The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and Gene Therapy Advisory Committee (GTAC) have given Advanced Cell Technology the go ahead to start treating patients in a Phase I/II clinical trial evaluating the use of human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cells as a transplantation therapy for Stargardt macular dystrophy (SMD). Start of the U.K.-based trial marks the first time an hESC-based product has progressed into human studies anywhere in the world outside the U.S.
A Phase I/II clinical trial evaluating transplantation of hESC-derived RPE cells for the treatment of SMD and age-related macular degeneration (dry MD) has been initiated in the U.S., where the first two patients underwent transplantation in July. Both the Stargardt trial and the dry AMD trial in the U.S. will enroll 12 patients each.
The U.K. trial will be carried out at Moorfields Eye Hospital in London. “There is real potential that people with blinding disorders of the retina including Stargardt disease and age-related macular degeneration might benefit in the future from transplants of retinal cells,” comments James Bainbridge, M.D., principal trial investigator and chair of retinal studies at University College London. “The ability to generate retinal cells from stem cells in the laboratory has been a significant advance, and the opportunity to help translate such technology into new treatments for patients is hugely exciting."
ACT has two other cell therapy-based products in its pipeline. The myoblast program for the treatment of congestive heart failure is based on autologous myoblasts derived from the patients’ skeletal muscles. Four Phase I studies have been completed and FDA has cleared the start of a Phase II study, the firm says. Its preclinical-stage hemangioblast (HG) program aims to generate functional hematopoietic and angiogenic hemangioblast cells from hESC, for use in the potential treatment of blood and cardiovascular diseases. ACT claims preclinical studies have shown that hemangioblasts can help repair tissues and/or help blood flow in animal models of diabetes-related retinal damage, ischemia-repurfusion injury of the retina, myocardial infarction, and hind limb ischemia.