CORRECTING and REPLACING Advanced Cell Technology & Collaborators at The Casey Eye Institute Present Promising Data Supporti...
13 June 2009 - 3:28AM
Business Wire
Link to article should read:�http://www3.interscience.wiley.com/cgi-bin/fulltext/122455604/PDFSTART
(sted
http://www3.interscience.wiley.com/journal/121640180/issue?CRETRY=1&SRETRY=0)
The corrected release reads:
ADVANCED CELL TECHNOLOGY AND
COLLABORATORS AT THE CASEY EYE INSTITUTE PRESENT PROMISING DATA
SUPPORTING SAFETY AND EFFICACY OF STEM CELL THERAPY TO COMBAT
RETINAL DISEASE
Study suggests hESCs could serve
as a safe source of cells to treat diseases of the eye
Advanced Cell Technology, Inc. (OTC:ACTC) and its collaborators
at OHSU reported today the long-term safety and efficacy of human
embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE)
produced under manufacturing conditions suitable for human clinical
trials. Two important early potential hESC applications are the use
of RPE for the treatment of age-related macular degeneration and
Stargardt disease, an untreatable form of eye disease that leads to
early-onset blindness. The research, which appears online ahead of
print in the journal Stem Cells, shows long-term functional rescue
using hESC-derived cells in both the RCS rat and Elov14 mouse,
animal models of retinal degeneration and Stargardt, respectively.
The cells survived transplantation for prolonged periods (>220
days) and sustained visual function without tumor formation or
untoward pathological reactions. Near-normal functional rescue was
also achieved in the �Stargardt� mouse. To further address safety
concerns, a study was carried out in the NIH III immune deficient
mouse model. Long-term data (spanning the life of the animals)
revealed no evidence of tumor formation after transplantation.
�We�re delighted with these results,� said Robert Lanza, Chief
Scientific Officer at ACT, and co-senior author of the study.
�Everything looks great so far. Based on these and other studies,
we�re on schedule to file an IND with the FDA to begin human
clinical trials sometime in the next 3 or 4 months. Barring any
surprises, Stargardt disease and macular degeneration are likely to
be the next two clinical applications of ES technology.
Since their discovery over a decade ago, hESCs have been
considered a promising source of replacement cells for clinical
studies. However, problems continue to plague clinical translation,
including the risk of teratoma formation and the need for powerful
drugs to overcome the problem of immune rejection. Until induced
pluripotent stem (iPS) cell technology is further developed,
hESC-derived therapies are likely to be limited to immunoprivileged
sites such as the central nervous system and the eye. In the
retina, compromised RPE function can lead to deteriorated vision
and photoreceptor loss in both age-related macular degeneration and
other forms of degenerative eye disease. In this study, the cell
manufacturing and documentation process complied with the
guidelines set forth by the FDA, collectively referred to as Good
Manufacturing (GMP) and Good Tissue Practices.
The cells were prepared for clinical application and tested for
safety and efficacy in several different in vitro and animal models
in collaboration with researchers at the Casey Eye Institute at
Oregon Health and Science University (OHSU) headed by Dr. Raymond
Lund. Dr. Lund, co-senior author of the paper, is widely considered
one of the world�s foremost experts in retinal cell physiology and
vision restoration. RPE from different hESC lines were tested in
the RCS rat, an animal in which vision deteriorates over several
months due to an RPE functional defect. The studies focused on
longevity of effect, dosing effect, and evidence of untoward
pathology. After three months, the treated retina had 5-6 layers of
photoreceptors, whereas the untreated animals only had a single
layer of cells remaining (they were essentially blind). Based on
these data, a cell batch was chosen for a GLP safety study using
the NIH III mouse model, an immune deficient animal model.
Importantly, we also showed that vision can be rescued in a mouse
model of Stargardt�s disease, which is a form of early-onset
macular dystrophy that leads to blindness despite an intact Bruch�s
membrane (critical for RPE cell attachment and potential clinical
efficacy).
�We have worked with Dr. Lund and his team for over three years
investigating the safety and efficacy of our RPE cell therapy,�
stated William M. Caldwell IV, Chairman and CEO of ACTC. �We are
now completing the finishing touches to seek FDA approval to
conduct human clinical trials with a cell therapy that may be able
to address many of the 200 plus known retinal diseases.�
The researchers of the paper from Advanced Cell Technology,
collaborated with scientists from Casey Eye Institute at the Oregon
Health & Science University, Portland, Oregon. The paper�s
other authors are Bin Lu (co-equal first author), Shaomei Wang
(co-equal first author), Sergej Girman, and Peter Francis at OHSU;
and Christopher Malcuit (co-equal first author) and Linda Lemieux
of ACT.
Link to article: http://www3.interscience.wiley.com/cgi-bin/fulltext/122455604/PDFSTART
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company
applying cellular technology in the field of regenerative medicine.
For more information, visit http://www.advancedcell.com.
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