Multiple Sclerosis: How Stem Cells Could Help

There are 2.3 million people around the world who are suffering from Multiple Sclerosis, according to the National Multiple Sclerosis Society. In the US alone, over a million people over the age of 18 are afflicted with this disease.

Battling this disease has never been easy for patients and their families who often have to put their lives and plans on hold.

There is currently no cure for Multiple Sclerosis. Alongside established treatments, stem-cell-based approaches have become an area of active scientific interest, and early research has reported findings that may be relevant to how the disease is managed.

Over time, doctors and scientists have worked not only to address the symptoms of multiple sclerosis but also to influence the underlying disease process. Established care is directed by the treating neurologist and can combine several types of measures:

• Oral treatments. Several disease-modifying medicines, such as Fingolimod, Dimethyl Fumarate, Teriflunomide and Siponimod, are used to reduce the relapse rate; some may also slow progression. These are prescription-only medicines, and their suitability, benefits and possible side effects are assessed by the treating physician.
• Infusion treatments. Medicines such as Ocrelizumab, Alemtuzumab and Natalizumab are given intravenously and are used, depending on the situation, to reduce relapses or to treat more active disease. They are prescription-only and are selected and monitored by the treating physician.
• Injectable medications. Beta interferons and Glatiramer acetate are administered by injection to reduce the frequency and severity of relapses.
• Physical therapy. Exercises such as stretching, guided by physical and occupational therapists, are a routine part of MS care and can support a patient's mobility.

Alongside these established treatments, many of which address the symptoms of MS, stem-cell-based approaches have attracted growing scientific interest. Research has reported encouraging early findings, and further studies continue to investigate what these approaches may offer.

Hematopoietic stem cells, which are found in the bone marrow, have been studied for the treatment of Multiple Sclerosis since 1997. To understand why stem cells are of scientific interest in MS, it helps to understand how the disease works.

To make it simple, Multiple Sclerosis happens when the immune system mistakenly attacks the myelin sheath of nerve cells. The myelin sheath is a protective layer around the nerves that allow the brain to send commands to different parts of the body. So if the myelin sheath is damaged, a person's impulses slow down.

This is where stem cells become scientifically interesting. Research has explored two directions in particular: whether stem cells, or the factors they release, might support damaged nerve tissue, and whether they might help protect the myelin sheath. These mechanisms provide part of the scientific rationale behind stem-cell-based approaches.

Early-phase clinical studies have investigated stem-cell-based approaches in Multiple Sclerosis. Phase I and II trials have reported encouraging findings, including reduced inflammatory activity, improvements in visual function and fewer relapses. As these were early studies in relatively small numbers of patients, larger trials are needed to establish how broadly the results apply.

A study led by the University of California reported that a nanotechnology-based treatment reversed MS-like symptoms in mice, and a further study from the University of Cambridge reprogrammed skin cells into neural stem cells and reported reduced inflammation and repair of nervous-system damage in mice. These are preclinical studies in animal models and represent an earlier stage of research, but they add to the scientific rationale for continued investigation.

A common question most patients and their families have is: Does stem cell therapy work for primary progressive MS? Stem-cell-based approaches are not a cure. Research has, however, explored whether they might help reduce the impact of the disease, and some early studies in progressive MS have reported encouraging results.

These findings are still limited, and further clinical evaluation is ongoing to understand which patients may benefit and to what extent.

A frequently cited trial, the MS Stem Cell Transplant Trial, reported that in patients with active relapsing-remitting MS, a hematopoietic stem cell transplant was more effective than continued drug treatment. This raises the question of what a stem cell transplant for MS involves, and how it differs from other stem-cell-based approaches.

A stem cell transplant is merely a step or procedure in the overall therapy. The act of transplanting itself is just injecting stem cells into the patient's body, which may come from a host or from the patient themselves when they were still able to produce stem cells. (This is why cancer patients who have undergone chemotherapy or radiation need a stem cell transplant afterwards.)

It is worth being clear that hematopoietic stem cell transplantation is an intensive hospital procedure involving conditioning of the immune system, and it differs substantially from other stem-cell-based approaches. Results reported for this procedure therefore cannot simply be transferred to them.

In the trial, patients treated with their own hematopoietic stem cells showed a slowing of the disease, more remission, fewer relapses, less disability and fewer new MRI lesions. As these are results from an early trial in a selected group of patients, follow-up research is underway to confirm them.

There is no cure for Multiple Sclerosis, but research into stem-cell-based approaches has reported encouraging results. In some studies, particularly of hematopoietic stem cell transplantation, disease progression slowed or stabilised in treated patients. The evidence is still developing and results vary between studies, but the direction of these findings is one reason for the continued scientific interest in the field.

In the autologous hematopoietic stem cell transplantation study referred to above, 52 of the 55 patients who received transplantation showed no disease progression over one year, compared with 21 of the 55 patients in the control group who received medication alone.

Over the following years, the reported proportion of transplanted patients whose disease had progressed remained low: around 2% in the second year, 5% in the third year and 10% in the fourth and fifth years. These figures come from one early trial in a selected group of patients, and larger studies will help show how widely they apply.

The time frame depends on the individual patient's condition and medical history. For hematopoietic stem cell transplantation, sources such as the MS Trust describe that transplanted cells settle in the bone marrow and begin producing new blood cells within around 10 to 30 days.

Because people respond differently, the period over which any change might be noticed can also vary from one patient to another.

A lot of places around the world are offering stem cell treatment for Multiple Sclerosis patients. The price of the treatment varies due to the standards and policies of every country.

The average price range per treatment in the US is USD 7,000 to USD 10,000. Therapies outside the US are reported to be anywhere from USD 20,000 to USD 100,000, depending on the country.

In the UK, the National Health Service has not formally assessed stem cell transplants but there are several centers that started offering them under very strict regulations.

If you are considering a stem-cell-based approach for yourself or for a family member, it is worth remembering that any such decision depends on the individual medical situation and is best discussed with the treating physicians on the basis of a full medical assessment, rather than on cost alone.

Many countries allow stem-cell-based treatments for patients with Multiple Sclerosis. Here are some of these places:

• United States
• Canada
• United Kingdom
• China
• Russia
• Mexico
• Cayman Islands
• Germany

What we have to remember is that each country has its own policies and standards. There have been reports of clinics offering unregulated stem cell treatments in some parts of the US, Canada, and Mexico. This is why it's important to study the country and clinic where you plan to get your treatment.

Countries such as Germany apply strict regulatory standards to how cell-based treatments are manufactured and administered, which is one reason patients often look to well-regulated settings for these approaches.

At ANOVA IRM, stem-cell-based approaches are applied in selected patients following an individual medical assessment, and each treatment programme is tailored to the individual. If you have any questions about our procedures, feel free to contact us.

Stem-cell-based therapies for Multiple Sclerosis remain a developing area of medicine. The available evidence varies by indication, individual outcomes cannot be predicted, and treatment at ANOVA is offered only after an individual medical assessment.