Encouraging results of a reused Alzheimer’s drug for fragile X syndrome: shots


Fragile X syndrome involves changes to the X chromosome, which are seen in the four columns of chromosomes starting at the left. Two normal X chromosomes are shown in the far right column.

Richard J. Green / Science Source

An experimental drug intended for Alzheimer’s patients appears to improve language and learning in adults with Fragile X syndrome.

Drug called BPN14770, Cognitive scores increased by 10% in 30 adult males after 12 weeks, team Reports In the magazine Nature Medicine.

This is enough to change the lives of many people with Fragile X Mark Journey, CEO of Tetra Therapeutics, drug developer.

“People with impaired X with an IQ of 40 usually live with their parents or in a corporate environment,” says Gurney. “With an IQ of 50, in some cases they are able to ride the bus, be able to take a job with some help and be able to function better in their community.”

But it will take a much larger study to see if a drug is as good as it appears or not Mark BeerPicower, professor of neuroscience at MIT.

“This study is definitely not conclusive, but it is encouraging,” he says.

Fragile X syndrome is a genetic disorder that affects about 1 in 4,000 males and a smaller percentage of females. It is the most common genetic cause of intellectual disabilities and autism.

The idea for treating Fragile X with Alzheimer’s medication came from Gurney after he learned that both conditions affect a substance called cyclic AMP that helps transmit messages within cells.

Tetra was developing BPN14770 as an Alzheimer’s drug that works by treating cyclic AMP, “so we thought there was a strong possibility that this drug would be effective in Fragile X,” Gurney says.

call FRAXA, A foundation that has funded research on disorder since the mid-1990s. The Foundation arranged for Tetra to be tested on animals; Then I agreed to fund the study in individuals.

The apparent success comes after seven years of taking two other promising Fragile X drugs Did not work When testing it on people.

FRAXA co-founder Katie Clapp says the positive result “compensates for some of the devastation that occurred years ago when we faced such notable failures.”

Clapp says the results also give her new hope for her son Andy Tranvaglia, a 31-year-old with Fragile X Syndrome.

“The amazing thing about the results of this experiment is that they were able to show that learning improved,” says Clapp.

Dr Michael Tranvaglia, a dog husband and co-founder of FRAXA, says in some previous studies, drugs that had dramatic results in mice failed to behave the same way as humans.

With BNP14770, “we’ve seen an almost complete translation of these results that we’ve seen in mice into the human condition,” he says.

Tranvaglia says at least one promising candidate drug may have failed because people developed a tolerance to it.

“The only thing we know [BPN14770] He says the longer you work, the better. “It keeps doing better and better.”

Bear says that if the new drug’s effectiveness in adults is confirmed, it will likely be more successful in children.

“Fragile X can be visualized as a deviation from the normal maturation of the brain,” says Bear. “So the earlier we can get there and correct the course of development, the more dramatic the improvement will be.”

Tetra Therapeutics is working with its parent company, Japanese pharmaceutical company Shionogi, to launch a much larger study of the new drug this summer.

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