ASH 2018: Asciminib (ABL001) early-phase data of CML patients with T315I mutation
With five approved tyrosine kinase inhibitors (TKIs) and none of them being curative, one may think whether there are still gaps to be filled with new CML drugs that follow similar mechanisms of action and not a totally different approach to eradicate the disease.
There is still a proportion of CML patients that does not respond to current TKIs properly or cannot tolerate them – and with non-effective treatment, they are at risk of life-threatening disease progression. Some CML patients sadly can’t access current 2nd generation TKI (Dasatinib, Nilotinib, Bosutinib) or 3rd generation (Ponatinib) drugs even if they would potentially address unmet medical need, e.g. in case of multi-resistent mutations. Additionally, about one third of CML patients achieve a good response on current TKIs but never get into MR4 remission, so they will not be able to try treatment-free remission (TFR) – so a more potent drug would probably increase “TFR probability” . There is also some concern on all current 2nd generation TKI about side effects, e.g. cardiovascular side effects or pleural effusions, so a highly effective drug with a more favourable long-term toxicity profile and a better quality of life is certainly something CML patients would like to see. Of course the ultimate goal is cure of CML, which none of the current drugs achieve.
One new but also non-curative option is now on the horizon with Asciminib, formerly called ABL001, which was presented by Prof Delphine Réa from the Hôpital Saint-Louis, Paris, France at the ASH congress this year. Asciminib is in a way similar to other TKI, as it inhibits the CML-specific BCR-ABL and stops the proliferation of the CML cells. However, called an “allosteric inhibitor”, it binds at a different site of the protein than any of the other TKIs, and hence is not affected by the known mutations of the ATP pocket that actually render the current TKIs ineffective.
ASH 2018 CML Oral and Poster sessions
- ASH 2018 - CML Advocates Network Report by Giora Sharf -
Every year in the last years I have been asked by Haematologists and also patients, why do I leave home for 6 days and fly across the world to ASH in the USA, as CML seems to be a conquered disease where the life expectancy of a patient is similar to a healthy person. When I think about it I have a few possible answers.
First, ASH is the biggest and most important hematologic conference of the year where the newest data from clinical trials and real life are published at thousands of abstracts and posters in different sessions.
Second, as I run today not only the Israeli CML patients organization but also the Flute of Light which is the home for blood cancers patients in Israel, I feel it is my responsibility to be at the place where new hopes and optimism are published for patients in different cancers like Lymphomas, CLL, Acute Leukemias and MPN. In addition, ASH is the place to meet with the top management of all the companies we work with and discuss how to strengthen our collaboration for more patients centric activities like patients engagement in clinical trials for example and improved access.
ASH opens every year on Friday with many satellite symposiums sponsored by Pharma on different diseases. This year there was no symposium on CML so I was able to attend the Advocates breakfast organized each year by Pfizer and the ICMLF meeting led by Tim Hughes which included talks on pregnancy in CML by Dragana Milosevic from the Hammersmith, Pediatric CML management by Prof Suttorp from Germany and talks by Dr Benneh from Ghana on CML management challenges in low-income countries.
CML wise the conference included the following sessions: The CML educational session, 3 oral sessions with 6 abstract presentations each, and posters presented at the end of each day at the posters session.
This report will summarize the main topics I found to be interesting at the oral and poster sessions. The main topics were new drugs in CML, TFR and Pregnancy and CML.
If you want to read more info, you can find below each summary the link to the abstract presented.
Food effects on Nilotinib: Presentation of the NIFO Study at ASH 2018
Every medicine has different characteristics how the body converts the tablet or capsule into the active substance that treats the disease. Other medications taken in parallel can influence how much of one or the other drug is being absorbed by the body, which can either increase or decrease the blood levels, impacting efficacy or side-effects. In addition, the TKIs in use with CML have different mechanisms to interact with food taken at the same time as the drug. For example, Imatinib and Bosutinib need to be taken with food, as intake on an empty stomach can cause gastrointestinal irritation. Dasatinib and Ponatinib do not have significant interactions with food, so they can be taken while eating or fasting. Nilotinib and Asciminib need to be taken without food. A poster at this year's ASH provides some interesting new data on Nilotinib.
