To see our newsletters, Press release and TV coverage click here.
To see our research initiatives news click here
To see our webinars explaining the science behind the projects we are supporting click here
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:
***************************
*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
***************************
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
Mom Blazes Path to Cure for Son’s Rare Disease
Click here to read the article in WebMD about Dr Nicole Henwood, president of NF2 BioSolution.
Upcoming Events
Click here to Follow us on Facebook and don’t miss our updates:
Exactly one year ago this week, NF2 BioSolutions UK kicked off as an officially accredited charity focused on supporting research into cutting-edge gene therapies and treatments that will #endNF2.
The launch was marked with the participation of one of our two UK Ambassadors - Clare Goddard - in the Richmond Marathon, who finished the race in a solid four hours and fifty-six minutes
As she said when met at the finish line;
"I feel like I have achieved something really great and launched NF2 BioSolutions as a charity.
With this we can really get going and move forward and get lots of money; there are a lot of grants we can get from foundations for NF2 BioSolutions UK.
I'm definitely going to look into doing lots of different races because there is a lot of funds to be had at races like this.
My idea was to lead from the top, do a marathon and encourage others to do the same.
This is the first marathon I've run. I've been training since May and I decided that I had to do something challenging because all the people with NF2 face challenges every day and I'm healthy and well and this is something I can do to challenge my body to help all who are fighting NF2. They are my inspiration today. They really got me going, I was doing it for them and now we are going to go ahead and make something of this to accelerate the development of gene therapy for NF2."
And if you're in the UK and are interested in learning more, do head to bit.ly/3iCOPum to learn more about the work they're doing.
"I was reassured to hear from Dr. Marks that although he could not discuss the Complete Response Letter “nothing has changed when it comes to the pathway to approval of gene therapies for diseases with unmet need.”
The writer goes on to say that the FDA is " is ever more committed to this area of medicine knowing the promise it holds for patients who have no good options right now."
Given this climate, we should push all-out until we have something that can #endNF2
Dr. Peter Marks Clarifies FDA’s Approach to Gene Therapies After CRL for Hemophilia A Treatment
By Jennifer McNary, mother of two sons with Duchenne muscular dystrophy, patient advocate and founder of One Rare. For all the incredible progress we’ve made in the area of gene therapy, we are still very much in the pioneer days. While two directly administered gene therapies have been approved b...Thinking of doing a good deed to commemorate your birthday, or just thinking of celebrating it differently?
Well, why not set up a donation drive in support of NF2 BioSolutions - something that has been done by many, many people in the past! All funds received by us will be used to #endNF2 by supporting research in labs actively working on developing gene therapies that will address NF2 at its source.
If you are interested or have any questions, please contact Terri Rausch at terri@nf2biosolutions.org. And if you'd like our help to design a non-Facebook donation page or special link, we can help with that; please contact Gil Atlan at gilles@nf2biosolutions.org to find out more.
"COLUMBUS, Ohio – For the first time ever, a team of neurologists and neurosurgeons at The Ohio State University Wexner Medical Center and The Ohio State College of Medicine has performed a novel gene therapy brain infusion to treat a patient with Parkinson’s disease.
This multicenter, Phase 1b clinical safety study is sponsored by Brain Neurotherapy Bio, Inc. and funded by the California Institute for Regenerative Medicine to test GDNF gene therapy in patients with early to moderate stages of Parkinson’s disease. The one-time treatment involves infusion of a gene therapy solution into deep structures of the brain that are affected by the disease. "
The next disease beaten could be NF2, but it depends on us. We can beat NF2 as long as we stay the course and keep our eyes on the prize. As it is, we're also working with the Wexner Medical Center, collaborating as a partner in their research efforts to #endNF2.
Wexner Medical Center Performs First Gene Therapy Brain Infusion For Parkinson's Disease
wexnermedical.osu.edu
For the first time ever, neurologists and neurosurgeons at The Ohio State University Wexner Medical Center and The Ohio State College of Medicine have performed a novel gene therapy brain infusion to treat a patient with Parkinson’s disease.Greetings everyone! We at NF2 BioSolutions are excited to announce that we have two - yes, two - new members on our Board of Directors, and like all on our team, their families have been directly touched by the disorder.
