Rare Disease Day Announcement Brings Attention to Genetic Disorder that Affects 1 in 25,000 Births
The Children’s Tumor Foundation and NF2 BioSolutions have announced a joint initiative to advance patient-focused research efforts for the neurofibromatosis type-2 (NF2) community. The collaboration, announced in recognition of Rare Disease Day, is focused on recruitment for two key resources: the NF Registry and the NF2 Biobank. The two organizations are working together to raise awareness of NF2 and to encourage robust participation in these two important tools, which will help increase scientific understanding of NF2, accelerate drug development of effective treatments, and lead to improved lives for NF2 patients. For more information…
Due to the COVID-19 pandemic, we had to postpone our April 3rd, NF2 Novel Therapies consortium in Boston.
We had 45 NF2 and Gene Therapy researchers and clinicians that were planning to attend from all over USA and europe.
Here below was our agenda:
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*Welcoming statement +NF2 BioSolutions updates *Patients perspectives – living with NF2 *Guests introduction (per group)
*Merlin targeted gene-based therapy approaches to NF2:
1. Clinical advances in CNS-directed gene therapy. From target to drug.
2. Current molecular understanding of NF2-associated Merlin mutations and their impact on protein function. Panel discussion.
3. Data Present/Plans from Nationwide group – Dr Meyer
4. Data Present/Plans from Mueller group – Dr Meijboom
5. Data Present/Plans from Dr. Castellano/Dr. Blanco
6. Group Discussion: Challenges/Milestones/Timelines
*Merlin-independent cell- and immuno-based therapy approaches to NF2:
1. Clinical advances in cell-based and immuno-based therapies.
2. Data Present/Plans from Gary Brenner and Sherif Ahmed + discussions
3. Discussion: Challenges/Milestones/Timelines
*Testimony living with NF2 + Coffee Break
*Cementing the basis for research and clinical studies through collaborations: Tumor bank, Biomarkers, Animal models and Natural History
1. Human Schwann Cell Model Systems for NF2. + Group discussion
2. Tissue Bank Kick off Discussion/stakeholder/partnership commitments + Group discussion
3. NF2 Animal models landscape. + Group discussion
4. Natural history study: NIH in action+ Group discussion
5. Discussion: Challenges/Milestones/Timelines
*New Approaches
*Next Steps
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We hope we can reschedule the meeting asap once the pandemic is over. In the meantime we will continue to interact with the researcher remotely.
April 3rd 2020: Kickoff of the first Open-Access NF2 Tissue/Cell bank to accelerate existing research and attract new labs into the race of curing NF2. Today it is very complicated for a research lab to have access to NF2 tumors (frozen/fresh), cell lines and genome sequencing data. This initiative will solve the access problem, we are going to collect tissue sample from many surgeons, performing analysis on them and share the data as well as the tissues. NF2 Cell line will be created as well and shared. For more info go here.
Gene Replacement Therapy for NF2 is starting!
August 1st 2019: NF2 BioSolutions signed a contract in July 2019 to partially fund a NF2 Gene therapy Pilot Pre-Clinical Study at the Abigail Wexner Research Institute at Nationwide Children’s Hospital by Dr. Kathrin Meyer, who is serving as the Principal Investigator.
June 26th 2019: Dr. Mueller of the University of Massachusetts Medical School attended NF2 BioSolutions’s NF2 Gene Therapy consortium in Boston on March 29th. Being moved by the impact of NF2, Dr. Mueller decided to start a gene therapy pilot study to determine if his lab’s gene therapy platform could be a good candidate for tackling the NF2 mutation. The advantage of his platform is its ability to silence the mutated gene and then add a healthy one. Dr. Mueller, assisted by Dr. Karin Meijboom, will be financing the development of the gene therapy NF2 vector for the pilot. Once manufactured, it will be tested on NF2 in order to determine efficiency. NF2 BioSolutions will facilitate the collaboration between the NF2 experts and Dr. Mueller’s lab.
This is just the beginning, there is much more to come!!! We need your continuing support as we shine the spotlight on NF2. Our goal is to collaborate with several leading gene therapy labs and share findings as we go to increase our chances of finding a long term solution for NF2.
