The first Texas dispensaries are now opening and selling their products to eligible patients across the state. But Texans who are interested in pursuing the so-called CBD oil treatment say there are several obstacles to getting the medicine.

There’s roughly 30 doctors across the state eligible to prescribe the medicine. Also, CBD oil is still federally illegal, so people have to pay out of pocket to secure it. And there are only three Texas dispensaries who can sell the medicine — two in Austin and one in Schulenburg.

In this video documentary, The Texas Tribune followed two families as they began their journey in pursuing medical cannabis treatment.

Full story here.

A panel convened by the FDA voted 13-0 that the agency should approve the drug as treatment of these two rare forms of epilepsy. The FDA will make a final decision to approve the medicine by June 27.

Cook Children’s participated in several of GW Pharmaceutical’s clinical trials of Epidiolex used to treat children over the age of 2 with Lennox Gastaut and Dravet and continues to enroll children in a trial of the drug for treatment of seizures in Tuberous Sclerosis Syndrome. “The trials were the largest of their kind in the world and have provided the gold-standard evidence needed to prove cannabidiol can be effective as a treatment for epilepsy,” said M. Scott Perry MD, Medical Director of Neurology and principle investigator for the trials performed at Cook Children’s.

The FDA gave the drug a favorable review, stating that it provides “substantial evidence” of the drug’s effectiveness in treating Lennox-Gastaut syndrome and Dravet syndrome.

“Although the review is still ongoing, the risk-benefit profile established by the data in the application appears to support approval of cannabidiol for the treatment of seizures associated with LGS [Lennox-Gastaut syndrome] and DS [Dravet syndrome]," the FDA said in a report.

Dr. Perry explains that Epidiolex is an essentially pure CBD substance produced under strict standards to ensure the drug is the same with every batch. Once approved, the drug would be obtained from a pharmacy, prescribed by a doctor and more likely to be covered by insurance.

“My hope is this will be the first of many drugs derived from the cannabis plant,” Dr. Perry said. “This drug has gone through a rigorous process to be approved and I think it will help people feel safe taking the medication. I hope it also encourages more research into what the hundreds of other substances in the cannabis plant can do for patients.”

Previously On This Topic:

• 9 Facts about Cannabidiol (CBD) Oil and the Texas Compassionate Use Act
• Texas legalizes non-euphoric cannabdiol for seizures in epileptic patients
• Drug in Cook Children's epilepsy trial shows positive results in separate trial
• Success in CBD study: Cook Children's researchers play a vital role
• Cannabis oil trial ongoing at Cook Children's
• Study: Cannabis Oil Can Dramatically Decrease Epileptic Seizures

Compassionate Cultivation will be the first of the three licensed dispensaries to open in the state, but will be followed, in theory, by the other two companies that received licenses last year under the Texas Compassionate Use Act, which was passed in 2015.

Watch the story here. 

The bill required DPS to license at least three dispensing organizations by Sept. 1, 2017, should they meet requirements.

On Feb. 8, 2018, the first medical dispensary in the state of Texas will open.

What does all of this mean for children with epilepsy?

1. What is it in the cannabis plant that helps treat epilepsy?

The truth is we don’t yet really know. There are over 500 chemical compounds in the cannabis plant and over 100 of them are plant-based cannabinoids (phytocannabinoids) which may interact with the endocannabinoid system of the human body and treat disease. Cannabidiol (CBD) is a non-psychoactive component (i.e. does not produce a “high” feeling) and has been most widely studied so far for epilepsy. It does not interact with either of the two known cannabinoid receptors in the brain and its mechanism is still unclear. Tetrahydrocannabidiol (THC) is the component in the plant that produces a high and does interact with the cannabinoid receptors, but for many reasons, is not likely to be a good choice as an antiepileptic compound.

2. What is CBD oil and what types of CBD oil are now legal in Texas?

A CBD oil is an oil made from a cannabis plant that is high in CBD and typically lower in THC. The oil may contain amounts of multiple other compounds found in the plant as well. The 2015 Texas Compassionate Use Act allows for “high” CBD (more than 10 percent by weight)/”low” THC ( less than 0.5 percent by weight). This is slightly higher than the amount of THC (less than 0.3 percent) allowed in a plant to be considered hemp. Hemp oils are produced from these lower THC plants and are often a poor source of CBD but have been legal and widely available.

3. People on the Internet already say CBD oil works, so why aren’t all doctors OK with using it over typical medications prescribed for seizures?

All FDA approved antiepileptic medications have gone through rigorous studies to prove they work and they are safe to use. The studies are blinded and placebo-controlled which means neither the doctor nor the patient knows whether they are getting the real drug or a placebo until the study is complete. This helps remove bias (i.e. people feeling like their seizures are better because they know they are taking the real drug). It also helps separate which side effects are due to the drug and which occur randomly in the course of life (for example fevers, rash, nausea which can occur commonly regardless). CBD has only recently been tested in this type of rigorous study – more on this later – and the other components of the plant lack this type of research so far.

