Home

Selumetinib optic glioma

Background: Pediatric low-grade gliomas (pLGGs) are the most common childhood brain tumor. Progression-free survival (PFS) is much lower than overall survival, emphasizing the need for alternative treatments. Sporadic (without neurofibromatosis type-1) optic pathway and hypothalamic glioma (OPHGs) are often multiply recurrent and cause significant visual deficits Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma all patients with non NF-1 associated optic pathway glioma with or without tissue must be enrolled on stratum 4 - Patients with sporadic (non NF-1 associated), histologically diagnosed progressive, recurrent or refractory non-optic pathway pilocytic. This phase I / II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment (refractory). Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth

Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth Despite being generally slow-growing, low-grade optic pathway gliomas are challenging to treat without causing disability. This phase II trial was designed to evaluate selumetinib, a selective inhibitor of mitogen-activated protein kinase (MAPK), for the treatment of patients with optic pathway and hypothalamic gliomas

This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas. Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth Selumetinib was supplied as capsules administered orally at a dose of 25 mg/m 2 /dose twice daily, the recommended phase II dose. 22 Patient adherence was measured utilizing patient diaries and pill counts, both evaluated at the end of each course. Each course was 28 days, and the dosing was continuous without planned breaks between courses

A Phase 2 Trial of Selumetinib in Children with Recurrent

  1. 10504. Background: A greater understanding of the Ras-MAP kinase-signaling pathway in pediatric low-grade glioma (LGG) paired with the availability of potent selective inhibitors has enhanced the ability to target this pathway with therapeutic intent.Methods: The PBTC conducted a multi-institutional phase II study (NCT01089101) evaluating selumetinib (AZD6244, ARRY-142886), a MEK I/II.
  2. Selumetinib is a selective and potent orally available non-ATP-competitive small molecule inhibitor of MEK1/2. The Pediatric Preclinical Testing Program showed that selumetinib induced complete regression in a BRAFV600E xenograft glioma model. 20 In 2017, the Pediatric Brain Tumor Consortium (PBTC) completed a phase 1 tria
  3. Selumetinib, a drug that inhibits the RAS pathway in patients with NFI and inoperable plexiform neurofibromas, successfully decreased tumor size, reduced pain, and improved quality of life in a sample of children participating in the trial. but with a different tumor called an optic pathway glioma, which are tumors of the optic nerve. These.

We report 2 patients with optic pathway gliomas who developed outer retinal layer separation visualized by optical coherence tomography while taking the MEK inhibitor selumetinib. After discontinuation of selumetinib, the outer retinal layer separation resolved without visual sequelae To determine the effect of selumetinib on stable NF1 related optic pathway gliomas and other gliomas for subjects whose primary indication for treatment is a progressive plexiform neurofibroma using evaluation of CNS MRI imaging Functional Outcome of patients with Plexiform Neurofibromas affecting the airway - PFT [ Time Frame: 3 years This phase I/II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment (refractory). Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells

Selumetinib in Treating Young Patients With Recurrent or

Selumetinib in Treating Young Patients with Recurrent or

An optic pathway tumor is one that is found along the visual system. This system sends signals from the eye to the brain so a person can see images. The visual system includes the optic nerve, the optic tract, the chiasm and the optic radiation. Optic pathway tumors squeeze the visual system A: Selumetinib (Koselugo, AstraZeneca) is an MEK 1 inhibitor, and we've found that most tumors in patients with pediatric low-grade glioma have abnormalities in the MAP kinase pathway where MEK..

Glioma. Selumetinib was an effective treatment for paediatric neurofibromatosis type 1-associated and sporadic recurrent/progressive hypothalamic and optic pathway glioma in a phase II trial (NCT01089101) [19, 20] Selumetinib is a drug that works by blocking some enzymes that low grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by..

There is new hope for Cataleya because researchers believe that there are drug options that might work for optic glioma tumors the way they do for plexiform neurofibromas. Cataleya just joined an exciting selumetinib clinical trial for patients with optic gliomas, and her family hopes that it will preserve what sight she has left Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas. » Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss.

Optic nerve - nerve that controls vision; Spinal cord - cluster of nerves and glial cells that connects the nerves of the body with the brain. How common is low-grade glioma? Pediatric low-grade gliomas are the most common central nervous system (CNS) tumor in children. They make up 30% of all childhood CNS tumors They also want to see if selumetinib is better than CV for improving vision in patients with LGG of the optic pathway. Selumetinib works by blocking proteins needed for cell growth and killing cancer cells. Patients in this study will be randomly assigned to receive either standard CV therapy for 15 months or selumetinib for 27 months We report outer retinal changes in 2 patients undergoing treatment in a clinical trial with the MEK inhibitor selumetinib for their optic pathway gliomas. Case 1 A 13-year-old girl diagnosed with a juvenile pilocytic astrocytoma of the optic chiasm infiltrating the hypothalamus and left optic nerve was being cared for at Children's National. This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatine / vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by. Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by.

