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The Regeneration Station talks about... ES cell therapies begin clinical testing 08

NYSTEM Funding Committee meets today starting at 1:30 and the Ethics Committee meeting is scheduled for Feb. 22. See their agenda on the NYSTEM website, attend the public meetings or listen in via webcast, the discussions are serious and the subject complex and deep. Much is at stake in parallel to the rapid and important developments of what is happening in the reality in stem cell therapeutic research endeavors.
With the caveat that it is always best to get the full story and visit The Regeneration Station blog (1) and another favorite, the Stem Cell patent.com website (2) , here is some big news quoted directly from a commentary posted February 14, 2008 on the blog The Regeneration Station (1)

According to an article on CNN.com, Geron’s CEO, Dr. Tom Okarma, confirmed timelines for Geron to initiate human clinical trials with their embryonic stem (ES) cell-based treatment for spinal cord injury (SCI). He stated that the trial should start by the end of the second quarter (~June). This is really big news, as this will be the first-in-man studies using ES cells in a regulated clinical trial. Dr. Okarma made sure to mention the ‘high bar’ related to safety that the FDA has placed on this unprecedented therapeutic cell product. However, Geron has assured investors that they have worked very closely with the FDA, and Okarma’s public statements this week tells me that Geron believes that they have generated sufficient safety data to satisfy the FDA for initiating clinical testing.
You can get an idea of what this ‘high bar’ set by the FDA relates to by reading the recent press release from Advanced Cell Technologies, Inc. on the outcome to their pre-IND meeting with the FDA. That press release states that the FDA wants to see:

-studies performed under good laboratory practices (GLP)
-using cells manufactured under good manufacturing practices (GMP)
-examining several areas including general safety (check all organs and systems for health- typically in immunocompromised rodents for cell products)
-cell biodistribution (if you put the cells in the eye, where else do they go throughout the animal)
-tumorigenicity potential of the transplanted cells
-cell tolerability (how well does the host tissue tolerate the transplanted cells) and
-cell survival after implantation.

The press release by Advanced Cell Technologies, Inc. suggested that they had a lot more work to do before the FDA allows them to start first-in-man studies. I would imagine that Geron, in working with the FDA, has worked to set the bar extremely high for other ES cell-based products. Sometimes being first with a technology allows a company to set very high technical standards for followers to meet.

You may wonder why the safety bar is set so much higher for ES cell therapies than for many of the adult stem cell-based products that are working their way through the clinic. Bone marrow (the source for most adult stem cell-based therapies) has been used extensively in transplantation procedures for tens of years, and has a well documented safety profile with known risks. ES cell products, on the other hand, are very new technology and were initially characterized by scientists for their ability to form teratomas, or multi-tissue tumors, when transplanted into mice or rats. Teratoma formation raises all sorts of flags for the FDA, as it only takes one cell to make a tumor, so the FDA is going to want to see extensive safety profiling as it relates to biodistribution and tumorigenicity throughout the major tissues. If any of this data becomes public, or maybe it will be part of the discussion in the FDA guidance meeting in April, it will be interesting to see how long these safety studies will be required to go out. How long do you wait for a tumor to form in an animal model? For Advanced Cell, the answer to that question will likely be: “at least as long as Geron has taken their studies out.” Again, as the pioneers, Geron has the opportunity to establish industry standards.

So, 2008 is a turning point for ES cell-based therapies. This will be the year that ES cell therapies begin clinical testing in at least one product format, maybe two if Advanced Cell can execute quickly. The FDA is proactively addressing ES cell testing and is hosting an open discussion in April regarding safety issues related to these unique cell therapy products.

The Regeneration Station blog lists their favorite (18) related blogs on a sidebar--one very interesting website that has been around a while is the Stem Cell Patents.com. --even if you are not a scientist nor a patent lawyer, you can easily glean a lot of what is happening in the real world by reading their commentaries. There you find how the hype and hope of stem cell therapeutic research is becoming its own unique reality and get a feel for the tangible benefits and challenges of developing IP in the stem cell therapeutic research area.
Both blogs have regular news email alerts visitors can sign up for.

(1) http://regeneration-station.com/2008/02/14/the-developing-embyronic-stem-cell-clinical-landscape/
see NYSTEM at http://stemcell.ny.gov/

The Regeneration Station blog is about the technology behind Regenerative Medicine - including, but not limited to, stem cells therapies, biomaterial-based devices, and tissue engineered products. Jon Rowley, has a PhD in Biomedical Engineering from the University of Michigan.

I have been in the Regenerative Medicine field since 1994. I have a strong background in both early stage research and the development aspects of regenerative medicine. I am currently an employee of a publicly traded adult autologous cell therapy company.

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This page contains a single entry from the blog posted on February 15, 2008 8:25 AM.

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