For the past few years, I have had a front row seat watching a world-class team at its very best here in Columbus. No, I have not been in Ohio Stadium, but in my office chair at the Research Institute at Nationwide Children’s Hospital.
Last week, the prestigious New England Journal of Medicine published the team’s promising results of a clinical trial in infants with a fatal genetic disorder called spinal muscular atrophy. The research was performed in our Center for Gene Therapy.
SMA, in its most-severe form, is a devastating disease. More than 50 percent of affected babies die by age 2 from severe muscle weakness. They cannot hold their heads up, roll over, sit up or walk.
The clinical trial found a single intravenous dose of a healthy SMA gene extends the lives of infants with SMA beyond what was previously believed possible. Two of 15 treated infants have even learned to walk.
Although not directly involved, I have watched with great interest as this research played out over many years. What began as parallel, unrelated research efforts converged over time into a remarkable discovery engine with a laser-like focus on an SMA treatment.
The story began more than two decades ago with foundational research on SMA at Ohio State University. Separately, in the early 1990s, scientists at Nationwide Children’s were studying unique adeno-associated viral (AAV) vectors — Trojan horses if you will — capable of carrying corrective gene payloads.
Thirteen years ago, a talented Ph.D. neuroscientist, Dr. Brian Kaspar, was recruited from southern California to join the Center for Gene Therapy. Early on, he found that a specific serotype of AAV — AAV9 — given by vein is exuberantly taken into the same spinal cord cells that are defective in SMA.
At the same time, prominent neurologist Dr. Jerry Mendell relocated from the main Ohio State campus to Nationwide Children’s to focus on developing gene therapies for children with neuromuscular disease. He convinced hospital leaders to construct a specialized facility to manufacture AAV for human use. Text full:http://www.dispatch.com/news/20171105/pediatric-research-teamwork-fuels-breakthrough-in-fight-against-fatal-genetic-disorder
Last week, the prestigious New England Journal of Medicine published the team’s promising results of a clinical trial in infants with a fatal genetic disorder called spinal muscular atrophy. The research was performed in our Center for Gene Therapy.
SMA, in its most-severe form, is a devastating disease. More than 50 percent of affected babies die by age 2 from severe muscle weakness. They cannot hold their heads up, roll over, sit up or walk.
The clinical trial found a single intravenous dose of a healthy SMA gene extends the lives of infants with SMA beyond what was previously believed possible. Two of 15 treated infants have even learned to walk.
Although not directly involved, I have watched with great interest as this research played out over many years. What began as parallel, unrelated research efforts converged over time into a remarkable discovery engine with a laser-like focus on an SMA treatment.
The story began more than two decades ago with foundational research on SMA at Ohio State University. Separately, in the early 1990s, scientists at Nationwide Children’s were studying unique adeno-associated viral (AAV) vectors — Trojan horses if you will — capable of carrying corrective gene payloads.
Thirteen years ago, a talented Ph.D. neuroscientist, Dr. Brian Kaspar, was recruited from southern California to join the Center for Gene Therapy. Early on, he found that a specific serotype of AAV — AAV9 — given by vein is exuberantly taken into the same spinal cord cells that are defective in SMA.
At the same time, prominent neurologist Dr. Jerry Mendell relocated from the main Ohio State campus to Nationwide Children’s to focus on developing gene therapies for children with neuromuscular disease. He convinced hospital leaders to construct a specialized facility to manufacture AAV for human use. Text full:http://www.dispatch.com/news/20171105/pediatric-research-teamwork-fuels-breakthrough-in-fight-against-fatal-genetic-disorder
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