To evaluate visual acuity (VA) outcomes utilizing Teller Acuity Cards (TAC) and HOTV letter acuity testing in previously-untreated optic pathway gliomas (OPGs). III. To describe the improvement in motor function as measured by the Vineland Scale in children with previously-untreated LGG that have motor deficits at enrollment Optic Nerve Glioma: Selumetinib: To evaluate the Maximum Tolerated Dose Objective response rate: NCT03741101: 6, 2019: 2: Sweden: 15: NF1 Plexiform Neurofibromas: Trametinib: Remission of tumor volume ≥20

Selumetinib in Children With Recurrent Optic Pathway and

  1. MEKi treatment of low grade glioma in Neurofibromatosis type 1. MEKi selumetinib and trametinib are currently employed in trials for low grade gliomas (LGG) including NF1 patients (NCT03363217, PNOC021, NCT03871257, NCT04166409, NCT04576117, NCT033263388, NCT01089101). LGG are common brain tumors in children
  2. o Optic glioma o Two or more Lisch nodules o A distinctive bony lesion o A first-degree relative with NF1 Member must have had at least on measureable PN, defined as a lesion > 3 cm measured in one dimension. Complete resection of PN is not considered feasible without substantial risk or morbidity (e.g.
  3. However if you google Selumetinib trial for optic glioma a lot of information will pop up and you will have a lot of information at your disposal. All the best, kieransdad. July 22, 2017 at 8:29 pm; Report; As far as fully tested, FDA-approved drugs are concerned, chemotherapy probably is your best course of action. Selumetinib has NOT been.
  4. Selumetinib is a small molecule oral drug that inhibits proteins called MEK1 and MEK2, which are involved a signaling pathway that affect cell proliferation and tumor cell survival. Chemotherapy drugs like carboplatin and vincristine work in different ways to stop the growth of tumor cells, either by killing the cells, by preventing them from.

Selumetinib in Treating Patients with Neurofibromatosis

In our cohort stabilization of the optic pathway, glioma also accompanied stabilization or improvement in visual impairment. By comparison, a phase 1 and 2 studies evaluated selumetinib (another MEK inhibitor) in children with PLGG and the authors observed a partial response after 13 cycles in 36%-39% of patients. 15,. Optic Nerve Glioma Selumetinib Change in the size NCT02407405 1, 2016 2 USA 60 NF1 Plexiform Neurofibromas Selumetinib Determine objective response rate NCT04461886 7, 2020 3 Japan 100 NF NPC-12G gel Discontinuation rate associated with adverse events NCT03871257 10, 2019 3 USA 290 Low Grade Glioma NF

phase II trial of selumetinib in children with recurrent

Key Eligibility • Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment • Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing • Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery • For patients with optic pathway. MEKi are indeed the only proven effective treatment for PNFs in NF1 patients , and have in particular been shown to be efficacious in NF1-related optic pathway gliomas in a phase 2 study . The response rates of NF1-related pLGG treated with selumetinib [ 18 ] appear to be similar to the response rates of NF1-related pLGG treated with. Optic pathway tumor is a type of glioma, a tumor that grows from glial cells which surround and support nerve cells. Optic pathway tumors in children are usually low-grade tumors. They grow along structures of the visual system including the optic nerve, optic tract, and/or optic chiasm

Selumetinib is a drug that works by blocking a protein that lets tumor cells grow without stopping. Vinblastine blocks cell growth by stopping cell division and may kill cancer cells. Giving selumetinib in combination with vinblastine may work better than selumetinib alone in treating recurrent or progressive low-grade glioma Pediatric low-grade gliomas (PLGG) are the most frequent brain tumors in children. Up to 50% will be refractory to conventional chemotherapy. It is now known that the majority of PLGG have activation of the MAPK/ERK pathway. The same pathway is also activated in plexiform neurofibromas (PNs) which are low-grade tumors involving peripheral nerves in patients with neurofibromatosis type 1 (NF1) The ancillary study will collect three sets of OCT measurements for 60 NF1 patients with optic pathway gliomas who are enrolled in the Phase III trial: before treatment with selumetinib, after 6 months of treatment, and after 12 months of treatment. Based on that data, they will determine whether OCT measurements can predict how patients will. showed that selumetinib induced complete regression in a BRAFV600E xenograft glioma model.20 In 2017, the Pediatric Brain Tumor Consortium (PBTC) completed a phase 1 trial21 of selumetinib in 38 children with recurrent, refractory, or progressive paediatric low-grade glioma, establishing the recommended phase 2 dose as 25 mg/m twice daily

