Estelamari Rodriguez, MD, MPH

 I feel that there are some glaring unmet needs that we as a community should address and focus on combating. For example, LMD, which is, as you know doctor, leptomeningeal disease. Unfortunately, LMD was a battle that my husband and I, alongside our medical professionals, had to overcome. It basically means that the cancer that was in my husband’s body crossed the blood-brain barrier and began to affect his brain, which could lead to very rapid decline in a patient. That is something I feel we need to bring awareness to, that the medications should and could cross the blood-brain barrier so that they can go after this monster of a disease wherever it tries to hide. We really need new therapeutics to accomplish that.

 Thank you for sharing that because that’s truly an unmet need. Many trials exclude patients with leptomeningeal disease because we are not confident the drugs will penetrate, and the trials the way they were designed want to see a response fast. Patients with LMD have been unfairly excluded, and we need trials and drugs that penetrate the blood-brain barrier better and are effective. We have seen from the EGFR story over time, we have patients who have done remarkably well systemically, all over their body except for the brain. That’s really where we have failed patients, so I agree with you, that’s definitely an area of more research. Also, finding treatments that are better tolerated is going to be another area where patient advocates have a say. I think that when we develop drugs, we’re looking for the more potent drug, but sometimes the more potent the drug, the more potent the side effects. There’s a happy medium that the laboratory doesn’t get right, but the patient will get right. We need more experience with that as well.

 That’s the need for that partnership. Another great disparity or unmet need is that we are finding that, in our historically underserved communities, there’s so much underdiagnosis or misdiagnosis that’s happening. Quite a bit of that, I’d say, is attributed to a lack of health literacy across the board, and I would love to focus some light in that area. I know you spoke to that a little earlier with regard to having some health equity there in all communities. I just wanted to bring attention to that again, and agree with you on that point.

 I think that people are looking at this closely, but it’s been there from the beginning. We know that not all patients are offered every option, and sometimes it has to do with some biases in the interaction, and there’s lack of health insurance and lack of health literacy. All these things play a role, and we need to do a better job to make sure that resources are equitable. I think biomarker testing is really at the core of this.

 Once that is covered like other things are covered, like your CT scan is covered, then we should be able to demand that it’s equitable, that we at least identify all the patients. I will say that, at least in the United States, we have access to these drugs. I tell my patients, these drugs are really expensive and it’s sticker shock, but in this country today, we have great compassionate use programs and we have great patient assistance programs. Regarding the patients I take care of, I can get the drug for all our patients. Now, if I never identify the driver mutation and never knew I needed this drug, then I would never get it. However, for the ones who need it, once you identify them, the pharmaceutical industry is really committed to making these drugs available, and that I can speak to. We see it every day that patients need drugs and we can get those drugs for them.

 That’s a wonderful thing for all of us here in the United States and abroad; once we identify what medication needs to be prescribed to the patient, there is a process to get that drug to that patient who desperately needs it. I think more attention needs to be given to the local community oncologists to educate and equip them to be the captain of their patients’, I suppose I’d say vessel, with regard to making sure that they let them know about the clinical trials. If they are not made aware of the biomarker testing and the clinical trials, then they’re going to miss out on an opportunity that they would otherwise agree to. However, you can’t agree to something that you either don’t know about, or you haven’t had an opportunity to have your questions or concerns about any risks addressed by the doctor, or if you don’t yet know about the benefits for doing such a thing.

: Definitely. I think patients should question their doctors and should question the plan that is being offered. I recently saw a young nonsmoker, a very young patient, with extensive lung cancer. The initial test that was provided to us showed no driver mutation, and that made no sense to me. The patient questioned the doctor, and he came to us, and we did another biopsy. We actually did a liquid biopsy, which is a blood test, which could have been done from the beginning. Sometimes either the test gets it wrong, or the biopsy specimen doesn’t get to that clone of cells that has that mutation. Patients who are younger, and every patient who has a very limited smoking history, should have a very in-depth test, and sometimes it requires another biopsy because it makes a big difference for patients.

