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The Network Edge

The Neurofibromatosis Network is delighted to bring you our new science column, The Network Edge, written by science writer Vanessa Merker, BS, providing a summary of recent highlights from NF research and clinical trials.  The Network, through advocacy on Capitol Hill and direct funding of research, plays a central role in progress toward a cure.
 

Every three months The Network Edge will bring you the latest research news as compiled by science writer, Vanessa Merker.  The goal is to keep you abreast of advancements in treatment and clinical care for all forms of NF.
 
The Network Edge is presented in bite-sized pieces, allowing you to easily find and focus on those topics of most interest to you, whether it is NF1 learning disabilities studies, NF2 clinical trial updates or schwannomatosis news.

Volume 13: Fall, 2016

Highlights

  • Congressional Update: Twenty-one new NF projects are being funded by the Congressionally Directed Medical Research Program - Neurofibromatosis Research Program (CDMRP-NFRP).
  • NF Clinical Trials: Pegylated interferon slows down the growth of plexiform neurofibromas in young people with NF1; vinblastine may be an effective treatment for optic pathway glioma.
  • NF1 Biology: Researchers study how neurofibromas begin and later transform into MPNSTs, with some success identifying potential genes and pathways that new drugs can target.
  • NF2 Clinical Management: Researchers review auditory brainstem implants in NF2.
  • NF2 Biology: Research using two FDA-approved drugs, celecoxib and crizotinib, to treat schwannomas shows promising results in the lab and in mice.
  • Schwannomatosis Update:  A special type of MRI reveals nerve abnormalities in people with segmental schwannomatosis.
  • Quality of Life:  The Relaxation Response Resiliency Program (3RP) delivered over Skype shows promising results in improving quality of life of adults with NF1, NF2, and schwannomatosis.

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Volume 12: Summer, 2016

Highlights
  • NF1 Learning Disabilities: Lovastatin may help improve memory in children and adults with NF1.
  • NF1 and Cancer: People with NF1 under 30 are the most likely to develop brain tumors or MPNST; women with NF1, especially those under 45, have an increased risk of breast cancer.
  • NF1 Brain Tumors: Children with NF1 and low grade gliomas have good short-term and long-term survival after treatment; controversy continues over MRI screening for optic pathway gliomas.
  • NF2 Vestibular Schwannomas: Treatment with bevacizumab can improve hearing in some patients with NF2; a new mouse model offers a good opportunity to test new drugs.
  • Schwannomatosis Update: Schwannomas may appear cancerous on PET scans even when they are not; advances in MRI techniques may improve our ability to understand this phenomenon.
  • Quality of Life: A preliminary study of Acceptance and Commitment Therapy (ACT) shows promising results helping adolescents with NF1 and chronic pain.

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Volume 11: Spring, 2016

Highlights
  • Congressional Update: $15 million in funding is secured for the Congressionally Directed Medical Research Program-Neurofibromatosis Research Program (CDMRP-NFRP) in 2016!
  • NF1 Learning Disabilities: Large brain size in NF1 may relate to learning and social disabilities; the challenge of translating findings in mice to clinical applications in humans is examined.
  • NF1 Tumors: Old imaging technologies to monitor MPNSTs are used in new ways; hormones and neurofibroma growth are examined; what makes plexiform tumors bleed during surgery?
  • NF2: Successful use of cochlear implants and auditory brainstem implants in people with NF2 is reported; researchers focus on children with NF2.  
  • Schwannomatosis: New cell lines will help open up research into this rarest form of NF.
  • Breast Cancer in NF1: Two different outcomes highlight the importance of this area.
  • New Roles for the NF1 Gene: The NF1 gene emerges as a regulator of brain progenitor cells and also plays a role in chromosome organization and very early cardiovascular development.

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Volume 10: Fall, 2015

Highlights
  • New insights into how Bevacizumab is used to treat NF2 schwannoma and ependymoma tumors.
  • How a new UK Registry facilitates exploration of the causes and predictors of mortality in NF2.
  • Impact of migraine and pain on NF1 quality of life.
  • An analysis of teaching approaches to improve reading skills in children with NF1.
  • How NF2 schwannomas arise in the brain, and their possible relevance to treatment decisions.
  • NF2 biology updates: new drug candidates for meningiomas, understanding merlin biology.
  • The value of early life monitoring for NF1 optic pathway gliomas using MRI.

