1#凤凰涅盘
Participants in the First Stem Cell Clinical Trials for Retinal Disease Show Improved Vision
anuary 24, 2012 – In the first-ever clinical trials of a stem cell treatment for retinal disease, two women – one with dry age-related macular degeneration (AMD), the other with Stargardt disease – demonstrated improved vision, including the ability to read more lines on an eye chart with their treated eyes.
Most important, the treatment is improving their independence and ability to perform daily activities. The 78-year-old woman with dry AMD says that she has begun making her own breakfast and shopping by herself. The Stargardt patient, a graphic designer in her fifties, reports doing more chores around the house and riding her bike again. She also notes improvements in her color perception and ability to adapt to dark settings.
Researchers from the Jules Stein Eye Institute at the University of California, Los Angeles, conducting the two Phase I/II clinical trials — one for each disease — are also pleased with the safety results for the first two patients. No adverse events have occurred, and overall safety has been demonstrated. Four-month results of the study were reported January 23, 2012, in the journal Lancet.
The treatment is being developed by Advanced Cell Technology (ACT), a biotechnology company. The Foundation Fighting Blindness funded earlier stem cell lab studies, including the innovative work of Dr. Ray Lund, which made ACT’s clinical trials possible.
“This is a promising report. We are very pleased to learn that the treatment has been safe thus far — demonstrating safety is paramount in these early human studies — and we are delighted that both patients are showing vision improvement,” says Dr. Stephen Rose, chief researcher officer, Foundation Fighting Blindness. “While the trials are still at an early stage, the results are good news for more than 10 million people in the U.S., and millions more around the world, affected by retinal degenerative diseases.
“We at the Foundation have been excited about the potential of stem cells for saving and restoring vision, and ACT’s results show that we are on the right track. We look forward to more results from this trial and future Foundation-funded clinical trials for treatments derived from stem cells.”
During these early stages, ACT’s treatment involves the transplantation of 50,000 retinal pigment epithelial (RPE) cells transplanted into each eye. The RPE cells, derived from human embryonic stem cells using a procedure that wouldn’t destroy the embryos, play a critical role in supporting photoreceptors, the cells that provide vision. In dry AMD and Stargardt disease, RPE cells degenerate, which leads to the dysfunction and death of photoreceptors. ACT will transplant as many as 200,000 RPE cells in each patient’s retina as the Phase I/II trials progress. Twelve people will be enrolled in each of the two trials.
Researchers note that the cells transplanted into the Stargardt disease patient appeared to integrate in the proper location of the retina and take on normal RPE structure and organization. The anatomical results for the dry AMD patient were not as favorable; the RPE cells did not integrate. But the researchers were encouraged by the vision improvement they appeared to provide.
Dr. Rose notes, “We need to keep in mind that these results are for the first two people with a retinal disease ever to be treated with a therapy derived from stem cells. They are very short-term results, so there is a lot more to learn. With that said, the news is very encouraging and exciting.”
These results have been covered extensively in the media, including this report by BBC News.作者: 雪山飞狐 时间: 2012-1-28 18:16
下面是两篇来自ACT公司官网的文章是对上述内容的补充，各位战友可以研究一下。
ACT Announces that Additional Patient with Stargardt’s Disease Has Undergone Embryonic Stem Cell Transplantation at UCLA’s Jules Stein Eye Institute
MARLBOROUGH, Mass. — Jan. 25, 2012 – Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today the the dosing of an additional patient in its Phase 1/2 trial for Stargardt’s macular dystrophy using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Tuesday (Jan. 24) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA’s Jules Stein Eye Institute. The patient successfully underwent the outpatient transplantation surgery and is recovering uneventfully.
“This is the fourth patient worldwide treated with ACT&rquo;s hESC-derived RPE cells,“ said Gary Rabin, chairman and chief executive officer of ACT. “We are pleased to be on schedule with our clinical programs to test the safety and tolerability of ACT&rquo;s stem cell-derived RPE cells. We are working with the best ophthalmology institutes to evaluate the capacity of our cell therapy which may have the ability to treat a variety of devastating diseases.”
