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
Here's some exciting news to start the week with! 59-year-old Andy White will be doing a 162-mile coast-to-coast walk that starts on Sept 21 to raise awareness about NF2 and raise funds for NF2 BioSolutions. Why? Well, as Andy says in his own words; "I’m Andy White. I’m 59. More importantly, I’ll be 60 in September 2021. To mark the occasion, I’m going to walk Coast-to-Coast in the UK along the classic Wainwright route to raise funds for research to find a cure for Neurofibromatosis Type 2 (NF2).In the middle of 2019, my amazing eldest daughter Rebecca, now 33, was diagnosed with NF2, which is a rare genetic disease. A faulty NF2 gene causes vestibular schwannomas - benign tumours that occur on the balance and hearing nerves. People with NF2 have an increased risk of other tumours of the nervous system that can lead to significant medical problems, especially if there are multiple tumours in or next to the brain. Whilst there are treatments to slow progression, there is no cure yet. The walk is to raise funds to help find that cure.About 1 in 40,000 people have NF2. Around 50% do not have any family history of the condition. They have a spontaneous mutation in the NF2 gene, which is the case for Rebecca. A goal of treatment is to preserve the person’s hearing for as long as possible. Surgery to remove tumours can be an option, whilst radiation therapy is sometimes used to shrink them. Rebecca has been lucky enough to receive Avastin, a chemotherapy drug used experimentally with NF2 that seems to stop tumour growth. It has kept Rebecca’s tumours stable - although not reduced them - for about a year now”We are grateful to Hudson Benelux who have agreed to be the corporate sponsor for Andy's Coast-to-Coast Walk For NF2. We will be sharing information about them in the next post.If you would like to donate to Andys fundraiser, the link isuk.virginmoneygiving.com/Team/AndyandMikewalkforNF2...
Here's something really interesting - and with this, we at NF2 BioSolutions have some questions. How many of you NF2 fighters out there are taking hypertension meds, and have you noticed a hearing difference? ...
New research led by investigators at Massachusetts General Hospital (MGH) and Massachusetts Eye and Ear indicates that the blood pressure drug losartan may benefit patients with neurofibromatosis type 2 (NF2), a hereditary condition associated with vestibular schwannomas, or noncancerous tumors alon...
It seems that with the march of time, gene therapy is moving forward to win the war against one genetic disorder after another.We at NF2 BioSolutions are committed to seeing that the war against NF2 can be won - but we can't do it without you! It's AADC deficiency today, and it can be NF2 tomorrow - with your help, our scientists are stronger! ...
Below is a message from one of our Ambassadors from Germany, Vanessa Gurguta. She shares the latest about her struggle against NF2;"Hey dear onesNormally I do not speak up when an operation is due as I just don't know whether it would be interesting.However, after well over 20 operations, interventions and Avastin along with other therapies - the time has come and my options are almost at an end. The countless menignomas in and around my brain have somehow managed to wrap my brain over the years in such a way that neither blood nor cerebrospinal fluid can drain away and a shunt no longer works. Basically, there are too many tumors it is difficult to make a sieve for me. As it is, the pressure has reached critical levels and there are days where I'm temporarily blinded and unable to eat anything. Of course, there are also good days and I can make up for it by eating all day. When it comes to my treatment, my doctors have given me two options; the first is to remove the bones of my skull from my hairline up and leave everything free, while the second is to expand my skull's circumference. However, before this I have a procedure set for Monday. This procedure will be to examine the blood vessels in my brain to see where the blockages are exactly and it will be done by running a wire up from my groin to my brain. It will be done under local anesthesia and it is not without risk, as I could bleed to death or get a stroke from this. However, as I'm 27, the risks are not exceedingly high and my chances are good. If you'd like, and as long as I'm able to do so - I'll post updates! Hope everyone has a nice weekend ahead and do stay hydrated in the summer heat!"If you'd like to read more about Vanessa's story, it's shared at bit.ly/3iUbqW9...
