Every day is a day closer to a disease being treated, or even cured, by a stem cell therapy. The medical field is in a transition with clinical trials that are ongoing using adult, embryonic, and other forms of stem cells derived and differentiated using various laboratory techniques. These two companies have the potential to be the first self-sustaining, profitable regenerative medical companies.
The first company is Advanced Cell Technology. In Advanced Cell’s pipeline is the hermagioblast program, which is the creation of blood cells using human embryonic stem cells derived from the company’s embryo friendly single-blastomere technique (The company extracts a single cell from an embryo and can create stem cell lines from that single cell, meanwhile the embryo itself is unharmed). ACTC maintains the rights for North America and their partner CHA Biotech, a Korean based company, has the rights for South Korea and Japan. An IND filing is expected in either the fourth quarter of this year or first quarter of 2012.
The myoblast program, heart cells using adult stem cells, is approved for phase two clinical trials in the U.S. but it is the third program that holds the key to near term promise of profitability. The program is the retinal pigment epithelium (RPE) program, which are retinal cells derived from human embryonic stem cells using the single-blastomere technique. ACTC started two trials using these cells last month for Dry AMD and Stargardt’s macular dystrophy. CHA Biotech President and Chairman, Dr. Kwang Yul Cha, actually expects phase three approval soon in Korea as the Korean government has less regulations and a more positive attitude to stem cell therapies than the U.S. If his timeline is accurate and shows safety and efficacy in phase three in Korea then a possible fast track could follow in the U.S. The aggressive timeline is due to the fact that the eye is an immune-privileged site of the body and doctors can see cells and disease progression of the eye in real-time.
In a recent quarterly conference call ACTC CEO and Chairman, Gary Rabin, expects to expand the trials in the U.S. soon by injecting more patients, currently one patient in each trial has been injected at UCLA, and getting more sites online. While ACTC is in a quiet period we can assume safety is not a concern right now, otherwise the company would not be looking to expand trials. An official update of the trials is scheduled in October.
The second company is International Stem Cell Corporation. ISCO uses a technique called parthenogenesis, which creates stem cells from unfertilized oocytes. What is interesting and intriguing about ISCO is the strategy they are using. Instead of going after a disease and clinical trials procedure first ISCO instead is focused on cosmetics, namely a day and night skin cream combo where stem cells are an active ingredient to rejuvenate skin cells. FDA approval is not required for this product and is already on the market in a limited basis.
On the company’s last quarterly conference call they expected to sell a thousand bottles in a limited launch. Instead the company sold seven thousand bottles. That is the good news. The bad news is that ISCO cannot sell the product again until sometime in the third quarter as they underestimated demand and the cells needed for the product take two to three months to grow. Now that ISCO knows more about the demand to expect for the product there should hopefully be no more gaps in product production and could turn profitable within the next three to five quarters in my opinion. With a profitable cosmetic line the company then plans to fund therapeutic research and future clinical trials for liver diseases, Parkinson’s and eye diseases.
Disclosure: I am long ACTC.OB, OTCQB:ISCO.