Posted on behalf of the Clinical Trials Committee of the International Clinical Council on FOP (ICC)

The ICC is aware of the recent publication by the Palhares group regarding FOPCon and a new YouTube video (available in some countries, but appears to not be available in the USA or Europe) supporting FOPCon. FOPCon is a proposed therapy composed of ascorbic acid (vitamin C) and propranolol.

At this time and with the currently published evidence, the ICC feels that the evidence presented for public review does not support the use of FOPCon as a therapy for FOP.

Specifically, the ICC has requested the following:

• FOPCon should not be advertised as a definitive therapy for FOP. The FOPCon paper and video do not provide proof of efficacy or safety because the evidence presented and available for review is insufficient to make this judgment. Although FOPCon may eventually provide insights into therapeutic options, we strongly recommend caution for patients with FOP and physicians who treat FOP against drawing therapeutic conclusions.
• FOPCon should be described as an investigative therapy, until its efficacy and safety have been reviewed by a national regulatory agency, and accepted by the international community.
• The risks of FOPCon need to be fully presented. These include, but are not limited to, concerns about increased risks of cardiac arrhythmias, slow heartbeat, low blood pressure, dizziness, passing out (syncope), increased risks of kidney stones, and death.
• Subjects receiving FOPCon are portrayed to be in a clinical trial; thus, all subjects should be treated according to internationally-recognized standards. This includes formal written and voluntary consents, a full discussion of potential risks and benefits, standardized clinical and safety monitoring, and standardized endpoints to understand the potential efficacy of an investigational therapeutic intervention. The ICC also confirms that all investigation studies should follow key clinical trial recommendations previously agreed on by FOP clinicians worldwide. This will help ensure the safety and ethical treatment of subjects with FOP and scientific validity of the results. This is important so that the community can understand how well FOPCon works compared to other potential therapies.

The ICC confirms that the treatment recommendations recently reviewed in detail and published in the 2019 FOP Treatment Guidelines are unchanged at this point. We and others await the review of additional published data about how the proposed therapy FOPCon may benefit the FOP community. We also encourage the FOP community to critically assess the risks, benefits, and strength of the supporting data for any other proposed therapy for FOP. Patients with FOP who are considering any medication changes should discuss their options, and potential risks and benefits, with their doctors and medical team before starting any potential treatment.

The International Clinical Council on FOP (ICC) is an autonomous and independent group of 21 internationally-recognized physicians who are clinical experts in FOP from 13 nations (Argentina, Australia, Brazil, China, France, Germany, Italy, Japan, Netherlands, South Africa, South Korea, United Kingdom and United States) and six continents (Africa, Asia, Australia, Europe, North America and South America). The ICC was established to coordinate and consolidate a global voice for the best practices for clinical care and clinical research for people who suffer from FOP. The ICC publishes the FOP Treatment Guidelines, which is used internationally to guide the management of patients with FOP. The ICC also has published key recommendations for clinical trials testing potential therapies for FOP.