Success in CBD study: Cook Children's researchers play vital role
Meet the faces of groundbreaking cannabidiol oil study
When you get past the years of hard work, science and sacrifice of the families involved, the largest study in the world of cannabidiol (CBD) comes down to that one word.
Cook Children's participated in GW Pharmaceuticals latest Phase 3 clinical trial for Epidiolex, a cannabidiol-based oil used to treat children with Lennox Gastaut syndrome, a rare form of childhood epilepsy.
Patients taking 20mg of Epidiolex saw seizures drop an average of 42 percent compared to a drop of 17 percent in patients taking a placebo. Patients taking 10mg of Epidiolex experienced a 37 percent drop in seizures versues the 17 drop among those taking the placebo.
For Scott Perry, M.D., medical director of the Epilepsy Monitoring Unit and Genetic Epilepsy Clinic at Cook Children’s Jane and John Justin's Neurosciences Center, taking part in a study of this magnitude is the conclusion of more than a decade of his life’s work.
The study enrolled 171 children (average age of 15) from around the world with Lennox-Gastaut Syndrome. Ten of those patients were from Cook Children’s.
Of the patients who completed this trial, 99 percent have opted to continue into an open-label extension trial. The success has prompted GW to seek Food and Drug Administrative approval to make the drug available for prescription by physicians.
With the results from GW Pharmaceuticals showing positive results, there is hope that this may be the answer that parents with children who have hundreds of seizures a day have been looking for.
“The Epilepsy Foundation is thrilled to learn about the recent preliminary results for an innovative new therapy from GW for LGS. LGS in so many cases is extremely difficult to treat, and is an incredible challenge for children and families. We feel a tremendous sense of urgency to stop seizures, and believe that the pursuit of new therapies offers hope to individuals who have no currently available therapy to effectively stop their seizures. The Epilepsy Foundation will continue to be a champion for GW’s efforts to pursue this innovative new therapy as studies progress. We thank GW and all our partners who invest in a better tomorrow for people with epilepsy,” stated Philip Gattone, President and Chief Executive Officer of the Epilepsy Foundation.
Further data will be presented in future publications and medical meetings.
Cook Children’s has participated in three studies that examine CBD as a treatment for severe cases of epilepsy. The worldwide studies gathered data to see if CBD is an effective form of treatment for two uncontrollable forms of epilepsy – Lennox-Gastaut syndrome and Dravet syndrome.
To learn more about the use of CBD, four families graciously agreed to allow us to follow them during the trial, involving Lennox-Gastaut syndrome. These are their stories.
At 15 years old, Vanessa Castro was a teen mother. Her daughter, Jordyn, was diagnosed with a rare form of epilepsy Lennox-Gastaut syndrome when she was an infant
Through it all, Vanessa never gave up on her daughter and her fight to see her little girl get better.
At 14 years old, Jordyn had the developmental abilities of an18 month old. She suffered 50 to more than 100 seizures per day and lived on multiple medications, none of which worked.
"If you’re a parent of a child with epilepsy, you know the routine: you start a new medication and the seizures reduce (if you’re lucky) and then a few months go by and they start to increase again," Vanessa said. "Over the years, your hope starts to diminish and you come to realize that this is your child’s life. Not to mention you live in constant fear because you never know if or when that one seizure will come and take your child from you."
Following brain surgery, Jordyn had a stroke that left her paralyzed. After months of rehab, she regained the ability to walk again. Unfortunately, the seizures continued.
Jordyn could only go a day or two, without having a seizure. But there were also days with more than 50 seizures. At a neurology appointment, Dr. Perry approached Vannessa about the CBD study.
The first appointment went from 9 a.m. to 5 p.m., and that was just the screening to make sure Jordyn could even partake in the program. Multiple day-long appointments followed. The study's structure was a challenge for the single mom.
"I have to call every day between 8 p.m. and midnight to report the type and quantity of seizures, which takes about 5-10 minutes," Vanessa said. "If I miss more than two calls, we are kicked out of the program. I work full-time and have two children, so my time is managed wisely. Remembering to call has been a challenge because by the time I’m done with family time, it’s 9 p.m. and I’m ready for bed. But I continue to try my best because my daughter deserves it."
But as time progressed, Vanessa saw results. It began with her daughter going four days with only one seizure. Then she went one full week seizure-free.
Even though this was a blind study, meaning she wasn’t sure if her child was actually receiving the drug or a placebo, Vanessa had little doubt her daughter was taking the medication because of the amazing success she saw throughout. Now, in the next open-label phase, she knows her daughter is taking the medication.
Although it may be the norm for most moms, watching her daughter walk to the bathroom and turning on the light by herself is nothing short of miraculous.
