CML Advocates Network with the Rare Disease Day 2019
Chronic Myeloid Leukemia is a Rare Disease
What a Rare Disease is?
According to the European Commission, any disease affecting fewer than 5 people in 10,000 in the EU is considered rare. Although this might appear small, it translates into approximately 246,000 people.
Most patients suffer from even rarer diseases affecting 1 person in 100,000 or more. Approximately 5,000-8,000 distinct rare diseases affect 6-8% of the EU population i.e. between 27 and 36 million people.
In the USA, a disease is defined as rare when it affects fewer than 200,000 Americans at any given time.
The actual prevalence of rare diseases can vary between populations, making it difficult to provide a precise numerical definition
- 80% of rare diseases have identified genetic origins whilst others are the result of infections (bacterial or viral), allergies and environmental causes, or are degenerative and proliferative.
- 50% of rare diseases affect children.
Click here to check more information about Rare Diseases at Eurordis, Rare Diseases Europe.
UPDATE: Falsified Iclusig traded globally, discovered in Malaysia (new), Turkey, Switzerland, Argentina and Internet sales with UK/Ireland product batch numbers PR072875 (45mg) and 25A19E09 (15mg)
The World Health Organisation (WHO) has issued a medical product alert today that falsified versions of ICLUSIG (Ponatinib) have been discovered in Turkey, Argentina and Switzerland, and in Internet sales. The falsified (counterfeit) medicines do not contain any of the CML drug ponatinib, but only low quantities of paracetamol. These falsified medicines are not going to have any effect against CML. The falsified English language packs contain the lot/batch numbers
- PR072875 (UK/Ireland pack, English language, 60 tablets á 45mg, Expiry date 12/2019) and
- 25A19E09 (UK/Ireland pack, English language, 30 tablets á 15mg, Expiry date 10/2020).
In addition, on 20 February 2019, we were notified of a new falsified Iclusig pack:
- PR0834170 (German pack, German language, 30 tablets á 45 mg, Expiry date 06/2020).
The WHO page which is available in English, French, Russian and Spanish provides photos of the packages and pill bottles so patients can find the batch numbers. The WHO page has not yet been updated on the third falsified pack, which is expected those days.
To identify falsified product batches both the batch number and packaging (language, country of the pack) are important.
No UK/Ireland ORIGINAL product has been produced by the manufacturers Incyte/Takeda that carries those batch numbers in languages from those countries and with these languages. Products with the above batch numbers, country and language are most likely falsified.
In case of PR072875, the counterfeiters used this genuine lot number originally issued on a German ICLUSIG box and created a falsified UK packaging. In case of 25A19E09 and PR0834170, no original products were manufactured wih those batch numbers.
And so far, the manufacturer and medicines authorities are NOT aware of additional FALSIFIED ponatinib products bearing different batch numbers, so patients taking Ponatinib with other batch numbers than those listed above should NOT yet be concerned.
Patients, physicians and distributors should make sure to obtain CML medication from validated and reliable sources that are able to demonstrate the authenticity of origin of the products.
In case of any concern, check the batch number against the two identified falsifications, and contact Incyte Medical Information in case of persisting suspicion at eumedinfo@incyte.com or +800 00027423.
Please let the CML Advocates Network know via info@cmladvocates.net if you come across the falsified product with these batch numbers, so we know whether the falsified medicines have been shipped to any other regions or countries. National CML patient groups are also encouraged to notify their local health authorities and leading physicians.
Important Notice: Changes to the CML Horizons 2019 Conference
Important Notice: Changes to the CML Horizons 2019 Conference 
Dear Members & friends of CML Advocates Network,
This is a very important announcement and follows up from our notice a few weeks ago named "emerging news regarding CML Horizons 2019" following the tragic events that took place in Nairobi, Kenya.
After careful consideration, discussions and investigation the CML Steering committee has decided that at this point of time it is better for the safety of our members to change the location of CML Horizons 2019 and at such short notice we will need to come back to mainland Europe. Horizons will now be held in Lisbon, Portugal at the Lisbon Marriott Hotel, Avenida dos Combatentes 45, Lisbon 1600-042 Portugal and the dates still remain the same 17-19 May 2019.
This does not mean that in future years we will not take Horizons to another continent and we will work hard to try and make this happen for 2020 and we will keep you updated.
What does this change of location mean for all our members? We will need you to start registration ASAP and depending on the country you come from if you need a Schengen Visa this process can take 6-9 weeks so we need you to take the following steps.
Step 1: Register for CML Horizons 2019 here: https://www.cmladvocates.net/cmlhorizons
Step 2: At the following link is a list of the nationalities that must process a Visa: https://www.vistos.mne.pt/en/short-stay-visas-schengen/general-information/who-needs-a-visa#countries-2
Step 3: Once you have registered, we will be in contact with you in regards to support documents for your visa application.
For those members who have already registered: Vanja and Lidija will be in touch with you soon and there is NO NEED to re-register. For those who need a VISA we will need to send you new documents for a Schengen Visa to Portugal and new flight itinerary.
Finally, please note that this decision did not come easy and does not take away from why we had chosen Nairobi as our original destination and that is to honour our dearly departed Ferdinand Micho who was the leader in the fight of CML in Africa and the Middle East but also for the challenges that CML patients face in law and middle-income countries in access to treatment and monitoring.
We look forward to seeing you all soon and please should you have any questions at all please do not hesitate to contact us.
