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Posted on Feb 27,2017

Where do you turn when the only thing more dangerous to your child than the disease she faces is the cure itself?

When emergency room physician and worried dad Rafael saw his own sick daughter quickly running out of options, he used his medical expertise and the support of Johns Hopkins All Children’s Hospital to find a solution.

Rafael and Monica’s 11-year-old daughter Natalie had been diagnosed with T-cell lymphoma and was having allergic reactions to her chemotherapy drugs. Rafael, who had once worked in the hospital’s emergency center, was well aware of its reputation—both for expert cancer care and academic leadership in research. He knew there was no question of where to take his daughter.

“The cancer institute at Johns Hopkins All Children’s is the best in the area,” he explains. “Plus, I knew most of the medical staff including her physicians so that gave us comfort.”
 

Finding a New Solution

Though T-cell lymphoma has an impressively high cure rate of nearly 95 percent, it was asparaginase, the standard chemotherapy medication, that ended up causing problems for Natalie. By the third dose, she started coughing and developed a rash around her face and arms. “The nurses reacted immediately and gave her medication to control the anaphylactic reaction,” her mother, Monica, recalls of that frightening day.

A substitute medication was given, but Natalie reacted to that as well. That’s when Rafael got busy. As a physician, he knew an unlikely alternative would need to be found. Relieved that his daughter was in an academic hospital known for research studies, he started asking questions and looking into drug trial options with the help of the experts at Johns Hopkins All Children’s Clinical and Translational Research Organization (CTRO.)

“In most cases once patients react to both treatments, there isn’t a third line chemotherapy drug we can give them,” explains Ashley Repp, senior clinical research coordinator at the hospital. “Natalie’s father asked for me to do some research and see if there were any clinical trials available for patients that react to both drugs. He also did his own research and used his medical contacts to successfully locate the right trial.”

The trial he found involved the original chemotherapy drug they tried, asparaginase, but it was the delivery method that made it unusual. Chemotherapy is typically offered intravenously or in pill form. This drug trial involves injecting the drug directly into donor red blood cells.

“Using Natalie’s blood type, donor red blood cells that matched were injected with a new form of asparaginase into the red blood cells,” Rafael explains. This encapsulation of the chemo prevents the medicine from interacting with the rest of her body. Then the red blood cells were placed into her central line and given back to Natalie like a blood transfusion. The chemo was then released more slowly into the body.

Johns Hopkins All Children’s helped Natalie get access to the trial treatment through “compassionate use.” Permission sometimes is given when no other options are available.

“Compassionate use is treatment of a seriously ill patient using a new, investigational, unapproved drug when no other treatments are available,” Repp says. “Investigational drugs are generally available only to people who are taking part in a clinical trial—which is a research study that is testing the drug. Natalie was the first child accepted, though technically she was not part of the trial.”
 

It Takes a Hospital

Working with a drug that also happens to be a blood product—after the chemo is injected into the blood cells—proved to be a challenge for the hospital. But all involved departments rose to it. Typically blood products are stored and entered into the system and infused separately from drugs—especially particularly toxic drugs such as chemotherapy. Special considerations had to be planned in advance for how the product would be handled, where it would be stored and how it would be tracked in the computer systems. The Johns Hopkins All Children’s Blood Bank, pharmacy, Hematology/Oncology, Infusion Center and Research departments were all involved in keeping the system flowing.

“All of these steps were well worth taking so that we could offer our patient the most innovative care possible for her diagnosis,” says Dee Mcmichael, lab section supervisor. “We coordinated with the manufacturer of the blood product/drug combo to ensure accurate testing, processing, and shipping needs were met and everything would be ready for the day of infusion. We also worked to verify accurate labeling and documentation both from the blood product and drug perspective. It was very heartwarming to hear how appreciative and grateful the family was.”

It worked. Natalie, now 12, received the entire protocol without complications. She remains in remission with no sign of reoccurrence but faces another year of treatment. “We were nervous about another reaction,” Monica remembers. “But the nurses were very good about explaining each step of the process and easing our fears.”

Most of Natalie’s treatments are in-home. She was able to take advantage of the Homebound program and with the help of her teachers, has been able to keep up with her honor classes. In fact, Monica explains, she even organized a fundraiser at school to raise money for pediatric cancer research—because she understands research is what helped her. Though results from the drug trial are not yet in, what helped Natalie was her body’s ability to accept the medication through this alternative method.

“When my dad found the drug trial, I was really happy,” Natalie recalls. “I am very blessed that my dad is a doctor. Without him, or anyone else in my family, I would have been really, really scared. My family has always been very close, but in a way, I think we have gotten closer and more open with each other.”


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