Pediatric Cancer Research

Putting Hope Within Reach

Cancer Center team makes innovative treatments available to the children who need them most

On Feb. 7,2008, CHOP researchers discovered that mutations in the ALK gene cause neuroblastoma, one of the deadliest childhood cancers.

On Sept. 21, 2009, they started a clinical trial of a drug that they believe will kill cancer cells by deactivating the ALK gene.

The drug is a promising new treatment option for a group of patients who have very few. And researchers were able to get it to them remarkably quickly.

"If you ask most people how long it takes to go from a discovery to a clinical trial, they'll tell you it typically takes ten years," says John M. Maris, M.D., chief of the Division of Oncology. "We want this to be the new paradigm."

Maris says he and his colleagues were able to make the journey from discovery to treatment in less than two years because they have a team that spans the bench to the bedside. The laboratory team, led by Yael Mosse, M.D., includes scientists who focus on getting the best drugs to patients as quickly as possible, working closely with a group of clinical professionals — two nurse practitioners, a social worker and a clinical research associate — who help set up and run clinical trials. The goal is to make discoveries and prioritize those that give patients access to the newest and most innovative treatments.

For many neuroblastoma patients, the clinical trials offered at CHOP are their last hope — and Maris' staff helps put thai hope within reach. His team finds families a place to stay near the Hospital, puts them in touch with financial assistance programs, and works with patients' insurance companies to ensure that their treatments are covered — a task that families often find overwhelming.

"These families have an extra layer of stress and need," says Dana Vass, M.S.W., L.S.W, the social worker on Maris' team. "If you don't have a team that's going to do this day in and day out on a consistent basis, some of these patients aren't going to get here."

The clinical research team relies heavily on philanthropy: the Alex's Lemonade Stand Foundation, the friends and family of CHOP patient Andrew Accardi, GlaxoSmithKline, and the Evan T.J. Dunbar Neuroblastoma Foundation have all made significant donations to support the team's work.

The result is that Maris and his colleagues are able to offer a wide selection of the very best clinical trials, giving patients more options at every stage of their disease. The team works closely with each family to identify those options and develop a customized treatment plan, which often includes several different clinical trials.

It's an approach that's working for 8-year-old Danielle Blascovich, who was diagnosed with neuroblastoma in 2005. When doctors at her local hospital told her parents there was nothing else they could do, Maris' team arranged for Danielle to travel to CHOP, where she enrolled in a clinical trial that combined targeted radiation therapy with chemotherapy.

That treatment helped, but it was the next trial they tried that put Danielle's cancer into remission. And it was the third one that has kept her in remission for more than two years.

More Hope to Give

CHOP'S neuroblastoma program has been hugely successful on both a local and a national level:

treatments like the ones Danielle received are now used in hospitals across the country — and Maris' team receives referrals from all 50 states.

Now, Richard Aplenc, M.D., M.S.C.E., and Rochelle Bagatell, M.D., want to use the same team approach to help more children who have brain tumors, sarcomas, lymphomas, and the most common childhood cancer, leukemia.

Maris believes research on these cancers has advanced to the point where significant treatment breakthroughs are possible. It's just a matter of getting a robust team in place to make them happen.

A two-year grant from Alex's Lemonade Stand Foundation will help Aplenc and Bagatell begin to build that team, but additional funding is needed. "Philanthropy is very critical to us because it costs more to treat patients on new drug trials than insurance will reimburse. So we can't develop and run these trials without philanthropic support," says Aplenc.

"What we're trying to do is offer people treatments that they wouldn't otherwise have access to," says Bagatell. "These are people who would otherwise be told, 'We have nothing left to offer.' "

It's something Danielle Blascovich's parents remember hearing a few years ago.

So much has changed for Danielle since then. "She's feeling great — she's running around playing with friends, she's in school," says her mom, Diane. "There's hope."

Source: Cause for Hope: CHOP Cancer Center Annual Report 2009