A Look Back at Research in 2024

Marosh; Flickr

The beginning of a new year brings with it a renewed sense of optimism and hope.

It’s a time when we often ask ourselves, did we do better, and did we do more. When it comes to pancreatic cancer research, the answer to both of those questions is yes. In the past year we gained a better understanding of the fundamental biology of the disease. And with that understanding, the promise of new and better treatments has either come to fruition or is on the horizon.

Here is a brief recap of just some of the new research during 2024. As we enter 2025, the Let’s Win team will continue to cover these breakthroughs so that you can make more informed decisions as you move forward.

A Potential Game Changer for Treatment of Cachexia

An experimental drug called ponsegromab developed by Pfizer may be an effective treatment for a wasting syndrome called cachexia that often affects people with cancer, according to results from a clinical trial published in September in the New England Journal of Medicine. Cachexia is the unintentional loss of a significant amount of weight, both from fat and skeletal muscle, which harms quality of life and is estimated to cause as many as 30 percent of deaths in some cancer types.

In the clinical trial, 187 people with advanced lung, colorectal, or pancreatic cancer had to have unintentionally lost at least 5 percent of their body weight over the previous six months. The participants also must have had elevated blood levels of GDF-15, a type of protein known as a cytokine, which helps cells adapt and respond to changes in their surroundings. Participants treated with ponsegromab gained anywhere from two to five pounds on average over 12 weeks, depending on the dose they received. By comparison, people treated with the placebo lost an average of one pound. The group of participants treated with the highest dose, 400 mg, gained back more than 5 percent of their body weight.

A larger trial of the drug is planned for 2025. And if the trial produces similarly positive results, it could lead to the drug’s approval by the Food and Drug Administration.

Targeting RAS

Once considered “undruggable,” KRAS research is booming, and data continues to look promising.

Of particular interest is the investigational agent RMC-6236, a drug developed by Revolution Medicines (Redwood City, California). This is the first targeted drug designed to directly inhibit all major forms of oncogenic RAS, the major drivers of pancreatic cancer. It has shown antitumor activity and a favorable safety profile in a broad population of patients with previously treated pancreatic cancer.

Based on positive earlier data, a global, randomized, open label, phase III study is now open. It is designed to evaluate whether treatment with RMC-6236 will improve progression-free survival or overall survival compared to standard of care chemotherapy in patients with metastatic pancreatic cancer who were previously treated with one prior line of therapy with a 5-fluorouracil-based or gemcitabine-based regimen.

More Treatment Options in Metastatic Disease

The FDA approved irinotecan liposome (ONIVYDE) in combination with oxaliplatin, fluorouracil, and leucovorin, for the frontline treatment of metastatic pancreatic adenocarcinoma. The combo, dubbed NALIRIFOX, has been shown to improve overall survival for a disease that has far too few treatment options. This is the first new treatment approved for first-line treatment of metastatic pancreatic cancer since the September 2013 approval of nab-paclitaxel (Abraxane) in combination with gemcitabine.

Landmark Study Moves Forward

Biomarker-based subtypes of pancreatic cancer have become an important focus of research efforts. Many scientists now think subtypes may be the key to more effectively treating the disease. The landmark PASS-01 study, a collaboration between a group of the foremost cancer centers in North America, focuses on exploring personalized medicine in pancreatic cancer using cutting-edge organoid technology. As reported in 2024, the researchers continue to see consistent differences in overall survival between the FOLFIRINOX (FFX) cohort and the gemcitabine/nab-paclitaxel cohort (GnP). At this point, there is better overall survival with the GnP group. Molecular profiling data is available for 90 percent of patients, which allows researchers to look at subgroups and predictors of response. What they have found is the basal-like subtype is the most predictive prognostic variable with worse outcomes. This group responds poorly to FFX. Epidemiological risks, such as smoking and obesity, among others, are not associated with basal or classical subtypes.

Trial Finder Debuts

Clinical trials not only advance our understanding of pancreatic cancer. In many instances, these trials offer the best chance of improving and prolonging life. Finding clinical trials can be daunting. To better serve our community, Let’s Win developed its very own Trial Finder in late 2024. Our Trial Finder makes identifying potential trials simple. Patients and caregivers can search for trials by exploring on their own, answering a few questions to help direct the Trial Finder, or getting in touch with a dedicated Trial Navigator. Patients and caregivers can easily explore available clinical trials based on location, prior treatment, diagnosis, tumor type, and more. Our Trial Finder is available in English and Spanish. Please remember, clinical trials are not the last resort. Rather, pancreatic cancer patients are encouraged to investigate clinical trials as part of their initial treatment.

On behalf of Let’s Win, we wish you and those you love all good things in 2025. And we thank you for your unwavering support.