LustgartenLIVE! Best of 2021: Research Highlights

LustgartenLIVE! webinar is made possible by the support of Ipsen and Elevation Oncology

It’s truly the most wonderful time of the year—a time to reflect on the last 12 months and recognize our accomplishments; and to build momentum and excitement for the work ahead.

We often proclaim, “pancreatic cancer research is progressing faster than ever,” and it remains true. Like the proverbial snowball barreling down the mountainside, the speed of pancreatic cancer discoveries accelerates as the base of knowledge—the snowball—builds over time and distance. And because time is everything to pancreatic cancer patients and their families, the Lustgarten Foundation is committed to advancing the most innovative and intelligent research possible to transform pancreatic cancer into a curable disease.

The Foundation also is committed to sharing that knowledge and excitement with supporters. In April, we invited you to join us for LustgartenLIVE!, quarterly interactive discussions with the best researchers investigating the disease. We kicked off the year with an update of Lustgarten-funded research into earlier detection, then explored the transformative power of personalized medicine and later shared our unique approach to spurring the rapid development of clinical advances. In December, we bookended the inaugural series sponsored by Ipsen and Elevation Oncology with Chief Scientist David Tuveson, MD, PhD, of Cold Spring Harbor Laboratory, and Chief Medical Advisor Elizabeth Jaffee, MD, from Johns Hopkins, with a compelling look back at what has been an exceptional year of research progress and offer a thrilling preview for 2022. Watch the webinar here.

Ambition, Need and Progress

Beginning with the January appointment of new CEO Linda Tantawi, 2021 was a year of change, growth and transformation. In addition to LustgartenLIVE!, the Foundation created several exciting equity and diversity initiatives, including the 2021 John Lewis and Ruth Bader Ginsburg Career Development Awards and the Lustgarten Equity, Accessibility and Diversity (LEAD) project, which will begin in 2022. We developed new partnerships, funded ground-breaking clinical trials and doubled down on identifying and funding research in the areas of greatest need for patients—earlier detection, new therapeutics and personalized medicine—all of which depend upon closing the gaps in scientific knowledge.

Strategic Research Priorities

“Tying together the areas of need and progress leads to patient impact,” said Andrew Rakeman, PhD, VP Research. “Lustgarten’s focus on creating patient impact is paying off, through the development of what we call liquid biopsies to detect all kinds of cancers, including pancreatic cancer, as well as other research advancements.”

As the largest private funder of pancreatic cancer research in the world, the Lustgarten Foundation is steadfast in our focus on patient impact. 100% of donations fund high-risk, high-reward pancreatic cancer research in three distinct priorities:

• Earlier Detection
• Drug Development
• Personalized Medicine

The focus on and early investment in basic, foundational science and understanding the biology underlying genetics and genetic changes is why we can confidently claim Lustgarten-funded science has been a driving force in every major advancement in pancreatic cancer. In funding the basic science of the disease, we have laid the groundwork for research to follow.

Earlier Detection

“The investment in developing an early detection test has been going on for several decades,” said Dr. Tuveson. “It started with Lustgarten investing in determining what genes are mutated in pancreatic cancer, studying the cancer cell genome leading to the development of tests looking for changes in the blood that could tell us the patient has a tumor, even a tiny tumor.

“The tumors we’re trying to detect are the size of a grain of rice; however, there aren’t tests in medicine where you can see a grain of rice on a scan. We are trying to come up with the most sensitive ways to do this, like CancerSEEK,” Tuveson added. The CancerSEEK research lays the foundation for a single blood screening test for multiple cancers that could be offered as part of routine medical checks and represents a significant step forward in how pancreatic cancer may be diagnosed in the future.

CancerSEEK has been acquired by Exact Sciences, a major test developer in the U.S., now conducting the clinical tests necessary to eventually bring the science to market.

Tuveson noted, “We know genes are part of cancer—you need bad genes to develop cancer—but that’s not enough. Other things have to happen, so we’re looking for those other things, other events, and we now have a pretty good idea of what they might be: inflammation, development of stroma (supportive tissue around the cancer cell), changes in metabolism and metastases.

Drug Development

Diagnosing pancreatic cancer in patients is one thing, but patients need safer, more effective treatments they can tolerate to eliminate pancreatic cancer at any stage.

Dr. Rakeman said one of the most exciting developments in the last year is the launch of the Clinical Accelerator Initiative (CAI), under Dr. Liz Jaffee’s leadership. “The groundbreaking project was born on the recognition that there is lots of great science coming out of the lab, where there are potentially new ways to get at pancreatic cancer.”

Dr. Jaffee, an internationally renowned expert in immunology, agreed. “Pancreatic cancer patients historically have not had a lot of clinical trial options based on understanding pancreatic cancer. We often repurposed drugs that were working in other cancers for this reason, because we didn’t have a lot of science enlightening us as to how pancreatic cancers develop and metastasize.”

With a deeper understanding of the biology of pancreatic cancer, we can now create rapid test agents, new drugs developed based on insight from patients with pancreatic cancer. “Designed to shorten the time from clinical trial concept to launch, the Clinical Accelerator Initiative brings fast and nimble clinical trials to current patients and holds the promise of discovering new treatments at an accelerated pace to improve patient outcomes,” she added.

These “smarter” clinical trials generate large volumes of data “so if a trial’s working, we’ll continue it,” Jaffee said. “But if a trial isn’t working, we’ll stop it, learn from the information and then make the trial, make the drug better, or add new drugs so that that drug can work more effectively.”

