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Science

NYC Cancer Trial Delivers 'Unheard-of' Result: Complete Remission for Everyone (nbcnewyork.com) 79

A small NYC-led cancer trial has achieved a result reportedly never before seen - the total remission of cancer in all of its patients. From a report: To be sure, the trial -- led by doctors at Memorial Sloan Kettering and backed by drug maker GlaxoSmithKline -- has only completed treatment of 12 patients, with a specific cancer in its early stages and with a rare mutation as well. But the results, reported Sunday in the New England Journal of Medicine and the New York Times, were still striking enough to prompt multiple physicians to tell the paper they were believed to be unprecedented. One cancer specialist told the Times it was an "unheard-of" result. According to the NEJM paper and the Times report, all 12 patients had rectal cancer that had not spread beyond the local area, and their tumors all exhibited a mutation affecting the ability of cells to repair damage to DNA.
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NYC Cancer Trial Delivers 'Unheard-of' Result: Complete Remission for Everyone

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  • by OffTheLip ( 636691 ) on Wednesday June 08, 2022 @01:51PM (#62604480)
    While cautious optimism is warranted here I would say this offers a glimmer of hope for the future treatment of these insidious diseases. But I am a skeptic by nature so I will wait for a bigger trial and results.
    • Yes, this requires a larger sample size. It is quite amazing that there have been a number of advances against cancer in the recent years.

      • by arglebargle_xiv ( 2212710 ) on Wednesday June 08, 2022 @04:23PM (#62604890)

        Also, note that it's an incredibly specific type of cancer. Very specific cures aren't that rare, for example for one specific form of leukaemia, chronic myelogenous leukaemia (CML), the genes on two chromosomes get switched and use a molecule called ATP to activate other proteins that cause cancerous growths. Blocking ATP using a drug called imantinib deals with this, so it cures cancer, as long as the cancer is chronic myelogenous leukaemia.

        It's nice to see that there's yet another very specific treatment for a very specific type of cancer, but it's not the news you think it is.

        • It's my understanding that mismatch repair cancers also occur at least in the colon, brain, and blood (leukemia and lymphoma).

          From my lay person understanding, it seems likely that a drug that is so effective for this type of cancer on the rectum will probably also work a few centimeters way in the colon, or other parts of the body. I wonder if you might have more knowledge than I about this and might be able to offer an informed opinion.

          I also notice that it's an injection, meaning it travels everywhere th

          • >I also notice that it's an injection, meaning it travels everywhere the blood goes.

            Not exactly. I'd guess that you're right about the rest of the body, but since you specifically mention brain cancer, it's worth noting that the blood-brain barrier is pretty good at keeping the brain free from foreign material that's in the blood.

            I'm not well versed in the particulars though - and it might depend on the cancer. E.g. a metastasized liver tumor that takes up residence in the brain might well be on the "bl

          • This paper gives a good background on previous studies of the PD-1 pathway.

            https://www.sciencedirect.com/... [sciencedirect.com]
        • by az-saguaro ( 1231754 ) on Wednesday June 08, 2022 @08:49PM (#62605586)

          Cancer trials for new therapies, like anything else, have to start somewhere, and small exploratory studies are just the prelude to more clinical investigation for promising drugs.
          Why the investigators designed this study with this specific tumor at such and such specific staging, I could not find the info, but it seems like a rather typical study.

          In the past decade, we are seeing many new mab's (monoclonal antibodies) come to market, with some remarkable treatment profiles against many cancers. It is a whole new and better world for cancer pharma therapy than ever before, and the whole art is still in its infancy. It is all very promising.

          As for this drug and trial, the trial appropriately used a well defined tumor model, but the treatment per se is not so specific.
          The drug is dostarlimab. It's effect is to disinhibit T-lymphocytes and thereby allow the immune system to again recognize tumor cells and kill them. T-cells can be inhibited by receptors on their surfaces that bind to ligands that tumor cells can make. In essence, the tumor can turn off the cancer cops. The drug blocks the receptor, so the T-cells cannot be turned off.

