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Medical Cannabis: A Possible Adjunctive Therapy for Gaucher Disease – Part I (Podcast Recap)

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In the treatment of Gaucher disease (GD), there are primary therapies (enzyme replacement therapy and substrate reduction therapy) as well as adjunctive therapies. An adjunctive therapy, or adjunct therapy, is a treatment that is used together with a primary treatment method to assist the primary treatment and improve results.

Nutrition supplements, meditation, breathing, and exercise are examples of adjunctive therapy. And now, there is the possibility of adding medical cannabis to this arsenal of adjunctive therapies, which may improve the primary medication therapy results, thereby improving quality of life.

But why medical cannabis? And why now? In this month’s podcast (Part I of II), we discuss the history of cannabis, including its long and zigzagging path in the medical world, and how cannabis relates to the important homeostatic system in the body.

Joining us is Dr. Robin Ely, co-founder of the National Gaucher Foundation (NGF) and currently serving as the clinical director of NGF’s diagnostic initiative. In addition to her work with NGF, since 2016, Dr. Ely has devoted her efforts to cannabis medical research and clinical practice in the states of Maryland, New York and Florida.

Dr. Ely is an investor, member of the board, and clinical advisor to iCAN (Israel Cannabis), a premier cannabis space accelerator that powers the internationally renowned CannaTech Conference.

History of Cannabis

Why is Dr. Ely involved in a cannabis organization in Israel? To understand the answer, we need to understand a brief history of cannabis.

Cannabis has been around since the earliest civilizations—archaeologists have found remains of cannabis seeds buried along with human remains in Egypt and India. Throughout history, people recognized the positive effects of medical cannabis. The cannabis plant was used widely for medicinal purposes, with great success.

The legal history of cannabis hit a rough patch beginning in the early 20th century. With the advent of President’s Nixon’s war on drugs in the 1970s, all cannabis plants, both industrial and medicinal, were banned in the U.S. From approximately 1940 – 1996, medical cannabis in the U.S. was no longer legal to use, and it was placed on the controlled substances ban.

There were many reasons for this, none of which had anything to do with the healing properties of the plant. Many countries followed the U.S. in prohibiting cannabis and cannabis research. When California passed Proposition 215 in 1996, legalizing medical cannabis, avenues for research began to reopen and patients with a number of medical conditions started to receive medical cannabis as part of their treatment.

However, one country never banned cannabis research: Israel. In the early 1960s, led by Dr. Raphael Mechoulam, Israeli scientists were the first to isolate and chemically characterize both CBD and THC, two components of cannabis.

In the late 1980s, Israeli scientists discovered cannabinoid receptors in the human brain, and in the 1990s they discovered that human beings secrete cannabinoid chemicals within the body. This discovery of our own cannabinoid production—called the endocannabinoid system (ECS)—paved the way for understanding why medical cannabis is effective for so many ailments.

Because of Israel’s robust cannabis research and clinical program, Dr. Ely got involved in iCAN, lending her expertise and skills.

“Cannabis” or “Marijuana?”

Language matters. And when you hear Dr. Ely talk about the cannabis plant, you won’t hear the term “marijuana.” She says, “Marijuana is a term that is associated with all the negative features that people have been attributing to the plant over the course of the last 70 years.”

And the word “cannabis” is actually the more accurate term—the word, or one similar to it, crops up again and again in various languages. Dr. Ely believes that by using the word cannabis, “We are pulling stigma away from the conversation and identifying cannabis as something medicinal.”

What Is Cannabis?

Cannabis Sativa L. refers to several subspecies of plants that provide fuel, fiber, and food, as well as other subspecies that are used for medicine. The names for these subspecies have varied over time and include: hemp, marijuana, ganga, pot, weed, and many others.

The cannabis plant contains more than 115 cannabinoids and 120 terpenes that have healing properties. Two of the most familiar cannabinoids are:

  • CBD, which has been shown in numerous clinical trials to reduce inflammation and pain. It is also used to treat epilepsy and has many other clinical applications. It is not psychoactive but has a relaxing effect.
  • THC, which is the primary psychoactive molecule in cannabis. It possesses many healing capabilities, including favorable effects on inflammation, pain modulation, malignancy, nausea, and muscles spasms.

Medical Benefits of Cannabis

Dr. Ely’s name is practically synonymous with Gaucher disease. Because three of her six children have Gaucher disease, she devoted significant time to the field and co-founded the NGF.

Her son, Brian Berman, was diagnosed with GD. Brian was the first child to receive enzyme replacement therapy (ERT), which was developed by Dr. Roscoe Brady, at the National Institutes of Health. Based on Brian’s favorable response, Dr. Brady’s laboratory, in association with the corporation Genzyme, set the stage for treating the full spectrum of lysosomal storage diseases.

In addition to her work with Gaucher disease, Dr. Ely is the director of the Center of Integrative Medicine, which focuses on complementary approaches to healing. In this context, Dr. Ely researches many alternative approaches.

