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The PancChat Podcast is a collaborative effort from Let’s Win Pancreatic Cancer and the Pancreatic Cancer Action Network (PanCAN), inspired by the long-running #PancChat Twitter/X chat.
Hosted by award-winning journalist Alisyn Camerota, each episode features conversations with leading researchers, clinicians, patients, and advocates who are shaping the future of pancreatic cancer care and research. Together, we deliver expert insights, personal journeys, and the latest breakthroughs—bridging the gap between science and lived experience.
Whether you’re a patient, caregiver, healthcare professional, or simply want to learn more, join us to connect, be inspired, and learn how you can help to accelerate progress in the fight against pancreatic cancer.
Julie Fleshman: Hi, I'm Julie Fleshman, President and CEO of PanCAN. On today’s podcast, we will learn about the pancreatic cancer clinical trials and emerging treatments coming down the pipeline within the next year.
Alisyn Camerota: Hi everyone, welcome to PancChat. I'm your host, Alisyn Camerota. We want to thank our sponsor, Revolution Medicines. Now, in our last episode, we talked about all the advancements in pancreatic cancer treatment that have come into practice this year. Today, our focus shifts to the emerging trials and treatments that are on the horizon next year and what those mean for patients and their families.
We're talking once again to two doctors on the front lines of emerging treatments, Dr. Allyson Ocean and Dr. Anna Berkenblit. Dr. Ocean is the director of the Pancreatic Cancer Research Program and professor of clinical medicine at Weill Cornell Medical College of Cornell University, as well as a medical oncologist and attending physician in gastrointestinal oncology at New York Presbyterian Hospital, Weill Cornell Medical Center. She is a co-founder of Let's Win and chair of the Scientific Advisory Board.
Dr. Berkenblit is a medical oncologist and has more than two decades of experience in clinical development of novel anticancer therapies in the life science industry. She is the chief scientific and medical officer for PanCAN and is responsible for the organization's overall scientific and clinical strategy. Welcome back, both of you.
Allyson Ocean: Thank you.
Dr. Anna Berkenblit: Thank you.
Alisyn Camerota: Thank you. Great to talk to you again. Okay, so I always learn so much whenever we're all in conversation, and I know this will be the same. Okay, so let's start with talking about vaccines. We'll just pick up where we were talking about them last.
So, Dr. Berkenblit, you were describing these two different paths, or types, of vaccines. They're both in clinical trials. Do you know yet the outcome?
Anna Berkenblit: So these two vaccine strategies are intended to prevent the cancer from coming back again after it has been surgically resected. And both of them have been studied in early studies that looked promising. They were called single-arm studies, where patients received the vaccines after their tumor was resected. And it looked like the immune system responded, which is good. So the first question is, does the immune system respond to the vaccine?
And if the answer is yes, that's great. The next question is: Does that immune response work so well that it prevents the cancer from coming back again? And in order to answer that question, you really need to do a randomized study, which means some patients get the vaccine and some patients are observed. And if it turns out that the people who get the vaccine, if it takes longer for their cancer to come back again, which is called disease-free survival, that is a win because maybe it even means that the cancer will never come back again, and maybe they're cured because their immune system mopped up whatever little bits of cancer there are. So we need randomized trials to demonstrate the efficacy and better understand the safety of these vaccines.
So both of them have been in randomized trials. There's one that is still ongoing. It is the Genentech BioNTech bespoke vaccine that is made from the tumor's specific new mutated bits of protein. The antibodies are generated to that. And that study is still ongoing.
We look forward to the results of that randomized trial. The one randomized trial that we do have data for now is from a company called Elicio Therapeutics, their phase 2 AMPLIFY-7P study. It's called 7P because it has seven different little bits of protein from the RAS protein that are mutated most commonly in pancreatic cancer. And the vaccines are directed toward those seven most common types. So that AMPLIFY-7P study, unfortunately, in the entire group that it was studied in, compared with observation, it did not prevent the cancer from coming back again in terms of disease-free survival.
But it turned out that there were more patients who received the vaccine who had a worse prognosis. They were probably not going to do so well because when the cancer was removed by the surgeon, there was either tumor right at the margin. So the surgeon cut through the tumor, and there was leftover tumor, or it was just within one millimeter of the margin, the edge of what the surgeon resected. So in those patients, it looks like the vaccine didn't work so well, but in the patients who had what's called an R0 resection, where there was no tumor within, or near the surgical resection margin, those patients did seem to do better in terms of their disease not coming back again. And so those data are going to be used to design the next trial for us to really understand who is going to benefit from these vaccine strategies.
Alisyn Camerota: Dr. Ocean, anything you wanna add to that?
Allyson Ocean: No, I thought that was a great explanation. I think that this is by no means the end of vaccines in this space. I think that sometimes negative information is very helpful and helps us. Like Dr. Berkenblit said, design the next trial where the right patients are going to be enrolled so that we can see the most benefit from the intervention. And having had that trial open at Cornell and seeing the patients receive this vaccine, just being on the trial was a form of hope for these patients. I feel like that may have helped somewhat, anyway, just being on the trial, knowing that they're getting something novel and trying to get something to prevent it from coming back.
