In the summer of 2016, Norman and his family return to the Netherlands after a long stay abroad. Their youngest daughter Louisa, eight months old, suddenly becomes very sick. First they think it’s a flu, but doctors in the Sophia Children’s Hospital quickly make a different diagnosis: Louisa has acute lymphatic leukaemia (ALL).

Moving train

Norman: ‘To make matters even worse, Louisa had a very rare form of the disease. Children under the age of one year have a higher chance of abnormalities in the so-called MLL gene. This makes treatment more difficult, and significantly reduces the odds of a cure. It feels like you’ve jumped onto a moving train. Everything is defined in protocols. Louisa qualified for a two-year treatment program with a fairly intensive chemotherapy regimen; she needed to stay in the hospital for the first two months of that. After the diagnosis, she received her first dose of chemotherapy. Only a few months later did the realisation strike: this is a long lasting disease, it’s not going away easily. The process is gruelling.’

Side-effects

‘In general, things went fairly well. But there were complications. She responded very poorly to some cytostatic medicines; others she did better with. But she also suffered indirectly due to the chemo, in the form of serious infections. Her immune system was minimal, so her body could not deal with bacterial and viral infections properly. There was a phase during which she was quarantined fully.’

Surviving

‘Your life comes to a standstill. The only thing that matters is caring for your child. But we also had two other daughters, three and five years old. They had also just returned from England, and were building new lives here. We made the conscious decision to make sure their lives were as normal as possible – school, swimming lessons, visiting friends. During the first period, my wife spent every day in the hospital, and I was there after work whenever I could be.’

Recurrence

‘In September, Louisa was put on less intensive treatment. The signs were hopeful; no more leukemia cells could be detected. But in December, we received the news we were all dreading. A bone marrow and lumbar puncture showed that the leukemia was back. The fact it recurred during treatment was considered a bad omen. Unfortunately, it also showed that there were leukemia cells in the brain fluid.’

Tailored treatment

‘You’re suddenly thrust into uncertainty, into a domain without protocols or manuals. The only option we had in the Netherlands was a bone marrow transplant. The question remained whether the treatment would be effective for Louisa, since she was so severely weakened and due to the leukaemia cells in the brain fluid. The chances of success in her case were also very poor. We talked to doctors about this, but we did not have a good feeling.’

Immunotherapy

‘Our doctor then pointed us towards an experimental treatment at the Children’s Hospital of Philadelphia: CAR T therapy. Children with ALL receive treatment with CAR T-cells. This is a one-time treatment, in which specific immune cells belonging to the patient – the T-cells – are collected and modified genetically in a laboratory so that they can recognize and destroy CD19-positive leukaemia cells. We were lucky – Louisa was given a spot in an ongoing clinical trial. Things moved very quickly, and before we knew it we were in an airplane heading for an intake meeting.

Philadelphia

‘The treating team decided that Louisa’s T-cells could be collected in the Netherlands, in the Wilhelmina Children’s Hospital in Utrecht. The cells were then shipped to the USA. The lab at the Children’s Hospital of Philadelphia then worked to modify her T-cells for six to eight weeks. Finally, in May 2018, the big moment arrived – the administration of the modified T-cells. The whole family temporarily moved to Philadelphia for her treatment. Fortunately, her condition had not worsened significantly in the meanwhile.’

Suspense

‘Of course, the moment her processed T-cells were returned is tense, but the treatment itself is no big deal. For Louisa, it consisted of a single injection. Fortunately for her, other than a bit of fever, there were no side-effects. I’m still amazed at how easily it went. If you see what chemotherapy does to your body, and if there is an alternative that appears to work for my daughter, that’s great news. We also realised that Louisa was extremely lucky; the treatment does not work well for half of patients.’

Going to school

‘We have recently returned from a follow-up visit in Philadelphia. Louisa has had a clean bill of health for a year and three months now. She has a full head of hair, is growing quickly and enjoying life to the fullest. In March 2020 she will start kindergarten. That’s something we never dared to? dream. CAR T therapy literally opened up a new world for her. Before, she led a very sheltered life due to the risk of infections. It’s the first time she can visit the supermarket or a restaurant now. Her mental and physical development is moving in leaps and bounds.’

Future

‘Only now are we really processing everything that has happened. We are trying to start living a normal life as a family again. But the uncertainty re-mains. I’m afraid that’s just part of life as a parent of a child with cancer. You can never be sure it’s completely gone. But every quarter we receive good news, means a longer ‘clean’ bill of health. We are thankful every day for the opportunity we were given. We never dared to? dream she would respond so well to treatment.’

Photo: The girl in the photo is not Louisa.