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.