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


Related Stories

Recommended for you

Study of smoking and genetics illuminates complexities of blood pressure

February 15, 2018
Analyzing the genetics and smoking habits of more than half a million people has shed new light on the complexities of controlling blood pressure, according to a study led by researchers at Washington University School of ...

New mutation linked to ovarian cancer can be passed down through dad

February 15, 2018
A newly identified mutation, passed down through the X-chromosome, is linked to earlier onset of ovarian cancer in women and prostate cancer in father and sons. Kunle Odunsi, Kevin H. Eng and colleagues at Roswell Park Comprehensive ...

A gene that increases the risk of pancreatic cancer controls inflammation in normal tissue

February 14, 2018
Inflammation is a defensive response of the body to pathogens, but when it persists, it can be harmful, even leading to cancer. Hence, it is crucial to understand the relationship between inflammation and cancer. A group ...

Scientists develop low-cost way to build gene sequences

February 13, 2018
A new technique pioneered by UCLA researchers could enable scientists in any typical biochemistry laboratory to make their own gene sequences for only about $2 per gene. Researchers now generally buy gene sequences from commercial ...

New insights into gene underlying circadian rhythms

February 13, 2018
A genetic modification in a "clock gene" that influences circadian rhythm produced significant changes in the length and magnitude of cycles, providing insight into the complex system and giving scientists a new tool to further ...

Clues to aging found in stem cells' genomes

February 13, 2018
Little hints of immortality are lurking in fruit flies' stem cells.


Adjust slider to filter visible comments by rank

Display comments: newest first

not rated yet Nov 22, 2010
I read about this on Google news last night. Didn't know Physorg was so slow.
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.
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

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.