November 11, 2014

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When you think about our outstanding neurology team at Texas Children’s Epilepsy Center – one of the largest Level 4 epilepsy centers in the nation – one particular attribute comes to mind: their passion for innovation.

“Our focus has always been to discover new and innovative ways to treat and cure children with various forms of epilepsy,” said Dr. Angus Wilfong, a Texas Children’s neurologist who is leading the hospital’s first worldwide clinical trial of a cannabis-derived experimental drug to treat pediatric Dravet Syndrome.

The syndrome, which occurs in about one in every 30,000 births, is a rare and catastrophic form of intractable epilepsy that causes patients to experience dozens or even hundreds of seizures a day. If left untreated, children struggle with developmental delays, serious health complications and death.

That’s the grim reality 6-year-old Izaiah Ruiz faces daily. His grandmother, Lori Fountain, says Ruiz’s epilepsy is so severe that despite taking numerous seizure medications, nothing seems to produce long-term relief. Ruiz’s first seizure occurred when he was 2 months old, and since then, he’s almost lost his life twice. “I want him to be a normal little boy without having to worry about a seizure every time he goes to the playground or when he gets a runny nose,” said Fountain.

Ruiz is one of 30 patients worldwide enrolled in Texas Children’s study to examine the safety and efficacy of Epidiolex®, a highly purified cannabis extract that contains pure Cannobidiol (CBD). Although it is derived from the cannibas plant, Epidiolex contains no tetrahydrocannabional (THC), the psychoactive chemical in marijuana. GW Pharmaceuticals, who will fund the trial, is the only company in the world that produces pure plant-derived CBD, which is believed to help control seizures in children. The CBD medication is administered orally as a suspension or via feeding tube.

Wilfong, who is the study’s lead investigator and the first physician to administer Epidiolex in Texas, says the trial will be conducted in two stages. The first phase will determine the ideal dosage for children with Dravet Syndrome and the second phase will evaluate the drug’s safety and efficacy in this patient population.

“We are hopeful that in the next year, the results of this trial will show this drug has a positive impact on enrolled patients and that it will have implications for patients with other forms of intractable epilepsy,” said Dr. Gary Clark, chief of Neurology and Developmental Neuroscience at Texas Children’s.

Texas Children’s first began using Epidiolex in April of this year under a compassionate use protocol approved by the Food and Drug Administration for patients with catastrophic forms of epilepsy who were not responsive to seizure medications.

Wilfong said, “Initial trials of Epidiolex demonstrate promising signs of efficacy in children with treatment-resistant epilepsy. We are excited to partner with GW Pharmaceuticals in the first worldwide trial for this group of patients with such a catastrophoic form of epilepsy.”

Fountain hopes her grandson receives the experimental CBD medication rather than a placebo, but she knows that if he doesn’t, he’ll get the drug soon thereafter.

“If this drug could reduce Izaiah’s seizures and give him any semblance of a normal life, I can’t tell you how ecstatic I’ll be,” said Fountain.

Currently, Texas Children’s Epidiolex trial is accepting only pediatric patients with confirmed Dravet Syndrome. To learn more, click here or call Christina Tally, BS, CCRP, at Ext. 2-1276.

Click the link for more information about Texas Children’s Epilepsy Center.

August 26, 2014

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It wasn’t a typical Monday morning for Dr. Timothy Lotze, a neurologist at Texas Children’s. Besides making his usual hospital rounds to see patients, Lotze paid a visit to 14-year old Joseph Garza’s room. But, this visit was extra special.

Trading in his white lab coat for a comfy red T-shirt, Lotze participated in the Ice Bucket Challenge to help raise money and awareness for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that severely impacts a person’s ability to move.

Lotze accepted the challenge from Texas Children’s Muscular Dystrophy Association representative Dalia Deleon on behalf of Garza, who suffers from an extremely rare form of pediatric ALS.

View the video:

“I am so grateful to Dr. Lotze for taking on this challenge to help raise awareness for ALS,” said Lorrie Garza, Joseph’s mom. “Hopefully, they’ll find a cure so no other family or child has to go through this terrible disease.”

A year before Garza’s diagnosis, Lorrie noticed her son was having trouble lifting his arms and began walking with a limp. His condition grew worse, but doctors in his hometown of Brownsville weren’t sure what type of neurological disorder he had. Once Garza was transferred to Texas Children’s, he and his family met with Dr. Lotze, who diagnosed Garza with ALS in May of this year.

While ALS primarily affects adults, there are children who suffer from this debilitating disease that attacks nerve cells in the brain and spinal cord which control voluntary muscle movement. Patients with ALS progressively lose their ability to eat, speak, walk and eventually breathe.

“Since the disease impacts voluntary muscle action, patients in the later stages of ALS may become completely paralyzed,” said Lotze. “It’s important for us to continue to support research and raise community awareness about ALS so we can find a cure for this horrible disease and improve the quality of life for these patients.”

