8 ms·

Some heart-wrenching comments from the page: "Husband and father of 3, age 31, high grade spindle cell sarcoma, stage 4 with mets- help! nuclearcouple@hotmail.

by wwwtyro 14y ago

Some heart-wrenching comments from the page: "Husband and father of 3, age 31, high grade spindle cell sarcoma, stage 4 with mets- help! nuclearcouple@hotmail.com or find me (spouse) on fb, Heather Cimino in Fort Myers, Fl, willing to travel anywhere, just save my husband!" "my wife has tumor that are killing her will you hurry up and get this sorted - is there anyway one can volunteer for a trial tim.parry@xtra.co.nz" There has got to be a way to address this need.

ceejayoz 14y ago
There is, "expanded access" or "compassionate use". http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocat...
- maxharris 14y ago
  How many months or years does that process take? Morally speaking, the judgement of a patient (and the physician he has chosen) must not be impeded even for an instant. The FDA renders individual judgement on these matters irrelevant, and the result is often death and slowed medical progress.
  - wwwtyro 14y ago
    I imagine these people are desperate enough to find other means to acquire an antibody that blocks CD47. I wonder what quasi- or il- legal means are available to them? Care to speculate? Edit: perhaps a set of crowd-sourced, extremely explicit instructions about how to manufacture, purify, and test such antibodies would be a start. With such a set of instructions, they might be able to find experts to help them under the table.
    - maxharris 14y ago
      Well, you could try ordering from Fischer or any of the usual research supply places out there, but they will not ship to a non-institutional address. Even theft isn't an option here, unless you're somehow certain that a particular lab has exactly the right antibody. (In my lab, we often have trouble keeping track of that for ourselves...) And don't forget that you need to be able to maintain sterile conditions, and have whatever other reagents you need to dilute things, etc.
      - wwwtyro 14y ago
        Wait - are you saying people could (were it legal) simply order this stuff from a company? Can you give me some kind of reference for this? If so, I could see people reaching out and finding others capable of placing the order.
        maxharris 14y ago
        Here's CD47, conjugated to a fluorophor (when you have a cell covered in a conjugated antibody, it fluoresces when exposed to laser light - this is done so that an instrument called a flow cytometer can identify cells). http://www.biolegend.com/percp-cy55-anti-human-cd47-4338.html http://www.biolegend.com/percp-cy55-anti-human-cd47-4338.htm... Of course, to get CD47 depletion, you'd probably want an antibody that isn't conjugated (functional grade): http://www.ebioscience.com/resources/in-vivo-use-of-antibodies.htm http://www.ebioscience.com/resources/in-vivo-use-of-antibodi... Also, some more help on the terminology: CD47 (and most, if not all molecules that fall under the Cluster of Differention [CD] nomenclature) vary on a per-species basis. So an antibody that targets CD47 in mice is not going to stick to human CD47. "Anti-" in this context means that the antibody in question will stick to specific molecule in the species that comes after the "anti-". Note what it says on the bottom of the page. That's there because of regulations.
        wwwtyro 14y ago
        Perfect, thanks for all this.
    - deleted 14y ago
      [deleted]
    - car 14y ago
      Strong word of warning! These antibodies are not human, but most likely mouse. They will illicit a strong immune response, since they are foreign to the human immune system. Furthermore, antibodies can be directed against many different locations on their target molecule (epitopes), CD47 in this case, and therefore it is very unlikely that they will compete with the ligand they are trying to block. In brief: you will hurt people doing this and achieve nothing.
      - wwwtyro 14y ago
        Thank you.
  - ceejayoz 14y ago
    I'm only aware of the process because of a NYTimes (IIRC) article, which said they had a roughly two week wait for approval. Getting the pharmaceutical company to provide the med was significantly harder.
maxharris 14y ago
There has got to be a way to address this need. Yes, there is. Fight for the right of each individual to follow his best judgement in regard to all matters pertaining to his own life. (If someone doesn't know enough to make the call, he knows enough to choose a physician who can.) Right now, timely experimental treatments are basically outlawed. What good is it to say that there's an FDA procedure to get around the controls if you're treated too late? What right does anyone have to deprive anyone else of what might be their only shot at life?
- wwwtyro 14y ago
  Didn't know it was this bad, but couldn't agree more. It should be entirely up to an individual what they put in their body. Monumentally frustrated right now.
- roc 14y ago
  The problem is that lay people have essentially no ability to distinguish between potential 'last shots'. And exemptions will invite snake-oil salesmen. It can be frustrating, but the rules weren't created in some blind bureaucratic power-grab. They're responses to actual problems that existed in their absence. They aren't without their downsides, but they remain better than the alternative on balance.
  - wwwtyro 14y ago
    They aren't without their downsides, but they remain better than the alternative on balance. That's not a credible claim. Tell that to either of the two people that left those comments. Sure, there's risks inherent with experimental drugs, but these people are dying or losing loved ones. They don't give a damn, and none of us has any right to stop them from taking those risks with their own persons. Their bodies are their own, they are not yours. And frankly, the snake-oil salesman claim strikes me as little more than FUD. People can get recommendations from multiple MDs before making up their minds.
    - rubyrescue 14y ago
      The history of snake-oil salesmen is well documented. http://www.authentichistory.com/1898-1913/2-progressivism/8-quackery/ http://www.authentichistory.com/1898-1913/2-progressivism/8-...
sunnybunny 14y ago
Well, the treatment is moving to Stage 1 Human trials, these people should clearly get in touch and volunteer to be a part of the trials.
- pcrh 14y ago
  Here is a link to a related trial in South Carolina currently recruiting participants to examine a role for CD47 in myeloma. It is not a trial using the antibody cited in the OP's link, but a study to examine if targeting CD47 (and some other potential targets) makes sense in a human context, rather than just in a mouse.

