A stem cell treatment for ALS and other incurable neurodegenerative diseases gets early efficacy proofs and is entering Phase II clinical trials in the US. The treatment that has been developed by an Israeli clinic BrainStorm Cell Therapeutics is based on the patent’s own stem cells and is called NurOwn.
To develop the medication, the company used stem cells from the patient bone marrow enhanced to produce increased levels of nerve growth factors. These growth factors are responsible for protecting existing motor neurons and growing new ones, as well as for restoring of interaction between muscles and nerves.
The therapy showed promising preliminary effects in two initial trials that involved 24 patients and were conducted at Jerusalem’s Hadassah University Medical Center. For some parameters, 60 percent beneficial response after the treatment was observed.
In particular, four patients suffering from ALS in an advanced stage have been treated with NurOwn transplants as “compassionate use” cases starting in 2012. All the patients demonstrated improve in their muscle power and breathing for 3 to 6 months. Some of the patients demonstrated significant slowdown of the disease progression, while others even started walking and talking again.
In summer 2014, the company was notified of the approval of its US patent application. The double-blind, randomized trial that is being launched in the US will involve 48 patients, 36 of which will receive NurOwn treatment and the remaining 12 patients – placebo. The study results – in particular on safety and efficacy - will become available in early 2016.