Matt Bellina is the face of the Have A Heart Foundation’s fight for the Right To Try and raising funds for ALS Research for a Cure!

As featured in the Courier Times and the Intelligencer | 8/11/17

Matt Bellina is dying. But he hopes he lives long enough to play a game of touch football with his oldest son.

It’s a wish that his 5-year-old son, JP, made on a dandelion he picked during a walk with his dad last year.

“Like any dad, I knew my job was to help him make his wish come true,” Bellina said.

But medical textbooks suggest that Bellina, a 34-year-old father of three from Northampton, is nearing the end of his life. He has amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig’s disease, an incurable and fatal disorder that progressively attacks the central nervous system. Those with the condition eventually lose their ability to speak, eat, move and breathe. The former U.S. Navy pilot can feed himself, but he can no longer write. He uses a walker and a wheelchair to get around.

When Bellina was definitively diagnosed with ALS in 2014 — eight years after he first showed symptoms — his illness was too far along for one federally approved drug for ALS patients and not advanced enough for the other. Clinical trials for new drugs are underway, but Bellina hasn’t qualified for them. Drug companies typically seek participants in early stages of a disease since the approval process can take at least 10 years — double the average life expectancy of an ALS patient.

And while federal law gives Bellina and others with terminal illnesses access to clinical trials of treatments and drugs that haven’t been approved by the U.S. Food and Drug Administration, the “compassionate use” process to get that access is time-consuming and burdensome.

“If I’m not going to make it anyway, why shouldn’t I get to try (experimental drugs)?” asked Bellina, who has testified before Congress and campaigned online to get people with terminal illnesses better access to potentially lifesaving drugs.

Thirty-seven states and the U.S. Senate agree.

They’ve passed so-called Right to Try bills and state laws, which are designed to ease the process for terminally ill patients to get experimental drugs and treatments.

Bills are in various stages of action in the Pennsylvania and New Jersey legislatures. However, since state laws can’t supersede federal law, they are mostly symbolic.

The U.S. Senate bill, which passed earlier this month, would let terminally ill patients circumvent the FDA’s lengthy “compassionate use” application to get speedier access to experimental drugs.

A patient’s doctor could directly request such medications from drug companies that have successfully completed preliminary safety testing, said Starlee Coleman, a spokeswoman for the Goldwater Institute. The Libertarian think tank developed the template on which the federal and most state bills are modeled.

Drug companies would continue to advise the FDA of adverse effects on terminally ill patients as they do on everyone in a clinical trial, but data on “compassionate use” patients couldn’t be used against pharmaceutical companies in the drug approval process as it is now, Coleman said. The Senate bill also protects drug companies and prescribing doctors from liability for bad reactions to these experimental drugs.

The House version of the bill, co-sponsored by Bucks County Congressman Brian Fitzpatrick, R-8, of Middletown, was introduced earlier this year and is expected to be voted on by the full House, though there’s no indication when. Fitzpatrick described the measure as another avenue to expand access beyond the existing FDA protocol.

“For too many, access to these potentially lifesaving treatments will come too late, or not at all,” Fitzpatrick said in a statement. “Whether it’s a father courageously battling ALS or a brave child living with Duchenne muscular dystrophy, they deserve the right to try.”

Journey to diagnosis

Bellina was 28 and a new dad when a neurologist first told him the symptoms that had plagued him for years might be ALS. He experienced balance problems, persistent muscle aches and hand cramping when he was a Navy pilot flying electronic-warfare jets.

The Navy grounded Bellina about three years after he first noticed the twitching in his hands. In 2012, he was placed on administrative duty and deployed to east Africa and the Korean peninsula to work as an anti-terrorism officer. He medically retired with the rank of lieutenant commander in 2015.

Obtaining his diagnosis was complicated by his youth and lack of a family history of ALS, Bellina explained. When it was finally confirmed in 2014, he was 31. JP was almost 2 and Kip was 2 months old.

Bellina has outlived others with his diagnosis and his doctors suspect he may have a slower progressing form of the disease than most people.

“I’m really lucky to do what I can do,” he said.

And what he can do lately is more than what he could do earlier this year.

Two weeks before his youngest son, Paxson, was born 5 months ago, Bellina started taking an experimental drug provided by a manufacturer he declined to identify. It was the first and only drug company to grant his “compassionate use” out of the nearly 20 he approached.

Since he started the medication, Bellina and his wife, Caitlin, said they’ve noticed improvements. He can get out of a chair unassisted. He can whistle again. His breathing is better. He has more energy. He no longer drags his feet. And his muscle cramps have eased.

Vomiting, the drug’s main side effect, is tolerable.

“When you’re dying, side effects are … eh,” he said.

Longtime state Rep. Robert Godshall, R-53, of Souderton, knows what that’s like.

He’s the driving force behind the Pennsylvania bill, which was inspired by his firsthand experience as a patient with a terminal diagnosis and no traditional medical options. Twelve years ago, Godshall had an unapproved stem cell transplant for multiple myeloma, a fatal, incurable blood cancer. The FDA age cutoff for the procedure was 65. Godshall was 72.

Now 84, Godshall said he used his political clout to pressure doctors at the University of Pennsylvania Health System to ignore medical protocol. The approval process took two months and involved legal paperwork that absolved the hospital and doctors from liability.

The transplant, which he said his health insurance covered, kept the disease at bay for two years. That was enough time for Godshall to enroll in a Penn clinical trial of a new drug. He was in the trial until recently, when the drug stopped working after 10 years and his cancer returned “full-blown,” Godshall said.

“I don’t think you should have to be in the Legislature to save your life,” he said.

Ethical concerns

Some medical ethicists, including Art Caplan, argue there is no need for Right to Try legislation.

The FDA has historically approved 99 percent of “compassionate use” requests, which typically number fewer than 2,000 a year, he said, and the agency recently streamlined the process to address time lags. The real obstacle for patients is drug companies that may reject compassionate use requests for a variety of reasons, including fear an adverse effect could delay FDA approval as it could under current law, he said.

“The decisions are still in the hands of drug companies that have the research and nothing about these laws shapes their willingness to give the drug,” said Caplan, founding director of the medical ethics division at New York University’s Langone Medical Center.

Right to Try laws generally don’t address the price of some experimental drugs or require insurers to cover them, or treat people for potential medical complications from experimental drugs, Caplan said. There’s no funding to help lower income people afford the drugs or to assist smaller drug companies to make them available, he added.

Finally, Caplan said, using experimental drugs could risk patient eligibility for end-of-life supports, including hospice and home health care — depending on their insurance coverage.

“I don’t see (the federal law) making a wit of difference for access,” Caplan said. “It’s nice to pass feel-good laws, but families will quickly find if they want to get drugs from little companies, they may have to pay a lot of money and travel to get the drug.”

Jo Ciavaglia: 215-949-4181; email: jciavaglia@calkins.com; Twitter: @JoCiavaglia

Photo credits:

Kim Weiner | Photo journalist