One Last Try: Compassionate Use Drugs Give Cook Children's Patients Another Chance

To the parent of a child with a terminal illness, the value of a day, a week, a month or a year can not be measured. Each minute of life is precious as they and their child fight for every single one. Sometimes that fight includes incorporating the use of drug treatments that can only be offered under the label of compassionate use. This is the use of an investigational drug to treat a patient with a serious disease or condition when they have run out of available treatment options.

That was the case for Zander Hunt, the 11-year-old son of Aaron and Emily Hunt of Amarillo, Texas. Diagnosed with kidney cancer at age 7, Zander spent years undergoing grueling chemotherapy and radiation treatments, but the cancer continued to spread. By January 2020, it was classified as stage four. Zander’s prognosis was grim.

The family made a last-ditch effort with an aggressive chemotherapy treatment which shrank the tumors, but Zander needed surgery to completely remove them. The procedure required specialized care. That’s when the family turned to Cook Children’s Medical Center and found Marty Knott, D.O., Ph.D., pediatric surgeon, and Kelly Vallance, M.D., MPH, pediatric oncologist.

Dr. Knott performed surgery to remove a number of tumors from Zander’s body.

“The care we received at Cook Children’s was a breath of needed fresh air,” Aaron said. “We didn’t know what to expect after our first hospital experience but the attention and expertise that Zander and our family received was superior. We will be forever grateful!”

Dr. Vallance pledged to the Hunts to do everything in her power to find a drug treatment for Zander’s cancer. Finally, the family had a glimmer of hope.

“I remember feeling almost desperate for anything,” Emily said. “At that point you’re tired. The chemo and radiation have taken every bit of life out of them at some point. They’re always recovering from treatment. You just see your kid for this prolonged period of time withering away. I was desperate for anything that could help him without hurting him.”

Finding a treatment match proved to be difficult. Turns out a medication for Zander’s specific cancer markers and tumors does not exist. Dr. Vallance presented the family with one last option — try a medication that is commercially available for adults, but not approved for children or his specific type of cancer. Because of this, getting the expensive treatment covered by insurance was a long shot. The hospital would need to be granted compassionate use to trial this drug with Zander.

Dr. Vallance turned to Cook Children’s research pharmacy team, led by Micha Koentz, PharmD, and they went to work to obtain compassionate use approval.

Koentz and her small team, which includes another pharmacist and a pharmacy administrator, are responsible for dispensing investigational drugs being used in clinical trials and research at Cook Children’s. There are currently more than 100 medication studies open at the medical center. The research pharmacy team also handles any requests for compassionate use medications and works through a gauntlet of regulatory bodies for approval to use them. These drugs are either still being studied by drug companies but not yet on the market, or are available but approved for adults only, like in Zander’s case. Sometimes medication is available for children but not covered by insurance and is far too costly for most.

Once a request by a physician is made, one of the research pharmacists contacts the drug company to get their buy-in. Then, a request must be made to the Food and Drug Administration (FDA). After FDA approval, the request is sent to Cook Children’s internal review board for final approval. If all agree that the benefit outweighs the risks for the child, the drug can then be issued by the drug company, which they provide free-of-charge to the hospital. That means it’s also provided at no cost to the patient.

“It truly means access to a drug that is otherwise not available or affordable,” Koentz said. “This can buy the patient and family more time.”

Managing compassionate use requests — contacting drug companies, pleading the patient’s case, writing treatment plans and working with regulatory bodies — has become a personal passion for all three members of the research pharmacy team. The process can take anywhere from a few days to a few weeks, but the small but efficient team has streamlined the process to a point where they can usually obtain approval from all within two weeks or less. They know that when a child is facing the end of their life, time is of the essence.

The compassionate use request for Zander was approved. In September, he will have been using the medication for two years. All the while his scans have been clear. There is no sign of cancer advancing in his body. Best of all, he has gained back his childhood.

“He goes to school, plays on a club soccer team, has friends and a normal life,” Emily said. “This medication is working for him. We've gotten two years that we didn't know we would get, and they've been two years with clear health, like he was a healthy little boy. We're so thankful to all of the people we've worked with at Cook Children’s, because it changed our entire experience with kid cancer and with medical treatment.”

Sometimes a patient using a compassionate use drug lives another few weeks or months. Even so, there is comfort for the family in knowing every treatment option was exhausted, Koentz explained. Other times, the medication adds years to a patient’s life.

“It truly is the last option,” Koentz said. “Under compassionate use, we’re looking at anything we can do to really give the patient one last try at something that might work.”

In addition to one last try, these drugs give kids the opportunity to go home.

“A lot of our drugs are oral agents,” Koentz said. “It’s a pill that you swallow by mouth. So if you are at the end of your life, you really don’t want to spend that time in a hospital hooked to IVs. This really is a nice way for patients to go home, take an oral drug and hopefully add some weeks, months or years to their life.”

Koentz has issued compassionate use requests since her arrival at Cook Children’s research pharmacy in 2014. She said back then they would do one here and there. Because of her dedicated team, they now request and receive approval for multiple a year. In 2020 and 2021, nine compassionate use requests were approved. This year, the research pharmacy team is already working on their sixth request.

Pediatric cancer patients have fewer options for treatment than adults. While just as devastating, when compared to the prevalence of adult cancers, pediatric cancers are rare. So drug companies tend to focus on the research and development of adult cancer medications because the market for those is greater. That’s why compassionate use medications are critical in pediatric cancer care.

It’s also why Koentz went into research pharmacology. In pharmacy school all she learned about were drugs that are FDA-approved and commercially available at a pharmacy.

“Then I started working in pediatric oncology and quickly learned that some of our patients have to get research drugs because there’s just not a whole lot of options out there if they progress or if they never have their disease depressed,” Koentz said. “They really have to try other options. A lot of times our pediatric cancer patients will go to clinical trials. So I got really into clinical trials because I have a passion to further advance pediatric oncology. I want more options for our kiddos.”

As for Zander and his family, including his five brothers and sisters, they’re making the most of the time they’ve been given for more moments, more smiles, more laughter and more memories, and they’re relishing the normalcy.

“Zander told me the other day, he doesn't remember what it's like to be sick anymore,” Emily said. “I thought that was really impactful. He said he can remember being in the hospital and all of the doctors and the surgeries, but he can't remember what it feels like to be sick anymore.”