Wisconsin

Penn Med and CHOP Are Beating Cancer With Gene Therapy

“I do believe in miracles.”

Austin Schuetz was just 2 years old when doctors diagnosed him with acute lymphoblastic leukemia (ALL), a rare and potentially fatal form of cancer. His parents were stunned. Schuetz was like any other little boy that age - active, healthy and showing few signs of illness.

Too young to understand his diagnosis, Schuetz told his parents that he had “cancer cooties,” his mother, Kim Schuetz, said.

“I felt like I was looking into somebody else’s life,” she told NBC10.

Chemotherapy, radiation and a bone marrow transplant followed, but nothing seemed to work. His parents enrolled Schuetz in a clinical trial at the Children’s Hospital of Philadelphia and, within one month, their luck began to change.

Schuetz had an adult leukemia patient named Bill Ludwig to thank.

Like Schuetz, Ludwig made his way to Philadelphia in hopes of beating his cancer 10 years after first being diagnosed. Chemotherapy no longer worked, and Ludwig and his wife sought some kind of miracle at Penn Medicine.

“I was hoping for a day, a week or a month,” he said.

Ludwig became the first patient to try an experimental treatment called chimeric antigen receptor (CAR) T-cell therapy, or CAR-T, that would eventually save his, and Schuetz’s, life.

“I asked God every night to give me the strength to fight,” he said.

And fight he did.

Using a modified version of the HIV virus, doctors removed Ludwig’s healthy cells and modified them to search for and destroy cancerous cells. The so-called hunter cells were infused back into Ludwig’s body and left to do their work.

The subsequent side effects were almost worse than the initial diagnoses, however. Within days, every major organ stopped functioning properly, Ludwig said.

With his help, doctors began to understand and address the unexpected side effects. Just a few weeks later, Ludwig was cancer-free.

“I’m a bless individual,” he said.

Ludwig has been in remission for seven years now. Since then, doctors at Penn Medicine and CHOP have applied what they learned from the 72-year-old patient to kids with leukemia.

CHOP became the first institution to study T-cell therapy on ALL, the most common type of childhood cancer. Typically, 20 percent of those diagnosed with ALL do not respond to conventional treatments.

But a 2015 global trial involving 68 children and young adults saw positive results in 83 percent of patients.

In August, the Food and Drug Administration approved the therapy for patients 25 years and younger.

“The big question is can we make these kinds of cells work for other kinds of cancers?” Dr. Stephen Grupp, who oversaw the first clinical trial, said.

For Schuetz’s family, every check-up can feel like the very first one. He still flinches when doctors draw blood, and his mom worries the cancer could resurface. But thanks to Ludwig and the clinical trials, the little boy from Wisconsin remains cancer-free four years later.

“You just want to have that one last hope that this is going to work and this is going to save him,” Kim Schuetz said. “I do believe in miracles.”

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