The Challenge
We are not done
Chronic myeloid leukemia (CML) has been transformed by tyrosine kinase inhibitors, turning what was once a fatal disease for many into one that can often be controlled over the long term. Yet this progress has also revealed three continuing challenges.
- Inequity: many patients still face barriers to timely diagnosis, molecular monitoring, consistent treatment, and specialist expertise, particularly in low and middle income countries.
- Burden of lifelong therapy: even when treatment is available, long term TKI use can bring significant physical, emotional, and financial impact.
- The need to move beyond long term control toward cure.
Together, these challenges show why progress in CML must be measured not only by what is possible, but by who can benefit from it, at what cost, and with what future in view.
The inequality gap
The treatment of chronic myeloid leukemia has changed dramatically over the past two decades. For many patients, modern therapies offer long term disease control, near normal life expectancy, and in some cases the possibility of stopping treatment successfully.
But these advances have not been shared equally.
For too many people, outcomes are still shaped by where they live, the resources available to them, and whether specialist expertise can be accessed when it is needed. These barriers are often greatest in low and middle income countries, where health systems may be under significant pressure and resources must be stretched across many competing priorities.
Key challenges include:
Limited Access to Diagnostics: delayed diagnosis and inconsistent or unaffordable molecular monitoring result in difficulty making timely treatment decisions
Limited Access to Treatment: the high price of therapies limits treatment choices leading to poorer outcomes.
Lack of Expertise: Shortages of trained specialists and reduced ability to access to up-to-date training and knowledge on the latest advancements in CML, further widening disparities.
Geographical Barriers: Inadequate healthcare infrastructure means patients often travel long distances to access care, adding financial and emotional strain on families.
For many patients, where they live still shapes the care they receive and the outcomes they can expect.
"The improved survival in CML directly attributable to the use of tyrosine kinase inhibitors is so dramatic that these new agents must be made available to the greatest possible number of eligible patients as rapidly as possible throughout the whole world"
- Professor John Goldman, April 2010
The burden of lifelong therapy
The success of tyrosine kinase inhibitors has transformed survival, but effective treatment does not remove the burden of disease entirely. Many patients remain on therapy for years, and often for life. Even when treatment is successful, the long term impact can be considerable for patients, families, and health systems.
Lifelong Medication
Chronic side effects
Financial and emotional burden
The constant risk of relapse
From the patient perspective, challenges include non-responsiveness or developed resistance to current therapies, the need for ongoing monitoring, long term toxicity to therapies and the absence of treatments that fully eradicate the disease. Innovative, curative therapies are urgently needed to address drug resistance, reduce toxicity, and ultimately improve the quality of life for CML patients.
Even for those who do well, lifelong therapy is not the same as cure.
The cure imperative
Alongside the need to reduce inequity and treatment burden, the iCMLf is also focused on a bold long term goal: CURE
Today, only a 20-25% of patients achieve the deep molecular responses needed to attempt stopping treatment, and only half of those remain in remission once therapy is withdrawn. To change this, the rates of deep, sustained response and successful treatment cessation must increase substantially.
The central scientific challenge is the persistence of leukemic stem cells, which can survive despite long term therapy and lead to disease recurrence when treatment stops. Understanding why these cells persist is essential to advancing cure.
This work now enters an exciting new phase. By bringing together international research teams and using advanced technologies, including single cell approaches, scientists are gaining unprecedented insight into the biology of residual disease, the bone marrow environment, and the immune factors that influence whether remission is sustained.
These advances are bringing the field closer to identifying the mechanisms that allow resistant cells to survive and to developing new strategies that can translate this knowledge into clinical trials and, ultimately, lasting cure.
The iCMLf: a way forward
The International CML Foundation works to address these challenges through education, collaboration, and global partnership.
By sharing expertise across borders, supporting physicians in resource constrained settings, and strengthening international networks, the iCMLf helps improve knowledge, decision making, and patient outcomes. Through programs such as preceptorships, regional discussion groups, expert collaboration, and global research partnerships, the Foundation is helping close the gap while also advancing progress toward treatment free remission and cure.
Find out more about the different ways you can get involved with the iCMLf and help transform lives by improving care for people living with CML worldwide.
>Get involved