As the opioid crisis continues to worsen, many have suggested that marijuana-based medicine could be used as an alternative treatment — particularly for pain management. Currently, however, marijuana is listed as a “schedule one” drug by the Drug Enforcement Agency (DEA), which is defined as a “drug with no currently accepted medical use and a high potential for abuse.” In August of 2016, the DEA responded to a request by the governors of Rhode Island and Washington to reclassify marijuana as a “schedule two” drug — reclassification which could have significantly expanded research and access to treatment — by asserting that there was still no currently accepted medical use for marijuana and a high potential for abuse, and thus marijuana would remain a schedule one drug.
Over the past five years, studies have shown that a non-psychoactive compound of marijuana, called cannabidiol (CBD), can play a role in the modulation of brain functioning. This pharmacodynamic action has been conclusively determined to reduce seizures in children with epilepsy. London-based pharmaceutical company, GW Pharmaceuticals, has filed a new drug application (NDA) with the FDA for its CBD drug, epidiolex, which could be approved as early as this summer, citing a reduction of epileptic seizures by 27 percent in children with epilepsy treated with epidiolex over a placebo.
The same brain modulation effects observed with CBD-based treatment in epilepsy could also ameliorate symptoms in children with autism (a disorder also believed to involve brain regulation). In an Israeli-based study to be published later this year in the journal, Pediatrics, nearly half of children treated with CBD compounds saw a marked reduction in the core symptoms of autism, and nearly a third of children in the study started speaking for the first time (or began communicating nonverbally for the first time).
In the United States, the supply of marijuana for federally funded research is currently regulated under the Controlled Substances Act (CSA). The University of Mississippi is the only entity authorized to supply marijuana for federally-funded research under an exclusive contract with the National Institute on Drug Abuse (NIDA), pursuant to the Single Convention on Narcotic Drugs (1961).
Research institutions (sponsors) who wish to utilize this supply of marijuana and initiate an investigation into the effects of marijuana in human subjects would first need to obtain approval from their Institutional Review Board (IRB), which would evaluate and approve protections for the human subjects involved in the research. Sponsors should expect a higher level of scrutiny due to the psychoactivity of marijuana and the probability of vulnerable patients such as pediatric patients, patients with brain disorders, and patients who may be susceptible to abuse of opioid drugs due to chronic pain.
Following IRB approval, the sponsor would need to file an Investigational New Drug Application (IND) with the FDA pursuant to 21 C.F.R. 312. Because marijuana research is potentially more complex from traditional pharmaceutical research, which typically uses what are called “small molecule” drugs, the sponsor of the IND would wish to consult the non-binding “Botanical Drug Development Guidance for Industry” posted by the FDA prior to submitting an IND for approval. The FDA would have thirty days to communicate any reservations about the study, during which time the sponsor would not be permitted to allow the medical investigators to commence research.
Because marijuana is a schedule one drug, additional requirements would also apply under 21 C.F.R. 1301.18, which regulates controlled substances used in research. The medical investigator and the research site(s) used in the study would be required to obtain special licensure and registration from the DEA. The DEA requires that sponsors file three copies of the IND with the DEA (in addition to the FDA) and a statement of the security provisions to be used in the study. The DEA also requires prompt reporting of any modifications to the study.
The FDA lists a typical seven step process that sponsors would follow for researching marijuana, which can be found here. As the global medical marijuana market is currently valued at $30 billion, it will be exciting to follow the progress made by United States based researchers who wish to contribute to the development of new and innovative medical treatments.
image credit: https://www.dea.gov/galleries/drug-images/marijuana