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:10/10/2017)... Los Angeles, CA (PRWEB) , ... ... ... Pharmaceuticals, Inc., a development-stage cancer-focused pharmaceutical company advancing targeted antibody-drug conjugate (ADC) ... all uses of targeted HPLN (Hybrid Polymerized Liposomal Nanoparticle), a technology developed ...
(Date:10/10/2017)... ... October 10, 2017 , ... USDM Life Sciences , ... life sciences and healthcare industries, announces a presentation by Subbu Viswanathan and Jennifer ... “Automating GxP Validation for Agile Cloud Platforms,” will present a revolutionary approach to ...
(Date:10/9/2017)... ... 2017 , ... The Giving Tree Wellness Center announces the ... of consumers who are incorporating medical marijuana into their wellness and health regimens. ... operators of two successful Valley dispensaries, The Giving Tree’s two founders, Lilach Mazor ...
(Date:10/6/2017)... ... October 06, 2017 , ... The HealthTech Venture Network ... at their fourth annual Conference where founders, investors, innovative practitioners and collaborators are ... pitch competition showcasing early stage digital health and med tech companies. , This ...
Breaking Biology Technology:
(Date:5/23/2017)... first robotic gym for the rehabilitation and functional motor sense evaluation of ... Italy . The first 30 robots will be available from June ... . The technology was developed and patented at the IIT laboratories and ... thanks to a 10 million euro investment from entrepreneur Sergio Dompè. ... ...
(Date:5/6/2017)... 2017 RAM Group , Singaporean ... breakthrough in biometric authentication based on a ... to perform biometric authentication. These new sensors are based ... by Ram Group and its partners. This sensor will ... chains and security. Ram Group is a next ...
(Date:4/18/2017)... , April 18, 2017  Socionext Inc., a global expert ... a media edge server, the M820, which features the company,s hybrid ... software provided by Tera Probe, Inc., will be showcased during the ... the NAB show at the Las Vegas ... ...
Breaking Biology News(10 mins):