The FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy

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The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the same disease. Jaci has a rare form of ALS. The mutation is called FUS P525L.

The disease is progressing so quickly that even her doctor expressed surprise at her rapid decline.

In only a few months after diagnosis Jaci was no longer able to ride her favorite horse, Bud. Jaci always dreamed of someday having a horse arena. But now she is coping with her doctor’s suggestion that she should consider the use of a ventilator to assist with breathing.

According to a recent article in STAT, Dr. Neil Shneider, Jaci’s physician, who is head of the ALS clinic at Columbia University, contacted the Hermstads about antisense oligonucleotide (ASO), a new therapy some call “a miracle”. He said this treatment could halt production of the proteins that are suspect in killing ALS patient’s motor neurons.

That was unbelievably exciting news for the Hermstads. But what felt like “divine intervention” quickly turned to a struggle for Jaci’s life.

They were told the therapy was untested and that they could not access it. It had not even been tested for toxicity. That would mean getting the drug through FDA’s program called “expanded access.”

The Hermstads could not count on the government’s right-to-try law because the law required at a minimum, completion of at least a Phase 1 clinical trial.

Jaci’s Crusade

Jaci’s parents went into high gear. They handed out petitions, sent a tweet to President Trump, and contacted a few powerful politicians. These contacts included Speaker Nancy Pelosi, Sen. Chuck Grassley, and their Iowa representative Steve King, who has recently been under fire for his purportedly racist comments during an interview.

The Hermstads started a group on facebook called “Cowgirl Up for Jaci,” and an online petition to the FDA. The goal was to get the FDA to waive their initial studies so Jaci can get the (hopefully) lifesaving injections.

As it turned out, the FDA never denied Jaci access to the therapy but simply requested that toxicology tests be conducted. Jaci’s own physician, Dr. Shneider, agreed that the FDA was cooperative.

Powerful Arguments on Both Sides

The main job of the FDA is to make a decision about the efficacy of a drug and whether it is safe for Americans. The current debate over Jaci’s proposed therapy is a replay of previous debates in Washington that culminated in the passage of the “right-to-try” law.

The law effectively removes the FDA from deciding whether patients with terminal illnesses may be allowed to use experimental drugs that are being tested in clinical studies.

The complexity of gaining access to experimental drugs in the U.S. lends itself to a continuous debate regarding:

  • The role of the FDA in deciding when patients who are terminally ill should have access to experimental therapy;
  • Whether politicians should be involved with the “how and when” regarding therapies never before tested on humans;
  • What is the role of lawmakers when it comes to members of Congress intervening on behalf of just one constituent?
  • The relationship of crowdfunding to research;
  • Who is responsible for assisting a young woman faced with the prospect of dying and unable to get therapy that might possibly save her life?

Experts on one side of the debate question if we are allowing the FDA to carry out its mission. Others have ethical questions such as asking about patients who do not have the same contacts or resources to attract the attention of legislators and the media.

One of the primary issues on the other side of the debate is simply that a life is “on the line” so let’s save it if there is even the slightest chance of doing so.

One Sentence Legislation

Congressman King took an unusual step by introducing a bill that consisted of only forty words. The bill would require the federal agency to grant an exception in its rules to one individual: Jaci Hermstad.

Although the bill was never under consideration by Congress, King did get the attention of Nancy Pelosi who eventually had discussions with the FDA.

Washington’s influential Sen. Grassley also urged the FDA and the White House to consider Jaci’s request. About one week after Pelosi and King’s intervention the FDA showed the first sign of backing down and indicated to Jaci and her parents that she may be receiving the therapy.

GoFundMe

The Hermstads have already raised $200,000 of the $700,000 needed for the treatment but it will not be paid to the developer, Ionis Pharmaceuticals.

Instead, it will be used to pay for the treatment basics such as paying a contract treatment agency for the balance of the requisite tests, cost of infusions, hospitalizations, and travel to the hospital.

About Ionis Pharmaceuticals

The developer of ASOs, Ionis Pharmaceuticals, produces drugs that control the on/off switches that govern protein production. This is in contrast to most traditional drugs.

Ionis will be conducting clinical trials for Parkinson’s, ALS, Alzheimer’s and Huntington’s. The company indicated their willingness to cooperate with physicians and scientists by releasing their early data and offering advice about antisense technology as it applies to ALS.

Race Against Time

The good news finally arrived from the FDA. It approved testing for ASO. The manufacturing of this specific treatment, just for Jaci, had been completed as of May 7, 2019.

The family’s anxiety continues however, as testing the drug for safety may take at least six weeks. As they wait, Jaci’s condition continues its decline.

And the debate is still swirling in the political and scientific worlds. Many questions arise when they speak on behalf of people who do not have the contacts or funds to access these experimental drugs.

But then they speak of Jaci’s parents losing one child and desperately trying to save their other daughter.

Where do you stand on this debate?

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Rose Duesterwald     June 6, 2019

 

 

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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