The clinical trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. It is a prospective, open-label study, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt&rquo;s macular dystrophy at 12 months, the study&rquo;s primary endpoint. The patient, a 47-year-old male, was treated with the smallest dose of 50,000 cells. The hESC-derived RPE cells are currently also in Phase 1/2 for dry age-related macular degeneration. Preliminary results for the first two patients in each of the Phase 1/2 clinical trials were recently reported in The Lancet.
A third Phase1/2 trial for Stargardt’s macular dystrophy was recently initiated at the Moorfields Eye Hospital in London, treating the first patient on Jan. 20.
Stargardt&rquo;s Macular Dystrophy (SMD) is one of the most common forms of macular degeneration in the world. SMD causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium or RPE cell layer.
Further information about patient eligibility for the Stargardt&rquo;s macular dystrophy study is also available on www.clinicaltrials.gov
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 www.advancedcell.com.
Forward-Looking Statements
Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates,” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company’s periodic reports, including the report on Form 10-K for the year ended December 31, 2010. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company’s clinical trials will be successful.作者: 凤凰涅盘 时间: 2012-1-29 08:55
10#凤凰涅盘 http://www.advancedcell.com/news ... -patients/index.asp
ACT Publishes First Report of Embryonic Stem Cell (ESC)-Derived Cells Transplanted Into Patients
Study in The Lancet Shows No Safety Concerns and Improved Vision in Patients with Macular Degeneration
MARLBOROUGH, Mass. — Jan. 23, 2012 – Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that Phase 1/2 clinical data published in The Lancet as an early online publication demonstrate the safety of ACT’s human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cells for the treatment of Stargardt’s macular dystrophy (SMD) and dry age-related macular degeneration (dry AMD). Results were reported for two patients, the first in each of the Phase 1/2 clinical trials. In addition to showing no adverse safety issues, structural evidence confirmed that the hESC-derived cells survived and continued to persist during the study period reported. Both patients had measurable improvements in their vision that persisted for more than four months.
At four months following treatment, no hyperproliferation, tumorigenicity, ectopic tissue formation, or apparent rejection were observed in either patient at any time. Detailed clinical and diagnostic laboratory assessments were performed at multiple post-transplantation evaluations. Abnormal growth (or tumor formation) would be considered a significant safety concern for stem-cell based therapies, in particular those derived from hESCs due to their pluripotency; it is therefore critical to control the differentiation of hESCs. Results reported indicate that stem cell differentiation was well controlled in these patients. No adverse safety signals were detected.
Anatomic evidence of successful stem cell derived RPE transplantation was observed clinically and with high resolution imaging technology in the patient with SMD. This evidence included increasing pigmentation at the level of RPE, within the area of the transplant, beginning one week after transplantation and throughout the follow-up period. Transplanted stem cell derived RPE appeared to engraft in the proper location and assume normal RPE morphology. Engraftment and increasing pigmentation were not detected in the dry AMD patient. However, both patients showed some visual improvement at the four month follow-up period.
Measuring visual improvement in patients with very low vision is difficult, and no regulatory consensus exists regarding on how best to measure visual changes in these patients. As reported in The Lancet, the visual acuity of the Stargardt’s patient improved from hand motions only to 20/800 vision. Before treatment, the patient was unable to read any letter on the ETDRS visual acuity chart. However, by two weeks post-transplantation, she was able to start reading letters, which improved to five letters at one to three months in the treated eye.