          This drug works by a mechanism that is not directed against a specific metabolic or genomic alteration that creates a specific type of tumor. It is a global anti-tumor effect. Whether that translates into universal anti-tumor clinical success remains to be seen. For example, if a tumor does not depend on evading the immune system to grow, then this drug might not work. Nonetheless, this drug has a mostly generic approach to tumor killing. Compare to the many drugs now on the market that inhibit vegf (vascular endothelial growth factor). Nothing can live without blood supply, and all cells in the body can make vegf, so under conditions of growth, cells that outstrip existing vascular supply can summon a new blood vessel. This is crucial for embryogenesis, all pre-adult growth, wound healing, and tumor growth. So, inhibit vegf, and normal adult tissues that have their established stroma are not affected, but rapidly growing tumors that cannot generate a blood supply die. This is about as good as it gets for a "lowest common denominator" anti-tumor treatment. In contrast, dostarlimab does not inhibit tumor growth, it dis-inhibits tumor killing, so a different mechanism, but still a nearly universal mechanism.

          Based on this study, I think we can be expecting an avalanche of new studies for many types of cancer.

        • Many moons ago I was doing cancer research with the Feds, one of the first things we learned was that there is no such thing as "cancer". There's many cancers, that are functionally all separate diseases, with separate (albeit broadly similar) treatments. Course, this 20 years ago before the targeted immunotherapy stuff - but that just emphasizes the fact even more.

          My work on glioblastoma multiforme was useless to my wife when she was diagnosed with cervical cancer, as it would be for these tumors.

    • by kqs ( 1038910 )

      Agreed. We need more (and higher-quality) trials. However, this result guarantees more trials. The treatment is expensive, but much less expensive than cancer treatment. I have many issues with drug companies, but they often do fantastic work.

      • by jellomizer ( 103300 ) on Wednesday June 08, 2022 @02:58PM (#62604650)

        Drug companies have two parts. The scientists who actually try to make a good product and cures the problems. Then the Sales and Executives who mark up the prices because they know people are sick are going to pay nearly anything to get better. The execs will pay the politicians to look away, if the politicians refuse to take the "bribe", then they will just give money to their opposition who can stop their agenda.

        We as citizens in general will just follow the party that we relate to, willing to ignore or consider only a small problem of the aspects we dislike about the party, while clinging on on the parts we like them, and putting it in high esteem. While doing the opposite of the party we dislike.

        In a representative democracy we don't vote on issues, just for people who hold onto a large bucket of issues. If there is an issue but people from opposing parties do not give it much weight (other than probably just a 30 second talking point, but do nothing in the job) then that issue no matter how big it is will not get resolved. Because it won't effect the person you vote for.

        • This is why you need above-board-lobbying laws...
          • by ceoyoyo ( 59147 )

            And sane health care systems.

          • by dada21 ( 163177 )

            No, you donâ(TM)t.

            Lobbying is 100% free speech.

            What you need is to downsize governments by 90%, so that lobbying is ineffective because government doesnâ(TM)t have the power.

            • Lobbying is 100% free speech.

              Lobbying is 100% bribery aka corruption.

              • by torkus ( 1133985 )

                Lobbying is 100% free speech.

                Lobbying is 100% bribery aka corruption.

                The problem is you're both correct. Factor in corporate personhood too...yeah, what could go wrong? /s

            • by tragedy ( 27079 )

              What you need is to downsize governments by 90%, so that lobbying is ineffective because government doesnâ(TM)t have the power.

              Just out of curiosity, in your system, where does all that power you took away from government go?

            • by khchung ( 462899 )

              No, you donâ(TM)t.

              Lobbying is 100% free speech.

              If a US business did US-style lobbying in a foreign country, e.g. a US-owned Brazilian subsidiary "donate" money to a Brazilian political party in Brazil, then US parent company would ran afoul of US laws against bribery in a foreign country.

              That means US lawmakers are fully cognisant that US-style lobbying IS bribery. They just don't want anyone else getting the money.

