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 Burlo-Garofolo children’s hospital in Trieste. Among the
authors we find very well known names in the field of people fighting NF: Dott.
ssa 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 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!