Stem cell therapy for ALS fast-tracked by FDA

A stem cell therapy for amyotophic lateral sclerosis (ALS) could have a shorter route to market after being fast-tracked by the FDA.

Israeli firm BrainStorm Cell Therapeutics has been granted the status for its NurOwn therapy, which involves harvesting stem cells from patients’ bone marrow, modifying them to secrete substances that stimulate neuron growth and transplanting them back into the spinal cord or muscles.

The news is one of the first developments in the field since ALS was catapulted into the spotlight by the ‘ALS Ice Bucket Challenge’. The craze swept through the internet and social media in August and helped raise millions of dollars around the world for research into the condition, known as motor neurone disease (MND) in the UK.

Fast-track status means that the company will have a higher level of interaction with the FDA as it ushers NurOwn through development for the disease, and could also make it eligible for a shorter review time or a rolling marketing application.

NurOwn is one of only a few cell-based therapies to be fast-tracked by the FDA and is currently in a Phase IIa trial at the Hadassah Medical Center in Jerusalem – which is already fully enrolled with 32 patients – as well as a Phase II study being carried out in the US at the Mayo Clinic Massachusetts General Hospital (MGH) and the University of Massachusetts (UMass) Memorial Hospital that is still recruiting with a target population of 48 patients.

The Israeli trial should generate data later this year while the US results are expected in 2016, according to the company, which is in a race to market with rival NeuralStem for an MND stem cell therapy, with the latter’s NSI-566 also in Phase II.

“We expect fast-track designation, which recognises the potential of NurOwn … to address an unmet medical need in ALS, to help speed and improve our development programme,” said BrainStorm’s chief executive Tony Fiorino, who took over the helm of the company in June.

Shortly after taking over, Fiorino said that MND/ALS represents a multi-billion dollar market opportunity, even with “unheroic assumptions for annual cost”, pointing out that the only approved therapy for the disorder – Rilutek (riluzole) – has only a modest survival benefit and provides no benefit in terms of muscle strength or neurological function.

In addition to MND/ALS, the company is also developing its stem cell therapy for Parkinson’s disease and multiple sclerosis.

The announcement comes as stem cell therapies appear to be gathering momentum after years of promise that has not translated into clinical success. For example, in August StemCells reported a major milestone as it advanced its therapies for blindness and spinal cord injury into proof-of-principle trials.

Problems with access to funding also seem to be receding with StemCells raising $20m in July and BrainStorm now sitting on a $10.5m balance sheet after a financing round in June while NeuralStem raised $20m in January.

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