Researchers in Italy, funded by Curestarters, have uncovered the process our body uses to create blood cells as early as before you were born. This is the first time ever that researchers have been able to pinpoint this process. They are hopeful this discovery can help expose the causes of a rare childhood cancer, and better yet, lead to the development of better more targeted treatments for this difficult disease.
I would like to say a huge thank you to all the Worldwide Cancer Research Curestarters for making our work possible. Thanks to your support we have recently made a breakthrough in understanding how blood cells develop in the embryo and it is crucial to uncovering how blood cancers begin. Bringing us a step closer to preventing and curing JMML a rare type of paediatric leukaemia. Because of you I have so much hope that in the future children facing cancer will have access to much better treatments giving them what matters most, precious time to just be kids alongside their families. Thank you.
What is juvenile myelomonocytic leukaemia (JMML)?
JMML is a rare childhood blood cancer with limited treatment options. Sadly, around three quarters of cases are diagnosed in children below the age of three.
It's technically classified with a group of blood cancers called myeloproliferative and myelodysplastic disorders. These are conditions where the body makes too many white blood cells and the blood cells made are abnormal and haven’t developed fully.
In JMML it is a specific type of white blood cells called monocytes that are abnormal. Monocytes are part of the immune system and help the body to fight infection, and in JMML, too many are produced, and they are too underdeveloped to work properly.
How treatable is JMML?
JMML is one of the rarest blood cancers that affects children, accounting for 1% of all paediatric leukaemias, meaning about 1-2 cases per million people. And sadly, survival rates for JMML have been really poor historically, with 90% of patients dying from the disease.
With the development of bone marrow transplants this number has bettered to 50% but clearly more work is still needed to support patients.
Unfortunately, the exact underlying causes of JMML are still unknown to scientists, which is why Dr Emanuele Azzoni and his team have been focussing their research on it, and why this discovery is so exciting.
The more we know about how these cells form, the closer we are to understanding what it is that goes wrong to cause blood cancers like JMML to develop, and to a day when no life is cut short by them.
What does this discovery mean for the future of JMML?
Scientists believe they can build on this research to find new targeted treatments for JMML.
Dr Azzoni and his team originally started this project wanting to investigate new clues they had discovered that linked inflammation and the development of JMML. Following these clues, they started to examine particular molecular pathways - microscopic networks our cells uses to do their jobs and communicate with each other.
Through this pursuit, they became some of the first scientists to figure out the mechanisms behind how white blood cells are first produced while an embryo is forming. Dr Azzoni is hopeful that this discovery provides the groundwork to fully understand the origins of blood cancers, particularly paediatric leukaemia.
Why is discovery research so important for blood cancer research?
Understanding the basic biology of different types of leukaemia is the catalyst that kicks off all advancements in treatment, prevention and diagnosis.
Without understanding the disease on a molecular level we cannot continue to develop and improve new drugs that can target childhood leukaemias like JMML and that is why international cancer research like this is so important.
We stand on the shoulders of all the research that has come before us. We know that every breakthrough made will join the full and important history of cancer research and help inform and push the next generation of scientists and breakthroughs.
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1999We funded research into what drives leukaemia on a cellular level
This set the stage for more projects looking at how we can differentiate between the subtypes of leukaemia and what that could mean for how we treat them individually.
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2014You supported a project that discovered more about how leukaemia grows
This research discovered how the structures in a cell can be affected by mutations and help leukaemia grow, and unlocked the potential for more researchers asking: “Now we know more about how these leukaemias develop, could we exploit this and create drugs that are more targeted?”
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2023You helped co-fund research to create targeted treatments for infant leukaemia
Alongside Leukaemia UK, we began funding Professor Katrin Ottersbach in Edinburgh, who is aiming to exploit genes known to be involved in the development of the most common type of infant leukaemia, in order to create new targeted therapies.
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2026Two of the researchers we are funding began collaborating on infant leukaemia research
Dr Azzoni and his team began working on their project and have been collaborating with Professor Ottersbach, sharing knowledge and laboratory techniques. We are so proud that our mission to fund research anywhere in the world is seeding collaboration and helping to accelerate cures.
What could the impact of your support be?
Dr Azzoni has hope that his teams' work will lead to children in the future having access to much better treatments for JMML.
Understanding more about the very earliest stages of the disease could shift the focus from reactive to proactive and targeted care - meaning more effective and kinder treatments.
If Dr Azzoni and his team can follow the clues once again and figure out how errors in this process can lead to blood cancer development, they will then be able to pinpoint druggable targets with the final goal of developing new targeted therapies for JMML.
By focusing research on how JMML develops at its earliest stages, we could also help clinicians identify disease development earlier. This would mean more treatment options for patients and hopefully better outcomes. Even better than this, early detection could lead to new prevention strategies for JMML and diseases like it.
It's only thanks to generous donations like yours that research like Dr Azzoni’s is made possible. Your support is helping to create real change for all leukaemia patients, including those who might not even have been born yet.
Join our united effort to stop cancer.
There are so many different ways to support our search for new cures. Will you join the 90,000+ Curestarters who are already helping us get closer to a day when no life is cut short by cancer?