Treating acute lymphoblastic leukaemia

Ana Rita Fragoso

Instituto de Medicina Molecular (iMM), Lisbon, Portugal

Cancer is now the leading cause of premature death worldwide, according to the World Health Organisation (WHO). In 2020, almost 10 million deaths were caused by cancer, accounting for one in six recorded deaths. Current estimates predict that the incidence of this set of diseases will increase throughout this century, leading to higher mortality rates and a greater burden on public health systems. Therefore, it is essential to develop new, more effective medical therapies with fewer side effects.

In this regard, the project aims to develop a novel gene therapy that selectively identifies cancer cells and induces their death. The researchers are building on results obtained in preliminary studies, which have shown that the expression of molecules specific to cancer cells are capable of regulating the function of a gene that induces cell death.

They now intend to obtain proof of concept by validating the therapeutic potential of gene therapy in T-cell acute lymphoblastic leukaemia, which is a very aggressive cancer with a high relapse rate and a dismal prognosis. In the long term, the idea is to explore the potential of the therapy not only for leukaemia, but also for other types of cancer.

Development of a microRNA-regulated cell death-inducing gene therapy (miRGT) for cancer

Stage 2