ASH report on news about generic imatinib
When generic imatinib was introduced between 2013 and 2016 when the Glivec patent expired, generic formulations have been used recently as a more cost-effective treatment, but there are few studies that have prospectively evaluated the efficacy and safety of these drugs. By the time of introduction of generics, many patients and physicians were concerned about whether generic versions of the drug are as effective as the original, but no publications from the more strongly regulated markets have substantiated these concerns.
At this year's ASH congress, two publications addressed the topics of generic imatinib - one observational study from Brazil, Argentina and Italy on first-line treatment with branded and generic imatinib, and one observational study from Italy on switching from Glivec to generic imatinib.
ASH 2018 CML Education Session: With great success comes great responsibility
“June 2018 was the 20th anniversary of the clinical use of the first Tyrosine Kinase Inhibitor (TKI), Imatinib, in CML patients. Since then, the change in prognosis is one of the major success stories of modern cancer medicine”. Having this as an introduction of the CML section in this year’s education book of the ASH reminded me how far we’ve come in the past 20 years. Two decades have passed since first CML patients tested STI571, now called Imatinib, on initiative by CML experts Brian Druker and John Goldman against much resistance of established experts who thought this whole TKI approach is never going to work.
Today, as long as they can access current CML treatments, patients and doctors are no longer concerned with delaying the inevitable progression of the disease or the time towards bone marrow transplant, as it was in 2001. Today’s focus on the best choice of therapy based on the individual molecular biology of the disease, as well as individual comorbidities, side effects, quality of life. The main treatment goals are mainly to reduce disease burden quickly to prevent risk of early progression, and then achieve deep molecular remission and good quality of life. For about one fourth of patients, therapy-free remission after years of treatment is already an attainable goal.
“With great success comes great responsibility” was the motto of this year’s CML Education Session at ASH 2018 – reflecting that with all the choice we have, we must do our best to leverage all therapeutic options to give each patient the best care possible, to live up to the promise that any CML patient can live a normal, long and good life. The three speakers, Prof Jane Apperley from the Hammersmith Hospital in London, Prof Tim Hughes from Adelaide and Prof Charles Craddock from the University of Birmingham made this very practical by outlining therapeutic decision making on two hypothetical cases: 55 year old overweight, smoking Bob with additional chromosomal abnormalities, and 22 year old Sue who was pregnant. The three CML experts touched upon three important areas: choice of first-line treatment, molecular monitoring schedules, and advances in stem cell transplant for patients where TKIs have failed.
[Click on "read more" to read the full article, and click here for the slides of the session.]
ASH 2018 Report (I): Treating CML during pregnancy
The 60th American Society of Haematology Annual Meeting and Exposition is taking place from 1-4 December in San Diego, USA.
The meeting is providing a huge educational experience and the opportunity to review thousands of scientific abstracts highlighting updates in the hottest topics in haematology with the attendance of more than 25,000 health-care professionals as well as many patient advocates.
We are pleased to share with you our first "ASH 2018 Report", contributed by our co-founder Jan Geissler.
Fellowships available to attend the DIA Europe 2019 Conference
DIA Europe 2019 is an annual conference organized by the global association DIA that connects life science professionals from many different areas with patients, peers and thought leaders.
From 5th to 7th February 2019, key thought leaders and healthcare innovators as the industry, academia, regulatory, government agencies, patient, and philanthropic organizations will come from across all disciplines involved in healthcare products and therapies. The meeting is intended to strengthen professionals’ understanding of the value of cross-discipline integration and to foster innovation for better health outcomes.
For more information, please visit the Dia Europe 2019 website.
Apply for a fellowship by Wednesday, the 5th of December 2018, 12:00 CET
This year's conference will take place at the Austria Center Vienna from the 5th to the 7th of February 2019.
DIA Europe 2019 is offering up to five Full Fellowships and up to fifteen Registration Fee waivers to include the patient representatives input in the conversation.
