ASH 2017- personal report, impressions and summary

Jun 4, 2018

– Written by Sharf Giora, CML Advocates Network –

ASH 2017 is the 10th ASH conference I am attending in a raw, and I am being asked many times why do I like this conference and travel such a long distance to attend it every year.

AshGiora1The main reason that I, as a patients representative, leave home and travel to ASH is to hear and learn about the last innovations in the treatment of blood cancers and bring these news to patients in my country and across the globe, to empower them with knowledge, and mainly hope. that research in the various diseases is ongoing and if they are in a need for a new treatment, maybe it is just around the corner. The second reason why I go to ASH, which is not less important, is that this is the best place and opportunity to meet in a short time all the stakeholders that I am working with, like Drs and scientists from Israel and the globe, Pharma representatives from medical and patients relations, and other patients advocates, and to discuss with all of them how to continue and strengthen our collaboration.


– Giora and Nadav, a board member of

Flute of Light at ASH 2017  –

59th ASH Annual Meeting & Exposition

The ASH meeting which takes place at the beginning of December each year is the most important Hematology scientific meeting in the world, and it brings together about 30,000 Hematologists, researchers, clinical development leaders and also few dozen patients advocates. This is the place where the latest news about clinical trials results, and new treatments and directions are published for the first time.

Ash GioraSharf CMLThe conference is Huge and consists of thousands of sessions and presentations. It always impresses me to see the thousands of Drs Running from one session to another in a very big conference centre, where getting from one side to the other can take 15-20 minutes of walking.

This year the conference took place in the Southern part of the USA, Atlanta, but due to extremely stormy weather with 3 days of snow, the city was taken by surprise and the feeling was we are in Northern Canada. The airport was closed for a couple of days which prevented from some of the participants to arrive as planned.

– Israeli researcher presenting her poster –

The conference program is built from 3 main types of sessions: The first one is the Educational session in each disease area where the 3 main important topics are presented.The second is the oral presentations where 6 important abstracts are presented for 15 minutes each, on topics carefully chosen by the conference committee, and the last one is the Poster sessions where each day hundreds of researchers are presenting a poster on their work according to disease type.

Messages from ASH 2017

One of my main takeaway messages from ASH was, not for the first time, that Medicine is not Math, and there are different opinions and approaches among the experts on various topics. There isn’t always right or wrong. I saw it in the discussion about TFR, where some experts say that MR4 is enough to stop CML treatment and others claim that MR4.5 is required.

Another example was after Dr Delphine Rea presented their observational study on reducing Nilotinib dose and changing to once daily regiment, (discussed below), and other experts were very much against this concept claiming it is even dangerous for patients. We, as patient’s representatives, can only stand and listen to all sides and try to understand the logic behind before we make up our mind.

CML topics at ASH 2017

Here are some of the most important CML topics and news I have gathered from presentations and posters at ASH 2017:

Nilo-Red real life observational study- Molecular responses of CML patients after being switched from twice daily NIlotinib to once daily reduced dose- presented by Dr Delphine Rea, France

The standard dose for newly diagnosed CML patients treated with Nilotinib is twice daily 300 Mg and in 2nd line, it is twice daily 400 mg. Dose reduction is recommended only for patients with significant side effects. Since patients taking Nilotinib need to fast 2 hours before and 1 hour after taking the drug, this makes it quite difficult for the patient to adhere to treatment.

Ash GioraSharf CMLThe goal of this observational study was to check if patients will maintain their MMR after switching to a more convenient reduced dose and only once daily regimen. The study was done in 2 centres in Paris and Bordeaux and included 82 patients who were switched to once daily, reduced the dose of Nilotinib, mainly 450 Mg. The median follow-up time for the whole cohort since dose reduction was 25 months (0-73). 71 patients have followed up of at least 12 months and none of them experienced disease progression. 2 patients lost MMR after 4 and 6 months and continued to take the reduced dose, and regained their MMR spontaneously.

