Philanthropy at Work: Clinical trial gives hope to kids with brain cancer

Donor support launches trial, unlocking millions more in funding to expand treatment. 

An Urgent Mission 

“I'm not going to stop until I never have to tell another parent that their child has an incurable cancer,” says Leo Wang, M.D., Ph.D., a pediatric hematologist-oncologist at City of Hope. 

Leo Wang, M.D., Ph.D.
Leo Wang, M.D., Ph.D.

Wang treats kids who have brain tumors, the most common cause of childhood death from cancer. While there are effective therapies for some brain tumors, others can be difficult to treat. Certain tumors like diffuse intrinsic pontine glioma only have a median survival rate after diagnosis of nine to 15 months. 

“The conversation where I tell the family that their child has this disease is also the conversation where I tell them that we cannot cure it, that the best we can hope for is to slow it down, and that their child will probably not live much longer,” he says. 

It is a crushing conversation – one that drives Wang forward with the greatest sense of urgency.  

As a dad, he understands the stakes. As a scientist, he knows better outcomes are possible. And time is of the essence. 

Critical Seed Funding 

Wang began working at City of Hope in 2016 with the hope of developing a clinical trial for pediatric brain cancers using chimeric antigen receptor (CAR) T cell therapy. CAR T cell therapy is a type of immunotherapy that engineers a patient’s own T cells to fight cancer. City of Hope has one of the most comprehensive CAR T programs in the world. 

The Food and Drug Administration has approved several CAR T cell therapies to treat blood cancers, and the technique shows significant promise with solid tumors as well.  

Yet Wang says conventional funding agencies weren’t initially interested in funding a CAR T trial for pediatric brain tumors. They felt it was unlikely to succeed and too risky, especially for children.  

He remembers a time 25 years ago when people thought immunotherapy was radical and unproven. City of Hope scientists saw its potential and persisted in the research. Now, immunotherapy has made once-fatal forms of cancer curable, and it is considered one of the biggest medical advances of a generation. 

City of Hope donors generously supported that early work in immunotherapy and they would become critical in moving Wang’s work forward, too. 

“It is impossible for me to overstate the importance of philanthropy in the work that we do here. I came here to cure pediatric cancer. That is an extraordinarily ambitious goal, and I recognize that, but if we don't set our sights high, then we're limiting what we can accomplish,” he explains. 

So when Wang needed seed funding to initiate his most recent trial, longtime City of Hope supporters, including the National Business Products Industry group and the Panda Cares Foundation, once again stepped in to advance the research. 

Although the results were modest, the first two patients showed that the therapy was safe, and there was some indication that the reengineered cells might kill tumor cells. It was enough to convince the California Institute for Regenerative Medicine to provide an additional $8.4 million grant to back the Phase 1 trial. 

“It really takes the commitment and investment of our donor community. They understand that we're here to do great things and are willing to help us get there.” 

Hope for the Future 

Wang knows there is still a long way to go, but the research results are encouraging. He says none of it would be possible without donor support. 

“What we have accomplished thus far is truly remarkable,” he says. “Our supporters are giving families the precious gifts of hope, time and the possibility of a future. I'm not going to stop until we start curing patients. And I believe with the help of donors, we can get there.”