Navigation Links
Assessment of safety and efficacy of human embryonic stem cell therapy
Date:6/12/2009

Worcester, MA June 11, 2009 (EurekAlert)--Advanced Cell Technology, Inc. (OTC:ACTC.PK) 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."


'/>"/>

Contact: Kathy Singh
ksingh@advancedcell.com
508-756-1212 x653
Advanced Cell Technology
Source:Eurekalert

Related biology technology :

1. Serial Monitoring With ImmuKnow(R) is Useful in the Risk Assessment of Kidney Transplant Recipients
2. PDHI Launches Short Form of Its ConXus Profile Health Assessment
3. The Coalition to Prevent Deep-Vein Thrombosis (DVT) RV Tour Takes to the Road to Educate the Nation About the Importance of DVT Risk Assessment
4. New Program Offers Breast Cancer Clinicians and Patients Support for Pre-Operative Clinical Assessment and Ongoing Surveillance of Lymphedema
5. 4C Controls Announces the Appointment of Dr. Philip Hayden as Vice President, Security Assessment & Threat Analysis
6. United BioSource Corporation and CogState Ltd. Form Strategic Partnership to Support Cognitive Assessment in Clinical Research
7. VAP(R) Cholesterol Test Bolsters Biophysical(R) Health Assessment
8. Simbionix Releases Powerful New Learning Management System With Enhanced True Assessment System(TM)
9. OncoGenex receives completed Special Protocol Assessment for primary registration study of lead drug candidate OGX-011
10. Follow-up Phone Surveys: Effective Customer Service Assessment Tool for Improving Satisfaction
11. Expert Consensus Panel Recommendation for Incorporating Lp-PLA2 Testing into Cardiovascular Disease Risk Assessment Guidelines
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/15/2017)... , ... August 15, 2017 , ... ... biosensors that accelerate pharmaceutical and biotherapeutics development, announces the launch of the new ... steps needed to gain kinetic binding data for a wide range of molecules, ...
(Date:8/15/2017)... ... August 15, 2017 , ... ... of 6” modular downlights designed to stay tightly sealed and perform efficiently for ... damp and wet location listings just aren't enough, such as: hospitals; behavioral health ...
(Date:8/15/2017)... Allentown, PA (PRWEB) , ... August 15, 2017 ... ... and encrypted shopping cart. Now mobile responsive, the new website makes it ... lab or anywhere in between. Users can now find detailed product information, educational ...
(Date:8/14/2017)... ... August 14, 2017 , ... The Conference Forum has confirmed the ... place on September 6, 2017 at the Marriott Copley Place in Boston, MA. , ... Informatics, and Regulatory Strategy, Pfizer Innovative Research Lab, Pfizer, who leads 19 industry speakers ...
Breaking Biology Technology:
(Date:4/13/2017)... SANTA MONICA, Calif. , April 13, 2017 /PRNewswire/ ... New York will feature emerging and ... Innovation Summits. Both Innovation Summits will run alongside the ... variety of speaker sessions, panels and demonstrations focused on ... east coast,s largest advanced design and manufacturing event will ...
(Date:4/11/2017)... 2017 Research and Markets has announced the ... to their offering. ... eye tracking market to grow at a CAGR of 30.37% during ... Market 2017-2021, has been prepared based on an in-depth market analysis ... and its growth prospects over the coming years. The report also ...
(Date:4/6/2017)... LONDON , April 6, 2017 ... Control, RFID, ANPR, Document Readers, by End-Use (Transportation & ... Energy Facility, Oil, Gas & Fossil Generation Facility, Nuclear ... Healthcare, Educational, Other) Are you looking for ... Authentication sector? ...
Breaking Biology News(10 mins):