Congress acts to help Alabama family save their son who ‘doesn’t know he’s dying’

WASHINGTON — A 10-year-old Alabama boy who already inspired the Alabama legislature to take action to help individuals suffering from terminal illnesses has managed to repeat the impressive feat, this time in the United States Congress.

Gabe Griffin, a boy fighting a rare form of muscular dystrophy, visited Washington, D.C. with his family this week to advocate for the 21st Century Cares Act. The bill would let the Food and Drug Administration automatically approve “follow-on drugs” – prescription medications that use the same technology as drugs that have already been approved but target different structures to treat certain conditions.

Advocates say the bill could help anyone with a rare terminal illness that does not have any known cures or treatments.

The bill overwhelmingly passed the House by a vote of 344-77, and now moves to the Senate. During their visit to D.C., the Griffins met with the bill’s sponsor and Chairman of the House Energy and Commerce Committee, Fred Upton of Michigan, as well as Alabama Congressmen Bradley Byrne (R-AL1) and Martha Roby (R-AL2). Rep. Upton is confident that the bill will pass the Senate.

Last year the Alabama legislature passed “Gabe’s Right to Try” law, which will allow new medications already in the human trial portion of FDA approval to be tried by patients with terminal, life-threatening illnesses.

Gabe suffers from a rare form of muscular dystrophy called Duchenne disease. Duchenne, which is caused by an absence of a protein that helps keep muscles intact, causes weakness and muscle deterioration that worsens over time.

“At 10 years old, Gabe walks, hugs, breathes, and feeds himself like any other typical child that age,” said Gabe’s father, Scott Griffin, “but that will be taken away in the next few years if we don’t find a cure. Without a cure, Gabe is expected by doctors to be in a wheelchair by the time he is 12 years old and lose his life around the age of 20. Traci and I are willing to accept all risks to save our son and we will do anything to keep from having to put him in a wheelchair in a couple of years.”

The Griffins hope that the passage of the 21st Century Cares Act will give Gabe access to drugs that are based on eteplirsen, a drug that has shown to be effective in stopping the progression of his particular subset of Duchenne.

Gabe’s story also inspired Michael Staley, a former congressional staffer from Alabama, to cycle across the entire United States to raise money and awareness for the Hope For Gabe foundation.

Through it all, the Griffins remain hopeful.

“Gabe doesn’t know that he’s dying, and one day I’m going to have to tell him that,” his father said. “I pray every day that that day doesn’t come.”