{"id":13386,"date":"2021-07-01T11:29:58","date_gmt":"2021-07-01T16:29:58","guid":{"rendered":"https:\/\/last-drum.flywheelsites.com\/?p=13386"},"modified":"2024-04-28T17:45:52","modified_gmt":"2024-04-28T22:45:52","slug":"alzheimers-drug-appears-to-benefit-adult-men-with-fragile-x","status":"publish","type":"post","link":"https:\/\/autism.org\/alzheimers-drug-appears-to-benefit-adult-men-with-fragile-x\/","title":{"rendered":"Alzheimer\u2019s drug appears to benefit adult men with fragile X"},"content":{"rendered":"

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A drug intended to help people with Alzheimer\u2019s disease may also be beneficial for individuals with fragile X syndrome, according to a new study.<\/p>\n

Fragile X syndrome is the most common known genetic cause of autistic-like symptoms and the most common cause of inherited intellectual disability. It affects about 1 in 4,000 males and 1 in 8,000 females.<\/p>\n<\/div>

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Fragile X syndrome is the most common known genetic cause of autistic-like symptoms and the most common cause of inherited intellectual disability. It affects about 1 in 4,000 males and 1 in 8,000 females.<\/p>\n

In a 24-week randomized, placebo-controlled, two-way crossover study, Elizabeth Berry-Kravis and colleagues tested the Alzheimer\u2019s drug, called BPN14770, on 30 adult male patients with fragile X syndrome. Study co-author Mark Gurney became interested in the drug when he learned that both autism and Alzheimer\u2019s affect a substance called cyclic AMP (cAMP), which helps transmit messages inside cells and plays a critical role in memory formation. Researchers in Germany had developed BPN14770 to inhibit the activity of an enzyme called PDE4D, increasing the levels of cyclic AMP in the brain.<\/p>\n

The drug was first tested on animals before being tested on humans. Unlike several other drugs that worked well in animal models but were ineffective in people with fragile X syndrome, BPN14770 appears to be effective for both animals and humans.<\/p>\n

The researchers found that cognitive scores increased by about 10% in participants receiving the drug, and language and daily functioning skills improved significantly. Moreover, no differences in side effects were seen between the active and placebo groups.<\/p>\n

Berry-Kravis says, \u201cThe majority of clinical outcome measures were in favor of the drug. These measures included performance-based assessments, biomarkers, and parent and physician-rated scales, which in combination suggest a meaningful impact on the global [fragile X] disease process.\u201d She adds that it is exciting that the drug \u201cpotentially addresses a core biochemical deficit in [fragile X], a deficiency of cAMP,\u201d which has been documented in individuals with fragile X syndrome. The researchers note, however, that larger trials are needed.<\/p>\n

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\u201cInhibition of phosphodiesterase-4D in adults with fragile X syndrome: a randomized, placebo-controlled, phase 2 clinical trial,\u201d Elizabeth M. Berry-Kravis, Mark D. Harnett, Scott A. Reines, Melody A. Reese, Lauren E. Ethridge, Abigail H. Outterson, Claire Michalak, Jeremiah Furman, and Mark E. Gurney, Nature Medicine<\/em>, April 29, 2021 (online). Address: Elizabeth Berry-Kravis, Department of Pediatrics, Neurological Sciences, and Biochemistry, Rush University Medical Center, Chicago, IL 60612.<\/p>\n

\u2014and\u2014<\/p>\n

\u201cTreatment found to improve cognitive function in patients with fragile X syndrome,\u201d news release, Rush University Medical Center, April 29, 2021.<\/p>\n

\u2014and\u2014<\/p>\n

\u201cAn Alzheimer\u2019s drug may boost cognition in people with fragile X syndrome,\u201d Jon Hamilton, NPR, April 30, 2021.<\/p>\n

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\nThis article originally appeared in Autism Research Review International, <\/em><\/a>Vol. 35, No. 2, 2021<\/em><\/p>\n<\/div>

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