Update from the Clinic: October

Author: Mark Curtis, 11/27/14

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Welcome to your Update from the Clinic for the month of October. Northwest Biotherapeutics continues to receive support for its cancer vaccine platform in Europe. Neuralstem and StemCells provided updates on their respective clinical programs for spinal cord injury. Pluristem continues to advance its second product, PLX-RAD, into large-scale manufacturing. Read on to find out more.

Northwest Biotherapeutics (NWBT) received notification that patients with Glioblastoma multiforme are now entitled to use the company’s DCVax® technology for treatment at hospitals in Germany, making it the first immunotherapy to be granted a hospital exemption under German Drug Law. The exemption will cover patients with high-grade (severe) and low-grade glioma brain cancers and be allowable whether patients are newly diagnosed or recurrent. The technology requires the removal of the tumour from the brain, and subsequent shipping to the Fraunhofer Centre where it is processed and used to prime a patient’s own dendritic cells.

Asterias Biotherapeutics (AST) and StemCells, Inc. (STEM) both have clinical programs underway for the treatment of spinal cord injury. The latter initiated the first clinical study to investigate the safety and efficacy of stem cells as a treatment for cervical spinal cord injury (Pathway study). Asterias is in possession of the old Geron intellectual property, which protects the use of human embryonic stem cell-derived oligodendrocyte progenitors. The company received $14.3 million in non-dilutive funds from CIRM this past month to progress its Phase 1/2 clinical study.

Neuralstem (CUR), which is advancing NSI-566, a human neural stem cell product, treated its first patient of four in a small, first-in-human, trial being conducted at UC San Diego. The patient, who had a thoracic spinal cord injury, has left the hospital without any reported adverse events. The company’s product is intended to “bridge the gap” above and below the injury and allow for neurons to traverse the damaged section of the spinal cord.

bluebird bio (BLUE) announced that is has treated its first patient with sickle cell disease using lentiglobin BB305, an autologous modified hematopoietic stem cell (HSC) product. The first patient is enrolled in a clinical site in Paris. Another group of eight patients will be treated in the United States. Sickle cell disease can take decades off a patient’s life. So, the indication provides generous therapeutic headroom. In June of this year, the company reported that the product was able to rapidly induce transfusion independence in patients with beta-thalassemia.

Isreal-based Pluristem Therapeutics (PSTI) has brought its second cell therapy line, PLX-RAD, into large-scale manufacturing. The name comes from the ability of the cells to mitigate damage induced by radiation in mice. The company now has two allogeneic bulk products derived from the placenta, the first of which, PLX-PAD, has been deployed across a number of different disease areas, including cardiovascular, orthopedic, pulmonary and hematology. The therapeutic effect of PLX cells is exerted via the secretome, or release of cytokines, growth factors and chemokines. Case Western Reserve University will be investigating PLX-RAD cells in a preclinical setting for the purposes of enhancing engraftment of cord blood for treating bone marrow failure.

Cell therapies for the amelioration of spinal cord injury are now progressing in the clinic, as are other cellular products being developed for disorders of the nervous system, including neurodegenerative diseases such as amyotropic lateral sclerosis (ALS) and multiple sclerosis (MS). Brainstorm Therapeutics (BCLI), is developing NurOwn™, an autologous bone marrow-derived MSC-based cell therapy for this application, and was granted a Fast Track Designation for the technology. These are ideal targets for the commercialization of cell therapies given the significant decrease in quality-of-life they are associated with.

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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
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