Since launching earlier this year, four clinical trials have been funded through the CIA, including a project from Tyler Jacks, PhD, who leads the Lustgarten Dedicated Pancreatic Cancer Research Lab at MIT. By looking at the immune system and why it wasn’t effectively targeting pancreatic cancer, scientists discovered a promising triple drug combination “effective at targeting one of the immune cells critical to fighting pancreatic cancer that can redirect the immune system to recognize the cancer and kill it,” Dr. Jaffee explained.

Meanwhile, in concert with the CAI, Lustgarten’s new Therapeutics-Focused Research Program is advancing the understanding of the causes and progression of pancreatic cancer to identify new potential therapies.

“Pancreatic cancer is a pretty tough warrior, as you all know,” said Dr. Tuveson. “But even tough competitors have an Achilles heel” and that’s essentially what the Therapeutics program is about: seeking new ideas for discovering and exploiting pancreatic cancer’s weaknesses. A total of 50 proposals underwent a rigorous review process by external experts and ten were selected for funding—an investment of $11.2 million over the next three years.

“Liz and I bring two different perspectives to the table about how to tackle pancreatic cancer,” he said. “I’ve been trying to pose potential avenues that then Liz and her team of really sophisticated and excellent medical oncologists and surgical and radiation oncologists can work on in small clinical trials.”  The target being the disease’s Achilles heel, where arrows from the scientific quiver can hit, and ultimately defeat, pancreatic cancer.

“That we can talk about launching multiple science-driven new trials in a year and thinking about different ways to target the disease demonstrates just how far we’ve come,” said Dr. Rakeman. “So how, when do we know we have enough trials? What’s the goal here?”

Dr. Jaffee responded, “We’re not done learning.

“Until every single patient that I’ve ever seen, or am going to see, can tell me they don’t have metastatic pancreatic cancer anymore, that they feel good, that they’re living a normal life, I don’t think we’re done. But what I will tell you is that more than ever, I am optimistic that we’re making progress.

“We’re learning how to make these drugs better and…patients are already benefiting. Even 10 years ago, when I signed someone up for a clinical trial, I’d have to say, ‘this is experimental. It’s never going to work.’ I don’t have to say that anymore because our drugs have been working—not as well as we’d like—but clinical trials are out there. And it’s very important that patients consider them as part of their therapy, that this is really part of the entire therapeutic plan for cancer patients,” Dr. Jaffee enthused.

Personalized Medicine

2021 was a big year for personalized medicine in pancreatic cancer, particularly the launch of the PASS-01 trial. An international collaboration between the Pancreatic Cancer Collective—Lustgarten’s strategic partnership with Stand Up To Cancer—and Pancreatic Cancer Canada, testing the two standard-of-care chemotherapy treatments for advanced pancreatic cancer (FOLFIRINOX and a combination of gemcitabine and Abraxane, commonly referred to as Gemabraxane). Both treatments benefit about 35% of patients.

“The problem is we don’t know who is going to benefit upfront. And just because you benefit from one, that doesn’t mean you’re going to benefit from the other,” said Dr. Jaffee. Participants are newly diagnosed with metastatic pancreatic cancer, allowing scientists to obtain pre-treatment biopsies. Additional biopsies are collected during treatment to help the team better understand what is happening inside the tumors.

“We’re doing genetics. We’re doing inflammatory analysis. We’re looking at the stroma. We’re looking at panels of biomarkers that both David (Tuveson) and others have predicted might be a benefit to these patients. And we’re hoping that at the end of this study, we will have specific biomarkers to tell a patient: Go on the FOLFIRINOX. Go on the Gemabraxane.”

Another significant, cutting-edge component is the additional step of building organoids from each patient’s tumor. Cells from the pretreatment biopsy are sent to the Lustgarten-funded dedicated organoid lab at Cold Spring Harbor Laboratory. “These organoids are basically 3D structures of the patient’s tumor…that we’re able to test a whole panel of different drugs on to determine which of these drugs might be the next viable treatment.

“If this works, we’ll be able to better predict for patients, what is the best treatment for each individual,” Dr. Jaffee said.

“That’s a great example of how because of investment in this early science—whether it was the development of the organoids or how to apply some of these other profiling tools that really enables this to move forward—right now is such an exciting time to make progress and to turn discoveries into impact for patients,” responded Dr. Rakeman.

Going Forward

The Lustgarten Foundation is truly the lynchpin of the pancreatic cancer community. We’ve been funding the best and brightest researchers in the world for years. That investment is now paying off in real results and improved outcomes. Like the snowball, we’re gaining speed to bring more hope and better options to patients today and in the future.

LustgartenLIVE! will continue—and expand—in 2022. Mark your calendars for the following dates, sponsored by View Ray, a global MRI-Guided Radiation Therapy cancer treatment company:

• March 8: Women Rule the (pancreatic cancer research) World!
• March 30: Doctors Day — From the Clinic to the Lab to You: How Research, Led by Lustgarten, is Transforming Patient Care at Your Local Cancer Center
• June 20: A Juneteenth Conversation: Catalyzing Workforce Diversity to Understand Disparities in Pancreatic Cancer Among African Americans
• November TBD: Pancreatic Cancer Awareness Month — The Intersection of Science & Technology
• December TBD: The Best of 2022: Research Highlights