Finding a Cure
In a race against time, the search for a cure is now international.
The search for a treatment for FOP is continuing at a number of academic institutions around the world, with the main sites being in the US, United Kingdom, Argentina, Japan, Italy, France, and the Netherlands.
In order to further the knowledge and understanding of FOP, the Drug Development Forum was set up. The first event was held in Boston, MA, USA in 2014 and hosted by the IFOPA. The event was created to bring together the best minds in FOP researchers, from universities, academic institutions, and bio-pharmaceutical companies around the world, to share knowledge and discuss the important issues and questions surrounding the challenges they face in trying to find a treatment and a cure for FOP.
The aims of the DDF are to:
- Advance research collaboration through the sharing of relevant and timely FOP data
- Solve key issues facing FOP drug development
- Strengthen the FOP research network and stimulate new ideas to help advance therapeutic drug development
Chris represented FOP Friends when he attended the 4th Drug Development Forum in November 2019 which was held in Florida, USA. There were representatives and families there from 19 different countries.
Chris sat on a panel with other patients and families affected by FOP to answer questions about how FOP impact their lives, and their family life.
Here, some of the attendees reflect on the importance of such events for the FOP community: DDF 2019, Florida, USA
There are currently eight clinical trials underway in the search for a treatment for FOP (September 2020). Some are still in the very early stages of phase 1/healthy volunteers, while others are moving slowly towards applying for approval. The IFOPA has a comprehensive page about Clinical Trials for FOP.
FOP Friends does not endorse or promote any trial or drug treatment path. If you are considering particpating in a clincial trial, please contact your medical practitioner for further guidance.
The National Institute for Health Research has more information on trials available in the UK: Be Part of Research
For general guidance and information to support your decision on whether to take part in a clincial trial, please visit the NHS website: https://www.nhs.uk/conditions/clinical-trials/
Image credit: IFOPA 2020
The University of Oxford has been a major centre for FOP research since the mid-1970s when Professor Jim Triffitt and Dr Roger Smith began their collaborative studies that culminated in the co-discovery of the causative gene for FOP, ACVR1, in 2006. Now with Emeritus status, Professor Triffitt is collaborating with Dr Alex Bullock to develop ACVR1 inhibitors as an effective treatment for FOP.
The University of Oxford FOP Research Team is investigating precisely how ACVR1 is activated in FOP patients to induce unwanted bone formation and how drug-like molecules might stop this devastating process. With the help of the Structural Genomics Consortium, the team has elucidated the 3D structures of the ACVR1 protein bound to a variety of different drug-like molecules. This information is being used to design safer and more specific molecules that are strictly necessary before patient clinical trials can be considered.
The research efforts for FOP are entirely supported at the University of Oxford by monies raised by FOP Friends, as well as other patients and their families and friends. The research team owes a great debt to these individuals for their extensive efforts to support this work. They are greatly encouraged by their contact with patients and families and are striving to accelerate knowledge on FOP to achieve the therapeutic goal.
Much FOP research takes place at the University of Pennsylvania School of Medicine, where a research laboratory devoted to FOP exists. The FOP Research Laboratory, created in 1992, is led by Frederick Kaplan, M.D., and Eileen Shore, Ph.D., with the dedicated research team including three principal investigators with many post-doctoral fellows, scientists, students and staff. This core team collaborates with physicians and scientists worldwide to develop treatments and – someday – a cure for FOP.
The FOP Research Lab’s seminal work has been highlighted in many prestigious medical publications, including the New England Journal of Medicine, Nature Genetics, The Journal of Bone and Joint Surgery, Clinical Orthopaedics, and The Journal of Bone and Mineral Research.
Click here to read the most recent annual reports from the FOP Research Laboratory, written by Professor Fred Kaplan, Professor Eileen Shore, and Dr Mona Al Mukaddam.