Tennessee families impacted by ALS become advocates for Congressional action

As Allie Schmidt awaits for the government to approve new treatments that could help her battle with amyotrophic lateral sclerosis (ALS), she’s left to pursue her own remedies.

For Schmidt, that means spending $300 per month from her modest disability insurance stipend to pay for vitamin bundles “in the hopes that these vitamins slow my disease progression.”

Schmidt is one of a growing number of Tennessee residents who have joined a coalition of advocates to push for the passage of new federal legislation that would cut the red tape that slows down access to promising new treatments. Most of the advocates are people diagnosed with ALS and their family members.

In addition to slashing the red tape, the ACT for ALS Act legislation, filed earlier this year in the U.S. House and just a few weeks ago in the Senate, would also increase research funding for ALS treatments and possible cures.

The legislation would establish a federal government collaboration between the National Institutes of Health and the Food and Drug Administration, while also committing grant funds to promising research similar to what the federal government already does for cancer research.

The increased scientific focus in recent years on ALS, the rare and mysterious neurodegenerative disease that affects the brain and spinal cord, has yielded promising treatments.  Approval for those treatments can take up to 10 years, which leaves Schmidt hoping her expensive vitamin regimen works.

“But when there’s so much red tape surrounding access to treatment, what other choice do I have?” said Schmidt, who is documenting her experiencing on a blog called Disability Dame. “It can be just as discouraging seeing promising results from ALS clinical trials, knowing that the likelihood of getting access is so slim. What good is having a promising treatment when the people who need it don’t have access to it?”

Schmidt and other Tennessee families have seen their advocacy pay off. Six of Tennessee’s nine U.S. House members have co-sponsored the ACT for ALS Act. Republican Sen. Marsha Blackburn joined the Senate’s ALS Caucus earlier this year.

U.S. Rep. Jim Cooper, D-Nashville, said he agreed to co-sponsor the legislation after hearing the story of Nashville resident Evan Campa, who is also diagnosed with ALS and has taken a leading advocacy role with the national group I Am ALS.

“We need to fully fund all types of scientific research, especially now during the pandemic,” Cooper said. “I’m founder of the Golden Goose Awards, which promotes federal funding for research that makes an impact. Nashvillian Evan Campa and many in the ALS community have been an inspiration to sign on to this bill.

“They’ve shared their stories about what works and what doesn’t, and the importance of experimental treatments. We have to further accelerate research to fight ALS.”

As Campa puts it, “the only way we can beat ALS is if we move at the pace of ALS.” The average life expectancy for someone diagnosed with ALS is between two and five years.

“That urgency has built momentum for a policy that creates new pathways for what will one day be an effective treatment,” Campa said.

The success of Operation Warp Speed in approving and distributing a vaccine for COVID-19 accentuates what can be gained from cutting through the bureaucratic process.

“ACT for ALS invests in novel therapies and increases the opportunity for patients to access investigational treatments outside of clinical trials,” said Campa, a 39-year-old mother of two children. “My only hope to see my kids grow up will be an expedited treatment. FDA approval can take 10 years — we don’t have that kind of time.”

The advocacy strategy helped gain a supporter in Rep. Scott DesJarlais, R-Jasper. The conservative DesJarlais added his name to the list of cosponsors after speaking to advocates earlier this year.

“While there are some encouraging developments in the fight, we need to make sure we are doing everything to accelerate this research and make those potential new treatments available to those suffering,” DesJarlais said. “ALS patients and their families know and accept what they’re fighting against, and they are willing to be on the forefront of accessing and trying new treatments with the hope of one day finding the silver bullet to kill this insidious disease.

“As we also see with COVID and Operation Warp Speed for vaccine development, we need a system that can be flexible, nimble, and accelerate care and therapeutics to patients who need it.”

In addition to Cooper and DesJarlais, the other members of Tennessee Congressional delegation to co-sponsor the ACT for ALS Act are Rep. Steve Cohen, D-Memphis, Rep. Tim Burchett, R-Knoxville, Rep. Chuck Fleishmann, R-Ooltewah, and outgoing Rep. Phil Roe, R-Johnson City.

“It’s not just in Tennessee – nationally people with ALS are organizing and building momentum to push for treatments and hopefully one day a cure,” Campa said. “I Am ALS, a national, patient-led advocacy group, has brought many of us together with a clearly defined goal – end ALS. We are so grateful to the six representatives who support the ACT for ALS bill and hope that our two senators will join them.”