Encouraging results from Italian groups on the treatment of tumours of young patients suffering from Neurofibromatosis type 1 have been just published. The work entitled “Selumetinib in the Treatment of Symptomatic Intractable Plexiform Neurofibromas in Neurofibromatosis Type 1: A Prospective Case Series with Emphasis on Side Effects” has been published in the prestigious scientific journal Pediatric Drugs by the team of the Buro-Garofolo children’s hospital in Trieste. Among the authors we find very well known names in the field of people fighting NF: Dr. Irene Bruno, Dr. Egidio Barbi and Dr. Francesco Baldo Pediatr Drugs 22, 417–423 (2020). https://doi.org/10.1007/s40272-020-00399-y ).
The Trieste group is already well known nationally and internationally for its long experience and commitment in the care and treatment of children suffering from neurofibromatosis.
The article describes the beneficial effects of the drug called Selumetinib (it is a MEK inhibitor!) administered to 9 children who together had 17 tumours. The children did not show any worrying side effects, in fact, in some cases, acne, gastrointestinal problems appeared and paronychia (infection of one or more toes or hands), while 16 tumours regressed and one growing stabilized.
Good news therefore in the field of treatment of plexiform tumours, in addition to those recently illustrated by Linfa in the work of the American group led by Dr Widemann.
Our hopes for at least partial treatment of neurofibromas are increasingly strengthened by new, very positive data, and we hope that in the near future other groups will publish their results and others will be encouraged to start treating suffering children.
A crucial question emerges overwhelmingly, but when will we think about adults?
In two American hospitals a trial (trial) for adults has started, we are talking about the Birmingham Cancer Center University of Alabama and the National Institutes of Health Clinical Center. This study started because the American drug agency, the Food and Drug Administration has approved Selumetinib for treatment in both children and adults (Children’ Tumour Fondation Conference 2020, https://www.ctf.org/get-involved/nf-conference).
In Europe, we are awaiting approval of the drug by the European Medicine Agency (EMA). In the absence of this approval, the drug is administered only for so-called “compassionate” use (this is said when there is not yet approval from the EMA, which must first analyze all experimental data and ascertain the actual efficacy of the drug and exclude toxic effects) and for now only to children.
Let’s keep our fingers crossed!