Verification of efficacy in in vivo model of AML

Our project, ConfirmAML, aimed to address the urgent need for life-saving treatment for a type of blood cancer called acute myeloid leukemia (AML). AML is a particularly aggressive form of leukemia that primarily affects adults. Mutations in a gene called FLT3 are responsible for driving the growth of cancer cells in 30-35% of AML patients. Currently, patients with FLT3 mutations have a low survival rate, with only about 15% of them surviving beyond five years. We want to change that. FLT3 has been identified as a promising molecular target for treating AML, and inhibiting FLT3 has shown positive results in increasing survival rates in patients. However, existing FLT3 inhibitors on the market face a challenge: drug-induced resistance. This means that over time, the cancer cells can become resistant to the effects of these inhibitors, rendering them less effective. To prepare for successful pre-/clinical development of new and potent FLT3 inhibitors, we demonstrated the efficacy of our compounds in a xenograft model of AML. We used a mouse model that closely mimics AML, using a specific cell line which is driven by FLT3 mutations. We succeded to independently confirm that our compounds have a good potential to work effectively in vivo. This step was essential for reducing the risks associated with our technology and gaining the necessary support to continue our research.

The xenograft study performed in this project confirmed the previous results. With this result, the risk of our project will be significantly lower and the chances to attract additional investments and funding are increased. The outcome of the project contributes to the development of novel FLT3 inhibitors.