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Pioneer in Brain Disorders Honored With the Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in Developmental Psychobiology

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Dr. Huda Zoghbi's Research Has Advanced Learning on Rett Syndrome, Autism and Adult Neurodegenerative Diseases

NEW YORK, NY (March 27, 2015) — Weill Cornell Medical College and Columbia University College of Physicians and Surgeons have announced that the Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in Developmental Psychobiology has been awarded to Dr. Huda Zoghbi, an investigator with the Howard Hughes Medical Institute and a professor in the Departments of Pediatrics, Molecular and Human Genetics, Neuroscience, and Neurology at Baylor College of Medicine in Houston.

Dr. Huda Zoghbi

Dr. Zoghbi, who is also the director of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, has done groundbreaking work on Rett syndrome and other rare brain disorders, and has advanced research of more common conditions including autism and adult neurodegenerative diseases.

"We're thrilled that Dr. Zoghbi is this year's recipient," said Dr. B.J. Casey, director of the Sackler Institute and the Sackler Professor of Developmental Psychobiology at Weill Cornell Medical College. "She's dedicated herself to this field for decades and in the process has made remarkable achievements. But we're also recognizing her leadership in the field of neuroscience and her track record of mentoring young scientists as they embark on their own careers."

"Dr. Zoghbi's work truly exemplifies the importance of having a neurodevelopmental framework when considering how to understand mental health and mental disorders," said Dr. Jay Gingrich, director of the Columbia Sackler Institute and the Sackler Institute Professor of Developmental Psychobiology at Columbia University Medical Center and the New York State Psychiatric Institute. "Her work in these areas is of profound significance, and we are delighted to be able to recognize her achievements with this prize."

Dr. Zogbhi, for her part, was both surprised and thrilled to have been chosen.

"To have a prize bearing the name of someone who's made such remarkable achievements in this field is a great honor," she said. "There are so many deserving scientists and physician-scientists out there who have done fantastic work. I feel truly honored to have been selected."

Dr. Zoghbi, a member of the National Academy of Sciences, was born and raised in Beirut, Lebanon, started medical school at the American University of Beirut and completed her medical studies at Meharry Medical College in Nashville, Tenn., and completed her residency in pediatrics and neurology and postdoctoral training in molecular genetics all at Baylor College of Medicine, where she's remained since. She says that she became interested in Rett syndrome and neurological problems after seeing patients in a clinical setting who were suffering from serious issues for which doctors didn't know or understand the cause. With Rett syndrome, infants who appeared healthy for the first year or two of their lives would start deteriorating and their brains would stop developing normally, Dr. Zoghbi said, adding that she was driven into research by the nagging question: Why is this happening?

From Rett syndrome, Dr. Zoghbi also moved on to study other neurodegenerative diseases, including some late-onset diseases that allow a person to remain healthy for decades before developing symptoms later in life. All of her work, she says, is about "getting insight into disease mechanisms" with the hope that her research and work will "make a difference in the patient's life."

"I've been fortunate to be able to find the root causes of the diseases that I am studying," Dr. Zoghbi said. In the case of Rett syndrome, this has meant identifying the gene that causes Rett syndrome and further revealing that its protein product is critical for the function of every single brain cell. She calls this the "Goldilocks Protein" because "you can't have too much of it or too little of it," she said. "It has to be at exactly the right level for the brain to be healthy," and its discovery has been the most unexpected insight from her work, she said.

When asked about the prize, Dr. Zoghbi says that she is donating the $100,000 Sackler Prize money to a mentorship fund that she established to help young female scientists get their work – specifically research on "bold, new ideas" – off the ground. Her hope is that with this support, recipients will feel emboldened to take risks and pursue the work that they love, as she was able to do.

"It's an amazing time for women in science," Dr. Casey said, pointing to Dr. Zoghbi's award and mentorship fund as proof. "They're finally getting the recognition that they have deserved for many years, and it's great that Dr. Zoghbi is championing this cause, too, and supporting women in science."

The Sackler Prize is selected by a committee of 14, including faculty from each of the seven Sackler Institutes, programs and centers: Weill Cornell Medical College; Columbia University College of Physicians and Surgeons; Universities of Edinburgh, Glasgow and Sussex; King's College London; and McGill University.

