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Why Clinical Trials Are Critical

Breakthroughs, Breakthroughs • Spring 2024
by Naedine Hazell


Talk with Dr. Patricia LoRusso, president of American Association for Cancer Research

The halls of Yale Cancer Center rarely ring with anything other than footsteps, hurried hellos, and the occasional polite mention that a meeting has let out and there are leftover bagels in the kitchenette. The day to day action takes place in 16 Smilow Cancer hospital locations, the labs, and the research programs.

But on some days, Patricia M. LoRusso, DO, PhD(h), professor of Medicine (Medical Oncology), Yale School of Medicine, chief of the Early Therapeutics Program, and Associate Cancer Center Director for Experimental Therapeutics is in her YCC office and she brings a unique energy.

“Hey, how you doing? Yeah, it’s been a while,” Dr. LoRusso says, smiling into her phone. She is behind her office desk with the door open, but it is as if she is beside the person on the other end of the phone. “Hey listen, I’m calling about…yeah, that trial. Is there any way we can fit one more? I’ve got this patient, just saw her today….”

Dr. LoRusso’s heart is always in clinic. And when she’s not physically there, it seems she’s talking about clinic, or bargaining to improve patient support in clinical trials, or advising why there should be more or greater access to clinical trials.

Clinical trials are among the critical final steps in bringing a new treatment to patients with cancer and they typically follow years of earlier research, modeling using mice, other preclinical model systems and published findings. Successful clinical trials with patients are necessary to obtain federal approval for a treatment’s wider user. Dr. LoRusso’s message about the importance of clinical trials is similar whether she’s talking to a patient or is in an advisory meeting at the White House, as she was a few months ago, or part of a panel at the annual "People v. Cancer" symposium hosted by the national magazine, The Atlantic.

“Less than 3% of [cancer] patients are going on clinical trials and the numbers are declining,” Dr. LoRusso told The Atlantic audience in November.

As potential drug treatments become increasingly focused, the need for more trials is critical and they need to include more diverse patients. “There was a publication last year in JAMA that showed that up to 40% of clinical trials had no underrepresented patients. It’s important to have representation of these populations because we have to understand, before these drugs become FDA-approved for commercial use, how these drugs are going to act in all patients” in terms of benefits and toxicities.

An internationally recognized expert in drug development and early-phase clinical investigation of novel therapies, Dr. LoRusso has always done clinical research.

At present, YCC has 26 actively enrolling phase 1 trials, and Dr. LoRusso and team are working to increase accessibility and participation. The intent is to improve education tools, including “myth busting” messaging about trials on monitors in Smilow clinics; continued coordination with Community Engagement and Health Equity (the COE program at YCC); and less cumbersome informed consent forms.

In addition to recruiting additional faculty, as the early phase program is growing along with the excitement in the novel drug therapy landscape, Dr. LoRusso hopes to recruit 250 patients to early phase trials of novel therapies in 2024 and to expand efforts beyond the Smilow site in Bridgeport/Trumbull.

It’s a heavy lift for 2024 given that, in April, Dr. LoRusso became president of AACR—the American Association of Cancer Research—the largest cancer research program internationally, with a membership of 58,000. But the pull of her patients will remain.

“I love what I do,” she said. “It humbles you. It makes one realize how blessed we are to be healthy, and how very much of a hero each of patients are, for helping to advance new treatments.

“You’ve got to remember that the patients, for the most part, have advanced disease…many of them have no other options. They are the heroes. They are giving so much time, of what little time that they have left. They go on trials in the hopes that they respond to the new therapies, often having failed all standard drugs that are typically given to patients with their disease. They are hoping that participation will allow them to live longer, but they also are helping develop new drugs for mankind,” Dr. LoRusso said.

Dr. LoRusso uses the word “phenomenal” to describe the leap forward in cancer research enabled by the groundbreaking work on the human genome, new lab technologies and computational advancements. The drugs being developed are more selective for specific targets and tumor types, meaning they benefit thinner slices of the cancer patient population, but bring the potential for more targeted treatment and hopefully all this translates to a greater response and a longer life.

As for the always hopeful prediction that a cancer cure is closer every day, Dr. LoRusso allows that “We know a lot, but I don’t think we are there. We are closer, but we’re not on the cusp — we have quite a long way to go.”

“As we become more knowledgeable, we realize the many different faces and diseases of cancer. Each patient, even with a similar diagnosis, has a different disease. We are not at the level of personalization yet that I think we need to be to have the greatest impact,” she said.

“That’s going to come with more translational research, more bedside to bench research, more clinical research in terms of novel treatments, designs and approaches to giving drugs—which I think is hopefully going to translate to better individualized therapies for patients,” she said, emphasizing the importance of the process being bi-directional—from lab bench to clinical bedside and then back to the bench.

Still, Dr. LoRusso is quick to note the impressive advances and she’s seen many since she began. Her career in cancer research can be described as beginning before she even graduated high school. That was when she lost both parents to cancer in her youth.

“I always look at cancer as my enemy. It’s been my enemy for many, many years. When my parents were sick, there were no drugs,” she told an AACR interviewer in a video produced in 2022. The video was made when she won the prestigious Joseph H. Burchenal Award for Outstanding Achievement in Clinical Cancer Research.

“For clinical research, your greatest resource is the patients — the most precious, valuable resource.”

So, while the quest for cures continues, breakthrough treatments that sabotage or frustrate cancer’s return or its growth still translate to lives saved and years lived. “That’s the fuel that has kept me going,” Dr. LoRusso said.

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