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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 2011 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
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