“It has been over a decade since the discovery of human embryonic stem cells,” said Robert Lanza, M.D., chief scientific officer of ACT, and co-senior author of the paper. “This is the first report of hESC-derived cells transplanted into patients, and the safety and engraftment data to date look very encouraging. Although several new drugs are available for the treatment of the wet type of AMD, no proven treatments currently exist for either dry AMD or Stargardt’s disease. Despite the progressive nature of these conditions, the vision of both patients appears to have improved after transplantation of the cells, even at the lowest dosage. This is particularly important, since the ultimate goal of this therapy will be to treat patients earlier in the course of the disease where more significant results might potentially be expected. We would like to thank the patients for their willingness to participate in these safety studies. It has provided the scientific community with important data and experience that will help advance efforts in the regenerative medicine field.”
Human embryonic stem cells can provide a superior source of replacement tissue by producing an unlimited number of healthy “young” cells with potentially reduced immunogenicity. The eye is an immune privileged site due to the protection of the subretinal space by a blood-ocular barrier, and as a result only low and transient doses of immunosuppression were used. No signs of rejection or inflammation were observed in either patient, and doctors will continue to monitor both patients.
“We are extremely pleased with these first clinical results from our ongoing studies to determine the safety and tolerability of subretinal transplantation of hESC-derived RPE cells,” said Gary Rabin, chairman and CEO of ACT. “This represents an important milestone not only for ACT and UCLA”s Jules Stein Eye Institute but also for the field of regenerative medicine. The publication of these data in The Lancet demonstrates their quality and importance. We would like to thank the team, patients and principal investigator for their contributions to this study which have resulted in this outstanding publication. The data underscore the potential of stem cell therapies and regenerative medicine to realize the possibility repairing or replacing tissues damaged from disease. We are looking forward to the continuation of our clinical programs and the generation of additional data.”
The hESC-derived RPE cells underwent extensive safety studies prior to transplantation. The cells were confirmed to be free of animal and human pathogens, and a high sensitivity assay was performed to rule out the presence of any undifferentiated hESCs in the final product, a risk factor for tumor formation. Controlled hESC differentiation resulted in near-100 percent pure RPE. A central feature of hESCs is that the stage of in vitro differentiation can be controlled to maximize survival and functionality. The data here show that the extent of RPE maturity and pigmentation may dramatically impact subsequent attachment and growth of the cells after transplantation.
“It is an honor to initiate the translational research process as we begin to take stem cell biology out of the laboratory and into the operating room,” said Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA’s Jules Stein Eye Institute, principal investigator of the study and author of the publication. “The scientific and regulatory teams, as well as the leadership at ACT have been exemplary. Recognizing that we are reporting positive preliminary safety data, and a functional signal that there may be a biological benefit to patients in terms of visual increase, makes this is an exciting time for ophthalmology and regenerative medicine.”
Both trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with SMD and dry AMD at 12 months, the studies’ primary endpoint. Each trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. Both the SMD and dry AMD patient had subretinal transplantation of the smallest dose (50,000 cells) of fully-differentiated RPE cells derived from hESCs. In addition to the two clinical trials in the U.S., the company has obtained clearance from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin treating patients as part of a Phase 1/2 clinical trial for SMD. Patient enrollment has begun and the first patient was treated at Moorfields Eye Hospital in London last Friday.
The paper’s other authors are Jean-Pierre Hubschman, Gad Heilwell, Valentina Franco-Cardenas, Carolyn K. Pan, and Rosaleen M Ostrick at UCLA and the Jules Stein Institute; and Edmund Mickunas, Roger Gay, and Irina Klimanskaya at ACT.
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 www.advancedcell.com.
About Jules Stein Eye Institute
The Jules Stein Eye Institute at UCLA is focused on teaching, research and patient care. For more information, visit http://www.jsei.org/.作者: 南国乞儿 时间: 2012-1-29 08:58
谢谢楼主的分享.请哪位高手翻译一下.作者: 凤凰涅盘 时间: 2012-1-29 09:01
11#凤凰涅盘
Forward-Looking Statements
Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates,” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company’s periodic reports, including the report on Form 10-K for the year ended December 31, 2010. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company’s clinical trials will be successful.作者: 寒冰吹雪 时间: 2012-1-29 10:13
