Decentralized Science vs. Rare Diseases: A Game-Changer? | Ethan Perlstein

Mar 24, 2025

Ethan Perlstein, founder of CureTopia, discusses revolutionary approaches to addressing rare diseases that affect over 30 million people in the U.S. He highlights the misleading notion of rarity and the neglect from Big Pharma. Perlstein advocates for decentralized science and blockchain as transformative tools to empower patient communities and streamline drug development. The conversation also explores drug repurposing, community engagement, and the emotional journeys of affected families, all while pushing for innovative funding models and greater awareness.

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INSIGHT

Rare Diseases Are More Common Than Thought

Rare diseases affect over 30 million people in the U.S., more than cancer and HIV combined.
The term "rare" is misleading; collectively these diseases are common but are underfunded and overlooked.

ANECDOTE

Parents Become Scientific Experts

Ethan Perlstein met a rare disease parent who was deeply knowledgeable about science and biotech.
Parents often become scientific experts while fighting to save their children from fatal diseases.

INSIGHT

Rare Disease Fragmentation Limits Unity

The umbrella term "rare disease" unites many diverse conditions with little common purpose.
This fragmentation hinders the formation of a unified social movement or collective voice.

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Welcome to another episode of the DeSci Podcast. Today, we are discussing a critical issue that affects over 30 million people in the United States—Rare Diseases. Despite being more prevalent than cancer and HIV combined, rare diseases remain underfunded, overlooked by Big Pharma, and trapped in a broken system.Our Guest: This episode features Ethan Perlstein, founder of CureTopia, an initiative using Decentralized Science (DeSci) and blockchain to revolutionize rare disease research. Why does the current system fail millions of patients, and how can decentralized models change that?What You’ll Learn in This Episode:-The reality of rare diseases and why the term "rare" is misleading-How patients and parents are taking control of scientific research-Why Big Pharma does not prioritize rare diseases and the funding challenges involved-How DeSci and blockchain can disrupt traditional biotech and accelerate drug development-The vision behind CureTopia and how it is reshaping research for rare disease treatmentsResources Mentioned:CureTopia: https://curetopia.xyzDeSci Podcast: https://www.molecule.xyzFeel free to navigate through any of the topics discussed in the podcast using the timestamps below.___⭐ Timestamps:0:00 - Introduction: The Rare Disease Crisis2:14 - Why Rare Diseases Are More Common Than People Think5:13 - Challenges in Rare Disease Advocacy9:25 - How Parents Become Scientific Experts in Their Child’s Disease13:50 - Why Big Pharma Ignores Rare Diseases17:16 - The Role of Decentralized Science (DeSci) in Drug Development22:41 - The Funding Challenges in Rare Disease Research27:34 - How Blockchain and Smart Contracts Can Help Rare Disease Research33:04 - The Power of Community in Rare Disease Advocacy38:26 - What Is CureTopia and How Does It Work?43:57 - The Future of Rare Disease Research and Biotech Innovation49:14 - Ethan Perlstein’s Vision for CureTopia54:10 - Closing Thoughts and Where to Learn More______If you prefer listening to podcasts on audio apps, feel free to search for "The DeSci Podcast" on Spotify, Google Podcasts & Apple Podcasts. You'll also find all links to the podcast apps below.If you want to join us on the show as a speaker or have guest ideas, please shoot us an mail at podcast@molecule.toSpecial thanks to:Jillian Casalini: https://x.com/JillianCasaliniEthan Perlstein: https://x.com/eperlste____Let's connect: Website: https://bit.ly/3d6nh2CX: https://twitter.com/Molecule_daoDiscord: https://discord.com/invite/moleculedaoMolecule - The future of medicine belongs to everyone!