That’s exactly what Texas Children’s researchers Dr. Hugo Bellen and his team strive to accomplish. In their laboratory at the Jan and Duncan Neurological Research Institute at Texas Children’s and Baylor College of Medicine, they are examining the role of genes in ALS to help find ways to suppress degeneration and improve a patient’s functionality.

“A subset of ALS patients have mutations in a gene called VapB,” said Bellen. “We are trying to understand the specific role of this gene by studying fly and mouse models of ALS, caused by mutations in this gene.”

Bellen and his team have discovered that the VapB protein circulates in the blood and functions as a hormone. It binds to receptors on muscle cells to control the function of the muscle mitochondria, the cell’s powerhouse. In the mutants, the muscle mitochondria do not function properly anymore, and this in turn affects the function of the neurons that innervate the muscles, ultimately causing these neurons to die.

“Although the causes of ALS are not well understood, we are making significant progress on how the disease may develop, which could lead to better therapies or possibly a cure in the future,” said Bellen.

As for Garza, his mom says he’s not giving up hope. “He’s already told me he’s going to fight,” said Lorrie. “And I tell Joseph, “We will fight together.”

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Texas Children’s President and CEO Mark A. Wallace took a fun break to raise awareness for a very serious cause on Thursday. Watch him take the ALS ice bucket challenge and see who he’s called out.

August 5, 2014

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It was off to camp for several members of Texas Children’s pediatric neurology team and their patients. They made the trek to Camp For All, a beautiful, barrier-free recreational facility in Burton that helps children with special needs experience the thrill of camping and nature, just like normal kids their age.

Each summer, Camp For All hosts Camp Spike ‘n’ Wave, a weeklong camping expedition for children and teens with epileptic seizures. They build new friendships, unleash their independent, adventurous spirit, and focus less on their disability.

“We don’t want their medical condition to completely define who they are,” said Dr. Gary Clark, the camp’s lead physician and chief of Neurology and developmental neuroscience at Texas Children’s. “Wheelchair bound or not, every child can accomplish just about anything they set their minds to.”

Unlike traditional camps, Camp For All has no barriers. The entire 100,000 square foot facility – including cabins, bathrooms, showers, and sidewalks – are completely wheelchair accessible. The sidewalks are wide enough to accommodate side-by-side wheelchairs. And, every child is able to participate in all activities, regardless of the severity of their disability. If a wheelchair bound child wants to canoe, the child is placed in a sling and lowered into the canoe with a crane so they can explore nature on the water, just like their able-bodied peers.

“I will never forget how one of our wheelchair bound patients got to go on the big swing, which is essentially a big zipline,” said Sterling Myers, a camp volunteer and pediatric neurology nurse at Texas Children’s. “It made me smile to see the campers cheer each other on at every activity and boost each other’s self-esteem.”

More than 120 children with epilepsy participated in adrenaline-pumping activities like rockwall climbing, zip lining, horseback riding, rope courses, archery, biking, fishing, canoeing, swimming, karaoke, and arts and crafts.

8614Campinside640“It was great to see their fearlessness,” said Dr. Shannon Dicarlo, a pediatric neurology physician and former fellow at Texas Children’s. “They didn’t let their epilepsy get in the way of going about their lives, making friends, and having fun.”

Behind the scenes, our Texas Children’s medical team made sure the kids received superior care away from home. Every child at camp required medication for their seizures. Texas Children’s pharmacists filled and labeled prescriptions onsite according to the patients’ electronic medicine administration record generated at the hospital.

“When you think about it, we operated a mobile hospital right here at the campground,” said Clark. “It’s all about meeting our children’s needs.”

Camp Spike “n” Wave provides unique training opportunities for fellows to observe how their clinical decisions can impact their patients. It’s not every day that physicians see patients have seizures or struggle to swallow a fistful of pills. When these happen, highly trained camp counselors, along with Texas Children’s physicians and nurses, spring into action.

“This year, we trained a 6-year-old how to swallow pills,” said Dr. Geetanjali Rathore, a pediatric neurology fellow at Texas Children’s. “He shed a few tears the first time, but by the end of the week, he became a champ at swallowing pills.”

Camps, like Camp Spike ‘n’ Wave, wouldn’t be possible without the diligent efforts of Texas Children’s own, Dr. Robert Zeller, chief of the Blue Bird Circle Clinic, who collaborated tirelessly with other physicians and organizations to create Camp For All in 1993.

“I had a patient with epilepsy who couldn’t go to camp because camps wouldn’t accept children with this condition,” said Zeller. “This prompted me to develop a camp for disabled children who can discover life without barriers. It’s my way of giving back to my patients.”

