BetaStem Therapeutics Inc
April 28, 2014 Issue
The Most Powerful Name In Corporate News and Information
Stem Cell Therapy for Diabetes and Cancer
BetaStem Therapeutics Inc
The complications of diabetes can be devastating. Diabetic Retinopathy (DR) is one of the leading causes of blindness in adults. 438 million people are projected to suffer from diabetes and its complications by 2030. The number of patients with diabetes is roughly doubling every 10 years. DR currently affects over 5.3 million Americans age 18 or older. A study in 2010 stated that more than 27 million Americans are affected by diabetes now, with 67 million estimated to have pre-diabetes.
BetaStemís technology is derived from discoveries that CD34+ stem cells become activated gaining highly advantageous regenerative properties when treated with a proprietary antisense to block a major activation inhibitor, namely transforming growth factor-beta, (TGF-β1). The activated stem cells home into the damaged vascular regions of the retina, initiating a process of blood vessel regeneration and thereby preserving eye health.
the first real solution for DR by preventing and repairing micro-capillary
Sausalito, CA 94965
Interview conducted by: Lynn Fosse, Senior Editor, CEOCFO Magazine
Dr. Bartelmez: The concept is trying to address a problem in diabetes. It is a problem called diabetic retinopathy in which the blood vessels of the patientís cells are damaged severely over time with diabetes and there is no treatment for it. The only treatment that is going to work is some sort of regenerative treatment. We have been working on a stem cell treatment based on stem cells from the patients to repair the retinal vessels. We are trying to enter our first clinical trial after seven years of preclinical work.
CEOCFO: Has your approach been tried in the past?
Dr. Bartelmez: No, we discovered this approach.
CEOCFO: What led you to believe this was the best way?
Dr. Bartelmez: In 1997 in my lab at the University of Washington, we discovered something that was extremely striking and that was that you could not just take bone marrow stem cells and transplant an experimental animal with them without somehow activating the cells because the stem cells took weeks in order to become activated in the animal. Our approach was to modulate gene expression in the stem cell. The modulation only occurs over about two or three days, but it is enough to activate the stem cells. We first used this approach experimentally for bone marrow and umbilical cord stem cells transplantation. We next tried our approach to induce blood vessel formation from the stem cells. In fact, the first experiments we did showed strongly that the same mechanism works to repair damaged vessels in the eye.
CEOCFO: Has the medical community paid attention yet or is it too early?
Dr. Bartelmez: It is not too early anymore. We have generated about $2.5 million in government small business grants. Reviewers have embraced this particular approach and wanted to know if it could become a clinical reality. We have published on this. Until we get through an initial clinical trial, investors will be skeptical because we do not actually have the therapy established in humans. The only way we are going to show treatment safety and efficacy is to do a clinical trial. We have previously completed extensive human stem cell-into-mouse animal models. Clinical Trials are designated Phase I, II and III and by Phase III you are showing safety and efficacy at that point everyone jumps on the investment bandwagon. If our therapy is proven to work in patients we will get funding.
CEOCFRO: What gives you the confidence, personally, that you are on the right track?
Dr. Bartelmez: Because of the results that we have gotten to this point. Our data from our animal models shows that our modified stem cells are safe and effective. We can show investors efficacy of the procedure. We are using human stem cells transplanted into mouse eyes and following retinal microvascular. Importantly, UNTREATED stem cells essentially do not repair vascular. The stem cells need to be activated.
CEOCFO: What will you be providing and selling when all is through?
Dr. Bartelmez: We are going to be selling a patented method to treat diabetic retinopathy that works like this: The patient will go into a blood bank and have blood drawn, the stem cells will be isolated, they will be treated with a morpholino antisense (which we have patent rights to) and then the cells will be injected back into the patientís eye. Without morpholino antisense the procedure will not work. Most stem cell therapies that are in place today do not modulate the cells at all or they will not work in the eye.
CEOCFO: What is morpholino antisense and what does it do that allows the stem cells to work?
