The Machinery of Disease: Scientist Invents a ‘Molecular Wrench’ That Thwarts Cancer
Science Spotlight
Courtney Miller, Ph.D., is pioneering new drug discoveries for brain cancer, addiction and PTSD, all through a powerful protein family called myosin.
By targeting myosin motor proteins in the brain, Courtney Miller, Ph.D., is developing new therapies to disrupt relapse, trauma and the spread of aggressive brain tumors. Photo by Jason Nuttle
From trauma and addiction to aggressive brain cancer, a single protein may offer creative ways to tackle difficult medical challenges. Professor Courtney Miller, Ph.D., director of academic affairs and neuroscientist at The Wertheim UF Scripps Institute, is developing treatments that target a vital family of proteins called myosins.
Myosins help shape memory, help muscles move, and control how cells grow. They also play a role in serious diseases and conditions, including cancer and post-traumatic stress disorder, or PTSD. Miller’s elegant work has revealed how myosins can be targeted with precision to stop diseases, providing new hope for people with addiction, PTSD and glioblastoma, one of the most aggressive cancers.
Who is Courtney Miller?
Miller is a neurobiologist, which means she studies the molecular biology of the brain and nervous system. She also advocates for people fighting the diseases that her science addresses. Miller is so committed to moving her discoveries to patients that she launched a company with two of her Wertheim UF Scripps colleagues that is focused bringing new medicines to the clinic. At The Wertheim UF Scripps Institute, she leads a team of researchers focused on both basic science and drug development, to address many unmet needs affecting brain health.
What are Myosins?
Myosins are a family of motor proteins that move along tiny, flexible protein strands called actin filaments. They help cells change shape, divide and communicate. While best known for their role in muscle contraction, some play key roles in cell movement and shape, memory formation in the brain, and how cancer cells invade nearby tissue.
Why it Matters
PTSD and methamphetamine addiction are notoriously difficult to treat. As of 2020, an estimated 2.5 million Americans used methamphetamine in the previous year, and 13 million were living with PTSD. Glioblastoma, an aggressive brain cancer, has a five-year survival rate of just 6.9%. In all of these conditions, the brain’s ability to form memories and change with experience plays a central role. Miller’s research shows that targeting myosin proteins may offer a potential path to treatment.
Dendritic spines receive the majority of the brain’s excitatory input. Miller’s group has targeted myosins as a way of diminishing addiction-related dendritic spine density. From CNS & Neurological Disorders – Drug Targets, DOI: 10.2174/1871527314666150529145531
How it Works
In addiction and PTSD, strong memories can overpower the brain’s ability to adapt, leading to relapse or flashbacks. In glioblastoma, brain cancer cells hijack the body’s natural movement tools to crawl through brain tissue and invade healthy areas. This ability to spread makes glioblastoma especially difficult to treat. Miller’s work is rooted in the idea that one mechanism—myosin motor activity—can be targeted to reshape how cells behave, whether to weaken harmful memories or stop a tumor in its tracks.
In the Lab
With support from the National Institutes of Health, Miller and her team spent many years developing MT-110, a first-in-class drug that targets the receptor for non-muscle myosin II to treat methamphetamine use disorder. In 2021, alongside Mayo Clinic researchers, Miller’s team discovered that non-muscle myosin IIA and IIB could also be promising targets for cancer treatment. That breakthrough led to the development of MT-125, a lead drug candidate for glioblastoma and other cancers, which olds promise to block both tumor invasion and growth, stopping the cancer in its tracks.
Because Miller’s research focuses on genes involved in brain function, her work could also inform future treatments for conditions including autism and dementia.
The Big Picture
By understanding and targeting myosins, Professor Miller is rewriting the rules for treating memory-linked diseases and cancers. Her work brings powerful new tools to the fight against addiction, PTSD and brain cancer, and is helping tranform bold ideas into clinical realities.
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Twenty Years of Innovation
A Proud Legacy of Discovery
Read about the innovators of The Wertheim UF Scripps Institute and their outsized contributions to science and to people awaiting cures.