Be a part of the Patient Advocacy Program for the 13th Annual Patient, Family and Donor Day
The 45th EBMT (European Society for Blood and Marrow Transplantation) Congress will be held in Frankfurt, Germany during March 24th to 27th, 2019. The EBMT is happy to announce that they have added a new segment to the Congress and would like to introduce the new parallel session "Patient Advocacy Program" which will run as a part of the 13th Annual Patient, Family and Donor Day, taking place on Sunday, March 24th, 2019.
Be a part of the Patient Advocacy Program for the 13th Annual Patient, Family and Donor Day that is held at the annual EBMT Congress in Frankfurt, Germany.
Patient advocates are invited to participate in this remarkable and essential annual event of the: Patient, Family and Donor Day, that will now include a Patient Advocacy Track and it is free to attend.
There is also the opportunity to be able to have access to the full EBMT Congress (March 24th – 27th), by applying for a free registration. You will need to be quick to apply as there is a limited amount of 30 available for patient advocates.
RCE-ESO-ESMO training course for rare cancer patient advocates: Travel grants available
Are you are Patient Advocate for Rare Cancers? If so, you need to know about the Training Course from RCE-ESO-ESMO with travel grants available.
From 01-03 December 2018, the Rare Cancers Europe (RCE) initiative, in collaboration with the European School of Oncology (ESO) and the European Society for Medical Oncology (ESMO), is organising a specific training course for Rare Cancer Patient Advocates.
The 2018 edition of the event will bring together patient advocates from across Europe, to exchange views and ideas with clinicians and researchers, who are attending the parallel ESO-ESMO-RCE Preceptorships and Clinical Update on Rare Adult Solid Cancers.
Patient advocate travel grants available: Deadline 11th November
Report from the first European Hematology Community Advisory Board (Hem-CAB) on 18 June 2018 in Stockholm, Sweden
The European hematology patient community held its first hematology community advisory board (HEM-CAB) meeting in Stockholm on 18 June 2018. The hematology patient community is a very well-connected patient community in the area of rare diseases and oncology, with 12 pan-European patient umbrella organisations, including the CML Advocates Network, that have a network of over 250 member organisations that provide information for patients and carers, engage in EU and national policy and engage in research, development and regulatory affairs.
As an immediate follow-up action from the European Hematology Association (EHA) Congress, the first Hem-CAB meeting brought together leaders from the 12 pan-European umbrella patient organisations and representatives of eight pharmaceutical companies working in hematology. The CML Advocates Network was represented by Sarunas Narbutas and Jan Geissler.
The 8-hour Hem-CAB meeting was preceded by a 3-hour training session, preparing patient advocates about the structure and purposes of the meeting. While the meeting was held under confidentiality terms from all parties to allow for an open and frank discussion, key findings and action points are summarized in this report.
Welcome, Association Fanomezana LMC! Now 119 patient organisations from 89 countries in the CML Advocates Network
We are proud to welcome a new member organisation to the CML Advocates Network: Association Fanomezana LMC, based in Madagascar. The CML Advocates Network now totals 119 member organisations in 89 countries worldwide! See the list of CML groups in the Network on our world map.
TFR 4 CML patients survey is closed!
The TFR for CML patients survey is now closed!
The CML Advocates Network launched the online global survey “TFR 4 CML Patients”, available in eleven languages (English, Spanish, German, Italian, French, Finnish, Hebrew, Arabic, Danish, Japanese and Russian) to measure the viewpoints, needs and experiences of CML patients that are considering or have already started Treatment Free Remission (TFR). From March to July 2018, the survey has collected the feedback of more than one thousand CML patients worldwide.
The findings of the survey will help us to better understand what the concerns, challenges and motivations are during the decision phase, the first probation phase after stopping, and when attempting TFR has either worked or failed.
Now, we start working on the analysis of the results of the treatment-free remission survey to provide the CML global community with a powerful evidence-based patient advocacy tool. Stay tuned for more information about #TFR4CML!