            • Then drug companies can mark up prices however they want, experiment on whoever they please. You anarchists give morons a bad name...
        • In a representative democracy we don't vote on issues, just for people who hold onto a large bucket of issues. If there is an issue but people from opposing parties do not give it much weight (other than probably just a 30 second talking point, but do nothing in the job) then that issue no matter how big it is will not get resolved. Because it won't effect the person you vote for.

          Quite right. We vote for our Political Team in much the same way as sports fans root for their football or baseball team... fanatically and very forgivingly. Often, not even in our own best interest.

          This cancer drug trial is astonishing, if it's being reported accurately. 12 out of 12 patients into spontaneous remission? If no truth has been adulterated, even though the sample size is very small, that would be a statistically unlikely coincidence.

          • >Quite right. We vote for our Political Team

            Which is why I'm a big fan of some form of Direct Representation where you could vote for which of all sitting Representative should actually get to vote on your behalf. Then come bill-voting time the most popular Reps cast a huge number of votes, one per supporter, while the least popular don't cast enough votes to be worth bribing.

            Keep the Reps competing with the other members of their own team for our support, and they'd have a lot more incentive to actually

            • by ixuzus ( 2418046 )
              Interesting idea but how do you prevent your political animals becoming so populist and short sighted (even more than they are now) that they become completely incapable of making any decisions that will have short term pain but are in the long term best interests of the country?
              • 1) You really think they're looking out for the long-term interests of the country now?
                2) You really think there's so few citizens that care about the long-term interests of the country, that such Reps couldn't collectively get enough support to have real influence?
                3) There's no reason *all* Reps have to use the system. E.g. you might keep the Senate as-is, while making the House, which is supposed to represent the people, actually do so. Then any legislation needs both the support of the people, *and* th

        • Then the Sales and Executives who mark up the prices ...

          Don't know if this is true, but I've read several articles that say drug prices are (usually) based on what the company would have earned by investing the development costs, rather than the actual (usually lower) development costs. If so, I can see the logic from a shareholder perspective, except for the fact that they're drug-development companies, not investment firms and (I think) shareholders should base expectations on the former, not latter. Wonder how many people invest in pharmaceutical companies

          • Maybe sometimes, but certainly sometimes not. Remember this guy? "Shkreli was CEO of Turing Pharmaceuticals - later Vyera - when it jacked up the price of Daraprim from $13.50 to $750 per pill, after obtaining exclusive rights to the decades-old drug in 2015."
          • Investing in R&D is one of the earliest and purest forms of investment. Spending time and energy figuring out a good way to do something, rather than grunting and heaving. Yes you look at investing in medicine R&D vs investing in something else - such as building a toilet paper factory.

            Here's a question - do we *want* people to invest their money into finding a cure for cancer?

            The money for that R&D of course comes from people investing their retirement savings. Suppose you're saving for retirem

        • If the drug companies are so evil why has no one else anywhere in the world come up with a better way to create drugs? Over half the useful new drugs that are sold are funded by American companies yet Americans are only 4% of the world population. I'm not a fan of patents (I can't even make any sense of the patents that have my name on) and having worked in certification, I know that having the company producing a product pay the certification entity is a huge conflict of interests. But it is the best mod
    • If I flip a coin 12 times it might end up all heads.

      The probability of this remission won't be exactly 50% per patient but the same math still applies.

      • by sconeu ( 64226 )

        The probability of this remission won't be exactly 50% per patient but the same math still applies.

        Yeah, but this result has P 0.0003

        Agreed, it's small sample size, but definitely worth a bigger trial.

        • by sconeu ( 64226 )

          Dammit. That's P < 0.0003

        • The probability of this remission won't be exactly 50% per patient but the same math still applies.

          Yeah, but this result has P 0.0003

          Agreed, it's small sample size, but definitely worth a bigger trial.

          Provided it isn't confounded by anything, such as the the environment in the hospital and the food they were being supplied with.
          The smaller the P value, the bigger the impact of confounders on the value.