I know that isn't statistically proving anything, but I just feel like when patients are able to be on the cutting edge of science by entering clinical trials, it really does put hope into their journey, and really makes it – there's just hope that better things can come, and I think that that's really important because always having hope that better things are coming our way.
Alisyn Camerota: Well, I agree with you. I think that hope is a big part of the equation of success. But like you, I can't prove that with the data, but I certainly felt that when my husband was going through it.
So, to both of you, can you just explain for our listeners the process of a scientist having a great idea? So in other words, the kind of concept of maybe a new cure for some kind of cancer, to the clinical trial, to trying it out, to market, and to actually it being used to treat patients. How long is that process?
So, Dr. Berkenblit, do you want to start?
Anna Berkenblit: Sure. That process takes years. It takes years to go from an idea to a drug that is approved and available to patients who will benefit from it. And so in order to do that, you need to have the scientific understanding.
You need to have all of the data to show where you think the drug is going to be most helpful, like which patients.
You need to understand the mechanism of action. You need to understand, in early models – in the lab – how to dose the drug based on early data. What potential side effects to anticipate once you get into patients? You need to understand if there are any potential drug-drug interactions because sometimes, if you take one drug with another drug, you can get a bad reaction. You need to understand how the drug is absorbed. How it is metabolized in the body, how the body breaks it down, and how it is excreted from the body.
All of the things I just said require scientific studies and experiments before the drug can ever get into patients, where we wind up testing our hypotheses to make sure that we understand in an early phase, first-in-human study. How do we dose the drug? What are the side effects? And which patients are going to benefit the most based on the scientific mechanism that we understand? Once we have all that, then we go into larger studies to really understand what we call benefit-risk. So there's no such thing as a drug that is 100% safe.
But at the end of the day, FDA is going to assess the benefit-risk of whatever drug it is that we're talking about in the intended population. And so that takes years of work and hundreds of people with different kinds of expertise, scientists, chemists, formulation experts, biologists, statisticians, clinicians. It takes a whole host. It's really a team sport, if you will. And many drugs that are developed along the way sometimes have setbacks because of decisions that are made.
And sometimes you have to do a do-over. Or sometimes you need to figure out a different way to formulate the drug. Or sometimes you need to figure out a different population because now you understand the biology better than you did a few years ago. And all of the things that I just described really are responsible for every drug that is on its way to approval or getting approved.
And clinical trials are fundamental.
We need patients to participate in clinical trials. They need to understand their treatment options. They need to provide informed consent, which means they understand what the trial is about. They understand the risks associated with participating, and they give consent to participate in the trial.
And when they do, they're monitored more closely than when they are on standard-of-care therapy that's widely available. And that extra monitoring is to make sure that patients get through safely, because sometimes there are new things that happen, and we want to make sure that we understand what's happening and that patients are safe as they are participating in the clinical trials.
So often that requires more visits, more blood tests, maybe more scans.
But at the end of the day, that is so that we generate a big enough data set that is high quality so that FDA can review all of the data and assess the benefit-risk.
And if it's thumbs up, then the drug gets approved, then it's commercially available, and then the physician can write a prescription for it.
Alisyn Camerota: That's excellent. What an excellent explainer for us. And Dr. Ocean, I know that it must be so frustrating to know that something's on the horizon, that some company is working on something that's a breakthrough, and you're so good at knowing what's in the pipeline, but not being able to get it or give it to patients yet.
Allyson Ocean: Right. It is a challenge for sure, because the patients need it, like yesterday, and all of these new treatments that are promising. But we have to wait until they're validated in a way that makes the information sound, safe, and meaningful for patients when they get these drugs. It is a little bit of a waiting game.
That's why, as soon as something is available and we're able to use it, is to get people on it as fast as we can.
It is a waiting game a lot of the time, but it's waiting for the right reasons, unfortunately. It's hard to acknowledge that when you're sick, but it really is waiting so that we can give someone something that works and that is safe for them to take.
Alisyn Camerota: Yeah, I understand. Is there anything else that you wanted to add to the timeline or to the process of getting it from concept to treatment?
Allyson Ocean: Right. I think what people may not know about – is that there are different phases of these trials that were mentioned. And when you hear a trial is a phase 1 trial, that means the first time that they're studying this drug in people. And the goal of the trial is not to see if the drug works or not because we kind of think it works in the lab, that we have data that's showing that it works on animals or works on cells, but we don't know yet if it works in humans. But before it even works, we want to make sure that it's safe.
So the goal of a phase 1 study is safety.
And if safety is met and we find the dose at which is the safest for patients, that becomes the dose that people use when creating a phase 2 trial.
A phase 2 trial is done for efficacy. Does this intervention help people? Does it change survival? Does it reduce a symptom? Does it make people live longer? Etcetera, those are what phase 2 studies are the first question being asked, “Is this intervention, drug, device helpful for patients?” We know it's safe. Now, is it helpful?
