Leukemia genetics: The genetic and chromosomal abnormalities in AML make treating this disease particularly difficult. Identification of these genetic variations is necessary in order to adapt treatment options to the distinct genetic characteristics of the leukaemia cells. New techniques of gene sequencing have revealed mutations that may have a role in the development of AML. Researchers will be able to develop new, precisely targeted therapies with help of the information provided by this.

New drugs and treatment regimens: Treatments for AML are being created that are safer and more effective. They are investigating either new medicines and the delivery of current medications in different dosage and delivery methods. The overall survival of AML patients has risen over the last few decades as a result of improvements in the understanding of disease genetics. However, to greatly increase survival, researchers are investigating chemotherapy drug combinations with much more current targeted therapies and continuing to modify and reformulate traditional chemotherapy drugs. The following treatment modalities are now being explored for use in AML patients:

Novel Targeted Therapies: A targeted therapy uses drugs or other substances to block the function of certain enzymes, proteins, or other molecules important in the growth and survival of cancer cells, with fewer side effects on healthy cells.

FLT3 Inhibitors Newer generation FLT3 inhibitors, such as quizartinib (previously AC-220) and crenolanib, are being investigated in combination with chemotherapy to treat patients with newly diagnosed and relapsed AML with FLT3 mutations.

p53 Inhibitors Tumor suppressor genes, which prevent healthy, normal cells from getting cancer, are inhibited by p53 gene mutations. For the treatment of AML with p53 mutations, APR-246 (eprenetapopt), a novel small molecular inhibitor, is being investigated. The mutated gene's function is targeted and restored by APR-246.

Menin inhibitors: Menin protein has been related to the growth and development of some leukemias due to mutations in the KMT2A gene (formerly known as the mixed-lineage leukaemia or MLL gene) and the NPM-1 (nucleophosmin-1) gene. Menin inhibitors have been shown to have anti-leukemic effects in AML with KMT2A and NPM-1 mutations in the lab. Clinical trials are being performed on two menin inhibitors, KO-539 and SNDX-5613, that could be treatment options for these specific genetic types of leukaemia.

Focused on the metabolism of cancer: Devimistat (CPI-613®), a brand-new medicine, targets the enzymes involved in the energy metabolism of cancer cells and increases the sensitivity of cancer cells to various chemotherapies.Combining existing treatments with devimistat has the potential to improve their effectiveness and allow for the use of lower doses of generally toxic drugs.

Immunotherapy: This is a type of biological therapy that helps the body fight cancer by either increasing or reducing the immune system as needed. It uses substances produced synthetically in a lab or naturally by the body to improve, target, or restore immune system function.

The use of monoclonal antibodies: To treat AML, this type of targeted therapy is being studied. Part of the immune system are antibodies. Antibodies are normally created by the body in response to antigens such as bacteria, viruses, and cancer cells. The antibodies that bind to the antigens and help in their destruction. Certain antigens, such as CD123, which is found on the majority of AML cells, are now being investigated by researchers as potential targets. Magrolimab (an anti-CD47 antibody), which works by making malignant cells recognizable to macrophage cells, is a promising example. These are immune system cells whose mission it is to "eat" sick and unhealthy cells and remove them from the body.

Even in those with mutations like p53, treatment with magrolimab and azacitidine appears to be effective in older patients with newly diagnosed AML. Cusatuzumab is a different antibody that targets specific leukaemia stem cells, the kind of cells that trigger the disease to relapse, as it express the CD70 antibody. In older AML patients, treatment with cusatuzumab with azacitidine is being investigated.

Antibody therapy for the bispecific T-cell engager (BiTE): AML cells are simultaneously attacked by special antibodies that also encourage the patient's immune system to combat these malignant cells. AMG-330 is one such medication that makes use of T cells to specifically target AML cells that exhibit the CD33 antigen. Another, flotetuzumab, directs T lymphocytes to CD123, which is expressed in chemotherapy-resistant AML cells.

Vaccine Therapy: To generate a potent immune response to a patient's cancer, researchers are developing vaccines that could be customized to each individual patient. Patients who are in remission are the intended audience for these vaccines. They work by generating an immune response against the leukaemia cells in an effort to, hopefully, prevent the disease from recurring. The WT1 (Wilm's tumour 1) antigen, which is expressed on many AML cells, is one of the vaccine's targets.

Chimeric Antigen Receptors CAR T-Cell therapy: This type of immunotherapy genetically changes the patient's own immune cells to detect and fight cancer cells. Researchers are investigating whether these treatments are effective in AML patients. They are also investigating if it is possible to cure patients more quickly by using someone else's modified immune cells in a "off the shelf" approach, instead of the patient's own cells.

Best Regards
Ruby Thomson
Editorial team