Statins rescue dwarfism?

In the past few days, several Chinese media said statins may be effective for dwarfism (侏儒症). In fact, this idea originated from a study published on September 17 in Nature[1]. However, it is overstated and misleading.

Gain-of-function mutations in the FGFR3 gene result in skeletal dysplasias, such as thanatophoric dysplasia (TD, 致死性骨发育不全) and achondroplasia (ACH, 软骨发育不全). Japan’s scientists established iPS cells (a kind of stem cell) from skin cells of both healthy individuals (wild type, WT) and TD patients. Chondrocytes differentiated from TD-iPS cells produced less cartilage than those from WT-iPS cells.

The authors found that both FGFR3 neutralizing antibody and statins rescue the cartilage formation in TD-iPS cells. They point out that statins may accelerate the degradation of FGFR3 protein. The team then injected ACH model mice with 1.0 mg/kg rosuvastatin, demonstrating this drug is effective in vivo.

Statins are a class of common drugs used to lower cholesterol. Because cholesterol is important for the growth and development of children, I don’t think statins are safe enough for children. The author Noriyuki Tsumaki also urged that patients not take statins on their own.

Moreover, the authors said “1.0 mg/kg in model mice is equivalent to 70 mg per day in a 70 kg human”. This conversion may be oversimplified. Body surface area is a better parameter than body weight for extrapolating the animal dose to the equivalent human dose.

[1] Nature. 2014, doi: 10.1038/nature13775.

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