Gene Replacement Therapy for NF2 is starting!
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).
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Article on WebMD about Nicole Henwood, founder of NF2 BioSolutions
Mom Blazes Path to Cure for Son’s Rare Disease
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11 hours ago
We are thrilled to announce that leading NF2 neurosurgeon and NF2 researcher Dr. Michel Kalamarides is now on our scientific advisory board!
Michel Kalamarides is a Professor of Neurosurgery at Pitié-Salpetrière Hôpital (Sorbonne- Université), Paris, France. He specializes in vestibular schwannoma, meningioma and spinal ependymoma surgery.
He heads the French NF2 reference clinical center located at Pitie-Salpetriere Hospital and the French NF2 network of four affiliated NF2- clinics across France. The clinic has been organized to allow NF2 patients to have their annual check-up during a single half-day visit and to be able to perform a fast-track complete assessment for new patients.
De. Kalamarides is also involved with the French NF patients association-Fondation CAP NF (scientific advisory board member).
Within a large INSERM unit- U 1127 at the Brain and Spine Institute (Pitié-Salpetrière), he leads a research group that studies a type of brain tumor common to NF2, meningiomas, using mouse models to analyze the mechanisms of meningioma initiation and progression.
To see our full scientific advisory board, head to nf2biosolutions.org/about-us/advisory-board ...
5 days ago
It is with deep sorrow that I would like to share the news of my beloved friend, a true and fierce NF2 warrior, Maria Kathrina Lopez Yarza or simply Kcat, has just passed away from complications due to NF2. She was the one who established the NF community in the Philippines and is the reason and inspiration why I am here in this fight in the first place. She was the one who encouraged me to pursue science to cure NF2. She is a real fighter and inspiration to all. Please, if you have a moment say a prayer for her and her family.
She made a great effort to be able to say "I need a cure" in the video www.youtube.com/watch?v=AqXb9S7kzYI . For Kcat we need to keep our promise and all work hard together to boost research for NF2.
NF2 BioSolutions Ambassador in Philippines and Scientific Advisory Board member. ...
6 days ago
We are delighted to welcome Dr. Chernoff to NF2 BioSolutions's Scientific Advisory Board. Dr Chernoff is one of the few that has experimented on gene therapy for NF2 in the form of gene editing.
As Chief Scientific Officer, Dr. Chernoff coordinates and charts the future course of research at Fox Chase Cancer Center.
Dr. Chernoff joined the staff in 1991 as an associate member and was promoted to member with tenure in 1996. In 2002 he was promoted to be a senior member in Fox Chase Cancer Center's Basic Science division, the equivalent of a full professor in a university.
A molecular oncologist as well as a board-certified medical oncologist, Dr. Chernoff has a special interest in factors that control cell growth and movement, including oncogenes and anticancer or tumor-suppressor genes, and has made fundamental contributions in this research. His group helped establish a link between the Rac/Pak signaling system and the Merlin protein, and also a connection between fatty acid synthesis and growth of Merlin-deficient Schwann cells.
Dr. Chernoff earned his MD and his PhD in biochemistry in 1984 at Mount Sinai School of Medicine in New York City. He completed his residency in internal medicine at the University Health Center of Pittsburgh and a clinical fellowship in medical oncology at Johns Hopkins Oncology Center. He then held a postdoctoral fellowship in cellular and developmental biology at Harvard University before coming to Fox Chase.
In recognition of his national reputation in molecular oncology and biochemistry, Dr. Chernoff also holds the Stanley P. Reimann Chair in Oncology.
Please check out the current NF2 BioSolutions Scientific Advisory Board. nf2biosolutions.org/about-us/advisory-board/ ...
I would like to share my story about volunteering.
A friend and colleague of mine, Lauren Hanrahan, was diagnosed this past year with NF2. When she had announced her diagnosis to all the staff at our school, my heart felt for her. When she went on to say that if she loses her hearing altogether, she hopes that the last sound she hears is that of her babies - I knew I needed to do something.
On a total whim, I made a video directed to Ellen DeGeneres in some crazy hope of a miracle that she would feel my heartfelt reach of hope for Lauren. I wanted her to showcase this disease to bring it to the forefront and raise awareness and therefore donations to help find a cure.
From that moment, my friends, colleagues, students parents, and my family all began to rally around me in my passion toward this cause. I knew when I got home that day that I emailed the video to Ellen that God had a plan. It literally gave me goosebumps just thinking about it. I knew really great things were to come.
Since then, Lauren and I have had so many people and local business step up to raise funding for NF2 BioSolutions. One idea has led to another and we have interviewed with local new stations, newspapers, and are now working on making NF2 BioSolutions a charity in the January 11th runDisney Marathon Weekend.
