You can do a lot with $10 million. Yet when Jeffrey Randall Allen won the largest prize in Amazon Prime Video’s Beast Games, he wanted to use it for one purpose: supporting research that might help cure his son’s rare disease.

“When my youngest son, Lucas, turned eight months old, we noticed he was missing milestones,” Allen said. “As a parent, it’s hard. All these thoughts run through your mind: What’s wrong? Did we do something wrong? What can we do differently? That started a 20‑month diagnostic odyssey. It was full of valleys—with few ups. It was hard.”

The family’s journey and their search for a cure inspired the undergraduate, master’s, PhD, and postdoctoral students who heard Allen speak at the 2025 CIRM Trainee Networking Conference. The conference brought together students from CIRM’s training programs. It included talks from patient advocates who reminded them how their regenerative medicine work impacts real people and families.

No energy for the brain

When Allen’s son Lucas was two, the family finally got a diagnosis. Lucas was born with creatine transporter deficiency (CTD), a rare genetic disorder on the X chromosome with only a few hundred known cases worldwide. The disease disables the gene that transports creatine—the brain and muscle tissue’s main energy source.

Lucas’ body produces creatine normally, but without a working transporter gene, he can’t move creatine across the blood‑brain barrier to the brain cells that need it. This leads to a range of neurological problems.

Therapies like speech, physical, and occupational therapy, dietary supplements, special diets, and thickened liquids can ease symptoms, Allen said, but Lucas needs a cure.

“The diagnosis was bittersweet,” Allen said. “It was fantastic to finally know what was going on. It was bitter because there’s no therapy.”

At least, not yet.

Allen believes increasing awareness of his son’s disease and greater funding for CTD research will lead to a cure. He is using his newfound fame to teach people about CTD, and he is spending his prize money on research through the Association for Creatine Deficiencies, where he serves as vice chair and director of impact and donor relations.

Quest for cure and connection

CIRM is also funding research to develop therapies for CTD and many other rare and common diseases. In 2023, CIRM awarded $6.9 million for stem‑cell‑based therapies, including $2.3 million to Gerald Lipshutz and his UCLA team to develop a gene therapy for CTD.

With CIRM support, Lipshutz’s team is creating viral vectors that act like tiny delivery trucks to carry gene therapies to the parts of the body that need them. The researchers hope their CTD work will provide a blueprint for developing similar therapies for other genetic diseases.

Speaking to students, Jeffrey emphasized the importance of connection.
“Before the cure comes connection, and I encourage researchers to connect with the people affected by the disease and the problem they’re trying to solve,” he said.

Sharing their story

The audience was filled with students—future doctors and researchers participating in CIRM-created educational programs. These initiatives provide mentorship, funding, and training for careers in regenerative medicine.

For Allen, sharing his insights with the next generation of researchers was powerful, meaningful, and hopeful.

When asked what he wanted to say to the students who might one day find a cure for CTD, Jeffrey said, “People and parents like me need help, but we have a ton of hope. It’s up to you to use your gifts, talents, and brilliance to build cures. Together we can do amazing things.”

Allen also stressed why everyone, regardless of career, should understand basic biology and science.

“Biology was probably my least favorite science in high school,” he said. “But understanding it matters, especially now. We’re seeing discoveries move into practice faster than ever before.”

These rapid advances give Allen hope. “At the end of the day, we need to decide where to put our attention and funds. We’re going to find a therapy for this, and it’s up to us to help direct resources so it happens sooner rather than later.”

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