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.
The Phase I trial
Phase I clinical trials aim to establish if a potential treatment is safe, and are usually only tested in a small number of people. You can find out more about the different stages of clinical trial testing below or via our website.
The results of the first American Food and Drugs Administration (FDA) approved phase I trial of Neuralstem’s stem cell treatment for MND were presented in April 2013. The treatment was tested in 18 people living with MND and found to be both safe and well tolerated, with surgery shown to not accelerate disease progression.
The long-term follow up data news
Three trial participants from the phase I trial were followed up for over three years post-treatment. The news announced by Neuralstem on 22 September refers to these participants.
Neuralstem announced that there was a significant slowing of disease progression for over three years after treatment with Neuralstem, highlighting the potential beneficial effects of the treatment.
The phase I trial involved 18 people. Of these, only three people were followed-up to establish the long-term survival of the transplanted cells. These people were found to have experienced slower progression of MND compared to before they had taken part in the trial. The three participants who experienced a slowing of disease progression also had similar disease characteristics (eg no bulbar symptoms) and as the phase I trial was only assessing the safety of the treatment the scientific and medical community will need further evidence as to whether or not the treatment is beneficial.
Larger numbers of participants are needed to establish if the treatment is truly beneficial in a phase III trial (only 18 people took part in the phase I trial above). Because MND presents differently, a more detailed trial with more people is needed to establish if the treatment is truly beneficial.
A phase II trial, involving 18 people, giving injections into the cervical spinal cord at increasing doses (numbers of cells), is funded and is approved by the FDA. This trial began in September 2013, with the last surgery taking place in July 2014. Results are expected in late 2014/early 2015 following a six month observational period.
Once the phase II results have been announced we will know whether or not Neuralstem will be pursuing further testing, and whether or not there will be a phase III trial to establish if the treatment is beneficial or not. This stage of testing will likely involve large numbers of people (hundreds) and if Neuralstem wish to conduct a trial in the UK, the MND Association will work with them in the design and implementation of this trial.
Find out more:
You can find out more about clinical trials in our research information sheet D. Information about the Neuralstem clinical trial and stem cells is available on our website, as well as information on unproven treatments.
The different stages of clinical trial testing:
Clinical trials are divided into four phases:
- Phase I examines the safety of the potential new treatment, often in just a few (5 – 20) people. In many cases, this phase involves healthy volunteers rather than patients. Participants are monitored for adverse reactions or side effects; if any appear that are judged to be too dangerous, the drug will not advance any further through the clinical trials process.
- Phase II determines the optimal dose size, timing of doses and drug delivery route (eg by mouth, by injection) for the next phase of testing. Although Phase II testing may provide some indication of the drug’s ability to treat the disease, the numbers of patients involved in this phase is too small for such findings to be relied upon.
- Phase III aims to show whether or not the drug actually has a beneficial effect on patients. This stage of testing will usually involve hundreds of patients, which is enough to allow a reliable assessment of the drug’s efficacy. Phase III results will determine whether or not a drug is approved for use to treat the disease.
- Phase IV occurs after the drug has been approved for sale. With the drug in general use, further data can be gathered on its effects in an extremely large number of people over an extended period of time.