Advertisement

If you have an ACS member number, please enter it here so we can link this account to your membership. (optional)

ACS values your privacy. By submitting your information, you are gaining access to C&EN and subscribing to our weekly newsletter. We use the information you provide to make your reading experience better, and we will never sell your data to third party members.

ENJOY UNLIMITED ACCES TO C&EN

Biological Chemistry

Antibody Treats Rare Bone Disorder In Mice

Drug Discovery: Researchers develop antibody to treat rare genetic disease in which soft tissue turns to bone

by Judith Lavelle
September 7, 2015 | A version of this story appeared in Volume 93, Issue 35

[+]Enlarge
Credit: LiQin Xie and Nanditha Das/Regeneron
This CT image shows abnormal bone formation (yellow) in a skeleton of a mouse with fibrodysplasia ossificans progressiva.
Abnormal bone formation in a skeleton of a mouse with fibrodysplasia ossificans progressiva.
Credit: LiQin Xie and Nanditha Das/Regeneron
This CT image shows abnormal bone formation (yellow) in a skeleton of a mouse with fibrodysplasia ossificans progressiva.

No treatments currently exist for fibrodysplasia ossificans progressiva (FOP)—a rare disease in which a person’s soft tissues ossify, or turn to bone. The genetic mutation that most commonly causes FOP swaps an arginine for a histidine in a bone-producing protein receptor called ACVR1. This leads to receptor hyperactivity and, in turn, abnormal bone growth. Now, a team from Regeneron Pharmaceuticals, in Tarrytown, N.Y., and Brigham & Women’s Hospital, in Boston, has created a mouse model to study FOP. The team tested whether activin A—a protein the body produces when injured—promoted bone growth in FOP mice (Sci. Transl. Med. 2015, DOI: 10.1126/scitranslmed.aac4358). Researchers soaked collagen sponges either in activin A or the bone-producing protein BMP2 and implanted them in FOP and control mice. The BMP2 sponges caused ossification in all mice, but the activin A sponges did so only in FOP mice—indicating that activin A triggers the severe ossification seen in patients with FOP. The team created human monoclonal antibodies that target activin A. Untreated FOP mice developed ossifications in three weeks, but mice treated with the antibody did not show significant ossification six weeks into testing.

Article:

This article has been sent to the following recipient:

0 /1 FREE ARTICLES LEFT THIS MONTH Remaining
Chemistry matters. Join us to get the news you need.