Do No Harm!
Research is a strange thing. Some people think research is too slow and too complicated. Others are sceptical and wouldn’t like to be the guinea pig. From the point of a clinician the most important goal in doing research is:
Do no harm!
Issue Nr. 4-2021 of the OIFE Magazine is dedicated to research and future perspectives, even if this will take a lot of time and money. And it is true that research is sometimes slow. Many of the currently investigated drugs and therapeutic options will only be “Standard of care” in many years from now. Precise investigation and evaluation of side effects are needed to offer a treatment, which is accepted by people with OI and follow the guidance: Do no harm!
Past and present
But changes have happened in the past, and they did improve the care for people with OI. Many adults never received bisphosphonates during childhood, because this treatment, which is now standard of care, was not available at that time. At the beginning of the “bisphosphonate-era” the drug was used more restrictive than today, and a lot of additional investigations (blood draws, biopsies etc) have been performed to make sure that researchers and clinicians do not harm with their treatments. Another huge improvement of care that most of the adult readers are too old to benefit from, was the development of telescopic rods. Today we know that they reduce the total number of surgeries. They also improve mobility and self-care of people with OI. Nonetheless, at the beginning there was extensive discussions if the long surgeries and the risk of dislocation would be acceptable to justify the use of telescopic rods. Other inventions like bone marrow transplantations, have not turned out to be beneficial. After treatment of a few patients, the trials with this kind of therapy have been stopped.
Role of authorities
The development of new drugs was simplified by the authorities (EMA and FDA) a few years ago by implementing the so-called “Orphan disease status”. This encouraged some companies to develop drugs specific for OI or at least to investigate the effect of drugs already approved in other indications in proper clinical trials for the use in OI. Even with this orphan disease status, the process of developing new treatments and therapies is still slow. A clinical trial takes years. You need to find the required number of volunteers to participate, but this is completely necessary. In the end we aim for a drug which is hopefully effective, but without severe side effects. The importance of close monitoring of drugs which are under development is indispensable because some side effects might only occur after some time of treatment.
New drugs and therapies
Currently more ideas are being researched than ever before, to improve the situation of people with OI. New drugs as antisclerostin and new antiresorptives are being investigated. Additionally, completely new therapeutic pathways are considered, such as stem cell treatment or gene therapies. These are promising approaches, at least in basic research and in animal models. Time is needed to assess the safety and efficacy of these new agents to make sure that we do not harm patients.
The importance of networking
During the last years an additional change in therapeutic strategies has happened, which is definitely safe, will not harm anybody and will speed up improvements in the care of people with OI. The cooperation between researchers has changed and improved dramatically. 20-30 years ago, every OI-researcher was working on his own, competing with other researchers, hiding the results until they were published. Today many (maybe too many) networks have been established to connect clinicians, clinical and basic researchers. Some are also including patients themselves, people from health care providers, authorities and ethical committees. This is an extremely promising approach, especially because patient organizations can play an active role in these networks. OI-organizations are not only commenting on initiatives presented by researchers anymore, but have the opportunity to guide these networks and to prioritize areas of special interest.
To conclude – we live in very exciting times, in which more research on OI is done than ever before. Nonetheless many people think that research is still too slow. Different approaches are currently being investigated. And research must be accurate and reliable, so that patients can trust their clinicians, when new treatments become available. This issue will give you a glimpse of what is going on in the world of OI-research. I hope you will enjoy reading.
Chair of the Medical Advisory Board of OIFE (OIFE MAB)