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Leukaemia treatment drug developed by MU receives US patent

The success highlights excellent basic research with great application potential. Obtaining a patent is a prerequisite for the development of new drugs and for potential collaborations with pharmaceutical companies.

Associate Professor Marek Mráz’s research team

Associate Professor Marek Mráz’s team at Masaryk University and the University Hospital Brno has been granted a US patent for the use of GAB1 inhibitors in the treatment of haematological malignancies. This achievement builds on more than a decade of research into the GAB1 protein and its role in the biology of B-cell cancers.

MU CEITEC researcher Marek Mráz has achieved a major milestone in the study of chronic lymphocytic leukaemia. His team received the first US patent for the potential use of GAB1 inhibitors as a treatment for haematological malignancies (such as leukaemia and lymphomas). In 2018, Mráz and his team won the ERC Starting Grant, which is highly coveted in the scientific community. This allowed the research group to focus on intensive research into the development and treatment of chronic lymphocytic leukaemia – the most common type of leukaemia in adults. The disease is caused by the uncontrolled growth of B lymphocytes, immune system cells that normally defend the body against pathogens by producing antibodies.

The US patent application was filed in 2020, and the MU Technology Transfer Office team has been working on it ever since. “The patent confers a monopoly in the United States for the use of GAB 1 inhibitors in the treatment of haematological malignancies (leukaemia). The rights are owned by Masaryk University and the University Hospital Brno, which is a necessary condition for the development of new drugs and potential collaboration with pharmaceutical companies,” says Markéta Vlasáková, IP manager at the MU Technology Transfer Office, who guided Mráz’s team through the entire patent process.

Marek Mráz – Research Group Leader Senior, Marek Mráz Research Group

The development of new drugs is a multidisciplinary, long-term process, and Mráz’s team has a certain advantage over other research groups looking for ways to fight leukaemia. “Our key advantage is the possibility to draw on the expertise of Associate Professor Kamil Paruch at MU, a very experienced medicinal chemist, with whom we plan to further develop GAB1 inhibitors. Another huge asset is the close collaboration with clinicians at the Department of Internal Medicine, Haematology and Oncology of the University Hospital Brno, which allows us to study the biology of GAB1 using primary samples from patients with CLL, lymphomas or acute myeloid leukaemia,” adds Mráz.

The granting of the US patent is an important milestone, but it does not end there. “Therapeutic strategies in oncology have evolved rapidly over the past decade. Therefore, our ultimate goal is to develop a new inhibitor that specifically targets the GAB1 molecule, has a favourable pharmacokinetic profile, is patent-protected and has the potential to be used in patients with one of the blood cancers we are studying,” says Mráz.