Page URL:

Motor neurone disease stem cell trial advances to next phase

22 April 2013
Appeared in BioNews 701

A neural stem cell therapy aimed at treating the symptoms of amyotrophic lateral sclerosis (ALS) is set to advance in the USA, after the Food and Drug Administration (FDA) gave its approval for it to proceed to the next stage of clinical trials.

Up to 15 patients with ALS will be enrolled to receive injections of neural stem cells derived by US company NeuralStem from the spinal cord of an eight-week-old aborted fetus. It is hoped that the treatment will combat the symptoms of the disease by halting its progression or even reversing some of the effects.

The first phase of the clinical trials, carried out at Emory University, Atlanta, established safety of the procedure in humans. The technique involved a series of injections consisting of up to 100,000 cells each, given directly into the grey matter of a patient's spinal cord. The results of the safety tests came back highly positive. A small patient subgroup also demonstrated some interruption to their disease progression after long term treatment during the first stage.

The next stage of the trials, which will take place at both the Emory ALS Center, as well as the University of Michigan, is designed to test the efficacy of the neural stem cells as a treatment for ALS, while still constantly monitoring safety aspects. Patients will receive up to 40 injections of up to 400,000 cells per injection.

Professor Jonathan Glass, director of the Emory ALS Center said: 'We are looking forward to progressing the dosage to the maximum safe tolerated dose, where we could also hope to see meaningful effects for the patients'.

While the injection sites in the first stage trial were mostly confined to the lower back region of the spine, in the second stage injections will also be given at the upper neck in the cervical spine area, where it is thought that the therapeutic action of the neuron stem cells will help preserve breathing function. Twelve patients will receive cervical injections and three will receive injections at both areas.

Principal investigator Professor Eva Feldman, a neurologist from the University of Michigan, said: 'I am overjoyed that finally there will be a new trial to see whether stem cells could possibly lead to a new treatment for ALS, and it will be happening here in Michigan'.  

ALS, also known as Lou Gehrig's disease, is a late onset neurodegenerative disease where nerve cells that control voluntary muscle movements are damaged, leading to paralysis in those affected. The eventual loss of function of chest wall muscles and the diaphragm results in breathing complications, which is the cause of most deaths. ALS affects around one in every 50,000 people.

Approval for the trial from the University of Michigan Review Board is expected by June this year, now that phase two testing has been given the go-ahead by the FDA.

Mich. University joins Lou Gehrig's clinical trial
USA Today |  18 April 2013
Neuralstem Receives FDA Approval To Commence Phase II Stem Cell Trial In Amytrophic Lateral Sclerosis
Neuralstem (press release) |  17 April 2013
U-M to join ALS stem cell research
The Detroit News |  18 April 2013
1 August 2016 - by Jenny Sharpe 
Donations from the ALS 'ice-bucket challenge' have funded research that has led to the discovery of new genes linked to the disease...
5 October 2015 - by Dr Hannah Somers 
Reactivation of an ancient virus embedded in the human genome may trigger the onset of motor neurone disease...
27 October 2014 - by Dr Shanya Sivakumaran 
A man who was paralysed from the chest down has begun to walk again, with the use of a frame, following the transplantation of cells into his spinal cord...
13 October 2014 - by Rhys Baker 
The US Food and Drug Administration (FDA) has awarded 'fast-track' status to a potential stem cell therapy for Amyotrophic Lateral Sclerosis (ALS), or motor neurone disease...
26 August 2014 - by Dr Lux Fatimathas 
Scientists have generated the specialised cells in the embryo that go on to form spinal cord, muscle and bone tissue and used them to produce nerve and muscle cells...
2 April 2012 - by Dr Linda Wijlaars 
A clinical trial to test the safety of a stem cell treatment for amyotrophic lateral sclerosis, the most common form of motor neurone disease, suggests that the new therapy is safe and well-tolerated. Encouragingly, one of the 12 patients participating in the study showed some improvement, although the trial was not designed to test the treatment's efficacy...
28 November 2011 - by Dr Marianne Kennedy 
On 18 November, Richard Grosjean became the first patient to receive a pioneering stem cell treatment in the upper part of the spinal cord. His procedure is part of an ongoing US-based clinical trial aimed at assessing the safety of injecting neural stem cells taken from eight-week-old fetuses into the spinal cords of patients with amyotrophic lateral sclerosis (ALS)...
28 September 2009 - by Dr Marianne Kennedy 
The US Food and Drug Administration (FDA) has granted approval to NeuralStem Inc., a Maryland-based biotherapeutics company, to conduct the first human trial using neural stem cells for treatment of amyotrophic lateral sclerosis (ALS). ALS is a type of motor neuron disease often referred to as 'Lou Gehrig's' and 'Maladie de Charcot'. The late-onset condition, of unknown cause, affects approximately two in 100,000 people, including the UK physicist Stephen Hawking and US rock gui...
21 September 2005 - by BioNews 
US scientists have used nerve stem cells to treat mice affected by severe spinal cord injuries. The team, based at the University of California at Irvine, said the treated animals regained the ability to walk just a few weeks after receiving the injections. The findings, published in the Proceedings of...
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.