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2nd human hESC trial?

A second company has requested permission from the U.S. Food and Drug Administration (FDA) to conduct a human clinical trial using embryonic stem cells. Human embryonic stem cellsImage: Wikimedia commons, Nissim Benvenistylinkurl:Advanced Cell Technology;http://www.advancedcell.com/ (ACT) filed an Investigational New Drug (IND) application yesterday (November 18) to conduct a phase I/II trial using hESCs to treat a genetic eye disease. "I'm cautiously optimistic," said linkurl:Marco Zarbin;h

By | November 19, 2009

A second company has requested permission from the U.S. Food and Drug Administration (FDA) to conduct a human clinical trial using embryonic stem cells.
Human embryonic stem cells
Image: Wikimedia commons,
Nissim Benvenisty
linkurl:Advanced Cell Technology;http://www.advancedcell.com/ (ACT) filed an Investigational New Drug (IND) application yesterday (November 18) to conduct a phase I/II trial using hESCs to treat a genetic eye disease. "I'm cautiously optimistic," said linkurl:Marco Zarbin;http://njms2.umdnj.edu/eyeweb/CVs/zarbin.html of the Institute of Ophthalmology & Visual Science of University of Medicine and Dentistry of New Jersey, who would participate in the proposed trial. "Of all the places in the body where I can imagine cell-based therapy working, first -- because it's so seemingly simple -- is in the eye." If approved, this would be only the second clinical trial involving hESCs. The first -- Geron's study of stem cell treatment for spinal cord injury -- has been linkurl:on hold;http://www.the-scientist.com/blog/display/55898/ by the FDA since August due to linkurl:microscopic cysts;http://www.the-scientist.com/blog/display/55950/ that developed in an animal study, and linkurl:doesn't expect to resume;http://www.the-scientist.com/blog/display/56126/ until the third quarter of next year. The proposed trial aims to treat patients with Stargardt's Macular Dystrophy (SMD) -- a genetic eye disease for which there is currently no treatment and one of the most common causes of juvenile blindness. The disease results in the degeneration of the retinal pigment epithelium (RPE) -- cells that support the photoreceptors needed for vision -- which in turn causes the degeneration of the photoreceptors and leads to vision impairment. ACT proposes to transplant hESCs that have been differentiated into retinal pigment epithelial cells into SMD patients in hopes of improving visual acuity. Previous work at ACT has shown dramatic improvement in the visual performance of rats who received implants of these differentiated RPE cells, according to a linkurl:2006 study.;http://www.ncbi.nlm.nih.gov/pubmed/17009895 Furthermore, they found no adverse effects (such as teratomas) in hundreds of treated animals, and the cells are "almost 100% RPE," said linkurl:Robert Lanza,;http://www.advancedcell.com/senior-executive-officers/#Robert%20Lanza,%20M.D. ACT's chief scientific officer, meaning there are virtually no undifferentiated cells that could potentially behave differently after implantation. "From the data that we've seen, I think we're in a good position to take this into the clinic in an effective and safe fashion," said linkurl:Peter Francis;http://ordc.ohsu.edu/staff/pfrancis/index.php of the Casey Eye Institute in Portland, OR. The Casey Eye Institute is one of the clinics that would participate in the study, and Francis also contributed to the preclinical research leading up to this IND. "We're in good shape," agreed Lanza. "I'm sure [the FDA is] going to have some questions and want some additional information, but, knock-on-wood, we'll be able to start early next year with the clinical trial." The trial will involve a total of 12 patients and three clinical sites, Lanza said. "Once you show that the patients tolerate the cells, we can go in to larger numbers." In addition, given the similar pathological processes between Stargardt's Macular Dystrophy and age-related macular degeneration, ACT also hopes to file an IND in the next few months to use hESCs in age-related macular degeneration, a disease that affects millions worldwide.
**__Related stories:__***linkurl:Geron trial may resume next year;http://www.the-scientist.com/blog/display/56126/
[30th October 2009]*linkurl:Profiting from Pluripotency;http://www.the-scientist.com/article/display/55372/
[February 2009]*linkurl:FDA mulls embryonic stem cell therapy;http://www.the-scientist.com/blog/display/54544/
[11th April 2008]
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Comments

Avatar of: Pablo de Felipe

Pablo de Felipe

Posts: 6

November 20, 2009

It is a pity that the space for comments is been taken over with spam... I guess The Scientist should be able to remove it!
Avatar of: Alison McCook

Alison McCook

Posts: 68

November 20, 2009

Yes, sometimes advertisements sneak into our comments section -- we have the ability to delete any ads, but that can take a few hours to occur. I have deleted the spam that snuck into this comment thread.\n\nThanks for the feedback,\n\nAlison McCook\nDeputy Editor
Avatar of: andree goodman

andree goodman

Posts: 1

November 20, 2009

I have two daughters aged 14 and 18 and a 13 year old niece with stargartds disease and this could be a great breakthrough for them. We live in australia and would be keen to travel to the US for treatment if the trial proves successful. Please keep us informed. This is a fantastic development and hopefully it will be given approval soon so the many people afflicted with vision loss because of Stargardts disease can see again normally. Both of our girls are legally blind.\nAndree Goodman

October 30, 2010

My colleagues and I have just concluded one of India's most in-depth studies on "Clinical Trials in India". We have looked at various areas such as:\n- Market Trends\n- Growth Drivers\n- Regulatory Bodies and Framework\n- Major Players.\n-Etc.\nWe interviewed over 200 individuals and firms to collect the data in what we believe is one of the most detailed study on the subject in India. If you are interested in a copy, you may email me at infoalcpl@gmail.com. \n\nAshritha\ninfoalcpl@gmail.com

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