9 ms·

A "problem" with this treatment is that, for most patients, the current standard of care works. To the extent that this treatment is effective, it is likely eff

A "problem" with this treatment is that, for most patients, the current standard of care works. To the extent that this treatment is effective, it is likely effective for most leukaemia patients, including those who would respond well to current treatments. It is very plausible that this treatment (or a refined version of it) will eventually be shown to be superior to the current standard of care, in which case it would become the standard of care, and the current treatment will be relegated to the niche patient that does not respond to gene edited t-cells. More broadly, the technique of precisely editing DNA of select cells to perform specific engineered tasks is very broadly applicable. But that is a technique for developing treatments, not a treatment itself.

walkerbrown 4y ago
> will eventually be shown to be superior to the current standard of care, in which case it would become the standard of care How does this overturning occur? Do care providers agitate for change? Or is it typically a response to headstrong patients and families?
- Someone 4y ago
  It starts with researchers convincing their ethical review board that they should be given the opportunity to experiment with the different approach. That’s why new procedures almost always are done on patients who are as good as dead (gene editing to change one’s hair color won’t soon get past ethical review, for example, but if gene editing gets used in mainstream medicine, enough data may be collected to make a review board say it’s acceptable to give it a try) The first experiments typically only prove the medicine or procedure works. Long term survival of the patient the exception. From there, it’s step by step towards treating healthier and healthier patients. If it turns out the approach is better for the typical patient, patient groups (who may even have paid for part of the research) start lobbying for its use. If the new procedure is both better and cheaper, insurers also will lobby for it.
throwuwu 4y ago
Isn’t this treatment simply accomplishing the same thing that the current treatment does but in a targeted way with hopefully much fewer side effects? When you have cancerous T cells the only option is to eradicate all of them and start over with a bone marrow transplant. Normally that would mean frying them with radiation or poisoning with chemo which also effects a lot of other tissue. This treatment is supposed to only kill the T cells.
- specialist 4y ago
  That's my noob understanding too. This is a more precise form of autologous transplant. I assume quick adoption of base editing for trickier cases, eg for T-cell acute lymphoblastic leukemia, like this patient had. Note that she had a previous transplant, using the more conventional treatment of her T cells. Just starting with base editing will be much less onerous to future patients. This is one tough person. Surviving chemo then two transplants is amazing. Just thinking about it crushes my heart. Source: Had bone marrow transplant 30+ years ago. My then experimental treatment became the default option (as applicable). Don't know much about biology or medicine, but I can repeat what I've heard and read.
noobermin 4y ago
Remember what "works" means in this case. Chemotherapy, or for other cancers, radiation, surgery that removes large, healthy parts of organs that might have a tumor. Chemotherapy has hosts of side effects, of course (hair loss, nausea, ruined immunity, CIPN, etc) some permanent for some people. It "works" when you look at what KPIs oncologists like, very reductionist things like "5-year overall survival" whilst you ignore things like quality of life both in the short term and long term. We should be moving away from chemotherapy. It really is poison (cytotoxin) that has "treatment" of cancer as a side effect, just because it will readily kill cancer cells more than healthy cells whilst also taking a lot of the healthy cells with it. But, it's the "established" standard of care, which many doctors built their careers on, so I suppose it won't go away soon.
- wholinator2 4y ago
  Surgery to remove part of an organ, followed by chemotherapy gave my mother a few more years of life. Are you suggesting there is some other alternative, currently accepted and understood, that would've worked better? Because while those side effects are terrible they are usually more temporary than the extension of life that they afford people. Face the question, would you rather chemo for a couple months and then live a couple years, or die in a couple weeks?
  - tpm 4y ago
    No, they are suggesting we should strive for better therapies.

walkerbrown 4y ago

> will eventually be shown to be superior to the current standard of care, in which case it would become the standard of care How does this overturning occur? Do care providers agitate for change? Or is it typically a response to headstrong patients and families?

Someone 4y ago
It starts with researchers convincing their ethical review board that they should be given the opportunity to experiment with the different approach. That’s why new procedures almost always are done on patients who are as good as dead (gene editing to change one’s hair color won’t soon get past ethical review, for example, but if gene editing gets used in mainstream medicine, enough data may be collected to make a review board say it’s acceptable to give it a try) The first experiments typically only prove the medicine or procedure works. Long term survival of the patient the exception. From there, it’s step by step towards treating healthier and healthier patients. If it turns out the approach is better for the typical patient, patient groups (who may even have paid for part of the research) start lobbying for its use. If the new procedure is both better and cheaper, insurers also will lobby for it.

throwuwu 4y ago

Isn’t this treatment simply accomplishing the same thing that the current treatment does but in a targeted way with hopefully much fewer side effects? When you have cancerous T cells the only option is to eradicate all of them and start over with a bone marrow transplant. Normally that would mean frying them with radiation or poisoning with chemo which also effects a lot of other tissue. This treatment is supposed to only kill the T cells.

specialist 4y ago
That's my noob understanding too. This is a more precise form of autologous transplant. I assume quick adoption of base editing for trickier cases, eg for T-cell acute lymphoblastic leukemia, like this patient had. Note that she had a previous transplant, using the more conventional treatment of her T cells. Just starting with base editing will be much less onerous to future patients. This is one tough person. Surviving chemo then two transplants is amazing. Just thinking about it crushes my heart. Source: Had bone marrow transplant 30+ years ago. My then experimental treatment became the default option (as applicable). Don't know much about biology or medicine, but I can repeat what I've heard and read.

noobermin 4y ago

Remember what "works" means in this case. Chemotherapy, or for other cancers, radiation, surgery that removes large, healthy parts of organs that might have a tumor. Chemotherapy has hosts of side effects, of course (hair loss, nausea, ruined immunity, CIPN, etc) some permanent for some people. It "works" when you look at what KPIs oncologists like, very reductionist things like "5-year overall survival" whilst you ignore things like quality of life both in the short term and long term. We should be moving away from chemotherapy. It really is poison (cytotoxin) that has "treatment" of cancer as a side effect, just because it will readily kill cancer cells more than healthy cells whilst also taking a lot of the healthy cells with it. But, it's the "established" standard of care, which many doctors built their careers on, so I suppose it won't go away soon.

wholinator2 4y ago
Surgery to remove part of an organ, followed by chemotherapy gave my mother a few more years of life. Are you suggesting there is some other alternative, currently accepted and understood, that would've worked better? Because while those side effects are terrible they are usually more temporary than the extension of life that they afford people. Face the question, would you rather chemo for a couple months and then live a couple years, or die in a couple weeks?
- tpm 4y ago
  No, they are suggesting we should strive for better therapies.