MLD is a serious genetic disease caused by a defect in the DNA. This gene therapy is the only effective treatment for a severe variant of MLD in which children deteriorate rapidly. The Dutch government has recently started to reimburse this therapy for patients who are not yet symptomatic. UMC Utrecht and UMC Amsterdam have collaborated to provide access to this treatment.
Reimbursement
Young children with the rare metabolic disease MLD can now survive thanks to a new treatment. The gene therapy was developed in Milan by academic centers and a pharmaceutical company. It is a one-time treatment with the product 'Libmeldy'. The reimbursement applies to children with a severe form who do not yet have symptoms. For patients who already have symptoms, it is being investigated whether they can start treatment through conditional access. UMC Utrecht and UMC Amsterdam have collaborated to provide access to this treatment.
Utrecht Stem Cell Transplant Center
In the Netherlands, a few children per year qualify for this treatment, which takes place at the Stem Cell Transplant Center for Children in Utrecht. This is a joint initiative of the Princess Máxima Center for Pediatric Oncology and the Wilhelmina Children's Hospital, where stem cell transplants are performed for patients with metabolic diseases. Recently, Utrecht became one of five stem cell transplant centers in Europe for this treatment. The Leukodystrophy Center of Amsterdam UMC is heavily involved as an international MLD Center of Expertise.
Peter van Hasselt, Head of Metabolic Diseases, Wilhelmina Children's Hospital: 'This new form of stem cell transplantation is more effective than conventional stem cell transplantation and makes it possible to treat the most severe forms of this metabolic disease, for which until recently there were no treatment options. That is why it is so important that this treatment is now available in the Netherlands. We hope that this more powerful form will also make treatment possible for a number of other metabolic diseases.’
Caroline Lindemans, stem cell transplantation physician at the Princess Máxima Center: 'It is fantastic that the expertise within the joint stem cell transplantation center of the UMC Utrecht and the Princess Máxima Center also provides the space to enable innovative gene therapies for children with non-malignant diseases.’