"From the bench to the bedside" is a phrase often used to describe a drug discovery's journey from the laboratory to the clinic, where patients benefit.
MD Anderson's Therapeutics Discovery division, however, takes a different approach, by beginning with the bench at the bedside. This drug discovery engine conducts research informed by the clinic, from start to finish.
"We are completely driven by unmet needs we see in the patients that come to MD Anderson for help," says Phil Jones, Ph.D., vice president of Therapeutics Discovery. "Guided by the expertise of our world-class clinicians, our efforts begin with the patient and their cancer. The Therapeutics Discovery model is designed to develop new treatments to meet their needs."
Composed of three Moon Shots Program™ platforms and the Neurodegeneration Consortium, Therapeutics Discovery is working hard to bring transformational, life-saving medicines to patients quickly, safely and effectively. These medicines range from new chemical compounds to antibodies and cell-based therapies.
Unlike typical pharmaceutical companies, Therapeutics Discovery was built within the walls of MD Anderson, placing drug development expertise in unparalleled proximity to patients and leading physicians. It's a recipe for success that already is yielding promising results.
And at the heart of it all are more than 100 scientists, driven by a passion to see their work one day save a patient's life.
The Institute for Applied Cancer Science (IACS)
IACS is devoted to inventing new small-molecule drugs, or chemical compounds, that target specific vulnerabilities in cancer cells.
Principal research scientist Mick Soth, Ph.D., is a lead chemist for one of IACS’ drug discovery projects, and responsible for designing the safest and most effective compounds possible.
Soth spent more than a decade working for a major pharmaceutical company, but grew increasingly frustrated by limited successes and a lack of meaningful collaborations. He chose to join MD Anderson five years ago to find a new, more productive environment for drug development.
"I'm very excited about building something here," Soth says. "We're in a great spot to do drug discovery, with direct connections to MD Anderson clinicians, and we're building up what could become an operation that will elevate the institution, Houston and the state of Texas. That's really cool."
Soth already has two projects advancing to clinical trials early next year. That type of early success, coupled with tremendous cross-communication in the division, was exactly what he was looking for.
But what ultimately makes it all worthwhile?
"The first patient that is actually helped," Soth says.
Oncology Research for Biologics and Immunotherapy Translation (ORBIT)
The ORBIT platform develops antibodies that recognize specific targets to either seek and destroy cancer cells or stimulate the immune system against a tumor.
Dongxing Zha, Ph.D., associate director of ORBIT, brought decades of research and antibody development experience with him when he joined MD Anderson four years ago. He also brought personal experience, having previously lost his father to gastric cancer.
"Cancer is a horrible, horrible disease that affects almost everyone directly or indirectly," Zha says. "I really want to contribute and help find new drugs to help those patients who desperately need new therapies."
Zha was excited by the possibilities at MD Anderson, knowing he'd work closely with leading investigators and draw from their deep understanding of cancer biology. With his team's expertise in drug development, he felt it was a win-win opportunity.
In a few short years, his team already has developed two drugs, one now in clinical trials and another soon to be.
"I'm very excited and extremely proud of our team, and it's only possible at MD Anderson," says Zha. "We couldn't deliver this anywhere else."
Translational Research to Advance Therapeutics and Innovation in Oncology (TRACTION)
Therapeutics Discovery's TRACTION platform works at the crossroads of preclinical testing and validation of new drugs, which leads to a better understanding of how the drugs that are developed work and who will benefit most from them.
Angela Harris, an associate scientist on the in vivo pharmacology team, was born and raised in Houston. For her, working at MD Anderson was a "dream job." She worked for 12 years with a Houston-area pharmaceutical group, but jumped at the chance to bring her expertise to MD Anderson four years ago.
"It's a great privilege to interact and collaborate with all the amazing scientists, project leaders and clinicians here," she says. "It's all teamwork, and that collaborative environment is so important to success."
Harris conducts preclinical experiments with new therapeutics to learn how safe and effective they might be for treating cancer in humans. The results of her team's work form the basis for decisions on whether or not to move into clinical trials and which patients should be treated with the new therapies.
"That's what we're all here for," she says. "I think we will make a difference in patients' lives. That's what motivates me to be here. If anything, if what we're working on is able to help patients, that would be tremendously fulfilling."
Neurodegeneration Consortium (NDC)
The NDC is a multi-institutional initiative established in 2012 by an inaugural gift of $25 million from the Robert A. and Renee Belfer Family Foundation to better understand neurodegenerative diseases and develop new therapies to treat them. The NDC includes researchers from Therapeutics Discovery, Baylor College of Medicine, the Massachusetts Institute of Technology and the Icahn School of Medicine at Mount Sinai.
Paul Acton came to the NDC with a personal and professional passion to take on Alzheimer's disease. Having watched four people in his family diagnosed with or succumb to the disease, he chose to devote himself to developing new drugs more than 25 years ago.
"It's definitely something that is real for me and gets me out of bed in the morning," Acton says.
He has worked for a number of major pharmaceutical companies over the years, but he never thought he'd work at a cancer center. However, he was impressed by the talent of the colleagues in Therapeutics Discovery and the infrastructure available at MD Anderson.
As a senior research scientist, Acton leads drug discovery efforts for the NDC. While the focus of the NDC’s work is Alzheimer’s disease, the drugs developed there may also benefit cancer patients suffering from the neurodegenerative and neurocognitive side effects of chemotherapy.
Acton has lost several family members to cancer and has witnessed these side effects. Driven by this experience, he has made it one of his goals to help these patients.
"My hope is to get a drug not just into the clinic, but through the clinic and to the patients to make a difference in their lives."
Recent Therapeutics Discovery accomplishments
Researchers in IACS and TRACTION have discovered a drug that blocks a vital metabolic process required for cancer cells' growth and survival. The drug, IACS-10759, is now in clinical trials for AML and solid tumors.
In 2018, MD Anderson and Accelerator Life Science Partners launched Magnolia Neurosciences, a company developing neuroprotective medicines based on discoveries made by IACS and the NDC.
Researchers in the ORBIT platform developed a novel antibody, h8F4, which targets and selectively kills AML cancer cells. With the support of Astellas Pharma Inc., the new therapeutic will soon enter clinical trials.