A phase II prospective study of selumetinib in children

Protocol Description. Through this Pediatric Brain Tumor Consortium investigation, researchers will study the effects of selumetinib (AZD6244) in treating or re-treating young patients with a low-grade glioma that has come back (recurrent) or does not respond to treatment (refractory) Phase I and II study of the MEK inhibitor Selumetinib given twice daily on 5 out of 7 days in children with NF1 and inoperable plexiform neurofibromas or progressive/relapsed optic pathway gliomas. This study will test the early and late toxicities of selumetinib when it is given in this intermittent schedule (in 5 out of 7 days) and will also test the effectiveness of the drug in reducing the. Selumetinib is a drug that works by blocking some enzymes that low grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by. Due to this new visual complaints with associated retinal changes, selumetinib was discontinued. Her symptoms resolved within 2 days of the discontinuation. The OCT findings resolved and other examinations were stable after 12 days. A 6-year-old boy, who had a history of optic pathway glioma, received treatment with selumetinib

c-Jun N-terminal kinase inhibitor, SP600125, has also been utilized in preclinical trials of optic pathway gliomas and there was reduced proliferation and motility of neurofibromatosis deficient microglia and reduced size of glioma. 40 This presents a potential site for future therapeutic interventions in optic nerve gliomas NCI Definition: A glioma that affects the optic nerve. This condition can be seen in association with neurofibromatosis 1. It is most commonly seen in the pediatric age group. Clinical Trials View Clinical Trials for Optic Nerve Glioma . Significant Genes in Optic Nerve Glioma. Patients with optic pathway gliomas and prior BRAF or MEK inhibitors treatment were excluded. Pharmacokinetics were measured after the first dose of selumetinib. Pathology specimens were tested for MAPK pathway activation using immunohistochemistry for ERK (downstream from BRAF) A Study of the Drugs Selumetinib Versus arboplatin/Vincristine in Patients With for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). ACNS1831 NCT03871257 linical Trials for NS Tumors Onl

Optic Pathway Gliomas in Children with Neurofibromatosis Type 1 . Robert Listernick, MD Children's Hospital of Chicago . Optic pathway gliomas are benign (low grade) pilocytic tumors. They may involve any location in the optic pathway, starting on the optic nerve in the orbit (the eye socket), the optic chiasm (the area o Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma. This phase I/II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment (refractory)

We highlight recent successes with selumetinib while acknowledging ongoing challenges for NF1 patients and future directions. Recent Findings: MEK inhibitors have demonstrated efficacy for NF1-related conditions, including plexiform neurofibromas and low-grade gliomas, two common causes of NF1-related morbidity Inclusion Criteria: - Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment - Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing - Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery - For patients with optic pathway gliomas. This trial compares the treatment response of a new targeted therapy (selumetinib) to the present standard of care (carboplatin and vincristine) in children with newly diagnosed or previously untreated NF1 low-grade gliomas, including NF1 optic pathway gliomas A phase I/II study of Selumetinib for recurrent or refractory Low Grade Gliomas (including Optic Gliomas) in children, with or without NF1 (National Cancer Institute) So, what is selumetinib and why might it be useful to treat symptoms associated with Neurofibromatosis? Selumetinib belongs to a class of drugs called MEK inhibitors

Selumetinib is active in recurrent, refractory, or progressive pilocytic astrocytoma harbouring common BRAF aberrations and NF1-associated paediatric low-grade glioma. These results show that selumetinib could be an alternative to standard chemotherapy for these subgroups of patients, and have directly led to the development of two Children's. Second primary tumors in neurofibromatosis 1 patients treated for optic glioma: substantial risks after radiotherapy. J Clin Oncol. 2006;24:2570-5. Donahue B. Short- and long-term complications of radiation therapy for pediatric brain tumors Historical note and terminology. Chiasmatic gliomas arising in childhood have been the subject of many reports, with various recommendations for treatment (44; 24; 78).The first descriptions of the various types of orbital tumors were by Byers (17).Hudson was the first to suggest that optic pathway tumors were hamartomatous rather than neoplastic (46), a concept that was debated heatedly over.

