Nearly $1 Million Drug Helps Temple Student Battle Deadly Disease
PHILADELPHIA (CBS) — There's new hope for people with a rare and deadly genetic disease. It costs $750,000 a year, but it is being covered by most insurance plans.
There's been a lot of controversy about the price of this drug as it was developed to treat a very rare disease that usually strikes babies. Now, it's also being used to treat adults.
Eric Murphy, 24, is feeling a little stronger since he's been getting a new drug to treat spinal muscular atrophy.
"I've noticed my grip has gotten better," he said, explaining that he has seen improvements.
Murphy's been in a wheelchair for almost a decade.
As a little boy, he sometimes had trouble walking. He was eventually diagnosed with SMA, a genetic disease that causes muscles to weaken, and it's eventually fatal.
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"They told my mom; my mom started crying and my dad cried too. It was a really dark day," Murphy recalled.
Spinraza, the first drug approved to treat SMA, works on the underlying genetic cause, and enables a backup gene to produce more of a missing protein.
"It's giving hope where there wasn't hope and it's revolutionized treatment," Temple neurologist Dr. Terry Heiman-Patterson said.
Dr. Heiman-Patterson says Spinraza was shown to be effective on babies, and while approved for adult use, in patients like Murphy, it's not clear if it will work as well.
"It's too soon to tell but I'm encouraged," Dr. Heiman-Patterson said.
Murphy, who's a senior at Temple, hopes the treatment will at least stop progression of the disease. His insurance is covering the cost $750,000 for the first year, a steep price for his first stab at hope.
"It's just incredible we live in a world where science was able to get to this point, where I could potentially walk again. It really is amazing," Murphy said.
