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Until research unlocks medical understanding of marijuana, patients experiment

July 6, 2016 at 6:15 PM EDT
Lenny and Amy’s 5-year-old son has epilepsy. When conventional medications caused terrible side effects, they started giving him a daily drop of cannabis oil, with dramatic results. But it’s a calculated risk: While there is anecdotal evidence of cannabis’ effectiveness, scientists face research roadblocks because it’s a schedule 1 controlled substance. Science correspondent Miles O’Brien reports.

JUDY WOODRUFF: A big question: What are the benefits and drawbacks of medical marijuana?

In some cases, the law is ahead of the research.

Miles O’Brien explores. It’s part of our weekly reports on the Leading Edge of science.

MILES O’BRIEN: A sunny day, a scooter, and a little boy tearing up the driveway, a simple pleasure for him, a cherished moment for his parents.

LENNY LOPEZ: I see him do it, and I said, I can’t believe he’s riding a scooter by himself. It’s not a big deal for most people. That’s what kids do. But for us that’s a big deal.

MILES O’BRIEN: Lenny and Amy Lopez’s adopted 5-year-old son, Isaias, has epilepsy Dravet’s syndrome, which besets its young victims with incessant seizures.

How often would that happen?

LENNY LOPEZ: In the beginning, it happened all the time. He would have hundreds. So…

AMY LOPEZ: And it’s all day long.

LENNY LOPEZ: He wouldn’t be able to play. He wouldn’t be able to eat. He would just fall. And then when he would have like the more severe ones, he would literally just like shut off and fall down and smack his head.

MILES O’BRIEN: Amy and Lenny noticed a near-instant improvement for Isaias after this became a part of their daily routine, a squirt of cannabis oil. It is mostly CBD, a chemical in marijuana that doesn’t alter consciousness.

Isaias’ seizures have reduced in intensity and frequency by about 80 percent.

AMY LOPEZ: What about Rocky?

We went through a couple of different strains, and then just the dose, how much do you give, what time of day do you give it, so definitely trial and error.

MILES O’BRIEN: For all intents and purposes, Amy and Lenny are experimenting with their sick little boy. It is a calculated risk born of desperation and fear.

911 OPERATOR: Nine-one-one recorded line.

LENNY LOPEZ: Hi. I’m calling about — my son is having a seizure.

It’s a helpless feeling, because, no matter what you do, nothing seems to be working.

AMY LOPEZ: When your child is turning blue, and he’s not responding to you, and you know he’s not getting any air, like my husband said, you don’t know if that’s — that’s it, that’s the last one. So you just hope and pray that he pulls through it.

MILES O’BRIEN: They tried conventional pharmaceuticals designed to curb the seizures. But the side effects were horrible. They made him either unresponsive, or caused him to act out and hurt himself. It was not a real solution.

DR. SANJAY GUPTA, CNN: Our journey begins here in this small-town home.

MILES O’BRIEN: Then, one day, they saw the CNN documentary “Weed.” It told the story of a family in Colorado using a high CBD strain of cannabis to treat their daughter’s Dravet’s epilepsy. The marijuana was working.

PAIGE FIGI, Mother: She didn’t have a seizure that day, and then she didn’t have a seizure that night, and then she didn’t have one the next day, and then the next day.

MILES O’BRIEN: Was it like a revelation?

AMY LOPEZ: Yes, I was like, we have to get this. I knew it wasn’t legal here at the time, but we have to get this.

MILES O’BRIEN: Easier said than done. The doctor they had at the time would have nothing of it. So they found their way to a more sympathetic ear.

DR. ELIZABETH THIELE, Massachusetts General Hospital: For the past three years, I don’t think there’s been a single patient I have seen that hasn’t asked about CBD or medical marijuana.

MILES O’BRIEN: Dr. Elizabeth Thiele is director of the pediatric epilepsy program at Massachusetts General Hospital.

DR. ELIZABETH THIELE: When this first came up, I think that there were a lot of — I sometimes use the word haters, as the medical profession, I think, wasn’t necessarily welcoming this at first.

MILES O’BRIEN: But Dr. Thiele shared in the Lopezes’ frustration, even though the number of conventional medications aimed at treating epilepsy has more than doubled in the past few decades.

DR. ELIZABETH THIELE: Most children with Dravet don’t respond to the medications we have. So there continues to be a significant unmet need for epilepsy therapies.

MILES O’BRIEN: While there is plenty of anecdotal evidence that cannabis helps children with Dravet’s, the Drug Enforcement Administration classifies marijuana among the most dangerous drugs, a Schedule 1 controlled substance.

