5 ms·

Desperate, Prof Long formulated a radical plan to treat Prof Scolyer based on what had worked in melanoma, but which had never been tested in brain cancer. I d

Desperate, Prof Long formulated a radical plan to treat Prof Scolyer based on what had worked in melanoma, but which had never been tested in brain cancer. I don't know how Aus works, but, in the U.S., we need a much more serious right-to-try regime, particularly for fatal diseases like GBM. The FDA is way too slow and paternalistic, a topic that I've been writing about: https://jakeseliger.com/2024/01/29/the-dead-and-dying-at-the-gates-of-oncology-clinical-trials/ https://jakeseliger.com/2024/01/29/the-dead-and-dying-at-the... and https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-cell-carcinoma-and-the-treatments-that-might-save-me-are-just-out-of-reach/ https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-ce... because I'm dying from a squamous cell carcinoma infestation that, it turns out, should be much more treatable than it has been so far. For example, MCLA-158 / petosemtamab should, based on existing phase 1a and 1b data, be approved: https://beta.clinicaltrials.gov/study/NCT03526835 https://beta.clinicaltrials.gov/study/NCT03526835. But, from what I understand, the FDA is going to make Merus proceed with phase 3 trials, despite the incredibly positive phase 1b and 2 data. Madness. The FDA killed during the pandemic: https://moreisdifferent.blog/p/fda-devastation-during-the-pandemic https://moreisdifferent.blog/p/fda-devastation-during-the-pa... and continues to kill today. And they worried the experiment could kill him faster. It's a reasonable trade-off to decide to take the risk. Immunotherapy drugs are quite toxic, especially when mixed, so he could be poisoned I wonder which the author is talking about, because e.g. pembrolizumab (Keytruda) is not. I assume the author isn't talking about e.g. CAR-T therapies? If you're in the US and know someone with GBM, look into DCVax: https://www.uclahealth.org/departments/neurosurgery/phase-iii-clinical-trial-evaluating-dcvaxae-l-autologous https://www.uclahealth.org/departments/neurosurgery/phase-ii....

rswerve 3y ago
Good luck to you.
anonymouskimmer 3y ago
> The FDA killed during the pandemic: https://moreisdifferent.blog/p/fda-devastation-during-the-pa https://moreisdifferent.blog/p/fda-devastation-during-the-pa... and continues to kill today. There are potential trade-offs where experimental treatments which fail preclude other experimental treatments that work. Ethically this is a minefield. Personally I agree that if I have a disease I'd want to take any experimental treatment that seemed like a good idea. I think people's action during the pandemic are a bit more responsible for additional deaths than were FDA delays. : Rapid tests are actually quite sensitive during days ~2-4 of illness when patients are most infectious. Was this known beforehand, or only discovered post-hoc? From your blog: > Regarding drugs, particularly drugs for people who are already effectively dead, like me, we should be moving closer to a surgical model. This seems like a good idea. Though there have been some really bad surgical interventions (lobotomy, sex assignment surgery for intersex conditions or botched circumcisions).
agumonkey 3y ago
IIRC former NCI head, Vince de Vita, mentions that early oncology research was pretty much right-to-try by default because they had no data on anything. And it made some people live longer because they could change strategy after the first one failed. Nowadays you have a standard care and if it fails the system will almost drop you out..
- hiddencost 3y ago
  My grand father was a hematologist and he definitely just tried pet theories on terminal patients
  - JPLeRouzic 3y ago
    Isn't this both unethical and illegal?
    - austinjp 3y ago
      Maybe now, but it hasn't always been so. Attitudes change. If you're lucky enough to chat with someone in their 90s about dying, you may hear stories of how doctors used to "help people on their way". In other words, casual euthanasia was quietly accepted and expected. Not quite what GP is describing, but it illustrates the point.
      - toomuchtodo 3y ago
        My mother, long since passed, was a registered nurse (and also carrying far more empathy than most human). She explained morphine pushes and “slow codes” (taking your time so people who wanted to die could) to me in my teens (early to mid 90s). Good terms of art to know when end of life creeps up on you.
mcbain 3y ago
I'm not sure if they've said publicly what immunotherapy they used. I have seen it referred to as a combo, so I would guess ipi/nivo - that is Nivolumab (Opdivo) + Ipilimumab (Yervoy). They've been trialling those as neoadjuvant for melanoma: https://melanoma.org.au/paper/neo-adjuvant-immunotherapy-emerges-as-best-medical-practice-and-will-be-the-new-standard-of-care-for-macroscopic-stage-iii-melanoma/ https://melanoma.org.au/paper/neo-adjuvant-immunotherapy-eme... Nivo is generally well tolerated, ipi less so. I only got to my 2nd cycle of ipi/nivo before we discontinued it. (I'd already had more cycles of nivo on a trial.) It can have neurological effects and encephalitis in rare cases, so maybe that's what they are referring to. I wouldn't say "poison" though. As it happens, Prof Long is my medonc. We don't sit around chatting about Richard though, she's a bit busy on clinic days!
- mcbain 3y ago
  If it doesn't come out beforehand it'll likely be presented at ASCO: https://x.com/profglongmia/status/1753650640847597930 https://x.com/profglongmia/status/1753650640847597930
- Spooky23 3y ago
  My wife recently fought and unfortunately lost a battle with metastatic melanoma. I would agree that the nivo/ipi combo isn’t poison, but it’s no joke, and given that disease like melanoma sprouts mets like crazy, and the side effects of the treatment can result in significant side effect to critical organs. I think it’s fine to critique the FDA process, but I also think that a conservative approach to approvals is important, because ultimately bad outcomes will cause the pharmaceutical people to get more conservative internally - imo we’re better with a external entity putting the brakes on. It’s a tough business. I’m uncomfortable with the “fuck it give me the newest whatever” attitude that you find in online forums. I’m an IT exec, my wife was a finance professional in healthcare. It was difficult for us to navigate the decisions and risks. Most people are not well equipped to make risk decisions, and dumping experimental consent and other decisions on people facing death with adequate support is a really vicious thing to do. One thing that Molly felt deeply was that the hope she was able to have about beating the cancer was built on the shoulders of those before her. And folks even 10-15 years ago had no hope at all. We have a duty to honor the sacrifice made and be cognizant of what is real and what is not.
n3storm 3y ago
I am sorry about your situation, but existing so many companies and startups scamming and trying to scam people I don't feel it's slow and paternalistic (here in EU is even "slower" than in USA) Perhaps scammers should stop using health for their "getting rich fast" plots so this rules could be relaxed, but that is asking pathological narcisists to think about others which is not going to happen anytime soon :(
pkaye 3y ago
What about the "right to try" option in the US? https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try https://www.fda.gov/patients/learn-about-expanded-access-and...

