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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
***************************
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.
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The strategist Sun Tzu once said that "if you know the enemy and know yourself, you need not fear the result of a hundred battles."
Indeed, to win against any enemy - one must first understand the basics of what one is up against, even if the enemy in this case is a rare disease.
This free course here has the potential to empower a person with some of the fundamentals needed to understand genetic diseases, essential if one is to understand how gene therapies work and why the research supported by NF2 BioSolutions is important in the fight against NF2.
Genetics Concepts for Rare Disease Patients & Families
rareuniversity.com
Many of us within the RARE community find empowerment in educating ourselves about genetics and learning more about the condition that impacts ourselves or our loved ones. With advances in genetics like gene therapy and new genetic testing technologies many of us want to be able to keep up with the....Are you a NF2 fighter who's just had surgery or are you gearing up to have a surgery soon?
You can help advance the global fight against NF2 by filling up a simple form at redcap.chop.edu/surveys/?s=F9L8ED7W33 - and please do consider doing so, because by contributing your tumor to our tissue bank, you will be helping to boost research into NF2 and hopefully treatment through gene therapy.
This graphic here basically shows what will happen once you fill our form.
And if you need to learn more, you can go to nf2biosolutions.org/gene-therapy-and-nf2/open-access-nf2-tissues-cells-bank/ for more detailed information.
Let us welcome Jonathan Gooch from the United Kingdom into our Professional Advisory Board
Jonathan is the loving father of a 18-year-old son who was diagnosed with NF2 in early 2019, and he has chosen to step up and use his background as an accomplished Global Regulatory Affairs professional to help his son and other NF2 fighters around the world #endNF2 by working to support our effort to make gene therapy for NF2 a reality.
His career - which spans the last 25 years - has been focused in the the pharmaceutical industry in a variety of companies and settings. Jonathan has experience of small molecules and biologics in multiple therapeutic areas.
Please read his bio at nf2biosolutions.org/about-us/professional-advisory-board/
nf2biosolutions.org
NF2 BioSolutions is proud to receive the support of these amazing advisors, guiding us through legal, financial and industry matters. M. Jaysson Eicholtz – FDA Advisory Committee Jaysson grad..."In one case in the Medi-Cal pilot project, a newborn girl had a life-threatening irregular heartbeat, but cardiologists didn’t know the underlying cause. Within two days, sequencing detected Timothy syndrome, a rare genetic disorder that affects the heart. Doctors were able to target a medicine to control the heartbeat and then implant a pacemaker. 'That’s the kind of power these tests can provide,' says Henry Garlich, director of health care value solutions and enhanced clinical programs at Blue Shield.
Ultimately, as many as 40,000 newborns each year in the US may be candidates for this whole genome sequencing to find a diagnosis", is a fact outlined in this Wired article here at bit.ly/2x7BbfE
It might begin with genetic sequencing, but gene therapies - like the ones supported by NF2 BioSolutions - are the necessary next step and can be a reality if we stay the course and follow through
Due to the COVID-19 pandemic, we have no choice but to postpone our NF2 Novel Therapies consortium in Boston, which was originally set for April 3. We had 45 NF2 and Gene Therapy researchers and clinicians who were planning to attend from all over the United States and Europe.
Our planned agenda was as follows:
***************************
*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 soonest possible once the pandemic is over. In the meantime we will continue to interact with researchers remotely.
We have two new NF2 BioSolutions ambassadors, Mary Sell in Iowa and Amy Meyer Williams in Kentucky. If you are from these states, do not hesitate to contact them and join the fight
against NF2!
More information on Mary is available at nf2biosolutions.org/about-us/your-local-ambassadors/usa-nbs-team/iowa/ and more information on Amy can be found at nf2biosolutions.org/about-us/your-local-ambassadors/usa-nbs-team/kentucky/
This article is definitely worth reading because it really shows how the future of medicine lies with gene therapies, especially when it comes to treating rare disorders.
Indeed, if we look at the human body as a biological machine - wouldn't it make sense to look at the source code when something's gone wrong?
If DNA is like software, can we just fix the code?
http://www.technologyreview.com
In a race to cure his daughter, a Google programmer enters the world of hyper-personalized drugs.This article here drives home a hard truth; that if we want something great, we're going to have to fight for it - and that includes gene therapy to treat NF2.
We are all stronger when we work together, and together we will #endNF2!
Rare Disease Parents Must Fight Hard for a Miracle - Future of Personal Health
www-futureofpersonalhealth-com.cdn.ampproject.org
Securing funds, advocating research, and raising awareness are critical steps for creating treatments. In this paradigm, rare disease patients are often left to fight for their own miracles.The article here shows that gene therapy for rare disorders isn't some medicine of the future, as governments are beginning to approve its use to treat disorders such as spinal muscular atrophy.
And yes, it begins with spinal muscular atrophy - but it doesn't have to stop there. With your continued support for all the ongoing research that NF2 BioSolutions is backing, gene therapies for NF2 can also become a reality.
Japanese government panel OKs expensive gene therapy for rare disease | The Japan Times
A health ministry panel approved the production and sales of a gene therapy for treating spinal muscular atrophy, a rare incurable disease that destroys muscle function.Two days ago, we asked you for the first three words that came to your mind when you think of NF2. We got many responses, and we put them together in a little graphic here.
All said and done, we want to bring hope to all of you by pushing hard for gene therapy to fight NF2 - and we can't do this without you. So thank you from all of us NF2 BioSolutions volunteers, for it is your continued support that gives us the energy to keep striving every day. Because of you, we won't stop until we #endNF2 together!
A spotlight is now shining on the hard work of our two Florida ambassadors - Lauren Hanrahan and Stephanie Ham - and rightfully so!
Thank you for all that you did in Washington DC, raising awareness about our struggle! May you move from strength to strength as we all work to #EndNF2
The article in the Englewood Sun is available at shorturl.at/mTW05
Below is a Rare Diseases Day post from the two NF2 BioSolutions Ambassadors from Australia, Billie Harvey and Emily Block. These two valiant NF2 fighters would like to share the following message with you;
"Rare Disease Day is February 29, and we need your direct support in our newest endeavour - establishing an NF2 tissue bank. By giving scientists direct access to NF2 tumours, it can help speed research and development of specific drugs and gene therapies to help in our fight for an eventual cure.
The images below show cell slides from two common NF2 tumours - an ependymoma and a schwannoma. Slides like these can be stored in our new NF2 Tissue/Cell Bank to help scientists in targeted, specific NF2 reasearch; something we have never had before!"
So, having said that please head to www.facebook.com/donate/218691646200995/2229635803998369/ and share what you can. Every little helps!
This Saturday is Rare Disease Day, and we at NF2 BioSolutions will be launching the first open-access NF2 Tissue and Cell bank (tumors, cell lines), a project that will drastically speed up research and bring in new research labs to the race in finding a treatment for NF2.
However, we cannot do this without your help - and this is why we will be launching a fundraiser totally focused on raising the funds needed to launch and maintain this bank.
So stay tuned, stay ready for Rare Diseases Day this Saturday!
In the meantime,please do drop a comment below if you have any questions.
