Three men, aged 68, 76, and 82, who suffered from heart failure due to the buildup of sticky, toxic proteins, have experienced an unexpected reversal of their condition.
This phenomenon, reported by a team at the University College London (UCL) and the Royal Free Hospital, is groundbreaking as their condition, a form of amyloidosis, was previously considered progressive and irreversible.
Unraveling Amyloidosis
Transthyretin (ATTR) amyloidosis, the condition the men suffered from, is caused by the accumulation of amyloid deposits composed of a protein called transthyretin.
These deposits, when built up in the heart, lead to ATTR amyloid cardiomyopathy (ATTR-CM), causing heart failure.
Current treatments only relieve the symptoms but don’t tackle the amyloid, making the spontaneous reversal of the condition in these three patients remarkable.
The Unlikely Reversal
This study was initiated when the 68-year-old patient reported an improvement in his symptoms.
This led the researchers to comb through the records of 1,663 patients with ATTR-CM, where they found two more similar cases.
Through the use of multiple imaging techniques and blood tests, it was confirmed that the heart function of these patients had returned to near normal and the amyloid had almost completely cleared.
The Immune Response Link
Upon investigating further, the researchers discovered an atypical immune response in these patients, indicated by antibodies that specifically targeted amyloid.
These antibodies were not found in patients whose condition progressed as normal.
While it’s not definitively proven that these antibodies caused the patients’ recovery, the findings indicate this as highly likely and open potential for these antibodies to be replicated in a lab for therapeutic use.
A Future of Potential Treatments
The discovery of these antibodies could enhance new therapies being trialed that suppress TTR protein production.
In essence, clinicians could potentially not only prevent further amyloid deposition but also clear away existing amyloid.
A promising therapy under investigation is a novel gene-editing treatment based on CRISPR/Cas9, which may halt disease progression.
The Bigger Picture
This study represents a significant breakthrough in understanding cardiac amyloidosis and offers hope for more effective treatment options.
The UCL Centre for Amyloidosis, one of the world’s leading centers for amyloid research, continues to delve deeper into this disease, striving to bring innovative treatments to patients sooner.
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The study was published in the New England Journal of Medicine.
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