The International Clinical Council on FOP

Optimizing care for the FOP patient

ICC_Final_Final_Logo2_27_18.jpgThe International Clinical Council on FOP (The ICC) is an autonomous and independent group of 19 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 Council was officially established and its Constitution unanimously ratified on June 21, 2017.

The ICC will independently establish its rules, committees, and criteria for membership and will meet at least twice annually, either in-person and/or by teleconference. The ICC looks forward to a very proactive agenda. Formal announcements, updates and activities will be presented at relevant meetings and on relevant websites.

The detailed background and rationale for the ICC is described in the Preamble of the Constitution of the ICC:

“During the past 25 years, the Fibrodysplasia Ossificans Progressiva (FOP) community has moved from the wastelands of a rare disease to the watershed of clinical trials. Together, we identified the genetic cause of FOP and used that knowledge to spearhead worldwide research efforts to develop therapies that will transform the care of individuals with FOP. We have expanded the frontiers of discovery and drug development, dismantled the physical and perceptual barriers that have impeded progress, and inspired global research in small molecules, antibodies, and gene therapy for FOP. We have formulated best practices and assembled teams of experts to optimize ambulatory and in-patient care of the FOP patient."

Research scientists at university laboratories, pharmaceutical companies, biotechnology firms, and government agencies are racing to create effective treatments and a cure of FOP. Presently, at least 30 independent laboratories, pharmaceutical or biotechnology companies are working on the development of kinase inhibitors, target cell inhibitors, ligand traps, antibodies, small inhibitory RNA technology, gene editing – all propelled by the historic discovery of the FOP gene and by the identification of its robust therapeutic target – the mutant ACVR1 kinase, its upstream activators, downstream targets and side-stream modulators.

The recent seismic activity in FOP basic and clinical research presents exciting challenges for clinicians caring for FOP patients worldwide. Importantly, ongoing clinical care and emerging clinical trials present medical and logistical challenges for individuals with FOP. Additionally, the pharmaceutical - biotechnology complex continually seeks expert advice from our ranks on clinical trial development – all of which hinges on critical clinical studies on the natural history and biomarkers of disease activity.

There is clearly an urgent unmet need to consolidate and coordinate clinical knowledge and advice on clinical care, symptomatic treatment, and clinical trial development into a framework that best serves the needs of FOP patient community worldwide. In the past, this function has been performed informally by the various members of the International Clinical Consortium – the authors of the popular FOP Treatment Guidelines. It is time now to formalize this emergent opportunity as leaders in the care of FOP patients and in the robust clinical activities of the FOP community internationally.” 

Mission of The ICC:

  1. To educate on best practices for the care of individuals with FOP.
  2. To advise on the design and conduct of interventional trials in FOP patients.
  3. To publish from time-to-time the FOP Clinical Guidelines.
  4. To advocate for a robust infrastructure for data sharing and collaboration on vital and emerging matters of clinical concern to the FOP community.
  5. To identify less explored areas of FOP patient care and issues that may drive insight into research.
  6. To share valuable clinical experiences from the care of patients with classic and variant FOP.
  7. To better understand the variable phenotype of FOP and the systemic nature of FOP pathology.

The ICC has five standing committees that shall meet regularly in person and by teleconference:

Governance & Membership Committee (Chairperson: Zvi Grunwald, MD)
Function: To establish the ICC governing rules, membership terms, auditing processes, bylaws.

Ethics Committee (Chairperson: Rolf Morhart, MD)
Function: To guard the health and safety of FOP patients by supporting transparency and compliance with Good Clinical Practices.

Communications & Relations Committee (Chairperson: Mona Al Mukaddam, MD)
Function: To provide the external communications to the public.

Publications Committee (Chairperson: Frederick S. Kaplan, MD)
Function: To revise and publish the clinical guidelines and provide the resource for all materials published on behalf of the ICC.

Clinical Trials Committee (Chairperson: Edward Hsiao, MD, PhD)
Function: To provide guidelines for clinical trials in support of safe and transformative treatments for FOP.

For more information, please contact the following committee chairs:

Chair of the Council, Frederick S. Kaplan, MD
Governance & Membership Committee, Zvi Grunwald, MD
Ethics Committee, Rolf Morhart, MD
Communications & Relations Committee, Mona Al Mukaddam, MD
Publications Committee, Frederick S. Kaplan, MD
Clinical Trials Committee, Edward Hsiao, MD, PhD

Founding Members

Mona Al Mukaddam, MD
Philadelphia, PA, USA

Genevieve Baujat, MD
Paris, France

Matthew Brown, MBBS, MD, FRACP, FAHMS, FAA
Brisbane, Australia 

Tae-Joon Cho, MD
Seoul, Republic of Korea

Carmen L. De Cunto, MD
Buenos Aires, Argentina

Patricia L. R. Delai, MD
São Paulo, Brazil

Maja Di Rocco, MD
Genoa, Italy

Marelise W. Eekhoff, MD, PhD
Amsterdam, The Netherlands

Zvi Grunwald, MD
Philadelphia, PA, USA

Nobuhiko Haga, MD
Tokyo, Japan

Edward Hsiao, MD, PhD
San Francisco, CA, USA

Frederick S. Kaplan, MD
Philadelphia, PA, USA

Richard Keen, MD, PhD
London, United Kingdom

Rolf Morhart, MD
Garmisch-Partenkirchen, Germany

Coen Netelenbos, MD, PhD
Amsterdam, The Netherlands

Robert J. Pignolo, MD, PhD
Rochester, MN, USA

Christiaan Scott, MBChB
Cape Town, South Africa

Michael Zasloff, MD, PhD
Washington, DC, USA

Keqin Zhang, MD, PhD
Shanghai, China

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