And if we get signals from phase 2 that it is efficacious, then that means the drug or intervention can go on to phase 3.
Phase 3 is the trial that has to be done in order to get FDA approval.
What is a phase 3 trial? A phase 3 trial is a trial that compares the intervention to standard of care.
We have a standard of care because that was found out in clinical trials all along.
Whatever is standard of care became standard of care because it was proven in this phase 3 trial process.
So a phase 3 clinical trial takes standard of care as one group getting that. We know that that helps people, and we're asking the question: “Does this new intervention work better against your cancer than the standard of care?”
And if it does and the data is positive, and it meets its endpoints, that data goes to the FDA, gets reviewed, and then, ultimately, if the magnitude of difference in the new drug is enough, that leads to FDA approval.
And then that's a whole other thing waiting for that. But you can see why it's a long process.
But that's the process of how a drug goes from idea to preclinical – meaning in the lab testing – to clinical trials, to FDA approval, to access.
Alisyn Camerota: I so appreciate that primer because I've never known the difference between phase 1, phase 2, and phase 3. And my husband was on phase 1, and I think a phase 1 and either a phase 2 or phase 3, separate clinical trial. And I never knew the distinctions between all of that. So that's very helpful.
Allyson Ocean: Sorry, patients, Alisyn. I just wanted to say that some people they're waiting around for a phase 3 trial because that drug is further along in study, and they want to go on a phase 3 trial to be able to get the drug that's minus one step away potentially from approval.
But I tell patients that every phase of a clinical trial is important because if you're on a phase 1 trial, that means you're going to get the drug. We don't know exactly what dose it is, but it's done safely.
They enroll three patients in a trial. They see if those three patients have any side effects from that dose. If all three patients do well and have no side effects to that dose, they raise the dose a little bit. Then they enroll three more patients. Exactly like that. So it's a very slow process because it has to be done looking at safety.
And so if a phase 1 trial, if the science behind it is good, it could very well be that you're getting a drug that may be one day FDA approved. We don't know. But you're actually getting it. There's no randomization. There's no group that doesn't get it.
You get it, but you're getting it at a controlled dose.
And then phase 2. Some phase 2 trials are not randomized, so you can get a drug that is being investigated. Or you can be on a trial that is randomized, where you're randomized to getting it or not getting it.
Patients may also come across the term blinded in a clinical trial. Whether a trial is blinded or not blinded. A blinded clinical trial means that no one knows what the person is getting, meaning that the doctor doesn't know, the patient doesn't know, only the company knows what the patient is getting. That blinding has to be done for the integrity of the trial, for validity of the trial, and to make sure that the statistics and the data that come out of it are very well validated and actually mean the data means what it represents.
And so blinding is an important process within the clinical trials process.
Alisyn Camerota: Thank you. And again, this is all really helpful because the words were undistinguishable to me until now. Okay, ladies, what else would you like to add that we haven't touched on?
Anna Berkenblit: I'll just make another plug for considering clinical trials at every step of a patient's journey. That's the only way we're going to make progress.
Alisyn Camerota: Here, here. I'm a big fan. I mean, particularly with pancreatic cancer, these are new, cutting-edge things, and standard of care hasn't proven to be great.
So my husband, I know, was happy, very happy that we had this opportunity, and we felt lucky to have these.
Allyson Ocean: As Dr. Berkenblit mentioned, when you're on a clinical trial, you're watched very, very carefully. So you have more visits than you normally would with your doctor. You have more blood tests. I think that's a good thing that you're getting watched so closely, that it's care at its best, I think, and it's really a reason to consider a clinical trial.
Alisyn Camerota: Such a great point. I mean, you even have like an after-hours number that you can call. You have staff that's available to you, more so, than just the way regular standard of care would. I forgot about that. You really are treated in this kind of little verified group.
Okay, well, ladies, thank you very much for all of the information. That was super helpful. We want to thank Dr. Ocean and Dr. Berkenblit for being here today.
Thanks also to our sponsor, Revolution Medicines, and to you, our listeners. We really appreciate you joining us.
I'm Alisyn Camerota, and I'll see you next time on PancChat.
Cindy Gavin: Thank you, Dr. Berkenblit and Dr. Ocean, for that enlightening discussion. I'm Cindy Gavin, CEO and co-founder of Let's Win. If you or a loved one has been diagnosed with pancreatic cancer, navigating this journey can feel extremely overwhelming.
But you don't have to do it alone. To learn more about the latest breakthroughs in pancreatic cancer treatment, be sure to explore the wealth of resources available for patients and caregivers at PanCAN and Let's Win. You can find PanCAN at pancan.org and Let's Win at letswinpc.org. Our sites continuously post the latest news about emerging pancreatic cancer treatments. Together, Let's Win and PanCAN are committed to guiding you through every step of this journey, offering support, information, and hope.
In our next episode, we're going to be learning more about the relationship between diabetes and pancreatic cancer. Please follow PancChat to get new episodes delivered twice a month right in your podcast feed. PancChat is available on all major platforms. See you next month.