We're a little crazy, about science!

Fragile X and a new autism treatment


There are many roads to autism, none of them involve vaccination. I get tired of saying that, but it’s simple science, vaccines do not cause autism. Thankfully science knows this and is looking at not only what actually causes autism (Since again there are many different ways to develop on the spectrum). Well they may have found a new treatment for people affected by a common inherited form of autism by using a drug that is being tested as a treatment for cancer.

Researchers who have identified a chemical pathway that goes awry in the brains of Fragile X patients say the drug could reverse their behavioural symptoms. The scientists have found that a known naturally occurring chemical called cercosporamide can block the pathway and improve sociability in mice with the condition.

The team identified a key molecule – eIF4E – that drives excess protein production in the brains of Fragile X patients. This can cause behavioural symptoms that include learning difficulties. It can also lead to more serious intellectual disabilities, delays in speech and language development and problems with social interactions.

They found that eIF4E regulates the production of an enzyme called MMP-9, which breaks down and re-orders the connections between brain cells called synapses. This disrupts communication between brain cells leading to changes in behaviour.

Treatment with cercosporamide blocks the activity of eIF4E, and therefore reduces the amounts of MMP-9, and reverses the behavioural symptoms in mice with a version of Fragile X Syndrome, the team showed.

Cercosporamide is being tested as a treatment for lung cancer and acute myeloid leukemia. The new findings suggest that it could also have a use as a treatment for patients with Fragile X Syndrome.

Fragile X Syndrome is the most common genetic cause of autism spectrum disorders. It affects around 1 in 4000 boys and 1 in 6000 girls. Currently, there is no cure.

“Our findings open the door to targeted treatments for Fragile X Syndrome. By designing treatments that block just this pathway, it is hoped that we can limit the potential side-effects and develop therapies that are more efficient than general treatment approaches,” said Dr Christos Gkogkas, researcher of Fragile X syndrome and intellectual disabilities.

“Dr. Gkogkas is an important addition to the Patrick Wild Centre. His research provides important insights into the brain processes underlying the symptoms of people with Fragile X Syndrome and other intellectual disabilities. Only with this knowledge can better medicines to treat these conditions be developed,” said Professor Peter Kind, Director of the Patrick Wild Centre at the University of Edinburgh.

“We found that eIF4E regulates the production of an enzyme called MMP-9, which breaks down and re-orders the connections between brain cells called synapses. Excess MMP-9 disrupts communication between brain cells, leading to changes in behaviour,” Professor Nahum Sonenberg said.

Once again I need to say this, since it may not have been clear in the intro, VACCINES DO NOT CAUSE AUTISM!! We know this because we know lots of things that do cause autism, none of which has anything remotely to do with the immune system. In fact if the people who thought vaccines caused autism understood the immune system (or autism for that matter) they would see it would be all but impossible.

So in short, vaccinate your children don’t risk their lives because you are scared. Vaccines do not cause autism and really as anyone with an autistic child will tell you, they are just as special and lovable as any child. Sure being a parent is scary and when something like autism gets thrown into the mix it can be even more scary, but we are finding more and more ways to give autistic children a normal life.

People are working hard to help find ways to treat and prevent autism, don’t take that away by not vaccinating your children out of fear, support the science, not the people trying to scare you because they want fame or money.

Gkogkas, C., Khoutorsky, A., Cao, R., Jafarnejad, S., Prager-Khoutorsky, M., Giannakas, N., Kaminari, A., Fragkouli, A., Nader, K., Price, T., Konicek, B., Graff, J., Tzinia, A., Lacaille, J., & Sonenberg, N. (2014). Pharmacogenetic Inhibition of eIF4E-Dependent Mmp9 mRNA Translation Reverses Fragile X Syndrome-like Phenotypes Cell Reports DOI: 10.1016/j.celrep.2014.10.064

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