Standard Care-Frontier Gap

Siqi Chen shares his experience researching treatments for his daughter's rare brain tumor, revealing the significant gaps in the medical system and how motivated individuals can go beyond standard care to find solutions.

Medical System Insights

• Standard Care vs. Frontier Gap:

  • There's a huge gap between what's available as standard of care in hospitals and what's available at the frontier of medical research
  • For rare diseases, the amount of research and available treatments is even more limited

• Research Economics Problems:

  • Rare diseases don't have enough "critical mass" for research to be financially worthwhile
  • IP issues create perverse incentives - money goes to patentable molecules even when existing drugs might work
  • Example: A pinworm drug from the 1970s shows promise for cancer treatment but gets no clinical trials because it can't be patented

• Personal Research Journey:

  • "I'm doing a lot of primary research to find repurposable drugs that might already exist that could treat a distantly rare brain tumor"
  • For rare diseases, a motivated parent can become more knowledgeable than specialists who must study many conditions
  • "I am like more knowledgeable about the disease than anyone in the room because they have to like study 50 different cancers"

• Working Outside Standard Care:

  • Proposed a third treatment path beyond what doctors initially suggested
  • "I straight up said I do not care what the standard of care is I think the standard of care is crap"
  • Found doctors willing to consider alternative approaches, especially for serious rare diseases
  • To use your own discovered treatment, you need to "convince one doctor" to prescribe it off-label on a compassionate use basis

• Not Alone in This Approach:

  • Other founders have taken similar paths - "I'm not the first founder type who's been in this situation"
  • References Rohan (Clubhouse cofounder) who runs the Lydia Foundation and manufactures his own drugs
  • "For very rare diseases you can do a lot... end of one cures exist"
  • "If you're sufficiently motivated and there's no one more motivated than a dad with a sick kid you can go so much further than what is available as standard of care"