2020 CDMRP NF Research Highlight: Pain in Neurofibromatosis
For the past several years, the Neurofibromatosis Research Program (NFRP) has listed pain as an area of emphasis in its program funding opportunities, encouraging research applications aimed at addressing this critical need in the NF community. Each year, pain research continues to progress with new studies supported by the NFRP.
MGH Seeking Participants for Resiliency for NF Studies
Researchers at the Integrated Brain Health Clinical and Research Program at Massachusetts General Hospital are seeking participants for their studies, comparing 2 stress management programs for adults and adolescents with NF to see which one best improves quality of life and well-being for those living with NF.
The MEK inhibitor selumetinib may reduce spinal neurofibroma burden in patients with NF1 and plexiform neurofibromas
In a new publication, researchers detail the positive effect the MEK inhibitor, selumetinib, has on reducing the spinal neurofibroma burden in patients with NF1 and plexiform neurofibromas.
DermResearch and NFlection Therapeutics Seeking Participants for Cutaneous Neurofibroma Clinical Trial
DermResearch and NFlection Therapeutics are currently seeking new participants to enroll in their clinical trial for a new topical drug treatment. This trial is to study a gel treatment for cutaneous neurofibromas.
Technion discovers how to reduce cognitive damages of brain diseases
Technion-Israel Institute of Technology, in partnership with Cincinnati Children’s Hospital Medical Center (CCHMC), found out how to rescue functional and structural connectivity, and reduce cognitive impairments of brain diseases with drug treatment.
Please take this new NF survey for adults!
You are being invited to participate in an anonymous online survey to help researchers design psychosocial trials for adults with neurofibromatosis. We also have some important questions on how the current coronavirus pandemic is affecting you.
New Targets for Childhood Brain Tumors Identified
Findings at the Washington University School of Medicine in St. Louis indicates neurons and immune cells work together to promote tumor growth in neurofibromatosis type 1 (NF1). These findings point to new potential treatments for low-grade brain tumors in people with NF1.
FDA Approves First Treatment for Neurofibromatosis
The U.S. Food and Drug Administration has approved Koselugo (selumetinib) to treat plexiform neurofibromas in pediatric patients with NF1 ages 2 years and older. This approval means Koselugo is the first approved treatment for neurofibromatosis!
FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease
The U.S. Food and Drug Administration has approved Koselugo (selumetinib) to treat plexiform neurofibromas in pediatric patients with NF1. This approval means Koselugo is the first approved treatment for neurofibromatosis.
New Research Study for Understanding Chronic Pain and Work Barriers within NF Population
Dr. Frank Buono leads a new research study investigating the impact of NF pain and, as a result, the quality of life and work-related barriers.
Encouraging News for Kids with Neurofibromatosis Type 1
30 years ago, Dr. Collins led a team that discovered the gene that underlies NF1. Today, he is pleased to share what promises to be the first effective treatment to help young people affected by NF1.
In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1
In a trial sponsored by the NCI Cancer Therapy Evaluation Program (CTEP) and conducted by the NIH intramural program, the drug selumetinib improved outcomes for children with NF1 and plexiform neurofibromas.
Neurofibromatosis gene provides new insight into breast cancer resistance to hormone therapy
An international team of researchers led by scientists at Baylor College of Medicine has new insights into the function of neurofibromin, a tumor suppressor produced by the NF1 gene.
Bevacizumab Treatment Reportedly Increases Quality of Life in Adults & Children
Study Finds That Bevacizumab Treatment for NF2-Related Vestibular Schwannomas Increases Reported Quality of Life in Adults and Children.
SpringWorks' NF1 Plexiform Neurofibroma Trial
The ReNeu trial is a Phase 2b clinical trial designed to evaluate the safety, effectiveness, and tolerability of an investigational medicine called mirdametinib in children 2 years or older and adults with an inoperable NF1-PN.
