Matt Gethers/20.380 HIV Project/Technical Paper/Jessie InVivo

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Jessie: In Vivo and Clinical Studies


  • Purpose of In Vivo Studies/Animal Models
  • Purpose of Pre-clinical studies
  • Brief history of clinical trials

Proof of Concept - In Vivo Studies

  • Animal Model: Most likely macaque
    • Can use pig-tailed macaque with the stHIV that has a 95% similarity to HIV-1, but infection only lasts a couple months and does not progress to AIDS
      • Can be used to test prophylactic effect
    • Can use macaque model with SIVmac that will allow macaque to develop SAIDS
      • Are SIV and HIV similar enough for this to be a proof of concept?
  • Things to look at:
    • Varying doses of HSCs - what is the optimal/plausible number of engineered HSCs needed?

Pre-clinical Studies

  • Biggest concern is safety
  • Can engineered HSCs become cancerous? - Drug super-sensitivity will be helpful
  • What other safety concerns are there with regards to stem cell therapies?
  • Need to prove natural functionality of engineered HSCs
    • Differentiate properly
    • RBCs function properly
  • What else do we need to prove before getting FDA approval?

Clinical Studies

  • Phase I
    • Prove mainly safety of treatment in humans
    • Should we do similar to Koronis and divide into Ia and Ib where Ia is in healthy volunteers and Ib is in people who have failed other HIV treatments?
    • Where should we conduct our trial? US or Africa?
  • Phase II
  • Phase III