Clinical Progress in Regenerative Medicine

06 August 2013 Personalized Medicine Bulletin Blog

Two separate clinical initiatives were recently announced that support the therapeutic use of regenerative medicine. The first utilzing induced pluripotent stem cells and the second, a state-wide network in support of researchers, physicians and patients.

Induced pluripotent stem cells (iPSCs) possess the regenerative potential of embryonic stem cells without the associated ethical controversy. To date, application of the technology has been focused on the use of iPSCs as research tools to test new drugs or model systems.

Pilot Study – Treatment of Macular Degeneration 

Dr. Masayo Takahashi, M.D., Ph.D. of the Laboratory of Regenerative Medicine, and RIKEN,Japan’s largest comprehensive research institution, have launched a pilot study to assess the safety and feasibility of the transplantation of iPSC-derived retinal pigment epithelium (RPE) cell sheets in patients with exudative (wet-type) age-related macular degeneration. The study was approved by two institutional review boards and will be conducted at theInstituteofBiomedical Research.

Severe age-related macular degeneration is a common cause of blindness that affects at least 1 % of people over 50. Dr. Takahashi, will lead the study. He has been studying the potential of iPSC for more than a decade. The form of the disease that is the subject of the study occurs when blood vessels invade the retina, destroying the retinal pigment epithelium that supports light-sensitive photoreceptors. Current drug treatments work by blocking the growth of new blood vessels, but the drug must be directly injected into the eye.

Dr. Takahashi’s approach will take cells directly from a patient’s upper arm and reprogram them to iPSCs. The iPSCs will then be transformed to a monolayer sheet of retinal cells that are placed under the damaged area of the retina. Once in the eye, the cells are hoped to grow and repair the eye. Initial transplants are to be conducted at intervals of at least eight weeks to be followed by three additional transplantations after the initial transplantations have been evaluated for safety. Transplant sites will be monitored for functional integration and potential adverse reactions during an initial one-year intensive observation period, with subsequent follow-up observation for three years. Production and validation of the autologous iPSCs and subsequent RPE cell sheets will take 10 months, and will be conducted at a certified clinical-grade cell processing center.

CIRM’s Network Supporting Clinical Trials                           

Across the Pacific, the California Institute for Regenerative Medicine (“CIRM”) recently announced a $70 million plan to create a new statewide network of sites that will act as a hub for stem cell clinical trials. The stated goal of the “Alpha Stem Cell Clinics Network” is to support and accelerate clinical trials for programs. As reported on the CIRM web site, the delivery of approved new stem cell therapies will become another focus as the field evolves.
The initial network will consist of up to five clinic sites at established academic institutions and a coordinating center that will help the clinics streamline challenging processes such as enrolling patients, managing regulatory procedures, and sharing data.

Another reported goal of the network is to educate citizens seeking stem cell therapies and to guide potential patients toward available studies and therapies. The Alpha Clinics network will provide packages of reliable information as well as patient counselors who can explain the reliable information and data that this initiative will create.

More information is available on the CIRM website.



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