National Brain Tumor Society (NBTS) is pleased to announce it has awarded an Innovation Research Grant to Tocagen Inc., which is developing an innovative therapeutic approach to the treatment of glioblastoma multiforme (GBM). Tocagen is preparing for clinical trials and the NBTS grant supports the evaluation of its technology in those clinical trials.
NBTS is excited about this award to Tocagen, believing it will represent a major step forward in driving brain tumor research towards therapies. It demonstrates NBTS’s fierce commitment to improving the lives of those affected by brain tumors and goes beyond the significant contributions that NBTS and its legacy organizations have already made to the field.
NBTS and research
The NBTS research strategy is to find, fund, and follow brain tumor research that leads to translational testing in clinical trials. NBTS encourages multiple research approaches, including focusing on understanding tumor complexity through the NBTS Systems Biology Brain Tumor Research Initiative. In contrast, the grant to Tocagen will support technology that concentrates on a general property of GBM and does not rely on an in-depth understanding of the specific and complex aberrations that drive tumor growth. By balancing these two alternative approaches, NBTS seeks to enhance the likelihood of finding effective brain tumor therapies.
About Tocagen
Tocagen is a small, private biotechnology company focusing exclusively on the treatment of GBM. Its research is the highest quality at all levels, from evaluating relevant biological questions through the production of its therapeutic agents for clinic trials, to its clinical trial plans. Tocagen is managed by an extremely experienced biotechnology business team that has enjoyed previous success. The team is capable of driving their brain tumor technology toward therapeutic application and fulfilling its vision of developing effective brain tumor therapies that will improve the lives of patients.
Tocagen’s research study
Tocagen’s approach to the treatment of glioblastoma is based upon the general nature of GBM cells to divide rapidly. It differs from other potential interventional approaches because it is based upon the inhibition of specific molecular targets.
In Tocagen’s technology, gene therapy is coupled with a prodrug, which must undergo a chemical conversion by metabolic processes before becoming an active pharmacological agent. In the gene therapy portion, a replicating viral vector is injected into a recurrent GBM to deliver a particular gene to tumor cells. The virus replicates in dividing tumor cells and the newly formed virus is able to spread throughout the tumor without infecting normal, non-dividing cells. The gene that is now in the tumor cells allows those cells to produce an enzyme called cytosine deaminase (CD).
In preclinical animal studies, Tocagen has demonstrated that when those tumor cells that express the CD enzyme are treated with the non-toxic prodrug, 5-FC, the tumor cells convert the prodrug into the toxic drug, 5-FU. The 5-FU drug then kills the tumor cells while leaving normal cells unharmed.
Tocagen has received full approval by the FDA and the Institutional Review Boards of UCSF, UCLA and the Cleveland Clinic to start their Phase I clinical trial and has tested this technology in its first patients in clinical trial.
The NBTS Innovation Grant
The Innovation Grant of $100,000 per year, for two years was awarded following a stringent review of the scientific and clinical approach used Tocagen by the NBTS Scientific Advisory Council (SAC). By supporting Tocagen with this grant, NBTS is demonstrating the belief that this approach has real potential in the treatment of brain tumors. It does not represent an endorsement of the Tocagen technology over others, only that it is promising in itself.
The grant is unique in that it supports the Tocagen research program as a whole with a focus on the correlative and biological studies in the clinical trial. These studies include evaluating those parameters within clinical trial patients that would be able to tell why the therapy did or did not work. To perform the evaluation, the studies will look at genes and enzymes that control the metabolism of the toxic 5-FU agent; the control of the spread of the viral vector through the tumor; the ability of the viral vector to efficiently replicate in the tumor cells, and the level of cell division within the tumor.
