A Plea in a Deadly Family Affair: Lingering Fallout from California's Hold on Applications for Millions in Research Dollars

Rare disease research affected, including a man named Jeff

Jeff Klassen’s family is featured on this podcast.

“Jeff should not die” is the plea in a letter this week to the directors of California’s $12 billion stem cell and gene therapy program. 

The urgent request came in response to a five-month hold on applications for clinical research awards at the agency, formally known as the California Institute for Regenerative Medicine (CIRM).  

“One patient in particular, Jeff, has followed the development program closely over the last couple of years. When Jeff heard this news (about CIRM) he broke down crying on the phone. Jeff has no other option, and now Jeff's program will be significantly delayed due to this decision by CIRM to pause funding,” said Rich Horgan, founder and president of Cure Rare Disease, in an email to CIRM.

“Jeff has three beautiful children, a loving wife. Jeff may die from this delay. Unfortunately, Jeff is but one of thousands of stories all united by a lack of therapeutic options and now, a worsensed situation.”

Jeff Klassen has SCA3, an ultra-rare neurodegenerative disease that is characterized by a toxic accumulation of protein that is fatal, usually by asphyxiation.

He has been caught in the wake of a “tsunami” of clinical applications that