Industry update: Clinical Trials

Written by Dusko ILIC

Latest developments compiled from 1—30 September 2014

Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions 1—30 September 2014, scheduled to be published in Volume 10 Issue 1 of Regenerative Medicine.

Clinical trials

Advanced Cell Technology

Advanced Cell Technology (MA, USA;, has completed treatment of the final patient in its UK-based Phase 1 clinical trial for Stargardt’s macular degeneration. The successful transplantation of the Company’s proprietary RPE cells in the last patient of twelve represents the completion of the enrollment stage of this trial. The trial is a prospective, open-label study designed to determine the safety and tolerability of the Company’s RPE cell therapy following sub-retinal transplantation into patients. Further information about this clinical trial are available at (ID: NCT01469832).


BioTime’ (CA, USA; subsidiary Asterias Biotherapeutics (CA, USA; has reached an agreement with Cancer Research UK ( and Cancer Research Technology (, the charity’s development and commercialization arm, to conduct a clinical trial of Asterias’ novel immunotherapy treatment AST-VAC2 in subjects with non-small cell lung cancer. AST-VAC2 is an allogeneic cancer vaccine designed to stimulate patients’ immune systems to attack telomerase, a protein that is expressed in over 95 percent of cancers but is rarely expressed in normal adult cells. AST-VAC2 is derived from human embryonic stem cells (hESC), meaning it can be produced on a large scale and stored ready for use, rather than having to produce a specific version of the drug for each patient. The trial of AST-VAC2 will evaluate the safety and toxicity of the vaccine, feasibility, stimulation of patient immune responses to telomerase and AST-VAC2, and clinical outcome after AST-VAC2 administration in patients with resected early-stage lung cancer and in patients with advanced forms of the disease. Under the agreement, Asterias will complete development of the manufacturing process for AST-VAC2. Cancer Research UK will then produce the vaccine and conduct the phase 1/2 clinical trial in the UK. On completion of the clinical trial, Asterias will have an exclusive first option to acquire a license to the data from the trial on pre-agreed terms including an upfront payment, milestones and royalties on sales of products. If Asterias declines this option, CRT will then have an option to obtain a license to Asterias’ intellectual property to continue the development and commercialization of AST-VAC2 and related products in exchange for a revenue share to Asterias of development and partnering proceeds.


Cellular Biomedicine Group (CBMG; CA, USA; launched a study on human adipose derived mesenchymal progenitor cell (haMPC) therapy for cartilage defects resulting from osteoarthritis or sports injury. Both arthroscopy and the use of magnetic resonance imaging will be deployed to further demonstrate the regenerative efficacy of ReJoinâ„¢ on cartilage defects. The clinical study is a singleblind, randomly assigned, controlled, clinical research study that will enroll thirty patients with the purpose of evaluating the safety and efficacy of haMPCs combined with lavage, debridement, and/or microfracture under arthroscopy treatment for cartilage defects. In addition, this study shall serve as a supporting study of ReJoin or the treatment of knee osteoarthritis so as to bring additional clinical data to the protocol of future knee osteoarthritis trials. The study will be conducted in Shanghai Ninth People’s Hospital. The company performed a similar safety/efficacy clinical study on 18 patients in Shanghai Renji Hospital a year ago (“Autologous Adipose Tissue Derived Mesenchymal Progenitor Cells Therapy for Patients With Knee Osteoarthritis’) and it is running another one on 48 patients (“Clinical Trial of Autologous Adipose Tissue-Derived Mesenchymal Progenitor Cells (MPCs) Therapy for Knee Osteoarthritis”) at the same hospital comparing effects of haMPC and sodium hyaluronate. Further information about these two clinical trials are available at (ID: NCT01809769 and NCT02162693).


On September 12, a Japanese woman in her 70-ies was the first person to receive tissue derived from iPSC, In a two-hour procedure, a team of three eye specialists implanted a 1.3 x 3.0 mm sheet of retinal pigment epithelium cells into an eye of the patient, who suffers from age-related macular degeneration. The procedure took place at the Institute for Biomedical Research and Innovation (Japan; The patient had no effusive bleeding or other serious problems after the surgery.


Osiris Therapeutics (MD, USA;, announced interim data from an evaluation of Grafix® in the treatment of chronic venous leg ulcers. The analysis evaluated 18 wounds treated with Grafix and compression therapy compared to 13 case matched control wounds with similar wound area and wound age treated with standard compression therapy alone. Overall healing rates in the Grafix patients were 50% compared to 15.4% in the control group (p<0.05). In addition, patients in the Grafix group had significantly reduced mean time to complete healing (11.9 weeks vs. 29.9 weeks, p<0.05) and mean length of therapy (12.6 weeks vs. 106.3 weeks, p=0.015) compared to the compression only group. The on-going evaluation will include up to 50 total patients. Grafix is a cryopreserved placental membrane for acute and chronic wounds. It is a flexible, conforming membrane that is applied directly at the site of the wound. Grafix is produced using Osiris’ BioSmart Intelligent Tissue Processing, which maintains the integrity of the extracellular matrix, growth factors, and endogenous fibroblasts, epithelial cells and MSC of the native tissue.


UC San Diego (CA, USA: will be initial site for the first-in-human Phase I/II clinical trial of a hESC-derived therapy for patients with Type 1 diabetes. The trial will assess the safety and efficacy of a new investigational drug called VC-01, which was recently approved for testing by the U.S. Food and Drug Administration. The 2-year trial will involve four to six testing sites, the first being at UC San Diego, and will recruit approximately 40 study participants. Further information about this clinical trial are available at (ID: NCT02239354).