The Interdisciplinary Laboratory is contributing to the development of new treatments for acute myeloid leukemia (AML)
Cure rates for childhood leukemias are very high, particularly for acute lymphoblastic leukemia (ALL). Unfortunately, treatments for children with acute myeloid leukemia (AML) are often less effective. High-risk patients have survival rates of less than 50%. The research group led by Prof. Boaz Tirosh at the Case Western Reserve University School of Medicine in Cleveland (Ohio, USA) has developed a new class of drugs known as mTOR activators. AcTor, the first mTOR activator, was able to sensitize acute myeloid leukemia cells to the approved drug ixazomib. This was also confirmed in primary patient samples.
In the Interdisciplinary Laboratory of the Frankfurt Foundation for Children with Cancer, the combination of AcTor and ixazomib was tested in resistant AML cell lines from the Resistant Cancer Cell Line (RCCL) Collection. The combination of AcTor and ixazomib is just as effective in resistant AML cell lines that have adapted to drugs used in clinical practice.
An important factor in AML therapy is p53 status. p53 is a tumor suppressor gene; alterations in this protein can contribute to increased tumor growth and make treatment more difficult. The combination of AcTor and ixazomib demonstrated the same efficacy in AML cell lines with altered p53 and wild-type p53. This makes its clinical application particularly promising.
Dr. Florian Rothweiler from the Interdisciplinary Laboratory comments on the recent publication: “We are pleased that, thanks to the unique RCCL Collection, we are able to contribute to the development of a new class of drugs that will hopefully help children with cancer in the future—children for whom there are currently no effective therapies.”
The scientific publication on this topic has appeared in the journal *Molecular Cancer*:
