A New Drug for Hypertrophic Cardiomyopathy: One Patient’s Remarkable Recovery

Someone emailed me this week with a question about whether lung conditions affect the heart (and vice versa!) and it struck me that would be a great topic to cover. I’ll start with this week’s video, then write a follow up next week.

However for this week’s blog, I have to write about something I saw with my own eyes this week the likes of which I don’t think I’ve seen in over 20 years of being a doctor. I’ve written before about a new drug for the condition hypertrophic obstructive cardiomyopathy. Whilst actually relatively common (1 in 500 people have a gene that could cause it, and you may well have heard of it – it’s the commonest inherited heart condition and is often behind tragic sudden sports deaths in young people), this particular treatment is pretty niche. Nevertheless, this patient story is really powerful.

Many patients with the disease have significant symptoms of breathlessness, chest pain and dizziness, which can be challenging to manage. For a proportion, the ‘gold standard’ treatment is open heart surgery. However that is all set to change with the new drug Mavacamten. It is the first drug of its kind, working on the actual force-generating component of heart muscle cells. Only certain hospitals and specialists in the UK have been chosen to prescribe the drug, and I’m honoured to be one of them. This week, my first two patients who I have given it to came back for their one month heart scan and review.

As this was a big deal, the hospital’s media team came along to film it, so there will be some video available in due course. I was a little nervous to meet my first patient in case he felt no better, but nothing could have been further from the truth. This was a man in his middle age, who only a month earlier had to stop several times with me as we walked the few hundred yards to the pharmacy to collect his first prescription due to breathlessness. When he told me he now had no symptoms whatsoever, I admit I was a little sceptical, and actually wondered if there was an element of placebo effect going on. 

However, when we did his heart scan – his heart had totally changed. 

In one month. 

The blockage that was there was gone!

No wonder he felt so much better! I read medical scientific papers each week, and am very familiar with the various measures and statistics used to evaluate new treatments. However to see and hear a fellow human who has had their life transformed in front of you is an altogether different experience, and incredibly humbling. It really is ‘laboratory bench to bedside medicine’. 

Notably, he said he had gone from being anxious to leave the house for fear of symptoms, to being anxious that he wouldn’t be able to access his new medication for some reason, and said he could sympathise with illicit drug users who would do anything for their next fix!

Now I totally appreciate the niche aspects of this particular drug, but as a story, this is one well worth sharing. This month is world heart month, so whilst most of us have no need for this specialist medication, now is a great time to think about your own heart health. All of us, myself included, could improve their heart health. There is no simple hack, no one treatment or supplement that will forever cure us. However the little steps we can each take each day are what add up to long-term benefit. 

For this heart health month, I’ll round off with a few questions:

Have you taken your blood pressure in the last 6 months?

Have you had your cholesterol taken in the last year?

Have you had a diabetes check in the last year?

Have you measured your height, weight and body mass index in the last 6 months?

If the answer is no, do yourself a favour – check them this month!

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