107 pointsby giuliomagnifico3 hours ago7 comments
  • jawns2 hours ago
    I have been a supporter of Cures Within Reach, a nonprofit that focuses on repurposing drugs, especially for rare diseases. https://www.cureswithinreach.org

    They have funded some important repurposed-drug studies for Huntingtons Disease, which runs in my family. For a disease like this, it's never going to make sense for major pharmaceutical companies to invest the effort to develop entirely new drugs, but by repurposing existing drugs, it gives people living with rare diseases a chance to ease symptoms.

    • petesergeant3 minutes ago
      > For a disease like [Huntingtons], it's never going to make sense for major pharmaceutical companies to invest the effort to develop entirely new drugs

      This is ... not correct.

      Roche, Regeneron, and Novartis all have novel HD drugs under development in tandem with smaller labs (Ionis, Alnylam, and PTC respectively), and then smaller labs like uniQure and Wave Life Sciences do too. Novartis have already dropped $1bn on the partnership with a committed $2b more. In addition, there are a bunch of incentive schemes for diseases like HD: both the FDA and EMA have offered orphan-drug designation to therapies for HD, the FDA does expedited programmes and can offer RMAT designation for drugs like AMT-130.

    • dmichulke2 hours ago
      I'm on my cell phone and I couldn't figure out what diseases can be treated on the site. I'd say it's donor focused and not patient focused
    • unsupp0rtedan hour ago
      Have you looked into UniQure / Clearpoint Neuro ?

      Exciting stuff, if it gets FDA approved.

  • functionmousean hour ago
    Related, one of my all time favorite articles: https://www.propublica.org/article/revlimid-price-cancer-cel...
  • oezi3 hours ago
    Such studies are great but there is no regulatory pathway to extend the use of existing drugs for new indications of use without the consent of the manufacturer (or becoming a manufacturer yourself).

    This means such studies can give more clarity on which off-label use is beneficial but it can't be an officially allowed usage.

    • intrasight2 hours ago
      There tens of millions of people being treated off-label.
      • oezian hour ago
        The doctors are assuming liability or let the patient sign waivers, which I guess is fine. But the lack of a pathway prevents a lot of commercial possibilities. For instance it is almost impossible to build a business around supporting off-label use cases (for instance selling necessary accessories).
        • jnovek42 minutes ago
          I have some difficult-to-treat medical issues and about 1/3 of the handful of pills I take every day are off-label. I think when you get into less common medical concerns off-label use becomes much, much more frequent.

          E.g. gabapentin is an anti-seizure medication that has been found to reduce neuropathic pain as well. It has shot up to the 5th most prescribed medication in the last decade as it has replaced long-term opioid use for new chronic neuropathic pain patients. This is 100% off-label and is prescribed by everyone from NPs all the way up to neurosurgeons for this purpose.

          I’ve never been asked to sign a waiver and I can’t imagine that for-profit hospitals would allow their doctors to prescribe off-label medications willy-nilly if they represented big liability. (I don’t know this for sure, this is just what my experience implies.)

        • Aurornis44 minutes ago
          Off label prescribing is extremely common.

          This isn’t a new or novel concept. Doctors manage patients with off-label prescriptions all day long.

        • forgetfreemanan hour ago
          The business is already built given the drug in question is already being manufactured and available on the market.
      • uberexan hour ago
        Can confirm. LDN user here.
        • cjbgkaghan hour ago
          Also a LDN user, that drug saved my life. I bought it from a shady dude online, the initial effect was so strong that I thought they shipped me meth instead - that wore off after a week but the lifting of the brain fog persisted.
          • jnovekan hour ago
            It didn’t have that sort of immediate, intense effect for me (though yours isn’t the only account like this that I’ve seen) but I’m still happy with the outcome; it dropped my average 1-to-10 pain score by about two points at three months.
            • cjbgkagh17 minutes ago
              It is a rare side effect, I helped a bunch of people (~50) get LDN, primarily for LongCovid, and only one other had a similar ‘too much energy’ effect and both of us have multiple TNXB SNPs and a very similar rare personality profile. I think the brain is so starved for dopamine at that point that it becomes hyper sensitive. I do miss the extra high energy but I also know that isn’t maintainable and I try to err on the side of caution.

              I’ve had ME/CFS my whole life but the third covid vaccine shot sent me to new lows, to the point LDN just wasn’t cutting it anymore. These days I take a combo of modafinil in the morning and amitryptiline at night. And low dose ozempic has been super helpful as well. I was researching GLP1As prior to the current craze because I was worried about hyper sensitivity so I waited for more data before trying, I started at 1/100th the normal starting dose and still got temporary gastroparesis. These days I take a more regular dose of 1mg/wk but it also seems that my body has largely normalized as the hypersensitivities have worn off. Probably a good sign that I’ve successfully addressed actual deficiencies.

    • vidarh2 hours ago
      The article points out that it is typically after patent expiry that the universities and hospitals start looking at repurposing.
      • oezian hour ago
        The patents aren't hindering off-label use. The hinder commercial exploitation by others.
        • vidarhan hour ago
          Yes, but that isn't relevant to what happens after the type of trials described in the article, which tends to happen when the patents are no longer relevant. As the article points out, while the drugs are patented, the manufacturers themselves are generally very interested in repurposing because it broadens their market. The article is discussing trials that mostly happens once the patents have expired.
    • ktallett3 hours ago
      This isn't completely true at least in the UK. It is simply that the manufacturer is no longer responsible legally. The GMC allow prescibing of unlicensed meds. However the change needs to be made to the pathway. So many issues in the NHS has been due to pathway problems.
    • boxed3 hours ago
      That seems wild. Do you have a citation to back that up? And in what country/countries?
      • amanaplanacanal2 hours ago
        I'm in the US, and there are a couple of wrinkles to this: insurance typically won't cover off label use, so patients end up paying the full cost of the medication, and if there is a big enough market, I believe companies can patent the new use even if the patents for the original use have run out.

