Can GSK’s epilepsy drug help ALS patients?

The search for a new treatment for the progressive neurodegenerative disease ALS has turned to an existing epilepsy drug marketed by GlaxoSmithKline (GSK).

New treatments for the disease are desperately needed, as the only drug approved for treating ALS (known as Motor Neurone Disease in the UK) is Sanofi’s Rilutek (riluzone), but it cannot cure or prolong the life of people with the condition.

Awareness of the disease and funding for research received a massive boost last August when the internet craze the Ice Bucket Challenge helped raise more than $100 million for the ALS Association, and numerous new and existing molecules are being considered as possible treatments.

The charity is teaming up with GSK, the Harvard Stem Cell Institute (HSCI) and Massachusetts General Hospital Neurological Clinical Research Institute (MGH NCRI) to investigate the pharma company’s drug retigabine.

Developed by Valeant and GSK, the drug was launched in the US in 2010 as Potiga and then in Europe as Trobalt. It has a unique mechanism of action, working to calm the ‘hyperexcitability’ of neurons thought to cause epileptic seizures.

These same hyperexcitable neurons are also thought to play a role in ALS, and a new trial of the drug will be performed across 12 academic centres in the US.

The research partners say a large body of data supports the hypothesis that neurons become hyperexcitable in ALS, firing more than they normally do. This may, in turn, contribute to death of nerve cells, considered a root cause of the disease. In cell models of ALS, retigabine has shown the ability to reduce excitability and prolong survival in the lab.

The 12 academic centres are part of the Northeast ALS Consortium, an international, non-profit group of researchers who collaborate on clinical research in ALS and other motor neuron diseases. GSK will provide the drug, and funding support will come from HSCI, The ALS Association, the MGH NCRI and GSK.

Lucie Bruijn, chief scientist for The ALS Association hailed the new research partnership, and said it would also use a cutting-edge technique to speed up drug development.

Alongside the clinical trials, researchers will also for the first time create stem cells from these patients to see if they can be used to determine in advance which patients could benefit from the medicine.

Among the other drug candidates being tested against the disease are Novartis’ multiple sclerosis drug Gilenya (fingolimod), Teva’s Azilect (rasagiline), VM BioPharma’s DNA-based VM202, NeuroNova’s sNN0029 and NeuralStem’s cell therapy NSI-566.

The brightest hopes rest with two companies, Brainstorm Cell Therapeutics and Neuralstem, which are developing stem cell harvesting techniques to treat ALS and a range of central nervous system conditions.

Both companies are now testing their therapies in phase II studies, with Brainstorm’s NurOwn therapy having been fast-tracked by the US FDA in October.

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