Meet the architects who design treatment-advancing clinical trials
A cancer patient who is enrolled in a clinical trial that very well may save their life gets to know their oncologist or specialist, their nurses, and the many members of the care staff they see and speak with during their visits.
But they aren’t likely to meet an important group of people that plays a crucial role in designing that life-saving study, ensuring the proper information is included and analyzed. Most people are unaware that behind all successful clinical research are experts in biostatistics and bioinformatics.
“Biostatisticians and bioinformaticians are key players on the clinical research team, and an integral part of the clinical trial enterprise,” says Prithviraj Bose, M.D., associate professor of Leukemia. “The entire validity of the results of a clinical trial becomes questionable if the trial is not adequately ‘powered’ to assess its endpoints.”
This often involves complex calculations of study sample size and taking into account the many factors related to disease treatment, the drugs being tested and more, he adds.
Anirban Maitra, M.B.B.S., professor of Translational Molecular Pathology, agrees.
“No clinical trial can be designed without input from biostatistics. A poorly designed clinical trial is worse than no trial at all. We are keenly dependent on biostatisticians for ensuring that our clinical trials yield useful information for future generations of patients,” says Maitra, who is the scientific director for the Sheikh Ahmed Pancreatic Cancer Research Center. “Bioinformatics is critical in deciphering the wealth of new genomic data. The bioinformatics team uses sophisticated algorithms to elucidate markers of response and resistance to therapy, as well as determine how patients can be stratified appropriately for the most relevant therapies under study.”
Biostatisticians and bioinformaticians must have a high level of understanding of the research question being asked, and design the trial accordingly with the information available, setting reasonable expectations for a new treatment based on input from the clinical investigator.
Someone who knows a thing or two about the importance of data collection is Michael Andreeff, M.D., Ph.D., professor of Leukemia, who was a pioneer in the develop of flow cytometry, a method for counting and sorting cells that is commonly used in today’s clinical trials.
“In clinical studies, we are looking for genetic patterns that aren’t obvious. It is simply too much information and too complex,” says Andreeff. “With 20,000 genes, how is it even possible to understand what you must know in researching disease? Bioinformaticians are able to help us with that understanding through analysis of large databases such as The Cancer Genome Atlas, to see if data correlates with other studies.”
Deluged with data
The accessibility to information that has come with the growth of the Internet has been a boon for many industries and endeavors, and it’s been especially important for clinical trials. But the enormous amount of data available is too much for doctors hoping to find reliable treatments or even cures for cancer to take in. There is simply too much coming at them at a high rate of speed.
Enter biostatistics and bioinformatics.
“Modern medicine has generated unprecedented amounts of data. A combination of clinical, environmental and public health information, proliferation of associated genomic data, and increasingly complex digital information from sources such as electronic health records, social media, mobile health and imaging,” says Veera Baladandayuthapani, Ph.D., professor of Biostatistics. “Biostatistics maximizes access to, and usability of, such data to enhance, improve and inform decision-making, and is one of the pillars of cancer research. Today, more than ever, precision medicine is a data-driven and data-dependent endeavor.”
While biostatisticians help ensure a clinical trial is designed for success, bioinformaticians translate massive databases of cancer-related information that was virtually unknowable just two decades ago when the ambitious Human Genome Project. The effort, which began in 1990 and ended in 2003, mapped out the intricate details of every human gene, revolutionizing how clinical trials were designed, and what they were able to accomplish.
“Biostatisticians are vital because they help us separate truth from fiction, offer unbiased information, and are able to answer in-depth questions a clinical investigator wants to ask.”
Genomic explosion
For scientists like Han Liang, Ph.D., associate professor and deputy chair of Bioinformatics and Computational Biology, the Human Genome Project ignited a passion for big data that continues to this day.
“I was so fascinated by the fact that we could know each detail of the genome and use this information to understand our evolutionary journey of millions of years, and cure diseases for future generations,” says Liang. “Genomics has revolutionized modern medicine over the last decade, especially for cancers. As a result, bioinformatics is one of the fastest moving fields. New data, technology and analytic tools have emerged at an incredible speed, which excites me and makes me want to dig into them. I feel so lucky to be a bioinformatician in the era of ‘big data.’”
Work by John Weinstein, M.D., Ph.D., chair of Bioinformatics and Computational Biology, was critical to the creation of The Cancer Genome Atlas (TCGA), an ambitious project funded by the National Cancer Institute and the National Human Genome Research that has provided a wealth of genomic data related to cancer. His early work spearheading the comprehensive molecular profiling of 60 cancer cell lines presaged what became TCGA. The Human Genome Project, TCGA and other titanic scientific undertakings paved the way for collaboration among clinical researchers and today’s biostatisticians and bioinformaticians. Those projects have provided novel approaches to clinical trial design and the development of potential new therapies.
“Today we have a plethora of innovative and creative opportunities that range from early detection, biomarker discoveries and adaptive clinical trial designs,” says Baladandayuthapani. “Cancer is one of the most advanced and well-characterized disease systems. Certainly studies today must be data-driven.”
No clinical trial is successfully completed without a host of participants with their own individual expertise. It truly takes a team to create a cure from the seemingly endless mounds of interwoven data available today.
“I have worked with multiple clinical and science groups at MD Anderson that cut across multiple cancer types,” says Baladandayuthapani. “One of the things that drew me into this field was the ability to connect with a broad range of scientists and find diverse, high-impact problems to work on. This modern era of big data has presented considerable challenges and opportunities that will keep us occupied for years to come.”
