Beyond Achondroplasia

Growing together with Clara

Statins for achondroplasia update

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I contacted Professor Noriyuki Tsumaki, the head researcher for the study of the use of statins in achondroplasia, also mentioned in a post here.

I kindly got  the following asnwers:

Statins should not be used in children because statins decrease cholesterol, an essential substance for the growth and development of children. We need to do further examination on the appropriate delivery and dose for skeletal dysplasia treatment“.

Statins are a class of drugs renown for their action against cholesterol and investigated because they have anabolic and protective effects on chondrocytes. Anabolic effect is the set of metabolic pathways (chemical reactions occurring within a cell) that construct molecules from smaller units.

In this study, authors used iPS cells generated from the fibroblasts of both healthy individuals (WT-iPSC) and TD patients (TD-iPSC). Statin treatment was found to accelerate the degradation of the FGFR3 protein in chondrogenically differentiated TD-iPSC, a process inhibited in TD cases.

Tsumaki_Nature_140918-thumb-650x291-1469

Image from CiRA newsroom, Kyoto University. Statins were given daily by intraperitoneal injections (inside the bone).

Dr. Tsumaki’s group shows that an injection of 1 mg per kg of rosuvastatin into the ACH mouse model restored bone growth in the limbs and head. However, this dose would translate into 70 mg per day for a 70 kg human, which far exceeds the 20 mg limit in Japan and 40 mg limit in Europe and the U.S.

The major feedback was that:

Regarding to his future plan, Prof. Tsumaki group hopes to conduct a clinical study if we can confirm that appropriate delivery and dose to children cause no side effects and improve their symptoms.

“This study is an important research result, as it demonstrates that patient-specific iPSCs can be an effective tool for drug repositioning. I hope this approach will be a model to help us find new cures for other intractable disease”

Here you can find the full press release on the study.

Prof. Tsumaki is the principal investigator at the Center for iPS Cell Research and Application, Kyoto University in Japan and his studies are focused in iPS cell technology, induced pluripotent stem cells.

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