MARCO ISLAND, Florida—When it comes to cannabis, Bonni Goldstein, MD, certainly knows her terpenes from her tetrahydrocannabinol (THC).
A California-licensed pediatrician based in Los Angeles since 1990, Dr. Goldstein is also an expert on neurological disorders. About 80% of the 18,000 kids she’s seen over the last 15 years have had refractory epilepsy—meaning patients have seizures that don’t respond to antiseizure medication—including rare types such as Dravet syndrome and Lennox-Gastaut syndrome (LGS).
That’s why Dr. Goldstein has become a fierce advocate for using medical cannabis to treat these diseases.
“Saying we don’t know enough about cannabis is really cruel,” she said. “We know it works.”
Dr. Goldstein spoke to Rare Disease Advisor during the CannMed 23 Innovation & Investment Summit here. The medical director and owner of Canna-Centers Wellness, she’s also the author of a 368-page book, Cannabis is Medicine: How Medical Cannabis and CBD are Healing Everything from Anxiety to Chronic Pain.
The reason cannabis is effective, she said, is that it targets the human endocannabinoid system, which helps maintain homeostasis, or balance in brain messaging.
“In many patients who have neuroexcitatory conditions, there can be seizures, or in children with autism, it can manifest as abnormal or unwanted behaviors,” she said. “When you take cannabis, it targets this endocannabinoid system as well as numerous other areas in the brain to send a message to decrease the excitatory message. It’s like turning the volume down on your radio.”
In June 2018, the US Food and Drug Administration (FDA) approved an oral cannabidiol solution known as Epidiolex® to treat both Dravet and LGS in patients 2 years of age and older. That marked the first time the agency had ever given its blessing to any purified drug substance derived from marijuana.
But Dr. Goldstein said Epidiolex is an isolate that contains CBD but lacks the other compounds which occur naturally in the cannabis plant.
“The reason they did that was because they were seeing what was happening with families in Colorado, California, and other states where CBD was used to control child epilepsy,” she said. “A pharmaceutical company saw that it had already been working in the cannabis space and decided to go after that particular market.”
She added: “We know phytonutrients are good for us. Why would I want to take away compounds in the plant that are actually beneficial for the patient? That makes no sense unless you have a patent and are concerned with your shareholders.”
Even so, Dr. Goldstein said she doesn’t anticipate the FDA approving botanical forms of cannabis for the simple reason that they contain at least 500 known compounds.
“That means you have to test each and every compound. We are talking about another 200 years of research,” she said. “What we need to do is recognize that cannabis medicine is unique and does not fit the pharmaceutical model. It is a botanical medicine, and it must have its own lane. It does not need to be shoved into randomized clinical trials.”
Complicating the situation is the nationwide patchwork of laws regulating cannabis use. All but 6 states—Idaho, Kansas, Nebraska, North Carolina, South Carolina and Wyoming—permit residents to use cannabis for medical reasons, though under various conditions.
For example, Alabama allows medical cannabis only in the form of nonpsychoactive CBD oil for children with epilepsy; all other use is punishable by up to a year in prison and $6000 in fines. South Carolina allows only CBD oil with less than 0.9% THC for people with Dravet, LGS, or refractory epilepsy. In Idaho, marijuana is illegal for any use; possession of more than 3 ounces is a felony.
The federal government still bans cannabis, meaning that most clinical trials which involve cannabinoid compounds are conducted overseas, mainly in Canada, Israel, and the Netherlands.
For example, Israeli pediatric neurologist Adi Aran, MD, has done groundbreaking work with autistic children at Jerusalem’s Shaare Zedek Medical Center.
In 2017, Dr. Aran launched the world’s first-ever randomized, double-blind, placebo-controlled clinical trial on cannabinoids for behavioral problems in children with autism spectrum disorder. It involved 150 autistic individuals aged 5 to 21 years defined as low- to medium-functioning. Israel’s Ministry of Health approved the trial, but only after overcoming religious objections from both Arab Islamic politicians and, to a lesser extent, Orthodox Jewish groups.
For 12 weeks, half the participants received either a whole plant extract including terpenes, flavonoids, and other cannabinoids, or just CBD and THC. The other half got only placebo. Then, after a 4-week washout period, the groups were reversed. The result: half the patients in each cannabinoid arm improved, while in the placebo arm, improvement was seen in only 20% of the participants. Dr. Aran is now preparing a second clinical trial for 100 children aged 4-12 years.
“Our medical community has bought into the idea that something needs to be FDA‑approved to be medicine,” Dr. Goldstein said, cautioning that American doctors will not prescribe anything the agency has not authorized. “And cannabis is not going to be approved until it becomes descheduled off our Controlled Substance[s] Act. It is still categorized as a Schedule I controlled substance, which means that the definition is very important.”
Dr. Goldstein suggested that the US pharmaceutical industry hypes lies about cannabis because it fears competition with expensive antiseizure medications that are often ineffective.
“No medicinal value, untrue. High addiction potential, untrue. Lack of safety, untrue. High abuse, untrue. I can say all that about tobacco and alcohol, which are not on the schedule but are free to buy and kill people every year,” she said. “Cannabis kills no one and is still stuck in that category, and this is all propaganda.”