Amy Williams lives in Louisville, Kentucky with her husband Rob and between them they have been blessed with 4 wonderful boys. Amy is a retired Dental Hygienist and decided 8 years ago to go back to the dental world where she is office manager to a prominent pediatric dental office. Amy’s son Parker was diagnosed in 2018 at the age of 19, and as Amy says;
“He is the first in my family and his father's family to have this diagnosis. You can only imagine our shock and disbelief when we learned of this.
He is being seen by his team of doctors once every six months for a repeat brain MRI and he has a yearly spine MRI to monitor his bilateral vestibular schwannomas, spinal cord tumors and peripheral tumors.
Since his diagnosis, he has had Gamma Knife radiosurgery to a tumor on his right side in August 2018, and he started Avastin in April 2019.
Currently, there is no cure for NF2 and I want to help change this; I believe wholeheartedly in the approach of the doctors, scientists and the amazing team that has come together to form NF2 BioSolutions."
Amy is also our Kentucky Ambassador and she is the one who sends out the warm thank-you letters in response to all the generous donations we receive mailed-in.
As she says in closing;
“I am honored and humbled to have been approached by NF2 Biosolutions to be part of the Board of Directors. I will do everything I can to help educate ,spread awareness, raise funds and to hopefully create a network of support for each other. Let’s help end NF2 together."
Amy can be contacted at amy@nf2biosolutions.org
---
Chantelle Nobis lives in upstate New York with her husband and three children. She is currently a middle school music and chorus teacher going into her 17th year of teaching, having earned a Bachelor of Science in Music Education and a Master’s Degree in Literacy. Her husband Peter was diagnosed with NF2 in 2008 and their daughter Amelia was diagnosed in 2015, with Peter's case of NF2 being a spontaneous one.
Peter has a very mild case of NF2 in the form of bilateral vestibular schwannomas while Amelia seems to be a very rare, more unpredictable case. Since her diagnosis, she has had a golf ball-sized malignant brain tumor (a meningioma) removed in 2015, spinal cord tumors, a cataract, cafe-au-lait spots, intraneural perineuromas, and an additional meningioma in her brain.
Since Peter and Amelia's diagnosis, Chantelle has made it her mission to find others with the same disorder and fight for a cure. She truly believes that gene therapy is the answer to curing NF2, and this - along with her family - is the reason she has decided to join and support NF2 BioSolutions.
As she says;
“I look forward to meeting and working with other families affected by NF2, so we can educate, spread awareness, and fundraising for research and a cure. It is an honor to be added to the Board of Directors, and I will go above and beyond to join them in our mission to cure NF2.”
She can be contacted at chantelle@nf2biosolutions.org
The best way to motivate and support our researchers, read below:🚨I am an ambassador for NF2 BioSolutions, and I need your help! ‼️I am looking for any artwork/message/photo that can be hung on the wall of the research labs (I will frame them for you) currently working to help us find a cure for NF2. 🌟 We want our researchers to see as many faces of NF2 - to give them some energy and put a face to the “why” for their research!
💙 Each of you has a story, an NF2 hero/warrior and a passion/fight bigger than anyone around you, with the desire to cure this monster of NF2. 💙 Help me share your story💙
💡 *Theres an example below
📬Once you are finished, message Chantelle Nobis for the address to physically mail them! 📫 by October 28th! 🗓
👉If you want more info on the labs, the research continuing, or would like to donate check out our website: nf2biosolutions.org/
or our Facebook page: www.facebook.com/NF2BioSolutions/
As a patient, NF2 fighters can strike a hard blow against the genetic disorder that is NF2 by donating tumor and tissue samples that can be used for research.
Indeed, every time NF2 BioSolutions talks with leading research labs, these labs ask for NF2 tissues and cell lines made from fresh tissues.
The following was written by NF2 BioSolutions vice president Gilles Atlan;
.
