Tag Archives: fragile X syndrome

Marijuana-like brain chemicals could be key to treating fragile X syndrome.

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“In an international collaboration of research centers from America and Europe, scientists have revealed that increasing chemicals in the brain that act similarly to marijuana can help repair the debilitating symptoms associated with fragile X syndrome.

The overall success of this study could lead to future treatments for the condition, which has been identified as the most common genetic basis for autism spectrum disorders.  The research was published in Nature Communications.

The marijuana-like compound, called 2-AG, is a part of a class of chemicals called endocannabinoid transmitters.    These compounds are naturally made by the brain, and they act by combining to receptor proteins in the brain that marijuana chemicals also bind with.

Fragile X syndrome is the result of a mutation of the FMR1 gene in the X chromosome passed on by the mother.   The condition occurs mostly in males because females typically have another X chromosome to compensate for the faulty X chromosome.  Symptoms of fragile X often include mental disability, walking and language delays and hyperactivity – as well as certain physical characteristics such as an elongated face and large ears.”

Read more: http://www.foxnews.com/health/2012/09/25/marijuana-like-brain-chemicals-could-be-key-to-treating-fragile-x-syndrome/#ixzz2DStbqb00

Marijuana- Like Compound in Brain Reduces Anxiety Associated With Fragile X Syndrome

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“Increasing levels of a marijuana-like compound in the brain may help reduce some behavioral problems seen in people with Fragile X Syndrome.
Researchers say that the marijuana-like compound may help reduce some of the anxiety and learning-related issues in people with this condition. The compound, called 2-AG, falls under a class of chemicals in the brain called endocannabinoids transmitters.

 Fragile X syndrome is a genetic disorder that causes learning disabilities. Children with this condition have characteristic physical features like long and narrow face, large forehead and ears, flexible fingers and flat feet that become more apparent as the child ages. The condition is caused by a change in the FMR1 gene that codes for a protein that helps the brain grow properly.”

Read more at http://www.medicaldaily.com/articles/12334/20120926/marijuana-compound-brain-reduces-anxiety-associated-fragile.htm#tFdDDvKazcBZ1gFG.99

  • mike

    “YES, finally its time… i have autistic spectrum and i have incorrect behaviors that cannot be fixed unless you tell yourself 24/7 and behaviors and thought process that is incorrect that you will never even know is incorrect. Ive tried marijuana before and the first time ive tried it ive done some self explaining to myself that was very different and i didnt know why. after using marijuana several times which i find relaxing if not used too much at once, i started realizing the difference in thought process and realizing the off things that i do and it got to the point where i actually started figuring out my problems with cannabis and i cant believe the correction in thought process when using this significant plant. there was never a cure for autism but this is the CLOSEST to it. i have a lot more things to say about this but it would take too long to write but all i have to say is this is THE best medication for personality disorders and autistic behaviors and correction to the thought process of such.”

Marijuana-Like Chemical May Help Autism And Fragile X Syndrome Symptoms

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“American and European researchers have found that increasing natural marijuana-like chemicals in the brain may help correct behavioral issues related to autism.

Daniele Piomelli of UC Irvine and Olivier Manzoni of INSERM, the French national research agency, led the study, which could result in treatments of anxiety and cognitive defects in individuals with fragile X syndrome, the most common known genetic cause of autism, according to a press release by UC Irvine.

The study examined 2-AG, which naturally occurs in the brain and is in a class of chemicals called endocannabinoid transmitters. These transmitters allow for the efficient transport of electrical signals at synapses, which is severely limited in people with fragile X syndrome.

The researchers treated mice that exhibited symptoms of fragile X syndrome with novel compounds that correct 2-AG protein signaling in the brain. And the results were promising–the mice showed “dramatic behavioral improvements in maze tests measuring anxiety and open-space acceptance,” UCI reports.

Piomelli said this is the first study to identify the role of naturally-occuring endocannabinoids, which share a similar chemical structure with THC, the primary psychoactive component of marijuana. “What we hope is to one day increase the ability of people with fragile X syndrome to socialize and engage in normal cognitive functions,” said Piomelli, a UCI professor of anatomy and neurobiology.

“It would be either an oral or injected drug but that’s at the very end stage of drug discovery, and we are at the very early stage of drug discovery,” Kwang Mook Jung, a researcher on the study and UCI professor, told The Huffington Post.

In addition, his study of endocannabinoids could result in new treatments for anxiety, pain, depression and obesity, according to UCI.”

http://www.huffingtonpost.com/2012/09/27/marijuana-chemical-autism-fragile-x_n_1920320.html

 

Boosting Natural Marijuana-Like Brain Chemicals Treats Fragile X Syndrome Symptoms

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“ScienceDaily (Sep. 25, 2012) — American and European scientists have found that increasing natural marijuana-like chemicals in the brain can help correct behavioral issues related to fragile X syndrome, the most common known genetic cause of autism.

