If you have a social media account, chances are you aware of the Ice Bucket Challenge, an activity that went viral in July-August of this year to promote awareness and raise funds for ALS, Amytrophic Lateral Scleroris, otherwise known as Motor Neurone Disease. A global phenomenon, people are nominating their friends and family (celebrities included) to drench themselves in a bucket of ice water for the sake of this neurodegenerative disease that many are hearing of for the first time.
While this challenge has certainly raised global awareness of the disease, it can only do so much – in fact, the campaign has drawn criticism for its self-congratulatory nature and focus primarily on fun rather than donating to charity. Other developments in the ALS sphere, however, are remaining focused on the primary goal: that of treating the disease. And Israeli biomedical company, BrainStorm Cell Therapeutics, is one of the players driving these developments.
BrainStorm is laying the foundations for the most comprehensive treatment seen so far for this disease. Their product, NurOwn, uses adult stem cells to help ease symptoms and halt the progression of neuromuscular diseases such as ALS. This platform was used to successfully treat four advance-stage ALS patients at Hadassah Medical Centre in Israel, and is at present going into Phase 2 clinical trials in three major US medical centres. 48 patients will participate in the double-blind, randomised trial across Massachusetts General Hospital, the University of Massachusetts Memorial Hospital and the Mayo Clinic.
The treatment works by adding chemicals to stem cells from the ALS patient themselves until they are able to autonomously grow new neurons, re-injecting the cells once this process is complete. The technique was developed by Profs. Daniel Offen and Eldad Melamed at Tel Aviv University, who explain that “These nerve cells secrete a growth hormone that supports existing nerve cells, slowing down, and even halting degeneration.”
Though there are many companies in the stem-cell space, CEO Tony Fiorino explains that what differentiates BrainStorm is that “we’re in the clinical space. Secondly, many others have a ‘shotgun approach’ in which they use early stem cells with the potential to turn into differentiated cells. We have taken our cells and converted them into factories that pump out growth factors. We know exactly where we want to use them; there are no mysteries.”
The patients treated at Hadassah Medical Centre showed dramatic improvements in their speech, walking, muscular strength and overall wellbeing within a few weeks of the injection of NurOwn cells. However, improvement fades within three to six months, underscoring the importance behind the new clinical trials in the US. They focus around improving protocol in order to do multiple injections for the longer term and discovering how to tailor administration to individual patients.
Last June saw BrainStorm raise $10.5 million in private investments, with it being notified in July of the allowance of its US patent application.
For more information, visit BrainStorm’s website.
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