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RI children get access to new therapy for neurofibromatosis type 1
Indonesian children with Neurofibromatosis Type 1 (NF1), a rare genetic condition where tumors grow on the nerves and skin, now have access to Selumetinib, the world’s first recognized therapy for NF1.
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Approximately 27 million Indonesians are currently at risk of rare diseases, with 50 percent of cases affecting children and 30 percent of those children not surviving past age 5. (Shutterstock/File)
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ndonesian children with Neurofibromatosis Type 1 (NF1), a rare genetic condition where tumors grow on the nerves and skin, now have access to Selumetinib, the world’s first recognized therapy for NF1.
NF1 is one of the most frequently occurring rare diseases in young children.
NF1, 96 percent of all neurofibromatosis cases, affects 1 in 3,000 children worldwide, with around 120 babies born with the condition every day. In Indonesia, there have been 140 documented cases of NF1, affecting children as young as 8 and 12 years old, as well as adults with tumor complications.
“It’s good news for us to learn in 2022 about the approval of a new treatment [for NF1 patients with plexiform tumors]. In Indonesia, this treatment [Selumetinib] was registered at the Food and Drug Monitoring Agency [BPOM] in 2023,” said pediatrician Damayanti Rusli Sjarif of state-run Cipto Mangunkusumo hospital.
She said breakthrough therapy is desperately needed to tackle NF1.
“A gene called neurofibromin 1 normally functions to control and prevent excessive cell growth in the body. Because of a mutation in the neurofibromin 1 gene, cells in skin and nerves can grow rapidly and uncontrollably,” said Damayanti, who is also a diet and children’s metabolic diseases consultant.
In its early stages, NF1 may appear mild, with symptoms such as café-au-lait or brown-colored spots and freckles in the armpits or groin area. But it can lead to the growth of tumors in various locations, such as the face, internal organs, nerves in the eyes and even in the brain.
