A combined diagnostic and targeted gene therapy platform for brain tumor relapse

Emergence of therapy resistance is a major cause of death following conventional chemotherapy and radiation for children and adults with malignant brain tumors. It is imperative to find novel options to target therapy-resistant brain tumor populations to save lives. We propose a novel platform providing a prognostic tool and a suicide gene therapy tool using engineered viruses.

It involves targeting freshly biopsied cells from brain tumor patients with our strategy in combination with standard therapy to predict risk of patients developing relapse and a gene therapy suicide tool to remove cells causing relapse. Previous suicide gene therapies have only used viruses hitting dividing tumor cells.

Our strategy will use an enigneered virus targeting non-dividing tumor cells that we know escape standard therapy.

After removing the tumor bulk using standard treatment, we propose to use our virus with a marker for diagnostic purposes or a with a suicide gene that will be activated by a prodrug to eradicate therapy-resistant cells.

Prodrug administration before recurrence will eliminate relapsed cells once they start dividing and become dangerous.The impact of our strategy will be high, providing health care professionals with a better tool to identify patients at high risk of developing recurrence.

Such patients can receive a better, more tailored treatment that will not only increase their chance of survival but also reduce their time under hospital care and rehabilitation. We protected our strategy with a filed patent and assessed our regulatory needs. We completed a market analysis to evaluate market size and potential benefit for cancer patients.

We have disclosed competitors but also potential collaborators that can cofinance and move our strategy forward to in vitro diagnostic device registration and initial clinical trials.

In our proposal we will apply for funding covering lab work to test efficacy and safety, help with registration and negotitation strategies.