Dr. Bartelmez: It is a very small molecule that is chemically synthesized that can gain access into the stem cells and binds to the messenger RNA after which a the messenger RNA can no longer be translated into the protein. Thus we transiently block the production of transforming growth factor-beta type 1 (TGF-B1) in the stem cell for 4-5 days using antisense phosphorodiamidate morpholino oligomers (PMO) to TGF-B1 in diabetic stem cells which potentiates their ability to repair retinal vessels in diabetic. TGFbeta1 is in that family of similar factors and is heavily over expressed in diabetic stem cells, so we reduce the amount of TGF-beta1 in the stem cells before we inject it in the eye and this is highly effective.
CEOCFO: Is there much training involved for the physicians?
Dr. Bartelmez: This is going to have to be done by a surgical ophthalmologist because the best administration route will be directly into the eye. Currently, there are tens of thousands of patients going through intravitreal injection of anti-VEGF antibodies in saline solution for advanced stages of retinopathy. Instead of putting the drug into the syringe, we are putting stem cells into the syringe. As far as the physician is concerned, they cannot see any difference and it does not make any difference. In one case, they are injecting drugs and in the other case, they are injecting stem cells.
CEOCFO: What is the timetable?
Dr. Bartelmez: We are trying to generate the money for a Phase I trial which is basically going to be a safety trial but we will also get some determination of efficacy from of this trial. We have had meetings with the FDA and they want to show that the safety of this procedure is second to none and the only way to prove it is to inject the cells into patients eyes that are sick with retinopathy, so a compassionate based trial.
CEOCFO: Are you looking at potential partnerships or would you prefer funding that keeps you in control?
Dr. Bartelmez: I think partnerships are probably the strongest way to go. We have looked at potential partnerships.
CEOCFO: Different areas of healthcare and research tend to fall in and out of favor with the investment community. Are you working in an area that has interest these days?
Dr. Bartelmez: Well letís just say it is an area that is gaining interest. The stem cell therapy field is just starting to get some traction. One must not forget that bone marrow stem cell therapy was established in the 1960ís and has saved 100,000s of patients with leukemia. There have been quite a few clinical trials done with different stem cell types. The interest is gaining but big pharma has control of these treatment areas (using small molecules) for years. We love big pharma but small molecules are only good for certain indications; you cannot regenerate tissue with small molecules, it requires cells. This is just going to be a matter of time before big pharma turns around and starts partnering with stem cell companies like us.
CEOCFO: Are you on a holding pattern?
Dr. Bartelmez: We are continuing to do research. We are in the middle of a raise of $2 million right now that is going through Optimum Investments in North Carolina.
CEOCFO: How do you deal with the frustration when you have something that could potentially make such a difference and at yet it is such an arduous process to get it in place?
Dr. Bartelmez: Everything is based on shear persistence and money. I learned that initially many years ago in academic science where I had to generate all my own money from grants, otherwise I could not do any research. The universities were somewhat supportive, but basically raising money for research was up to the principal investigator (me). Now in business it is the same thing. I founded BetaStem seven years ago. Money of course has been a major problem going forward and most of our funding has come from NIH grants. However, I am also a major investor in BetaStem which has proved necessary to keep the development going ahead during low funding periods.
CEOCFO: Why should BetaStem standout?
We have the ability to help a huge population of diabetics where there has
been no treatment at all and basically many patients are going blind. The
BetaStem therapy is predicted to block retinal cell death preventing the
blindness and increasing visual acuity for a very large number of patients.
As far as the investor is concerned, the potential of generating money from
the BetaStem therapy is substantial. Furthermore, we have other therapies in
the pipeline to address peripheral vascular disease and heart disease. Once
the procedure is available to patients (i.e., after clinical trials) the
therapy will generate over $100 million a year. Now that is a good
investment and as the investors say, it is only a good investment if it is
safe and works and we agree 100%, so we are trying to get in to show that it
works on the patients in need.
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