About the organization
Fundraising and becoming an active member
Gene therapy and its role in NF2
About the organization:
Question: How was NF2BioSolutions born?
Answer: We were born because we were needed. There was no group dedicated solely to NF2 in the US and certainly no other group looking at gene therapy. The need was there and we came into the NF2 community at the right time.
Question : What are the short-term objectives of NBS?
Answer: Our short term objective is to raise enough money to start a pre-clinical program to study how gene replacement therapy will work to slow or halt disease progression in NF2. Once we get something that works in cells and animal models then we can head toward clinical trials.
Question: How can patients and their community help achieve that goal?
Answer: The most important thing patients can do is educate themselves and their friends and family about the power of gene therapy. We have a page dedicated to gene therapy successes in other diseases on our website, this is good place to start. Then they need to become part of the solution and actively support by volunteering and donations, because no one will do it for us.
Question: Are the members of the organization being paid by NF2 BioSolutions or any other organization for managing NF2 BioSolutions ?
Answer: No, nobody at NF2 BioSolutions gets paid. We are an all volunteer organization, and are very motivated to find a cure since most of us are directly impacted by NF2 (patients, family members and close friends).
About fundraising and become an active member:
Question: How is it possible for NF2 BioSolutions to raise the millions of dollars needed to fund gene therapy trials?
Answer: Many other rare disease foundations, some even rarer than NF2, successfully raised millions of dollars and financed dedicated research into their rare disease. NF2 BioSolutions is in contact with these other rare disease organizations and will follow a similar approach, having learned from their experiences. For example, if we actively engage 200 NF2 families, each raising an average $10,000 per year, we reach $2 million and this is without corporate support or investors. That’s why we need you to be part of this effort of finding a gene therapy cure for NF2.
Question: If I want to be an active member and help raise money for NF2 BioSolutions, how do I get involved?
Answer: We will guide and support you and explain the different ways you can reach your fundraising goals by using social media, and workplace, personal, and school networks. Please contact Jill at jill@nf2biosolutions.org
Question: What is the next short-term goal for NF2 BioSolutions?
Answer: It is to organize a first meeting in March 2019 that will bring together gene therapy and NF2 experts, among others, to get their opinion and commitment to focus on gene therapy research. To fund this meeting, our goal is to raise $50k.
Question: If I am an active member, what about my privacy?
Answer: You can be an active member and still keep your privacy and/or be anonymous. We always seek approval before any names or personal information are published.
Gene therapy and its role in NF2:
Question : How would you explain what gene therapy is to someone who does not know anything about it?
Answer: First I would send them to our website because we have a lot of information about this on the site. But in short, gene therapy is any therapy that involves genes/DNA. If the problem is caused by a missing gene, there are ways of replacing that missing gene, using something called a vector, to carry the missing gene into the cells. If there is a mutation, or mistake, there are ways to fix or edit the gene, for example with a product like CRISPR-cas9. There are many other ways to use gene therapy but these two are the most common.
Question : Gene therapy, is it a future therapy or something that can already accessed?
Answer: It’s no longer the future, it’s happening now. In 2017 the FDA approved the very first gene therapy to replace a nonfunctional gene in a rare disease that causes blindness. Many other applications are already waiting to be reviewed by the FDA and thousands of clinical trials are taking place to treat genetic disorders.
Question : If a gene therapy cure or treatment is found, will I or my loved one have access to it?
Answer: We are dedicated to helping all NF2 patients access any treatments developed through the programs at NF2 BioSolutions. When such a time comes, we pledge to direct our resources to getting treatments to those who need them.