Amyotrophic lateral sclerosis (ALS) researchers at The Methodist Hospital in Houston believe that experts trying to find a way to deliver gene therapy may finding their research interesting.
Study leader Dr. Stanley H. Appel says that such "Trojan horse" cells may improve the ability to deliver gene therapy to the brain and spinal cord.
The researcher, who is the co-founder and co-director of the Methodist Neurological Institute at The Methodist Hospital, has revealed that the original intent behind the study was to see whether transplanted bone marrow stem cells in six patients with sporadic ALS would suppress neuroinflammation, and improve the patients' clinical outcomes.
Though the study showed no benefit in fighting the disease, the researchers found that transplanting bone marrow stem cells that were 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," he added.
Appel said that his team focused on bone marrow because of all stem cells, which can offer a readily available source for potential use in the development of therapies, drug delivery, and drug administration in ALS and other neurodegenerative diseases.
The study has been published in the journal Neurology.