A Phase I SBIR program of preclinical development is proposed focusing on the development of an active specific immunotherapeutic called Cellarium"""""""" for treatment of glial cell-based cancers. Successful completion of this project will establish the minimum level of therapeutic feasibility and lead to further development of the vaccine in preparation for first-in-human studies. Upon demonstration of safety and efficacy, Cellarium would provide a greatly- improved, targeted treatment for glioma patients, which currently have dismal survival prognosis, are expensive to treat, and for whom current treatments can be debilitating and painful. Gliomas are widespread deadly diseases for which current therapies are inadequate. In the US gliomas accounted for 44% of the 22,070 cases of brain and other nervous system cancers diagnosed and 12,920 associated deaths in 2009. Recent studies have also shown trends toward increased incidence of glioma in US adults. Currently, gliomas are predominantly treated with an aggressive three step protocol. Despite this regime, patients with Glioblastoma Multiforme (GBM), the most common and deadly malignant brain tumor, still have a median survival of less than 15 months. Moreover, the estimated cost of treatment for each patient with a malignant brain tumor ranges from $30,000 to several hundred thousand dollars annually. Based on the Cellarium"""""""" technology platform developed at Harvard University, this in situ vaccine, which incorporates well-established biological cofactors and patient specific cell lysate, attracts dendritic cells (DCs), exposes them to the tumor specific antigens, and then releases them to direct a bolstered, targeted immune response against malignant tissue. Support is requested to refine vaccine formulation and demonstrate initial efficacy and safety in an in vivo therapeutic glioma model, an in vivo tumor resection model8 and biocompatibility studies. If Phase I studies are successful, the Cellarium vaccine will continue to be developed for clinical trials which the eventual goal being scaled manufacture in the US and sale as a commercially available specific immunotherapeutic vaccine to treat glioma patients.
We propose to develop an active specific immunotherapeutic vaccine that helps the body's own immune system identify and then attack cancerous glial cells. This device, if validated, could give physicians and their patients a much needed therapy prolong survival and improve treatment of glioma, a disease which currently affects around 21,560 US citizens, has a 15 month median survival time and can be both painful and harmful to treat. Furthermore, this therapy would offer a significant cost reduction to the health care system by not only supplanting the need for the current, expensive treatment regimes but also preventing additional expenditures that stem from the side effects of current treatments.
