UK scientists getting ready to work on major international trials to investigate the safety and effectiveness of stem cells in slowing, stopping or reversing brain and spinal cord damage in people with multiple scleroris (MS) have received £1 million in funds from the MS Society and the UK Stem Cell Foundation (UKCSF).

Many scientists believe this unique international collaboration will significantly reduce the timescales for finding out if stem cells are safe and effective in treating MS.

In a statement issued today, 29 July, the MS Society said that the funding is for three UK studies within the international trials.

One of these will investigate the use of autologous mesenchymal stem cells as a form of immunotherapy to prevent and potentially reverse neurological deterioration in relapse-remitting MS. The trial, a collaborative phase II study, will involve 150 to 200 MS patients from across the world, including UK, Italy, the US and Canada.

In the UK, the trial will be sited in London and Edinburgh, with Dr Paolo Muraro from Imperial College London as study leader.

Muraro and colleagues will collect bone marrow stem cells from 13 MS patients, grow them in the lab, then re-inject them back into the same patients, such that each patient receives a large boost of his or her own stem cells.

The hope is that the stem cells will travel to the brain and start to repair the damage caused by MS, including that currently in progress in “active lesions”.

Another project, led by Professor David Baker of Queen Mary University of London, will evaluate the use of transplanting neural stem cells as a therapy for optic neuritis, a symptom of MS that impairs sight because the optic nerve in the eye becomes inflamed.

And in a third project, study leader Professor Cris Constantinescu of Queen’s Medical Centre (QMC) Nottingham, and colleagues, will be finding out more about mesenchymal stem cells, which influence the immune system and have potential to protect and repair brain cells and nerves. They will be using ultra-high field imaging and looking in particular at mesenchymal stem cells’ phenotypic characteristics and immune interactions, so we know more about these types of cells for future trials.

In the UK, MS affects about 1 in every 650 people. Having parents or siblings with MS reduces a person’s risk of developing the disease to about 1 in 100.

MS is caused by the body’s own immune system attacking and damaging the protective myelin sheath that stops nerve fibers short-circuiting in the brain and spinal cord. The damage stops messages travelling to and from the brain and the body, so that patients eventually become sight-impaired, get stiff muscles, become disabled and lose control of their bladder and bowels. Although more and more drugs are becoming available that treat some aspects of MS, none as yet can reverse the damage or limit the progression of the disease.

Stem cells are cells that have the potential to become other cells in the body, under the right conditions. Thus they have great potential as therapies where tissue can be repaired by replacing damaged cells with new cells. And because they have the same DNA as the patient, there are no problems with rejection as there might be with donated cells.

However, there are some risks, such as what if the cells grow uncontrollably, like cancer, and this is one of the things the scientists will be trying to control and look out for in the trials.

It is interesting that the chance of developing MS if you have an identical twin with MS is about 1 in 4 and not 100%. If one identical twin has MS it does not necessarily mean that the other will develop it, even if though they have 100% genes in common. MS is not simply a genetic disease, it seems, hence the importance of exploring phenotypic characteristics, as these are to do with how genes are influenced by environment.

Simon Gillespie, Chief Executive of the MS Society, told the press that:

“Stem cells hold tremendous potential as a future treatment option for people with MS. We are delighted to be funding this world leading research which shows the power of an international research collaboration and joint working between charities.”

Lil Shortland, Chief Operating Officer of the UKSCF, said thanks to the joint effort with MS Society, it has been possible to raise the vital funding these new projects need more quickly:

“MS is such a debilitating condition that devastates the lives of so many people, particularly in the UK. We hope these projects will lead ultimately to the development of successful new treatments for multiple sclerosis within the next three to five years,” she added.

Sources: MS Society, UK Stem Cell Foundation, (MS).

Written by: Catharine Paddock, PhD