Through exclusive world-wide licenses to more than 30 issued patents and patent applications, we have secured the rights to a broad patent estate within the Treg field, covering natural Tregs (nTregs), induced Tregs (iTregs) and methods of treating or preventing certain conditions and/or diseases by use of Tregs. Both types of Tregs have been shown in pre-clinical studies to be important in modulating autoimmune and inflammatory diseases. nTregs have been evaluated by others in early phase human clinical trials and shown to be safe with suggestions of clinical benefit in graft-versus-host disease. Both nTregs and iTregs have demonstrated the ability to treat conditions like diabetes, inflammatory bowel disease and organ transplant tolerance in animal models of disease.
NeoStem’s Immune Modulation Program is establishing methods to isolate and expand human nTregs for large scale manufacturing to enable our planned clinical trials.
Type 1 Diabetes Program Development
Type 1 diabetes (also known as insulin dependent diabetes or juvenile diabetes) is caused by the autoimmune destruction of insulin-producing beta cells of the pancreas.
We have established a collaboration with UCSF and the laboratories of Drs. Jeffrey Bluestone and Qizhi Tang, to collaborate on the development of a therapy for the treatment of type 1 diabetes. This collaboration is advancing the Company’s Immune Modulation Program towards a Phase 2 trial, expected to be initiated in 2014 to evaluate the efficacy of autologous Tregs in type 1 diabetes.
In this collaboration, NeoStem will sponsor and manufacture a Treg product consisting of polyclonally expanded Tregs for the planned Phase 2 trial to treat patients newly diagnosed with type 1 diabetes. The collaboration also includes research efforts to develop the next generation of Treg products for therapeutic use. Dr. Bluestone, the Study Director, and Dr. Kevan Herold, the Study Principal Investigator (Yale University), completed the Phase 1 study of autologous Tregs in type 1 diabetes and expect to report the Phase 1 results at the American Diabetes Association Scientific Sessions in Chicago being held on June 13-17, 2014. These results are expected to serve as the basis for the initiation of the Company’s planned Phase 2 study.
Steroid Resistant Asthma Program Development
Asthma, another condition caused by an imbalance in the immune system, occurs when excessive inflammation is triggered in the lungs resulting in constriction of the airways and difficulty breathing. The causes of asthma are complex, however it is known that over-activity of T-helper type 2 (Th2) cells is a common feature. Th2 cells secrete the inflammatory signals that lead to the symptoms of asthma. Existing evidence indicates that Treg cells may regulate Th2 activity and therefore may have a beneficial effect on severe asthma. We are planning a pilot clinical trial to assess the effect of Treg therapy on severe asthma. We expect to initiate in Canada, a Phase 1 trial of Tregs to support a steroid resistant asthma indication in 2014.
Other Potential Therapeutic Targets
Other potential therapeutic targets for T Regulatory cells could include prevention of organ transplant rejection and graft vs. host disease (GVHD). Tregs have been evaluated in early phase human clinical trials and have shown clinical results in cases of GVHD, lupus and multiple sclerosis.