4 ms·

Here's a press release from the company: http://asteriasbiotherapeutics.com/asterias-biotherapeutics-announces-positive-efficacy-data-in-patients-with-complete-

by stenl 10y ago

Here's a press release from the company: http://asteriasbiotherapeutics.com/asterias-biotherapeutics-announces-positive-efficacy-data-in-patients-with-complete-cervical-spinal-cord-injuries-treated-with-ast-opc1/ http://asteriasbiotherapeutics.com/asterias-biotherapeutics-... They're doing a dosing study, with escalating number of cells injected. Results are promising, but the study includes no controls, so there is no way to know if the cell-based treatment is better than no treatment. The current study is open-label (i.e. not blinded) single-arm (no controls). Once they establish the right dose, they will need to do a double-blind randomized controlled study to find out if it works.

JshWright 10y ago
Blinding isn't especially important for a procedure like this. An RCT would be a sufficiently strong study design, in my opinion.
- robbiep 10y ago
  If you were going to RCT, in this instance, why would you not blind? Sham injections - it's a no brainer. Why get the second best level of evidence if you can get the best?
  - Darwin226 10y ago
    It's a needle to the spine...
    - Noseshine 10y ago
      Hmm... maybe use people who require surgery/anesthesia and just tell them they were injected? I've taken a few courses on clinical trial design just out of curiosity, but now I find I can't really say anything substantial about my own idea. Back to the courses?
      - JshWright 10y ago
        Sham surgery is a thing (there have been some interesting studies looking at the efficacy of knee surgery compared with sham surgery for a variety of common knee issues...) https://www.youtube.com/watch?v=P_pAdg8Y70Y https://www.youtube.com/watch?v=P_pAdg8Y70Y
  - JshWright 10y ago
    People much smarter than me have been wrong about the placebo effect in the past, but I think it's pretty unlikely that a placebo effect will result in the restoration of function after a traumatic spinal cord injury... This procedure clearly needs to be tested against "doing nothing" (but it's impossible to blind against that, obviously). You only need to blind a study when you are testing against a placebo.
    - lisper 10y ago
      > I think it's pretty unlikely that a placebo effect will result in the restoration of function after a traumatic spinal cord injury... You would be amazed. The placebo effect is surprisingly powerful. https://en.wikipedia.org/wiki/Sham_surgery https://en.wikipedia.org/wiki/Sham_surgery
      - JshWright 10y ago
        Yeah, I'm familiar with the effect (I linked to a Healthcare Triage video on that topic in another leaf). I still think the placebo effect is likely to be very small when it comes to the restoration of movement (as opposed to reduction in pain, or other symptoms).
        lisper 10y ago
        Yes, but the only way to know for sure is to do the experiment. Wouldn't it be cool if you turned out to be wrong?

by stenl 10y ago

JshWright 10y ago

Blinding isn't especially important for a procedure like this. An RCT would be a sufficiently strong study design, in my opinion.

robbiep 10y ago
If you were going to RCT, in this instance, why would you not blind? Sham injections - it's a no brainer. Why get the second best level of evidence if you can get the best?
- Darwin226 10y ago
  It's a needle to the spine...
  - Noseshine 10y ago
    Hmm... maybe use people who require surgery/anesthesia and just tell them they were injected? I've taken a few courses on clinical trial design just out of curiosity, but now I find I can't really say anything substantial about my own idea. Back to the courses?
    - JshWright 10y ago
      Sham surgery is a thing (there have been some interesting studies looking at the efficacy of knee surgery compared with sham surgery for a variety of common knee issues...) https://www.youtube.com/watch?v=P_pAdg8Y70Y https://www.youtube.com/watch?v=P_pAdg8Y70Y
- JshWright 10y ago
  People much smarter than me have been wrong about the placebo effect in the past, but I think it's pretty unlikely that a placebo effect will result in the restoration of function after a traumatic spinal cord injury... This procedure clearly needs to be tested against "doing nothing" (but it's impossible to blind against that, obviously). You only need to blind a study when you are testing against a placebo.
  - lisper 10y ago
    > I think it's pretty unlikely that a placebo effect will result in the restoration of function after a traumatic spinal cord injury... You would be amazed. The placebo effect is surprisingly powerful. https://en.wikipedia.org/wiki/Sham_surgery https://en.wikipedia.org/wiki/Sham_surgery
    - JshWright 10y ago
      Yeah, I'm familiar with the effect (I linked to a Healthcare Triage video on that topic in another leaf). I still think the placebo effect is likely to be very small when it comes to the restoration of movement (as opposed to reduction in pain, or other symptoms).
      - lisper 10y ago
        Yes, but the only way to know for sure is to do the experiment. Wouldn't it be cool if you turned out to be wrong?