October 26, 2009

Nerve transplants possible treatment for ALS-related respiratory failure

Because the inability to breathe is an ultimate cause of death of patients with ALS, Johns Hopkins scientists are targeting the diaphragm as a therapeutic target by transplanting stem cells directly into rats’ cervical spinal cords, precisely where the motor neurons that control this respiratory muscle are located.

“We are transplanting stem cells that will become astrocytes because these cells play an important role in maintaining the health of motor neurons,” said Angelo Lepore, a postdoctoral fellow in the lab of Nicholas Maragakis, an associate professor of neurology at the School of Medicine.

The team showed that transplanting rat-derived stem cells into the rodents’ cervical spinal cords helped slow down the decline of diaphragm function and therefore extended survival in the rat model of ALS. Their findings show that a targeted delivery of stem cells to the cervical spinal cord is a promising therapeutic strategy because even the partial rescue of motor neurons resulted in a decreased loss of respiratory function.

“I think that we first need to examine the potential of human-derived cells following transplantation before we can say whether this strategy will work in human patients,” Lepore said. “While the initial results are promising, a number of key experiments must be conducted before this therapy is translated to the clinic, including testing the efficacy of a similar class of human stem cells.”

The team’s work was presented at the annual meeting of the Society for Neuroscience, held Oct. 17 to 21 in Chicago.

Related Web sites

Angelo Lepore and his work on ALS: www.hopkinsmedicine.org/stem_cell_research/coaxing_cells/at_the_bench.html