Home Forums Chat What next for the Trust?

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    While we’re still getting on our feet as an organisation we’ve also started thinking about next steps once we’ve registered with the Charities Commission and the HMRC (so can start fund-raising). Ideas we’ve got include things like doing research with LFS families to see where the gaps are in services, starting a formal registry of LFS families and individuals so new trials can have a set of people they can approach easily. fund-raising for research projects etc.

    We’d be really interested to get opinions and ideas at this stage. If you’ve got some ideas please post them here so we can share and help set the direction of where we’re going!


    When you say new trials do you mean by external hospitals/ doctors? Could we get particular doctors on board and do our own trials? Eventually have our own facilities? (Although I am sure that would be way in the future!)


    There were two things I was thinking of.

    The first is that if some new treatment comes along – say some new form of gene therapy that can replace mutated TP53 with normal type – then how can doctors quickly set up a clinical trial with a reasonable population of patients? With rare conditions like LFS they could wait for years until they get enough people on board. But if there was a central registry then there’d be a ready list of people to contact to join the trial.

    Secondly, the emphasis on clinical research in LFS at the moment is on screening and surveiilance. That’s a start, but it’s not enough. In other genetic cancer pre-disposition syndromes, like Lynch Syndrome, they’ve moved to do trials of drugs to prevent sufferers from getting cancer in the first place. That’s what we need to be doing for LFS as a matter of some urgency. And for that I think we’ll need to push as a group to get things moving.


    I was reading something a while ago, not sure whether it was on this site or the anticancer site but it was about the way that certain chemotherapy drugs work and how some kind of use tp53 to help the process. I won’t even pretend to understand it properly but I did find it interesting that there might be some chemotherapy drugs which may not work or not work as well without the fully functioning tp53 so therefore some treatments might be ‘better’ for Li Fraumeni patients than others. As I say this was way too technical for my little brain but maybe more research into this type of thing or even a list of some kind which indicates which drugs might be ‘preferred’ for Li Fraumeni patients, it could even go as far as to suggest alternatives? I know a lot of cancers will have a treatment protocol specific to them but could these be tailored for those with Li Fraumeni?

    The thing that has got me thinking about this is that my son had Rhabdomyosarcoma, he has been off treatment for almost three years now but I am now wondering whether his treatment was the best it could have been. The protocol that was used to treat him included Radiotherapy at the end. It was decided not to have this because of the associated risks but this has meant that he has in fact had less/worse treatment than someone else with the same tumour but without LFS. We had never heard about proton therapy (which again I first heard about on this site) and it was never offered or discussed as an alternative to the radiotherapy which is something else that I am now struggling to deal with. As there was a full clearance during the operation to remove the tumour we thought, at the time, we were doing the best thing for his long term health by not having the radiotherapy but I now feel I have let him down by not knowing about or pushing for the proton therapy instead. Had we been offered or told about it I would have jumped at the chance to have it, effectively completing the treatment protocol but it now feels ‘unfinished’ if you know what I mean, it is obviously part of the protocol for a reason.

    Hope some of that made sense!!

    I definitely think a LFS registry is a good idea. As I am not under either Manchester or the Royal Marsden I wonder whether I would ever have found out about the SIGNIFY study had I not found this website. My geneticist had not seen the Canada/USA study until I asked her about it and it was through my questions about this that she found out about SIGNIFY. What if I had never asked??


    Sandra, here’s another way to think about your son’s treatment for his rhabdo – because he didn’t have radiotherapy it means there’s another trick up your sleeve if it ever recurs…

    My son, George, also had a rhabdomyosarcoma of the temporalis muscle. He was treated with chemo and after surgery we were told there were clear margins. But it recurred in just a few months. It’s an aggrressive disease and recurrence becomes less likely the further on you go. In our case when it did recur George was treated with second-line chemo, radiotherapy and more surgery. More than ten years later his new tumours were all in the area where he’d been treated with rdiotherapy (we didn’t find out that he had LFS until after the third primary cancer).

    Remember also that proton therapy is still a form of radiation, so although it’s much better than standard radiotherapy, it’s still not as good as no radiation therapy at all. So rather than thinking of his treatment as unfinished, I would view it as finished but you’ve still got room for manoeuvre if necessary in the future (heaven forbid!).

    Anyway, I like the idea of trying to list those chemo drugs that work better for LFS patients. I’m also really glad that you’ve found the site useful, sometimes it’s hard getting a handle on how useful a service we’re providing to people.

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