Impressions from Climb for a Cure Mount Kilimanjaro participants

"We have made great progress over the past two decades but we can't sit back and say the job is done when so many CML patients are still not receiving the best drug for them, and so few are achieving treatment-free-remission. With the Climb for a Cure Mount Kilimanjaro 2019 we wanted to do something a bit out of our comfort zone to celebrate 10 years of work by the iCMLf and to mark a new phase ...

"Researchers, patients, patient advocates, physicians and supporters of the Foundation joined us on the Climb for a Cure Mount Kilimanjaro 2019. For me this represents the whole CML community. It's how the community works together and responds together" (Nicola Evans, Chief Executive of the iCMLf)

"I spend my days treating patients with CML and we can treat them sucessfully, but the rest of the world is not like that. We have a bigger obligation to patients in other parts of the world that don't have what we have. I like this focus of the iCMLf" (Kendra Sweet - researcher and hematologist from the US)

"I pledged to do this for me and for all CML patients in Nigeria. CML is not known in Nigeria, the government doesn't know we exist because this is a rare cancer. What I really want to do is to make a change. It might not be possible in a lifetime but at least I will give it a try. It's better than not trying. From there I'll see what happens"(Andy Ojelabi - CML patient from Nigeria)

"The most amazing experience I've ever had, not only about climbing Kilimanjaro, but making this as a group and sharing our experiences" (Katia Pagnano - researcher and hematologist from Brazil)

"The perfect way to meet new friends, discover new places and challenge myself while supporting a noble cause. I would have never dreamed of climbing Mount Kilimanjaro. If I can do this, CML researchers can find a cure for CML" (Rihab Nasr - researcher and hematologist from Lebanon)

"Patients do this mountain climbing every day if they have the diagnosis. I wanted to show my small tribute to their daily struggle" (Vamsi Kota - researcher and hematologist from the US)

Climb for a cure 2019 - Ayla, Conny and Yunus Borowczak

"Thanks to effective medication, CML has changed from a fatal to a chronic disease. This is a great achievement and gives CML patients hope. But this is not enough – more can be done!Our children need a healing therapy! This is our highest priority as a family and we want to reach the top and achieve a cure for CML."(Conny Borowczak with her daugher Ayla and her son Yunus - a CML patient)

Maximising achievement of TFR while minimising failed TFR attempts and other negative outcomes

The iCMLf also formed a second Alliance under the banner of CURE - the iCMLf Treatment-Free-Remission (TFR) Alliance. The mission of the iCMLf TFR Alliance is to maximise achievement of TFR while minimising failed TFR attempts and other negative outcomes.

Background

The dramatic success of tyrosine kinase inhibitors (TKIs) has led to the widespread perception that chronic myeloid leukemia (CML) has become another chronic disease, where lifelong commitment to pharmacological control is the paradigm. Recent clinical trials demonstrate that some CML patients who have achieved stable deep molecular response can safely stop their therapy without relapsing. Furthermore, those who are unsuccessful in their cessation attempt can safely re-establish remission after restarting their TKI therapy. Hence, the concept of treatment-free remission (TFR) is currently being discussed intensively in the CML community. This is changing our approach for the many CML patients who have achieved a stable deep molecular response on long-term TKI therapy as well as the many CML patients who have not achieved a stable deep molecular response. A greater understanding regarding the key determinants of TFR is needed to enable us to make TFR an achievable goal for the majority of our CML patients.

Planned priorities of the TFR Alliance

Develop and assess clinical predictors of TFR
Develop and assess biomarkers of TFR
Clinical registries and trials of TFR
Virtual tissue bank of TFR samples
Disseminate a correlative study collection and storage protocol for TFR studies
Assess safety of current TFR practice
Frequency, management and mechanisms of withdrawal syndrome
Assess long-term outcomes - is TFR safer than long-term TKI therapy for patients in deep molecular response (DMR)?

iCMLf Global TFR Registry

The iCMLf is calling for applications to build and maintain a global registry of TFR cases, for the predominant purpose of improving our understanding regarding the determinants of TFR.
The call for expressions of interest is now closed.

Expressions of interest should be no more than a total of 2 pages outlining briefly:

Project proposal including specific aims
What makes their proposed registry unique
Methodology and statistical analysis plan
Relevant experience and achievements including any existing data from retrospective or existing patients
Potential sources of additional funding
Sustainability of the registry and broad budget outline

Selected applicants will be invited to submit detailed proposals for the registry in early March 2021 and would have 8 weeks to prepare a final application. These will be peer reviewed by a panel of CML experts.

More information on the call for proposals can be found here

It is envisaged that the establishment phase of the registry will take place from June 2021 to June 2022, but a timeline and a detailed budget will be required for full applications.

Aim of the iCMLf Global TFR Registry

The aim of the iCMLf Global TFR Registry is to build an International registry of patients who have (1) attempted to achieve TFR and/or (2) will attempt to achieve TFR, by integrating clinical data from the collaborating members of the TFR alliance who have established local or regional TFR registries. The purpose of the global registry is to develop and validate predictors of TFR success, look at other aspects of TFR outcomes (late relapse, etc.), and (critically) facilitate exchange of clinically annotated samples from registry patients to further our understanding about the biological and molecular features of TFR.

Current iCMLf TFR Alliance members

Timothy Hughes (SAHMRI / University of Adelaide) (Chair)
Jane F Apperley (Imperial College, London)
Charles Chuah (Duke-NUS Medical School, Singapore and Department of Haematology, Singapore General Hospital)
Richard Clark (University of Liverpool)
Simone Claudiani (Imperial College Healthcare NHS Trust, London)
Jorge Cortes (Georgia Cancer Center, Augusta)
Michael Deininger (Huntsman Cancer Institute, University of Utah, Salt Lake City)
Brian J Druker (Oregon Health & Science University Knight Cancer Institute, Portland and Howard Hughes Medical Institute, Portland)
J. Valentin Garcia Gutierrez (Hospital Universitario Ramón Y Cajal)
Jan Geissler (CML Advocates Network)
Nobuko Hijiya (Columbia University, New York)
Carlo Gambacorti-Passerini (University of Milano Bicocca, San Gerardo Hospital, Monza)
Andreas Hochhaus (Universitätsklinikum Jena)
Dennis Kim (Princess Margaret Cancer Centre, Toronto)
Dong-Wook Kim (Seoul St. Mary's Hospital Seoul)
Francois-Xavier Mahon (University of Bordeaux)
Michael J Mauro (Memorial Sloan Kettering Cancer Center, New York)
Dragana Milojkovic (Imperial College Healthcare NHS Trust, London)
Satu Mustjoki (University of Helsinki)
Tiong Ong (Duke NUS Medical School)
Carolina Pavlovsky (Fundaleu, Buenos Aires)
Jerry P Radich (Fred Hutchinson Cancer Center, Seattle)
Delphine Réa (Hospital Saint-Louis, Paris)
Susanne Saußele (University of Mannheim)
Giora Sharf (CML Advocates Network)
Naoto Takahashi (Akita University, Akita)
Ravi Bhatia (University of Alabama at Birmingham)

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Registered Address: International CML Foundation - 20 Eversley Road - Bexhill-On-Sea, East Sussex, TN40 1HE - UK