METHODS FOR THE TREATMENT OF NEUROFIBROMATOSIS

The invention relates to a PI3K inhibitor for use in the treatment of neurofibromatosis type 1 and type 2 in a subject in need thereof. Currently there are no treatment of neurofibromatosis type 1 and 2. Patients mainly received supportive care to treat severe symptoms including surgery to remove tu...

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Bibliographic Details
Main Author CANAUD, Guillaume
Format Patent
LanguageEnglish
Published 17.02.2022
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary:The invention relates to a PI3K inhibitor for use in the treatment of neurofibromatosis type 1 and type 2 in a subject in need thereof. Currently there are no treatment of neurofibromatosis type 1 and 2. Patients mainly received supportive care to treat severe symptoms including surgery to remove tumors compressing nearby tissue or damaging organs, stereotactic radiosurgery or cochlear implants. Inventors have worked on immortalized NF1−/− cells and shown that PIK3CA pharmacological inhibition (BYL719) was associated with increased apoptosis as assessed by PARP cleavage in a dose dependent manner. They also decided to expose NF1−/− cells to a multitargeted therapy including BYL719+selumetinib or BYL719+selumetinib+IPA-3. Importantly, they found that both combinations led to severe apoptosis with double strand DNA break as assessed by the phosphorylation of H2aX. This new approach with either BYL719 alone or in combination with other therapeutics seem to be very promising in patients with neurofibromatosis.
Bibliography:Application Number: US201917274600