Scientists may be one step closer to a breakthrough that uses stem cells to replace damaged skull and facial bones in patients who experience a head trauma or undergo cancer surgery requiring repair and reconstructive surgery.
Researchers have discovered and isolated stem cells capable of repairing these bones in mice. The research, led by Takamitsu Maruyama and the research team at the University of Rochester Medical Center in Rochester, N.Y., could also help patients born with a skull deformity known as craniosynostosis, which can lead to developmental delays and pressure on the brain.
In the study, scientists investigated the role of the Axin2 gene in bone formation and regeneration. They also examined a specific mutation that causes craniosynostosis in mice. Their finding show that stem cells involved in skull formation are contained within this cell population. These cells are specificto the bones in the head and are very different from other stem cells involved in the formation of the bones in the legs and other parts of the body.
Tests to uncover these cells could also help physicians detect bone diseases caused by stem cell abnormalities, according to the researchers.
The research was published Feb. 1 in the journal Nature Communications.
The British Broadcasting Corporation (BBC) recently reported that stem cell transplant treatments normally used for cancer patients are helping Multiple Sclerosis (MS) patients in the UK. According to the January 18, 2016 report, 20 patients received bone marrow stem cell transplants using their own stem cells, and that at least some of the patients who were paralyzed by MS are able to walk again post-treatment.
Approximately100,000 people in the United Kingdom suffer from MS, with most new patients diagnosed between the ages of 20 and 30 years of age.
“To have a treatment which can potentially reverse disability is really a major achievement,” says Prof Basil Sharrack, of Sheffield’s Royal Hallamshire Hospital in Sheffield, England.
The treatment, known as autologous hematopoietic stem cell transplantation (HSCT), involves the intravenous infusion of autologous or allogeneic stem cells harvested from the patient’s own bone marrow to reestablish hematopoietic function (formation of blood or blood cells) in patients whose bone marrow or immune system is damaged or defective by chemotherapy. Using stem cells harvested from the patient’s bone marrow helps rebuild the immune system. The theory is that these newly harvested cells are at such an early stage in development that the cellular defects that result in MS do not exist.
“The immune system is being reset or rebooted back to a time point before it caused MS,” says Prof John Snowden, consultant hematologist at Royal Hallamshire Hospital.
The BBC’s Panorama program spoke to several MS patients who have undergone the stem cell transplant.
Steven Storey was diagnosed with MS in 2013 and, within a year, went from being an able-bodied athlete to wheelchair dependent and losing sensation in much of his body.
“I went from running marathons to needing 24-hour acute care. At one point I couldn’t even hold a spoon and feed myself,” Storey says.
Within a few days of the transplant Storey was able to move his toes, and after four months he could stand unaided.
While Storey still needs a wheelchair for mobility, he calls his progress astounding.
“It’s been incredible,” he says. “I was in a dire place, but now I can swim and cycle and I am determined to walk.”
The Royal Hallamshire Hospital along with hospitals in the United States, Sweden and Brazil, is part of an international clinical trial called MIST that is assessing the long-term benefits of the stem cell procedure on MS patients. Study participants all have relapsing remitting MS (RRMS), and received intensive chemotherapy to completely destroy the patients’ immune systems.
Treatment costs are about the same as the annual cost for existing treatments, and the stem cell treatment does not require the use of new or existing medications.
Prof Richard Burt of Northwestern University in Chicago carried out the first hematopoietic stem cell transplantation for MS in 1995, and is coordinating this current MIST international trial, which began in 2006.
“There has been resistance to this in the pharma and academic world,” Burt says. “This is not a technology you can patent and we have achieved this without industry backing.”
A study published last year involving MS patients in Chicago showed significant reductions in neurological disability, and for some the improvements persisted for at least four years, although there was no comparative control group.
The outcomes of the current international trial will be reported in 2018, and may determine whether the stem cell transplant becomes a standard in the United Kingdoms health care system for many MS patients.
“Ongoing research suggests stem cell treatments such as HSCT could offer hope, and it’s clear that in the cases highlighted by Panorama they’ve had a life-changing impact,” says Emma Gray, M.D., head of clinical trials at UK’s MS Society.