Welcome to your Update from the Clinic for the month of April. Athersys provides disappointing interim results for its MultiStem® cell therapy in ulcerative colitis patients. Sernova continues to demonstrate safety and biocompatability of its Cell Pouch technology in patients with Type 1 diabetes, with one patient now passed the 180-day mark. StemCells completes enrollment in a Phase I/II for spinal cord injury. Read on to find out more.
In a Phase 2 study of Athersys’ (ATHX) MultiStem® cell therapy, in patients with moderate to severe ulcerative colitis (a form of inflammatory bowel disease), being conducted by Pfizer, there was no significant difference between treatment and placebo groups. While significance was observed in responders at four weeks post-treatment a small subset of patients experienced declines at this juncture, resulting in a lack of significance in the overall data.
Primary endpoints, in this study of 88 patients, included reduction in number and severity of adverse events, change in endoscopic score, and changes in intestinal bleeding score. Patients are being administered a second dose of MultiStem® at eight weeks. So, there is a chance for better results in the second half of the study, but things don’t look promising at this point. Athersys is targeting a challenging patient population in this study – those individuals with both a chronic and severe form of the disease. MultiStem® is a bulk product (off-the-shelf) derived from human bone marrow. It is also being investigated in neurological and cardiovascular conditions, including acute myocardial infarction.
Toronto-based Sernova (SVA), which continues to pioneer a next generation therapy for diabetes, announced another set of interim results in its Phase 1 study of the Cell Pouch™ technology in 20 patients with unstable diabetes. Last September, Sernova announced a world-first and the first interim results following the 30-day mark; one of the patients is now past 180 days, with similarly promising results in terms of safety and biocompatibility.
Following transplant, the Cell Pouch™ undergoes vascularization and becomes in-filled with a network of capillaries to bring blood flow into the device. Islet cells are then transplanted into small reservoirs contained in the pouch and exert their effect through the release of insulin and glucagon, a peptide hormone that raises blood glucose levels. With the current results it seems almost certain Sernova will move to a Phase 2 study, which be a real landmark for the company.
StemCells (STEM) has completed enrolment of 12 patients in an open-label multi-national Phase 1/2 study investigating the company’s Hu-CNS-SC cells (human neural stem cells) for treating thoracic spinal cord injury (mid-back). Patients enrolled in the study have sustained chest-level injuries to the spinal cord, seven of whom have complete paralysis (no motor or sensory function) below the point of injury, while the remaining five have no motor function and limited sensory function. The clinical study, being run by StemCells Inc., is the first to complete enrolment to evaluate stem cells for treating spinal cord injury. The three sites include University Hospital Balgrist in Zurich, Switzerland, Foothills Medical Centre in Calgary, Alberta, and Toronto Western Hospital in Ontario.
NeuralStem (CUR) is also in the race to create a cell therapy for treating spinal cord injury. The company recently announced approval of a Phase 1 study, at the University of California, San Diego, to investigate its neural stem cell product NSI-566 in patients with thoracic spinal cord injury. Both NeuralStem and StemCells are using a tissue-derived approach, where human neural stem cells are extracted from human central nervous system tissue (the brain or the spinal cord) and then sorted, expanded in vitro, and banked in doses. This prevents cells from differentiating into unwanted cell types following transplantation, due to their terminal cell fate. It will be interesting to see how comparable the results of these studies are when they are made available in 2015.
Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visit http://ccrm.ca/industry-consortium