4. If CBD has already been studied, why are some doctors still hesitant to recommend CBD oil?

When people are treated with typical antiepileptic drugs, they are taking a single compound, like carbamazepine or levetiracetam for example. When you get a CBD oil, you get a substance that may have a high amount of CBD, but it also can contain varied amounts of the multiple other compounds from the plant. We do not know how effective and safe those other compounds are, so we must be cautious to recommend it to treat your child. Currently, there are no testing standards to make sure the CBD oil you get from a company contains what the label says, thus the formulation may change some from month to month – something akin to getting a different generic antiepileptic medication each month. While all CBD oils contain the similar CBD compound, they may be produced from different types of plant, using different growing techniques/conditions, and manufactured with different processes – thus creating different medications with different other compounds included. Think of it as being prescribed a sodium-channel drug like carbamazepine, but potentially getting one of multiple other sodium channel drugs such as oxcarbazepine, phenytoin, rufinamide, lamotrigine, etc. So doctors aren’t necessarily averse to CBD oils, we just have to be realistic about their limitations.

5.What did the studies of CBD show?

First, understand that the Greenwich Biosciences CBD compound Epidiolex is not the same as the CBD oil you get from a dispensary. It is a nearly pure CBD substance produced under strict standards to ensure the drug is the same with every batch. In 120 patients with Dravet syndrome, 43 percent on CBD had more than 50 percent reduction of convulsive seizures versus 27 percent on placebo. This was actually not a statistically significant difference. In 171 patients with Lennox-Gastaut syndrome, 44 percent on CBD had more than 50 percent reduction of drop seizures versus 23.5 percent on placebo and this was significant. From these results we can conclude that CBD does appear to have some antiseizure effect, but it is not necessarily any better than most other available drugs. It is also not without side effect, as diarrhea (19%), sleepiness (15%), fever (13%), decreased appetite (13%), and vomiting (11%) were reported. So CBD is an option for epilepsy treatment, but is not likely to cure every patient.

6. How does the Texas Compassionate Use Act work?

Under this law, doctors can register to recommend CBD oils only to patients with intractable epilepsy. To register, a doctor must be board certified in Neurology or Child Neurology and must also be certified in Epilepsy/Neurophysiology or spend more than 50 percent of their practice treating patients with epilepsy. If a registered doctor feels CBD oil is appropriate for their patient because the patient has tried all reasonable, better-studied and available treatment alternatives, the doctor can recommend an amount of CBD to be taken. This recommendation must be approved by a second registered doctor who reviews the case and agrees with the decision. Registered patients can then go to one of three dispensaries in the state to get a CBD oil.

7 .Will the CBD oil be covered by insurance?

No. As this is not an FDA approved treatment, it is unlikely any insurance company will cover the cost. The price is typically several hundred dollars per month depending on the amount of CBD recommended.

8. Now that we have this law, it is legal right?

The cannabis plant and all the components of the plant remain a schedule 1 substance on the Drug Enforcement Agency’s list- meaning it has no medicinal value and is illegal. CBD is part of the plant and still falls under this category for federal law despite what a state law may say. While the 2013 directive from the Department of Justice indicated they had better things to do than prosecute people following the laws of their state, Jeff Sessions reversed this opinion several weeks ago – thus it is unclear how the federal government will choose to deal with CBD oils now.

9. When will a pharmaceutical grade CBD oil be available?

Greenwich Biosciences has submitted their drug, Epidiolex, to the FDA for approval December 2017. While the FDA could take years to rule, they have indicated their plan is to rule on approval by summer 2018. Once approved, this drug would be obtained from a pharmacy, prescribed by a doctor, and more likely to be covered by insurance.

Previously on this topic:

• Texas legalizes non-euphoric cannabdiol for seizures in epileptic patients
• Drug in Cook Children's epilepsy trial shows positive results in separate trial
• Success in CBD study: Cook Children's researchers play a vital role
• Cannabis oil trial ongoing at Cook Children's
• Study: Cannabis Oil Can Dramatically Decrease Epileptic Seizures

"I don’t know that she’s ever had a time where she’s been completely seizure-free," says her mother, Laci.Kadience has a genetic disorder called Tuberous Sclerosis, or "TS."

It affects about one in every 4,000 people, says Dr. M. Scott Perry of Cook Children's Medical Center. He says it affects almost every single organ system and often leads to debilitating seizures.

Read full story here. 

The Food and Drug Administration has published results of an ongoing study that found cannabis oil can dramatically decrease the number of seizures in some epileptic children.

A Fort Worth family, who is part of the trial, says it changed their lives.

Read the full story here.

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.