        • 12 good gets you a trial of 50, 50 gets you a 1k, etc. each level brings new funding. right?
          • The researches were looking at this treatment. When used together with chemo and surgery, it wasn't showing major benefits. But the way it worked should have been more effective if used before chemo and surgery - but getting support to delay cancer treatment while you do a drug trial was hard - hard to get approval from ethics committees, hard to get funding from companies.

            That makes this astounding result so valuable - with this result being so unambiguously positive, they should be able to go straight to

      • by Synonymous Cowered ( 6159202 ) on Wednesday June 08, 2022 @02:35PM (#62604586)

        Cancer patients tend not to just go into remission. It does happen but it's much rarer than your 50% coin flip. A little Google searching turned up some articles that put the odds around 1 in 100K. So good luck repeating that 12 times in a row.

        • by Xenx ( 2211586 )
          The unheard of part isn't that it's effective at all. You would only need to compare the odds of remission against spontaneous remission if you're trying to see if it's effective at all. The unheard of part is HOW effective it is. A more realistic comparison, for why it's unheard of, would be against the remission rate of other treatments.
    • Well it is part of the process.
      It would be unethical to start out with a large size test. However 12 people is a good number to see if roughly it is 33%, 50%, 66%, 75%... effective and if there are outlier data, there is enough people to still show general effectiveness.

      If the success is strong, as well the side effects are not worse then the condition or with other established methods. Then you can go up to a higher number 120, 1200... Until you have reached a statistical optimal point.

      Statistically if t

    • by jonadab ( 583620 )
      Sure, but when the results are this good with the small sample size, you highly prioritize the follow-up study with a rather larger sample size, as soon as it can be scheduled. AND you put out a press release, because why pass up the chance to make the stock jump.
    • It's not the sample size that is the problem, it is that they selected for a specific kind of rectal cancer that was in stage II and III.

      In general, stage IIIa rectal cancer cure rate is around 95% with standard treatments. Also, the study was done without a control.

      • As a relatively young victim of Cancer, I think it is a blessing that people are continuing to investigate and create lifesaving treatments. It allows people to have HOPE.

  • You get remission! And you get remission! You ALL GET REMISSION!

  • More importantly (Score:5, Informative)

    by fahrbot-bot ( 874524 ) on Wednesday June 08, 2022 @02:20PM (#62604550)

    Noted in the NYT article [nytimes.com] referenced in TFA, the trial subjects were "patients who could be cured with standard treatments", but hadn't yet received any chemotherapy, radiation or surgery yet -- all ended up not needing any further treatments.

    These rectal cancer patients had faced grueling treatments — chemotherapy, radiation and, most likely, life-altering surgery that could result in bowel, urinary and sexual dysfunction. Some would need colostomy bags.

    They entered the study thinking that, when it was over, they would have to undergo those procedures because no one really expected their tumors to disappear.

    But they got a surprise: No further treatment was necessary.

    The researchers ran into difficulties getting sponsored because non-successful outcomes might allow the cancers to grow beyond what could then be cured:

    Dr. Diaz began asking companies that made checkpoint inhibitors if they would sponsor a small trial. They turned him down, saying the trial was too risky. He and Dr. Cercek wanted to give the drug to patients who could be cured with standard treatments. What the researchers were proposing might end up allowing the cancers to grow beyond the point where they could be cured.

    “It is very hard to alter the standard of care,” Dr. Diaz said. “The whole standard-of-care machinery wants to do the surgery.”

    Finally, a small biotechnology firm, Tesaro, agreed to sponsor the study.

    • by GFS666 ( 6452674 ) on Wednesday June 08, 2022 @02:44PM (#62604612)

      Dr. Diaz began asking companies that made checkpoint inhibitors if they would sponsor a small trial. They turned him down, saying the trial was too risky. He and Dr. Cercek wanted to give the drug to patients who could be cured with standard treatments. What the researchers were proposing might end up allowing the cancers to grow beyond the point where they could be cured.

      “It is very hard to alter the standard of care,” Dr. Diaz said. “The whole standard-of-care machinery wants to do the surgery.”

