Research update

Winter 2020  

Gene Addition: Dr. Meyer’s lab, Abigail Wexner Research Institute, Nationwide Children’s Hospital (Columbus,OH)

NF2 BioSolutions sponsored the pre-clinical NF2 gene addition program at Dr. Kathrin Meyer’s lab in 2019, a top gene therapy laboratory located at Nationwide Children’s Hospital in Columbus, Ohio. The team consists of gene therapy researcher Dr. Kathrin Meyer, NF2 researcher Dr. Long Sheng Chang, post-doctoral associate Dr. Oksana Pavlyuk, and research assistant Krizelle Alcantara, who is also a member of the NF2 BioSolutions scientific advisory board. 

Several viral constructs have been designed and manufactured to insert a healthy copy of the NF2 gene into the cells of NF2 patients.  By adding a healthy copy of the NF2 gene, we will restore the production of the NF2 protein product Merlin; if absent, the result is the growth of tumors in the nervous system. The next step is to simultaneously test these vectors in two labs specializing in NF2 mouse models in the next few weeks.

Gene Replacement: Dr. Flotte’s lab, University of Massachusetts (Worcester, Massachusetts)

NF2 BioSolutions introduced the NF2 disease challenge to Dr. Mueller who decided to launch a pre-clinical NF2 gene replacement research approach. University Dean Dr. Terry Flotte took over the project, where postdoc Dr. Katharina Meijboom is designing the vectors (virus) that will silence the mutated NF2 genes and add a healthy copy. The vectors are currently being produced for testing on NF2 mice. This research program is partially funded by NF2 BioSolutions, NF NorthEast, NF Michigan.

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. 

Here is a webinar where Dr Brenner and Dr Mekalanos explained their research:

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 Resnick 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 BioBank:

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.

These achievements were done thanks to the donations to NF2 BioSolutions. 
In order to have this research moving faster, please donate here

NF2 BioSolutions, is a patient volunteer-run, nonprofit organization that effects change through patient advocacy by bringing the smartest minds in various research fields together to find an NF2 treatment or cure.


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