As far as the federal government is concerned, cannabis has no currently accepted medical use and a high potential for abuse. As a result, scientists have trouble accessing the drug legally to do the research, and so we know precious little about the medicinal value of marijuana to treat epilepsy, or anything else.

That’s starting to change. The British pharmaceutical company GW is testing a version of cannabis oil called Epidiolex. Designed for children with seizure disorders, it is nearly pure CBD. It’s early, but the results are promising. If they can be repeated in larger studies, Dr. Thiele says it could be a breakthrough.

For now, she can’t even be sure precisely what is in the cannabis her patients are taking.

DR. ELIZABETH THIELE: Sometimes, the batch will be effective, will make the seizures better. Sometimes, the next month, it will be worse, the seizures will get worse. So, I don’t feel comfortable prescribing something that I don’t understand better than that.

NORTON ARBELAEZ, New England Treatment Access: To grow the pharmaceutical-grade cannabis that we’re growing, it takes quite a bit of effort.

MILES O’BRIEN: Norton Arbelaez is on a mission to change this. He is a consultant for New England Treatment Access in Massachusetts, one of 25 states and the District of Columbia that have voted to make medicinal marijuana legal.

He gave me a tour of NETA’s cultivation and manufacturing facility, where they grow about 60 distinct strains of marijuana. At any given time, there are 14,000 plants here at various stages of development. They work hard to maintain precise, consistent conditions.

NORTON ARBELAEZ: So, these are clones. Initially, we grew it from seed.

MILES O’BRIEN: What’s the advantage of cloning, as opposed to planting seeds?

NORTON ARBELAEZ: Well, that we have control of the genetic stock. The patients are looking for a very specific strain to deal with a very specific type of illness, so we want to be able to reproduce that each and every time.

MILES O’BRIEN: Strains are identified by color. And every bar coded batch is tested by a third party before it is sold to patients.

But analyzing batches of marijuana for a handful of chemicals only scratches the surface. A Massachusetts-based genomic testing firm, Courtagen Life Sciences, is trying to learn the genetic makeup of various strains of marijuana.

KEVIN MCKERNAN, Courtagen Life Sciences: What we do down here in the laboratory is run some robotics that then go into the genome and extract the regions that we want to sequence.

MILES O’BRIEN: Kevin McKernan is the chief scientific officer. He hopes to inject some objective science into the conversation.

KEVIN MCKERNAN: It’s about as scientific as a wine, oaky, woody, earthy. And if we want medicine to absorb this, we need to start talking about the cannabis in the language that physicians like, which is about genotypes, not woody, earthy, oaky smells.

MILES O’BRIEN: They have sequenced 600 strains of marijuana so far.

But what are we seeing here?

KEVIN MCKERNAN: So what you’re looking at is the genetic distance between different cannabis strains and the length of this line. If you follow this in and out is the length of the genetic distance between these. And then each of these names are the names of the strains.

MILES O’BRIEN: After all these years, this familiar plant remains a mystery. Marijuana contains more than 400 chemicals, including 80 cannabinoids.

Scientists are not sure how they may interact with each other to provide some medicinal benefit. They are trying to understand if isolating the compounds works, or if there is a so-called entourage effect.

KEVIN MCKERNAN: I think it’s this entourage effect, that the other compounds that are there in the whole plant extract are doing something we don’t understand yet. There’s something about isolating the compounds that seems to not replicate what the plant does on its own.

MILES O’BRIEN: The real answers won’t be forthcoming unless research dollars flow and more human trials begin. But that won’t happen until the federal government reconsiders whether marijuana appropriately belongs in the same category as heroin.

The Centers for Disease Control and Prevention says there has never been a documented overdose death from marijuana.

So, Amy and Lenny Lopez see much more benefit than risk.

AMY LOPEZ: He makes us…

LENNY LOPEZ: It’s definitely worth it.

AMY LOPEZ: He makes my blood flow.


AMY LOPEZ: That kid make me breathe. I would do anything for him.

MILES O’BRIEN: The proof is in the scooter.

Miles O’Brien, the “PBS NewsHour,” Lowell, Massachusetts.

JUDY WOODRUFF: The DEA has been evaluating proposals to loosen the classification of cannabis, which would make it easier for scientists and doctors to do research. But the agency missed its own July deadline, and it is not clear when there will be a decision.

In his next piece, Miles will speak with the Israeli chemist whose pioneering work into medical cannabis 30 years ago is now being embraced by the scientific mainstream.