Two Stories, One Cause: A Right to Try

Congressman’s Notebook — May 1, 2016

“I want to be able to say I didn’t leave any chips on the table. I want to know I did everything I could to try to grow old with my wife and watch my boys grow up.”

Matt Bellina’s words touched my heart as I sat in a crowded conference room in Hyattsville, Maryland.

An Air Force veteran battling ALS, also known as Lou Gerhig’s Disease, Matt lives in Holland, Bucks County, with his family. Last week, he and Frank Mongiello, of Yardley, joined me in Washington, D.C. along with other local ALS families to advocate for new legislation that would give those suffering with ALS access to potentially life-changing treatments even if those therapies are in the clinical stage.

Too often, those suffering from terminal illnesses — including ALS — are obstructed from trying these drugs by both the pace of the trials and current federal rules. Once a new drug is created in the United States, it can take an average of two and a half to eight years for it to become widely available and that timetable may be longer than the life expectancy for most individuals with ALS.

What’s more, even when a drug passes the clinical trial phase when a patient could have it, the FDA only allows a limited number. In addition, those who have had a disease for over two years usually won’t qualify for any trial drug.

But those with ALS want to fight rather than succumb to a fate that might be changed if they are given more options. For those like Matt and Frank, the right to try represents hope in the face of despair.

The aptly named Right to Try Act [H.R.3012] would force the FDA to acknowledge both hope and reality and remove the needless barriers to potentially groundbreaking care.

If there is a drug or treatment available that could improve the life of a patient with a terminal illness, who is the FDA to stand in the way? Even if the efficacy is uncertain, don’t patients and families in dire circumstances deserve this opportunity?

The noisy room in Maryland filled with as many as 900 fell quiet as the FDA’s Advisory Committee opened the floor to speakers. I rose; the first to address the group of agency advisors and doctors.

The hearing was intended to seek comment on the potential approval of a drug that had promising clinical returns in the treatment of Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease that impacts about 13,000 children, mostly boys.

I had come to urge the panel to approve this potentially life-saving drug, although it had a small clinical trial size, the data appeared to indicate it was helping and certainly not causing more harm. Unfortunately, the FDA expressed concern about its efficacy and so far has refused to allow access.

I thanked the panel, recognizing the agency’s ongoing commitment to use its full range of tools and authority to expeditiously review candidate therapies for rare but devastating diseases, like Duchenne.

Then I introduced them to Jake Wesley of Trevose.

Jake is 15-years old and lives each day with this terrible disease. Sadly, like so many in his condition, the decline of his health has been precipitous. The risk of doing nothing for someone like Jake is unacceptable. I have seen his disease progress year after year, robbing him of independence. At least, he deserves the right to try and the hope that comes with it.

I told the panel I am not alone in this advocacy. Nearly 110 bipartisan members of Congress joined me in writing to the FDA, urging them to act for Jake and those like him. After I concluded, 50 other speakers — many parents, and even boys confined to power wheelchairs by Duchenne — made similar pleas.

It was beyond disappointing when the Advisory Committee voted against the approval — removing these individual’s right to try by a mere one vote. As is the case with ALS, the decision further highlights the need for a legislative fix.

Whether for Matt or Frank or Jake or millions of others throughout Pennsylvania and the nation, who are battling a terminal disease, the status quo is immoral and impractical. Each of our sons, daughters, mothers and fathers deserve their right to try. Personally, I will not relent until each is afforded that right.