Bio-Science: André Rogatko, PhD

"I wanted to know about the important features of the world, so I'd make that not-easy trip," said Rogatko, director of the Biostatistics Shared Resource at Cedars-Sinai.

That youthful drive to do what is important but not easy has never waned. Today Rogatko is widely recognized as a talented biostatistician and accomplished investigator. He trains his arsenal of complex statistical methods at an immeasurably important but decidedly not easy target: the reduction of human suffering.

His efforts, over four decades, have earned Rogatko the esteem of his colleagues, who benefit from his advice and collaboration. Most recently, he was awarded $3.4 million from the  National Cancer Institute (NCI) to study the adverse impacts of experimental therapies on clinical trial participants—one of the few biostatisticians to receive an NCI grant.

Rogatko has been the primary investigator or co-investigator for some 50 studies; published nearly 130 research papers; contributed chapters to more than 10 books; and developed nine computer applications in biostatistics and genetic epidemiology.

One of his singular contributions is a clinical trial design known as Escalation With Overdose Control (EWOC).

Early in his career, Rogatko concluded that the standard dose-finding method most investigators were using in Phase I clinical trials was inefficient and put patients at risk for receiving excessively high doses and experiencing severe side effects. In 1998, he countered with EWOC, a Bayesian adaptive design enabling investigators to continually refine doses based on patients' responses and other data, while simultaneously guarding against the toxic effects of overdosing.

Rogatko and his nine-person team have since built an EWOC web portal and further refined EWOC's design. EWOC tools have been downloaded thousands of times by investigators worldwide.

"I'm always thinking about those patients who took part in a clinical trial and not only weren't helped by the experimental drug, but also suffered serious side effects. Then there are others who benefited from the treatment, but were left with long-term side effects like neuropathy or frequent falling," Rogatko said.

He also has developed novel statistical approaches to help diagnose certain diseases and isolated disease biomarkers that have proven to be predictive of both adverse and positive responses to treatment.

Rogatko's patient-centered mindset is partially rooted in his family tree. His mother was a dentist, and her grandfather, Ludwik Rydygier, MD,  (1850-1920) was widely recognized as the "father" of gastrointestinal surgery in Poland. Rogatko's parents emigrated from Poland to Brazil, where his father was a diplomat at the Polish Consulate in São Paulo before the Second World War.

Rogatko has fond memories of growing up in São Paulo. When he wasn't trekking to historic cities, he often was scuba-diving, a pastime he still enjoys. He also is drawn to airborne adventures. For many years he flew single-engine planes, and this past summer he had a "fantastic experience" gliding high above Southern California's high desert in an engine-free plane.

While he still visits Brazil, Rogatko has called America home for more than 30 years.

After earning a PhD in genetics and statistics at the University of São Paulo, Rogatko did a postdoctoral fellowship at New York City's Memorial Sloan Kettering Cancer Center, where he spent several years as a biostatistician.

At Philadelphia's Fox Chase Cancer Center, he rose to become chairman of the Department of Biostatistics. Rogatko then spent several years at Atlanta's Emory University as a professor in the Department of Biostatistics and Bioinformatics, where he helped the university's Winship Cancer Institute receive an NCI comprehensive cancer center designation.

Since joining Cedars-Sinai 11 years ago, Rogatko and his team have fulfilled more than 1,600 requests from colleagues for biostatistics and bioinformatics support and authored or co-authored nearly 370 papers published in peer-reviewed journals.

"We probably review 10 study designs per week so we can spot typical errors," said Rogatko, who advises investigators to visit his team early in the process, when they have their hypotheses.

Among those who have collaborated with Rogatko is Patrick Lyden, MD, professor of Neurology and leader of a multicenter research program called the Stroke Preclinical Assessment Network (SPAN). Cedars-Sinai is the coordinating center.

"When I started thinking about applying for the SPAN grant, André was the first person I called," Lyden said. "André is the brains behind that grant. One of the things the SPAN reviewers liked most about my proposal was the statistical design André contributed."

Noel Bairey Merz, MD, professor of Medicine, director of the Barbra Streisand Women's Heart Center and the Linda Joy Pollin Women's Heart Health Program in the Smidt Heart Institute, has collaborated with Rogatko on multiple studies and clinical trials. "André has great ideas and is very disciplined," she said. "He's one of my favorite people."

The current project Rogatko is pursuing with his NCI grant epitomizes his patient-focused career trajectory. He is working with Patricia Ganz, MD, professor of Medicine in the David Geffen School of Medicine at UCLA.

Together, they are analyzing data from past and present clinical trials with an eye toward developing statistical methods to better assess the adverse impact of experimental therapies on trial participants and incorporate patients' feedback about their treatment.

"We have a chance to give patients more power in how they want to be treated," Rogatko explained.