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FDA approves trial of neural stem cells to treat Lou Gehrig’s disease

28 September 2009
Appeared in BioNews 527

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 guitarist Jason Becker.

The planned trial will involve 12 patients who will undergo several stem cell injections in the lumbar region of the spinal cord. The patients will be examined regularly post-surgery and a final review of the results is expected in two years. Currently, the research team is awaiting permission from the patient safety board at Emory University in Atlanta, Georgia, where the trial is proposed to take place.

Dr Eva Feldman, who will direct the clinical trial, explains: 'In work with animals, these spinal cord stem cells both protected at-risk motor neurons and made connections to the neurons controlling muscles. We don't want to raise expectations unduly, but we believe these stem cells could produce similar results in patients with ALS'.

Mr Richard Garr, NeuralStem Chief Executive and President, stated: 'The beginning of our clinical trial program is a major step towards achieving Neuralstem's goal of treating ALS, a fatal neurodegenerative disease for which currently there is no effective treatment or cure. While this trial aims to primarily establish safety and feasibility data in treating ALS patients, we also hope to be able to measure a slowing down of the ALS degenerative process'.

The approval comes eight months after the FDA placed the trial on hold following reports of tumour development in a boy who underwent unregulated stem cell therapy in Moscow in 2001. A month ago, the FDA also halted plans of another stem cell trial for spinal cord injury, by the Californian-based company, Geron, over potential safety concerns following animal studies. However, the animals in questions were found to have benign cysts, rather than tumours caused by the treatment, and Geron are hoping to restart the trial soon.

The new therapy developed by Geron uses cells derived from human embryonic stem cells, while the NeuralStem treatment uses neural stem cells taken from a single eight-week old aborted fetus and grown in the laboratory.
FDA green lights stem-cell clinical trial for Lou Gehrig’s disease
The Great Beyond (Nature blog) |  22 September 2009
Neuralstem gets FDA OK to test stem cell drug in humans
Reuters |  21 September 2009
University of Michigan researcher will lead first human trial of using stem cells to treat ALS
MedCity News |  22 September 2009
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...
18 February 2013 - by Reuben Harwood 
What would be the first clinical trial to use induced pluripotent stem cells has been granted ethical approval in Japan...
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...
15 October 2012 - by Joseph Jebelli 
Scientists have successfully implanted human neural stem cells into the brains of children with a rare neurological disorder...
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 September 2009 - by Nishat Hyder 
Information has come to light regarding the US Food and Drug Adminstration (FDA)'s freeze on the clinical trails of GRNOPC1, a groundbreaking therapy for spinal cord injury derived from human embryonic stem cells (ES cells) being undertaken by biotech company Geron...
23 August 2009 - by Alison Cranage 
The US Food and Drug Administration (FDA) has delayed the start of a clinical trial that plans to use human embryonic stem cell(ES) cells to treat spinal cord injury. The trial is being run by Californian based company Geron. The FDA originally gave the go-ahead for the trial in January, but now has halted the start in order to review new data submitted by Geron....
26 January 2009 - by Antony Starza-Allen 
The US Food and Drug Administration (FDA) has allowed the biotech firm, Geron, to begin clinical trials using embryonic stem (ES) cells to help treat spinal cord injury sufferers who have become paralysed from the chest down. The product known as GRNOPC1 will be tested for its...
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...
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