– New CML management by Dr Delphine Rea –

In most patients, molecular responses continued to improve after the dose reduction and switch to once daily dosing. 15 patients stopped treatment of Nilotinib after the dose reduction, and after 19 months only 3 lost their MMR and restarted treatment.

Dr Rea concludes that switching CML patients who achieved optimal molecular responses on twice daily dosing, to a reduced dose once a day is feasible. The anti Leukemic efficacy of the reduced dose is maintained and molecular responses continue to improve for most patients. This study paves the way for additional studies aiming at a more convenient regimen of Nilotinib for patients and avoiding overtreatment with 2nd generation TKI. At the end, Dr Rea presented a new possible way to treat newly diagnosed CML patients. Start with full dose TKI to achieve optimal response induction. Then lower the TKI dose for optimal response consolidation. Patients who don’t achieve DMR continue for life low dose and those who achieve DMR attempt TFR.

GS note- As I mentioned above this is only an observational study from real life and not a planned comparative clinical trial, and the data is not enough at this point to base dose lowering decisions. Patients must consult their Drs before taking any action in regards to changing treatment does.

PF-114 – new 3 ed generation TKI of BCR-ABL

Dr Jorge Cortes from MD Anderson presented data on a new compound in clinical trial, with similar activity and structure like Ponatinib. The compound was designed to avoid inhibition of numerous off-target Kinases and thus avoid life-threatening side effects. In a Phase 1 trial, 24 heavily pre-treated patients were recruited out of which 11 had the T315I mutation. The compound exhibits anti-leukemia activity in this difficult patients population including the ones with the T315I mutation. The side effects profile seems to be safe with only one patient developing grade 3 rash. Unlike Ponatinib, no cardiovascular events have been observed. A Phase 2 multicenter international study is planned for 2018.

Bosutinib Vs Imatinib for newly Diagnosed CML – the BEFORE Trial- 18 months follow up- presented by Dr Carlo Gambacorti

Dr Gambacorti from Italy presented this 2 arms study- in one arm 246 patients received Bosutinib as first-line treatment and in the comparative arm 241 patients received Imatinib. Bosutinib showed higher efficacy than Imatinib and after 18 months follow up, 57% of patients on Bosutinib achieved MMR compared to 48% on Imatinib. The better responses were also observed for MR4 and MR4.5. Achieving a PCR of lower than 10 % after 3 months of treatment initiation is associated with better long-term outcome. In this trial, 75 % of the patients on Bosutinib achieved this goal compared to only 57% on Imatinib. Dr Gambacorti concludes that Bosutinib is a good first line treatment option for newly diagnosed CML patients.

GS comment- following this study the FDA has approved on 20.12.2017 Bosutinib as a first line treatment for newly diagnosed CML patients. We now have, at least in the US, 4 options for 1 st line treatment- Imatinib, Nilotinib, Dasatinib and Bosutinib.

Preliminary experience of Imatinib after Nilotinib in first-line treatment of CML

This study of a French group was presented at the CML poster session on Sunday. The study included 74 patients who started Nilotinib as first-line treatment and achieved a full Cytogenetic response. 10 patients were switched to Imatinib for comparison with the rest of the patients who were left on Nilotinib.

The preliminary results indicate that there is no substantial difference in maintaining the response between the patients who stayed on Nilotinib and those switched to Imatinib, and in both groups, the molecular response continued to improve.

Since about 40% of newly diagnosed CML patients suffer from metabolic and cardiovascular comorbidities, a long-term Nilotinib treatment might be an issue for them. The deeper and faster response achieved on Nilotinib as first-line compared to Imatinib makes the option of giving short-term Nilotinib induction treatment, reaching a good molecular response and then switching to maintenance Imatinib treatment, an attractive option for this group of patients and will also allow a future possibility of stopping treatment.

ASH GioraSharf CML

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