Challenging Medical Consensus

Siqi Chen shares the story of his daughter's rare brain tumor diagnosis and his journey to find treatments.

"My daughter was diagnosed with a rare brain tumor last September and we've been doing everything we can to find new treatments for this. Because it's so rare, you really get deep insight into the sentiments and structure of the medical community.

One of the big learnings is that there's a huge gap between what's available as standard of care—meaning what's available if you go to a hospital or talk to a doctor—and what's available at the frontier.

When you have a rare disease, the amount of research, data, and treatments available is also just bad because you need enough critical mass for the research to be worth it so they can recoup costs.

We learned that IP issues are really weird. For example, there's this drug that is FDA approved, non-prescription, been out since the seventies to treat pinworms. Over the past twenty years, there's a huge amount of compelling data this might be a pretty good treatment for different kinds of cancer. But there've been no clinical trials for it because there's no money in it—you can't patent a pinworm drug. So all the money, hundreds of millions, billions of dollars, are going to new molecules that are patentable.

Once you get into this, you realize it's super broken. So I'm doing a lot of primary research to find repurposable drugs that might already exist that could treat this extremely rare brain tumor.

The last time we met with our primary care team, they were proposing two particular paths—one involving pretty aggressive surgery and radiation, another involving this drug. I proposed a third path. They were discussing it—these were neurosurgeons and clinicians—and they came back a week later saying, 'Actually, your path is more sensible.' The reason I was able to do that is because this disease is so rare, I am more knowledgeable about it than anyone in the room because they have to study 50 different cancers."