Using a Human Stem Cell Model to Study Schwannomas
Summary
- Dr. Diane Jung is investigating new drug targets for NF2-related vestibular schwannomas using an innovative human stem cell model.
- Radiation and surgery are the only treatment options for vestibular schwannomas.
- Dr. Jung and mentor Dr. Pei-Ciao Tang developed the stem cell model for their research.
What if a stem cell breakthrough could unlock a treatment for a rare genetic disorder that robs people of their hearing and threatens their brain function?
At the University of Miami Miller School of Medicine, research resident Diane Jung, M.D., Ph.D., is teaming up with stem cell biologist Pei-Ciao Tang, Ph.D., to do just that.
Backed by a prestigious resident research grant from the American Academy of Otolaryngology–Head and Neck Surgery, Dr. Jung is investigating NF2-related vestibular schwannomas, tumors with life-altering consequences and no approved drug therapies. Their mission: to uncover new drug targets using an innovative human stem cell model that could redefine how scientists approach tumor research and treatment.
NF2 and Schwannomas
NF2 is a rare genetic disorder that affects about one in every 33,000 people.
“If a patient has an NF2 mutation, they often develop these vestibular schwannomas in both ears and lose their hearing,” says Dr. Jung. “It can also result in brainstem compression and other serious complications such as loss of balance.”
Unfortunately, there aren’t any approved drugs to treat vestibular schwannomas. Radiation and surgery are the only treatment options.
To identify drug targets, researchers need to better understand the molecular mechanisms driving cellular proliferation. However, there are no effective models to explore how manipulating the expression of different genes might impact tumor development.
Human Stem Cell Models
Dr. Jung and her primary mentor, Dr. Tang, a research assistant professor in the Department of Otolaryngology-Head and Neck Surgery at the Miller School, will be using a newly-developed human stem cell model.
Dr. Tang’s lab previously developed human stem cells with the NF2 mutation and then transformed them into Schwann cells. It’s an ideal model to probe the molecular mechanisms of tumor proliferation.
“Through the collaboration among surgeon-scientists and stem cell biologists, we now have a good team with various specialties to initiate this project,” says Dr. Tang. “We established a collection of human stem cells carrying NF2 mutations and a Schwann cell model derived from those human stem cells. With these tools, we now can investigate the role of NF2 in the human Schwann cell environment directly.”
The study is early phase. It is likely to be years before a treatment is developed from it and progresses through clinical trials.
“The more we understand about the molecular processes of NF2,” said Dr. Tang, “the more likely we’ll be able to find targets for drug development.”
In addition to advancing treatment for schwannomas, Dr. Jung and Dr. Tang hope that the work will remind other scientists of the potential for human stem cell models in disease research.
“The human stem cell-based model is a powerful tool not only for studying the biology of the tumor but also providing a platform for testing various hypotheses and treatments,” said Dr. Tang.
Tags: Department of Otolaryngology, Dr. Pei-Ciao Tang, NF2, otolaryngology, stem cell study, vestibular schwannoma