derived cells have profound neurogenic potential.”(6)
Since this time, we have witnessed the watershed study of cord blood stem cells for cerebral palsy at Duke University, which resulted in improved motor skills in some children: “We are encouraged by the results of this study,” said senior author Joanne Kurtzberg, M.D., director of Duke’s Pediatric Blood and Marrow Transplant Program, “which shows that appropriately dosed infusions of cord blood cells can help lessen symptoms in children with cerebral palsy.”(7)
With so many trials underway, and over 80 conditions already routinely treated with umbilical cord blood stem cells, including blood cancers and diseases of the immune system, scientists are cautiously optimistic about the future of regenerative medicine. A major limitation, of course, is finding a good match between patient and stem cell sample. Birth is a once in a lifetime opportunity to store your child’s cord blood and tissue stem cells, giving your family peace of mind and protection should a matching family member become unwell.