An innovative stem cell treatment developed by Hadassah-University Medical Center and NeuroGenesis has led to significant clinical improvements in patients with progressive multiple sclerosis, according to the results of a Phase II clinical trial.
The results of the placebo-controlled, randomized double-blind trial, which found that treatment not only halted progression of the disease but led to improvement in neurological ability, were published in Oxford University’s peer-reviewed journal Brain.
Multiple sclerosis (MS) is an autoimmune disease that plagues more than 2.3 million people worldwide, according to the US-based National Multiple Sclerosis Society. The disease causes damage in the myelin – the covering that protects the nerves and promotes efficient transmission of nerve impulses – as well as in the nerve cells of the central nervous system, ultimately leading to a neurological disability.
People with MS often experience tingling in their limbs or numbness, can have trouble walking and using their hands, and eventually can even become paralyzed. The condition is also associated with visual problems.
To date, not only is there no known cure, but people with progressive MS have few if any working treatments. Those that do exist are meant to reduce the incidence of relapse or marginally slow the progression of the disease.
Hadassah’s development, known as NG-01, uses an “autologous proprietary subpopulation of mesenchymal stem cells.” In layman's terms, the treatment uses cells derived from the bone marrow of MS patients themselves, ensuring that there is no element of rejection. The cells are injected into the spinal cord fluid of the patients’ central nervous system.
The cells then travel to the site of injury and create a “repair ecosystem” in the lesion area, explained NeuroGenesis CEO Tal Gilat.
The process is relatively simple and takes about 20 minutes under local anesthesia. The patient spends a couple of hours in the hospital for observation and is then free to go home.
The technology was further developed by NeuroGenesis, following a license from Hadasit, Hadassah’s technology transfer company.
“The patients’ improvement was in many cases quite dramatic,” Gilat said.
Nearly 60% of patients did not show any evidence of disease activity during the entire treatment period, compared to 10% in the placebo group, which translates to a more than 80% reduction in the progression of the disease.
Some 20% of those treated with the stems cells in their spinal fluid showed improvement after a first injection. After six months, 73% not only did not experience disease progression but also improved.
Improvements included a better ability to walk, increased muscle power, neurological improvements in cognitive function and improvements in the dexterity of their hands. A functional MRI also showed that there was improvement in the motor function.
Gilat described patients who could walk again, including one who went from difficulty moving to running 10 miles.
“Although we currently have several good treatment options for relapsing remitting MS, we fall short in providing effective treatment for progressive MS that could substantially suppress the progression of disability,” said Hadassah’s Prof. Dimitrios Karussis, who led the study.
“This trial provides encouraging results and suggests the potential for a new approach that may not only slow down the progression of the disease but even induce improvement and promote repair mechanisms in progressive MS,” he said.
The Phase II trial involved 48 patients and lasted for three years. However, some 140 patients have received the treatment, during a Phase I trial and through compassionate use at Hadassah.
In none of the studies have there been any serious, treatment-related side effects. Currently, the results are short term and require continual treatment to remain effective. Gilat noted that the treatment does not "deal with the underlying cause of the disease, which is yet unknown. We are improving the symptoms."
Karussis added that “on the one hand, we should always be cautious interpreting positive effects from medium-size studies – and large-scale trials are needed to confirm these findings.
On the other hand, when we see such actual neurological improvements in patients with active and progressive disease, we cannot be restrained from being enthusiastic,” he said. “These are certainly very strong findings.”
Gilat said that Hadassah and NeuroGenesis have also conducted early-stage trials using the treatment in patients with amyotrophic lateral sclerosis (ALS) with promising results.
NeuroGenesis and Hadassah have met with the US Food and Drug Administration and shared the data from their first trials. Next, they intend to launch a multicenter Phase III trial in the US, Europe and Israel, and then start the application process for approval. Karussis estimated that it could happen in two to five years.