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Step forward in pioneering stem cell trial for motor neurone disease

28 November 2011
Appeared in BioNews 635

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 to assess the safety of injecting neural stem cells taken from eight-week-old fetuses into the spinal cords of patients with amyotrophic lateral sclerosis (ALS).

ALS is a type of motor neurone disease also referred to as Lou Gehrig's disease and Maladie de Charcot. It is a late-onset condition affecting roughly one in 50,000 people. Cambridge physics professor Stephen Hawking is perhaps the best-known ALS patient in the UK.

Mr Grosjean's operation took four hours and included five injections to the cervical region of the spine, which runs from the head to the shoulders. Each injection consists of more than 100,000 stem cells. The treatment was developed by NeuralStem, a US biotech company, and uses neural stem cells - which give rise to brain and nerve cells - extracted from the spinal cord of an aborted eight-week-old fetus.

Dr Eva Feldman, director of research of the ALS Clinic at the University of Michigan Health System, who designed the trial, explained: 'The ultimate goal of transplanting cells into this region is to preserve or even enhance breathing capacity for the patients. This treatment is essential to improve the quality of ALS patient lives and potentially lengthen them'.

The current study started in January 2010 and is a phase I trial to assess the safety of the transplantation method. There is no evidence as yet as to whether the treatment is effective. The first 12 patients have already received stem cell transplants in the lumbar (lower back) region of the spine and the trial has now progressed to the final six patients, all of whom will be treated in the cervical region.

The California-based company Geron was also attempting a stem cell-based clinical trial for ALS but announced last week that it was stopping this, apparently due to financial reasons. Geron was to have trialled the transplantation of brain cell precursor cells in ALS patients, with the brain cell precursors having been developed from embryonic stem cells taken from human embryos at four or five days old.

Such research is controversial because it relies on material from human embryos - or in the NeuralStem trial, fetuses - which are destroyed after use. Last month the European Court of Justice decided to ban the patenting of any stem cell process that involves destroying a human embryo.

Talking about the progress of the NeuralStem trial, study leader Dr Jonathan Glass, a neurologist at Emory University in Atlanta, said that things were 'moving forward', but added: 'We don't have a treatment yet, we don't have a cure yet and there's no evidence yet even putting these stem cells on the spinal cord is going to either slow the disease or prevent progression or even make it better'.


Note: This article was corrected on 29 November 22053 from an earlier version which mistakenly labelled both the neural stem cells used by NeuralStem and the embryonic stem cell-derived oligodendrocyte precursors used by Geron as 'embryonic stem cells'. Our thanks to Dr Dusko Ilic, senior lecturer in stem cell science at King's College London, for alerting us to the error.

After Geron, Stem Cells’ New Saviors
The Daily Beast |  18 November 2011
Emory University Spine Surgeons Inject First Stem Cells Into Cervical Spine for ALS Treatment
Becker's Orthopedic, Pain and Spine Management |  22 November 2011
First Patient Dosed in Cervical Region in Neuralstem ALS Stem Cell Trial
Neuralstem Inc. (Press Release) |  23 November 2011
Operation marks another step forward in stem cell research
CNN |  21 November 2011
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...
22 April 2013 - by Matthew Young 
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...
7 January 2013 - by Dr Tamara Hirsch 
Neural stem cells were shown to be effective against amyotrophic lateral sclerosis (ALS), or motor neurone disease, in 11 independent studies on mice with the disease...
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...
6 February 2012 - by Cathy Holding 
Mouse skin cells have been converted directly into neural precursor cells which go on to form the major cells in the brain...
21 November 2011 - by Rosemary Paxman 
A US drug company is abandoning its research into a novel paralysis treatment using embryonic stem cells, due to current economic conditions....
24 October 2011 - by Antony Blackburn-Starza 
The European Court of Justice (ECJ) has ruled that stem cell processes which require the prior destruction of human embryos or are based upon the use of human embryos are not patentable. The decision may have wide implications for scientists engaged in embryonic stem cell research....
1 June 2010 - by Victoria Kay 
A new research project, which will use human stem cells to artificially create the diseased brain cells affected in Motor Neurone Disease (MND) could pave the way for a cure for sufferers....
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...
15 May 2009 - by Heidi Colleran 
By Heidi Colleran: An international team lead by UK researchers at the Centre for Neurodegenerative Research (CNR) at Kings College, London, have discovered a genetic variation that prolongs survival in people affected with motor neuron disease (MND). In a study of over 5,000 people in six countries - almost half of...
16 October 2006 - by Dr Jess Buxton 
US researchers have used human stem cells to lessen the symptoms of motor neurone disease in rats bred to have symptoms of the condition. The scientists, based at Johns Hopkins University, Baltimore, showed that injecting fetal nerve stem cells into the spines of rats with amytrophic...
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