Mesenchymal stem cell therapy in the spotlight

An upcoming visit to Australia by Professor Karussis from the Hadassah Medical Centre, Israel, is once again shining the light on mesenchymal stem cell therapy for the treatment of MS.

What are mesenchymal stem cells?

Mesenchymal stem cell therapy is a different treatment to the more widely recognised therapy autologous haematopoietic stem cell transplantation (AHSCT). AHSCT is primarily a chemotherapy treatment, that completely or substantially depletes the immune system, and then rebuilds the immune system through the re-infusion of the patient’s own blood and immune stem cells (haematopoietic stem cells).

Mesenchymal stem cell (MSC) therapy does not involve chemotherapy and uses a different type of stem cell which can be isolated from different tissues, including bone marrow and fat.

In laboratory studies, mesenchymal stem cells have shown the potential to develop into a range of other cell types including muscle, connective tissue and nerve cells. However, they also secrete growth factors and other factors that can influence the immune system. The evidence to date from animal studies and very early stage human trials suggests that the therapeutic potential of MSCs may come from the chemicals they secrete that appear to calm the immune system and contribute to an environment that is more supportive for self-repair of the central nervous system.

The Haddassah Neurological STEM trial

Professor Karussis is considered to be one of the leaders in this field and has been carrying out early stage clinical trials in Israel testing this therapy in MS and other neurological conditions. He is visiting Australia in an effort to raise funds for his latest trials.

It is important to note that at this stage Professor Karussis work is in the early phases and despite reports and case studies of patients that may have received some early or temporary benefits, it is too early to determine whether this will be an effective treatment to stop or reverse the effects of MS. It is important that careful research is undertaken to ensure the safety and efficacy of this treatment.

Professor Karussis, has previously carried out an open label single arm phase 1 trial – ‘open label’ means people knew they were getting the active treatment, and there wasn’t a group that got a placebo or sham treatment. In this trial, 15 people with MS and 19 people with Amyotrophic Lateral Sclerosis (motor neuron disease) were treated with MSC. In this trial MSC were extracted from the bone marrow from each patient, and grown in a laboratory dish before being injected back into the body by direct injection into the spinal fluid (intrathecal).

Other international MSC trials

This trial is one of a handful of trials which have looked at the safety and feasibility of mesenchymal stem cell therapy in MS. Other trials include the MESIMS trial (Mesenchymal Stem Cells in Multiple Sclerosis) and an American trial which have involved 10 and 20 people with MS respectively.

These previous trials are early phase and contained only a small number of patients. They were primarily designed to determine whether this treatment was safe before enrolling a larger number of people. This is standard practise to ensure patients aren’t put at undue risks. However, from these trials we cannot robustly conclude whether there is any benefit or improvement in MS or not. However, these studies did not report any serious adverse events or deaths.

Together, the results from the various trials support the case for further bigger trials to continue to test the safety, but also start to test the effectiveness of this treatment for MS.

Professor Karussis and his team are currently carrying out a clinical trial assessing the safety and potential benefits of injecting the cells directly into veins (intravenously) or directly into the fluid within the spinal cord (intrathecally). The previously mentioned early phase trials used a mix of intrathecal and intravenous injections, which may have different efficacies and potentially different risks. For example, intrathecal may ensure the cells get closer to the brain but injecting directly into the spinal column can be risky.  Work is also ongoing to investigate different methods to grow the cells outside the body to improve their safety and efficacy, and further trials are planned.

In addition to the Hadassah study, other international groups are also testing MSC therapy for MS. One such group is a large international collaboration known as the International Mesenchymal Stem Cells Transplantation Study Group (IMSCTSG) which includes centres in America, Europe, the UK and Australia. These groups use the same clinical trial protocol and share some key centralised procedures. This is a very useful way to allow independent small trials to be combined for a more robust result to accelerate this area of research. Their study, MESEMS  (mesenchymal stem cells for MS) is testing the intravenous delivery of MSCs

The Tisch MS Research Centre in New York has also been conducting early phase trials of MSCs for MS. They are currently running an FDA approved Phase I study of intrathecal delivery (injection directly into the spinal fluid) of MSCs in progressive MS (More details here)

What’s happening in Australia in MSC research?

Australian researchers working in this field include researchers led by Professor Claude Bernard at Monash University. Professor Bernard has strong international collaborations with others in the field, including the International Mesenchymal Stem Cells Transplantation Study Group.

MS Research Australia has previously funded the research of Professor Bernard and others in this field who have been conducting fundamental research into the properties and potential of MSCs as a treatment for MS. We are also funding other similar research which falls into the broad category of cellular therapies. An example that we are currently funding is the work of Dr Natalie Payne at Monash University.

We look forward to the results of all these trials and studies which will give us a better understanding of the role that MSC therapy may play in reducing the impact of MS.

MS Research Australia is not funding Professor Karussis’s work, but is supportive of any well designed and ethically conducted research that may lead to better treatments and ultimately a cure for MS.



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Mesenchymal stem cell therapy in the spotlight