We have so much exciting news to catch you up on since our March 2019 Team Leah Newsletter was issued!
As the newsletter was shipped off to the printer, John and Linda Manth boarded a plane for Boston, MA to attend the inaugural NF2 Gene Therapy Consortium, with other representatives from lead sponsor, NF2 BioSolutions. John Manth sits on the board of the Boston based non-profit, founded in 2018 by Dr. Nicole Henwood, mother of 7 year old A.J., who has NF2. The Consortium brought together over 40 gene therapy experts from academia and biotech, NF2 researchers and clinicians from across the USA and the United Kingdom with an aim to broaden knowledge, foster collaboration and set a vision for gene therapy as a realistic method for treating and maybe even curing NF2 tumors.
"Until recently, gene therapy was more of a theoretical dream than a reality. However, due to rapid medical advances, gene therapy is now becoming a possibility for many medical conditions and genetic disorders. We seek to support and fund research and clinical trials for NF2, with the goal of bringing gene therapy to those patients with NF2 who desperately need a treatment outside of surgery and radiation".
It's been only 4 short months since the Consortium and NF2 BioSolutions has funded THREE NF2 gene replacement therapy initiatives:
NF2 Gene Therapy Pilot Pre-Clinical Study
NF2 BioSolutions signed a contract in July 2019 to partially fund an NF2 Gene Therapy Pilot Pre-Clinical Study at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (NCH) by Dr. Kathrin Meyer, who is serving as the Principal Investigator. NF2 BioSolutions is sponsoring two researchers to work along with Dr. Meyer, and they will be 100% dedicated to NF2 research in order to facilitate this project in the most timely manner possible. (read more)
Mueller Lab for Gene Therapy
Having attended NF2 BioSolutions’s NF2 Gene Therapy Consortium on March 29th, Dr. Chris Mueller was moved by the impact of NF2 on Leah and other NF2 patients and families, as presented through their stories and personal accounts of living with NF2. Dr. Mueller decided to run a gene therapy pilot study to identify if his lab’s gene therapy platform could be a good candidate for tackling the NF2 mutation. Dr Mueller, assisted by Dr. Karin Meijboom, will be financing the development of the gene therapy NF2 vector for the pilot. NF2 BioSolutions will facilitate the collaboration between the NF2 experts and the Mueller’s lab. (read more)
Schwannoma Suicide Gene Therapy Approach – MGH – Dr. Brenner Lab
NF2 BioSolutions is supporting Dr. Gary Brenner’s lab to move towards a toxicology study prior to submitting an IND (investigational new drug) application to the FDA.
Schwannomas are benign tumors composed of Schwann-lineage cells that normally form a protective sheath around nerves in the cranium (skull) and peripheral nervous system. Most people with schwannoma-associated disease have multiple tumors which generally first appear during adolescence and continue to develop throughout life. For Leah, these types of tumors were first diagnosed via MRI when she was just 7 years old. Schwannomas are associated with 3 distinct diseases, one of which is NF2. The focus of Dr. Brenner's lab is to develop a gene therapy that specifically targets schwannomas, which are composed entirely of Schwann-lineage tumor cells. (read more)
Gene replacement therapy is offering new hope to Leah, our family and NF2 families around the world!