While no cure exists for ALS, researchers are finding ways to use unmodified human bone marrow CD34+ cells for spinal restoration.
The renowned physicist Stephen Hawking influenced how countless scientists worldwide view the universe. He also demonstrated his remarkable ability to survive amyotrophic lateral sclerosis (ALS) for 55 years. He is thought to be the longest survivor of ALS. According to the ALS Association, 50% of those with ALS survive for about 3 years after diagnosis, 20% survive for 5 or more years, and 10% survive for 10 years or more. There is no cure for ALS, and therapies that can slow or delay progression, decrease symptoms, and repair damaged spinal-cord tissue are continuously sought.
Research has suggested that dysfunction of the blood brain and blood spinal-cord barriers (BBB and BSCB) is an important factor in the progression of ALS. Inflammatory and other molecules can pass through the damaged barriers and cause motor neuron degeneration. Scientists from the University of South Florida have conducted a number of studies in mice that show how transplantation of human bone marrow cells can repair the BSCB and delay disease progression. One study showed that injection of mouse models of ALS with human bone marrow CD34+ cells led to BSCB restorative properties.
The transplanted unmodified human bone marrow CD34+ cells differentiated into endothelial cells and grafted into spinal cord capillaries. There was decreased permeability of spinal cord capillaries and even some restoration of myelin. Although CD34+ cell treatment led to a level of capillary engraftment and beneficial effects on motor function, significant capillary damage was still evident weeks after treatment. Therefore, additional studies were conducted using endothelial progenitor cells to determine if this would improve BSCB restoration.
Transplantation of human bone marrow-derived endothelial progenitor cells, provided enhanced spinal cord and brain capillary engraftment, decreased capillary leakage, increased survival of spinal cord motor neurons, and improved behavioral outcome disease measures in the mice. Using the restricted-lineage cells provided, improved BSCB restoration while improving nerve function. These findings can provide a promising treatment option to repair the BBB and BSCB, improve motor neuron function, and delay disease progression in patients with ALS.
Reference: Human Bone Marrow Cells Hold Promise as ALS Therapy — Mouse Study. (2019). ALS News Today. Retrieved 21 May 2019, from https://alsnewstoday.com/2019/04/05/human-bone-marrow-cells-hold-promise-als-therapy-mouse-study/