Almost 69% of patients with relapsing-remitting multiple sclerosis after high-dose immunosuppressive therapy with further stem cell transplantation managed to achieve a stable 5-year remission of the disease.
The promising results of a small clinical trial of Phase II HALT-MS is reported by the American journal Neurology.
The project was led by Dr. Richard Nash, a researcher at the Colorado Blood Cancer Institute, an oncologist at St. Luke's Presbyterian Hospital (Denver).
The authors recall that the share of relapsing-remitting multiple sclerosis accounts for about 85% of all cases of the disease. With multiple sclerosis, the immune system of patients damages myelin - a substance that forms the protective envelope of neurons. The disease is characterized by impaired conduction of nerve impulses in the brain and spinal cord.
Symptoms of multiple sclerosis (MS) can be muscle weakness and stiffness of movements, gait and balance disorders, chronic pain, weakness, dizziness, cognitive deficits and vision problems.
The clinical picture is very variable.
The peculiarity of relapsing-remitting flow of MS are periodic exacerbations (relapses) of the disease. These relapses of different duration and intensity are followed by periods of partial or complete remission.
To date, it is impossible to completely cure multiple sclerosis, but there is a mass of drugs for controlling symptoms.
A new study showed that a single high-dose immunosuppressive therapy (VIST) with further transplantation of autologous hematopoietic stem cells (TSCC) does provide a long-term remission.
We should note that similar therapy (VIST + TGSK) with varying success is experienced since the 1990s by scientists from Canada, Israel, Germany and a number of other countries.
The goal of the therapy is to "reset" the diseased immune system, that is, the removal of immune cells that attack the nerve fibers and replace them with new cells.
For treatment from the patient's own blood, hematopoietic stem cells are extracted, after which his immunity is destroyed by an overdose chemotherapy. In the future, autologous ("native") stem cells return to the body.
In 2014, Dr. Nash and his colleagues reported on the successful 3-year Phase II trial results.
Then Medbe. ru covered in detail the details of this work.
In the project, 24 volunteers were recruited with a relapsing-remitting form of multiple sclerosis (MS) aged 26 to 52 years. All received standard medical treatment, but completely unsuccessfully, which prompted them to participate in the experimental program VIST + TGSK.
After treatment, 80% of patients had no exacerbation or deterioration for three years, no new foci in the brain. Now, after five years after therapy, 69% of patients remain in the phase of stable remission.
Particularly inspiring is the fact that none of these patients take medication.
Their quality of life and ability to work increased significantly.
The main side effects of VIST + TSCC were cytopenia and infections, since during the "reset" of the immune system the body is deprived of protection for a certain time. Also in 5 years of observation, three patients died, but the researchers do not connect their death with experimental treatment.
Although further studies are needed to study the long-term effectiveness and safety of stem cell therapy, Dr. Nash is optimistic.
"If the results are confirmed by larger studies, VIST + THSC will be a valuable therapeutic option for MS patients who are not helped by standard drug treatment," says Dr. Daniel Rotrosen, a representative of NIAID.
The National Institute of Allergic and Infectious Diseases (NIAID) has provided funding for the study of new therapy for multiple sclerosis.
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