rswerve 3y ago

Good luck to you.

anonymouskimmer 3y ago

> The FDA killed during the pandemic: https://moreisdifferent.blog/p/fda-devastation-during-the-pa https://moreisdifferent.blog/p/fda-devastation-during-the-pa... and continues to kill today. There are potential trade-offs where experimental treatments which fail preclude other experimental treatments that work. Ethically this is a minefield. Personally I agree that if I have a disease I'd want to take any experimental treatment that seemed like a good idea. I think people's action during the pandemic are a bit more responsible for additional deaths than were FDA delays. : Rapid tests are actually quite sensitive during days ~2-4 of illness when patients are most infectious. Was this known beforehand, or only discovered post-hoc? From your blog: > Regarding drugs, particularly drugs for people who are already effectively dead, like me, we should be moving closer to a surgical model. This seems like a good idea. Though there have been some really bad surgical interventions (lobotomy, sex assignment surgery for intersex conditions or botched circumcisions).

agumonkey 3y ago

IIRC former NCI head, Vince de Vita, mentions that early oncology research was pretty much right-to-try by default because they had no data on anything. And it made some people live longer because they could change strategy after the first one failed. Nowadays you have a standard care and if it fails the system will almost drop you out..

hiddencost 3y ago
My grand father was a hematologist and he definitely just tried pet theories on terminal patients
- JPLeRouzic 3y ago
  Isn't this both unethical and illegal?
  - austinjp 3y ago
    Maybe now, but it hasn't always been so. Attitudes change. If you're lucky enough to chat with someone in their 90s about dying, you may hear stories of how doctors used to "help people on their way". In other words, casual euthanasia was quietly accepted and expected. Not quite what GP is describing, but it illustrates the point.
    - toomuchtodo 3y ago
      My mother, long since passed, was a registered nurse (and also carrying far more empathy than most human). She explained morphine pushes and “slow codes” (taking your time so people who wanted to die could) to me in my teens (early to mid 90s). Good terms of art to know when end of life creeps up on you.

mcbain 3y ago

I'm not sure if they've said publicly what immunotherapy they used. I have seen it referred to as a combo, so I would guess ipi/nivo - that is Nivolumab (Opdivo) + Ipilimumab (Yervoy). They've been trialling those as neoadjuvant for melanoma: https://melanoma.org.au/paper/neo-adjuvant-immunotherapy-emerges-as-best-medical-practice-and-will-be-the-new-standard-of-care-for-macroscopic-stage-iii-melanoma/ https://melanoma.org.au/paper/neo-adjuvant-immunotherapy-eme... Nivo is generally well tolerated, ipi less so. I only got to my 2nd cycle of ipi/nivo before we discontinued it. (I'd already had more cycles of nivo on a trial.) It can have neurological effects and encephalitis in rare cases, so maybe that's what they are referring to. I wouldn't say "poison" though. As it happens, Prof Long is my medonc. We don't sit around chatting about Richard though, she's a bit busy on clinic days!

mcbain 3y ago
If it doesn't come out beforehand it'll likely be presented at ASCO: https://x.com/profglongmia/status/1753650640847597930 https://x.com/profglongmia/status/1753650640847597930
Spooky23 3y ago
My wife recently fought and unfortunately lost a battle with metastatic melanoma. I would agree that the nivo/ipi combo isn’t poison, but it’s no joke, and given that disease like melanoma sprouts mets like crazy, and the side effects of the treatment can result in significant side effect to critical organs. I think it’s fine to critique the FDA process, but I also think that a conservative approach to approvals is important, because ultimately bad outcomes will cause the pharmaceutical people to get more conservative internally - imo we’re better with a external entity putting the brakes on. It’s a tough business. I’m uncomfortable with the “fuck it give me the newest whatever” attitude that you find in online forums. I’m an IT exec, my wife was a finance professional in healthcare. It was difficult for us to navigate the decisions and risks. Most people are not well equipped to make risk decisions, and dumping experimental consent and other decisions on people facing death with adequate support is a really vicious thing to do. One thing that Molly felt deeply was that the hope she was able to have about beating the cancer was built on the shoulders of those before her. And folks even 10-15 years ago had no hope at all. We have a duty to honor the sacrifice made and be cognizant of what is real and what is not.

n3storm 3y ago

I am sorry about your situation, but existing so many companies and startups scamming and trying to scam people I don't feel it's slow and paternalistic (here in EU is even "slower" than in USA) Perhaps scammers should stop using health for their "getting rich fast" plots so this rules could be relaxed, but that is asking pathological narcisists to think about others which is not going to happen anytime soon :(

pkaye 3y ago

What about the "right to try" option in the US? https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try https://www.fda.gov/patients/learn-about-expanded-access-and...