The time is now

For a number of years treatment of OI was mostly limited to bisphosphonates, surgical interventions and physiotherapy. Well basically it still is, if you think of approved and standardized treatment (if such a thing exists in OI). The group of drugs called bisphosphonates actually celebrates its 50th anniversary this year. And for the last 25-30 years or so, it has been a very important factor in OI. The same category of drugs with different names and labels have given children growing up now, a completely different start compared to people my own age. We do however get on with our lives, even if we didn’t receive the gold standard treatment of today, when we were young.

The problem with bisphosphonates is that they only affect the quantity of bone and not really the quality. By slowing down the osteoclasts, the body builds more bone, but of the same questionable quality as we had before. It helps, but it’s far from a miracle cure.

But times are changing and new options are coming out on the horizon. I think it’s fair to say that there has never been so much research going on in OI and other rare bone diseases as today. Stemcells are being investigated as a treatment in utero or for newborns in the project Boost Brittle Bones Before Birth (BOOSTB4). The company Mereo Biopharma has finished the first phases of the Asteroid trial, which is investigating the human antibody BPS804 (antisclerostin). From what I’ve heard, the results so far are positive. Another human antibody called romosozumab was very recently approved by the FDA (US Food & drug administration) for treatment of severe osteoporosis, something which might be good news for BPS804 as well. As opposed to the drug romosozumab, the BPS804 (antisclerostin) is a treatment being developed specifically for OI. It has approval as a socalled orphan drug, which means that the drug is being developed specifically for a rare disease.

This week we also got news that a research project lead by dr. Nick Bishop from the UK had received funding for an international study which will investigate the drug Losartan in OI – a drug which is primarily used as a treatment for high blood pressure. Don’t ask me how that is going to work! But the experts have faith that it might have a positive effect on people with OI.

An increasing challenge for the researchers however is to find enough people who are willing to take part in the different studies that are recruiting in different parts of the world. All of us want new treatments options to be developed, but not that many want to try out medicines that has not been approved yet or even researched for a long time. The problem is – if nobody wants to go first and be the pioneers, we will never have the necessary knowledge. Certainly a dilemma that needs to be addressed…

Another increasing challenge for OIFE is to find and provide enough people who can take on the role as patient representatives and consultants in the different research projects. My impression is that the demand for patient representatives have increased dramatically during the four years I have been functioning as the OIFE president. Paying attention to the voices of people who have the diagnosis themselves, have become an increasingly important criteria when projects apply for funding. And the demand for OI-people with bright ideas and enough time to invest in various projects is huge.

OIFE and our member organizations really need to put our minds together and discuss how we can solve this challenge, which is basically a luxury problem. Why so? Because I truly believe that the outcomes of research will be substantially better and more efficient if the people who will benefit from it are directly involved from A to Z. How can we as organizations find and recruit enough people with an interest in research? How can we train and educate people to become patient experts when needed? Sometimes it’s enough to ask input from a person without that much knowledge as long as the person/parent has personal experience of living with OI. But sometimes the role as a patient representative is so challenging that you need both knowledge and organizational experience in addition to personal experience with OI. I’m thinking about patient experts who can represent the OI-community in steering committees, in meetings in the European Medicines Agency (EMA), in health technology assessments nationally and internationally and people who can contribute as ePAGs in the European Reference Network for Rare Bone Diseases.

Fortunately the OI-community has solved great challenges together before. The OI-organizations with its many engaged volunteers have a tradition for coming together and helping both each other and the professionals, when difficult challenges needs to be solved.

The next time we’ll come together and discuss this will be at the OIFE Annual General Meeting. It will take place after the topical meeting See, Hear, Smile! in Riga, Latvia. See, Hear, Smile! is another example of how good collaboration between engaged professionals and OIFE-volunteers can result in an innovative programme focusing on new and important topics – the eyes, ears and teeth of people with OI. Craniofacial issues and basilar invagination will also be covered at the seminar and we have managed to gather prominent lecturers from all over Europe and from Canada and the US.

Later in 2019 we’ll meet again in Amsterdam. The conference QualityofLife4OI will gather researchers, clinicians and people with OI to discuss how quality of life of people with OI can be measured more systematically in order to make research and development as efficient and good as possible. The conference is initiated and organized by the foundation Care4BrittleBones and is a collaboration between the organizations OIFE, OIF and the European Reference Network for Rare Bone Diseases (ERN-BOND).

Between Riga and Amsterdam some of us will meet in the International Conference for Rare Bone Diseases (ICCBH) in Salzburg, where different stakeholders are trying to establish a network between organizations, companies and other stakeholders interested in rare bone disebisphoases, including OI.

So much going on! Sometimes when my email explodes and my mind is spinning, it feels like too much. But the opposite would be worse, wouldn’t it? What if nobody was interested in OI and no development was happening at all? This is certainly not the case at the moment. The time is now. And we have to use it wisely…The time is now

Ingunn W. – OIFE President

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