Stem Cell Clinical Trials Get Going

A Brief Primer on Stem Cells

If you were to ask biologists what a stem cell is, chances are you will get several definitions with slight variations.  By and large, self-renewal (i.e. reproduce itself) and capacity of differentiation (i.e. produce different types of cells) are the two common denominators of a stem cell definition.  The National Institutes of Health hosts a stem cell resource website that contains easy-to-understand scientific information as well as political, ethical, and healthcare discussions about stem cell research and implications.  Despite the political headwind on Obama Administration’s stem cell policy, the burgeoning stem cell research activities, interests, and potentials cannot be ignored.

Five Stem Cell Clinical Trials Got Go-Ahead

The first FDA-approved stem cell trial using human embryonic stem cell (hESC) to treat spinal cord injury was given to Geron (Menlo Park, CA) in January 2009.  However, Geron’s preclinical animal study showed small cysts developed within the injection site, which prompted a hold in human clinical trial for further investigation.  Fast forward to July 2010, the FDA lifted the hold and gave the first stem cell clinical trial OK to Geron to treat spinal cord injury.  In October 2010, Geron began its first stem cell therapy in a human patient.

Advanced Cell Technology’s retinal pigment epithelium therapeutic cells received the FDA’s clearance in November 2010 and January 2011 for initiating Phase I/II clinical trials on two eye-related diseases, juvenile Stargardt’s Macular Dystrophy and dry age-related macular degeneration.  These two eye diseases affected ~30 million people in the US and Europe and may ultimately lead to blindness.

Aldagen, a biopharmaceutical company developing adult stem cell products on tissue repair and regeneration, received a green light from FDA in January 2011 to start Phase II clinical trials to test stem-cell therapy for stroke.  Finally, Pluristem therapeutics wins FDA-EMEA (European Medicines Agency) joined approval for stem cell trials in which the company will conduct a Phase II/III study of its product on blocked arteries, and a Phase II study on ischemia colitis (i.e. inflammation in the large intestine due to loss of blood flow).

Will Stem Cell Therapy Live Up to Expectation?

Gene therapy, once held a high hope in the 1990s, became a stymie in clinical implication after a patient died from multiple organ failures following a gene therapy trial in 1999.  One of the biggest challenges in gene therapy is unwanted immune responses elicited by gene therapy carries, mostly viruses.  As of this post is written, FDA has not yet approved any human gene therapy product for sale.  A 2006 Nobel prize winning discovery, RNA interference, has become a new therapeutic intervention candidate tested by many biotech/pharmaceutical companies.  Several RNAi-based clinical trials are in progress.  Recently, Roche announced to terminate RNAi research and development, sending uncertain signals to companies developing RNAi-based therapeutic agents.  The next wave of therapeutic intervention is no surprise landing on stem cell.  With private and public funding supports, adult human stem cells and induced pluripotent stem cells have been discovered and are under intense studies in the hope to replace human embryonic stem cells, mainly due to ethical issues.  Despite numerous challenges ahead to bring stem cell therapy into clinical implications, I remain optimistic to this new technology, but only time will tell.

Interesting Reports on Potential Application of Stem Cell

More on stem cell news and reports:

Until next post, keep on reading and writing!!


About fdabiomed
I like to read, organize, and write about biomedical related topics. I am passionate to learn new things, particularly news related to the Food and Drug Administration's decision and announcement, as well as the decision impacting on company's stock price.

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