Spinraza Stabilizes Spinal Muscular Atrophy

LOS ANGELES, Calif. (Ivanhoe Newswire) – SMA is a rare degenerative muscle disease. Patients are missing the nerve cells in the spine that tells muscles to move. Until now, doctors could only treat symptoms.

Shawn Stewart was diagnosed with spinal muscular atrophy when he was three.  

Stewart said, “I was walking until the age of 13. I broke my leg at that age. After that, I was no longer able to walk.”

He lost function in his arms, legs, and back. Just breathing became difficult. For more than 45 years, doctors only treated his symptoms. Then in 2016, the FDA approved Spinraza, a drug that increases production of the SMN protein needed for muscle control.

“I’ve had patients that had no arm function that can now move their arms. I’ve had patients that were ventilated, couldn’t breathe on their own, and they can now breathe,” Michael Muhonen, MD, Neurosurgeon, Head of Neurosurgery at Children’s Hospital of Orange County at St. Joseph Hospital said.

The problem was that many SMA patients like Stewart have had spinal fusion. Giving the drug through a spinal tap three times a year was painful and potentially dangerous. So Dr. Muhonen came up with this: “This is a port that allows us to inject the Spinraza through this tube, which has 20 holes in the tip of it. This tube goes into the spinal fluid.”

“It makes the injections so easy. It takes five to ten minutes to get the injection, and I’m able to do that sitting up in my wheelchair,” Dr. Muhonen said.

Stewart says in a year and a half on Spinraza, his breathing and speech have improved. And since Dr. Muhonen’s port simplifies drug delivery, he has high hopes for the future.

A company in Boston is working on improvements to Dr. Muhonen’s port right now. He believes delivering the drug in the upper body and having it drip down through the spine will prove to be more effective than having the drug move up from a lumbar puncture.

Contributors to this news report include: Wendy Chioji, Field Producer; Rusty Reed, Videographer; Cyndy McGrath, Supervising Producer; Dave Harrison, Editor.