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Study to explore whether cannabis compound facilitates severe symptoms of autism

  Neon Medical Marijuana Sign. Image Laurie Avacado

Neon Medical Marijuana Sign. Image Laurie Avacado

Researcher at the University The San Diego School of Medicine in California is preparing a first multidisciplinary study to determine if and how cannabidiol (CBD), a non-psychoactive compound in the cannabis plant, can bring therapeutic benefits to children with severe autism symptoms (19659006) Study, which is expected to start in a year, is funded by a gift from the Ray and Tye Noorda Foundation in collaboration with the $ 4.7 million Wholistic Research and Education Foundation. Private Gift for Medical Cannabis Research in the United States [19659006] The Center for Medicinal Cannabis Research of the UC San Diego School of Medicine (CMCR) is being investigated by a team of researchers n Physicians and physicians care for scientists who apply clinical assessments, coupled with basic and translational research, across a cohort of patients to better understand how CBD interacts with or modifies neural activity, and to address some of the more problematic ASD symptoms, such as alleviate aggressive behavior, effectively and safely. repetitive or self-injurious behaviors, hyperactivity and social and communicative deficits.

"We clearly need more treatment options for children with moderate to severe autism who are hardest hit and need the most help," said Igor Grant. MD, CMCR Director and Chair of the Department of Psychiatry at the UC San Diego School of Medicine. "It is becoming increasingly apparent that CBD has a number of central nervous system effects that may be relevant to autism, and there are isolated reports that suggest that CBD treatments may improve the functioning of an ASD child.

" Investigating the effects of cannabidiol on autism was a long-term goal for CMCR. We are excited to begin this clinical trial and to provide additional evidence-based knowledge on how cannabis and cannabinoids can be used as medicines not only for autism, but in the future for other neurodevelopmental disorders and neuropsychiatric disorders as well.

ASS affects a estimated one in 68 children in the United States, primarily boys.The neurodevelopmental disorder is complex with several known or suspected causative factors, from inherited genetic mutations to environmental conditions to metabolic dysfunction – an important consequence is the abnormal evolution and function of connectivity and communication between brain cells and between neuronal networks, leading to many of the observed social and cognitive impairments in individuals with ASD. "The most exciting part of this new study is that the findings could not only for children with autism, but also for children with other types of neurodevelopmental disorders, "said Doris Trauner, MD, Distinguished Professor of Neuroscience and Paediatrics at the UC San Diego School of Medicine and a pediatric neurologist at Rady Ch ildren's Hospital San Diego has particular expertise in neurodevelopmental disorders. As a project manager, Trauner will oversee the design and execution of the clinical trial.

CBD is an important chemical compound found in cannabis. It does not produce the effects of feeling "high" caused by tetrahydrocannabinol or THC (the psychoactive ingredient in marijuana), but as one of more than 100 cannabinoids in cannabis, CBD works with the body's own endocannabinoid system, a network of neurotransmitters which regulate various physiological and cognitive processes and reactions to stress. CBD may additionally affect other neural signaling pathways. Some data suggest that CBD may be effective in seizure control and may reduce disturbed thinking in schizophrenia and anxiety symptoms in a variety of conditions, including post-traumatic stress disorder.

Grant said that the endocannabinoid system is associated with four phenotypic characteristics known to be atypical in ASD: social reward response, neural development, circadian rhythm and anxiety symptoms. "This makes the endocannabinoid system a prime region for finding and testing new potential therapeutic compounds such as CBD."

The objectives of the study are threefold: Determine if CBD is safe and tolerable and if it relieves unwanted side effects Symptoms of ASD; to find out if and how CBD alters brainwave activity, neurotransmitters and / or brain networkability; and to reveal whether biomarkers of neuroinflammation (also associated with ASD) are altered by CBD.

The clinical trial will consist of 30 children aged 8 to 12 years with autism and severe symptoms. All will receive behavioral tests, MRI scans and electroencephalograms. In the first phase of the study, half of the children receive CBD and placebo. In the second phase, the groups are changed, and half of those originally given CBD receive placebo, while the initial placebo group receives CBD. Examiners will be blinded which children will receive which treatment until all tests are completed at the end of the study.

CBD is a controlled drug according to Schedule 1, as defined by the Drug Enforcement Administration. The availability for research purposes is strictly limited. The CBD for this study is provided by INSYS Therapeutics, Inc., an Arizona-based company dedicated to the development, manufacture and commercialization of pharmaceutical cannabinoids, with products for the treatment of various neurological and neurological developmental disorders.

Understanding the Mechanisms Of any potential clinical benefit of CBD, the Noorda Gift will support two closely linked basic scientific studies.

First, using skin cells from trial participants, a research team led by Alysson Muotri, PhD, a professor at the UC San Diego School of Medicine Departments of Paediatrics and Cellular and Molecular Medicine, director of the UC San Diego Stem Cell Program and member of the Sanford Consortium for Regenerative Medicine, will use induced pluripotent stem cells to create in vitro models of participants' neurons and organoids – simple but organized, functional, three-dimensional cell aggregates, sometimes referred to as "mini-brain" "In 2010, Muotri and colleagues created the first human cellular model for autism that can be used as a tool for testing, diagnosing and drug screening. Second, Gabriel A. Silva, PhD, Professor of Bioengineering and Neuroscience. and colleagues are using newly developed computer models to track and analyze signaling pathways between in vitro neurons and networks.

The Ray and The Tye Noorda Foundation is based in Utah and emphasizes philanthropy, which focuses on improving access to health and education. His gift for the CBD study was made in partnership with and based on recommendations from the Wholistic Research and Education Foundation based in La Jolla, California.

"We are excited to partner with the renowned scientists and researchers at UC San Diego on such a groundbreaking study," said Pelin Thorogood, president and co-founder of the Wholistic Research and Education Foundation. "The results have the potential to provide treatment options for countless children with neurological disabilities and may be fundamental to the emerging field of cannabinoid therapy."

Source: UC San Diego Health

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