BrainStorm Cell Therapeutics – an Israeli biomed company currently conducting Phase I/II clinical trials on patients suffering from ALS, reported that at least some of the patients treated with its NurOwn cell therapy shown considerable improvement including walking and talking after being unable to do so because of the progress of the disease.
Amyotrophic lateral sclerosis (or ALS aka Lou Gehrig’s disease after the New York Yankees baseball player who was diagnosed with the disease in 1939) is severe disorder which causes muscle weakness and atrophy throughout the body caused by degeneration of the upper and lower motor neurons. Most patients who have been diagnosed with ALS die from respiratory failure, typically within 3-5 years from the onset of symptoms. The median survival time from the most early signs of ALS to death is around 39 months, and only 4% survive longer than 10 years (maybe the most famous, is the well known physicist Stephen Hawking, who lived with the disease for more than 50 years although he is considered to be an extremely rare case). –
Currently there are about 25,000 ALS patients living in the U.S. and scientists do not know the cause of disease (in most cases) and so far no cure has been developed although a drug known as Riluzole does slow down the progression of ALS but only by a few months. –
The technology developed by BrainStorm known as NurOwn is based a novel differentiation protocol which reprograms the bone marrow derived mesenchymal stem cells (MSC) into neuron-supporting cells. These specialized cells, “MSC-NTF” are capable of releasing neurotrophic factors such as Glial-derived neurotrophic factor (GDNF) and Brain-derived neurotrophic factor (BDNF). –
In more simple terms, NurOwn uses stem cells from the patient himself (taken from the bone marrow) to create. Using a new technique developed by Professor Eldad Melamed, former Head of Neurology at the Rabin Medical Center in Tel Aviv and Professor Daniel Offen, Head of the Neuroscience Laboratory, Felsenstein Medical Research Center (FMRC) of Tel Aviv University. –
The technique is used to grow the stem cells taken from the patient and turn them into factories producing GDNF and BDNF which have been shown in research to prevent cell death (apoptosis) which supposedly takes place in ALS patients. The cells will be injected into the patient (into the legs or arms in early stages or directly into the spinal cord in patients with more advanced stage ALS). Since the source of the cells is from the patient’s body – there is relatively low risk of immune cell response. –
So far there has only been a Phase I/II clinical trial with NurOwn which took place in Hadassah Medical Center in Jerusalem with about 24 patients. This early test was done in order to test the safety of the treatment however even in this preliminary stage the results have been very encouraging. Some of the patients showed definite signs of disease regression and one patient who agreed to reveal himself on Israeli Chanel 2 TV regained his ability to walk and talk after he has been restricted to a wheel chair and had difficulty talking. –
According to Professor Dimitrios Karussis of the Neurology Department at The Hadassah Medical Center in Jerusalem, and Principal Investigator of BrainStorm’s current Phase I/II clinical trial: "Within a few weeks following injection with NurOwn cells, the patient showed dramatic improvement in a variety of functions including breathing, speech, walking, muscular strength, and overall well-being. While we cannot draw scientific conclusions based on the outcome of an individual patient, these results are extremely encouraging". –
Despite these encuraging results finishing the development of NurOwn will likely take at least 5 more years with more widespread trails currently scedualed to begin in two major clinics in Boston. The technology developed by BrainStorm might also prove useful for other disease such as Parkinson’s disease which effect over a million patients in the U.S as well as Multiple Sclerosis (MS). –
More information can be found on BrainStorm’s website.
Iddo has a B.A. in Philosophy and Cognitive Science and an M.A. in Philosophy of Science from the Hebrew University of Jerusalem. He is currently writing his Ph.D. thesis on the relationship between the scientific community and industry. Iddo was awarded the 2006 Bar Hillel philosophy of science prize for his work on the relationship between science and technology. He is a member of the board of the lifeboat foundation and was the editor of several high-profile science and technology websites since 1999.