We want to thank you from the bottom of our hearts for your kind and generous donation to NF2 Biosolutions. Fighting NF2 is near and dear to our hearts, and we want to share with you some exciting news thus far.
Here at NF2 BioSolutions, our goal is to find a meaningful treatment or cure for NF2 through the use of gene therapy and other cutting-edge techniques. Your generous donation will go a long way toward helping us end NF2. Here is a quick summary of our three current initiatives on the research front.
Thanks to the March 2019 NF2 Gene Therapy Consortium in Boston, where we invited 45 researchers (roughly half from NF2 clinic and research side and half from the best known gene therapy labs and biotech companies), we successfully jump started three gene therapy labs and they have begun working on NF2 and initiative to support these labs. These labs each have a different approach, as described below.
- Gene Therapy Center at Nationwide Children’s Hospital: The Meyer lab will use a gene addition approach (adding a healthy NF2 gene) to potentially stop or slow tumor growth and help prevent new tumors from forming. We signed a sponsored research agreement where we fund two lab researchers for two years with payments based on milestones of progress.
- The Mueller Lab at the University of Massachusetts: They will use a silence and replace gene therapy approach, which is characterized by first shutting down the mutated (bad) NF2 gene and then adding in a healthy copy. They have been using this platform with success in other genetic diseases. NF2 BioSolutions will sponsor the NF2 vector design and manufacturing and will fund the NF2 mouse model work in external labs once the vector is ready. We are planning to collaborate with other NF organizations for funding for funding additional stages of this project.
- Suicide Gene: Dr. Brenner’s lab, Mass. General Hospital/Harvard Medical School(Boston). NF2 BioSolutions is supporting the lab and research of Dr. Gary Brenner, which aims to accelerate the translation of their gene therapy to clinical trials. Experiments in NF2 mice models have demonstrated the efficacy of the therapy in destroying schwannomas. The team led by Dr. Brenner has designed a gene therapy strategy that uses an adeno-associated (AAV) viral vector that expresses the “cell death” gene ASC to attack the mutated Schwann cells that cause the tumors. Negotiations are in progress to license this technology to a biotech company for development of the therapy and advancement to clinical trials.
- Bacteriology-immunotherapy: Dr. Brenner’s lab & Dr. Mekalanos’s lab, Mass. General Hospital/ Harvard Medical School (Boston). NF2 BioSolutions is also supporting Dr. Gary Brenner’s lab at Massachusetts General Hospital for the development of bacteriotherapy to treat NF2 associated tumors.. This new approach utilizes a cell-based treatment strategy in which live attenuated – or in other words, less toxic – bacteria are directly injected into tumors. In this case, preclinical data show that following injection of these therapies into NF2 schwannomas the tumors shrink and in some cases, completely resolve. Currently, Dr. Brenner is collaborating with the lab of Dr. John Mekalanos to develop other genetically modified versions of the bacterial product “armed” with genes that enhance tumor cell death and the development of anti-tumor adaptive immunity. The next step is to test new bacterial products in Dr. Brenner’s schwannoma mouse models. Additionally, a NIH (National Institute of Health) STTR (Small Business Technology Transfer) grant has been awarded to Dr. Brenner and an industry collaborator to accelerate this project, thanks to the work funded by NF2 BioSolutions. We anticipate FDA Fast Track designation, which will help speed up and facilitate moving the product to the clinic.
- NF2 BioBank: Dr. Adam Resnick’s Lab, CBTN at the Children’s Hospital of Philadelphia (CHOP). Having access to NF2 tumors and NF2 cell lines is a key requirement for labs to progress towards a treatment. NF2 BioSolutions united researchers/clinicians and launched a NF2 Tissue/Cell bank in mid-2020. NF2 BioSolutions is getting NF2 surgeons who remove tumors and research labs that need tumors to participate and benefit from a large number of NF2 tissues/cells. To date, more than 30 patients have consented to donate their NF2 tumor and/or blood samples. Our goal is to reach 100 patients by mid-2021. More than 15 agreements have been signed between clinics, hospitals and our NF2 Biobank allowing the transfer of tissue samples and de-identified patient data. Biobank head Dr Adam Resnisk said; “NF2 BioSolutions is a real leader across the rare disease landscape in how we address the challenges of drug discovery and accelerate impact for patients”.Here is a webinar where Dr Resnick explained the NF2 Bionbank: https://youtu.be/-RhtOyEnakg
- Ependymoma mice model: Dr Kalamarides lab, Hospital Necker (Paris, France). Spinal ependymomas are the third most frequent tumor type in NF2. They are located within the spinal cord, predominantly at the cervical level. The growth of ependymomas is unpredictable and sometimes very fast and lead to pain, paralysis and even death. The goal of this new research program at the laboratory of Professor Michel Kalamarides is to develop a new mouse model for ependymoma that recapitulates the features of human NF2 spinal ependymomas; namely the same histological aspect, same cell of origin, same growth patterns. This will hopefully allow the preclinical evaluation of therapeutic interventions such as medical treatment or gene therapy approaches as advocated by NF2 Biosolutions. This model will be the first mouse model of a NF2 spinal cord tumor. NF2 BioSolutions is currently funding this very important effort that we will make available to all NF2 research labs; it is our hope that this will help them tackle this destructive type of tumor.
On behalf of the entire team and all those affected by NF2 we thank you again for your generosity. For up to date information go to Facebook.com/NF2biosolutions and our website homepage to sign up for our newsletter. Please continue to help share and spread the word about NF2 and NF2 BioSolutions.
For any questions, do not hesitate to contact Gilles Atlan at email@example.com