In April 2020 the U.S. Food and Drug Administration (FDA) approved KOSELUGOTM (selumetinib) for use in children over age 2 with neurofibromatosis type 1 who have inoperable plexiform neurofibromas. This is the first drug ever approved specifically for a complication of NF Type 1. Obviously this has been huge news!

The process of getting this drug tested and approved has been a long road full of many collaborations including those between scientists and researchers as well as between NF organizations, volunteers and donors. The research was led by the National Cancer Institute (NCI), a division of the National Institutes of Health (NIH) with clinical trials conducted there in collaboration with clinical trials at several pediatric hospitals. Also, much of the funding came either through the federal government budget for the NIH or from the Congressionally Directed Medical Research Program (CDMRP). NF Midwest, NF Northeast, Texas NF and several other NF organizations and volunteers collaborated over the years to advocate for federal funds for NF research through these programs.

As NF regional patient organizations dedicated to informing and educating our communities, NF Midwest, NF Northeast and Texas NF  collaborated along with several NF experts to bring you Frequently Asked Questions (FAQ) document on KOSELUGO.





2019 - $5,000 funded to Ethan Lester, PhD of Harvard Medical School / Massachusetts General Hospital for "In Need and Hard to Reach: Adaptation of an evidence-based neurofribomatosis resiliency training program for web-based delivery"


2019 - $30,000 funded to Jonathan Rios, PhD of Texas Scottish Rite Hospital for Children for "Identify and Characterize Molecular sub-types of NF1 Pseudarthoroses"


2019 - $45,000 funded to Kelia Torres, MD, PhD of University of Texas MD Anderson Cancer Center for "Determine combination therapies that leverage the genetic and epigenomic deregulations of MPNSTs"

2018 - $25,000 funded to Jonathan Rios, PhD of Texas Scottish Rite Hospital for Children for "Clinical Challenges in Treating Tibiad Pseudarthrosis"


2018 - $25,000 funded to Dr. Keila Torres of the University of Texas MD Anderson Cancer Center for "Methylation Profiling in NF1 Patients"

2017- $45,000 funded to Dr. Lu Le of the University of Texas Southwestern Medical Center for "Phase 2 Clinical Trial of BET Inhibitor for Malignant Peripheral Nerve Sheath Tumor"

2017- $20,000 funded to Dr. Keila Torres of the University of Texas MD Anderson Cancer Center for "Characterization of the Methylome in MPNST"

2016 - $44,081 funded to Dr. Laura Klesse of Children’s Medical Center Dallas and University of Texas Southwestern Medical Center for her research titled “Cardiovascular Abnormalities in Pediatric Patients with Neurofibromatosis Type 1." $19,543 funded to Keila Torres, MD, PhD of The University of Texas MD Anderson Cancer Center for her research titled  “Evaluation of PARP Inhibition in Malignant Peripheral Nerve Sheath Tumors”.

2015 - $20,000 funded to Dr. Steve Richards with Texas Scottish Rite Hospital for his research on tibial pseudarthrosis. $30,000 funded to Dr. Keri Smith with The University of Texas Health Science Center at Houston for her research titled "Molecular Modeling of Neurofibromatosis Response to mTOR Inhibition to Uncover Targets for Treatment." 

2014 - $50,000 funded to Kristine Vogel, Ph.D. - Assistant Professor, Cellular and Structural Biology The University of Texas Health Science Center at San Antonio . Project: DNA Damage Repair and Disease Severity in Neurofibromatosis Type 1.

2013 - $20,000 funded to Alice Ann Holland, Ph.D. with Children's Medical Center Dallas. Project: Health Correlates of Bullying in Pediatric Neurofibromatosis.

2013 - $20,000 funded to Dr. Lu Le with UT Southwestern Medical Center.  Project: Identifying Novel Therapeutics for Malignant Peripheral Nerve Sheath Tumors.  

2012 - $20,000 funded to Dr. Laura Klesse, with  Children's Medical Center and UT Southwestern Medical Center.  Project: Neurofibromatosis Associated Plexiform Neurofibromatosis Analysis for Novel Therapeutic Targets.

2012 - $15,000 funded to Dr. Dina Chelouche Lev with the University of Texas MD Anderson Cancer Center. Project: The Role of HDAC8 as a novel anti-MPNST therapeutic target. 

Congressionally Directed Medical Research Programs Fiscal Year 2011 Annual Report


2011 - $30,000 Sponsored a Drug Discover Initiative award through CTF to Dr. David Largaespada, Masonic Cancer Center, University of Minnesota.  Project: Combinatorial signal pathway inhibition in a novel model of high grade neurofibroma development.

2010 - $45,000 Partnered with CTF to fund a Young Investigator Award to Dr. Jody Fromm, Harvard Medical School/Brigham Women's Hospital. Project: To determine the therapeutic effects of combined sunitinib and rapamycin treatment on MPNST's.

2009 - $47,000 Awarded to Dr. Ian McCutcheon, Dr. John Slopis and Dr. Daniel Cahill at the University of Texas MD Anderson Cancer Center. Project Title: Genotype-Phenotype correlation predicting malignant tumors in NF 1



For any questions or inquiries please call 972-739-6086  or fill out the following form:

OUR MISSION: The Texas NF Foundation is committed to meeting the needs of people impacted by neurofibromatosis by providing comfort, support, education, advocacy and funding of research for a treatment, prevention or cure.



320 Decker Drive, Suite 100

Irving, TX 75062

Office: 972-739-6086 or 877-515-TXNF

Fax: 972-739-6087

EIN: 74-2138345

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© 2020 by The Texas Neurofibromatosis Foundation. All Rights Reserved.

This project was supported by

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