Fort Worth, Texas,
11:37 AM

Drug in Cook Children's epilepsy trial shows positive results in separate trial

Study focuses on use of cannabidiol (CBD) to treat severe forms of epilepsy

“What about the use of medical marijuana?”

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.”


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