Two weeks ago, my 12-year-old daughter Karen had 2 skin lesions - plexiform schwannomas - removed; one was removed from scalp and the other from the top of her foot. A large number of NF2 patients have these kind of skin bumps, and these tissues are very important for research since they contain the cells that are mutated.
After the surgery, we transferred Karen's tissues to the NF2 Tissue bank that is being run by NF2 BioSolutions and the Children Hospital of Philadelphia (CBTTC). Once the samples are there, it will be analysed, stored and the tissues and data will be shared with labs working on researching treatments for NF2. So far, more than 30 NF2 fighters have transferred their tissues to our NF2 tissue bank - and we want to reach the goal of 100 tissue samples collected in the first year. The process is very easy; it takes just one phone call to state your consent - and that's it! And if you're wondering, our phone lines are accommodating if you're Deaf or hard of hearing.
Here is the video of the removal of the lesion from Karen's foot. It was painless and fast, thanks to the professionalism of Dr Jean at Main Line Plastic Surgery ( www.facebook.com/mainlineplasticsurgery ) near Philadelphia. You can also see Catherine on the video from the NF2 tissue bank that will receive the fresh tissue.
For those interested in donating tissue samples, please contact Shannon Robins from the tissue bank to coordinate their transfer by ROBINSSM@email.chop.edu and CC-me at gilles@nf2biosolutions.org
For more inf, head to nf2biosolutions.org/gene-therapy-and-nf2/open-access-nf2-tissues-cells-bank/
Thank you
Gil Atlan, VP of NF2 BioSolutions
We are delighted to report that our Axe Out NF2 Campaign has reached and exceeded our minimum goal of $100,000! Thanks to the generous support of many benefactors, NF2 BioSolutions can accelerate research into the tumor-targeting bacteria approach at The Brenner Lab at the Massachusetts General Hospital.
The research spearheaded by Dr. Gary Brenner is a very promising approach, one that will certainly accelerate our efforts to find a cure for NF2.
This critical funding will lead to the ability to submit an IND (Investigative New Drug) application with the FDA, an important next step in finding a cure.
At NF2 BioSolutions, we believe in working towards a world where those living with a rare disease - including NF2 - will live full and productive lives, free from physical and emotional pain and suffering.
Visit www.nf2biosolutions.org to learn more about our efforts to support and advance existing and next-generation biomedical technologies by collaborating with three world-renowned labs.
We at NF2 BioSolutions are grateful and happy to announce that we have hit a big - and we mean big - milestone, which we couldn't have done without all of our supporters, so thank you!
Basically, we have raised more than US$1,000,000 since we kicked off in late 2018 and all these funds are being channeled to three labs working on four different approaches to either stop tumor growth or kill tumors that have already formed. We at NF2 BioSolutions will continue to scout for promising research into new therapies that could be applied against NF2 mutations. The managements and executives of NF2 BioSolutions are still 100% volunteers that receive no salaries or benefits, a policy that allows us to have the biggest impact when it comes to accelerating research.
Again, thank you from all of us at NF2 BioSolutions - we couldn't have done all we've done without your support!
Hi, our devoted NF2 BioSolutions volunteer Chantelle Nobis is organising a Pampered Chef Fundraiser.
You’re invited to an ONLINE Fundraising Pampered Chef Party, hosted by Chantelle Nobis, facilitated by Cindy Donaldson her Pampered Chef Consultant!
PARTY POSTING STARTS THIS MONDAY and PARTY ENDS FRIDAY 28 August at 10PM EST!! But play the games over the weekend! and earn tickets for the drawings!
This is an ONLINE Fundraising party - you never need to leave the couch to shop for some awesome kitchen products! and you are supporting NF2 BioSolutions as wel!!!
INTERACTIVE CATALOG:
www.pamperedchef.com/pws/divacin/shop-landing-page
but please order from her party
www.pamperedchef.com/party/chantellenobis0814
*
Even if you are unsure of your level of participation and/or plan to purchase a product, we want you to be part of this party!
*
This 5-day party will be filled with cooking tips and tricks, recipes, games, and information on Pampered Chef products that save people time in the kitchen.
