A cure?

In November Nic and I went up to the 7th annual Patient and Carer CML conference organised by CML Support. We attended with a mixture of trepidation and with a complete lack of expectation. We had no idea what we'd find and to be honest we feared it would be a big organised hug, full of people 'supporting' each other.

What we hadn't expected was the high level of engagement from the specialists in the area, with all the lead consultants in the area from the UK there and a couple from France and Italy, as well as the 3 main pharmaceutical companies involved in production of the three main drug therapies now used to fight the disease.

Thankfully it was all consultant led and while the drug companies were largely funding it, there was no overt manipulation of agendas. In short the three companies do not technically compete against each other, but rather there is a lead drug and two other different drugs that will combat different strains of the disease's mutations when and if they occur and become resistant to Imatinib (developed by Dr Brian Drucker - or God as CML patients like to call him - at Novartis) which is very much the lead. A little about Imatinib and it's wonder drug status is contained here.

Anyway, the day was frankly exhausting. It was a series of half hour lectures over 10 hours with a cut down 45 minute lunch break and couple of quickly snatched tea breaks. Not that I'm complaining the whole thing was fascinating and everyone found it so, hence the fact that we had to cut down the breaks and also overran on the room booking by 2 hours. They went into extraordinary medical and molecular biological detail and unfortunately most of the real brain twisting stuff came towards the end of the day, but we learnt an amazing amount. Not least that the word 'cure' has begun to be uttered - in very hushed tones let's be careful about this.

Again I'll try and explain this in very simplistic terms.

There is strong evidence after 7 years on the market that Imatinib can ensure that people not only reach full remission but stay there. By that I mean it is undetectable by any test that is available currently. Note any test currently, because they know that they are currently unable to test below a certain level (and therefore cannot say this is a medical cure), however they have so far been perfectly happy to accept this as a zero score.

There is precedent however of people coming off Imatinib for sustained periods when in remission and then staying there. The most common reason for this is in women who wish to become pregnant. A clssic example is Erin Zammett Ruddy an editor of Glamour Magazine in the States who when diagnosed started a column called 'Life with Cancer'. Erin came off Imatinib for a period during 2006/7 while she was trying to get preganant and then for the full term of her pregnancy a period of around 13 months. During that time she did not come out of her state of remission.

This is a story that appears to be increasingly common amongst patients who have achived remission fairly quickly and simply and remain resistance free. This in turn has started to raise the possiblity that Imatinib may actually be a cure.

In order to understand this further we need understand that Leukaemia is essentially a disease of the stem cells. In each active stem cell parts of two chromosomes (the 9th and 22nd) switch places. As a result, part of the BCR ("breakpoint cluster region") gene from chromosome 22 is fused with the ABL (the Abelson proto-oncogene)gene on chromosome 9. Imatinib effectively acts as an inhibitor to stop this process occurring and therefore proliferating and in fact it kills them off hence the move towards remission.

Now what the specialists don't know is whether this process occurs to the extent that the mutation stops completely and there are of course many factors to take into account, the main two in mind being as follows:

1. Do I have a pre-disposition to the chromosal mutation i.e. even if I get rid of every single leukaemic cell will it matter if my body just wants to continue operating in the wrong way.

2. What about dormant stem cells? Stem cells lay dormant for 20 years and because no one can actually read anything from a dormant stem cell and Imatinib does not effect these cells either we have no idea whether these cells are effected by the leukaemia, i.e. when they finally come to life will they be mutated or will they not have been effected because they were dormant. It's a great unknowable.

However they have started to do some trials in Scandanavia to test the cure hypothesis. Volunteers are coming off the treatment having been in full remission for a period of time to basically 'see what happens'. It's a fairly low risk strategy as it has been proven that those that have achieved remission quickly and come off for a period only for the leukaemia to return do in fact achieve remission again just as quickly when they return to the medication.

So there you have it, who knows eh. If Dr. Brian Drucker has found a cure for Cancer I can't think a nobel prize for medicine can be far away.