Fibrodysplasia Ossificans Progressiva

Roger Gale Excerpts

Monday 6th December 2021 Westminster Hall

[Sir Roger Gale (in the Chair)]

Before we begin, I remind Members that they are expected to wear face coverings when not speaking in the debate. This is in line with current Government guidance and that of the House of Commons Commission. Members are asked by the House to take a covid lateral flow test twice a week if coming on to the estate, which can be done either at the testing centre, formerly the Members centre in Portcullis House, or at home. Please give one another and members of staff space when seated, and when entering and leaving the room.

[Elliot Colburn (Carshalton and Wallington) (Con)]

I beg to move,

That this House has considered e-petition 590405, relating to research into Fibrodysplasia Ossificans Progressiva.

It is a pleasure to serve under your chairmanship, Sir Roger. The petition closed with 111,186 signatures, including 162 from my constituency. First, I thank the petition creators, the Bedford-Gay family, FOP Friends, Dr Alex Bullock and Dr Richard Keen, for meeting with my office to share their stories and experiences of, and expertise on, fibrodysplasia ossificans progressiva. I am incredibly grateful for their help preparing not only me but other right hon. and hon. Members for this debate. Many colleagues are keen to speak, not least my right hon. Friend the Member for Hemel Hempstead (Sir Mike Penning), who has been a champion for his constituents on this issue. I look forward to hearing his contribution. I will keep my comments brief to give others the opportunity to speak.

FOP is a very rare, genetic, degenerative condition that causes the body’s bone to develop in areas where normally it would not, progressively locking joints in place and making movement more difficult and, eventually, impossible. Those with the condition will eventually become 100% immobile, almost like a human statue, with a healthy mind locked inside a frozen body. It is one of the most debilitating and disabling conditions known to affect children in their early years, with no treatment, cure or prevention.

Once it progresses there is no way to reverse it, because trauma causes more activity. Something as small as a knock, a bump or a fall can trigger more bone growth. Likewise, the trauma of misdiagnosis and related medical treatments such as biopsies and injections can trigger bone growth. Even unrelated illnesses such as flu can trigger bone growth, so I can only imagine the stress and horror caused by the last two years of the covid-19 pandemic for families with children suffering from FOP. FOP does its worst damage in a child’s early years. While the condition will progress over time at different rates and no two individuals will have the same journey, most people with FOP are immobile by the age of 30.

The statistics and details of FOP are powerful, but not as powerful as the stories of those experiencing this condition. I am very grateful to the petition creators, Helen and Chris Bedford-Gay, for sharing Oliver’s story. When their son Oliver was three months old, he had what some medical professionals considered to be funny toes and a lump that began to appear on the back of his head. Oliver’s consultant concluded that the lump was not cancerous but should be removed none the less. Shortly after Oliver’s first birthday, the consultant diagnosed him with FOP. The family were led to believe that he would be fine as long as he avoided contact sports such as rugby. It was only later, when Oliver’s parents searched for more information, that they discovered the true implications of a diagnosis of FOP.

FOP results from a single gene mutation, which was discovered only in 2006, so there is very little information on or experience of this condition easily available to the public or medical professionals. With such a large barrier to access to relevant knowledge and guidance, the Bedford-Gay family were seemingly alone, with nowhere to turn for help and support. At that point there was just a small patient group but no dedicated UK charity to support families with FOP and fund research. That prompted the Bedford-Gay family to establish Friends of Oliver, now known as FOP Friends. In short, FOP Friends aims to further research into FOP and related conditions by supporting current and future research projects, to support families suffering from the condition and to raise awareness. Since the charity began, FOP Friends has raised more than £700,000 to help that work and has been able to work alongside the Royal National Orthopaedic Hospital, the FOP research team at the University of Oxford and other international FOP patient organisations in this fight. Since Oliver’s diagnosis, there have been leaps forward in research, awareness and treatment, thanks to those organisations. However, there remains so much more to be done, and it cannot be done alone. FOP Friends has three key asks of Government.

The first is to increase research funding into FOP. My right hon. Friend the Member for Hemel Hempstead will no doubt delve deeper into that topic, so I will not steal his thunder. However, I will say that the University of Oxford FOP research team, led by Dr Alex Bullock, has been investigating how the mutation that causes FOP is activated in patients and what might be able to prevent it from progressing, but that research receives no Government funding. The team’s research into a new drug that could treat FOP has been put on ice due to the covid-19 pandemic, and it is unlikely that external funding will be sourced to conclude this clinical trial.

As a rare condition that only impacts one in a million people, many consider there to be no commercial incentive to fund commercial research. However, because of the effects of FOP, research into it could help solve problems in unwanted bone growth, and conversely, how to encourage it in other major disease areas, including military injuries or surgeries, severe burns, osteoporosis or heart disease. FOP is just the tip of the iceberg of the research. Unfortunately, there is no mechanism for the Oxford team to obtain emergency funding for a clinical trial that is already under way. While the Government have pledged more than £6.6 million of funding via the National Institute for Health Research and UK Research and Innovation for more general bone disease research, there is some confusion about how this has or will be applied to FOP research. As I understand it, that funding has not been seen by the Oxford research team. I would be grateful if the Minister could shed some light on this issue and the potential mechanism for the team to access emergency research funding.

Secondly, the petitioners call for the Government to transform the standard of care that patients receive. The Government’s rare diseases policy, the UK Rare Diseases Framework, offers a vital opportunity to transform and improve standards of care for patients and families across the country. With only a handful of NHS clinicians with FOP experience, FOP patients receive varying levels of medical care and home support. I am aware that FOP Friends does amazing work assisting families in school settings with education, health and care plans. Carers of FOP patients are often parents or siblings as the specific needs of FOP patients can be tricky for others to understand or manage. Too often, the ability of those who suffer from FOP and their families to work, live and contribute to society is limited by the condition without wider institutional support. I would be grateful if the Minister could confirm and outline further how the UK Rare Diseases Framework could better support FOP patients and their families.

Thirdly, the petitioners call for the Government to help increase awareness of FOP and to transform diagnosis. As I mentioned, as it is a fairly newly discovered condition, there is a serious lack of knowledge and experience of FOP. Misdiagnosis and mistreatment, such as through biopsies and vaccinations and so on, can cause the condition to worsen and trigger irreversible bone growth. Early diagnosis is crucial not only to treat the condition but to prevent avoidable early progressions, which is why it is so important to raise awareness of FOP among medical practitioners. I understand that there have been calls to make the teaching of FOP mandatory in medical schools, so I would appreciate the Minister’s saying a few words on that.

A genetic test exists to confirm a diagnosis of FOP, but currently only specialist clinicians can request a test. An application has been made to include FOP as part of the roll-out of the NHS genomic medicine service, which is funded by NHS England, to allow a wide range of clinicians to request a test if they suspect FOP. I understand the directory of approved tests will be updated in April 2022, and I hope the Minister will enlighten us as to whether FOP will be included in that because that will increase access to genetic testing and reduce the time to diagnosis.

I want to once again pay tribute to Oliver and his family, as well as the many organisations, researchers, campaigners and other families who have worked tirelessly to fight FOP, many of whom I am sure we will hear about this afternoon. I appreciate that many other colleagues want to get in, especially my right hon. Friend the Member for Hemel Hempstead, who has a great degree of knowledge in this area, so I will bring my remarks to a close. I hope that we can have a productive debate on this issue and the key asks outlined by the petitioners.