A group of heart failure patients have seen their condition spontaneously reversed in an ‘unprecedented case’.
Each of the three men suffered from heart failure due to cardiac amyloidosis, a build-up of toxic proteins in the heart that makes it difficult for the organ to function properly.
The chronic disease worsens over time, is sometimes fatal, and until now was thought to be irreversible.
Scientists from University College London (UCL) and the Royal Free Hospital carried out medical tests on the men, aged 68, 76 and 82, after reports that their symptoms were improving.
The scans showed that some toxic proteins had cleared from their hearts, which appeared to be recovering from the disease.
The team also found promising evidence for antibodies that specifically target these proteins.
Further tests revealed that these antibodies were not found in other patients whose condition had progressed normally.
Medical professionals hope the findings could lead to new treatments for the condition.
Three men suffered from a progressive condition previously thought to be incurable
(PA)
Lead author Professor Mariana Fontana from UCL’s Department of Medicine said: “This is the first time we have seen that the heart can improve with this disease.
“This has not been known before and raises the bar for what may be possible with new treatments.”
In the study, researchers looked at a specific type of condition that is caused by the build-up of a blood protein called transthyretin (TTR).
In the UK, this variant, known as ATTR-CM, is currently unknown due to potential under-diagnosis and under-reporting.
Researchers looked at records from 1,663 patients with the condition after one of the men, aged 68, said his symptoms were improving.
Two more cases of improvement were identified and confirmed through blood tests, scans and medical imaging techniques.
The team found antibodies in the three patients that bind specifically to ATTR amyloid deposits in mouse and human tissue in the lab.
Using these antibodies could help clear the toxic proteins and potentially reverse the condition, the researchers said.
Scientists led by Professor Julian Gilmore from UCL’s Department of Medicine are investigating a gene-editing therapy that could halt the progression of cardiac amyloidosis.
Early results from the trial show it can stop the progression of the disease, the team said.
Prof Gilmour, who is also head of the UCL Amyloidosis Centre, based at the Royal Free Hospital, said: ‘It has not been conclusively proven whether these antibodies caused the patients to recover.
“However, our data show that this is very likely, and there is potential for such antibodies to be recreated in the laboratory and used as therapy.”
“We are currently investigating this further, although this research remains at a preliminary stage.”
The findings were published in The New England Journal of Medicine.
John Spiers, chief executive of the Royal Free Charity, which supported the research, said: “This work not only represents a major breakthrough in our understanding of cardiac amyloidosis, but crucially opens up new opportunities for more effective treatment options.”