Selumetinib in paediatric patients with BRAF-aberrant or

  1. The clinical study will collect three sets of OCT measurements for 60 NF1 optic pathway glioma patients enrolled in the Phase III trial: pre-treatment and after 6 and 12 months of receiving treatment
  2. (3) RAD001: RAD001 for Children with NF1 and Chemotherapy-Refractory Radiographic Progressive Low-Grade Gliomas This trial was a single-arm Phase II trial to evaluate the effectiveness of RAD001 (everolimus) in the treatment of pediatric patients with NF1 and low-grade gliomas (brain tumors and optic gliomas) that have not responded to standard.
  3. Selumetinib (Koselugo) is a mitogen-activated protein kinase (MEK) inhibitor for both MEK 1 and 2 that Optic glioma (OPG). 5. Two or more iris hamartomas (Lisch nodules - dome-shaped gelatinous masses developing on the surface of the iris). 6. A distinctive osseous lesion, such as sphenoid win
  4. A phase II prospective study of selumetinib in children with recurrent or refractory low-grade glioma (LGG): A Pediatric Brain Tumor Consortium (PBTC) study. Stratum I included children with non-NF-1 and non-optic pathway recurrent/refractory pilocytic astrocytoma (PA) harboring BRAF aberrations (BRAF V600e mutation or the BRAF-KIAA 1549.
  5. Selumetinib is in clinical development for children with neurofibromatosis type 1 (NF1), also called von Recklinghausen's disease. NF1 is a rare genetic disorder characterized by the optic glioma two or more neurofibromas or one plexiform neurofibrom
  6. eral density-Optic glioma Paired biopsy: PN, DNF pERK, pAKT, pMEK, kinome, transcriptome, PDX, immune infiltrate Target response rate / pts. 36%, 50 patients (stratum 1) 45% , max 35 patients (two-stage design

Video: FDA Approves Selumetinib for Children with Inoperable

Separation of outer retinal layers secondary to selumetini

  1. low grade glioma (other than optic pathway glioma [OPG]) with tissue available for BRAF analyses who cannot be classified into stratum 1, 2 or 5 due to inadequate tissue quality, assay failure, etc - Clarification: Stratum 4 was specifically designed for patients with hypothalamic/optic pathway gliomas; the intent is that if there is any optic
  2. selumetinib (Koselugo™) Policy # and gliomas. Clinical features of NF1 include skin fold freckling, distinctive osseous lesions, optic pathway gliomas, neurofibromas (including plexiform neurofibromas), and others with a variety of features present in each patient. Up to 50% of patient
  3. A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1) Study Opening Date. ACN1833 opened on 1/3/20
  4. Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma. Condition(s): Cutaneous Neurofibroma; Neurofibromatosis Type 1; Optic Nerve Glioma Last Updated: February 26, 2021 Recruitin
  5. without pseudoarthrosis), optic pathway glioma, neurofibroma (≥ 2 neurofibromas of any type or one plexiform neurofibroma), and a first-degree relative with NF1.2,4,5 In addition, children with NF1 may have developmental delays, learning difficulties, behavioral issues and attention-deficit hyperactivity disorder.2,4,

Intermittent Dosing Of Selumetinib In Childhood NF1

This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas. Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.. Clinical Trials Registry. ICH GCP The U.S. Food and Drug Administration last week granted breakthrough therapy designation for the MEK 1/2 inhibitor selumetinib. The designation is for the treatment of pediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic and/or progressive, inoperable plexiform neurofibromas (PN), a rare, incurable genetic condition Selumetinib (Koselugo®; AstraZeneca and Merck & Co.), an inhibitor of mitogen-activated protein kinases 1 and 2 (MEK1/2), increases the potential for successful tumor reduction in patients and could benefit over 2.5 million pediatric patients, 2 years of age and older, living with Neurofibromatosis Type 1 (NF1) and Symptomatic, Inoperable Plexiform Neurofibromas (PN). This is the conclusion [

UCSF Glioma Trial → Selumetinib in Treating Young Patients

As of today, Selumetinib is also being evaluated in clinical trials for other NF1-associated tumors, including optic pathway and other low-grade gliomas, cutaneous neurofibromas, and malignant peripheral nerve sheath tumors It is an exploratory aim of an ongoing COG phase 3 trial investigating selumetinib for the treatment of patients with NF1-associated low grade glioma. Drs. Avery and Fisher will test the validity of OCT as a tool to objectively assess the visual system and its response to treatment in NF1 patients with optic pathway gliomas Optic Pathway Glioma: Imaging Challenges. NF1 and LGGs . Childhood LGGs. Visual Pathway and Other Gliomas in NF1Irradiation. NF1 and LGGs: Treatment. Prospective Clinical Trials for Patients With LGGs With NF1. Selumetinib for LGGPhase 3 COG ACNSI838I Trial in Progress