Here's how it works:
1. Enter the Group www.facebook.com/groups/752668255532912/?ref=share - CLICK "JOIN" and "OK" to be added
2. Visit Chantelle's Show Site
www.pamperedchef.com/party/chantellenobis0814
3. Click "Shop Now"
4. WIN FREE PAMPERED CHEF PRODUCTS just for participating in the event.
***The more you participate the more chances you have to WIN FREE PRODUCTS during the party***
Thank you! I'm so excited!!!!
Chantelle’s NF2 BioSolutions Pampered Chef Fundraiser
Let’s fund raise together online! You can shop in yourA company with ties to angel investing and venture capital groups that wants to remain anonymous made a very generous donation of USD250,000 to NF2 BioSolutions; a family member of one of their employees is a NF2 fighter.
This donation will boost Dr. Gary Brenner's research into tumor-targeting bacteria - basically genetically modified bacteria that will be introduced into tumors caused by NF2 to kill the cells.
If you work for a company that has a policy of donating to nonprofits or has a donation-matching program, please let them know about the research being supported by NF2 BioSolutions. We are wholly aimed at supporting research into long-term treatment for NF2 treatment that will save lives for years and years to come.
The son of Nicole (NF2 BioSolutions's founder) AJ had a brain surgery on Thursday to remove a tumor and trying to save his hearing and facial nerve. Please give all your support to Nicole. Without Nicole's energy and knowledge, we won't have any hope today to have a future long term therapy for NF2.
“Tremendous progress has been made in the discovery of new therapies for debilitating diseases that are difficult to treat. This approval is yet another example of customized treatments that use a patient’s own immune system to help fight cancer, while using a scientific advance in this promising new area of medicine,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
First cancer, next NF2 - this will be the reality, and it's attainable within our lifetimes. Our scientists just need your support to make this possible!
globalgenes.org
Rare Daily Staff The U.S. Food and Drug Administration granted accelerated approval to Tecartus, Gilead subsidiary Kite’s cell-based gene therapy for treatment of adult patients diagnosed with mantle cell lymphoma who have not responded to or who have relapsed following other kinds of treatment. T...And here's some good news to kick off August - NF2 BioSolutions is now working with the Center for Data-Driven Discovery in Biomedicine or D3b, which is run by the Children's Hospital of Philadelphia. Under the cooperation, the following will take place:
> NF2 Biosolutions and D3b will collaborate to perform observational prospective longitudinal clinical research studies for patients with brain tumors, neuro diagnosis and birth defects for the collection of biospecimens and clinical data for use in D3b and for external collaborators and external investigators;
> create comprehensive harmonized clinical molecular disease datasets that are annotated with associated available biospecimens for use in D3b and for external collaborators and external investigators;
> create disease-specific preclinical models clinically annotated with associated biospecimens and genomic data for use in D3b and for external collaborators and external investigators including drug and CRISPR Screens;
> perform hypothesis based translational brain tumor research in D3b and with external collaborators and support independent external investigators research to develop, test and launch novel therapeutic strategies and
> provide a molecularly based clinical decision, clinical decision support for clinical trials, clinical trial development
d3b.center
Background Neurofibromatosis 2 (NF2) is a rare genetic disorder that is primarily characterized by noncancerous (benign) tumors of the nerves that transmit balance and sound impulses from the...Conner Curran was 4 years old when he was diagnosed with Duchenne muscular dystrophy, a genetic disease that causes muscles to waste away.
Conner's mother, Jessica Curran, remembers some advice she got from the doctor who made that 2015 diagnosis: "Take your son home, love him, take him on trips while he's walking, give him a good life and enjoy him because there are really not many options right now."
Five years later, Conner is not just walking, but running faster than ever, thanks to an experimental gene therapy that took more than 30 years to develop.
---
This is proof positive that gene therapies can work, do work and are the future - and with the strides that have been made, we don't have to wait for more than 30 years for a treatment for NF2. We just have to keep our scientists supported and in the fight - and that's what NF2 BioSolutions is committed to doing!
A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived
Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.