By Don C. Reed
December 20, 2023 UPDATE :
The study is reopening January 15th to treat an additional 28 patients to determine how well the therapy works (a phase 2 trial).
Original article:
People ask: what has the California stem cell program done with the money entrusted to it? Here is one example.
As the father of a paralyzed young man*, I sit up straight if someone mentions a possible cure for any kind of paralysis.
The most common form of lifelong paralysis is spina bifida, a vicious condition where the unborn baby’s spine does not completely cover itself. That gap in spinal covering brings devastating damage and loss of abilities: paralysis, cognitive malfunction, bowel and bladder problems.
However, after two decades of struggle, Dr. Diana Farmer of UC Davis may have brought the world an actual cure.
Farmer began science as a marine biologist, doing research at the famous Woods Hole Institute. On her way to receive an award, she suffered a car accident. This changed her mind, and she decided to work on human biology instead. She became the world’s first woman to perform surgery on a baby, while still inside its mother’s womb. (1)
“She and Aijun Wang received a CIRM grant to co-launch the world’s first human clinical trial using stem cells to treat spina bifida..” (2)
Doctor Farmer also had the help of fetal surgeon Shinjiro Hiroshi and other leaders in the field.
Surgery alone was not enough, “ …leaving 58% of patients unable to walk independently.”
And without sufficient funding, no amount of expertise could prevail. The California Institute for Regenerative Medicine (CIRM) came through with more than $9 million in research grants toward this goal.
The experts were ready; the funding was in place. How would the procedure work? The baby was operated on during the pregnancy. The mother’s uterus was opened slightly; a bioengineered mesh was inserted over the gap in the baby’s spinal cord covering. Mesenchymal stem cells (from a donor’s placenta) were placed on the meshing.
The procedure was tried first on animals born with spina bifida: including sheep and two bulldogs, Spanky and Darla, which were healed.
Human trials were funded by the California stem cell program.
Nothing was easy. It took four days for Priya Kumar, the scientist whose team created the stem cell patches, to develop and deliver them to the operating room.
Pregnant Emily Andrews and her child (diagnosed with spina bifida) went under surgery.