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Volume 9: Summer, 2015

Highlights
  • A new mouse model of NF1 dystrophic scoliosis: A new mouse model will allow closer study and drug testing.
  • Malignant peripheral nerve sheath tumors: New drug targets show promise; studies examine a “personalized medicine” approach to finding the right drug treatments.  
  • Increased age-specific breast cancer risk in NF1: A British population study suggests women with NF1 may be at greatest increased risk of breast cancer in their 30’s.
  • NF1 learning disabilities: A new candidate drug target is identified; studies examine impact of NF1 mutation type.  
  • Recognizing early life cognitive differences in NF1: Differences in brain function in children with NF1 may be seen in the first months of life, before they are recognized by parents.  
  • Quality of life in NF2: Complications of NF can prevent people from engaging with the world. New studies examine how challenges may be identified and addressed. 
  • NF2 Drug Treatments: Bevacizumab remains most promising; studies suggest tapering down the dose to reduce side effects while maintaining the drug’s tumor-treating effects.

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Volume 8: Spring, 2015

Highlights
  • A new NF1 patient registry highlights the value of patient-reported data.  
  • rhBMP-2, a candidate drug for promoting bone healing shows promise in NF1 mouse models.  
  • In NF1, understanding autism spectrum disorder and finding evidence for depression risk.  
  • SUZ12, a new genetic regulator of MPNSTs and candidate drug target; testing new MPNST drugs.  
  • Methylphenidate shows promise for treatment of AD(H)D in NF1. 
  • Children with NF1 have compromised motor skills, not necessarily due to attention deficits.
  • Implicating the NF1 gene in alcohol dependence. 
  • A closer look at Sirolimus treatment of plexiform tumors – do people respond differently to this?
  • What makes meningiomas become malignant? New candidate treatments for these NF2 tumors.
  • Hope comes from a promising single-case trial of Bevacizumab to treat schwannomatosis.

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Volume 7: December, 2014

Highlights
  • Survey of insurance claims shows NF1 may be linked to higher risk of other medical conditions.
  • Candidate drugs to improve bone mineralization and treat pseudarthrosis in NF1 are presented; sunshine exposure improves Vitamin D levels in NF1 but still falls short. 
  • From MPNST biology, a new candidate drug and two new drug targets emerge.  
  • Impact of NF1 on communication, academic success in early life, and later lifestyle is examined.  
  • In NF1 clinical trial, drug Sirolimus doesn’t shrink plexiform tumors, but it may help with NF1-related learning and behavioral challenges.  
  • Bevacizumab shows promise in clinical trial for treatment of the rare NF1 high-grade glioma.
  • New mouse model of NF2 represents human condition, opens the door to new research.  
  • Quality of life impact is a key consideration in selecting NF2 treatment regimes.

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Volume 6: Fall, 2014

Highlights
  • Understanding aneurysms and stenosis in NF1. 
  • Exploring Methylphenidate (Ritalin) for treatment of attention deficits in NF1.  
  • Real-life impact of NF1 learning disabilities on individuals and families.
  • Interpreting MRI hyperintensities in the NF1 and the NF2 brain.
  • New candidate drug targets and treatment approaches for NF1 and NF2.  
  • Surgical decision making in NF2 clinical management.  
  • Updates on clinical trial and drug treatment reports for NF1 and NF2.  
  • A new schwannomatosis gene is identified.  
  • Legius Syndrome updates; possible genetic links to leukemia.

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Volume 5: Fall, 2013

Highlights
  • Social challenges for those with NF1 and NF2. 
  • A new look at life expectancy in NF1 and NF2.  
  • Unique considerations of seizure and epilepsy management in NF1.  
  • NF1 learning disabilities – clinical trials update; new modes of early detection; a possible link with sleep disorders.
  • Updates on monitoring for MPNSTs; developing targeted drugs for these tumors.
  • New NF2 candidate drug targets.
  • The search for NF1-related gene mutations beyond the NF1 gene.  
  • A definitive link between autism and NF1.

