CML: A tiger in the cage? A (European) perspective on CML disease management, presented by Jan Geissler, co-founder CML Advocates Network, at the 5th Friends of Max Leadership Summit in (virtual) India on 21 Feb 2021, Session "Beyond Borders"

1. CML: A tiger in the cage?
A (European) perspective on
CML disease management
Jan Geissler, CML Advocates Network

2. Within 20 years, treatment evolution changed CML
from end of life care to a chronic cancer
Today
with current TKIs
1994
or untreated
German CML Study Group, update 2013
Survival
probability
Year after diagnosis
n = 3682
Imatinib, 2002 – 2012 (CML IV)
5-year survival 90%
10-year survival 84%
IFN or SCT, 1997 – 2003
(CML IIIA) 10-year survival 61%
IFN or SCT, 1995 – 2001 (CML III)
10-year survival 48%
IFN, ± HU, 1986 – 1994
10-year survival 27%
Hydroxyurea, 1983 – 1994, 10 yr surv. 18%
Busulfan, 1983 – 1994, 10-year survival 11%
(CML I, II)
2000

3. Imatinib
Nilotinib
Dasatinib
Bosutinib
Ponatinib
+Interferon
Asciminib
Transplant
Cure
Within 20 years, treatment evolution changed CML
from end of life care to a chronic cancer…

4. …but it’s still a tiger that kills if it escapes
(CML advanced, resistant, badly treated or untreated)

5. Patient perspective on CML 2021
• With access to current TKIs and good management,
most patients can live a long life, even though not
completely normal
• Still a chronic cancer, with all its psychological, social and
medical challenges
• Some patients have significant side effects
• Treatment for decades is unsustainable for many patients
and for healthcare systems
• Unfortunately, too many patients still die from CML
today. Either because they don’t have access to drugs
or PCR, or because they progress on resistance, or
because of insufficient management of disease
• We (still) need a cure.
• In the most wealthy countries, for ~45% of patients
stopping treatment in remission (TFR) is an attainable
goal. For 55%, it’s not.
30%
No stable MR4
35%
1st stop
successful
(50% of all
patients
stopping in
MR4)*
10.5%
(~35% TFR
rate in 2nd
stop)**
24.5%
TFR failure
after 1st
and 2nd stop*/**
* TFR failure rates vary from 45% to 65% in various STOP studies, e.g. STIM, EUROSKI, DASFREE, ENESTop, ENESTFreedom.
** RE-STIM.1
CML, chronic myeloid leukaemia; MR, molecular response; PCR, polymerase chain reaction; TFR, treatment-free remission;
TKI, tyrosine kinase inhibitor.
Therapy-free failure and
success

6. T
F
R
The chance for getting rid of side effects and financial burden
vs. leaving the “safe harbour” trying therapy-free remission…

7. Key priorities in CML 2021
• Access to second/third/fourth-generation drugs
• Minimizing long-term toxicity in our chronic cancer,
especially cardiovascular toxicity on 2nd generation TKI
• Returning to good quality of life: multi-intolerance,
low grade side effects, family planning
• Managing multidrug resistance, progression and death
for some
• Tackling (un)intentional non-adherence to therapy
(disease and therapy fatigue)
• Achieving a real cure – upcoming drugs like Asciminib
won’t fix that

8. Strategic priorities of the global CML
Advocates Network in close collaboration
with The Max Foundation
http://www.cmladvocates.net
Global voice for CML
patients
Represent voice of CML patients
tailor activities to regions,
address national / local needs
Leader of cancer patient
community
Be a role model for other
disease areas / rare cancers
Realise synergies with other
cancer organizations, e.g.
health/research policy, WECAN
Partnerships with other
stakeholders in CML
research
Conduct CML Community
Advisory Board (CAB)
meetings with academia and
pharma companies
Work with CML experts
Build members’ capacity
/ capability
Support regional initiatives
Provide tools (e.g. ELN
Guidelines in 22 languages)
Build knowledge through
CML Horizons

9. Patient
advocate
Remember: It’s on all of us -
“Nothing about us without us!”
Jan Geissler
jan@cmladvocates.net

10. Nothing about us
without us!
Jan Geissler, CML Advocates Network