      This is SO true. I was diagnosed with Prostate Cancer in 2018. I looked at all options, even those considered on the "cutting edge". As related above, the standard treatment by the Medical Industrial complex is surgery or Radiation Therapy (which has its own set of issues). The cutting edge options are Cryotherapy, HIFU (Hi Intensity Focused Ultrasound) or spot radiation therapy using highly radioactive "beads" to kill the tumor. Unfortunately, these cutting edge treatments are not universally accepted yet as treatments that insurance companies will cover. I eventually went with the HIFU procedure (had to pay for it out of my own pocket) and am very glad I did. Still cancer free with no side effects.

    • ..and everyone at Tesaro became very, very rich.
    • by jonadab ( 583620 )
      > The researchers ran into difficulties getting sponsored because non-successful
      > outcomes might allow the cancers to grow beyond what could then be cured

      With the 12/12 statistic from the first study, I'm guessing they can probably
      get the funding and volunteers for the larger follow-up study now.
    • What makes me guarded about this bit of good news is the qualifier "early stages". Everybody has cancer cells in their body. The problem is when these cells multiply beyond what the body's immune cells can kill off.

      Another thing is that cancer isn't a single disease, so this intervention might not apply to other cancer types.

  • and their tumors all exhibited a mutation affecting the ability of cells to repair damage to DNA.

    Why cancer is worse than thought [radiolab.org]

  • This is 2022, who here would be surprised if this "cure" played out exactly like Will Smith's I Am Legend?

  • With small sample sizes like this you need to use poisson statistics. Seeing zero failures in 12 samples means that you are 95% confident that the treatment is at least 69% effective.
  • by stabiesoft ( 733417 ) on Wednesday June 08, 2022 @03:21PM (#62604722) Homepage
    I read more and more where medical community is getting great results from immune system drugs instead of chemo and radiation. And the treatments have far fewer side effects. I saw a clip about a guy who had skin cancer I think it was, and the doc said it had metastasized. He told the guy get your things in order, 6 months probably. His wife knew about some trial that she got him into. After a few months he went into complete remission like these people. The interview was around 5 years after the diagnosis. He was still doing great. The formula for cancer seems to be let the body cut it out instead of external forces. Looks very promising. So it sounds like this drug is another one that can push the body to do the work.
    • Good point.
      Intermittent fasting was used in the old days help deal with chronic conditions like cancer.
      After 24 hours the body goes into ketosis where it’s starts moping up detritus for energy and burning fat. Sugar has been called cancer food and added sugar is only a recent phenomena.
      Interestingly, all religions have periodic fasting.

      Not saying it is a cure, but could be a helpful part a wholistic treatment protocol.

      • It's true that some cancers use carbs as fuel yes but far from all of them do. Never once have ketosis put any cancer in remission though, not even the ones that use carbs as fuel.
  • by kbahey ( 102895 ) on Wednesday June 08, 2022 @05:09PM (#62605042) Homepage

    In case you are wondering what the treatment is, and don't want to read more, it is dostarlimab [wikipedia.org].

    It is a monoclonal antibody that acts as an inhibitor for a cell death receptor.

    And it costs $11,000 every 3 weeks for 6 months.

    Cool stuff ...

    • And it costs $11,000 every 3 weeks for 6 months. My cost for non-Hodgkin lymphoma was $14,000 every 2 weeks @ initial treatment stage !! Time span got lengthen to once a month still @ 14,000/treatment fr 3 years. No cure - just reduced to 1 spot @ 6mm and cat scan once a year now.
  • This stuff has probably been around a while but the agenda and circle jerk fest between big pharma, government and corporations dont have our health at the frontlines, instead how much can they bleed from our wallets is whats important.
  • A more generally-effective approach would be a CRISPR-injected monitoring system within each cell which would test for the cell's current metabolic rate. If the rate exceeded a critical threshold, the monitoring system would kill its host cell (unless it was possible to repair it somehow). Such a monitoring system might function by producing a substance which could be metabolized by the cell, then measuring how long it took to decrease to a specific, lower concentration. To be on the safe side, each test
  • ...how many participants were dropped from the study while it was running, and for what reasons?

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