Routing gene therapy directly into brain

Researchers from Boston Children's Cancer and Blood Disorders Center have found a revolutionary way to transplant hematopoietic stem cells (HSCs) directly to a patient's brain. Traditionally, HSCs were thought to be transplanted only through an intravenous line into the bloodstream. This is problematic because the stem cells must mature and naturally circulate into the brain, which can be a slow process. In a mouse model, Dr. Alessandra Biffi of Boston Children's Hospital and her team found a way to deliver stem cells directly to the brain in a mouse model. The study involved treatment of mice that had a lysosomal storage disorder, which is enzyme imbalance that leads to a dangerous buildup of lipids, carbohydrates, and other materials that severely impacts the central nervous system. The researchers used the mice to transplant the HSCs directly to the ventricles of the brain and termed the method intracerebroventricular delivery. This approach enabled the stem cells to release signaling cytokines that counteract neuroinflammation caused by the lysosomal storage disorder. They found that the transplanted HSCs had not migrated to other parts of the central nervous system, which showed potential to create a chimera, a separate genetic profile within an organism. Researchers implicated that this capability to create chimeras within the brain could further treat neurodegenerative diseases such as Parkinson's, Alzheimer's, and ALS.
This innovative discovery has taken stem cell therapy to another level by optimizing its approach to be more effective. Direct delivery to the central nervous system is known to have a prompt sequelae. This could treat devastating and time sensitive diseases of the brain such as Huntington's disease and malignant brain tumors. The novel approach of intracerebroventricular delivery advances stem cell therapy and opens new windows for treating an array of medical conditions that affect the brain.
https://www.sciencedaily.com/releases/2017/12/171206174240.htm
http://advances.sciencemag.org/content/3/12/e1701211