Take a look at the impressive Mueller lab (the site is not yet updated with his new NF2 focus).
March 29th 2019: NF2 Gene Therapy Consortium
NF2 BioSolutions organized the first NF2 Gene Therapy Consortium where participated more than 40 biotech executives, researchers & clinicians from NF2 or leading Gene Therapy labs. Click here for more informations
Buy merchandise to support research
To buy merchandise, click here to see all the choices of t-shirts, cups, magnets… Fifty % of sales go to NF2 BioSolutions.
Article on WebMD about Nicole Henwood, founder of NF2 BioSolutions
If you need some convincing that gene therapies are the future, here you go - this graph by Pharmaprojects Informa from April 2021 really shows that more and more are in the pipeline at various stages of development.What does that mean for NF2? Basically, it means that we can #EndNF2 through gene therapies - but we can't do it without your support! ...
54 fighters joining this challenge!!! please signup: nf2biosolutions.org/get-involved/100-nf2-fighters-challenge-how-to-fundraise-online/ . Here is more information on why we raise these funds:People with NF2 have a genetic mutation that results in the growth of multiple tumors in the nervous system. They develop schwannomas, meningiomas and ependymoma tumors which generally first appear during childhood. These tumors cause deafness, pain, sensory/motor dysfunction, and eventually death through compression of peripheral nerves, the spinal cord, and/or the brain stem. Effective chemotherapy is essentially non-existent. Surgery and radiation will lead to nerve damage including deafness, blindness, chronic severe pain, loss of vocal chord and motor dysfunction. The paucity of therapeutic options, and the suffering caused by the limited options that do exist, makes the treatment of NF2 tumors a major unmet medical need.Dr.Brenner, a physician scientist at Harvard/MGH, developed and tested successfully on NF2 mice an AAV (adeno-associated virus) to deliver the ASC suicide-gene to schwannoma cells to induce the death of the tumor cells, leading to the shrinkage and sometimes resolution of the tumor. This gene therapy strategy utilizes a single transgene that potentially can: 1) induce cell death in the cells in which it is expressed, 2) propagate the death signal to adjacent tumor cells, and 3) initiate innate and adaptive immune responses that control subsequent future tumor formation.However, the use of the a virus to deliver this suicide-gene is challenging since it will be difficult to give several treatments to the patient. Overtime, the immune system develops neutralizing antibodies that block the drug effect. Today there is a new gene therapy delivery approach that will overcome the redosing challenge. It leverages the mRNA Covid19 vaccine delivery technique called LNPs (LNPs = lipid nanoparticules) as a mechanism to deliver genes to induce tumor cell death. These tiny particles can enter the cells and be used instead of a viral vector. LNP doesn’t have re-dosage limitations like we have with viruses.We need your financial support to start the use of LNPs as the delivery mechanism to destroy NF2 tumors. This project is in collaboration with University of Pennsylvania at the lab of Dr. Michael Mitchell. In this academic institution, the LNPs delivery technique originated. Step 1: $100,000 grant will fund a full time researcher and lab expenses for 12 months for developing the LNP that will transport the suicide-gene.Step 2: Another $50,000 grant will fund the next step that is the testing of the « suicide-gene LNPs » on NF2 mice at Dr Brenner Lab at Harvard Medical school/Massachusetts General Hospital. ...
Collecting biological specimens like brain tumors or skin bumps from people fighting NF2 is very, very important when it comes to accelerating the pace of research into NF2 and engaging more labs to work on NF2. So far, 55 of you have given your consent to share your tissues and blood samples. If you had surgery in the past or if you are going to have surgery in the future, please consider giving your consent as your tissue will be transferred to the NF2 Biobank.To sign up, please go to nf2biosolutions.org/open-access-nf2-biobank/...
