A rare antibody may be able to completely cure transthyretin amyloid cardiomyopathy, a hitherto incurable heart disease.
- A rare antibody may be able to completely cure a hitherto incurable heart disease, amyloid-transthyretin cardiomyopathy (ATTR-CM).
- To reach this conclusion, the researchers succeeded in identifying a specific antibody in three men who had spontaneously recovered from transthyretin amyloid cardiomyopathy.
- ATTR-CM is a fatal disease that causes damage to the heart and surrounding nerves.
According to a new studya rare antibody may be able to completely cure transthyretin amyloid cardiomyopathy, a hitherto incurable heart disease.
Three men recover spontaneously from transthyretin amyloid cardiomyopathy
Researchers have indeed identified a rare antibody that could potentially “reverse” the effects of this pathology. To reach this discovery, the scientists studied the cases of three men suffering from the disease who had recovered from it spontaneously. The researchers then isolated the antibody that would have potentially enabled their cure and determined that it was able to target and neutralize the transthyretin protein present in the heart.
The researchers then tested the antibody in mice with ATTR-CM and found that it was able to reverse the damage caused by the disease. They also administered the antibody to three patients with ATTR-CM in a preliminary clinical trial and observed a significant improvement in their health and a reduction in amyloid deposits in their hearts.
Recreating these antibodies in the lab
Despite the promising results of this study, further research will be needed to confirm the benefits of this antibody in patients with ATTR-CM. Researchers must also work to develop ways to produce this antibody in large quantities so that it is available to more people.
Study director Professor Julian Gillmore, head of the UCL Center for Amyloidosis based at the Royal Free Hospital, said: “It is not possible to conclusively prove that these antibodies cause patients to recover. However, our data indicate that it is very likely and possible to recreate these antibodies in the laboratory and to use them as therapy. We are currently continuing our research in this direction, although it is still at a preliminary stage”.
What is transthyretin amyloid cardiomyopathy?
Amyloid transthyretin cardiomyopathy (ATTR-CM) is a rare and fatal heart disease characterized by an abnormal buildup of protein in the heart. The protein involved, called transthyretin, interferes with the structure and function of heart muscle, leading to progressive heart failure.
Current treatments for ATTR-CM focus on managing the symptoms of heart failure, but there are no cures to reverse the damage caused by the disease. As a result, almost half of people with this condition die within four years of diagnosis.
