Project: Cardiomyopathy Drug: Brief description

Development of drugs for the untreatable diseases Hypertrophic (HCM) and Dilatative (DCM) Cardiomyopathy

HCM and DCM are relatively rare genetic heart diseases. HCM affects about 1:500 of the population. Over 1000 causing mutations have been discovered in various sacromeric proteins since 1995, which affect either directly or indirectly the interactions between the myosin and actin proteins thus modulating the heart muscle movement. The aim of our project, and suggested collaboration, is a development of drug against the untreatable diseases HCM and DCM but initially for cats and dogs not for humans.

In both the HCM and DCM the myosin – actin protein interactions are modulated but in a different way

Why now

Lack of solid scientific information how the genetic mutations affects the target proteins

  • There was no synonymous answer whether the HCM and DCM mutations provoke an increase or decrease in the myosin-actin interactions. Thus it was not clear what type of drug (an inhibitor or activator) should be developed. Such information became available in 2013.
  • The first gene directly linked to the PSORIASIS has been found in 2013 and the network of protein interactions became more evident in 2015
  • There was no sufficient innovation technology for the realization of such project outside of the big pharmacy companies.
  • Genetic screening became much cheaper in the last couple of years thus wider available to community

There was no sufficient scientific information was available up to 2013 and the first potential drugs for similar heart diseases have been developed after 2013. On the other hand there was no technology available such drug to be developed outside the big pharmaceuticals companies.

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