The recent announcement about the use of stem cells to treat a form of multiple sclerosis (MS), together with early results from the BrainStorm stem cell amyotrophic lateral sclerosis (ALS) clinical trial in Israel have raised the profile of stem cells as a possible treatment for motor neurone disease.
Stem cells are unspecialised cells in the body which do not yet perform a particular function. They can renew themselves and have the ability to give rise to different types of cell, including nerve cells (motor neurones and the surrounding support cells).
Both the ALS/MND study (ALS is a type of motor neurone disease) and the MS study used stem cells found in bone marrow taken from the patient, and then given back to the same patient later on in the process. The MND study gave a new use to the bone marrow stem cells, whereas in the MS study ‘corrupt/damaged’ stem cells were replaced with a new healthier set.
Below we look at both trials in more detail and describe what they mean for people living with MND.Read More »
This year the Symposium session on clinical trials looked at three drugs and one therapy. Dr Brian Dickie has posted a separate blog on one of these drug treatments – Edaravone.
A summary of the results from the drugs and treatments discussed is below. More information on each of them in detail is later on in this blog.
Ibudilast: This drug was safe and well tolerated in those who were not using non-invasive ventilation. However, these are results from an early stage trial so more research is needed to establish possible long-term benefit.
Methylcobalamin (Vitamin B12 injections): If this treatment is given early (within 12 months of diagnosis) then it showed an effect at increasing survival in a small sub-group of those taking part in the trial. This effect was not seen when the treatment was given further on from diagnosis.
Stem cell therapy: This small, early Phase 1/2 trial was testing the safety of bone-marrow derived stem cell injections into the spinal cord. The researchers found this treatment had no major side effects. Further studies are needed to evaluate the effectiveness and safety of this treatment over the long-term.
“On the fourth day of Christmas MND research gives to you… on the FOURTH month of 2014, we announced that we’ll fund an exciting new stem cell project”
During our April Biomedical Research Advisory Panel Meeting we agreed to fund seven new MND research projects. These projects included Prof Linda Greensmith’s research on Restoring muscle function with transplanted stem-cell derived motor neurones.
Based at University College London, this study will use stem cell technology to restore muscle function in a mouse model of MND. The researchers will transplant stem-cell derived motor neurones and then guide them to where they’re needed using light.
Prof Greensmith and her team aim to restore function to the muscles that are responsible for breathing and develop an optical stimulator, which can then be implanted into the body to stimulate the transplanted cells for long periods of time. If successful, this technique could form the basis of future treatments that could potentially restore muscle function in MND.
On 22 September 2014 Neuralstem announced the results from their long-term follow up of three participants who were involved in their initial phase I safety trial. Here’s what the news means and what may be next for Neuralstem’s stem cell treatment.
What is Neuralstem?
Neuralstem is the company behind an innovative neural stem cell treatment for MND. Stem cells are immature cells that have the capability to become any cell in the body. By transforming these in to ‘motor neurones’ it is hoped that they could be used to replace the damaged motor neurones in people living with MND.
Stem cells as a treatment for MND are currently unproven. This means that stem cells have not undergone rigorous testing, by means of a clinical trial, to establish if they are both safe and beneficial in MND. You can find out more about unproven treatments here.
Neuralstem’s stem cell treatment for MND involves several injections of these neural stem cells into the spinal cord of patients. Previous research in animals has shown that these cells make connections with healthy motor neurons and express nerve protecting factors, which could be potentially useful as a treatment for MND.
A question was submitted to the Association’s AGM last weekend, which could only be answered in brief at the time, due to the number of issues raised, some of which are of a technical nature. Below is a more detailed response from the Association’s Director of Research (in bold italics) to each point raised.