Research Highlights

Cholesterol lowering drug found to prevent brain damage

doi:10.1038/nindia.2019.74 Published online 9 June 2019

Lovastatin, a cholesterol-lowering drug, prevents brain damage in rats afflicted with fragile X syndrome (FXS), a genetic disorder that causes a range of developmental problems including learning disabilities and memory impairment, researchers have found1.

The finding suggests that early drug interventions could permanently and positively alter neural development in patients with FXS and potentially similar conditions.

Mutations in the FMR1 gene cause FXS, which affects one in 4,000 males and one in 8,000 females. The FMR1 gene provides instructions for making a protein called FMRP. This protein helps regulate the production of other proteins and plays a role in the development of synapses, which are connections between nerve cells that which relay nerve impulses, maintaining the functions of myriad brain circuits.

To better understand this disorder, an international research team, including scientists from the Centre for Brain Development and Repair, InStem, in Bangalore, India, studied a rat model of FXS and conducted several tests of associative memory and learning.

They first found that rats missing the Fmr1 gene showed delays in object recognition and associative memory at all ages. The scientists then administered lovastatin – a drug approved for treating high levels of blood cholesterol – to the Fmr1-deficient rats between five and nine weeks of age.

The researchers found that the drug restored the proper development of associative learning in the rats. Amazingly, these benefits persisted for several months after the treatment stopped, indicating that early lovastatin treatment could help preserve cognitive abilities in patients.


References

1. Asiminas, A. et al. Sustained correction of associative learning deficits after brief, early treatment in a rat model of Fragile X Syndrome. Sci. Transl. Med. 11, eaao0498 (2019)