HomeLifestyleHealth & FitnessA Medical Breakthrough: Heart Failure No Longer a Death Sentence and Can...

A Medical Breakthrough: Heart Failure No Longer a Death Sentence and Can Be Reversed

Published on

Cardiac Amyloidosis: Not Incurable Anymore? A New Medical Trial Has Seen Devastating Heart Condition Reversed in Three Patients

Breakthrough in heart disease research: for the first time a new study shows how “the heart can get better with this disease.” 

In an extraordinary turn of events, a trio of male patients, who had previously suffered from heart failure due to an accumulation of harmful proteins, have experienced a spontaneous reversal of their condition. This remarkable development has been reported by a research team from University College London (UCL) and the Royal Free Hospital.

These patients were diagnosed with a progressive form of amyloidosis – a condition that impacts the heart and was previously believed to be irreversible, leading to death in 50% of patients within four years post-diagnosis.

The latest findings, published today in The New England Journal of Medicine, focus on three men aged 68, 76, and 82 who were diagnosed with transthyretin cardiac amyloidosis and subsequently experienced recovery. Their personal accounts of symptom improvement were further substantiated by thorough medical evaluations, including cardiovascular magnetic resonance (CMR) scans, which revealed a noticeable decrease in amyloid protein buildup in their hearts.

Professor Marianna Fontana, the lead author of the study and a part of the UCL Division of Medicine, remarked, “We have seen for the first time that the heart can get better with this disease. That has not been known until now and it raises the bar for what might be possible with new treatments.”

In addition, the researchers identified a unique immune response within the three men that specifically targeted the amyloid proteins. This particular type of amyloid-fighting antibodies was not found in other patients whose condition followed the usual disease progression.

“Whether these antibodies caused the patients’ recovery is not conclusively proven,” remarks Senior author Professor Julian Gillmore.

“However, our data indicates that this is highly likely and there is potential for such antibodies to be recreated in a lab and used as a therapy.  We are currently investigating this further, although this research remains at a preliminary stage.”

Transthyretin (ATTR) amyloidosis is a health condition arising from the accumulation of amyloid deposits in the bloodstream, which are made up of a blood protein known as transthyretin (TTR). This condition can be genetic or non-genetic, commonly referred to as “wild-type.” When these proteins accumulate in the heart, it leads to a condition known as ATTR amyloid cardiomyopathy (ATTR-CM). Present treatments under the NHS aim to alleviate heart failure symptoms like fatigue, leg or abdominal swelling, and breathlessness during activities. However, they don’t address the underlying amyloid issue. Nonetheless, several experimental “gene-silencing” therapies are under trial, which aim to reduce TTR protein levels in the bloodstream and slow the amyloid buildup.

Significant advancements in medical imaging technology, some of which were developed at the UCL Centre for Amyloidosis, have allowed for increased diagnosis of the disease compared to two decades ago. In the past, a biopsy was necessary for diagnosis, involving heart tissue extraction.

These improved imaging technologies also facilitate better tracking of the amyloid burden on the heart and the disease’s progression. It becomes simpler to identify instances where the disease has reversed, as opposed to just remaining stable.

The current study, funded by the Royal Free Charity, was sparked by a 68-year-old man reporting symptom improvement. This led the research team to sift through the records of 1,663 patients diagnosed with ATTR-CM, revealing two additional similar cases.

These three men’s recovery was verified through blood tests, multiple imaging methods such as echocardiography (a type of ultrasound), CMR scans, scintigraphy (a nuclear medicine bone scan), and in one instance, an assessment of physical activity capacity. The CMR scans indicated the heart’s structure and functionality were near-normal, and the amyloid was almost entirely removed.

A detailed review of the records and assessments of the remaining 1,663 patients showed that these three men were the sole cases of disease reversal.

An intriguing aspect was that one of the three men showed an abnormal inflammatory response around the amyloid deposits (including macrophages – a type of white blood cells), indicating an immune response. Such a response wasn’t observed in 286 biopsies from patients with typical disease progression.

Further investigation revealed specific antibodies in the three patients that bound directly to ATTR amyloid deposits in both mouse and human tissue and synthetic ATTR amyloid. These antibodies were absent in another 350 patients with a standard clinical course.

If these antibodies could be utilized, they could be amalgamated with new therapies in trial that suppress TTR protein production, thus enabling clinicians to not only prevent further amyloid buildup but also remove existing amyloid.

One such promising therapy under trial is NTLA-2001, a unique gene-editing therapy using CRISPR/Cas9. Preliminary results of the trial, headed by Professor Gillmore, suggest it might halt disease progression.

“This work not only represents a major breakthrough in our understanding of cardiac amyloidosis, but crucially opens up new possibilities for more effective treatment options,” comments Jon Spiers, chief executive of the Royal Free Charity.

“It’s a hugely significant development that we welcome on behalf of all patients of the National Amyloidosis Centre and their families, many of whom have contributed to our research funding with their own fundraising efforts.”

Image Credit: Shutterstock

Latest articles

Here’s How and When Mount Everest-sized ‘Devil Comet’ Can Be Seen With Naked Eye

Mount Everest sized Comet 12P/Pons-Brooks, also known as "devil comet" which is making its...

Something Fascinating Happened When a Giant Quantum Vortex was Created in Superfluid Helium

Scientists created a giant swirling vortex within superfluid helium that is chilled to the...

The Science of Middle-aged Brain and the Best Thing You Can Do to Keep it Healthy, Revealed

Middle age: It is an important period in brain aging, characterized by unique biological...

Science Shock: Salmon’s Food Choices Better at Reducing Risk of Heart Disease and Stroke

Salmon: Rich in Health Benefits, Yet May Offer Less Nutritional Value - This is...

More like this

Here’s How and When Mount Everest-sized ‘Devil Comet’ Can Be Seen With Naked Eye

Mount Everest sized Comet 12P/Pons-Brooks, also known as "devil comet" which is making its...

Something Fascinating Happened When a Giant Quantum Vortex was Created in Superfluid Helium

Scientists created a giant swirling vortex within superfluid helium that is chilled to the...

The Science of Middle-aged Brain and the Best Thing You Can Do to Keep it Healthy, Revealed

Middle age: It is an important period in brain aging, characterized by unique biological...