An ongoing clinical trial at UC Davis Health shows that a new stem cell therapy appears to be successful in treating neonates with spina bifida-related paralysis and other issues.
The novel procedure, which is carried out while a foetus is still in the womb, has so far been administered to three newborns.
The principal researcher and chair of surgery at UC Davis Health, Diana Farmer, MD, told The Sacramento Bee that the most exciting moment was when a baby was born and began moving her legs and wiggling her toes, which far exceeded expectations.
She recalled saying, “Oh my God, I think she’s wiggling her toes,” as soon as she was born. “It was extremely evident the minute she was born that she was kicking her legs,” she said.
The official name of the clinical trial, which was started in early 2021, is “CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele.” According to the CDC, myelomeningocele is a severe form of spina bifida in which the spinal canal does not completely close before delivery, resulting in spinal cord injury. Children born with the illness frequently have paralysis and have no control over their bowel movements. An ultrasound is used to diagnose the illness, which affects 1,500 to 2,000 children annually in the United States.
The currently advised course of action, known as foetal repair, is for surgery to close the opening throughout pregnancy. The UC Davis researchers created a stem cell therapy that is applied during foetal repair surgery and extracted from the placenta to promote recovery.
The goal of the early-stage clinical experiment is to evaluate the safety of 35 patients receiving stem cell therapy versus neonates who receive foetal repair without the use of stem cells. The research team will follow the infants until they are 6 years old, with a crucial visit to see if they are walking and potty training at 30 months.
According to a press release from UC Davis, Emily, the first clinical study participant, remarked that “this clinical research could improve the quality of life for so many individuals going forward.”
Emily made the trip to the trial from Austin, Texas. When Robbie was born in September of last year, she could move her toes.
Before the diagnosis, “we didn’t know about spina bifida,” she claimed. “We are extremely appreciative that we were able to participate in this. We are providing our daughter with the finest opportunity for a successful future.
Farmer has been researching treatments for spina bifida for 25 years and was a pioneer in the use of prenatal surgery to lessen the neurological abnormalities associated with the condition, according to The Sacramento Bee. She conducted a clinical research in the early 2000s that shown how foetal surgery could lessen undesirable outcomes, albeit some kids still need assistance with walking.
Since then, she has persisted in looking into ways to assist children with spina bifida, bringing foetal surgeon Shinjiro Hirose, MD, and bioengineer Aijun Wang, PhD, to UC Davis in order to start a surgical bioengineering lab for stem cell research and a foetal treatment centre. For more than ten years, the research team has been working on the novel method of employing stem cells during foetal surgery, looking for the best stem cells to use and figuring out how to use them in sheep and dogs.
According to Wang’s remark, the newborn lambs who got stem cells were able to stand and move around virtually properly when they were born. It was incredible.
The first dogs in the world to undergo the procedure were two English bulldogs by the names of Darla and Spanky. The breed’s most prevalent birth abnormality, spina bifida, frequently renders dogs without functional hindquarters. But by the time Darla and Spanky were 4 months old and having their post-surgery check, they could walk, run, and play.
The study team then turned to human safety studies. The California Institute for Regenerative Medicine, the stem cell organisation that voters authorised in 2004 through Proposition 71, provided a $9 million funding for the present clinical research.
Although the experts have acknowledged reluctance to make final conclusions just yet, the treatment seems promising. Throughout the course of the study, they intend to provide updates on the babies’ progress toward several developmental milestones.
Emily added, “This experience has been bigger than life and has well above any expectations. “I’m hoping that this trial will improve patients’ quality of life for a very long time. Being a part of history being made is an honour.