DoD-Funded Study to Evaluate Pain Reduction in Adults with NF1
You are invited to participate in a group based focus group to discuss your chronic pain due to Neurofibromatosis Type 1 (NF1).
New Research Study at UMN and UWM for Children and Adolescents with NF1
Researchers at University of Minnesota, Twin Cities and University of Wisconsin, Madison are combining efforts to better understand the strengths and difficulties of children and adolescents with NF1.
The NFRP Presents the Fiscal Year 2018 Early Investigator Research Award Recipients
The Neurofibromatosis Research Program (NFRP) Early Investigator Research Award (EIRA) mechanism was offered for the first time in fiscal year 2018 (FY18).
SpringWorks Therapeutics' ReNeu Trial Press Release
SpringWorks Therapeutics Announces Initiation of Phase 2b ReNeu Clinical Trial of Mirdametinib in Children and Adults with Neurofibromatosis Type 1 (NF1)-Associated Plexiform Neurofibromas (NF1-PN)
New marker for tumor aggression in NF1
New molecular insights could mean more accurate prognoses and better selection of therapies.
Long-Term Outcomes of Stereotactic Radiosurgery...
The study confirmed a lack of significant difference in outcomes of SRS between NF2-associated and sporadic VSs.
A Greater Understanding of Tumor Cells
Through better understanding of the mechanical processes at work in tumors, Kristen Mills, an assistant professor of mechanical, aerospace, and nuclear engineering at Rensselaer Polytechnic Institute, hopes to support the development of more effective treatments.
SpringWorks Therapeutics Gains FDA Endorsement for Tumor Drug
Biotech firm SpringWorks Therapeutics has gained an endorsement from the U.S. Food and Drug Administration to help expedite the review of a drug that would treat rare tumors.
Couple give embryos, 'hope' to UM in fight against rare disease
The Cellucci family has donated seven embryos with the NF2 gene to the University of Michigan so researchers could create an embryonic stem cell line that other researchers could study, with the hope of one day discovering a breakthrough.
Investigating the Role of Fibroblasts in the Pathogenesis in Neurofibromas
With support from an Exploration-Hypothesis Development Award through the NFRP, Dr. Lu Le and his team are investigating the role of fibroblasts in the tumor microenvironment and hippo signaling pathway in neurofibromas.
Westerville father spearheads effort to fund research for daughter's disorder
The work of NF2 BioSolutions is now paying off in a big way.
Mueller Lab for Gene Therapy
Being moved by the impact of NF2, Dr Mueller decided to run a gene therapy pilot to identify if his lab’s gene therapy platform could be a good candidate for tackling the NF2 mutation.
New study reveals treatment pathway for neurofibromatosis
James Wymer, M.D. and his team have identified a novel pathway for potential treatment of NF.
Cerebellopontine Angle (CPA) Model
A Novel Tool for Investigating Immunotherapy in Neurofibromatosis Type 2 Vestibular Schwannomas
Dr. Vincent Riccardi Video
Dr. Vincent Riccardi is gaining momentum through Innovation and recruitment.
The Merlin Interactome Identifies Cell Junction Proteins
Proximity biotinylation identifies a set of conformation-specific interactions between Merlin and cell junction proteins
TOP NEWS: US FDA Grants Breakthrough Status To AstraZeneca Treatment
The drug selumetinib has been granted a breakthrough therapy designation in the US for neurofibromatosis type 1.
Neurofibromatosis Research Program Announcements
The NF Network plays a vital role in securing federal funds for Neurofibromatosis Research. One of the federal programs funded is the Congressionally Directed Medical Research Program, specifically the Neurofibromatosis Research Program Check out the NF Research Program Announcements for 2019.
Mice Sleeping Fitfully Provide Clues To Insomnia
Mice that sleep fitfully could help researchers unravel the mystery of insomnia.
Marker found for condition that causes tumors
UT Southwestern researchers have made a major advance in uncovering the biology.
