FOP research has made exciting strides lately. Several drugs under development show promise in slowing or preventing heterotopic bone formation (HO) and improving quality of life. Here’s what’s new:
🔬 Key Investigational Drugs
- Saracatinib (STOPFOP trial)
This oral small-molecule inhibitor works on ALK2 (also called ACVR1), the protein that misfires in FOP. Preclinical studies in mice showed that saracatinib can block excessive bone formation and preserve mobility. JCI Insight+2ifopa.org+2
The STOPFOP Phase 2 clinical trial is testing safety and efficacy in humans with the classic FOP mutation. Participants will undergo a randomized, placebo-controlled portion for 6 months, followed by extended treatment. ifopa.org+2ifopa.org+2 - INCB000928
This compound is in early trials targeting ALK2 as well. It aims to reduce the abnormal signalling caused by the mutant ACVR1 gene. The goal is to slow or prevent bone growth in soft tissues. Synapse+1 - DS-6016a, BLU-782, Fidrisertib, Zilurgisertib, Andecaliximab
These are additional ALK2 inhibitors or immune-modulatory agents under study. Some are small molecules, others are monoclonal antibodies. Their trials are at various stages (Phase 1 or 2). ifopa.org+2foptrust.org+2 - Palovarotene (Sohonos)
This RARγ agonist is an already approved treatment in some countries. It suppresses bone formation and has shown effectiveness in animal models and early human trials. But it has limitations and safety issues under review. Wikipedia+1
⚠️ What These Mean for Patients
- None of these drugs (except palovarotene) are widely available yet for routine use.
- Clinical trials are crucial; being informed allows patients to consider participation, where feasible.
- Safety, dosing, and long-term effects are being carefully studied.
🔍 Looking Ahead
Research continues at a fast pace. With each trial, we gain better understanding of how FOP develops and how best to intervene. Drugs like saracatinib and INCB000928 represent hope not just for treating flare-ups, but possibly slowing disease progression over time.
Stay in touch with FOP Trust India and IFOPA for updates—you might hear about new trials, experimental therapies, or compassionate use programs.
To learn more, visit: https://foptrust.org/
For international resources, visit: https://www.ifopa.org/
