A Small Fact About Kymriah
I'm writing this from our kitchen. It's Friday morning. I've had my coffee.
I want to write down one hopeful thing.
On August 30, 2017, the FDA approved Kymriah, a CAR-T cell therapy, for children and young adults with B-cell acute lymphoblastic leukemia that had relapsed or was refractory to standard chemotherapy.
The way Kymriah works is that a child's own T cells are collected, genetically re-engineered outside the body to target a protein on the surface of leukemia cells, and then infused back into the child. The re-engineered T cells hunt down and kill the cancer.
In the pivotal trial, 83 percent of the children and young adults treated went into complete remission within three months.
That is a group of patients who, in 1990, would have had almost no options after relapse. That is a group of patients who, in 2017, had a first-ever, FDA-approved gene therapy targeted at their specific disease.
That is what pediatric cancer research does when it gets funded.
I want to sit with that.
The distance between vincristine (1963) and Kymriah (2017) is 54 years. Most of what happened in pediatric leukemia treatment during those 54 years was the smarter use of the drugs we already had. Kymriah is the exception. Kymriah is what a targeted, well-funded, dedicated pediatric research effort looks like.
We need more Kymriahs.
That is the advocacy argument for pediatric cancer research funding, in one sentence.
I'm going to close the laptop. Our children are up. We're going to have breakfast.
I wanted to write this down because sometimes advocacy sounds like grief and rage, which it is. It's also sometimes hope. Kymriah is hope. There are researchers today working on the next Kymriah. Our advocacy funds them.
Dina
Mom of Max | Founder, Maxwell’s Toy Shoppe
Childhood Cancer Advocate 💛
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