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Researcher Provides Insight into Neurological Disorders

March 31, 2016

With the number of people suffering from Alzheimer’s disease expected to double in the next 20 years, the importance of the research being done by Dr. Huda Zoghbi in her lab at Baylor College of Medicine cannot be underestimated.

Dr. Huda Zoghbi Dr. Huda Zoghbi One of the world’s foremost researchers on neurological disorders, Zoghbi related aspects of her work and revealed some potential breakthroughs in prevention and treatments as the featured speaker for ASU’s 40th annual WTMA Distinguished Lectureship in Science Honoring Dr. Roy E. Moon on March 29. 

A native of Beirut, Zoghbi is best known for identifying the genes responsible for spinocerebellar ataxia (SCA1) and Rett syndrome. In her first ASU presentation, she detailed how her team discovered that SCA1 is caused by mutation of a particular gene that increases the level of the respective protein in brain cells. 

“That subtle increase is protein level is enough to cause neurodegeneration,” Zoghbi said. “If we can bring the level of that protein back to normal, it restores the balance in our animal models and makes them much better.” 

“So we then asked if that could be applicable to other adult neurodegenerative diseases, like Parkinson’s and Alzheimer’s,” she added. “We dug deeper and found that in all these diseases, protein levels do really matter, and it is different proteins for the different diseases. So if we can find ways to decrease those proteins to normal levels, maybe we can come up with therapeutics for Alzheimer’s and Parkinson’s.” 

With that discovery, Zoghbi shifted her focus to conceptualizing the human genetic data of Alzheimer’s and Parkinson’s. 

“Now we hope we have a path forward to develop new potential therapeutics for the future treatment of those diseases,” she said. “We are already in the process of developing a drug with a pharmaceutical company to help people suffering with SCA1.” 

“This is the generation that will benefit from the many scientific advances made in the last few years. Today’s students will have such a head start. But does that mean there won’t be any challenges? Absolutely not.”

Dr. Huda Zoghbi

Zoghbi’s second ASU presentation dealt with her celebrated work on Rett syndrome, an early-onset neurological disorder that mainly affects young girls and manifests in loss of speech, balance and motor control problems, constant hand-wringing, learning disability, anxiety, tremors and seizures. 

“It affects every aspect of the nervous system,” Zoghbi said, “so you can use it to understand many neuropsychiatric diseases.” 

In taking the audience through what her team has learned about the particular gene mutation that causes Rett syndrome, Zoghbi also detailed how they are using their findings to gain insight into which cells in the brain control our different behaviors. She also revealed both ongoing and upcoming tests using deep brain stimulation and neuromodulation in attempts to treat different symptoms of Rett syndrome. 

“The real joy that I will get is if I can develop some therapies that will help the people suffering from any of the diseases I study,” Zoghbi said. “That is the goal that I hold up.” 

Her team’s research findings also have implications for cancer research and have led to increased knowledge of bipolar and schizophrenia disorders and for distinguishing different types of autism. And she was pleased to be able to bring details of that research to ASU. 

Dr. Zoghbi in her lab at Baylor College of Medicine. (Photo courtesy of BCM) Dr. Zoghbi in her lab at Baylor College of Medicine. (Photo courtesy of BCM) “If I stay in my lab, affect advances and inspire my own students, that is great,” Zoghbi said. “But if I come to a place like ASU, where there are good students, but they may not have the opportunity to meet a diverse group of scientists, and I can inspire them, then I have increased what I can do on behalf of science and medicine.” 

One of her final statements included both comments and advice for budding ASU student scientists. 

“This is an exciting time to be studying science,” Zoghbi said. “This is the generation that will benefit from the many scientific advances made in the last few years. Today’s students will have such a head start. But does that mean there won’t be any challenges? Absolutely not.”

“With all the new technologies and everything at their fingertips, there will still be challenges,” she continued. “They will be able to try to answer even tougher questions and with that sometimes comes disappointment, but that is the nature of science. But those who stick with it will always make a discovery.”