Beyond Achondroplasia

Growing together with Clara

A summary of achondroplasia´s potential treatments

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I have been following for several months the search work of prof. Morrys Kaisermann around achondroplasia latest researchs. He keeps a blog about achondroplasia named “Acondroplasia-Achondroplasia” and his lastest post is a brilliant synopsis of the current achondroplasia potential treatments and I must share some of the most important information of his post here.

You can read the complete post  here

So, at this point, there are three drugs that will potentially treat or reduce with expected significance the anatomic effects of achondroplasia produced by the aberrant FGFR3 activity in the growth plate of long bones:

1- BMN-111 – a CPN analoge from BioMarin´s pharmaceutical, under evaluation in a clinical trial, now at phase 2, a cohort dose-escalating study.

2- Soluble FGFR3 – still with no published information of a pharmaceutical willing to take it to a clinical trial and produce it.

3-Meclizine – alrealdy at the market for several years and it´s a low cost anti-histaminic used to treat vertigo and motion sickness and also a research team found recently that also inhibits ERK, one of the enzymes located in the signaling cascade of FGFR3.

In the post, there was also mentioned some of the potential treatments/drugs that for one or other reason were left aside:

1- GH (Growth hormone)- doesn´t work in achondroplasia, showing just irrelevant changes in height.

2- PTH – parathyroid hormone

3- NC-2 – most probably, the interest in this molecule for further studies by a pharmaceutical is over (before it really began)

4- Vessel dilator – with no lab animal evaluation for the hypothesis of achondroplasia treatment

5- NF449 -anti-FGFR3 tyrosine kinase inhibitor

6- A31 – another anti-FGFR3 tyrosine kinase inhibitor that induces a large decrease in the FGFR3 expression in mutant femurs

7- Aptamers – not under research for achondroplasia


I still wonder for a genetic engineering solution for achondroplasia in a near future.

Let´s see and work for updates and news!





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