GW Pharmaceuticals announced positive results for the completed study of adjunctive Epidiolex (cannabidiol) in the treatment of drop seizures in patients with Lennox Gastaut syndrome (LGS, GWEP1414).

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.

Jordyn's story

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.

Rikki's story

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’s story

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."

Jaxon's story

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."

The future

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."

Learn more:

• Scott Perry, M.D.
• Jane and John Justin Neurosciences Center 
• Drug in Cook Children's epilepsy trial shows positive results in separate trial
• Texas legalizes non-euphoric cannabidiol for seizures in epileptic patients
• Cook Children's Epilepsy Monitoring Unit

Not a day goes by that Scott Perry, M.D., doesn’t hear that question from parents desperate to help their child. Dr. Perry says he probably discusses the pros and cons of marijuana use for intractable epilepsy at least three or four times a day.

Those questions may increase with the news an experimental cannabis-based drug has successfully treated children with a rare form of severe epilepsy, known as Dravet syndrome broke today.

GW Pharmaceuticals announced positive results from a 120-patient trial on Monday, March 14, 2016. The study shows that patients who took the drug known as Epidiolex saw a monthly reduction of convulsive seizures of 39 percent, compared to a reduction of 13 percent in patients on placebo.

Cook Children’s did not participate in this particular study, but Dr. Perry is leading a team studying the same drug with the same company in three additional trials of Dravet syndrome and Lennox Gastaut Syndrome.

“Many parents of children with epilepsy are interested in considering marijuana, but are afraid of the response they will get from their doctor when they ask,” said Dr. Perry, medical director of the Epilepsy Monitoring Unit and Genetic Epilepsy Clinic at Cook Children’s. “While providers are increasingly more open to the idea that marijuana has some place in medicine, many others continue to regard it as nothing more than an illegal drug with no health benefit.”

Cook Children’s is involved in two studies that examine cannabidiol (CBD) as a treatment for severe cases of epilepsy. The studies, the largest of its kind, are being conducted nationwide in an effort to gather data to see if CBD is an effective form of treatment for two uncontrollable forms of epilepsy – Lennox-Gastaut syndrome and Dravet syndrome.

For Dr. Perry, taking part in a study of this magnitude is the conclusion of more than a decade of his life’s work. It’s also been a passion of his, testifying in front of politicians and lawmakers. Not serving so much as an advocate for CBD, but more so as a scientist and caregiver. He wants to know is this works.

“Without doubt, there is a lot we don’t yet know about how marijuana can be used as a medical treatment, which diseases is it best for, at what doses, at what risks, etc.,” Dr. Perry said. “But to simply ignore the potential health benefits of this plant because of its popular use as a psychoactive drug is short-sighted.”

The experimental drug

The use of an experimental drug isn’t an uncommon practice at most major hospitals, including at Cook Children’s.

An experimental drug is a substance which may be useful to diagnose or treat disease or preserve or enhance health, but has not yet been approved by the U.S. Food and Drug Administration for the specific purpose of treating a patient.

“The drug might or might not be approved for one or a number of conditions, but it becomes ‘experimental’ when used to systematically investigate treatment for an unapproved condition,” said James Marshall, M.D.,Chief Research Officer at Cook Children’s.

The experimental drugs are used, most often, in two situations at Cook Children’s.

Most commonly, experimental drugs are used during the course of an investigation of the drug’s safety, effectiveness and/or how it impacts a patient’s system and well-being. During this time, the drugs are administered under careful supervision and the children are monitored at all times.

Another important, if not as common, use of experimental drugs occurs at hospitals such as Cook Children’s. Once a drug has been approved by the U.S. FDA, experimental drugs, known to be safe and effective in adults, may be used to help children under specific guidelines. This occasion is known as “expanded access” or “compassionate use.”

“In this case, a physician believes that the experimental drug may help his or her patient’s condition for which the drug has not been investigated or approved, but the data seems to indicate a chance it will benefit the child,” Dr. Marshall said.

The data obtained from the use of experimental drugs can be useful to future patients as doctors can see how beneficial they are the next time they are treating a patient with a similar condition.

At Cook Children’s, children participate voluntarily in all experimental drug investigation. Patients and their families, particularly in the case of minor children, must volunteer to participate in any experimental investigation through a process called, “informed consent.”

Informed consent for children is regulated heavily by the U.S. and international governments. This type of consent is managed by a local board concerned only for the safety of research subjects, and far more comprehensive than the normal treatment consent process. Informed Consent for children has several key elements:

• Education by the investigator and his/her team regarding all aspects of the research.
• Informed consent or actually signing a legal document by a minor child’s parent or legally authorized representative.
• Assent or documenting that a minor child understands and agrees to participation, and a continuous, open and welcome ongoing conversation between the participant/family and the investigational team to assure understanding through all phases of the research.

