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09/11/2014

Keeping up with Clinical Research with Human Embryonic Stem Cells

Unless one works daily in a specific area of biomedical research, it can be next to impossible to keep up with developments.  I have trouble keeping up with a reasonable amount of the progress in research for some cancers.  Trying to track stem cell research requires more bandwidth than I have.  Still, from time to time I do try to do at least a little catching up.  Among the fruitful resources are two I will mention here:

First, the website clinicaltrials.gov is a registry of biomedical trials.  By current law and regulation, almost all clinical trials of experimental drugs and devices involving investigators or sponsors (read: drug and device companies) in the developed world must be listed on this website.  It is something of a blunt instrument for searches, but still an important resource for anyone interested in checking what might be up in medical research.

If one enters “embryonic stem cell” on the website’s search page, one gets a list of 34 clinical studies.  The terminology involved is technical, but it is clear to me that this list includes studies with cells derived from human embryonic stem cells (hESCs), studies focusing on deriving hESCs from embryos, studies with induced (“reprogrammed”) pleuripotent stem cells (iPSCs), and studies with somatic, or “adult,” stem cells.  The search also identifies a couple of studies that appear to involve stem cells only indirectly, if at all.  And, from my experience, any one search on clinicaltrials.gov can fail to include “hits” of interest.  One sort of has to know what one is looking for, and try several strategies.  (Your correspondent doesn’t have time for an involved effort this evening.)

If one limits the search to studies that are currently open, the number of hits decreases to 23, 11 of which appear to involve hESCs or, more likely, cells derived from them.  (The technical details will vary, and may not always be public for confidentiality reasons that apply broadly to drug research, so it will not be readily apparent how many human embryos went into getting the cells actually used in the trial.)  Six of the eleven hESC trials involve the treatment of human retinal disease with cells that are the product of the Massachusetts-based company Advanced Cell Technology (ACT), which has other cell therapy programs being studied in the laboratory (but not yet the clinic).  Trials are in progress in the U.S. and the U.K., and it looks like the same cell product is being tested with a collaborator in Korea.  The five other open hESC trials are in Europe or Israel.

The second source is the informative blog California Stem Cell Report, which follows the joys and trials of the publicly-funded California Institute for Regenerative Medicine (CIRM).  Although the CIRM was initially conceived largely to support hESC research (and, as needed, so-called “research” or “therapeutic” cloning), it has supported a wide range of stem cell work, including with iPSCs and adult stem cells.  Recently, in response to criticism that hoped-for clinical treatments were slow in arriving, the CIRM committed $200 million of its funding to an “Accelerated Development Pathway” initiative intended to boost clinical trials and more rapidly turn research into treatments.  The first award from this—also recently reported in the general press—was to the San Diego-based firm ViaCyte for a trial of a “teabag”-like device that the company calls VC-01, that contains pancreatic cells derived from “an inexhaustible” hESC line; the device is inserted under the skin to sense blood sugar levels and secrete insulin to treat people with diabetes.  The California Stem Cell Report says this trial “is the first hESC trial backed by CIRM.  The trial is about to start, as the FDA recently “allowed” the Investigational New Drug application required by regulation before clinical trials of experimental drugs or devices may commence.  The recent award from CIRM, $16.6 million, brings ViaCyte’s total funding from CIRM to $55 million.   

My purpose here is simply to report a bit of what is in progress.  I reiterate my career interest in contributing to new, innovative treatments for human disease, and my settled conviction that it is unethical to create or destroy human embryos solely for research, or as “raw materials” for cell-based therapy.  I do not know, and cannot comment further on, how ViaCyte or ACT derived the cell lines they are using.

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