April 29, 2014

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In the summer of 2012, a then 6-year-old little girl, Mayah Villarreal, began to write with her left hand instead of her right. Her kindergarten teacher mentioned it to Mayah’s mom, Ashley, but told her not to worry as it was common in young children. A few weeks later while taking a bath, Ashley noticed that Mayah began to lean her body to the right and could not sit up straight. Soon she could not lift her right leg on her own to do simple, everyday tasks. Mayah, a formerly active young girl who loves gymnastics, had never shown any signs of a movement disorder and now her life had come to all but a halt, as she was unable to function normally.

Mayah was originally diagnosed in her hometown of Alton with a chiari malformation, and doctors recommended that Mayah undergo a craniotomy surgery, which she had in October 2012. A few days prior to the surgery, Mayah had stopped walking completely and was suffering from debilitating headaches.

After the surgery, there was no improvement in Mayah’s condition. In fact, it continued to worsen and she landed in a local hospital where she was admitted for two days and was told her condition was psychological. One of the doctors on call at the time called Texas Children’s Hospital and after a brief conversation, the Kangaroo Crew team picked Mayah up and flew her to Houston.

Mayah was admitted to the Blue Bird Circle Clinic where they diagnosed her with an extremely rare pediatric movement disorder called dystonia. Dystonia from each individual genetic etiology is rare, and in DYT 1, which is what Mayah has, the disease frequency in Ashkenazi Jews is estimated at 1:3000-1:9000. Among non-Jews, the prevalence is lower.

The team at the Blue Bird Circle Clinic, including Dr. Amber Stocco, former director of the hospital’s movement disorder clinic and Dr. Daniel Curry, director of pediatric surgical epilepsy and functional neurosurgery, developed a course of care that included an aggressive and innovative treatment plan. Stocco recommended deep brain stimulation (DBS) to treat Mayah’s condition. Commonly used for Parkinson’s patients, the use of DBS in pediatric patients is still limited as there are only a few experienced centers worldwide and few patients meet the criteria to qualify for DBS.

“Until recently there have been only case reports of DBS in children,” said Stocco, of this innovative approach. “It is starting to gain acceptance in pediatric centers; however, to date, there are likely less than 300 cases of DBS in children internationally.”

In order to administer DBS to Mayah, Stocco, Curry and the team implanted a small, programmable battery in the upper chest region, near her shoulder. The battery connected to a series of electrodes that targeted specific parts of her brain to block the abnormal brain activity that prevented her from controlling her own muscle movements.

Soon after surgery, Ashley noticed a change in Mayah’s behavior. “Soon after surgery, before the device was even fully programmed to a higher level, we saw Mayah’s condition improving. Every day, we watched Mayah become more and more independent.”

On August 10, 2013, Mayah walked unassisted for the first time since the summer of 2012.

Thanks to Dr. Stocco and Dr. Curry’s innovative approach, Mayah’s case proved to be an extraordinary outcome and story of success. The DBS has returned Mayah’s motor skills to normal and she is happily back enjoying gymnastics. Her implant requires minimal maintenance, needing a charge for just a few minutes every week.

Mayah’s case is amongst the most remarkable of recoveries. Until recently there have only been case reports about using DBS in children and is now starting to gain acceptance among pediatric hospitals, with Texas Children’s at the forefront.

April 22, 2014

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The BP MS 150 is a two-day bicycle ride from Houston to Austin organized by the National MS Society: Lone Star Chapter to raise funds to fight against the devastating neurological disorder multiple sclerosis. Since 2006, Texas Children’s Hospital has sponsored its own team of riders who have helped raise more than $1.53 million to date for the cause.

In 2014, Team Texas Children’s consisted of 218 registered riders, with 183 actually completing the ride on April 12-13. The 2014 team will raise more than $220,000 this year. In addition to the riders, approximately 100 employees and community volunteers gave their time to hand out snacks, haul luggage, blow up air mattresses, set up the overnight tent in La Grange and provide other much-needed and much-appreciated support.

Did you know?

  • The BP MS 150 is the largest event of its kind in North America.
  • The first MS 150 Houston to Austin bike tour was held in 1985, with 237 cyclists raising just over $100,000.
  • Since 2007, the wildly popular event has been capped at 13,000 riders.
  • Since surpassing the $15 million mark for the first time in 2008, the BP MS 150 has raised an average of $16 million per year.
  • This year’s ride is expected to raise around $18.5 million for MS.
  • A “fashion show” of sorts, teams compete to have the most creative, colorful and distinctive jersey designs.
  • Team Texas Children’s is among the most popular and most recognizable, winning multiple competitions for Best Jersey in recent years.
  • In 2014, four of the top 300 fundraisers rode on Team Texas Children’s.
  • In 2014, Team Texas Children’s riders ranged in age from 12-74 years old, with seven children under the age of 18 participating.