Our MLC Consortium, a group of clinical MLC experts founded this year, has just released their first statement in the Pediatric Neurology journal. Their editorial letter examines a recent publication of two patients with MLC who received a drug called Anakinra, typically used to treat arthritis in children. According to the published case report, the authors suggested that Anakinra may have had a positive effect on these two children. Notably, the children demonstrated some improvement and brain scans suggested less swelling in a part of the brain called the cerebellum.
However, our MLC consortium examined this case report in more detail and have published their findings here. Overall, the consortium cautions the interpretation of these results, as it is difficult to know if Anakinra really helped these patients as the study did not compare with a control i.e. patients who did not get the drug. Though the authors mentioned improvements were noticed in both patients, this is not uncommon as children with MLC often continue to achieve milestones even as disease deterioration continues, so the improvements may not be because of the drug. Though the report said the white matter in the cerebellum was less swollen, our experts mention that this part of the brain usually is not swollen in MLC, so these results are difficult to interpret. Since using Anakinra is not risk free – it requires daily painful shots and has possible side effects – the consortium suggests we need more research before giving this potential drug to MLC patients. Studies are currently underway to evaluate the effect of Anakinra in MLC mouse models.