From 2013 to before the trial, Rikki Adan took at least 13 medications to control his seizures.
Rikki suffered his first seizure at 3 months old in February 2013 and finally after battling Lennox-Gastaut syndrome for nearly three years, the family learned they would be a part of the CBD trial.
"We were all happy that maybe, just maybe, this medication would help my son,” said Sandy Adan, Rikki’s mother. “But sad, scared, upset and nervous also. But words of wisdom from my Jasmine, my daughter helped us. 'Mom, you have given him so many other medications, what if this is the one? If we don't try it, we'll never know.'”
The family began the study in August 2015. The first 30 days were hard, keeping a log of every seizure. For Sandy, mother of four, it made it difficult to keep up with a busy household and continue to chronicle Rikki's seizures.
The seizures impact the entire family. Virginia, age 12 and Rikki's sister wrote, "When I was first told my baby brother had epilepsy, I was really worried. I didn't really know what epilepsy was. When it was explained to me, I understood, but I still questioned why? I know that it is completely OK to be worried, but we still have to stay strong. He is a blessing and we will never give up on him."
The family made it through the trial and continue to take the medication. They are seeing results. Although his seizures aren't fully controlled, Sandy said she has seen a significant change in the severity and frequency of the seizures.
Cody Tabor, 12 years old, hit all of the milestones one would expect from a normal, well-developed child as a baby. Then, just like that, everything changed.
When he was 19 months old, Cody woke up one Sunday morning with a high fever. His mom, Tess, took him to an ER in Jasper, Texas, and he was admitted. He received antibiotic shots every four hours.
The next day Tess believed that Cody was on her way to recovery and she would be going home soon. Then she got one of the great shocks of her life.
“As I turned to pick up the phone, I heard a noise,” Tess said. “I turn back around to look at Cody and he was in the rocking chair. He had the most terrifying look on his face. To this day, I will never forget that look. It was the scariest thing I have ever seen in my life. I picked him up ran out of the hospital room screaming, ‘What is wrong with my baby? What is wrong with my baby?’”
Cody was having a seizure. The medical team ran back in the room with Cody, trying to help him. Tess stayed out in the hall scared and crying.
After his initial seizure, things seemed to be snowballing downhill for Cody. He had side effects to a medication that gave him near paralysis. He couldn’t move, even to roll over. Tess said it was “like all the muscles in his body just disappeared.”
In a matter of a few days, Tess, a single mom at the time, watched her normally developing baby suddenly having hundreds of seizures at a time.
Doctors told Tess that Cody did not have much longer to live and that everyone should begin saying their goodbyes.
Tess credits her faith for helping her cope with the way her life had suddenly turned upside down. For the next couple of years, Tess and Cody spent more time in a hospital in Houston than they did at their own home.
Life got better for Tess, but much more hectic. She married her husband, Tommy, and adopted his three kids. They moved to Lubbock, Texas in 2007.
Things were looking up for Cody too. He was going to school, taking speech therapy, physical therapy and occupational therapy. But he still suffered from seizures. After one particular bad one, he was air lifted to Cook Children’s where “we found the providers we love,” Tess said. “So that is where we stay to this day.”
It was then that Cody met Dr. Perry, who diagnosed Cody with Lennox-Gastaut syndrome. From there, other diagnoses came fast – cerebral palsy, developmental delays, innocent heart murmurs and immunoglobulin A (IGA) deficiency.
Tess was frightened at what she had learned about Lennox-Gastaut and yet relieved that at least now this thing that had plagued her son had a name and maybe they could fight it.
The next six years were filled with frustration and struggles. Cody’s treatment included trials of 13 different seizure medications twice daily, but the seizures continued.
It got to the point where Tess felt there was nothing left to do for her son.
“He has been on every medication that I am aware of out there, he has had the surgery to separate the two sides of his brain, he has had the vagal nerve stimulator surgery and nothing has worked,” Tess said.
She would try anything to bring back the little boy she’d known before the seizures began.
Then last fall, she found her first glimmer of hope in a long time when Dr. Perry told her Cook Children’s would be in a clinical trial for cannabidiol treating Lennox-Gastaut.
“My biggest concern about the trial was failure,” Tess said. “I was scared that the oil would not work or help him. If this oil fails then what happens to my son? Where do we go from here? How does he get better? These are questions that I have every day, scared to death that this oil is going to be another failed attempt to help him. It has to work. It just has to.”
Tess said her prayer is a simple one – for Cody to have a better life in some way being big or small. And she also hopes that this trial will make a difference for children in the future.
“I want every parent to be able to have the choice in trying the oil when nothing else has worked,” said Tess. “I want what we are doing to make a difference and make a pathway for others. The oil can help with so much more than seizures. It can help others out there that hit a wall with traditional treatments.”
