Feds OK 2nd human study of embryonic stem cells

November 22, 2010 By MALCOLM RITTER , AP Science Writer

(AP) -- For only the second time, the U.S. government has approved a test in people of a treatment using embryonic stem cells - this time for a rare disease that causes serious vision loss.

Advanced Cell Technology, a biotechnology company based in Santa Monica., Calif., said the research should begin early next year, following the green light from the U.S. .

Just last month another biotech company, Geron Corp., said it had begun preliminary testing in people for treating by injecting cells derived from .

Scientists hope to use to create a variety of tissues for transplant. But have to be destroyed to harvest those cells, which has made their use controversial.

ACT's experiment will focus on Stargardt disease, which affects only about 30,000 Americans. But the company hopes the same approach will work for similar and more common eye disorders like age-related macular degeneration, which affects millions.

Stargardt is an inherited disorder that attacks central vision used for tasks like reading and recognizing faces. Some patients go totally blind, even losing peripheral vision, while others are severely impaired and can only perceive light or see their hands moving in front of their faces.

The disease typically starts in adolescence. The key problem is that impaired scavenger cells fail to remove toxic byproducts from the eye, allowing them to build up and kill other cells. There is no proven treatment.

In the new study, 12 patients will be treated with healthy scavenger cells, created in a laboratory from human embryonic stem cells. This early phase of the research is primarily to test the safety of various doses, injecting only one eye of each patient.

"We're also hoping to see some improvement in visual acuity, but that's a bonus," said Dr. Robert Lanza, ACT's chief scientific officer.

The research will be performed at medical centers in Massachusetts, New Jersey and Oregon, ACT said.

Stephen Rose, chief research officer of the Foundation Fighting Blindness, said his group is "very, very glad" that ACT has permission to begin the study.

More information: ACT: http://www.advancedcell.com

Stargardt disease: http://bit.ly/bioNHS

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3 comments

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KomMaelstrom
not rated yet Nov 22, 2010
I read about this on Google news last night. Didn't know Physorg was so slow.
3432682
not rated yet Nov 22, 2010
The score for human therapy trials (at clinicaltrails.gov) is:
Adult stem cells - 3,185
Embryonic stem cells - 11

Adult stem cells (ASC) are obviously vastly more promising for human therapy, compared to embryonic stem cells (ESC). The research money is split about 50/50 between the two types. ESC have real problems with mutation and tumors. Research money ought to be directed in the more practical direction, toward ASC.
snoozzell
not rated yet Nov 22, 2010
The only thing you've proven with these "stats" is how ignorant you are of how this science works.

You need to reevaluate how you value research priorities. ASC clinical trials are proceeding quickly due to a relatively low barrier to meet human transplant requirements and as a result of primarily one procedure : bone marrow transplant (which has been going on for 40 years)

ES cells themselves may never provide a single functional therapy but are consistently proving to be hugely clinically valuable because they provide the only system in existence that can model human embryologic development in the lab. However, they have currently have a high barrier to clinical trial because of the necessities for animal-cell coculture and the lack of sufficient #'s of quality ES cell lines to meet immune system HLA matching requirements for transplant.

#'s of clinical trials is a terrible way to guage the value of a line a research

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