THE ESSENTIAL
- A combination of two existing treatments would be effective in curing juvenile myelomonocytic leukemia.
- The two treatments are 5-azacitidine and an MEK protein inhibitor.
- A clinical trial will soon be conducted on patients with juvenile myelomonocytic leukemia.
In France, chronic leukemia is very rare in children, with approximately 20 to 30 new cases per year, depending on the Arc Cancer Research Foundation. Juvenile myelomonocytic leukemia (JMLL) is particularly aggressive for most young patients. Unfortunately, treatment options are so far few.
Juvenile myelomonocytic leukemia, an aggressive pediatric cancer
Currently, the only treatment for JMML that can lead to a cure is allogeneic hematopoietic stem cell (HSC) transplantation. It consists of transferring the stem cells that make blood from a healthy person (donor) to the affected young patient. Often, in parallel, children have chemotherapy. Ihe overall survival rate with this care pathway is 50 to 60%, depending on the Arc Cancer Research Foundation.
But researchers may have found a treatment for juvenile myelomonocytic leukemia that may offer new hope: a combination of two existing therapies, 5-azacitidine and one MEK protein inhibitor. According to this work published in the journal Molecular Therapy April 5, 2023, their combination has reduced the number of cancerous blood stem cells as well as the cellular activity partly responsible for the disease.
Juvenile myelomonocytic leukaemia: ucombination of two already existing treatments
“Our research results demonstrated that the combination of two drug therapies reduced the number of cancer stem cells (…) and improved blood cell abnormalities often seen in patients with JMMLexplains Santhosh Pasupuleti, one of the authors, in a communiqué. These results provide hope for better treatment options for patients with JMML and highlight the potential of combination treatments in the fight against rare childhood diseases.“
For now, this treatment has only been tested in the laboratory on animals. But researchers have just obtained approval to conduct a clinical trial on patients with juvenile myelomonocytic leukemia for whom all other treatment options have been tested.
“We hypothesized that a combination of targeted drugs might be a better option than [les traitements actuellement] available, and we are delighted that our preclinical studies have shown this to be the case.”, concludes Reuben Kapur, another author of this study.