        Doctors here are allowed to prescribe them though.

        • Aurornis37 minutes ago
          > insurance typically won't cover off label use

          I’m in the US. This is not true.

          Insurance will have prior authorization rules for certain drugs that are expensive that require the doctor to submit documentation of the condition, but in most cases the common medication is simply covered if prescribed. The insurance company does not receive documentation of every condition for every prescription to determine if the prescription is on label or off label.

          Insurance companies can and do also support some off label treatments that are commonly used under their prior auth requirements.

          I don’t know why there are so many comments in this thread making confident assertions that off label prescribing or insurance or so uncommon. This happens all day long at doctors offices and pharmacies.

        • bonsai_spoolan hour ago
          > I believe companies can patent the new use even if the patents for the original use have run out.

          This is not true

          > insurance typically won't cover off label use

          Generally not true but it can be the case, especially for expensive medications

  • turtleyacht3 hours ago
    How do people needing (and willing to risk) treatment hear about repurposing studies?
    • Aurornis16 minutes ago
      Specialists keep up with the latest research in their domains and talk with other specialists in their field. They usually know about these things before their patients discover them spontaneously.

      You can read a lot of published papers on PubMed by searching for a condition or drug. You should be aware that there are a lot of papers published that say disease A can be treated with drug or supplement B that fail to replicate if anyone else tries it. It takes some practice to recognize when a treatment represents a real trend as opposed to a single quack doctor looking for a status boosting publication with some questionable claims.

    • giuliomagnifico3 hours ago
      I think in the same way they participate in other clinical trials.
    • astura3 hours ago
      Big medical systems will post their clinical trails quarterly or so. If you have a big medical system near you you can sign up for emails. I imagine universities would have a mailing list too, if they regularly did clinical trials.

      You can also search here: https://clinicaltrials.gov/

      I was a clinical trial participant once and it was a positive experience for me.

    • bell-cot2 hours ago
      Communities (on-line or off-line) of people who share your disorder, and maybe health care professionals who see many patients with your disorder.
  • xenophenes2 hours ago
    fascinating! I'm sure there's quite a bit that can be learned through appropriate research - pathways to solve problems that haven't been thought of before
  • ck22 hours ago
    the thing is while something is better than nothing, new drug development is critical

    there is absolutely no cure for certain types of long-covid and me-cfs right now

    no repurposing any drug is going to cure it, they've tried everything after six years

    it will take a decade to have anything even in the pipeline and won't emerge from the USA because all medical and science research investment by the government has been destroyed by Russell Vought and Heritage Foundation

    JAK-STAT inhibitors will be a big treatment, not a cure, but they cost thousands per month in the USA because generics aren't allowed

  • shevy-javaan hour ago
    The prices of drugs in the USA are especially high. This is interesting because the USA claims to pursue a maximum capitalistic society - but if this were the case, you'd have competition in a free market. But you don't have that. You have a cartel (or rather more than one).

    A pure capitalistic society works on assumptions that are not real. People are often cheaters. This would have to be taken into account. But when you have an orange Al Capone in charge, it is pillage day. Even before the orange King you had heavily overcharged prices in the health care system. You need to realise that you have a mafia in charge that does not want to change this system. Why kill the cow that you can milk for free?

    • wk_endan hour ago
      > This is interesting because the USA claims to pursue a maximum capitalistic society

      No it doesn't. This is silly.

      Drug prices in the US are high for non-generic drugs because patent law gives the patent holder an artificial government-granted monopoly, which is blatantly not "pure" or "maximum capitalistic".

      Generic drugs - where the free market does apply - in the US are as cheap or cheaper than in other countries. See [0]:

        U.S. prices for brand-name originator drugs were 422 percent of prices in
        comparison countries, while U.S. unbranded generics, which we found account for 90
        percent of U.S. prescription volume, were on average cheaper at 67 percent of
        prices in comparison countries, where on average only 41 percent of prescription
        volume is for unbranded generics.
      
      [0] https://pmc.ncbi.nlm.nih.gov/articles/PMC11147645/
      • none25854 minutes ago
        I believe you two are arguing the same thing. Maybe the poster could have better worded it "general thought among most people is that the USA ..."

        Because you are both absolutely right.

    • Aurornis32 minutes ago
      > This is interesting because the USA claims to pursue a maximum capitalistic society

      I don’t know why you think this. The US is not a maximally capitalist society. The reason drug prices are so high is due to regulations restriction who can manufacture them due to government-granted temporary monopolies through patent law.

      If the US was maximally capitalist it would be a free for all with no patent protection.

    • bluGillan hour ago
      The US is not a capitalist society, it is a liberal society. Capitalism is a consequence of that but it isn't the reason.
    • MrBuddyCasinoan hour ago
      Look at the development of price and quality of something that is outside the regulated medical system, like eg Lasik, and everything within that system. Its like night and day.

      If we had proper competition and price discovery, things would be much better.

      • Geeekan hour ago
        On the other hand, dental work, especially anything above basic filings, are prohibitively expensive. I'd say Lasik is the exception, not the rule.
        • MrBuddyCasino37 minutes ago
          Are you under the impression that dentists are under competitive pressure? The labour supply is artificially limited, similar to other doctor specialisations.