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Approximately one in thirty thousand individuals across the globe will be diagnosed with Neurofibromatosis Type 2 (NF2) in their lifetime. A defect in the NF2 gene causes this rare disease that results in tumor growth throughout the nervous system. Over time, these tumors severely impact the quality of life of affected individuals as they can often lead to hearing loss, severe balance problems, facial paralysis, blindness, and even death if the brain stem is compressed. Today there is no cure for NF2. Our mission is to jumpstart and accelerate novel therapies that can enable us to find one and find it as soon as possible.
Patients with any rare disorder face a harsh reality. The disorder they suffer from is competing for the attention of scarce resources. One of the biggest challenges is that pharmaceutical companies want to focus on research that can be monetized soon. On the other hand, researchers need to be interested in the cause and need funding to do the work. Once diagnosed, patients are not on a road to recovery but rather undergo treatments that only try to reduce the effects of the disease. For NF2 patients, their only choice is to take cancer drugs that come with many unbearable side effects or undergo invasive surgeries that severe nerves and radiotherapy. There is no long-term solution, and patients and their families must become the driving forces behind any progress.
However, there was a breakthrough. Gene therapy—the process by which defective or missing genes are replaced with normal ones— clinical trials were approved by the FDA for persons with SCID. As a result, more and more emerging therapies were researched for rare genetic diseases and progress began to be made. There was hope. Could gene therapy be the path to a cure for NF2 too?
In 2018, NF2 BioSolutions was founded by Dr. Nicole Henwood, the mother of a young son diagnosed with NF2, to accelerate this opportunity to cure NF2. We are the first organization to explore gene therapy—and now cell and immunotherapies too—to fight NF2. While there was new and exciting hope, NF2 BioSolutions knew it needed to raise funds for pre-clinical research as pharmaceutical companies would want to see promising results before becoming involved and supporting clinical trials. Our first step was to bring together the experts.
In 2019, NF2 BioSolutions hosted the NF2 Gene Therapy Consortium in Boston. Experts from both academia and industry gathered to discuss and determine the next steps in research. This important event connected the minds we needed and set the direction for our work.
Today, NF2 BioSolutions is supporting approximately 15 different novel therapy research projects. Every day NF2 patients are suffering without a cure. Because we do not yet know which approach is going to work, we seek to have the most impact through simultaneous exploration. As a nonprofit, patient-driven organization, NF2 BioSolutions has the ability to raise and focus its funds on the research we believe has the most potential, keep researchers connected and focused on a cure for NF2, and share the tools and data needed for impactful results.
How We Do It
Scientific research is a long and complicated process. NF2 BioSolutions funds a variety of studies at world-renowned institutions simultaneously—including cross-lab collaborations—and provides opportunities for researchers to share results. The projects target different therapy approaches and address multiple patient goals, including destroying existing tumors more effectively with fewer side-effect and preventing the formation of new tumors. Our sponsored research projects also support the development of needed research tools. NF2 BioSolutions also has set up its own open-access biobank to accelerate gene therapy research for NF2.
The research funded to date has yielded promising results in working toward a cure for NF2 and in the treatment of rare diseases more broadly. We are making progress in the development of key tools and resources including a biobank for NF2 tissues, cultures, and genetic data, 3D imagery and volumetric analysis of NF2 tumors, and the generation of NF2-specific mice models species.
“I feel very hopeful that within five years, we will have treatments that are far more effective than what we have now.” – Kaleb Yohay, M.D., Ph. D, Associate Professor of Neurology and Pediatrics (Clinical), New York University
How You Can Help
NF2 BioSolutions is led by patients or parents, those that are facing NF2 themselves or have a loved one affected. We are passionate about our work as it is part of our everyday lives, and all the funds we raise go to furthering our mission. We have volunteers and supporters around the globe who have helped us raise over $2,000,000 to date to support this work, but this is not enough for moving forward all the research projects.
Whether you have a few hours a year or each month, there are many ways to become involved in NF2 BioSolutions and help find a cure for this debilitating disease. Visit our Take Action page to see how you can help find a cure. Visit our Donate page if you would like to support research with a tax-deductible financial gift.
The National Organization for Rare Disorders (NORD) is pleased to announce that NORD’s Board of Directors has approved NF2 BioSolutions to become a Platinum NORD member organization