25th Annual NF Center Research Symposium
Washington University marked 25 years of NF research by hosting the NF Center Research Symposium.
Clinical response to MEK inhibitor I in a NF1 patient
Ecancer medical science reported their August 2018 issue about a case of a 12 year old child with NF1.
NF Center Researchers Identify Key Role for Immune Cells in Brain Tumors
In a new study they now show that microglia can be programmed to support tumor formation and growth by T cells.
Brain tumors occur often in kids with common genetic syndrome
Neurologists have estimated that only 15 to 20 percent of kids with NF1 develop brain tumors.
Fiscal Year 2016 New Investigator Award Recipients
The Neurofibromatosis Research Program (NFRP) utilizes the New Investigator Award (NIA) to introduce the next generation of investigators and their ideas to the NF research community.
Confronting the Challenge of Neurofibromatosis
Neurofibromatosis as a name encompasses at least three diseases in fact. They are called neurofibromatosis type one, neurofibromatosis type two, and Schwannomatosis.
Genomic Roadmap for Sarcoma Established
This landmark study, involving Drs. Brian Van Tine, David H. Gutmann and Li Ding from Washington University, characterized over 200 adult sarcomas representing six different types of these cancers.
Surgeons Preserve Patients’ Hearing with Innovative Brainstem Implant
FDA-approved device helps patients with neurofibromatosis type 2
New guidelines for the clinical care of adults with NF1
American College of Medical Genetics and Genomics convened experts to write new guidelines
Halting Intracranial Tumor Growth
Promising treatment identified for tumor that causes hearing loss and sometimes death.
Study finds key risk factor for severe NF1 symptoms
Research led by Ludwine Messiaen, professor of genetics at the University of Alabama at Birmingham, found a key risk factor for severe symptoms of NF1.
Turning piglets into personalized avatars for sick kids
A team of scientists wants to accelerate research into a genetic disorder by using CRISPR to copy unique mutations from affected children into pigs.
The Specific Genetic Mutation May Influence NF1 Brain Tumor Development
Researchers at the Washington University NF Center found that the location of the NF1 gene mutation correlates with the development of brain tumors.
Study finds increased breast cancer risk
A new study published by Dovepress suggests women with neurofibromatosis type 1 begin screening for breast cancer at age 30 due to increased risk between ages 30 and 50.
New cell of origin for optic gliomas identified
A new study revealed information about the development of optic gliomas, a type of tumor children with NF1 are susceptible to.
Genetic tests + coordinated care + research = HOPE
Life with an inherited disease sometimes brings unexpected twists and turns. Five-year-old Gus Erickson has navigated the gyrations with the help of Mayo Clinic’s Neurofibromatosis Clinic.
New research offers hope to neuro-tumor patients
New research could offer hope to the thousands of people affected by Neurofibromatosis 2.
Participate in NF research
Did you know that there are 167 clinical trials for neurofibromatosis (NF) in various stages of completion? People with NF can play a critical role in the development of new treatments and the search for a cure by participating in clinical trials.
Washington U professor receives $3.5M grant
David H. Gutmann, director of the Neurofibromatosis Center at the Washing University School of Medicine in St. Louis, was awarded a $3.5 million grant to study NF1.
Drug shrinks pediatric neural tumors
In an early-phase clinical trial, in most cases, children with neurofibromatosis type 1 and plexiform neurofibromas responded with tumor shrinkage.
Swedish teen walks using 3D printed hip implant
Fanny Fellesen, a Swedish 16-year-old with neurofibromatosis, was told she would never walk again.
Drug shows promise with hearing loss
A drug already in use for some cancers is not only halting hearing loss in some patients, but reversing it.
New drug approved for NF2 treatment
BioXcel announced the U.S. Food and Drug Administration has granted Orphan Drug Designation to BXCL101 for the treatment of Neurofibromatosis Type 2.