Stem cell therapies, despite their moral ambiguity, are gaining ground in the medical world. It is expected that the market for such therapies will be well over $300 million by 2020.
The field is still in the nascent stage and therefore, the Pharmaceuticals companies engaging in such research are more prone to market and regulatory risks. However, the payoff in the case of success is also expected to be equally steep.
One such potential winner in the field is Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI). The company touched an important milestone as it announced early this morning that it has validated its cryopreservation process for NurOwn® in preparation for an upcoming Phase 3 clinical study in Amyotrophic Lateral Sclerosis (ALS).
The validation study will involve comparing NurOwn® (MSC-NTF cells) derived from fresh mesenchymal stem cells (MSC) to those derived from cryopreserved MSC. The process of using cryopreserved MSC will let the company offer repeated doses of autologous NurOwn® from a single bone marrow aspirate in its upcoming multidose clinical trial.
The process is also more soothing for the patients as they would not be required to undergo multiple bone marrow aspirations.
Developing ALS treatment is one of the most challenging fields in the pharmaceuticals sector today. Globally, nearly 400,000 people are diagnosed with this ailment every year. Currently, there is only one drug, Riluzole, which is specifically prescribed for the disease. Use of stem cells is a rather novel idea being promoted by the company.
Brainstorm Cell Therapeutics has already administered NurOwn® to over 30 patients with ALS in clinical trials conducted in Israel. The company is likely to have strong prospects ahead if its trials in the United States go well. The treatment has shown good results at first and second stage trials.
Shares of BCLI are trading steadily this year as its year to date gains stand at over 11%. Shares have risen nearly 15% in the past 12 months. Brainstorm Cell Therapeutics is also on solid financial ground. It ended its September quarter with nearly $11.2 million in cash, cash equivalents, and short-term deposits.
One encouraging figure for the third quarter was the net loss of $1.6 million, down from $2.6 million net loss it had incurred for the corresponding quarter in the previous year. However, investors need to be aware of the fact that the company is focusing on a very specific and difficult segment, which reduces the chances of diversification and thereby increases the investment risk factor.
However, Brainstorm Cell Therapeutics has shown encouraging results for its clinical trials. NurOwn has also shown significantly good outcomes for its single dose and further optimistic news from stage III trials should provide additional support to the Company’s share price.
