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Dr. Benny Liu

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Dr. Liu researched NPC disease for around 7 years in the lab of Drs. John Dietschy and Stephen Turley at the University of Texas Southwestern (UTSW). In early 2006, Dr. Liu discovered cyclodextrin as a treatment for NPC disease.


Dr. Benny Liu is the Associate Division Chief of Gastroenterology at Highland Hospital in Oakland and an Assistant Clinical Professor at the University of California, San Francisco (UCSF). In addition to his work researching Niemann-Pick Type C disease, Dr. Liu's research interests include lysosomal storage diseases and cholesterol metabolism.

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Discovery of Cyclodextrin

Getting Involved with NPC

Dr. Liu began researching NPC shortly after an exciting finding at UCSF that claimed allopregnanolone extended the lives of NPC mice.

Research at UTSW

Although previous studies said cyclodextrin had no effect on NPC, Dr. Liu included a cyclodextrin-only control when he repeated the UCSF lifespan experiment at UTSW.

The Discovery

When Dr. Liu saw the results of the experiment, he realized it was actually cyclodextrin – not allopregnanolone – that was responsible for the lifespan extension of NPC mice.

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Mechanism of Cyclodextrin

"Cyclodextrin...actually released all that trapped cholesterol within the lysosome."

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Results of Cyclodextrin

Positive Effects

The results of cyclodextrin in mice were very good – more than double the lifespan as well as multiple improvements to their quality of life.

Adverse Effects

NPC mice treated with cyclodextrin displayed very few adverse effects.

A Question

There's one organ that didn't improve when NPC mice were treated with cyclodextrin, Dr. Liu observed.

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The Hempels' Story

"As we were developing cyclodextrin as a treatment...Chris Hempel on the other side was starting to really push this for kids with the disease."

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Cyclodextrin in NPC Patients

Bench to Bedside

Cyclodextrin's transition from the lab to the lives of NPC patients was swift.


Many families have seen NPC disease stabilization in their kids after cyclodextrin treatment.

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Roles After the Lab

"I'm still involved with cyclodextrin, building it as a treatment...I'm also heavily involved with medical education."

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"We want...to carry this to fruition and provide this drug to all the kids and all the people with NPC."