Huffington Foundation Focuses on Parkinson’s Research
By Michelle Flippin
A $2.5 million gift from the Huffington Foundation in September 2011 is providing an unprecedented opportunity for researchers at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital in Houston to advance research and treatment for Parkinson’s disease and other complex neurological illnesses.
Parkinson’s disease (PD) is the second most common neurodegenerative disease in aging populations, affecting some 10 million people worldwide — 1 million of them in the United States. Common symptoms include shaking (tremors) and difficulty with walking, movement and coordination.
The gift from the Huffington Foundation is enabling established and up-and-coming scientists to pursue three novel approaches to understanding the pathogenesis of PD. Andrea Ballabio, M.D., and Carmine Settembre, Ph.D., aim to modulate lysosomal activity to promote cellular clearance and prevent, or even reverse, neurodegeneration in PD. Hugo Bellen, D.V.M, Ph.D., and Hector Sandoval, Ph.D., hope to delineate the role of particular genes in mitochondrial dysfunction, which plays a critical role in the disease. Juan Botas, Ph.D., Jeehye Park, Ph.D., Thomas Westbrook, Ph.D., and the Institute’s director, Huda Y. Zoghbi, M.D., will use systematic genome-scale screening to identify genetic modifiers and targets suitable for therapeutic interventions.
“We so appreciate the generous support from the Huffington Foundation to allow us to launch this collaborative research program in Parkinson’s disease,” Zoghbi said. “Philanthropy like this enhances the breadth, depth and diversity of research we conduct every day.”
A portion of the Huffington Foundation gift also will enable the Institute to recruit another top researcher in the field of PD. Settembre, Sandoval and Park will be groomed to develop independent and expanded PD research programs. Collaborations within each team and among the three teams will help speed the process of discovery and development of new treatments for PD.
“My parents turned their attention to the needs of Houston’s elderly years ago when they established the Roy M. and Phyllis Gough Huffington Center on Aging at Baylor College of Medicine,” Terry Huffington said. “This investment at the Institute for research related to PD has the potential to impact not only the lives of elderly people in our community, but also people everywhere — young and old — with neurological diseases.”
W.M. Keck Foundation Funds Research into Neurodevelopmental Disorders
By Michelle Flippin
Girls with Rett syndrome — one in 10,000 — begin to experience loss of speech and purposeful hand movement and other symptoms within the first six to 18 months of their lives.
One of every 12,000 to 20,000 children born has Angelman syndrome, causing developmental delays, loss of movement or balance, speech impairments, and unique behaviors like frequent laughing.
While genetic defects have been linked to both, much remains to be discovered about each syndrome’s distinctive origin and development. Last year, the W.M. Keck Foundation invested $1,000,000 in a study at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital designed to push the frontiers of medical understanding of these diseases.
NRI Director Huda Y. Zoghbi, M.D., is working with Daoyun Ji, Ph.D., Jianrong Tang, Ph.D., Yuri Dabaghian, Ph.D., and Akash Patel, M.D., to analyze neural network activity in mouse models of Rett and Angelman syndromes. Theirs is the first study to use in vivo models of specific neurodevelopmental disorders to characterize disease-causing alterations in the neural network and to explore deep brain stimulation as a novel method for restoring normal function.
The multidisciplinary study at the NRI builds on a recent discovery in Zoghbi’s laboratory that highlighted the importance of inhibitory neurons for normal behavior and function in a model of Rett syndrome.
Characterizing the neural network activity in genetic models of these two disorders — which are considered prototypes for intellectual disabilities and autism spectrum disorders (IDs/ ASDs) — could reveal changes that underlie shared phenotypes (genetic traits). The preliminary data collected in this study of Rett and Angelman syndromes will allow the scientists involved to successfully compete for National Institutes of Health funding, and the understanding gained in these studies could eventually lead to treatments for multiple IDs/ASDs.
“The W.M. Keck Foundation’s gift helps us at the Institute move more quickly toward our ultimate goal of developing new interventions and treatments for individuals with IDs/ASDs,” Zoghbi said.
Disco Legends Set a Single-Night Fundraising Record
By Angela Dolder
In his book, You Gotta Dance Like No One’s Watching, Lester Smith shared six words that sum up his motto for life. “Anything worth doing is worth overdoing,” he wrote.
In 2012, Smith and his wife Sue lived up to this motto as chairs of the highest-earning, single-night gala in the history of Texas Children’s Hospital — An Evening with Disco Legends benefiting Texas Children’s Cancer Center.
