Stemcell therapy for multiple sclerosis is now a reality, not just a dream, says a leading neuro-immunologist.
Gianvito Martino, director of neuroscience at San Raffaele Hospital in Milan, Italy, said although the therapy was still experimental, it was yielding some exciting results.
Professor Martino said he did not believe stemcell therapy would be the solution to MS but an important treatment option with fewer side effects.
"To have the solution, we should know the cause of the disease but we don't know it," he said.
About 85 per cent of patients had relapsing remitting MS, which could be managed with current treatments, he said. However, within 10-20 years, about 90 per cent of these patients moved into the secondary progressive phase, for which there were no effective treatments, while about 10 per cent continued to have the so-called benign first phase.
"About 80-85 per cent of patients will need aid for walking in 20-25 years from diagnosis," he said.
The average age of diagnosis is 20-40. In Australia, three times as many females are affected. In the autoimmune disease, the insulating sheath of the nerve cells, called myelin, is attacked and destroyed and eventually the nerves are also destroyed, leading to progressive atrophy of the brain and spinal cord, which is the cause of disability. In Australia, the incidence of MS is about one in every 1000 people, with more than 21,000 people affected.
Professor Martino said there were two types of stemcells already being used in patients, both from blood. They were haematopoietic and mesenchymal stemcells.
Haematopoietic stemcells were those used in bone-marrow transplantations. The patient's immune system was destroyed by chemotherapy and then their own stemcells from the bone marrow were transplanted.
"The idea is to have new blood with no more cells capable of damaging your myelin," Professor Martino said.
"It is immuno- suppressive therapy, blocking the cells causing the disease."
About 500 MS patients worldwide had received the therapy since 1997 and in many, the progression of their disease had been halted.
"The results are very, very important because about 60 per cent of those patients do not worsen for up to four to five years after the transplants, they stabilise," Professor Martino said.
Even more exciting was the fact that only the patients with the worst prognosis and unresponsive to approved therapies had been eligible for the treatment, which was proving so successful.
Among those patients, the ones better responding to the transplant were the 5 per cent with the so-called malignant form of MS, who needed a wheelchair within five years of diagnosis.
The second type of transplant used mesenchymal cells, which are multi-potent stemcells taken from the blood and which can differentiate into a variety of cell types.
"They seem to help the immune system to block the body's reaction against itself," Professor Martino said. "You can just inject them intravenously and you don't need immuno-suppression or any therapy to avoid rejection."
While they seemed to block further damage from the disease, they did not repair nerve cells already damaged.
Apart from blood stemcells, there is another form of stemcell therapy which his group is testing, using neural stemcells taken from a foetus and grown in vitro as precursors of brain cells, which are then transplanted via a lumbar puncture.
"Those cells were not only able to become cells producing myelin once transplanted but they could also help cells resident within the brain, which were not damaged by the disease, to repair the brain," he said.
The trials conducted by his group to date have been in mice and monkeys. "We hope to start treatment in patients . . . within the next five years," he said.
He warned MS patients against going to the so-called stemcell clinics that were scattered worldwide, saying the therapy should be conducted only under rigorous clinical trial conditions.