Good examples

HOPE – unites healthcare and research to

benefit children with cancer

Anna Nilsson, forskare och specialistläkare i barnonkologi vid Astrid Lindgrens barnsjukhus i Solna.

Every year, 300 children in Sweden are diagnosed with cancer. Thanks to research and new therapies, the majority recover, but still around 20 per cent die from different types of cancer. At HOPE, a clinical trial unit for children at Karolinska University Hospital, initiatives are now taken on a broad front to ensure children have the opportunity to trial innovative therapies.


Over a few decades, the treatment opportunities for children with cancer have improved significantly. In the 1950s, only a minority received treatment that healed, but today around four out of five children with cancer survive. Despite advances in research and treatment, however, childhood cancer is the most common cause of death among children aged 1 to 14 years, according to the Swedish Childhood Cancer Fund. Every year, around 20 per cent of the children suffering from cancer die; children who do not respond to conventional treatment and who are in acute need of testing new types of therapy.

– In this situation, families are looking for hope and want to find something that can help their child. “We want to enable these children and their parents to find alternatives, and to quickly be added to studies where they can test a goal-directed treatment that can, at best, help them to survive their cancer, says Anna Nilsson, researcher and specialist in childhood oncology at the Astrid Lindgren Children’s Hospital in Solna.

European collaboration

Since 2017, Anna Nilsson has been working half-time at HOPE (haematology and oncology trials unit), a clinical trials unit for medicines at an early phase, where children with hard-to-treat cancer get access to new tailormade therapies, and thereby an increased chance of survival.

Medicine studies, or ‘clinical medicine trials’, are an important part of trying to improve survival rates and reduce the side effects of cancer treatments.

– At HOPE, we carry out studies and clinical medicine trials for all different types of childhood cancer, and we see patients from all over the country.

Anna is the “principal investigator”, or head of trials, and responsible for planning and coordinating the clinical trials conducted at HOPE. The unit is a member of ITCC (Innovative Therapies for Children with Cancer), a European umbrella organisation consisting of around 50 child oncology centres. The organisation works with pharmaceutical companies, politicians and patient organisations to give children the chance to take part in studies using new cancer medicines.

– ITCC coordinates child oncology centres that are members and that conduct clinical studies. It also has an expert panel that selects promising studies. “As because HOPE is one of the sites, we take part and run some of the ITCC studies. Because of HOPE, we in Sweden have been able to expand this type of multi-centre studies, says Anna Nilsson.

HOPE is also taking part in the INFORM study, a register-based study coordinated by Heidelberg University Hospital in Germany. A number of European countries are taking part in the study, which aims to develop better diagnostics of cancer tumours in children and youths. Another purpose is to develop systems for individual risk assessment and individualised therapy proposals for recurrent childhood cancer.

– We regularly send tumour tissue to the research laboratory in Heidelberg for whole genome sequencing. They characterise the tumour, and send back information that forms the basis for our patient conferences, where we discuss which targeted therapies that we can offer. Personally, I think this is the best bit of my job, to try to implement new knowledge and new studies at the clinic, says Anna Nilsson.

– Most of the medicines trialled are new types of cancer medicines, such as immune system modulating medicines that are well-known and used for cancer in adults.

– At HOPE, we try out treatments and medicine dosages that are specifically adapted for children. They are more sensitive than adults, and have a different metabolism, among other factors.

Matching the right study to the right child

A lot of preparatory work has to be done before you can begin a clinical study. It involves things such as completing various applications, contacts with pharmaceutical companies and the authorities involved, such as the Medical Products Agency and the Ethical Review Authority. We also have to identify the patients who are suitable as participants in the study.

– To succeed, it is important that a trials unit such as this is integrated in healthcare, and that there is close collaboration between research and those who work in the day-to-day operation. We have to work from both ends, otherwise there is a risk of prolonging the time before children with cancer can get access to new therapies and medicines, says Anna Nilsson.

According to Anna Nilsson, one major challenge is finding the right study to match the child’s specific type of cancer and need for treatment. This requires resources, comprehensive logistics and well-functioning infrastructure.

– We have built up this infrastructure at HOPE, with a well-functioning network. When we at the clinic identify a child who is not responding to conventional therapy, then we need to find alternatives quickly. This requires resources, someone who can administer all the practical details, and contact other units in Sweden or around the world that can offer a specific therapy.

Lost their son to cancer

HOPE, which is largely privately funded, started in 2016 and is based on a collaboration between the Entrepreneurs for Good foundation, the Swedish Childhood Cancer Fund, Karolinska Institutet and Karolinska University Hospital. The people behind Entrepreneurs for Good are Johanna Hoffstedt and Fredrik Malm. They started the foundation after losing their three-year-old son Nelson to cancer.

– When our darling died, we also died. But after some time, we wanted to try doing something focused and powerful to support the doctors who had been totally fantastic during Nelson’s care and treatment. And, as devastated as we were by grief, just as convinced were we that our painful experiences could make a positive difference for others.

Johanna and Fredrik chose to direct their strength and energy at identifying development areas and initiating new solutions. They started collaborating with the heads of child oncology at Karolinska University Hospital, and with Karolinska Institutet and the Swedish Childhood Cancer Fund.

– Our doctors identified building up a clinical trials unit for children with cancer as the most important initiative for rapidly becoming able to save more children from dying from cancer. Such a unit already existed for adult patients in Region Stockholm, but not for children. Now it does exist, and gives hope to children and their despairing parents. We do everything we can to spread knowledge about its importance, and to find additional funding. Cancer should no longer have to be the most common cause of death in children, says Johanna Hoffstedt.