Will stem cell regenerative medicine pivot in 2016 from its dosing elephant to embracing tissue stem cell counting?

Written by Asymmetrex LLC

Regenerative medicine with transplanted tissue stem cells is stuck in conventional denial about the importance of knowing the actual dose of tissue stem cells in treatment trials. Embracing a new technology from Asymmetrex for counting tissue stem cells in 2016 could change the future of stem cell medicine.

How remarkable, the current state of affairs in regenerative medicine! When a pharmaceutical company or physicians’ clinical trial group files an investigational new drug (IND) application with a regulatory agency (e.g., like the Food and Drug Administration [FDA] in the U.S.) for allowance to conduct a clinical trial to evaluate human effects of a new drug candidate, the dose — essentially the number of drug molecules to be given to trial subjects or trial patients — is an obligatory element for evaluation.

If the application is for a first clinical trial, it provides results of dosing studies performed in animals to project maximum drug doses allowed to limit toxicity and to estimate minimal drug doses required to achieve an efficacious effect. For secondary clinical trials, applications will prescribe doses of the drug based on experience from earlier human trials. Other important technical assessments are evidence that the drug can be supplied in sufficient quality and quantity and can be reliably administered with accuracy and precision from dose to dose.

Now, if regenerative medicine clinical studies had to meet these routine standards for pharmaceutical drugs, none of their applications would be allowed. Since the essential treatment principle in regenerative medicine stem cell transplantation trials is not drug molecules, but tissue stem cells, one would expect that knowing the dose — i.e., the number — of transplanted stem cells would be required for allowance. Yet, there are hundreds, approaching thousands, of allowed clinical trials involving unknown numbers of transplanted tissue stem cells under way right now. It follows that there are no projections of unsafe or efficacious doses; there is no verification of supply quality and quantity; and dose-to-dose reliability goes unmeasured. With such an obtuse absence of standards, how will such clinical trials ever deliver meaningful results?

So, regenerative medicine finds itself underneath a sizable elephant, which it mostly ignores, though often painfully, which is the way with intractable elephants. Many investigators have miscalled ambiguous biomarkers for tissue stem cells ‘stem cell markers’, for so long that now they accept them even though the vast majority of the cells they detect are not tissue stem cells at all. This resignation is somewhat understandable though, given that the same error is rampant in the peer-reviewed literature. Even more uniform preparations of tissue stem cells, like mesenchymal stem cells, are phenotypically heterogeneous, with a high fraction of cells that do not have the long-term tissue renewing capabilities provided by tissue stem cells.

At the close of 2015, Asymmetrex projected the following 5th advance in regenerative medicine in 2016, if its new first-in-class AlphaSTEM technology — for counting tissue stem cells from any self-renewing human tissue — were adopted by regenerative medicine companies and physicians’ clinical trial groups:

“5. Stem cell therapy clinical trials would no longer be performed without knowing whether different tissue cell preparations differ in stem cell number, the essential factor for the interpretation and success of the trials.” (http://asymmetrex.com/9-Improvements-We-Could-See-in-2016-Thanks-to-Biotechnology)

In science, the best way to decide whether to make a change in method is by experimentation. So, as in the previous RegMedNet post (https://www.regmednet.com/users/5937-asymmetrex-llc/posts/4598-introducing-hsc-dosing-to-hematopoietic-stem-cell-transplantation-medicine) focused for hematopoietic stem cell transplant centers and umbilical cord blood banks, Asymmetrex invites regenerative medicine companies and physicians’ clinical trial groups to learn more about its AlphaSTEM stem cell counting technology and evaluate it for use in their stem cell transplantation trials. As with drug molecules, knowing the actual dose of treating tissue stem cells can only improve the quality of the trials and their benefit to present and future patients. To learn more about AlphaSTEM technology, please, visit http://asymmetrex.com/our-products/drug-development-regenerative-medicine.

Interested in learning more? Check out the free webinar on counting adult tissue stem cells for applications in regenerative medicine and drug development.