In 2015, she learned about the growing body of evidence concerning the use of cannabis to treat many serious medical conditions. In this early research, three cases stood out and compelled her to devote more time and energy to the medical cannabis field:

Dravet syndrome

Charlotte, a young girl, had Dravet syndrome, a rare form of epilepsy that begins shortly after birth. People with Dravet syndrome have frequent and prolonged seizures. Charlotte was experiencing hundreds of seizures every day.

Despite taking many medications, her condition didn’t improve. The seizures hindered her development, and the medications had unpleasant side effects. Her parents looked into medical cannabis therapy and found out about the Stanley brothers in Colorado. These brothers had begun researching medical cannabis as an alternative treatment when their uncle was diagnosed with pancreatic cancer. They began to cultivate the plant for its therapeutic properties.

Charlotte’s family moved to Colorado to legally access medical cannabis. And Charlotte went from having hundreds of seizures per day to just a few per month. Free from the seizures’ grip, Charlotte was able to grow, achieve developmental milestones, and live her life.

Tourette syndrome

The next case study was about an Israeli soldier with Tourette syndrome, a type of tic disorder. People with Tourette syndrome have a combination of at least two motor tics and at least one vocal tic. The tics can be severe and debilitating.

The soldier filmed himself with all of his tics, then—on camera—inhaled cannabis. The tics disappeared, and he took out his guitar and started playing.

Debilitating stutter

A woman from Missouri had such a terrible stutter that she could barely talk. A single mother of two, the stutter made it difficult for her to take care of her kids. After she smoked some cannabis, she said to the camera, in a clear voice, “Now I can be a mother to my children.”

The life-changing results in these three stories propelled Dr. Ely to begin more in-depth research and involvement, both in Israel, the world’s epicenter for medical cannabis research, and in her practice. She began to include cannabis in her own repertoire of treatments. She also began to educate other medical professionals about the potential medical benefits of this plant.

Homeostasis and Disease

One chief benefit of medical cannabis has to do with homeostasis. One of the body’s most fundamental needs is to achieve homeostasis; to be in balance. All organisms experience stress from internal and external activities, causing imbalances.

Every living thing, from single-celled organisms to plants to humans, is in a constant state of being stressed—called dysregulation—and then returning to balance. So organisms have homeostatic mechanisms in place to bring them back to “center point.”

The endocannabinoid system (ECS) includes enzymes that synthesize chemicals. These chemicals are almost identical to the chemicals found in the cannabis plant. The ECS includes:

  • The chemicals themselves
  • The enzymes that both synthesize and break down the chemicals
  • The receptor proteins found on the cell membranes throughout the body, which the cannabinoids lock onto

Once the cannabinoids bind to the receptor proteins, a cascade of chemical reactions occur which governs and regulates almost all of the systems of the body.

In the last 10 years, the ECS has been recognized as the body’s primary homeostatic (regulating) system.

The discovery of the endocannabinoid system finally explains why cannabis has been used as a medicine for thousands of years—and why it can potentially address so many ailments that human beings experience.


One major process that the ECS uses is the inflammatory response. In a healthy ECS, inflammation produces healing. If the ECS is deficient, the inflammatory response will be too weak or too vigorous, both of which lead to illness.

Chemical Properties of Cannabis

To gain a greater understanding of the cannabis plant, we need a little basic chemistry. Terpenes are the most ubiquitous class of chemicals in all of nature. They are found in the oils of thousands and thousands of plants, including cannabis, giving plants their unique flavors and aromas, along with nutritional and medicinal effects.

In the cannabis plant itself, there are likely more than 200 terpenes, of which the cannabinoids are a non-aromatic subclass. The aromatic terpenes are the plant’s immune system. The terpenes help alleviate both internal and external phenomena that may cause harm, such as bacteria (internal) and mites (external). The cannabinoids function as one part of a system that includes the many other terpenes, phenols, and flavonoids working together in what is known as the Entourage Effect.

Better together

For decades, the goal driving drug research has been to isolate a single molecule from plants and then patent that molecule to treat a disease state. Take chemical X and use it to address a specific symptom or general disease. These isolated chemicals sometimes work, but frequently provoke unpleasant or even lethal side effects.

Proponents of whole cannabis plant therapy are seeking to capitalize on the Entourage Effect. Primary cannabinoids, such as CBD and THC, are found to be more effective when used in concert with other beneficial terpenes and related chemicals in the cannabis plant. Therefore, using whole plant therapy may decrease unfavorable side effects.

Now we have a basic understanding of homeostasis, which is the drive to wellness; the endocannabinoid system, which governs homeostasis; and the cannabis plant, which has so many healing chemical compounds that directly and indirectly interact with our own endocannabinoid system. We are ready to dive into the cannabis-Gaucher connection which will be addressed in Part II of our podcast.


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