The work indicates potential treatments for anxiety and cognitive defects in people with this condition. Results appear online in Nature Communications.

Daniele Piomelli of UC Irvine and Olivier Manzoni of INSERM, the French national research agency, led the study, which identified compounds that inhibit enzymes blocking endocannabinoid transmitters called 2-AG in the striatum and cortex regions of the brain.

These transmitters allow for the efficient transport of electrical signals at synapses, structures through which information passes between neurons. In fragile X syndrome, regional synapse communication is severely limited, giving rise to certain cognitive and behavioral problems.

Fragile X syndrome is caused by a mutation of the FMR1 gene on the X chromosome. People born with it are mentally disabled; generally experience crawling, walking and language delays; tend to avoid eye contact; may be hyperactive or impulsive; and have such notable physical characteristics as an elongated face, flat feet and large ears.

The researchers stress that their findings, while promising, do not point to a cure for the condition.

“What we hope is to one day increase the ability of people with fragile X syndrome to socialize and engage in normal cognitive functions,” said Piomelli, a UCI professor of anatomy & neurobiology and the Louise Turner Arnold Chair in the Neurosciences.

The study involved mice genetically altered with FMR1 mutations that exhibited symptoms of fragile X syndrome. Treated with novel compounds that correct 2-AG protein signaling in brain cells, these mice showed dramatic behavioral improvements in maze tests measuring anxiety and open-space acceptance.

While other work has focused on pharmacological treatments for behavioral issues associated with fragile X syndrome, Piomelli noted that this is the first to identify the role endocannabinoids play in the neurobiology of the condition.

About endocannabinoids

Endocannabinoid compounds are created naturally in the body and share a similar chemical structure with THC, the primary psychoactive component of the marijuana plant, Cannabis. Endocannabinoids are distinctive because they link with protein molecule receptors — called cannabinoid receptors — on the surface of cells. For instance, when a person smokes marijuana, the cannabinoid THC activates these receptors. Because the body’s natural cannabinoids control a variety of factors — such as pain, mood and appetite — they’re attractive targets for drug discovery and development. Piomelli is one of the world’s leading endocannabinoid researchers. His groundbreaking work is showing that this system can be exploited by new treatments to combat anxiety, pain, depression and obesity.”

http://www.sciencedaily.com/releases/2012/09/120925121349.htm

Toward Fulfilling the Promise of Molecular Medicine in Fragile X Syndrome

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“Fragile X syndrome (FXS) is the most common inherited form of mental retardation and a leading known cause of autism. It is caused by loss of expression of the fragile X mental retardation protein (FMRP), an RNA-binding protein that negatively regulates protein synthesis. In neurons, multiple lines of evidence suggest that protein synthesis at synapses is triggered by activation of group 1 metabotropic glutamate receptors (Gp1 mGluRs) and that many functional consequences of activating these receptors are altered in the absence of FMRP. These observations have led to the theory that exaggerated protein synthesis downstream of Gp1 mGluRs is a core pathogenic mechanism in FXS. This excess can be corrected by reducing signaling by Gp1 mGluRs, and numerous studies have shown that inhibition of mGluR5, in particular, can ameliorate multiple mutant phenotypes in animal models of FXS. Clinical trials based on this therapeutic strategy are currently under way. FXS is therefore poised to be the first neurobehavioral disorder in which corrective treatments have been developed from the bottom up: from gene identification to pathophysiology in animals to novel therapeutics in humans. The insights gained from FXS and other autism-related single-gene disorders may also assist in identifying molecular mechanisms and potential treatment approaches for idiopathic autism…

It is already clear that diverse molecular mechanisms can contribute to the synaptic abnormalities that underlie ASDs. In order to design appropriate therapeutic strategies for idiopathic autism, it will be critical to identify biomarkers that report the pathophysiological processes at work in the brains of the affected individuals.

Based on these findings, treatments that successfully target protein synthesis pathways in the single-gene disorders mentioned above, including mGluR5 modulators, may very well have broader therapeutic applications in idiopathic autism.”

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3100156/

 

Abnormal mGlu 5 Receptor/Endocannabinoid Coupling in Mice Lacking FMRP and BC1 RNA

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“Transcriptional silencing of the gene encoding the fragile X mental retardation protein (FMRP) causes fragile X syndrome (FXS)…

Our data indicate for the first time that mGlu5R-driven endocannabinoid signaling in the striatum is under the control of both FMRP and BC1 RNA. The abnormal mGlu5R/2-AG coupling found in FMRP-KO mice emphasizes the involvement of mGlu5Rs in the synaptic defects of FXSand identifies the modulation of the endocannabinoid system as a novel target for the treatment of this severe neuropsychiatric disorder.