These iconic Houston philanthropists (and champion ballroom dancers) not only underwrote all expenses for the evening but also matched each gift to the event through The Lester and Sue Smith Foundation, allowing every penny of the more than $30 million raised to support the Cancer Center.
“This transformational gift will accelerate pediatric cancer research initiatives here, including those that use genomic technologies to analyze samples and identify the specific genetic mutations responsible for the development and growth of tumors in pediatric cancer patients,” said David G. Poplack, M.D., director of Texas Children’s Cancer Center.
“Our goal is to study the biology of childhood cancers, how they develop, progress and spread,” said Poplack. “We also are studying why some tumors may respond to treatment whereas others do not.”
To honor the Smiths’ extraordinary generosity and years of support for pediatric cancer research and patient care, the outpatient clinic has been named The Lester and Sue Smith Clinic at Texas Children’s Cancer Center.
Smith, a two-time cancer survivor, rejoined Sue in the ballroom less than a year after completing treatment. In You Gotta Dance Like No One’s Watching, Smith recalls his personal struggles with cancer.
“My thoughts led me to all the things I would never get to do,” he said. “I’d never win another ballroom dance competition, climb Mt. Everest [or] chair a record-breaking gala. If anything, my cancer diagnosis spurred me on to crank up the heat and go for the gold. After more than eight years of hard work and fun, we made it to the top of our dance Mt. Everest.”
The Smiths were the 2002 and 2003 United States Grand Senior Latin Dance Champions.
“As chairs for Disco Legends, I hope that we transformed the shadow of cancer into a bright light of hope shining on the Cancer Center’s youngest warriors fighting the disease,” Sue said. “We knew that many children would be helped thanks to the tireless efforts of everyone involved with the magical evening that was Disco Legends.”
A standout moment at Disco Legends was hearing Gloria Gaynor’s ode to empowerment — “I Will Survive.”
“It’s such a great song with a lot of truth to it,” Lester said. “I will never forget the joy we felt that night dancing to that song — like no one was watching.”
From Hoops to Hope: The Juneau Family Helps Advance Congenital Heart Surgery
By Angela Dolder
Photo: Nhan Nguyen Photography
For as long as she can remember, 14-year-old Hailey Juneau has loved playing basketball. Last year, she was the starting point guard on her school’s team when she came down with a mysterious and aggressive cardiac condition caused by a virus that attacked her heart.
She required risky open-heart surgery, and the team that had one shot to save her was at Texas Children’s Hospital.
“Had she not received the expert care that this hospital provides, Hailey would not be here today,” said her father, Brad Juneau. “Without a doubt, Texas Children’s saved my daughter’s life.”
The surgeries included the installation of appliances that help Hailey’s heart maintain a normal rhythm.
“I have a pacemaker and defibrillator, a device that corrects abnormal rhythms of my heart and gives my heart an electrical shock if it needs it,” Hailey said.
In a gesture of thanks to Texas Children’s Hospital, Juneau and his wife, Melissa, have made a commitment of $2 million to establish the Brad and Melissa Juneau Endowed Chair in Congenital Heart Surgery. Jeffrey S. Heinle, M.D., Texas Children’s associate chief of congenital heart surgery and Baylor College of Medicine assistant professor of surgery, is the first to hold the newly established chair.
The Juneaus’ goal is to help ensure that the talented surgeons at Texas Children’s have the resources and tools they need to save more children like Hailey.
“We are grateful to the Juneau family for their support of congenital heart surgery and for their commitment to recruiting and retaining top talent to Texas Children’s,” said Charles D. Fraser, Jr., M.D., Texas Children’s surgeon-in-chief and chief of congenital heart surgery and Baylor College of Medicine professor of surgery.
“We feel blessed to give back to this wonderful hospital that did so much to heal Hailey,” Melissa said. “Supporting Dr. Heinle is an amazing opportunity for us because he was Hailey’s surgeon.”
While Hailey can no longer compete alongside her high school teammates, she still plays recreational games in her driveway. And she’s parlaying her hoop dreams into a shot at motivational speaking.
“First, I say push yourself — you can do more than you think you can,” Hailey said. “Then, I say to love your scars. Scars tell a story!
“I’m so thankful that my dad and my stepmom did something good for this hospital, because I love Texas Children’s. This hospital means so much to me.”