Roadmap Towards Cure

The CML patient community recognises that – while significant advances in the treatment of CML have been made – more needs to be done. The CML Community Advisory Board (CML-CAB) has set the achievement of a cure for CML as being one of their strategic priorities.

With CML having become a chronic disease for most patients thanks to 5 Tyrosine Kinase Inhibitors (TKIs) currently available, it is often considered a “lucky cancer”, a “done deal”, or a “ticked box”. However, in many countries still today there are severe access issues (both to therapy and PCR testing), and the side effects burden of a life-long therapy, the toxicity and non-adherence cannot be denied. Furthermore, TKI-treatment is a real burden especially on younger patients (since it seriously hinders family planning) as well as a financial burden on healthcare systems due to increasing prevalence.

Treatment Free Remission (TFR) is often presented as “cure”, however stopping treatment requires a stable deep molecular response which is only possible after many years of deep remission (every +1 year DMR -3% relapse rate). Furthermore, TFR success is only likely for at best 25-35% of patients, the reasons for this being: 1.) requirements to stop treatment aren´t met, 2.) no or limited access to PCR testing, and/or 3.) 50% relapse rate even with best TKI. That said, TFR can control the disease but not eradicate it.

Moreover, life-long PCR monitoring (if available at all) is a constant reminder of the disease. Last but not least, the stigma of cancer adheres to patients, treatment discontinuation causes fear, anxiety and depression, and there remains a risk of undetected (late) relapses that may lead to progression and death.

The CML patient community therefore calls for:

  • Recognition that cure is an unmet need in CML, not a done deal with TFR
  • Joint definition of the problem the community/CML-CAB trying to solve:
    •  All CML patients (which is not given with TFR)
    • can live a normal, long life (which is usually given with TFR)
    • with equivalent quality of life of a normal person (there is uncertainty if this is given with TFR)
    • in absence of any CML disease (which is not given with TFR)
    • without need of any CML therapy (which is given with TFR)
    • without need of continuous monitoring (which is not given with TFR)
  • Commitment and collaboration between all stakeholders, first and foremost patient community and CML researchers to finding a real cure for CML
  • More funding for research on a cure for CML
  • Building a joint, patient-centric “Research Network for CML cure” that helps coordinate, drive and fund research in that space

In October 2020, the CML community held its first Academic CML-CAB meeting on “Mechanisms to cure CML – beyond TFR” to which CML-CAB had invited 8 researchers, all of them working on novel areas of CML research. The main objectives of which were to:

  • Build a dialogue with academic researchers
  • Reach consensus on what the key issues are in CML (status quo)
  • Establish a vision of where we want to be and how a cure could look like (beyond TFR)
  • Look at different approaches to cure and learn which of these are most promising to cure CML
  • Identify what the community can contribute to achieve the vision of a cure for CML, and define a “roadmap to cure”

This first Academic CML-CAB revealed that there is a necessity for further research beyond TFR, as well as a sustained effort and commitment from all CML stakeholders to work together in placing a roadmap towards the ultimate achievement of a cure for CML. This Roadmap towards Cure is a strategic plan that will define the desired outcomes to be achieved and the major milestones needed to reach it. That way, the identified roadmap will take all stakeholders involved on a journey to advance our understanding of CML and ultimately provide a probable feasible route to a cure.

The development of a “Roadmap Towards Cure” is work in progress. We will provide updates as they become available.


In case of questions, please contact our Scientific Project Manager Eglys González-Marcano at