13-year-old Dylan Ward is currently in the midst of walking 100 laps around his UK neighborhood - that's 50 miles - to raise awareness and funds for NF2 BioSolutions, "so that more doctors can help us get a cure" for a monstrous genetic disorder called NF2.And he's being cheered on by people, both in his neighborhood as seen here and also by supporters around the world. And why is Dylan doing this? This is not just because he's the son of NF2 BioSolutions UK volunteer and NF2 fighter Joanne Ward. As he says in his own words; "I want to help my nine-year-old brother Oscar and all the other children in the world affected by NF2 so they can grow up with better lives, but I know that it won't happen without the money for research.So, I decided that I could make a difference by volunteering for the charity and raising some money while raising awareness. I've got a few other events happening later in the year too."Keep walking the good walk and fighting the good fight, Dylan! A grateful NF2 BioSolutions thanks you and is behind you - 1000% of the way! ...
We need a total 100 fighters to raise $1000 each. So far 50 fighters joined the challenge! Please join this challenge and together we will jumpstart our next novel therapy research at University of Pennsylvania.For more info and for signing up , go her, we need you: nf2biosolutions.org/get-involved/100-nf2-fighters-challenge-how-to-fundraise-online/...
We need a total 100 fighters to raise $1000 each. So far 48 fighters joined the challenge! Please join this challenge and together we will jumpstart our next novel therapy research at University of Pennsylvania.Signup here: nf2biosolutions.org/get-involved/100-nf2-fighters-challenge-how-to-fundraise-online/NF2 Biosolutions is putting the best labs and best minds in the race to tackle the cruelty of NF2. We continue nonstop at evaluating new approaches and techniques to tackle NF2. So far we support four different novel therapy approaches. Since we don't know yet if these approaches that are demonstrating promise in the lab will ultimately work as well in a human, we don't want to focus on just one area. We want to continue to onboard new labs and researchers with novel ideas and medical approaches.There is a new approach that we want to jumpstart. It is leveraging the Pfizer/Moderna Covid19 vaccine delivery technique (LNPs = lipid nanoparticules) as a mechanism to deliver genes to correct or induce tumor cell death .We need your financial support to start this research in collaboration with University of Pennsylvania (Philadephia) at the lab of Dr. Michael Mitchell. The University of Pennsylvania is the place where LNPs delivery technique originated. $100,000 will fund a full time researcher and lab expenses for 12 months.We will reach this goal if 100 people sign up here to this challenge of raising $1000 each.Deadline to raise $1000 is June 1st, 2021Signup here: nf2biosolutions.org/get-involved/100-nf2-fighters-challenge-how-to-fundraise-online/Thank you for joining this fight ...
Greetings, everyone! In the midst of all that is going on around the world, we have some news to share - one of our NF2 BioSolutions UK team, Ebony Jade-Barber will be speaking at the NFYA International Summit this Saturday at 2:45pm EST or 7:45pm GMTLet's log in through bit.ly/2RidU4R and give her some support! ...
Great newsletter here: nf2biosolutions.org/news/press-release-and-tv-coverage/spring-2021-newsletter/This newsletter is packed with updates from NF2 BioSolutions. You’ll learn about our latest fundraising efforts, get to check out the “Linked Together” video, hear more from Dr. Gary Brenner on his Bacteriotherapy lab, learn about Nissa Novas’ NF2 journey, watch our latest webinar on the surgical management of NF2, and plenty more! We’re keeping busy at NF2 BioSolutions working for a cure! We won’t rest until we have one. ...
The latest webinar recording is here! Watch NF2 BioSolutions and our esteemed panel of surgeons discuss the surgical management of NF2 tumors, including schwannomas, ependymomas, and meningiomas.You will find the link to the webinar transcript as well.Our expert panelists in the webinar :-Prof Lekovic and Prof Slattery, House Institute in Los Angeles, California-Prof Friedman and Prof Schwartz, University of California San Diego-Prof King, Salford Royal Hospital, University of Manchester, UK-Prof Kalamarides, Pitie-Salpetriere Hospital, Sorbonne University, Paris, France.This panel has significant experience with NF2 tumors and will discuss their approaches in treating and managing NF2. The webinar had live Q&A of your most burning questions! Please continue to support us so we can continue to boost research and NF2 awareness. ...
Join NF2 BioSolutions and our esteemed panel of surgeons as we discuss the surgical management of NF2 tumors, including schwannomas, ependymomas, and meningiomas.
