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Dr. Huda Zoghbi: Intown’s Medical Person of the Year

Dr. Huda Zoghbi: Intown’s Medical Person of the Year

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Our 2019 Medical Person of The Year is Dr. Huda Zoghbi. She is a Professor, Baylor College of Medicine and founding director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital. Her persistent and lengthy research has led to remarkable contributions to the study of the brain have earned her many awards, prizes accolades, and research funds for her institution. She began her career at the place she is today an unusual feat in this day and age of highly coveted talent, often leaving for a better deal.

Some of her prestigious awards include:
Vilcek Prize in Biomedical Science, 2009
March of Dimes Prize in Developmental Biology 2014
The Shaw Prize Laureate in Life Science and Medicine,
The Shaw Prize Foundation, Hong Kong, 2016
2017 Breakthrough Prize in Life Sciences
Canada Gairdner International Award, 2017

Born and raised in Beirut Lebanon she later enrolled in medical school at American University in Beirut. The Lebanese Civil War forced her parents to move her to Austin, Texas, where she later applied for med school at Vanderbilt. Her application turned down due to their policy of not accepting transfer students. Ironically, in 2015 she was awarded the Vanderbilt Prize in Biomedical Science. Instead of Vanderbilt, she attended nearby Meharry Medical College also in Nashville. Upon graduating, she went to Texas Children’s Hospital at the Baylor College of Medicine, where she still is today. Initially interested in becoming a pediatric cardiologist she was convinced by a department head the brain was a more interesting study. As it turned out that became a game changer in the field of neurology.

Breakthroughs began after many years of research on her pioneering work on Rett syndrome, a genetic neurological disease that affects young girls (males with the condition usually die in infancy) “Dr. Zoghbi’s contributions to our understanding of several entirely different neurological disorders, including her finding of the genetic basis of Rett syndrome, have opened new areas of research,” says Dr. Joe Leigh Simpson, senior vice president for Research and Global Programs at the March of Dimes, where she was awarded their top prize in biology development in 2014. “Her work influences the entire field of autism and other neuropsychiatric disorders,” stated Simpson.

Zoghbi was committed to searching for the genetic cause of the syndrome, and after a 16-year search, in 1999 she succeeded in identifying the Rett gene. This discovery provided a definitive genetic diagnosis for the condition and also opened the door to a biological understanding and a search for treatment. Zoghbi demonstrated that Rett syndrome causes a deficiency in a protein called MeCP2 and that each patient with the Rett mutation has a different pattern of healthy and mutant cells, explaining some of the variability of Rett symptoms.

In 2016 her a team of scientists at Baylor College of Medicine and Texas Children’s Hospital discovered the protein TRIM28 could promote the accumulation of two key proteins that drive the development of Parkinson’s, Alzheimer’s and related diseases. The discovery offered a new understanding of these diseases.

“Our work shows that by reducing the activity of TRIM28, we can reduce the accumulation of tau and alpha-synuclein in fruit flies and mouse models of the disease,” said Zoghbi. “These results encourage us to consider the possibility of developing drugs that could reduce the levels of TRIM28 to help prevent the development of Alzheimer’s, Parkinson’s, and related diseases.”

See Also

The Breakthrough Prize Foundation awards Breakthrough Prizes in Fundamental Physics, Life Sciences, and Mathematics.
Instituted in 2013 the winners receive a cash award of $3 million. It is a scientific award, funded by internet entrepreneurs: Mark Zuckerberg and Priscilla Chan of Facebook; Sergey Brin of Google; entrepreneur and venture capitalist Yuri Milner; and Anne Wojcicki, one of the founders of the genetics company 23andMe. The award is for research aimed at curing intractable diseases and extending human life and was awarded to Dr. Zogbhi in 2017. Moreover, the work continues. Mostly he was free of scandal or great controversy, with one troubling exception — his role as vice president in the Iran-Contra scandal. “I think over the years he fares well,” said presidential historian Henry Brands, an author and a professor at the University of Texas. “If voters have a referendum and they vote you down, that automatically puts you down a rung. It’s unfair.”

“The world was fortunate to have his background and instincts at a turning point,” said Robert Gates, who served as Bush’s CIA director and deputy national security adviser. “The collapse and end of the Cold War look sort of pre-ordained in hindsight, but for those who were there, it was not clear how it would happen.” Gates, who served in eight presidential administrations, suggested that Bush never received the credit he deserved for quietly “greasing the skids” that saw communists slide from power in the Soviet Union. “There is no precedent in all of history for the collapse of a heavily armed empire without a major war,” Gates said. “He was a figure of enormous historical importance.”

“What’s wrong with trying to help people,” he once asked. “What’s wrong with trying to bring peace? What’s wrong with trying to make the world a little better?” His one term arguably resulted in more significant legislative achievements than Reagan’s two, among them the Americans with Disabilities Act, a bolstered Clean Air Act, and an increased minimum wage. He was defeated in an unusual three-way contest with Democrat Clinton and Texas billionaire Ross Perot — a sour coda to a stellar career.

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