Stem Cells in Ophthalmology Update 17: Recent ACT Updates

While I was out of the country on vacation, Advanced Cell Technology issued four news releases about their ongoing human embryonic stem cell trials. Here is a compilation of what transpired during my absence.

Jan. 25, 2012 -- ACT Announces that Additional Patient with Stargardt's Disease Has Undergone Embryonic Stem Cell Transplantation at UCLA's Jules Stein Eye Institute

Advanced Cell Technology announced 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'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'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's macular dystrophy at 12 months, the study's primary endpoint. The patient, a 47-year-old male, was treated with the smallest dose of 50,000 cells.

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.

Jan. 30, 2012 – ACT Announces Aberdeen Royal Infirmary in Scotland as Additional Site for Phase 1/2 Clinical Trial Using hESC-Derived RPE Cells for Macular Degeneration

Advanced Cell Technology announced that the Aberdeen Royal Infirmary, the largest of the Grampian University Hospitals in Scotland, has been confirmed as a site for its Phase 1/2 human clinical trial for Stargardt's Macular Dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The Phase 1/2 trial is a prospective, open-label study designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with SMD.

"A leading medical institution in the United Kingdom, Aberdeen Royal Infirmary is an ideal partner for our European clinical trial for SMD," said Gary Rabin, chairman and CEO of ACT. "Moreover, we are particularly pleased that the lead investigator is Dr. Noemi Lois, a leading expert in SMD. We continue to forge ties with some of the best eye surgeons and hospitals in the world and work towards bringing this cutting-edge therapy closer to fruition. Our preliminary results to date keep us optimistic that we are on the right path both in terms of our science and the clinical team we are working with, particularly eye surgeons such as Dr. Lois."

This approved SMD clinical trial that Dr. Lois and her team will participate in is a prospective, open-label study designed to determine the safety and tolerability of RPE cells derived from hESCs following sub-retinal transplantation to patients with advanced SMD, and is similar in design to the FDA-cleared US trial initiated in July 2011.

"It is an honor to have been designated as a site for this path-breaking clinical trial," said Noemi Lois, M.D., Ph.D. "We could not be more pleased to be a part of this trial for a promising potential new treatment for SMD, using hESC-derived RPE cells." Dr. Lois is a is a member of the Department of Ophthalmology, NHS Grampian, and associated to the University of Aberdeen, Scotland, United Kingdom. Dr. Lois practices at the Aberdeen Royal Infirmary; she is an Ophthalmologist with special interest in Medical retina and Retinal surgery.

Editor’s Note: I am in the process of ascertaining whether Aberdeen Royal Infirmary will be operating under the European NCT that Moorfields is operating under, or the U.S. NCT which UCLA and Wills Eye Institute operate under. I expect it is the former.

Feb. 13, 2012 – ACT Announces Third Patient with Stargardt's Disease Treated in U.S. Clinical Trial with RPE Cells Derived from Embryonic Stem Cells

Final Patient from First Cohort in Phase I/II Trial Using hESC-Derived RPE Cells

Advanced Cell announced the dosing of a third patient in its Phase I/II trial for Stargardt's macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Monday (Feb. 6) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA) and retina division chief at UCLA's Jules Stein Eye Institute. The outpatient transplantation surgery was performed successfully and the patient is recovering uneventfully.

"With the treatment of this third Stargardt's patient at Jules Stein Eye Institute, we have now completed the treatment of the first cohort of patients under our clinical protocol for phase I/II of our U.S. SMD trial," said Gary Rabin, chairman and chief executive officer of ACT. "We will continue to regularly monitor the three SMD patients in this trial, and by early spring anticipate review of their progress and safety-related data by the Data and Safety Monitoring Board (DSMB). With approval of the DSMB, we would then advance to the next cohort of patients and administer a higher dosage of RPE cells. In the context of all three trials we have running, this patient is the fifth person worldwide to be treated with our hESC-derived RPE cells. To date, there have been no complications or side effects due to the RPE cells, and we remain cautiously optimistic that our ongoing clinical programs will demonstrate the safety and tolerability of ACT's stem cell-derived RPE cells."

Feb. 15m 2012 – ACT Announces Approval of Wills Eye Institute as an Additional Site for Stem Cell Clinical Trial for Stargardt's Disease

Leading Eye Institute Will Participate as Site for Both of ACT's Phase I/II Clinical Trials Using Human Embryonic Stem Cell-Derived RPE Cells for Macular Degeneration

Advanced Cell announced that the Wills Eye Institute in Philadelphia has received institutional review board (IRB) approval as a site for the company's Phase I/II clinical trial for Stargardt's Macular Dystrophy (SMD), a form of juvenile macular degeneration, using human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells. Earlier this year, the Company also announced that the IRB at Wills Eye Institute had approved the participation of the institution as a site for ACT's clinical trial for dry age-related macular degeneration (dry AMD).

"We thank Wills Eye Institute once more for providing their IRB and their invaluable contribution to our macular degeneration studies," said Gary Rabin, ACT's chairman and CEO. "We are very happy that we can now report that Wills Eye Institute has been approved as a clinical trial site for both our SMD and dry AMD clinical trials. Ranked as one of the best ophthalmology hospitals in the country by U.S. News & World Report, the Wills Eye Institute is a truly world-class institution. Our team is eagerly anticipating working with Dr. Carl Regillo, a renowned retinal surgeon and director of clinical retina research at Wills Eye Institute, as well as a professor of ophthalmology at Thomas Jefferson University, along with the rest of his team as we move forward with these ground-breaking trials."

Specific patient enrollment for both trials at the Wills Eye Institute will be determined in the near future. Further information about patient eligibility for the SMD study and the concurrent study on dry AMD is also available on www.clinicaltrials.gov; ClinicalTrials.gov Identifiers: NCT01345006 and NCT01344993.