RESEARCH

BOOSTB4 – study on stem cell therapy

Interview with Cecilia Götherström and Eva Åström

Who are you? What is your profession and what is your relationship to OI?

Cecilia Götherström:
I’m a researcher who have explored the potential use of stem cells for treatment of OI and other diseases for almost 20 years. My “relationship” with OI started in 2002, and since then it has been the closest to my heart.

Eva Åström:
I’m a pediatrician and pediatric neurologist and leader of the Swedish multidisciplinary pediatric OI-team and the main principal investigator of the clinical trial BOOSTB4.
I am also a member of the OIFE’s medical advisory board.

Can you tell us about your research project?

We have previously treated a few unborn babies and children with OI with stem cells, which has shown promising results. The Boost Brittle Bones Before Birth (BOOSTB4) project is a European network centred around four clinical hubs in Stockholm, London, Cologne and Leiden/Utrecht. We will investigate if stem cell transplantation before and/or soon after birth is a safe, feasible and effective treatment of OI. We hope that an early treatment of OI will result in a better effect and that maybe less treatment is needed later in life. If we see a good effect of the stem cells, we will explore their use in older people with OI. The project will also evaluate the acceptability of this type of stem cell therapy, both from the patient and family perspective as well as from health professional in the area, and from an ethical perspective. Finally, we will develop a method for non-invasive diagnosis of OI. This means that for diagnosis of OI during pregnancy one could take a blood sample from the pregnant woman instead of taking a sample from the fetus.

If you are interested in participating in the BOOSTB4 trial, you are welcome to contact us on BOOSTB4@clintec.ki.se
Children under the age of one year diagnosed with OI type III or severe type IV with a collagen type 1 mutation may participate in the trial.

How is the project financed?

The BOOSTB4 project is financed by the European Union’s Horizon 2020 research and innovation programme (grant agreement 681045) and by the Swedish Research Council. We have also received funding from Care 4 Brittle Bones for studies of the previously treated children with OI.

Do you have patient involvement in your project?

When we in 2015 wrote the research application to the European Union, we wanted to incorporate the views of patient organisations on the project. OIFE gave input on the project and kindly wrote a support letter that was included in the application. Since then the project has investigated stakeholders’ views to understand the believed benefits or concerns, identify ethical issues and establish protocols for support and counselling. This study involved adults affected with OI, with and without children affected with OI and parents of children affected and patient advocates from relevant patient support groups. We have also asked for input on the trial participant information and other trial documents from patient organisations.

Do you have a message for the readers of OIFE Magazine & our website?

Cecilia:
I have a motto that comes from Pippi Longstocking:
“I have never tried that before, so I think I should definitely be able to do that!”

Eva:
Always try to improve treatment options without causing harm, and remember that every individual is unique.