When Huda Zoghbi met 2-year-old Henry Engel, she saw an incredible opportunity.
“Henry captured Dr. Huda Zoghbi’s heart – but more than that, she thinks he could lead her to a scientific breakthrough,” reports Richard Engel, who is both NBC’s chief foreign correspondent and Henry’s father.
Henry suffers from Rett Syndrome, a rare genetic mutation that almost exclusively affects girls. Because the disorder is so rare in boys, Zoghbi realized that Henry’s case might hold the key to treating or even reversing it.
Huda Zoghbi First Discovered the Cause of Rett Syndrome
Zoghbi made a name for herself in the scientific community when she and her Israeli research partner Ruthie Amir discovered the specific gene mutation responsible for Rett Syndrome. Now, the Lebanon-native Zoghbi sees hope for a treatment in the near future – and not just for Rett Syndrome.
“In principle, we work on rare diseases, but what I’ve learned is that many of the principles that apply to rare diseases, you can actually apply to common disorders using similar strategies,” she explains. The research Zoghbi does on Henry’s genes may in fact also hold the key to treating a whole host of neurological disorders, from Alzheimer’s to Autism.
When Zoghbi first began investigating Rett Syndrome, she had a long road ahead of her. She began as a pediatric neurologist, not a researcher. But when she grew attached to a young patient named Ashley, she didn’t want to keep treating her disorder with the limited resources available. Instead, she wanted to find a way to cure her and others like her.
This dream is closer than ever. Zoghbi’s research has already found success in mice modeled with Henry’s gene. A successful treatment for humans may not be far off.
Civil War Interrupted Zoghbi’s Education
Zoghbi’s route to success was not a straight path. She studied biology at the American University of Beirut, but the growing sectarian tension in Lebanon made travel to and from the university difficult.
“Half way through my first year, civil war broke out, forcing those of us determined to continue our studies to sleep in double-walled rooms and basements on campus.” She recalls cooking her meals on a Bunsen burner, and sleeping in a small closet in the women’s bathroom.
The civil war also brought Zoghbi to the United States. While visiting relatives in Texas, Zoghbi found herself stranded as tensions in Lebanon grew to the point where airfields and travel shut down entirely.
She nonetheless persisted in her studies. After earning her degree from Meharry Medical College, she completed her residency at the Baylor College of Medicine.
New Research Meant Returning to School for Huda Zoghbi
This kind of risk and hard work are the bread and butter of Zoghbi’s success.
As a medical doctor, pursuing the research side of neurology meant starting back at square one. But this didn’t faze her. She was determined to find the cause of rare disorders, even if it meant several more years of schooling.
And in 1999, she discovered the gene responsible for Rett Syndrome. Over the next twenty years, she has continued to make breakthrough after breakthrough in the research of neurological disorders, earning countless awards and honors for her work.
“When you take a risk you are going where nobody has gone. That’s when you really can open a new path,” says Zoghbi. “I love taking risks in science.”
