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Marek Mráz, Credit: National Institute for Cancer Research

CEITEC Scientist Receives ERC Grant to Advance New Treatment for Aggressive Leukemia

Physician and molecular biologist Marek Mráz from CEITEC Masaryk University and Brno University Hospital has received a prestigious ERC Proof of Concept grant from the European Research Council. The funding will support the further development of a potential new therapy for acute myeloid leukemia (AML), one of the most aggressive forms of blood cancer.

The ERC Proof of Concept grant is awarded to researchers who have previously secured major ERC funding and is designed to help move scientific discoveries closer to practical application. In this case, it will allow Mráz’s team to build on earlier research into substances that block the GAB1 protein, which plays a key role in cancer cell survival.

Mráz and his research group at CEITEC MU and Brno University Hospital have long focused on understanding how cancer cells evade treatment. Their earlier work centered on chronic lymphocytic leukemia (CLL), where tumor cells can survive therapy by reshaping internal signaling pathways. During testing of new compounds developed under a previous ERC Starting Grant, the team made a key discovery: inhibitors targeting the GAB1 protein were effective not only against CLL, but also against AML and several other leukemias and lymphomas.

AML remains one of the most serious hematological diseases. It often affects younger patients, and despite current treatment options, more than half of those diagnosed die within a year. Researchers have shown that the GAB1 protein plays a central role in helping leukemia cells survive and adapt to treatment. Blocking this protein disrupts these processes and weakens the cancer cells.

“Targeting GAB1 is technically demanding because it is not an enzyme, which is the most common target of cancer drugs,” explained Mráz. “Instead, it acts as a signalling hub inside the cell. This means we need to use a different strategy to interfere with its function.”

The newly awarded ERC PoC grant will allow the team to develop and test second-generation GAB1 inhibitors, expand preclinical research, and assess their potential for future clinical use. The project will also support collaboration with industrial partners.

The work builds on a previously granted US patent for GAB1-blocking substances developed by Mráz’s team. While the path from laboratory discovery to an approved treatment is long and complex, the researchers see this project as an important step toward new therapeutic options for patients with AML and other hematological cancers.

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