In the United States alone, neurological diseases are estimated to be diagnosed in about 1.2 million people above the age of 18 annually1.
Spanning from Alzheimer’s Disease to different movement disorders such as Parkinson’s; the list of brain disorders, being vast, and seemingly never ending, makes it terribly hard for scientists and doctors alike to find their footing and make sustaining impacts.
In recent years, being in the age of technological advancement, researchers have been exploring a new treatment method which may just give us the footing we need.
Through the delivery of gene therapy directly to the brain, scientists have assisted four year old Rylae-Ann Poulin live the life many of us take for granted. At one year old, suffering from a disorder interfering with the communication between cells in her nervous system, Poulin was not even able to lift her head up. Now, years later, post-treatment, Poulin is able to walk, run, swim, read and even ride horses.
The disease Rylae-Ann possesses is AADC deficiency, which is caused by genetic mutations disrupting the synthesis of an enzyme that plays an important role in the production of the body's chemical messengers. During Poulin’s marginally invasive surgery, doctors administered a harmless virus carrying a functioning version of the gene. This direct gene delivery allows the brain cells to start making the needed neurotransmitters.
Research is currently being done with respect to other neurodegenerative diseases such as Alzheimer’s, Parkinson’s and Huntington’s. However, these more complex disorders are much tougher to treat as they are caused by a greater number of mutations.
As Jill Morris, program director with the National Institute of Neurological Disorders and Stroke mentioned, “when you’re correcting one gene, you know exactly where to target it”2.
Though there is still a long way to go, this success has put other researchers on what may be the right path to curing neurological disorders completely.
Sources
https://www.pbs.org/newshour/science/new-gene-therapy-delivers-treatment-directly-to-brain
https://www.caregiver.org/resource/incidence-and-prevalence-major-causes-brain-impairment/