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Volume 4: Summer, 2013

Highlights
  • New biological markers to better predict development of MPNSTs. 
  • Imaging brain structure: a new approach to measuring NF1 vision loss in NF1?
  • A potentially helpful role for radiotherapy following NF2 meningioma surgery.  
  • Café-au-lait marks may carry secrets of NF1 biology. 
  • Children with NF1 have reduced brain blood flow. 
  • New NF2 drug therapy shows promise. 
  • Growth factor TGF-beta contributes to NF1 bone abnormalities. 
  • Special considerations for surgical management of very large NF1 plexiform tumors. 
  • Sleep disturbances in NF1. 
  • A new mouse model for studying NF1 learning disabilities.  
  • Early screening for BRCA mutations in women with NF1 to detect breast cancer risk.
  • Multiple-spinal-menigioma disease, a new genetic condition defined.

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Volume 3: Spring, 2013

Highlights
  • Evaluating progress in the CDMRP NF Clinical Trial Consortium.
  • Bevacizumab is not effective in shrinking NF2 meningiomas.
  • Women with NF1: challenges faced in pregnancy.
  • Young adults with NF1 often overlook the importance of their clinical care.
  • New quality of life surveys for those with NF1 and NF2 could help detect health concerns.
  • High blood pressure and vascular health issues may affect children with NF1.
  • Breakthrough findings uncover the basis of nerve damage and pain in NF2.
  • International consensus update on schwannomatosis diagnosis and clinical care.
  • NF2 diagnosis in infancy may predict poorest outlook.
  • Update on genetic testing for Legius Syndrome.

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​​​​​​Volume 2: Winter, 2013

Highlights
  • CDMRP NFRP – A Critical Funder of NF Research. 
  • A Focus on NF1 Malignant Peripheral Nerve Sheath Tumors. 
  • NF2 Clinical Management Updates. 
  • NF1 Learning Disabilities: News from the Clinic and the Bench.
  • NF1 Clinical Management Updates. 
  • What’s New in NF2 Biology?
  • Heart and Blood Vessel Abnormalities in NF1: New Findings. 
  • What’s New in NF1 Biology?
  • NF1 Bony Abnormalities Update. 
  • The Evolving Link Between NF and Cancer.

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Volume 1: Fall, 2012

Highlights
  • Impact of the Neurofibromatosis Network on Federal NF Research Funding.
  • CDMRP NFRP Clinical Trials Network Expands.
  • NF1 Clinical Trials.
  • NF1 Clinical Management.
  • NF2 Clinical Trials.
  • NF2 Clinical Management.
  • NF1 Learning Disabilities.
  • NF1 Bony Abnormalities.
  • Schwannomatosis Update.
  • Increased Breast Cancer Risk in NF1.
  • Other Clinical Features of NF1.
  • What’s New in NF1 Biology and Drug Targets?
  • What’s New in NF2 Biology and Drug Targets?
  • Legius Syndrome Update.

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Volume 14: Spring, 2017

Highlights

  • NF1 Clinical Trials: In an early stage clinical trial, selumetinib shrank plexiform  neurofibromas in 71% of children, with anecdotal improvements in appearance, pain and motor function.   
  • NF1 Clinical Trials: Lovastatin does not significantly improve visual/spatial processing or attention in children with NF1.
  • NF1 Brain Tumors: Larger optic pathway gliomas are more likely to cause vision problems; research in mice suggests that estrogen might lead to more vision problems in females.
  • NF2 Clinical Management:  Bevacizumab can shrink fluid-filled cysts in spinal ependymomas, leading to clinical improvements in some patients.
  • Schwannomatosis Update: An international patient registry seeks to increase research opportunities and collaborations for schwannomatosis.
  • Quality of Life: Two new surveys specifically designed to measure quality of life in children, adolescents and adults with NF1 are being developed.

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