With CureWorks launch, they keep fighting for kids every day

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10/22/2020

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Dr. Jodi Skiles: “Being part of the revolutionizing of leukemia care is exciting for us both as physicians and scientists, to really get to contribute to the field of knowledge about how to continue to conquer this really awful disease.”

By Maureen Gilmer, IU Health senior journalist, mgilmer1@iuhealth.org

Dr. Jodi Skiles and Audrey Hopper go to battle every day for kids stricken with aggressive, life-threatening cancers. They embrace the mantra Fight Like a Kid, and now they have a new weapon in their arsenal.

It’s called CureWorks, and it’s ready to launch at Riley Hospital for Children at IU Health.

A year ago, news that Riley would be part of the revolutionary program to treat stubborn cancers in kids sparked hope in families around Central Indiana and the entire Midwest.

Now that program is poised to kick off with three groundbreaking trials reserved for the sickest kids, those whose cancers can’t be conquered with more traditional treatments.

As one of the selected hospitals, Riley oncologists and Indiana University School of Medicine researchers will have greater access to cutting-edge immunotherapy trials for pediatric oncology patients. Member hospitals are supported in launching and participating in exclusive clinical trials. CureWorks streamlines immunotherapy production, clinical trial enrollment and the trial coordination process.

CureWorks is a unique collaboration among five elite children’s hospitals in North America. Riley is the only member hospital in the Midwest. Others in the network are Seattle Children’s, Children’s Hospital Los Angeles, Children’s National (Washington, D.C.) and BC Children’s Hospital (Vancouver, British Columbia).

OPENING TRIALS AT RILEY

Dr. Skiles, director of pediatric stem cell transplant and cellular therapy at Riley, said her team is on track to open two of the three planned CureWorks trials for patients with acute lymphoblastic leukemia (ALL) at Riley by next month.

All three trials essentially use CAR T-cell therapy in a modified way to overcome some of the challenges experienced with the commercially available product Kymriah. CAR T-cell therapy is a form of immunotherapy that uses specially altered T cells — a part of the immune system — to fight cancer.

The hope is that the new therapies will be more successful at not only getting kids into remission but sustaining that remission.

“There are two Riley patients that need this therapy urgently,” Dr. Skiles said. “We are working closely with CureWorks to get these kids enrolled and started on treatment as soon as possible. We are working around the clock with our regulatory boards at IU so that patients will no longer need to leave home to get access to these groundbreaking and life-saving treatments.”

Dr. Skiles said the team is “cautiously optimistic and very excited about opening the trials here and the potential for getting these kids access to treatment.”

“Being part of the revolutionizing of leukemia care is exciting for us both as physicians and scientists, to really get to contribute to the field of knowledge about how to continue to conquer this really awful disease.”

“THESE ARE VERY SICK KIDS”

Audrey Hopper, clinical coordinator for Riley’s CAR T-cell program, is Dr. Skiles’ right hand in the rollout of the CureWorks trials.

“Audrey is helping us orchestrate all this,” Dr. Skiles said, explaining that coordination of care for the new program is a heavy lift. “She is a powerhouse, a huge advocate for cancer care. We are so lucky to have her.”

Hopper in turn relies on research coordinator Courtney Spiegel and nurse practitioners Jessica Harrison and Kim McGinnis, among others, to help make miracles happen.

A nurse in the Cancer Center at Riley since 2007, Hopper joined the CAR T team in 2018. Her tenacity and relationship-building skills are critical to the success of the program.

“I’m like a dog with a bone, I never take no for an answer,” she said of her advocacy work for patients and families, whether dealing with insurance companies, lawyers or the State Legislature.

One of those patients is Maria Lorenzo-Bartolo, who is recovering after CAR T-cell therapy. Hopper went to bat for her in a big way, getting all of her pre-approvals lined up in advance so when the time came for therapy to begin, they would not lose a minute of precious time.

“What’s been wonderful is I’ve met a lot of cool, interesting people and learned a lot about policy and programs and processes along the way,” Hopper said. “We’re not just nurses. We also do the financial piece, and that is a big barrier in CAR T because it is so new.”

The speed with which pieces have to fall into place can be dizzying. And the stakes are enormous.

“That’s why Dr. Skiles and I are so close, because it’s high pressure,” Hopper said. “These are very sick kids whose families’ worlds have been rocked. We fight for these kids.”

Hopper’s mind is always racing, thinking about the kids who need lifesaving intervention. Even on vacation, she is raising awareness of pediatric cancer, wearing Fight Like a Kid T-shirts with her own children.

MAKING CAR T SMARTER

Most of these families have been here before, hoping and praying that the latest experimental treatment will save their child.

The original FDA-approved CAR T-cell product targets a marker on the surface of the leukemia cell called CD19, but a newer version in one of the trials is bi-specific, targeting both CD19 and CD22, thus providing two mechanisms to attack the leukemia cells.

“Our hope is that targeting two markers on the leukemia cells will keep the leukemia from being able to out-smart the treatment,” Dr. Skiles said. “One of the problems with targeting a single leukemia marker (like CD19 with commercially available Kymriah) is that sometimes those leukemia cells just stop expressing CD19. If there’s no CD19 expressed on the surface of the leukemia cell, then the CAR-T cells don’t have a target to attack, thereby making treatment ineffective and allowing leukemia to come back with a vengeance.

“One of the CureWorks trials is working to overcome this problem by manufacturing CAR-T cells that have antibodies to two different markers on the surface of the leukemia cell,” she added. “This approach makes it harder for the leukemia cells to figure out a way around the drug.”

To participate in the CureWorks trials, patients must have either exhausted commercially available standard-of-care options, including stem cell transplants, or are not medically eligible for those options.

When a patient is identified as meeting the eligibility criteria, he or she is already critically ill, so fast action is necessary, Hopper said. That means ensuring the patient’s clinical needs are met, along with securing insurance approval and even temporary housing if necessary for family members.

“We go to war,” Hopper said, explaining how she and the team must move quickly through an obstacle course littered with red tape designed to slow them down.

That’s where her tenacity comes into play.

“When you have the autonomy to work at the highest level and you have the team behind you … you make a decision and go. In the end, it’s me and that family.”

As the CureWorks trials ramp up at Riley, Dr. Skiles acknowledges there will be a learning curve, so it is important to have all of the right supportive care measures in place. Her hope is to eventually have three to five patients participating in each trial at Riley.

Riley also recently opened its Children’s Oncology Group CAR T trial, which uses CAR T-cell therapy as an upfront treatment rather than a last-ditch effort.

“We are using it for patients who are at high risk for relapse,” Dr. Skiles said. “Instead of sending them to transplant, they go to CAR T first. It will be very interesting to see the results of that roll-out because if kids can avoid transplant all together, that would be amazing because it would allow them to avoid long-term toxicities of transplant,” she said.

“It’s awesome to be able to offer hope to families who otherwise would have nowhere to turn.”

Photos submitted and by Mike Dickbernd, IU Health visual journalist, mdickbernd@iuhealth.org

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CureWorks will be a ‘game changer’ for Riley kids with cancer -The unique collaboration among elite children’s hospitals means Riley oncologists and IU School of Medicine researchers will have greater access to cutting-edge immunotherapy trials for pediatric oncology patients.

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