Operational Milestone #3
Angiocrine Bioscience, Inc. has recently completed several IND-enabling preclinical studies for its experimental stem cell therapy, AB-110. The studies were funded by a grant award from the California Institute for Regenerative Medicine. AB-110 consists of E-CEL UVEC™ cells, which are engineered human endothelial cells obtained from umbilical cord tissue. Angiocrine’s proprietary technologies enable these cells, which are normally difficult to grow, to be produced at clinical and commercial scales. The E-CEL UVEC cells can multiply blood stem cells outside the body. That is because E-CEL UVEC cells mimic a natural process in the body called the ‘stem cell vascular niche’ where endothelial cells nurture, grow and instruct stem cells. Angiocrine is culturing E-CEL UVEC cells with cord blood stem cells outside the body and multiplying the latter to make AB-110. Because cord blood stem cells come in very small amounts, multiplying these stem cells with E-CEL UVEC cells can potentially provide a larger dose, and that may lead to a more effective and safer stem cell therapy. With many ‘expansion’ technologies, growing stem cells causes them to lose their quality. However, since E-CEL UVEC cells mimic the natural process found in the human stem cell vascular niche of bone marrow, E-CEL UVEC cells can multiply blood stem cells without loss of quality. AB-110 is first being studied in patients who have life-threatening blood and bone marrow cancers. AB-110 is designed to rebuild a new blood and immune system for the treated patient. The new blood and immune system not only would help the patient recover and live a normal life again but would also fight against any recurrence of the cancer. Hence, AB-110 is potentially curative.
The pre-clinical safety studies, which the FDA recommends for all cell therapies of this nature prior to entering a human clinical trial, have been successfully completed to the satisfaction of Angiocrine Bioscience and the FDA. Specifically, the toxicology studies consisted of two studies, one evaluating AB-110 and the other just the E-CEL UVEC cell component of AB-110. Across both murine studies, there were no test article-related deaths, clinical observed adverse reactions, hematological abnormalities, clinical chemistry findings, or changes in organ weight parameters.
Because E-CEL UVEC cells are genetically engineered, the capacity for generating tumors by either E-CEL UVEC cells alone or AB-110 was thoroughly tested. No tumor growth was seen in any animals injected with E-CEL UVEC cells alone or as part of AB-110 (E-CEL UVEC plus stem cells). Furthermore, the E-CEL UVEC cells were meticulously tracked in multiple biodistribution studies with an advanced molecular biology technique called qPCR which quantitatively measures genetic material at a molecular level. The E-CEL UVEC cells within AB-110 were found to be rapidly cleared, most within days after injection. These formal evaluations are in agreement with ten years of published basic science research on the safety profile of this cellular platform.
These pre-clinical studies were also supplemented with various analytical tests regarding the sterility of the materials used to generate this cell therapy as well as a highly specialized test to detect existence of any pathogens (e.g., unintended virus infection of cells). Collectively, Angiocrine Bioscience has completed animal and analytical tests demonstrating that AB-110, and E-CEL UVEC cells alone, have a safety profile appropriate to advance to testing via clinical trials in patients who have life threatening blood and bone marrow cancers.