Looking for some trendy new apparel while also wanting to support a really good cause? Well, head to nf2biosolutions.creator-spring.com/ for bags, tees, phone cases and more!50% of the purchase price of everything sold here goes towards helping NF2 BioSolutions support the fight against NF2! ...
Hi Fighters! I am Emily Mcdonnah, I want to share a few words about our fundraising experience in the 100 Fighters Challenge. I’m not good at just a few of anything, so here are perhaps too many words on the subject. I have NF2, and for the last nine years, I’ve been openly sharing my journey on a blog. It’s mainly followed by friends, family and acquaintances, but it (and I) have been received with so much love and understanding that I’m not sure I’d be in the place I am today without it. Within a few hours of sharing this fundraiser on my blog, I was over halfway to my $1000 goal. My mind was blown. By the following day, we had cleared the $1000 goal post, and today, a week later, have raised over $3000. I was asked if I could give any tips or motivation to others who have joined or are considering joining the challenge. I know I am uniquely blessed with the incredible support team around me, but I would say this: people want to help. They want to understand. If you or someone you love has NF2, don’t be afraid to let folks in on what that experience means for you. The daily hardships, the uncertainty, pain, appointments, where you draw strength and how you keep fighting! Not a soul with NF2 lacks an incredible and inspiring story. It’s part of the package. Don’t be afraid to own it. If you’re concerned about canvassing for funds during these uncertain times – honestly, every 5 dollars makes a huge impact. If folks can’t swing a financial donation, sharing your story and your campaign are every bit as helpful. You can make your outreach free from pressure in that way. A secondary part of my success has been selling sets of notecards. I’d had them printed a few years ago with my mom’s art on them for just such an occasion as this and they have sold like hotcakes! Everyone uses cards. If you or someone you know is artistically inclined, check out a printing service like Vistaprint and consider giving it a go! You could even design the cards, price them out and only order them after you have committed sales. NF2 can be a very lonely trail to walk, and there is often very little our circle can do to help. This is a thing they can do to help – and so many are more than happy to contribute, we just have to put ourselves out there and ask. Think auctions, bake sales, raffles… And don’t let that $1000 loom over you. If you raise $200, that’s $200 towards this incredible opportunity NF2 Biosolutions wouldn’t have without you! Good luck to you! It feels incredible to be directly impacting such a hope-filled research endeavour, and we all know hope can be hard to come by sometimes. We’ve got this friends, and here’s to brighter days ahead in the NF2 experience. ...
Welcoming Kathrin C. Meyer, PhD in our scientific advisory board. Dr Meyer is working on the NF2 gene addition constructs, research funded by NF2 BioSolutions. Today the gene constructs are being tested at Dr Clapp's lab and Dr Long-Sheng's lab on NF2 mice models.Dr Meyer studied Cellular and Molecular Biology at the Institute of Cell Biology in Berne, Switzerland. Her post-doctoral research was performed in Brian Kaspars laboratory at the Center for Gene Therapy in Columbus Ohio. During that time, Dr. Meyer established a new and fast reprogramming method for in vitro modeling of neurodegenerative diseases using patient skin cells. Moreover, Dr. Meyer developed intrathecal gene therapy programs for several neurodegenerative diseases including Spinal Muscular Atrophy and Batten Disease. Multiple clinical trials that are based on this work are currently ongoing at Nationwide Children’s Hospital. In 2017, Dr. Meyer became a Principal Investigator at Nationwide Children’s Hospital. She is also an Assistant Professor in the Department of Pediatrics at The Ohio State University, Columbus, Ohio.To see our NF2 BioSolutions Advisory board: nf2biosolutions.org/about-us/advisory-board/...
NF2 BioSolutions Chief Scientific Officer (CSO) Edgar Rodríguez-Lebrón, Ph.D. Dr. Rodríguez-Lebrón has over 18 years of experience in the use of AAV as a CNS gene delivery vector. His research has relied on the use of model systems to test novel gene-based therapies that prevent, halt or reverse...