Here's some amazing news from NF2 BioSolutions UK for everyone to take note of! NF2 BioSolutions UK are delighted to have been asked to be panel members at the first ever "NF International youth Summit" on 10th April 2021. Our Wales ambassador Ebony-Jade Barber is on the Q & A panel at this event which has been organised jointly by the Childhood Tumour Trust and the Littlest Tumour Foundation. All members of the NF1, NF2 and Schwannomatosis young adult community (ages 18-30) are welcome to join us on Zoom in welcoming NF young adults from around the world. Exciting speakers including Arooj Aftab, Kay Ashton MBE and two experts from the medical world - Dr. Gareth Evans and Prof. Eric Leigus. ...
In the UK, Thornbury Golf Club’s ladies vice captain Annie Griffin has walked an astonishing 370 miles since February 8 as part of a fundraising challenge.Annie is undergoing the challenge to raise money for NF2 Biosolutions through our UK chapter, NF2 BioSolutions UK.Ms Griffin, we at NF2 BioSolutions thank you for your effort, your commitment and your support. Together, we will #EndNF2! ...
Dylan Ward, 13 plans to walk 50 miles - 100 laps of his neighbourhood - over two days on April 17 and 18. Why? Dylan said he wants to raise awareness and funds for NF2 BioSolutions, "so that more doctors can help us get a cure".From a grateful NF2 BioSolutions, we thank you and your family, Dylan! Walk strong, walk proud! ...
Today's International Women's Day and it is no secret that NF2 BioSolutions would be nothing without the women who created it, without the women driving us forward towards the day when we can finally #EndNF2. From our innovating scientists like Dr. Kathrin C. Meyer of Nationwide Children's Hospital in Ohio to our Scientific Advisory Board member Krizelle Alcantara and our founder Dr. Nicole Henwood, our fight would be nothing without the efforts of all the women - the mothers, sisters, aunts, grandmothers, scientists, artists, experts and volunteers in a whole assortment of fields and skills and NF2 fighters driving our fight forward.To all of you, we at NF2 BioSolutions salute you, we thank you! ...
NF2 BioSolutions | 71 followers on LinkedIn. Accelerating Biologic Therapy Research for NF2 | We are a non-profit organization (Tax-ID 83-0940046) seeking a cure or preventative treatment for NF2 by vigorously supporting and advancing existing and next-generation biomedical technologies, such as gen...
Join NF2 BioSolutions and our esteemed panel of surgeons as we discuss the surgical management of NF2 tumors, including schwannomas, ependymomas, and meningiomas.
Due to the COVID-19 pandemic, we had to postpone our April 3rd, NF2 Novel Therapies consortium in Boston.
existing research and attract new labs into the race of curing NF2. Today it is very complicated for a research lab to have access to NF2 tumors (frozen/fresh), cell lines and genome sequencing data. This initiative will solve the access problem, we are going to collect tissue sample from many surgeons, performing analysis on them and share the data as well as the tissues. NF2 Cell line will be created as well and shared. For more info go here.
Gene therapy Pilot Pre-Clinical Study at the Abigail Wexner Research Institute at Nationwide Children’s Hospital by Dr. Kathrin Meyer, who is serving as the Principal Investigator.
consortium in Boston on March 29th. Being moved by the impact of NF2, Dr. Mueller decided to start a gene therapy pilot study to determine if his lab’s gene therapy platform could be a good candidate for tackling the NF2 mutation. The advantage of his platform is its ability to silence the mutated gene and then add a healthy one. Dr. Mueller, assisted by Dr. Karin Meijboom, will be financing the development of the gene therapy NF2 vector for the pilot. Once manufactured, it will be tested on NF2 in order to determine efficiency. NF2 BioSolutions will facilitate the collaboration between the NF2 experts and Dr. Mueller’s lab.
NF2 BioSolutions organized the first NF2 Gene Therapy Consortium where participated more than 40 biotech executives, researchers & clinicians from NF2 or leading Gene Therapy labs. Click here for more informations
To buy merchandise, click here to see all the choices of t-shirts, cups, magnets… Fifty % of sales go to NF2 BioSolutions.
