Revolutionizing Medical Research: The Power of Citizen Science

By Pulitzer Prize-winning reporter Amy Dockser Marcus, this moving narrative tells the story of a group of patient advocates who are revolutionizing the way medical research is conducted.

The Traditional Approach to Medical Research

For over half a century, medical advances have been driven by investigators launching experiments inside labs. Science has often been conducted in isolation, with a focus on long-term goals. However, this approach has its limitations, and a new movement is emerging to challenge the status quo.

The Niemann-Pick Disease Type C Revolution

A group of parents, whose children were diagnosed with the rare and fatal genetic condition known as Niemann-Pick disease type C, realized that they had to take action. This devastating disease prevents cells from processing cholesterol, leading to the progressive loss of brain and body function. Recognizing that traditional research timelines would be too slow to save their children, they decided to take matters into their own hands.

These determined parents formed a collaboration with researchers and doctors, working together to search for a cure. Through this partnership, they successfully accelerated the development of new drugs, challenging the traditional approach to medical research.

Citizen Scientists: A New Era of Medical Research

The parents didn’t just stop at funding research; they became citizen scientists, identifying promising new treatments and helping devise experiments. They recorded data about their children and co-authored scientific papers, sharing their findings with the medical community. They even engaged directly with the FDA at each step of the drug approval process.

This groundbreaking approach has sparked a radical new idea: that science must belong to us all. By working together, patients, researchers, and doctors can achieve far more than they could in isolation.

A New Pathway for Treating Disease and Conducting Research

Amy Dockser Marcus’s book reveals the extraordinary story of how a community joined forces with doctors and researchers to try to save children’s lives. This social experiment has opened up new pathways for treating disease and conducting research, and has the potential to change the face of medical science forever.

Published by Riverhead Books in February 2023, this 256-page hardcover book is a must-read for anyone interested in medical research, science, and the power of community-driven innovation.