NF2

• Neurofibromatosis Type II (NF2) is a genetic disorder that is caused by a defective gene that is missing a protein that is needed to fight off tumors. NF2 is usually characterized by the growth of bi-lateral tumors on the vestibular (balance) nerves and along the auditory nerves.  These tumors are located right at the brain stem and therefore as they grow they impinge on surrounding nerves that affect many bodily functions. Affected individuals frequently have numerous tumors along their spine as well as on the nerves behind the eyes.
• NF2 occurs in 1 of every 30,000 people. 50% of the cases are hereditary and each child born of an affected parent has a 50/50 chance of inheriting the disorder. The remaining cases are the result of spontaneous mutations.

Current treatments

Current treatment options include surgery, chemo therapy and radiation.  History has shown us that tumors frequently grow back following surgery. The surgery itself (often 12 hours or more) poses many risks aside from facial paralysis and other issues. There is a limit to how much radiation the brain stem can withstand and is not recommended for children as their brains are still maturing. All of these treatments are designed to try to “control” or slowdown tumor growth. None of them will cure the root cause of NF2.

Gene therapy

• The goal of gene therapy is synthesize the correct copy of the defective NF2 gene in the laboratory, insert it into the body where it will knock out the defective gene and put the healthy copy in its place, thus curing the disorder. Treated people will know that from then on, their NF2 will not cause any further damage. Can you imagine what this means to a newly diagnosed child? It will be a life changer!
• Gene therapy is approaching or has approached the clinical trial stage  of disorders such as ADA-SCID (also known as  Bubble Boy’s Disease), CGD which affects one’s ability to fight off infection, Hemophilia, Leber’s Congenital Blindness, Cancer, Mesothelioma, Parkinson’s, Huntington’s and other disorders. Being a comparatively rare disorder, NF2 has not attracted the financial resources and scientific expertise to find a cure for NF2

Goals of this project

• To finance a NF2 gene therapy Pilot Pre-clinical study at Nationwide Children’s Hospital in Columbus Ohio, one of the foremost gene therapy labs.
• To finance research at Dr. Gary Brenner’s lab at the Massachusetts General Hospital.
• To seek out other promising gene therapy labs and provide financing for specific research projects.

The results of the researches will be publicly available.

Myriad Canada is working with NF2 Biosolutions on this scientific project. NF2 Biosolutions is a US registered volunteer charitable organization, which has brought together a group of the foremost neurologists, neurosurgeons, geneticists and researchers to examine the ways in which gene therapy can be an effective cure for NF2.  Their mission is to identify and finance leading gene therapy approaches that were discussed at the consortium in Boston in March 2019. Because NF2 is a mono-genetic disorder (caused by a single defective gene), it has been identified as an ideal candidate for gene therapy.

Your donation will bring us one step closer to making the cure a reality and changing peoples’ lives forever.