Pediatric experimental cancer therapeutics program’s success is thanks to Four Diamonds support

Editor’s Note: Penn State’s THON Weekend is Feb. 20-22. Students will dance for 46-hours to support pediatric cancer patients. To date, $114 million has been raised and donated to Four Diamonds, a foundation that supports the families of pediatric patients at Penn State Hershey Children’s Hospital, and the cancer research done here. For more information on THON, or to watch the activities live, visit THON.org. For more information on Four Diamonds, visit FourDiamonds.org.

Dr. Valerie Brown

When Dr. Valerie Brown was hired as clinical director of the experimental therapeutics program in the Division of Pediatric Oncology/Hematology at Penn State Hershey Children’s Hospital, she had a vision: Develop a menu of experimental cancer treatment options not available in the region.

Through funding from Four Diamonds, her vision is becoming a reality, helping young cancer patients find alternatives when standard care isn’t enough.

Experimental therapeutics are typically phase 1 and 2 clinical trials. In phase 1 trials, researchers are looking for toxicity in the therapies. In phase 2 trials, the effectiveness of the therapies on specific cancer types is studied before testing in bigger studies.

“I really hit the ground running, and one of the things we needed to do was expand the portfolio because you don’t want to compete with other academic medical centers,” Brown said. “You want to offer things not offered at other places and be able to offer a variety of different studies for a large spectrum of cancer types.”

To help with that goal, Penn State Hershey joined several consortiums including the Neuroblastoma and Medulloblastoma Translational Research Consortium. The consortiums bring together several institutions all sharing the same goal by working together cooperatively, opening up access to a variety of clinical trials.

Brown has seen how the approach is working.

In one case, a child had neuroblastoma in remission and was set to participate in a study to keep the cancer in remission, called maintenance therapy. But as scans and imaging were completed, it was discovered that she relapsed and the cancer had returned.

“That meant she wasn’t eligible for the maintenance therapy study,” Brown said. “But instead of having to turn that child away with her disappointed mom, devastated with news that the neuroblastoma had returned, we had another protocol that is a treatment for relapsed neuroblastoma. If we didn’t have that portfolio of clinical studies ready, she would have had to leave and go somewhere else.”

In this case, the study is a personalized – or precision – medicine study. The tumor’s DNA and RNA are extracted from a piece of the tumor and are analyzed and compared against normal tissues in the body and other cancer type cells.

“Therapies are based on how the same tumor types typically react to treatment,” Brown said. “But each tumor is individual, and if the person has relapsed, we already know it isn’t reacting like a typical tumor. By analyzing the patient’s individual tumor, we try to find out what differences are making it react differently, and then we decide what we think will be the best difference to target for treatment.”

These results are then compared and prioritized by a computer program against a panel of about 200 agents – some of which are alternative like the spice curcumin, which is known to be active against cancers.

Those reports are then sent to primary investigators at the centers across the country that participate in the consortium. People are assigned to review the case and come up with a treatment plan based upon these reports, which is then discussed virtually through a tumor board.

“That day, as badly as I felt for that poor mom and child because she relapsed, I turned to our medical director and I said, ‘this is why we set up our program like we have. This is the vision we had, and it is benefitting our patients,’” Brown said.

Including studies in the Children’s Oncology Group, there are currently up to 40 trials available, with around 10 being early phase trials. Patients have travelled from nearby states to participate in the studies.

“People are coming from other states because the treatment options are not available there,” Brown said. “By word of mouth, and on social media from the parents, people have recognized that we are offering things that nobody is offering nearby.”

She continues to look for opportunities to connect Penn State Hershey doctors and scientists with peers at other institutions. She also actively looks for opportunities to move Penn State Hershey research in the laboratories into clinical trials through the consortiums.

“These parents are coming to us and are really at the end of the rope for their children,” Brown said. “You could offer, ‘I read a paper and they tried this and maybe…’ but the science side of me just can’t let that happen. We have to do this in a systematic way because we really want to make sure what we treat our children with is effective and not hurting them more. That can only be done in the context of studies and trials.”

All of this would not be possible without the support of Four Diamonds and the Penn State students who work hard throughout the year raising funds through THON.

“It takes time. It takes money. It takes resources,” Brown said. “Luckily I have a lot of those things here that I didn’t have at other places. Without the Four Diamonds’ backing, and its recognition of the importance of having an early phase program, none of this could happen and I wouldn’t be here. The money, in my opinion, has been well spent because even if these children don’t have the outcomes we want, we are contributing to the wealth of knowledge, and hopefully pushing it along so that the next child who walks through the door won’t have to go through a relapse or undergo such intense treatments.”

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