The study showed for the first time that the treatment was safe and showed an approximately 50% reduction in the amount of amyloid in the heart 12 months after treatment.
The results of this study were published in New England Journal of Medicine.
Heart failure due to ATTR amyloidosis is a serious disease with a poor prognosis. Patients have symptoms of shortness of breath and reduced exercise capacity due to deposition of protein aggregates (amyloid) in the heart. This causes a thickened and stiff heart muscle. Current treatment consists of stabilizing the disease with drugs. Until now, there was no way to clear away the amyloid already present.
"Capturing amyloid in the heart is a logical step to improve heart function," according to Peter van der Meer, cardiologist at UMCG and one of the co-authors of the paper published in the New England Journal of Medicine. "The results of this phase 1 study are promising, but more research is needed to determine the effectiveness of the drug on symptoms and prognosis."
Hans Nienhuis, internist at UMCG and coordinator of the Groningen Amyloidosis Centre of Expertise: "A treatment to clean up amyloid would be an important addition to current treatments that allow us to reduce the production of new amyloid and, in the most favourable case, stop disease progression. Patients often have a high burden of disease; this treatment has the potential to reduce that. This study shows that by administering an antibody directed against amyloid, it is possible to induce the immune system to clear the amyloid."
According to Van der Meer and Nienhuis, there will be follow-up research, which will focus on improving symptoms and prognosis. The UMCG will also play a role in this research.