Warm Regards
Giora and Lidija on behalf of the CML Advocates Steering Committee
CML Advocates Network
Falsified Iclusig traded globally, discovered in Turkey, Switzerland, Argentina and Internet sales with UK/Ireland product batch numbers PR072875 (45mg) and 25A19E09 (15mg)
The World Health Organisation (WHO) has issued a medical product alert today that falsified versions of ICLUSIG (Ponatinib) have been discovered in Turkey, Argentina and Switzerland, and in Internet sales. The falsified (counterfeit) medicines do not contain any of the CML drug ponatinib, but only low quantities of paracetamol. These falsified medicines are not going to have any effect against CML. The falsified English language packs contain the batch numbers
- PR072875 (UK/Ireland pack, English language, 60 tablets á 45mg, Expiry date 12/2019) and
- 25A19E09 (UK/Ireleand pack, English language, 30 tablets á 15mg, Expiry date 10/2020).
The WHO page which is available in English, French, Russian and Spanish provides photos of the packages and pill bottles so patients can find the batch numbers.
To identify falsified product batches both the batch number and packaging (language, country of the pack) are important.
No UK/Ireland ORIGINAL product has been produced by the manufacturers Incyte/Takeda that carries those batch numbers. - so products with the above two batch numbers in UK/Ireland and English language are most likely falsified. In case of PR072875, the counterfeiters used this genuine lot number originally issued on a German ICLUSIG box and created a falsified UK packaging.
And so far, the manufacturer and medicines authorities are NOT aware of additional FALSIFIED ponatinib products bearing different batch numbers, so patients taking Ponatinib with other batch numbers than those listed above should NOT yet be concerned.
Patients, physicians and distributors should make sure to obtain CML medication from validated and reliable sources that are able to demonstrate the authenticity of origin of the products.
In case of any concern, check the batch number against the two identified falsifications, and contact Incyte Medical Information in case of persisting suspicion at eumedinfo@incyte.com or +800 00027423.
Please let the CML Advocates Network know via info@cmladvocates.net if you come across the falsified product with these batch numbers, so we know whether the falsified medicines have been shipped to any other regions or countries. National CML patient groups are also encouraged to notify their local health authorities and leading physicians.
Emerging news regarding CML Horizons 2019
Emerging news regarding CML Horizons 2019
Dear members and friends of the CML Advocates Network,
You are probably aware of the terrible attack that took place yesterday by terrorists in the Dusit hotel in Nairobi.
These are terrible and sad news indeed. Our thoughts are with the lost innocent victims and their families. We live in a crazy world with crazy people. Terror is taking lives in almost every country and it is so difficult to understand and accept. We must not react rashly, and will not make a decision based on emotion, but rather consider the alternatives and then make a decision.
The steering committee will now study the facts and the extent of the sad event and will discuss the implications of CML Horizons in Nairobi in May.
Meanwhile, we are not stopping registration as we already have more than 20 delegates registered, but we are stopping any activity that increases our costs and commitment like we will not book more flights until the Steering committee discusses the issue and makes a decision.
We are in constant touch with our local member organization, Henzo Kenya, and with our logistics agency Safari Trails to better understand the Situation in Nairobi and all safety steps taken now and in the near future.
We are evaluating the situation and we will come back to our members with more information and the final decision as soon as possible within 2 weeks.
We must decide what is best for Horizons and for all our members and delegates. We will put the safety of our participants at the top of our priority.
It is a sad day.
With our hopes for a better future
Giora and all the CML Advocates Network steering committee members.
CML Horizons 2019 Registration Now Open - 17th to 19th May 2019
Register here - Preliminary Program - Conference fee - Scholarship Application
Call for Poster - Call for Nominations Travel - Visas - Venue & Accommodation - Acknowledgements
Registration is now open - Join us for the 17th “CML Horizons: Learn. Share. Grow" Conference
DATE: 17th to 19th MAY 2019
LOCATION: Lisbon, Portugal
Due to events beyond our control and with sad regret we have changed the location of this year's Horizons. Even though location has been changed to Lisbon, Portugal this does not take away from why we had chosen Nairobi as our original destination and that is to honour our dearly departed Ferdinand Micho who was a leader in the fight of CML in Africa and the Middle East but also for the challenges that CML patients face in low and middle-income countries in access to treatment and monitoring.
We have secured top speakers from Western countries like Dr. Francois Mahon & Dr. Gianantonio Rosti, also African and Asian countries such as Dr. Qian Jiang from China, Dr. Abinya from Kenya and Dr. Benneh from Ghana. More speakers are expected to confirm soon.
The CML Horizons conference is dedicated to the development of our members as patient advocates and deals with topics such as: - What is the goal of CML Treatment? Improving access, Key initiatives in CML treatment & advocacy and Living with CML and the future outlook. Dealing with these types of questions is the essence of Learn. Share & Grow and our conference offers the opportunity to support and facilitate the quality of education. This one of a kind conference also provides opportunities to be able to exchange ideas, interact & share best practice and provide a platform to start networking across borders, build alliances and so much more for you to bring back to your own organization.
We very much hope that you will be able to attend the 17th conference of its kind - with an exciting program on CML advocacy and CML science.
Our conference will provide you with knowledge on how to make a difference to the lives of patients, identify opportunities in patient support & advocacy and learn from the experts - both patient advocates and physicians by networking opportunities with CML advocacy leaders around the globe.
CLICK HERE TO REGISTER
Welcome, Ameleg, Mexico! Now 120 patient organisations from 90 countries in the CML Advocates Network
We are proud to welcome a new member organisation to the CML Advocates Network: Asociacion Mexicana de Leucemia y GIST, A.C. based in Mexico.
The CML Advocates Network now totals at 120 member organisations in 90 countries worldwide!
See the list of CML groups in the Network on our world map.
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.]
