Mission

Our mission is to bring safe, effective, innovative treatments to market for patients with serious and life threatening diseases, by deploying smart therapies in areas of significant unmet medical need.

Smart Weapons for the Fight Against Cancer

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A smart bomb, actively guided to the target by a ground observer, can destroy a deeply buried, heavily reinforced underground bunker, with absolute precision and minimal collateral damage. Cancer too is heavily protected, by a myriad of evasive and defensive mechanisms, against modern medicine. Today, for the first time, Bellicum’s drug-based remote control technology extends the physician’s reach beyond the point at which a treatment has been administered. Our mission is to leverage this smart technology to bring safe, effective, innovative cell therapies to market for patients with serious and life threatening diseases.

Cell therapy - the process of introducing new cells into a patient in order to treat a disease – comes in many forms. Hematopoietic stem cell transplants (HSCT) have been curing patients with hematologic malignancies for decades, and the first cellular immunotherapy for cancer was approved by the FDA in April 2010. In the future, pluripotent and multipotent stem cell therapies promise cures for conditions ranging from heart disease and diabetes to neurological disorders and spinal cord injuries. However, safety issues and confounding biological defense mechanisms are severely limiting the adoption and efficacy of these potentially curative therapies. Our smart technology addresses these challenges:

CLINICAL CHALLENGE

BELLICUM SOLUTION
Irreversible Toxicity
CaspaCIDe™ Self Destruct Switch
A remote self destruct switch is fundamental to the safety of a smart bomb. Non-replicating cells (e.g. dendritic cells) are eliminated by the body over time, like a drug, so any toxicity may resolve naturally. In contrast, T and stem cell therapies are designed to expand and persist, so toxicities can be irreversible. Known life-threatening side effects include GvHD in HSCT patients, cytokine storm in T cell recipients, and cancer in stem cell patients. The risk of lethal toxicity necessitates cautious, often sub-optimal dosing, so a safety concern leads to an efficacy constraint, further limiting adoption. A rapid, effective self destruct switch is widely recognized as a potential game changer. Cells have a built-in self destruct mechanism, called apoptosis. CaspaCIDe™ gives the physician control of Caspase-9 signaling, which directly activates apoptosis, allowing elimination of CaspaCIDe™ T cells previously transplanted into the patient, with no effect on the patient’s own tissues. The physician administers the CaspaCIDe™ activating drug if the cells become harmful, or after they have served their purpose. In an ongoing clinical trial in patients undergoing HSCT, we have demonstrated consistently rapid elimination of activated T cells and effective reversal of GvHD. Similar effectiveness has been shown in stem cell applications in published preclinical studies.
Therapeutic Vaccine Efficacy
DeCIDe™ Vaccines
One of the most important and impenetrable defenses adopted by cancer cells is evasion of the immune system. This multifactorial phenomenon allows cancer cells to escape detection and elimination by white blood cells. Therapies designed to boost the immune system to overcome these mechanisms are blocked by biological feedback systems that govern killer T cell activation. These checks and balances prevent autoimmune diseases such as lupus, MS and RA, but they have limited the effectiveness of cancer vaccines to a median overall survival benefit of 4-8 months. DeCIDe™ therapeutic dendritic cell (DC) vaccines give the physician control of Toll-Like Receptor (TLR) and CD40 signaling pathways, which govern activation of T cells and are normally tightly regulated. Critically, CD40 activation must occur in the patient’s lymph nodes, so remote control is essential. This is accomplished by administering the activating drug 24 hours after vaccination, following DC arrival in the lymph nodes. Thus, the physician guides the vaccine around the cancer’s immune defenses. Data from an ongoing clinical trial is establishing proof of principle.