The initial consent/assent process can take several hours, and investigators never really consider the conversation to be closed. Patients and their families often learn more about their condition during the consent/assent process than they would by receiving treatment only. Patients and families can decide to stop participation even before the experimental drug is administered.

However, many patient families see the importance of participating in these experimental drugs and the benefit it may bring to not only their child, but to patients in the future.

“At Cook Children’s, we believe that all children deserve the best, evidence-based treatment strategies available to manage their illnesses and maintain good health,” Dr. Marshall said. “Cook Children’s patients have the opportunity to contribute to the health of other children through safe and ethical clinical research. We treasure the fact that clinical research is the essence of people voluntarily caring for the future of mankind.”

Why a study of the use of Cannabidiol is needed

To prove a therapy is effective the following is needed:

• Controlled trials - studies where all the patients enrolled have similar conditions and in the process eliminate many variables that may affect the outcome other than use of the drug.
• Blinded studies - studies where neither the doctor nor the patient know what treatment they are on, to avoid bias.
• Placebo controls - some patients get the medicine and some get a “sugar pill” to make sure the improvement in seizures is not related to chance alone.

While early reports from families suggest CBD is well tolerated in most patients, standardized evaluation of side effects and long-term data will be very important. There is considerable concern about what impact CBD may have on the developing brain of a child and no trials in children have been sufficient yet to answer this question.

Finally, there is not yet adequate standardization of CBD production. Numerous states have CBD laws, all slightly different in their requirements, their oversight, and the conditions which can be legally treated.

“When you get a drug at the pharmacy, it is required by law to have certain standard properties to insure that each pill is similar to the next,” Dr. Perry said. “Families are often concerned about getting generic over brand name drugs – but even generic drugs have rules to insure they are very similar to their brand-name equivalent. Marijuana plants may have varied amounts of CBD and THC depending on the plant species, the growing conditions, and the extraction of the medication from the plant.”

Dr. Perry said the take home point is not whether or not CBD is a treatment for epilepsy, but is it ready to be used just as any other seizure medication now. Adequate evidence suggest that it warrants further investigation as a therapy, but there is inadequate evidence to wholeheartedly support it for more common use until the research is done.

The misconceptions of medical marijuana

The first question people often ask is “will it get my kid high?” Marijuana plants contain a number of substances which may hold potential to treat disease. There are two main chemicals in marijuana – the first and best known is THC. THC is the psychoactive part of marijuana – or the part that makes you “high.”

Cannabidiol (CBD) is another component and the one most people are interested in as a treatment for epilepsy. CBD has no psychoactive properties, so it doesn’t produce a “high.”

The second question is often “won’t smoking marijuana be bad for my child?” CBD is not administered by children smoking. It is typically formulated into an oil based liquid, so it can be given by mouth.

The next logical question is whether there is any evidence that CBD is effective at treating epilepsy – and this is where the research that Dr. Perry and his team are participating in comes into play.

“While the medical community is encouraged by the favorable reports from patients using CBD in other states, we understand that this type of ‘self-reported’ data is not adequate to prove a treatment works,” he said. “Often when a child improves with a medical treatment, the family will sing the praises of the miracle medication they found. We rarely ever hear from those people that tried yet another treatment that failed – as they simply have moved on looking for another potential cure. Therefore, while many people have reported success with CBD, we don’t know how many have failed using the same treatment.”

The Texas Department of Public Safety will be responsible for the licensing of at least three dispensing organizations by Sept. 1, 2017 before CBD will be available for patients.

CBD is the non-euphoric component of marijuana and contains no psychoactive properties, so it doesn’t produce a “high.” CBD is not administered by children smoking. It is typically formulated into an oil-based liquid so it can be given by mouth.

Texas agencies will regulate the distribution of the oils to patients whose symptoms have not responded to available treatments and the risks of treatment are outweighed by the potential benefits, according to their epileptologist. The treatment has to be agreed upon by two epileptologists before CBD can be administered.

M. Scott Perry, M.D., medical director of the Epilepsy Monitoring Unit and Tuberous Sclerosis Complex Clinic at Cook Children’s, estimates he discusses with parents and patients the pros and cons of marijuana use for epilepsy three or four times a day at minimum.

Dr. Perry said families are looking for any therapy that works for their child’s uncontrolled seizures once other options have failed, and some have either left the state or had plans on leaving Texas if the bill didn’t become law.

“I think we have seen enough in the other states that have already made CBD available to understand there is potential benefit to CBD use,” Dr. Perry said. “From what we’ve seen in the medical community so far, it’s very encouraging, but more research still needs to be done before CBD is a treatment of choice for most patients with epilepsy. For now, CBD may provide benefit for a small population with intractable (unmanageable) epilepsy, in which other treatments have been unhelpful. I’m glad that this bill will now afford me the same opportunity as other physicians in my field to prescribe CBD to this population of patients in Texas.”