Cody is currently in the open-label trial.
“Cody has not shown as much improvement as other participating patients in the trial,” Dr. Perry said."Unfortunately, this suggests not every patient is going to improve significantly on CBD treatment."
When Jaxon Lane Huffman was born on Dec. 17, 2010, he looked to be perfectly healthy boy.
At 2 ½ months old,Jaxon's parents woke up to notice he was having "weird movements" while he was sleeping. Doctors told the Huffmans that their son was probably suffering from acid reflux.
The Huffmans began to video Jaxon's movements while he was sleeping. After confirming he was having seizures, Jaxon's pediatrician sent the family to a hospital where he could be monitored for epilepsy.
Later that day, Jaxon was diagnosed with simple partial seizures with an unknown cause at that time. Jaxon was prescribed Phenobarbital for his seizures. He responded well to the medication, and continued to develop like any other baby boy would.
He was seizure-free on the medication, and his parents were hopeful that this was the end of the seizures for their son. Little did they know, that this was only the beginning for Jaxon. Things would soon get worse.
At 6 months old, Jaxon's seizures began again and this time he was having as many as 100 consecutive spasms. The seizures increased in length and he was diagnosed with infantile spasms (sudden bending forward of the body with stiffening of the arms and legs).
Neurologists tried several medications, but Jaxon suffered several side effects, including high blood pressure, a swollen face and weight gain. Frustrated, the Huffmans took their little boy to Cook Children's.
Dr. Perry found that Jaxon had atrophy of his brain and abnormalities in several areas.
"I cannot describe the feeling when we heard the news," Jennifer said. "As a parent, you have these hopes and dreams for your child to live a wonderful life. Not only was our child not developing, but now we find out that his brain is not developing properly. All we knew to do from that point on was to pray.”
Jaxon tried numerous treatments and was up to four seizure medications or more at a time, but the seizures continued. He also participated in physical therapy, occupational therapy and speech/feeding therapy two times each per week.
At age 4, Jaxon had very little head control, no trunk control, no use of his arms or legs, and was non-verbal. He had no muscle tone, and was not able to sit, crawl, stand or walk. He had 20-40 cluster seizures every day, which consisted of up to 100 spasms per cluster at times. He had three to five grand mal seizures daily and in some cases, stopped breathing.
After much discussion with Dr. Perry, Jaxon was referred to palliative care. "We have exhausted all treatment options for Jaxon at this time," Jennifer said. "We are asking for the opportunity for Jaxon to be able to try CBD. This is the only option left for Jaxon.”
Following the first 12 weeks of the trial, Jennifer saw little difference in Jaxon. Convinced that her son had been on the placebo, the family continued the next phase of the trial. This time they knew Jaxon would receive the CBD oil.
Since going on CBD, Jennifer has seen Jaxson's seizures decrease by as much as 50 percent. Jaxon still has clusters of seizures, but they are dramatically reduced. Where he once had 20 to 40 clusters a day, he now has an average of about six or seven. It now takes a week or more to have the same amount as he had in one or two days previously. He hasn't had a Grand mal seizure in close to three weeks.
The decrease in seizures has brought a sense of peace and normalcy that the Huffmans hadn't experienced in a long time. Jaxon is moving more, making noises and laughing more. He has stood in his walker and even moved it for the first time since the family brought it home when Jaxon was 2 years old.
Perhaps, most importantly, he's sleeping through the night on most nights. Before, the family, including Jaxon's brother and sister, would wake up because of seizures during the night.
"It's definitely been less stressful," Jennifer said. "Getting through most nights without the constant interruption of seizures has been a huge help for all of us. Knowing that he's getting rest and a good night sleep, honestly, that's the thing that means the most to us right now."
Jennifer said she went into the trial with realistic expectations. She didn't expect it to be cure all, but hoped that it would improve Jaxon's quality of life and that's what she has gotten for her little boy.
"I would have loved for it to have completely stopped his seizures," Jennifer said. "That would have been great. But I knew realistically that probably wasn't going to happen. Knowing that going in, this has not felt like a disappointment for us. We treasure all the little victories we've had during the trial."
Dr. Perry said the inititial research into CBD as a treatment of epilepsy is likely just the tip of the iceberg. He said there's enough evidence to suggest that CBD warrants further investigation as a therapy, but not enough enough evidence, yet, to wholeheartedly support it for more common uses until all the reserach is done.
"If the studies prove positive, there will likely be a whole new class of cannabis-derived drugs in the pipeline for the treatment of epilepsy and Cook Children’s will remain intimately involved in researching those treatments," Dr. Perry said. "Research to discover cures for epilepsy is a primary focus of the Cook Children’s Comprehensive Epilepsy program."