A Focus on Investigating Treatments for Cancer
NeoStem is developing an immunotherapy which uses a patient’s own tumor cells to maximize the ability of their immune system to identify and eliminate the cancer initiating cells (also known as “cancer stem cells”) that are capable of reconstituting or developing new tumors.
Our proposed therapeutics combine the patient’s own purified and irradiated cancer cells with specialized immune cells, educating the immune cells to destroy the cancer initiating cells from which tumors arise.
The company’s lead candidate, NBS20 (USAN: eltrapuldencel-T), an investigational treatment for malignant melanoma, has received Special Protocol Assessment, Fast Track & Orphan Drug Designations from the US Food & Drug Administration for its Phase 3 trial, the Intus Study, for which the company expects the first patient to be randomized in the second quarter of 2015.
In a Phase 2 randomized clinical trial in which an early formulation of NBS20 was subcutaneously injected, the product improved two year overall survival in patients with advanced melanoma (recurrent Stage III or Stage IV) to 72% compared to 31% for control patients treated with only their own tumor cells suspended in granulocyte macrophage colony stimulating factor (GM-CSF) (p=0.007). The toxicity profile was favorable with no grade IV and only one grade III (allergic reaction) event in the study. The allergic reaction was attributed to the granulocyte macrophage colony-stimulating factor (GM-CSF), an FDA-approved immune stimulant used in the final drug formulation. There were no other significant toxicities seen in either an earlier single-arm Phase 2 trial or this randomized Phase 2 trial. Local injection site reactions, such as skin irritation and itching, did occur, but the symptoms dissipated within hours after the injection. There were no significant adverse effects on hematopoietic cells or renal function, liver function, or patient performance status.
NeoStem’s immunotherapeutic approach is a platform technology that NeoStem believes could be expanded into other indications, such as hepatocellular carcinoma and other immune responsive tumor types.