Mother calls for better access to life-saving treatments

AUSTIN (KXAN) — Proposed federal and state laws could make it easier for terminally ill patients to get access to potentially life-saving drugs. Reforming the system by which medication or experimental therapies are made available to those patients, the system called expanded access or compassionate use, is gaining momentum and was a topic at this year’s annual Childhood Cancer Summit in Washington, D.C.

The issue is one the Guillot family knows well. Their son, Zach, was diagnosed with acute myeloid leukemia when he was 5 years old.

“When you’re in these desperate situations like this, you’re searching for options,” said Zach’s mom, Julie Guillot.

Zach went through the traditional treatments for AML, including several rounds of chemotherapy and two bone marrow transplants. But his leukemia returned for a third time and it was time for the Guillots to make some difficult decisions.

“Seattle Children’s agreed to take Zach’s case for some experimental therapies and a potential third bone marrow transplant, which was admittedly way out there,” Julie said. “It was very boundary pushing, but we had a lot of conversations with Zach. He wanted to live and so we packed up the family on a moment’s notice and left town and we went on a mission to find a cure.”

In Seattle, Zach was enrolled in a clinical trial for a medication his doctors thought was his best chance for survival. The little boy, who loved Batman and lived by the motto, ‘No Surrender’, responded well. But there was a complication during the trial when Zach became dehydrated. His dosage was lowered and the medication became less effective. After the clinical trial was over, the Guillots applied for compassionate use of the drug to be given to Zach at the higher dosage. They waited six weeks, time Zach did not have, to learn the drug company had denied his request.

“I think it is important that we look at how we can improve this process because really anybody can wake up tomorrow just like we did, just like Zach did, and you go through conventional treatment and you don’t have that many options,” Julie said.

“This would, I really think, transform the way we deliver medications, particularly to children with cancer.”— Congressman Michael McCaul

In Washington, D.C., Congressman Michael McCaul is leading the charge to reform the compassionate use system. He is preparing to introduce a bill called the Andrea Sloan CURE (Compassionate Use Reform and Enhancement) Act, which would help patients with life-threatening illnesses find clinical trials. It would also require pharmaceutical companies to have expanded access policies, along with requiring them to notify the Food and Drug Administration when they receive an expanded access request. If they refuse to grant expanded access of a drug, under the proposed legislation, companies would also be required to explain to patients the reason why their request was denied.

McCaul was a friend of Austin attorney Andrea Sloan, 45, who waged a public battle for compassionate use in 2013 while in the final stages of ovarian cancer. He said he is ready to introduce the new legislation in Congress in Sloan’s honor.

“It was very tragic to watch that experience, but I know that she would want us to move forward with a bill like this in the congress because she often advocated for it and I want to champion her and her cause by introducing this legislation,” McCaul said Friday. “This would, I really think, transform the way we deliver medications, particularly to children with cancer.”

Ultimately, the drug company in Zach Guillot’s case reversed its decision and granted compassionate use, although the Guillots had already decided to proceed with a third bone marrow transplant. Zach received the transplant, but died on February 8, 2014, from chemotherapy toxicity related to the procedure. He was 9 years old.

“It’s really hard to have to watch your kid go through so much and be so brave through all of these things,” Julie said. “I would do anything to save Zach, but what he had to go through as a kid is unacceptable. He spent over half his life fighting to live.”

In Texas, State Representative Eddie Rodriguez, D-Austin, also a friend of Sloan’s, is preparing to co-author bipartisan “Right to Try” legislation, that would remove the Food and Drug Administration from the compassionate use approval process. Three other states, Colorado, Louisiana and Missouri have already passed these laws. Rodriguez plans to introduce the legislation in the upcoming legislative session.

“I believe if someone is that sick, that ill, that they’ve exhausted everything else, that their own doctor who has been taking care of them for a long time and has determined that the best chance for you is to try this experimental drug without any guarantee of its success, but it’s their last chance, then I think we have to give them that chance,” said Rodriguez.

Caught in the Middle

Through her experience, Julie Guillot knows the issue is not a simple one.

“The process is difficult for all parties,” she said. “One as a family you’re terrified because this is one of your last tools in your tool belt, one of your last weapons, but it’s a big process for your doctor, it’s a big process for the hospital, all the way through the FDA and up the chain at the drug company. The beautiful thing about all this is individual freedom. I think that gets back to compassionate use, too. Not everybody wants to continue the fight and I think that is noble and that’s great, but for those people who want to continue to fight to have access to drugs and compassionate use and to push the boundaries, I think they help other people because doctors learn from them.”

At the Childhood Cancer Summit in Washington, D.C. last month, medical professionals and pharmaceutical company representatives discussed the challenge of compassionate use for doctors, hospitals and the drug industry.

“There have to be changes to the way people gain access to experimental medicines,” said Kenneth Moch, former CEO of Chimerix. “The current system of influence of social media or who you know or having a doctor who knows somebody simply doesn’t work on a broad basis for the patient populations in need. There’s a very difficult line between hope and what’s known about a drug and while you’re still developing a new pharmaceutical there’s many things you do not know and while people want hope when they or a loved one has a life threatening condition, the hardest thing to realize is that what’s not known about a drug may be as damaging or difficult as what is known about it.”

Since Zach’s death, the Guillot family has been fundraising for cancer research and supporting the use of an experimental therapy called targeted T-cell therapy, which has shown promise in patients with acute myeloid leukemia. A fundraiser is scheduled for October 23 in Austin.

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