Amyotrophic lateral sclerosis (ALS) researchers at The Methodist Hospital in Houston have shown that transplanted bone marrow stem cells can attach themselves to injured areas in the brain or spinal cord, possibly providing a way to deliver future gene therapy.
According to Dr. Stanley H. Appel’s (pictured), study, these "Trojan horse" cells may improve the ability to deliver gene therapy to the brain and spinal cord.
The original intent of this study focused on whether transplanted bone marrow stem cells in six patients with sporadic ALS would suppress neuroinflammation and improve the patients’ clinical outcomes, said Appel, co-founder and co-director of the Methodist Neurological Institute at The Methodist Hospital. While the results showed no benefit in fighting the disease, Appel’s research team found that transplanting bone marrow stem cells that are closely matched to the patients’ own bone marrow allowed for a significant percentage of those cells to travel to and reside in the brain or spinal cord. However, it is clear from the study that unless the bone marrow stem cells are engineered to secrete neuroprotective factors, such transplants are not likely to be beneficial in human ALS.
“Our courageous ALS patients committed themselves to helping search for meaningful therapies for this devastating disease,” said Appel, lead author and chairman of Neurology at Methodist. “With their help, we’ve shown that these transplanted stem cells can potentially be used to deliver future drugs. These findings open the window in terms of where we can go next.”
Appel and his research team have focused on bone marrow because of all stem cells, they offer a readily available source for potential use in the development of therapies, drug delivery, and drug administration in ALS and other neurodegenerative diseases.
Commonly known as Lou Gehrig’s disease, ALS is a progressive neurodegenerative disease characterized by degeneration of the upper and lower motor neurons in the brain and spinal cord, which stimulate skeletal muscle movement. As more motor neurons die, muscle weakness becomes progressively worse. Sporadic (non-inherited) ALS affects 90 percent of the ALS population, and is representative of the patients at Methodist’s MDA/ALS Clinic, the first and one of the largest multi-disciplinary ALS clinics in the nation.
Illustration: The Methodist Hospital.
The Methodist Hospital News Release (10/13/08)
Medical News Today (10/14/08)
Abstract (Neurology; 71, 1326-1334 (10/08))