An Acne Drug Might Be Able to Treat Heart Disease, Says New Study

Dilated cardiomyopathy (DCM) is a highly fatal disease of the cardiac muscle that predominantly affects the left ventricle of the heart. The left ventricle is responsible for pumping blood to the rest of the body to maintain adequate blood perfusion. It is the most common type of cardiomyopathy, affecting 1 in 250 people. It mainly affects adults between the ages of 20 and 60 years.

In addition to dilated cardiomyopathy’s effect on the ability of the heart to pump blood to the rest of the body, it can also result in severe arrhythmias, thrombus formation, and even sudden cardiac death (SCD). Dilated cardiomyopathy is one of the leading causes of Heart failure which is in itself a life-threatening condition.

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How to treat DCM and associated Heart Failure

Unfortunately, dilated cardiomyopathy cannot be reversed once it occurs. However, there are several supportive treatment regimens to prevent its progression to heart failure. Symptomatic treatment of high blood pressure or abnormal heart rhythm may also be prescribed to a patient suffering from DCM.

Heart rate and rhythm may be controlled using a pacemaker or an ICD. Despite several methods of treatment, if it reaches end-stage heart failure, none of these can be applied. In that case, only a heart transplant might be curative. However, heart donors are minimal and the need for them is maximal.

What does the new study suggest?

A new study was conducted as a collaboration between the European Molecular Biology Laboratory in Heidelberg and Stanford University with the objective of studying the genetic makeup of dilated cardiomyopathy.

The authors of the study emphasized the importance of this study by saying, ‘We need therapeutic strategies that target the cause of the disease in a personalized medicine approach.

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Methods of the study

In this study, researchers were presented with a unique opportunity to be able to study the genetic pathway of inherited dilated cardiomyopathy in an entire family that suffers from it. The team of scientists studied the genetic makeup of all the members of the family and divided them into three groups; those who had been diagnosed with DCM, those who were disease-free, and those who passed away due to dilated cardiomyopathy.

Results of the study

On examination of all members in these groups, the researchers found that the inherited dilated cardiomyopathy was associated with the protein, RBM20 encoded by the mutated gene, P633L. This gene played an important role in the development and progression of dilated cardiomyopathy.

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To prove that the mutation was responsible for the development of inherited dilated cardiomyopathy they performed gene editing to introduce the mutated protein to patient-derived induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs). The research has shown that splicing and cellular defects in the in-vitro models were due to the mutation in the iPSC-CMs.

Researchers ran the gene and the protein it encodes for through a database to find an inhibitor or blocker for this gene. Through this wide database search, they found a chemical compound, all-trans retinoids acid (ATRA) that could be used as a therapeutic agent for dilated cardiomyopathy.

This chemical compound, all-trans retinoic acid (ATRA), is used for the treatment of acne and for the treatment of acute promyelocytic leukemia. This compound is beneficial in the regulation of the protein encoded by the mutated gene.

Researchers are trying to use this compound as a treatment for mutation-dependent inherited dilated cardiomyopathy and the mutated gene to understand the non inherited dilated cardiomyopathy more.

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iPSC Modeling of RBM20-Deficient DCM Identifies Upregulation of RBM20 as a Therapeutic Strategy



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