UCSF Glioma Trial → A Study of the Drugs Selumetinib

Besides, they are in clinical trial now testing the incredibly effects MEK inhibitors on optic gliomas. I would much rather do a NF targeted treatment that often shrinks tumors, than a chemo treatment borrowed from cancer cases. Chemo would definitely not be effective unless the glioma is actively growing. Sometimes wait is powerful choice Chemotherapy may be used to treat optic pathway or other brain gliomas. The drug selumetinib (Koselugo®) has been approved by the U.S. Food and Drug Administration (FDA) to treat children older than two years old who have symptomatic but inoperable plexiform neurofibromas

Optic Pathway Gliomas | Ento Key

Separation of outer retinal layers secondary to selumetinib

NF1 is caused by a germline mutation in the NF1 tumor suppressor gene, which encodes neurofibromin, a GTPase-activating protein that functions as a negative regulator RAS [61, 89, 177]. 10-15% of children with NF1 will develop a low-grade glioma within the optic pathway, with an additional 3-5% arising outside of the optic pathway [14, 195, 196. gliomas and paediatric low-grade glioma harbouring either BRAF alteration.4 In The Lancet Oncology, Jason Fangusaro and colleagues 5 report the results of two of six strata of patients (aged 3-21 years) with recurrent, refractory, or progressive paediatric low-grade glioma from a phase 2 trial of selumetinib, a selective inhibitor of MEK1/2.

Glioma: Nf1 Optic Glioma

Selumetinib effective for pediatric low-grade gliom

Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by. Formula: C 17 H 15 BrC. l. FN 4 O 3. ChemSpider: 8303141 (Accessed: 2018) PubChem: CID 10127622. Selumetinib (AZD6244) is a drug being investigated for the treatment of various types of cancer, for example non-small cell lung cancer (NSCLC). [4 This phase 3 trial compares the effect of selumetinib versus the standard of care treatment with carboplatin and vincristine (CV) in treating patients with newly diagnosed or previously untreated low-grade glioma (LGG) that does not have a genetic abnormality called BRAFV600E mutation and is not associated with systemic neurofibromatosis type 1

The Use of MEK Inhibitors in Neurofibromatosis Type 1

A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1 The U.S. National Institutes of Health (NIH) announced that a Phase II clinical trial of AstraZeneca and Merck's selumetinib in neurofibromatosis type 1 (NF1) shrank inoperable tumors. NF1 is a genetic disorder that can affect multiple body systems. It is marked by skin changes, such as café-au-lait spots, Irish Lisch nodules, which are benign growths on the colored part of the eye.

A Study of the Drugs Selumetinib Versus Carboplatin

This study compares the treatment response of a new targeted therapy (selumetinib) with current standard of care (carboplatin and vincristine) in children with newly diagnosed or previously untreated NF1 low-grade gliomas, including NF1 optic gliomas - In addition to PN, subjects must have at least 1 other diagnostic criterion for NF1 as follows: 1. Six or more café-au-lait macules >5 mm in greatest diameter in pre-pubertal individuals and >15 mm in greatest diameter in post-pubertal individuals. 2. Freckling in the axillary or inguinal regions. 3. Optic glioma. 4 While selumetinib is the most advanced drug in terms of efficacy and toxicity data in the neurofibromatosis type 1 (NF1) population, other MEK inhibitors (MEKi) such as trametinib, cobimetinib, or binimetinib, are currently being tested in early phase clinical trials, or as compassionate use, in children with optic pathway glioma (OPG), with. • Optic pathway gliomas -unexplained visual loss, monocular or asymmetric nystagmus, Diencephalic syndrome, optic atrophy . Diagnosis : Imaging Rx with MEK inhibitors-selumetinib, Trametinib in Ph II studies • Additional alterations include RAF1 fusions, mutations, fusions o

Led by Michael J. Fisher, MD, researchers in the Fisher Research Program are using clinical outcomes analyses and molecular profiling to improve existing cancer treatment protocols. They are also evaluating new, targeted cancer therapies that can be used to treat pediatric solid tumors, including plexiform neurofibromas, optic pathway gliomas and medulloblastomas It is necessary to consider chemotherapy if an optic glioma causes vision loss and/or protrusion of the eyeball. Radiation should not be regarded as an option. Currently, a new treatment option using what are called MEK inhibitors (selumetinib and cobimetinib) for inoperable plexiform neurofibromas is receiving a lot of attention In particular, Surrogen's NF1 minipig displays café-au-lait macules, a diagnostic marker of NF1. In addition, the model also develops both optic pathway glioma and cutaneous neurofibromas, both of which are manifestations of the disease that have been challenging to study preclinically, until now