For patients

CureHeart - information for patients and families

https://www.youtube.com/embed/_hzPm2j7UFg?wmode=opaque&controls=&rel=0

Expand All

Cardiomyopathies are a group of diseases that affect heart muscle, affecting its ability to work properly and pump blood around the body. Approximately 260,000 people in the UK, and 1.5 million in the US, have cardiomyopathy; many are unaware that they may be at risk. 

Cardiomyopathies are a major medical, psychosocial and socio-economic burden for patients, families and society; sudden cardiac death and heart failure in the young are catastrophic and costly.

Find out more about cardiomyopathy from our patient involvement partners Cardiomyopathy UK, and read an introduction to the heart and circulatory system. 

You can also download a PDF about cardiomyopathies. 

We recognise the central importance of involving patients and family members at every stage of the CureHeart study. We have partnered with Cardiomyopathy UK and, in the US, the SHaRe Cardiomyopathy Registry, and will continue to work with patients and families throughout the study. The partner charities and the patient panel will be represented on the Scientific Steering Committee.

Throughout the study, patient input will be sought for activities including: 

  • development of patient/participant recruitment strategies
  • evaluation of attitudes to risk:benefit 
  • feedback on educational needs 
  • input to preparation of educational material
  • development of strategies to report findings

To begin to understand patients’ views, we set up a panel drawn from members of Cardiomyopathy UK, which met in two sessions in summer 2020. We provided an overview of the planned research and asked for panel views on the acceptability of the research, as well as understanding key issues of importance for patients and families. A similar exercise was also carried out in parallel in the US. 

We are currently running a survey to understand how our patient group might consider participation in a gene therapy trial in a range of different scenarios. 
 

Gene therapy is a new kind of treatment, and will need to be considered by a range of different organisations. One example is the Medicines and Healthcare products Regulatory Agency (MHRA), which regulates medicines and sets standards to ensure safety, quality and effectiveness in the UK. The US Food and Drug Administration (FDA) serves a similar role in the US. We will work with organisations like the MHRA and equivalent organisations elsewhere throughout the research programme.

We will establish an Ethics Advisory Group which will be chaired by Professor Mike Parker, the Director of the Wellcome Centre for Ethics and Humanities and the Director of the Ethox Centre at the University of Oxford. The Wellcome Centre for Ethics and Humanities conducts research on the ethical challenges that are posed by advances in neuroscience, data science, genomics and global connectedness, and their convergence. 

The patient panel and other relevant stakeholders will also be represented on a group, which will oversee the ethical framework under which the study will be conducted.  This will include providing a forum for discussion of ethical issues arising, and will feed into the Scientific Steering Committee. 

It is possible that CureHeart will raise new ethical and policy issues which we will need to study further. The Ethics Advisory Group and patient groups will work together with researchers, ensuring a seamless route for identifying and developing new issues. 

We are keen to hear from cardiomyopathy patients and their families, and we are currently piloting a survey to understand what patients with cardiomyopathy (and their families) think about participating in potential gene therapy trials. If you are a patient, or have a family member affected by cardiomyopathy, you can take part in the survey.  

The gene therapy treatment that our research team are planning to develop is not yet at the clinical trial stage. It will need to be trialled extensively before it is available in the clinic from your healthcare providers, and we will update this website when we begin recruiting for trials. 

The British Heart Foundation's Big Beat Challenge is a global competition, where international, multi-disciplinary groups of researchers were asked to identify and propose a transformational solution to a significant problem in a heart or circulatory disease.

The most compelling proposal will win a research award of up to £30 million, and the opportunity to accelerate breakthroughs that could transform lives across the globe. 

We are one of the four finalists for the Big Beat Challenge, and you can find out more about this research competition on the British Heart Foundation website