2015 ACR/ARHP Annual Meeting: Treatments for Transthyretin-Related Amyloidosis Generate Interest from Researchers, Pharmaceuticals

But the effects in other organs are not yet known.

“It’s a real question as to whether a heart involved with amyloid can tolerate immunologic chaos that might occur when you’re directing antibody attack on a major organ,” Dr. Berk said.

Overall, there is good cause for optimism in amyloidosis treatment, he said: “We haven’t identified all the therapeutic answers yet, but the future truly is bright.”

Thomas R. Collins is a medical writer based in Florida.

References

1. Berk JL, Suhr OB, Obici L, et al. Repurposing diflunisal for familial amyloid polyneuropathy: A randomized clinical trial. JAMA. 2013 Dec 25;310(24):2658–2667.
2. Suhr OB, Coelho T, Buades J, et al. Phase 2 Open-Label Extension Study of Patisiran An Investigational RNAi Therapeutic for the Treatment of Familial Amyloidotic Polyneuropathy. Presented Nov. 3, 2015 at the First European Congress on Hereditary ATTR Amyloidosis. Paris, France.
3. Richards DB, Cookson LM, Berges AC, et al. Therapeutic clearance of amyloid by antibodies to serum amyloid P component. N Engl J Med. 2015 Sep 17;373(12):1106–1114.