Open-Label Phase 2 Trial of ZYN002 in Developmental and Epileptic Encephalopathies

Zynerba Pharmaceuticals announced that it has initiated the Phase 2 BELIEVE 1 (Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy [DEE]) clinical trial.

As quoted in the press release: “DEE is a heterogeneous group of epilepsy syndromes that involve significant developmental impairment or regression of developmental progress, and are highly resistant to treatment,” said Dr. Liza Squires, Zynerba’s Chief Medical Officer. “Little attention is paid to DEE outside of Lennox-Gastaut and Dravet syndromes despite the high unmet need. The BELIEVE 1 study will assess children and adolescents with a variety of epilepsy syndromes that are characterized as DEE. Our goal is to deliver an effective, well tolerated, and easy to administer therapeutic option for individuals living with DEE and their families.”

Cannabidiol helps reduce seizures in people with severe form of epilepsy, study suggests

A key cannabis compound helped to reduce the number of drop seizures in patients with a severe form of epilepsy, newly published results of a clinical trial suggest.

The study, published by The Lancet, suggests that cannabidiol, alongside other anti-epilepsy treatments, reduced drop seizures in those who suffer from Lennox-Gastaut syndrome.

Drop seizures involve sudden falls due to loss of muscle tone. Lennox-Gastaut is a severe, lifelong form of epilepsy involving frequent seizures and cognitive impairment. The syndrome causes about one to four per cent of childhood epilepsy cases, researchers say.

In the 14-week clinical trial, the frequency of seizures was reduced in those who took a pharmaceutical formulation of cannabidiol. But researchers say the long-term efficacy and safety of the cannabis compound, as well its interaction with other epilepsy drugs, still need to be studied and confirmed.

The study’s lead author, Dr. Elizabeth Thiele, a neurologist who specializes in pediatric epilepsy at the Massachusetts General Hospital, said she and her colleagues are “pleased that our study has potentially found an additional option to add to patients’ existing treatment to reduce drop seizures.” In a news release, she noted that “it’s important to highlight that the drug used in this trial is a pharmaceutical formulation, and not medical marijuana.”

CURE Responds to DEA’s Decision on Marijuana Scheduling

Chicago, IL – This month the Drug Enforcement Administration (DEA) announced their decision to maintain marijuana as a Schedule I drug, despite petitions from Citizens United for Research in Epilepsy (CURE) and many other research and patient-focused organizations concerned with the federal barriers prohibiting researchers from fully understanding the potential of medical cannabis. Despite this setback, CURE is pleased with the DEA’s decision to expand the number of DEA-registered marijuana manufacturers for research, of which there is currently only one, so more researchers will be able to conduct much-needed research on cannabidiol (CBD) – the major non-psychoactive ingredient in marijuana, as well as the many other cannabinoids found in marijuana.

An estimated 3 million Americans currently live with epilepsy and for two-thirds of them, the cause is unknown, making treatment difficult if not impossible to pinpoint. The positive results that some people with epilepsy have been seeing from CBD-rich marijuana extracts are giving so many parents what they have been lacking for so long – hope. CBD may not be the answer for all of these families, but when available medications and surgeries do not stop the uncontrollable seizures, every avenue must be explored.

As the leading nongovernmental agency fully committed to funding research in epilepsy, we believe researchers should be encouraged and supported to test not only pure CBD, but also high CBD/low-THC cannabis, pure-THC and other types of medical marijuana to determine the efficacy of these and other combinations on seizure control and the genesis of epilepsy. We are hoping for more changes to marijuana access that makes it easier for researchers to continue their vital work.

The Fight to Improve Research Access to Cannabis Continues

WASHINGTON, D.C.— Due to its incredible medicinal potential, we continue to believe that more research must be done on marijuana rich in cannabidiol (CBD). In late April, CURE signed a group letter addressed to Chuck Rosenberg, the head of the Drug Enforcement Administration (DEA), urging him to remove cannabis from Schedule I in the Controlled Substances Act.

This would eradicate federal barriers to research, paving the way for more progressive research and new treatments. CURE has joined with other organizations in support of the Compassionate Access, Research Expansion, and Respect States Act (CARERS, S. 683, H.R. 1538) which would facilitate safe and legal access to medical cannabis for patients and physicians acting in accordance with state law and lift federal barriers to research. The CARERS Act would also remove low-THC cannabis from the CSA allowing individuals in states that have created protections for low-THC therapies to more easily access this potential treatment option.

Current regulatory hurdles make it difficult for researchers to gain access to marijuana rich in CBD. There is no debate that the hoops through which researchers must jump to acquire marijuana, or any chemical found in it, are hindering scientific advancement—and CURE is committed to helping researchers overcome these obstacles to advance research in this important area.