Hello All,
 
As promised here is part II
 
Before getting into part II I did want to mention another milestone that was celebrated at our meeting.
 
We all signed a card and celebrated Suzan McNamara's birthday.  A significant date for us all - since it was also the date 6 years ago that Suzan received the word from Novartis regarding the petition.
 
Dr. Laneuville spoke to us about a CML registry that has been approved for Canada.  This will be quite helpful to all of us as information on every patient will be entered into the database - blinded of course.  All sorts of information will be tracked, for instance a Hem/Onc could query the data for all patients with low Hgb to determine if it is of any prognostic value.  As you can imagine lots of information could be tracked and queried.  Hopefully we'll hear more about this in the future.
 
Now for Dr. Ravandi's part of the talk.
 
Most of my notes are from the oncology conference he spoke at here in Montreal the day before meeting with us, but then again, he presented much of the same thing.  If anyone who attended wants to add anything here, please feel free to do so.
 
Dr. Ravandi also covered the history and origin of CML, and presented more data from the IRIS trial and the from information at MDACC.
 
Dr. R. spoke about BCR ABL:
It is a tyrosine kinase oncoprotein
Auto and substrate phosphorylation
Highly plastic structure
"open" active versus "closed" autoinhibited state ( IM binds only to the active form)
State regulated intra-and extra-molecularly
 
In speaking about Gleevec - Dr. R. said:
other kinases inhibited by IM are:
ABL
Bcr-Abl
PDGFRβ
c-Kit
 
IM in the Phase I trial
 
With 25-85 mg/day - only 1 patient out of 10 achieved a CHR (complete hematological remission - meaning blood counts were alright)
140-250 mg/day- 10 patients out of 19 (53%) achieved CHR and 2 patients out of 19 (11%) achieved CCR
300-1000 mg/day - 53 patients out of 54 (98%) achieved CHR and 29 (54%) patients out of 54 achieved cytogenetic response 
17 (31%) patients of the 54 achieved Major cytogenetic response
No MTD (maximum total dose/day) was reached.
 
He also covered the results of the IRIS trial, which was covered already by Dr. L.
 
Dr. R. presented data on survival with IM in newly dxed CML comparing results with IRIS to MDACC
 
IRIS had 553 patients and 51 months out - ~ 95% were still alive
MDACC had 247 patients and ~ 98% were still alive.  
 
He showed data that was presented at ASH last year showing BCR-ABL levels continue to decrease with IM therapy in CP CML (28 pts on IRIS study Rx with IM 400 mg/d
 
12 mos median log reduction was 3.0 and 46% had a 3 log or better and 4% had a 4 log or better reduction
24 mos median log reduction was 3.4 and 68% had a 3 log or better and 7% had a 4 log or better reduction
36 mos median log reduction was 3.9 and 71% had a 3 log or better and 32% had a 4 log or better reduction
42 mos median log reductions was 4.2 and 71% had a 3 log or better and 54% had a 4 log or better reduction
 
Looking at IM 400mg versus 800 mg (presented at ASH 2004 also)
Of 175 patients chosen for the higher dose, 156 achieved Complete cytogenetic response
of the 50 patients chosen for the lower dose 39 achieved CCR
 
It is important to note that this was not a randomized trial.  That is why the Gleem trial is happening in Canada, as a randomized trial is needed to prove the higher dose strategy/theory.
 
The RIGHT study looked at various doses of IM  - 300mg, 400mg, 600mg and 800mg
 
It had the following endpoints:
Primary:  Percent of patients achieving molecular response
Secondary:  time to hematologic response, cytogenetic response, and complete molecular response
 - percent of patients with undetectable BCR-ABL transcripts at 12 mos
 - Safety
 
Interestingly this study also showed the level of non compliance (as it were) in that it showed actual dose of IM over time.  In the first 3 mos all patients were completely compliant at 3 mos - 36 patients were still at 800mg, 8 were at 600mg and 3 were at 400mg, by 9 mos, 15 were at 800mg, 5 were at 600mg and 5 were at 400mg, by 12 mos, 7 were at 800mg, 1 was at 600mg and 3 was at 400mg.
 
The results of the trial showed that 7 of the 11 patients who stayed at the 800mg dose achieved either a 3 log reduction or better and all 7 also achieved undetectable. So I guess there is something to be said about compliance!
 
He spoke about mechanisms of "resistance" to IM (Bcr-Abl mediated or not)
 - Patient not taking drug
- ABL point mutations going to conformation change of ATP binding site or modulators
- Increase in BCR-ABL, showing BCR-ABL kinase activity
- Metabolic, PK, drug interactions MDR clonal evolution
- ? other kinases - VEGF, mTOR, src
- CML no longer the main problem
 
Speaking about AMN107:
 
Showing the molecular structure he stated that AMN107 has a better interaction between ABL binding - improves the affinity of BCR-ABL to bind with AMN107.
 
The conclusions from the Phase I trial of AMN107 are:
Hematologic response rates >50% in  AP and BP
Cytogenetic responses in AP and BP
No DLT (dose limiting toxicity) found
    Well tolerated up to 1,200 mg daily
     Rare liver, skin, or marrow AE's (adverse events)
 
He spoke about Dasatinib - reviewing Talpaz's work presented at ASH last year:
 
Of the 10 patients in BP, 50% achieved CHR, 80% achieved MHR
Of the 32 patients in AP/ALL 28% achieved CHR, 69% achieved MHR
 
In the same group of CML AP/BP the CG response was as follows:
AP - 40% achieved CyR, 30% achieved CCyR, and 10% achieved PCyR
BP - 56% achieved CyR, 10% achieved CCyR, and 16% achieved PCyR
 
Of the most commonly reported non hematologic responses:
Elevated ALT (28%)
Elevated Creatinine (23%)
Diarrhea (18%)
Paresthesia (10%)
Headache (10%)
Nausea (5%)
Peripheral edema (5%)
Pleural Effusion (3%)
GI Hemorrhage (0)
These were of low grad 1 - 2
Note:  we have heard more about pleural effusion in the current trials, so it will be interesting to see how this gets updated at this years ASH.
 
He spoke about other things in the works such as Vaccines and things, but only briefly so.  I think I'll get much more information at ASH.
 
We finished off by presenting Dr. Ravandi with a Moose so he could tell all his friends he went Moose hunting in Canada.
 
I have to tell you this though, as I mentioned he had presented to a bunch of oncs the day before, he mentioned he got much better questions from our patient audience than from the docs that he presented to.  He was very impressed with our level of knowledge.
 
We thoroughly enjoyed his company.
 
Stay tuned for big news on more meetings coming in the future.
 
Cheers,
Cheryl-Anne 
 
 
 


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