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Welcome to October’s Update from the Clinic. Interesting developments this past month included the release of Phase 1/2a data from Living Cell Technologies that provides a first look at the safety and efficacy of its DIABECELL® product. We also saw an approval for Cytori Therapeutic’s Celution® System in Singapore and the first patient treated in bluebird bio’s Starbeam clinical study.

Australia’s Living Cell Technologies (LCT) announced findings from its Phase 1/2a safety/efficacy study of DIA-09 in patients with type-1 diabetes (T1D). DIA-09 is a preparation of encapsulated pig islet cells that, once transplanted beneath the abdominal skin of patients, is capable of regulating insulin levels in response to the patient’s glucose levels. Under normal circumstances, the host’s immune system would attack the foreign cells causing their destruction. However, the microspheres containing the islet cells are fine enough in their composition to exclude immune cells. As a result, patients can receive the foreign cells without requiring immunosuppression following transplantation, which is certainly favourable. Hypoglycemic events characteristic of T1D patients were reduced by 57% overall in both patient groups receiving islet cells, while 7 of 8 patients were able to reduce their daily insulin dose.

The Health Sciences Authority in Singapore granted approval to Cytori Therapeutic’s (CYTX) Celution® System for infusion of a patient’s own adipose-derived regenerative cells (ADRCs). The platform technology is designed to be amenable to a point-of-care setting and useful across multiple indications. ADRCs are a heterogeneous mixture of cells that have been shown to have beneficial medical effects via multiple mechanisms, including cell-to-cell signaling and improved vascularization. Management expects to see a material impact on sales as soon as 2014 as a result of the approval.

Massachusetts-based bluebird bio (BLUE) treated the first patient in its Phase 2/3 study of ALD-102 in patients with childhood cerebral adrenoleukodystrophy (CCALD), a rare X-linked inherited neurological disorder that eventually leads to the degeneration of the myelin cells insulating neurons in the brain. The therapy consists of a patient’s own hematopoietic stem cells that have been genetically modified with a lentivirus to include a functional copy of the ABCD1 gene. The product was granted orphan drug status by the FDA and the European Medicines Agency in 2012.

StemCells Inc. (STEM) released some very promising long-term follow-up data on patients treated with the company’s HuCNS-SC cellular product for Batten disease in its Phase 1 study initiated four years ago. The data set is quite valuable as it is the only multi-year follow-up study following transplantation of neural cells into the brain in humans. The data provides up to five years of safety data from three patients who survived the entire duration of the follow-up study. Six patients were initially treated.

In the U.S., Mesoblast (MSB) and its partner Teva Pharmaceuticals (TEVA) have been given the green light to launch a Phase 3 study of Mesoblast’s mesenchymal precursor cells (MPCs) in progressive congenital heart failure. The MPC technology is an ‘off-the-shelf’ platform product that can be used in any patient without immune rejection, and is being investigated across several indications including heart failure and heart attack. Mesoblast’s stock was up 9% off the back of the news.

Finally, after some discussion, Osiris Therapeutics (OSIR) and the FDA have come to an agreement on the regulatory path for the company’s Grafix® and Ovation® products. Grafix® is to remain on the market as a wound cover for both acute and chronic wounds. However, the product will require submission of a biologics license application (BLA) for certain expanded indications.

The data out on DIABECELL® are interesting and it is a progressive cell therapy product. However, there is contentious debate over the pros and cons of xenotransplantation – the act of transplanting animal cells into humans. While pigs provide a much more feasible source of insulin-producing islet cells for treating diabetes in humans, there are ongoing concerns at the FDA over public health issues relating to the transmission of animal viruses into the human population. As a result, the major medical markets are currently off-limits to xeno-based products. If the regulatory climate in the U.S. changes, Living Cell Technologies will be one to watch closely.

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


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Mark Curtis

Mark Curtis

Mark is a Business Development Analyst at the Centre for Commercialization of Regenerative Medicine (CCRM), where he collaborates with the team to help evaluate the commercial potential of regenerative medicine and cell therapy technologies. He began his career at Princess Margaret Hospital studying cellular reprogramming of human skin cells. Following this project, he left the laboratory and took on a role with Bloom Burton & Co., a healthcare-focused investment dealer. While there he supported the advisory team in carrying out scientific diligence on early-stage biotechnology companies. Prior to joining CCRM he was a consultant to Stem Cell Therapeutics, a Toronto-based biotechnology company focused on developing therapeutics targeting cancer stem cells. Mark received a Master’s degree from the University of New South Wales in Sydney, where he studied the directed differentiation of embryonic stem cells, and a Bachelor’s degree in Biology, from Queen’s University. Follow Mark on Twitter @markallencurtis
Mark Curtis

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