I have felt very blessed giving my time and heart to a nonprofit that I know is made up of beautiful people who just simply want a cure for their loved ones. I continued to feel the drive to do more and look forward to seeing what other ways I can contribute to what I believe in.
We are one world and we should all work together as such. That is my hope for this cause.
We have the pleasure of welcoming Lindsay Kantor to our professional advisory board.
Lindsay is a lawyer and partner at Torkin Manes LLP in Toronto, Canada; she received her undergraduate degree from McGill University and her law degree from the University of Toronto.
Having practiced mainly health law for almost 10 years, Lindsay has a keen interest in issues affecting the health care sector.
However, what has drawn Lindsay to support NF2 BioSolutions is her personal experience with NF2 as Lindsay is an NF2 patient herself, along with several other members of her family.
Lindsay believes strongly that connection and community are integral to human happiness and fulfillment. As she moves forward on her NF2 journey, she is committed to ensuring that she and other NF2 patients feel understood and connected to those around them. ...
Fantastic ideas for fundraising from the UK!
A Summer Road party and birthday party donating money instead of presents. Both events raising an impressive £797 that's $1004 on it's way to the lab to explore gene therapy for NF2.
Thank you to everyone that has donated. Please keep these great ideas coming. Having fun is the BEST way to cure NF2! 🥳 ...
***German version below***
My name is Friedrich from Germany, when I was 10 years old, I noticed that the pupil in one of my eyes was larger than the other. This led to the doctors ordering a MRI, and the scans showed that I had two vestibular schwannomas. One tumor was removed a few months later followed by the second vestibular schwannoma, again a few months later.
I was deaf after the surgeries and it changed my life. There was no chance of me getting my hearing back with a cochlear implant, and I had to change my school - which meant that I would lose my friends in the process. Sometimes, I wonder if my hearing would have been preserved if my surgeons were more familiar with NF2.
After this, I had surgeries on my brain and spine at intervals that were roughly two years apart. I survived and got back on my feet quickly.
This was the story of my life until I turned 19, which is when I had a major operation to remove a large tumor on my thoracic spine. It took several months of rehabilitation and daily training before I could walk again with the help of crutches.
After this, I had one tumor-removing operation almost every year and was able to walk until I was 29, which is when a spinal cord tumor put me back in a wheelchair - and I have been a wheelchair user since that point in my life approximately five years ago.
All said and done, I have had about 20 operations since I was ten years old and while the operations have pretty much ruined me physically, I tell myself that life could have gone much worse.
At the end of the day, I do not believe in self-pity and will always try to make the best from every situation.
Mit 10 Jahren ist aufgefallen, dass eine Pupille an den Augen größer ist als die andere. Es wurde kurz darauf ein MRT gemacht und dort wurden die beiden Akkustikneurinome entdeckt. Obwohl ich keinerlei Beschwerden hatte, wurde damals entschieden, beide zu entfernen. Paar Monate später dann die erste Operation und nochmal paar Monate später dann die zweite.
Und schon war ich Taub, ohne Chance darauf, wenigstens mit einem Cochlear Implantant etwas zu hören. Das hat mein Leben komplett verändert und ich musste die Schule wechseln. Wenn ich bei anderen Ärzten gewesen wäre, die sich besser damit auskennen, könnte ich heute noch vielleicht etwas hören.
Danach hatte ich alle zwei Jahre eine Operation am Kopf und am Rücken. Die habe ich gut überstanden und bin schnell wieder auf die Beine gekommen.
Mit 19 wurde ein großer Tumor am Rückenmark entfernt und ich konnte zum ersten mal nicht mehr laufen. Nach einigen Monaten in der Reha und täglichem Training, habe ich es geschafft mit Krücken wieder zu laufen. Die nächsten Jahre hatte ich dann fast jedes Jahr eine Operation.
Mit 29 Jahren wurde dann erneut ein Tumor am Rückenmark entfernt und dies brachte mich erneut in den Rollstuhl. Das ist nun 5 Jahre her, seitdem bin ich auf dauerhaft auf den Rollstuhl angewiesen.
Insgesamt hatte ich seit meinem 10. Lebensjahr ungefähr 20 Operationen. Auf der einen Seite hat es ziemlich viel kaputt gemacht, auf der anderen Seite hätte es auch schlimmer werden können.
Ich bin nicht wehleidig, sondern mache immer das Beste aus der jeweiligen Situation. ...
3 weeks ago
Something is definitely happening on the gene therapy front!! Thanks to your support, NF2 BioSolutions is putting the focus of gene therapy labs on NF2, this is our mission!
(IND application is filed usually after encouraging results in pre-clinical research). ...
NF2 BioSolutions is feeling excited.
3 weeks ago