Dr. Zoghbi will hold grand rounds at Weill Cornell at 11 a.m. on April 22, and will deliver a public lecture titled "Genetic, biochemical and circuit analyses to study neuropsychiatric disorders" at 5 p.m. on April 23 at the American Museum of Natural History. A public program to celebrate the prize with a series of short presentations by Dr. Casey and other Sackler Institute/Center directors, will take place from 3 to 5 p.m. on April 23 leading into Dr. Zoghbi's keynote address.

The Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in Developmental Psychobiology

The Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in Developmental Psychobiology recognizes researchers who have advanced our understanding of the developmental processes of mind, brain, and behavior that contribute to normal development and of the origins of mental illness. The prize aims to foster international cooperation among scientists and promote public understanding of their work. The prize is presented jointly every two years by the Sackler Institute for Developmental Psychobiology at Weill Cornell Medical College and the Sackler Institute for Developmental Psychobiology at Columbia University College of Physicians and Surgeons. The first prize was awarded in 2008 to Dr. Avshalom Caspi of King's College, London, and Duke University. Dr. Fernando Nottebohm of the Rockefeller University received the prize in 2010, and Dr. Carla Shatz of Stanford University received the prize in 2012.

The prize honors one of the most creative scientists in the field of developmental psychobiology, the late Mortimer D. Sackler, M.D., who began his career as a psychiatrist and pioneer researcher in biological psychiatry in the late 1940s at the New York State Department of Mental Health. During this early period, he published more than 40 papers in peer-reviewed journals, which highlighted the role of biology in psychiatric illness. In the early 1950s, Dr. Sackler, with his brothers, founded the pharmaceutical company known today as Purdue Pharma. The original prize was a gift in honor of Dr. Sackler's 90th birthday from his seven children, and the prize was endowed in 2009 by a gift from The Mortimer D. Sackler Foundation, Inc.

The Sackler Institutes at Weill Cornell Medical College and Columbia University Medical Center

The Sackler Institute for Developmental Psychobiology at Weill Cornell Medical College, established and endowed in 1996 by the Sackler Foundation-La Fondation Sackler and Mortimer D. Sackler, M.D., family members and related entities, is focused on research and training using state-of-the-art brain-imaging techniques, human and mouse genetics, and novel behavioral methods to study typical and atypical human brain development. The Sackler Institute for Developmental Psychobiology was endowed and established at Columbia University Medical Center in 2000 by the Sackler Foundation-La Fondation Sackler and is dedicated to research into the complex processes underlying normal development as well as the origins of psychiatric illness, working at levels ranging from the molecular to the psychological.

Columbia University Medical Center

Columbia University Medical Center provides international leadership in basic, pre-clinical, and clinical research; medical and health sciences education; and patient care. The medical center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians and Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Established in 1767, Columbia's College of Physicians and Surgeons was the first institution in the country to grant the MD degree and is among the most selective medical schools in the country. Columbia University Medical Center is home to the largest medical research enterprise in New York City and State and one of the largest in the United States. Its physicians treat patients at multiple locations throughout the tri-state area, including the NewYork-Presbyterian/Columbia campus in Washington Heights, the new ColumbiaDoctors Midtown location at 51 W. 51st St. in Manhattan, and the new ColumbiaDoctors Riverdale practice. For more information, visit or

Weill Cornell Medical College

Weill Cornell Medical College, Cornell University's medical school located in New York City, is committed to excellence in research, teaching, patient care and the advancement of the art and science of medicine, locally, nationally and globally. Physicians and scientists of Weill Cornell Medical College are engaged in cutting-edge research from bench to bedside aimed at unlocking mysteries of the human body in health and sickness and toward developing new treatments and prevention strategies. In its commitment to global health and education, Weill Cornell has a strong presence in places such as Qatar, Tanzania, Haiti, Brazil, Austria and Turkey. Through the historic Weill Cornell Medical College in Qatar, the Medical College is the first in the U.S. to offer its M.D. degree overseas. Weill Cornell is the birthplace of many medical advances — including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson's disease, and most recently, the world's first successful use of deep brain stimulation to treat a minimally conscious brain-injured patient. Weill Cornell Medical College is affiliated with NewYork-Presbyterian Hospital, where its faculty provides comprehensive patient care at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. The Medical College is also affiliated with Houston Methodist. For more information, visit

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