In conclusion, this is the first study addressing endocannabinoid system in a model of FXS. Our results show that dysfunctional mGlu5R signaling leads to abnormal 2-AG metabolism and physiological activity, and indicate that inhibition of 2-AG synthesis or activity at CB1Rs might be a useful treatment option in FXS patients. In this respect, recent investigations suggest that this modulation could be achieved not only by direct pharmacological blockade of CB1Rs, but also indirectly, for example through the inhibition of anandamide degradation or the stimulation of transient receptor potential vanilloid 1 (TRPV1) channels. These two components of the endocannabinoid system, in fact, have been shown to selectively interact with mGlu5R/2-AG coupling in striatal neurons, and might interfere with the synaptic alterations seen after FMRP ablation with less side effects than those of widespread pharmacological inhibition of CB1Rs, which control not only GABA but also glutamate synapses.”

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3055456/

Marijuana cannabinoids found to help combat autism

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“(NaturalNews) The cannabinoid compounds naturally found in many varieties of cannabis, also known more commonly as marijuana, may help children with autism spectrum disorders experience dramatic behavioral improvements, and potentially even full recovery from their symptoms. These are the findings of a new study published in the journal Nature Communications that help reinforce the growing body of evidence which shows that medicinal cannabinoids hold incredible potential in both treating and potentially curing chronic illness.

Daniele Piomelli from the University of California, Irvine (UCI) and her colleague Olivier Manzoni from Inserm, a French research agency, observed that marijuana cannabinoids are very closely related to the endocannabinoid transmitters naturally found in the brain that facilitate the transport of electrical signals between neurons. Known as 2-AG, these transmitters are responsible for regulating a whole host of important bodily processes, which include things like telling the body when it is hungry or when it is experiencing pain.

Children with autism spectrum disorders; however, including those who developed these disorders as a result of Fragile X syndrome, which is said to be the most commonly-known genetic cause of autism, often have poorly or non-functioning 2-AG, which necessitates chronic synaptic failure in the brain. Many children with Fragile X-induced autism end up becoming mentally disabled as a result of this synaptic failure, and have trouble developing basic motor skills like walking and talking, or learning how to behave in various social situations.

But taking marijuana cannabinoids, which as we pointed out in an earlier article are not psychoactive in the same way that tetrahydrocannabinol (THC) is (http://www.naturalnews.com/035759_cannabis_juicing_health.html), can help effectively block the enzymes that inhibit the proper function of 2-AG. In essence, marijuana cannabinoids essentially restore synaptic communication by feeding an ailing body the cannabinoids it lacks, which are absolutely vital for proper cell function and communication.

“Endocannabinoid compounds are created naturally in the body and share a similar chemical structure with THC … (and) are distinctive because they link with protein molecule receptors — called cannabinoid receptors — on the surface of cells,” explains the UCI report. “Because the body’s natural cannabinoids control a variety of factors — such as pain, mood and appetite — they’re attractive targets for drug discovery and development.””

Learn more: http://www.naturalnews.com/037445_marijuana_cannabinoids_autism.html#ixzz2DRNu5iDg

Cannabis-like chemical combats chief genetic cause of autism

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“Natural cannabis-like chemicals in the brain may help combat the leading genetic cause of autism, research has shown.

Scientists linked blockages in a signalling pathway dependent on the compounds, called 2-AG endocannabinoid transmitters, with symptoms of Fragile X syndrome.

Correcting the fault with drugs led to dramatic behavioural improvements in mice with a version of the condition.

Fragile X syndrome is the most common known genetic cause of autism.

It results from a mutation in the FMR1 gene on the female X chromosome. Men possess one copy of the chromosome, paired with a male Y chromosome, and women two.

Boys are much more likely to be born with Fragile X than girls. This is thought to be because with two X chromosomes, a defect in one may be compensated for by the other.

People with the syndrome suffer mental impairment, learning difficulties, and may be hyperactive or impulsive. They also possess notable physical characteristics such as an elongated face, flat feet and large ears.

“What we hope is to one day increase the ability of people with Fragile X syndrome to socialise and engage in normal cognitive functions,” said lead researcher Professor Daniele Piomelli, from the University of California at Irvine in the United States.

The study was the first to identify the role of endocannabinoids in the neurobiology of Fragile X, she said.

About endocannabinoids

Endocannabinoid compounds are created naturally in the body and share a similar chemical structure with THC, the primary psychoactive component of the marijuana plant, Cannabis.

Endocannabinoids are distinctive because they link with protein molecule receptors — called cannabinoid receptors — on the surface of cells. For instance, when a person smokes marijuana, the cannabinoid THC activates these receptors. And because the body’s natural cannabinoids control a variety of factors — such as pain, mood and appetite — they’re attractive targets for drug discovery and development.”

Read more: http://www.belfasttelegraph.co.uk/news/health/cannabis-chemical-combats-chief-genetic-cause-of-autism-16216090.html#ixzz2DRLsbjJO