Motor neurone disease stem cell trial advances to next phase

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.