Jeffrey and Marieke Rothschild gift establishes Stanford Center for Cancer Cell Therapy

The Stanford Cancer Institute has received a $10 million gift from Silicon Valley entrepreneur Jeffrey Rothschild and his wife, Marieke, to advance research in cancer cell therapy, which is considered the vanguard of cancer treatment today.

The gift launches the Stanford Center for Cancer Cell Therapy, which will support research and clinical trials of treatments that use the power of the patients’ own immune system to attack and kill tumor cells.

“This gift to establish the center will enable us to test new, targeted cell therapies, which have the potential to transform our fight against cancer,” said Lloyd Minor, MD, dean of the School of Medicine. “We are immensely grateful to Jeff and Marieke Rothschild for their commitment to our precision health vision and their foresight in supporting this exciting venture.”

“We were interested in supporting promising research, and this is an area that I had been paying attention to,” Jeffrey Rothschild said. “There’s not been as much progress in cancer therapy as people had thought there might be 20 years ago. Here’s something which looks like a path that holds real promise, harnessing the immune system. That just seems very exciting.”

A computer scientist by training, Rothschild was the vice president of infrastructure engineering at Facebook and has co-founded several technology companies, including Veritas Software and Mpath Interactive.

‘Appetite for risk’

Marieke Rothschild said she and her husband are attracted to projects that may be high-risk but have the potential for meaningful and enduring social impact. Their philanthropic investments have been focused on health care and education, including scholarships for students who might not otherwise attend school. Five years ago, they established an eye hospital in western Kenya, where clinicians now provide care at little or no cost to 3,500 patients a year with cataracts and glaucoma.

“We have an appetite for risk,” Marieke Rothschild said. “We ask the question, ‘What can really yield results? What are the projects that are not being funded? Where, with a relatively small amount of funding, can you have impact?’”

The Center for Cancer Cell Therapy was established to directly benefit patients and spur innovation in a field that is considered one of the frontiers in cancer care.

The center will be led by Crystal Mackall, MD, professor of pediatrics and of medicine and one of the pioneers in the field of cancer cell therapy. Mackall’s work has focused on CAR T cells, immune cells engineered to express receptors that lock onto and destroy malignant cells. While at the National Cancer Institute, she led several clinical trials using these modified T cells to treat children with leukemia whose condition hadn’t improved with other therapies. The response rates were remarkable, with 70 to 90 percent of children improving with a single treatment. A variation on this treatment, now being tested in young patients at Lucile Packard Children’s Hospital Stanford, was recommended for approval by a Food and Drug Administration panel July 12 and could be the first CAR T cell therapy to reach the market.

‘Potentially transformational’

“I think it has the potential to be highly impactful. That’s why I’m committing myself to it,” Mackall said. “If we can optimize the functioning of these cells, they have the potential to effectively kill an established cancer and to remain functional for years after one infusion. That’s the goal — to have a product that will work on behalf of the patient. When optimized, they could remain active for months or years, preventing a recurrence of cancer. So for me, it’s potentially transformational.”

Among the challenges of these therapies is that they work for some patients but not others, and aren’t effective in treating all types of cancers. Scientists at Stanford Medicine and elsewhere are looking at ways to apply the therapies more broadly and to minimize potential side effects of the treatment.

I think it’s the next frontier.

“This is an incredibly promising approach to immunotherapy,” said Beverly Mitchell, MD, professor of medicine and director of the Stanford Cancer Institute. “I think it has to overcome some problems, one of which is that there is occasional toxicity. And many solid tumors have yet to respond, so there is a lot of research that needs to be done to bring it to the broadest spectrum of patients. I have great confidence that it will become the mainstay of treatment for leukemias and lymphomas and will then extend into solid tumors, including brain cancers and sarcoma. I think it’s the next frontier.”

She said the Rothschild gift will enable Stanford Medicine researchers to move forward with a series of clinical trials using variations of CAR T cells in different types of cancers. Until this point, these trials at Stanford have been industry-sponsored; the new gift will support the first investigator-initiated trial using a therapy that Mackall developed in her lab. Expected to begin in August, this trial may enroll up to 50 children and adults with B-cell leukemias and lymphomas, using a CAR T cell that involves a “double-punch,” simultaneously targeting the CD22 and CD19 antigens on cancer cells, Mackall said. Previous therapies have been directed at the CD19 antigen alone and the CD22 antigen alone, but over time, some patients lose these targets and no longer respond to the treatment, she said. In 2018, trials will expand to children and adults with brain tumors and children with solid tumors, such as neuroblastoma and osteosarcoma, she said.

David Miklos, MD, associate professor of medicine and specialist in bone marrow transplantation, will co-direct the new center, which includes many other Stanford Medicine physician-scientists who care for both adults and children with cancer. With the Rothschilds’ support, Stanford also will recruit new faculty in cancer immunotherapy to join the effort.

The new center is expected to complement the work of the Parker Institute for Cancer Immunotherapy at Stanford, which Mackall directs. The institute brings together six academic medical centers nationwide working collaboratively to advance the field.

As for the Rothschilds, they say their goal is to contribute to knowledge in the field.

“We just hope to be able to affect things in a positive way — have some impact,” Jeffrey Rothschild said. “That’s all you ever can hope for.”