Thank you, everyone! The success of this fundraiser will allow us to start the development of a new non-viral gene delivery approach for NF2. #NF2 #nf2biosolutions ...
We at NF2 BioSolutions would like to thank these four great fighters! All of them completed their $1000 fundraisers; so thank you Terri Rausch, Deian Owen, Jon Kantor and Emily Mcdonnah! ...
We at NF2 BioSolutions want to thank these four amazing NF2 fighters!Currently, we have 76 fighters, but we need 100. Join us in this challenge to raise $1000 each to put an end to NF2 . signup here: nf2biosolutions.org/get-involved/100-nf2-fighters-challenge-how-to-fundraise-online/These donations will go towards our efforts to help accelerate new approaches and techniques to tackle NF2. The newest approach that we want to jumpstart 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. LNP doesn’t have re-dosage limitations like we have with viruses that are being attacked by our immune system. ...
Nissa Novas is a talented local who donates beautiful cakes for our guests to enjoy, but that's only one part of her extraordinary story. She lives with Neurofibromatosis Type 2 (NF2), a disorder that has resulted in the loss of her speech and hearing. She's channeling her experiences into empathy and kindness towards others. In honor of NF2 Awareness Month, read her story on our blog today here: nf2biosolutions.org/news/press-release-and-tv-coverage/nissa-novas-an-nf2-warrior/Nissa is supporting NF2 BioSolutions, an organization doing great work towards a cure for NF2 and Birthday Cakes for Free: MoCo, serving others through baking. ...
Crystals, beautiful crystals for a cause! Head to sites.touchstonecrystal.com/sites/sparklewithjamie/e/1588JRKG and pick up something for yourself, for a loved one or a friend and part of the proceeds will go towards supporting research into gene therapy and bacteriotherapy for NF2 - scientific progress that'll bring people fighting this genetic disorder to the light at the end of the tunnel! ...
We at NF2 BioSolutions want to thank these four amazing NF2 fighters!Currently, we have 76 fighters, but we need 100. Join us in this challenge to raise $1000 each to put an end to NF2 . signup here: nf2biosolutions.org/get-involved/100-nf2-fighters-challenge-how-to-fundraise-online/These donations will go towards our efforts to help accelerate new approaches and techniques to tackle NF2. The newest approach that we want to jumpstart 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. LNP doesn’t have re-dosage limitations like we have with viruses that are being attacked by our immune system. ...
Signup to jumpstart a very important project to stop this horrible rare disorder NF2: 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.Dr.Brenner, a physician scientist at Harvard/MGH, developed an NF2 therapy using an adeno-associated (AAV) viral vector to deliver the ASC suicide-gene to schwannoma cells to induce the death of the tumor cells, leading to shrinkage and sometimes resolution of the tumor. This gene therapy strategy utilizes a single transgene that can: 1) induce cell death in the treated cells, 2) propagate the death signal to adjacent tumor cells, and 3) initiate innate and adaptive immune responses that control future tumor formation. However, the use of a virus to deliver this suicide-gene is challenging since patients develop neutralizing antibodies to the viral vector. Thus, the therapy can only be used once. NF2 is a relentless and lifelong disease and any meaningful therapy requires multiple administrations. Recently, a new gene therapy technology that overcomes this redosing challenge has been developed. It leverages lipid nanoparticles (LNP) used in the mRNA Covid19 vaccine delivery technique to deliver genes. These tiny particles can deliver mRNA or DNA to cells instead of a viral vector. LNPs don’t have the re-dosage limitations of viruses because the body doesn’t recognize them as foreign. 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 are up to the challenge of raising $1000 each.#nf2biosolutionsuk #nf2biosolutions#ineedacure #nf2warrior #immunotherapy #changinglives#endNF2 #NF2awareness #neurofibromatosistype2 #NF2support #nf2 #nf2family #nf2warrior #LetstalkNF2 #nf2charity #nf2treatment #nf2awarenessday ...
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.
