Industry Update: Clinical trials

Latest clinical trial developments compiled from 1—30 June 2015

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 June 2015, scheduled
to be published in Volume 10 Issue 7 of Regenerative Medicine.

Asterias

Asterias Biotherapeutics (CA, USA; www.asteriasbiotherapeutics.com), a biotechnology company focused on the emerging field of regenerative medicine, today announced that the first patient was successfully dosed at the Shepherd Center (GA, USA; www.shepherd.org) in a Phase 1/2a clinical trial evaluating activity of escalating doses of oligodendrocyte progenitor cells AST-OPC1 in newly injured patients with sensory and motor complete cervical spinal cord injury. The open-label, single-arm study, which will be conducted at a total of up to eight centers in the United States, will test three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in 13 patients with subacute, C5 to C7, neurologically complete cervical spinal cord injury. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 will be administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging methods to assess the safety and activity of the product. Additional information on the Phase 1/2a study, including trial sites, can be found at www.clinicaltrials.gov (ID: NCT02302157).

Living Cell Technologies

Living Cell Technologies (New Zealand; www.lctglobal.com) has announced results from a Phase I/IIa clinical study of NTCELL®, immunoprotected (alginate-encapsulated) porcine choroid plexus cells for xenotransplantation in patients with Parkinson’s disease. The study, conducted in four patients in New Zealand, met its primary endpoint of safety, showing NTCELL implantation was well tolerated, with no adverse events related to NTCELL. NTCELL implantation also improved clinical features of Parkinson’s disease in the four patients studied, as measured by validated neurological rating scales and questionnaires, with the improvement sustained at 26 weeks post-implant. NTCELL, a unique cell therapy, is an alginate-coated capsule containing clusters of neonatal porcine choroid plexus cells that are sourced from a unique herd of designated pathogen-free pigs bred from stock originally discovered in the remote sub-Antarctic Auckland Islands. Choroid plexus cells are naturally occurring “support” cells for the brain and secrete cerebrospinal fluid, which contains a range of factors that support nerve cell functions and protective enzymes that are crucial for nerve growth and healthy functioning. In NTCELL, the porcine choroid plexus cells are coated with LCT’s propriety technology IMMUPELâ„¢ to protect them from attack by the immune system. Therefore, no immunosuppressive regimen is required for treatment.

Mesoblast

Mesoblast (Australia; www.mesoblast.com) announced that results from the Company’s Phase 2 trial in patients with diabetic nephropathy showed that a single infusion of its intravenously delivered allogeneic mesenchymal precursor cell (MPC) product candidate MPC-300-IV was safe, reduced damaging inflammation, and preserved or improved renal function over at least 24 weeks. MPC-300-IV was evaluated in a double blind, randomized, placebo-controlled, dose-escalating Phase 2 trial of 30 patients with type 2 diabetes and moderate to severe renal impairment, stage 3b-4 chronic kidney disease, who were already on a stable regimen of the standard of care therapy for diabetic nephropathy (renin-angiotensin system inhibition with angiotensin converting enzyme inhibitors or angiotensin II receptor blockers). Patients received a single infusion of 150 million MPCs, 300 million MPCs, or saline control.

StemCells

StemCells (CA, USA; www.stemcellsinc.com) has enrolled its first subject in Cohort 2 of its Phase II Pathway Study. The study is designed to assess the efficacy of the Company’s proprietary HuCNS-SC® platform technology (purified human neural stem cells) for the treatment of cervical spinal cord injury. Cohort 2 will enroll 40 patients and forms the single-blinded controlled arm of the Phase II study. The primary efficacy outcome being tested in Cohort 2 is the change in motor strength of the various muscle groups in the upper extremities innervated by the cervical spinal cord. The Pathway Study is the first clinical study designed to evaluate both the safety and efficacy of transplanting stem cells into patients with traumatic injury to the cervical spinal cord. Additional information about the clinical trial is available at www.sciresearchstudy.com and http://clinicaltrials.gov (ID: NCT02163876).

In independent news, the Health Canada (www.hc-sc.gc.ca) has authorized the Company to expand its Phase II clinical trial for chronic cervical spinal cord injury into Canada.