CF Foundation | Preclinical / New Technologies



preclinical research is the research that goes on before a new therapy ever reaches people with cystic fibrosis there is a lot of work that goes on way before you do a clinical trial all things to really determine could this drug potentially help people with CF and is it safe preclinical work at the CF foundation is particularly exciting right now and that's because some of the new therapies they're going to be transformative for down the road are now in the lab and we're seeing very exciting results some of these are CFTR modulators modulars which correct the underlying CFTR problem and are going to make a difference and transform CF in the upcoming years it's amazing when you look at the results of some of these preclinical studies they are significantly stronger than our current therapies and what this is going to do is it's going to make a bigger difference for those people taking modulator zhh but also increase the number of people and the number of different mutations that can benefit from these therapies another area where there's a large amount of preclinical work going on right now is an area of stop mutations these are harder to treat mutations that may not respond as well to CFG our modulators so preclinical work right now is trying to discover new therapies which will allow the stop mutations to be read through and to go ahead and make full protein we're also focusing quite a bit on other areas that we know need work these are new antibiotics new anti-inflammatories as well as new therapies which address mucociliary clearance and help clear mucus and make a difference for people with CF because we realized drug-resistant bacteria is an important topic for many people with CF we're doing research into trying to better understand why bacteria becomes resistant so this is basic science research in the lab we're screening for new compounds so we have a lab where we have samples of difficult-to-treat bacteria we have CF sputum we use these to actually test some of these new therapies to say which of these might be most effective against difficult to treat bacteria and then the last part of preclinical work is a particularly exciting one these are the the treatments of tomorrow these are things like gene editing gene therapy stem cells which aren't ready for primetime yet but if we start working on them now they're going to make a difference for people with CF in the future the reason that being involve a cystic fibrosis research is so great is because you know the things you're working on aren't just to try to promote a theory or something that would just help people understand but it's going to make a difference for people with cystic fibrosis we've seen that in just the last few years or one day you're working in the laboratory on a research project the next day you're going into clinical trials the following day you're seeing patients benefit from the therapy there's nothing better you

One Comment

  1. Oh no no said:

    First.

    Cf… Good luck on finding a cure

    June 30, 2019
    Reply

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