by wwwtyro 14y ago

ceejayoz 14y ago

There is, "expanded access" or "compassionate use". http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocat...

maxharris 14y ago
How many months or years does that process take? Morally speaking, the judgement of a patient (and the physician he has chosen) must not be impeded even for an instant. The FDA renders individual judgement on these matters irrelevant, and the result is often death and slowed medical progress.
- wwwtyro 14y ago
  I imagine these people are desperate enough to find other means to acquire an antibody that blocks CD47. I wonder what quasi- or il- legal means are available to them? Care to speculate? Edit: perhaps a set of crowd-sourced, extremely explicit instructions about how to manufacture, purify, and test such antibodies would be a start. With such a set of instructions, they might be able to find experts to help them under the table.
  - maxharris 14y ago
    Well, you could try ordering from Fischer or any of the usual research supply places out there, but they will not ship to a non-institutional address. Even theft isn't an option here, unless you're somehow certain that a particular lab has exactly the right antibody. (In my lab, we often have trouble keeping track of that for ourselves...) And don't forget that you need to be able to maintain sterile conditions, and have whatever other reagents you need to dilute things, etc.
    - wwwtyro 14y ago
      Wait - are you saying people could (were it legal) simply order this stuff from a company? Can you give me some kind of reference for this? If so, I could see people reaching out and finding others capable of placing the order.
      - maxharris 14y ago
        Here's CD47, conjugated to a fluorophor (when you have a cell covered in a conjugated antibody, it fluoresces when exposed to laser light - this is done so that an instrument called a flow cytometer can identify cells). http://www.biolegend.com/percp-cy55-anti-human-cd47-4338.html http://www.biolegend.com/percp-cy55-anti-human-cd47-4338.htm... Of course, to get CD47 depletion, you'd probably want an antibody that isn't conjugated (functional grade): http://www.ebioscience.com/resources/in-vivo-use-of-antibodies.htm http://www.ebioscience.com/resources/in-vivo-use-of-antibodi... Also, some more help on the terminology: CD47 (and most, if not all molecules that fall under the Cluster of Differention [CD] nomenclature) vary on a per-species basis. So an antibody that targets CD47 in mice is not going to stick to human CD47. "Anti-" in this context means that the antibody in question will stick to specific molecule in the species that comes after the "anti-". Note what it says on the bottom of the page. That's there because of regulations.
        wwwtyro 14y ago
        Perfect, thanks for all this.
  - deleted 14y ago
    [deleted]
  - car 14y ago
    Strong word of warning! These antibodies are not human, but most likely mouse. They will illicit a strong immune response, since they are foreign to the human immune system. Furthermore, antibodies can be directed against many different locations on their target molecule (epitopes), CD47 in this case, and therefore it is very unlikely that they will compete with the ligand they are trying to block. In brief: you will hurt people doing this and achieve nothing.
    - wwwtyro 14y ago
      Thank you.
- ceejayoz 14y ago
  I'm only aware of the process because of a NYTimes (IIRC) article, which said they had a roughly two week wait for approval. Getting the pharmaceutical company to provide the med was significantly harder.

maxharris 14y ago

There has got to be a way to address this need. Yes, there is. Fight for the right of each individual to follow his best judgement in regard to all matters pertaining to his own life. (If someone doesn't know enough to make the call, he knows enough to choose a physician who can.) Right now, timely experimental treatments are basically outlawed. What good is it to say that there's an FDA procedure to get around the controls if you're treated too late? What right does anyone have to deprive anyone else of what might be their only shot at life?

wwwtyro 14y ago
Didn't know it was this bad, but couldn't agree more. It should be entirely up to an individual what they put in their body. Monumentally frustrated right now.
roc 14y ago
The problem is that lay people have essentially no ability to distinguish between potential 'last shots'. And exemptions will invite snake-oil salesmen. It can be frustrating, but the rules weren't created in some blind bureaucratic power-grab. They're responses to actual problems that existed in their absence. They aren't without their downsides, but they remain better than the alternative on balance.
- wwwtyro 14y ago
  They aren't without their downsides, but they remain better than the alternative on balance. That's not a credible claim. Tell that to either of the two people that left those comments. Sure, there's risks inherent with experimental drugs, but these people are dying or losing loved ones. They don't give a damn, and none of us has any right to stop them from taking those risks with their own persons. Their bodies are their own, they are not yours. And frankly, the snake-oil salesman claim strikes me as little more than FUD. People can get recommendations from multiple MDs before making up their minds.
  - rubyrescue 14y ago
    The history of snake-oil salesmen is well documented. http://www.authentichistory.com/1898-1913/2-progressivism/8-quackery/ http://www.authentichistory.com/1898-1913/2-progressivism/8-...

sunnybunny 14y ago

Well, the treatment is moving to Stage 1 Human trials, these people should clearly get in touch and volunteer to be a part of the trials.

pcrh 14y ago
Here is a link to a related trial in South Carolina currently recruiting participants to examine a role for CD47 in myeloma. It is not a trial using the antibody cited in the OP's link, but a study to examine if targeting CD47 (and some other potential targets) makes sense in a human context, rather than just in a mouse.