 Absolutely. For the patients who are listening, family members, care partners, as well as clinicians, I again send out an invitation for you to join with us at the Exon 20 Group. Our website is exon20group.org. We welcome your partnership in providing services that you may or may not have access to, and in extending patient care, quality of life for patients, and in defeating this disease. We need everyone in our global community working on and toward this effort. Thank you so much, Dr. Rodriguez.

 Thank you, Katina Bland, for joining me today and for sharing your valuable perspective as an advocate and as a caregiver. To our viewing audience, we hope you found this 

program to be informative and rich, and that it helps open conversations for you and your families and your communities. Thank you so much.

Videos

Before closing out their discussion on treatment for EGFR Exon 20–Positive NSCLC, experts discuss prominent unmet needs in this setting.

Unmet Needs in the Management of EGFR Exon 20–Positive NSCLC

 In the future, there are other things to look ahead to. Like I mentioned, these drugs were not available 2 years ago for patients, but now they are. There are a lot of new drugs in the horizon. Poziotinib, which is a drug that your husband was treated with, has data, and the FDA is actually soon to get that drug available for patients. There are other drugs that are on trials closer by. I have patients who have had to travel to have another drug called CLN-081 that is on trials and also has showed responses around the same range of 40%, so you can envision that. We have different generations of these drugs. If patients don’t tolerate 1, or they progress on 1, hopefully they can respond to the other ones, because they have different mechanisms. That gives us the opportunity of treating patients with targeted options. We hope to have many for them.

I can mention CLN-081. I have a patient who is being treated on that. There’s another inhibitor of 

 exon 20 called BDTX-189. There are many other drugs that are currently being tested. A good place to start, and you can also speak to that, is clinicaltrials.gov, which is the database of clinical trials that every trial has to be registered with. You can do a search by state, by mutation, and then discuss that with your doctor who may be going too fast. Sometimes doctors are not even aware that these trials are close by.

 Can you share a little bit about how you found the trial and what things you think are there for the horizon?

Absolutely. CLN-081, now that you’ve mentioned that particular drug, we discovered that clinical trial for CLN-081, and that was the first oral medication and drug that my husband went on. He did really well with that medication until later when there was disease progression that was found. We connected with the Exon 20 Group and partnered with them and located a local clinical trial all for my husband and gained access to that particular medication. It’s a wonderful thing that the Exon 20 Group is doing and that they are continuously advocating, not just for the current medications that we have, but for a robust pipeline of medication and drugs. We want an endless supply. I’m so pleased that we’re in an era now where we do have 2 medications that have already been approved and we have more medications that are right behind them in the pipeline to also be approved. The more the merrier.

Until this disease can be cured, the battle’s not over. We still need to be pursuing more drugs. To that effort, the Exon 20 Group has an international research consortium, and what we’re doing there is working really hard to solve the problems in drug design and drug development issues in the hopes that in the future we’ll have more drugs. We want to have an endless supply, and not only have more of them, but speedier development, so that we can ramp up the different options that patients have to combat this disease. Our Exon 20 scientists are completely determinate at seeing that mission through that we don’t stop now and get comfortable because we have a few drugs, but we continue to push forward developing more and more until we find the drug that meets every need. Until that has happened, the fight isn’t over and we shouldn’t let up.

 You’re right. We are really at the beginning of the new treatments for 

 20 insertion. We’re not at the end or destination. We have seen that, from our history over the last 10 years with the common mutations, there are resistant pathways that emerge with the first agents, and then you can develop a better drug to treat the mutated receptor only, and potentially lead to less toxicity to other receptors in the skin and the GI [gastrointestinal] tract. There’s definitely a lot of room to have better, more effective drugs that target potential resistance, and also drugs that will cause less toxicity.

A shared discussion on the importance of enrolling in clinical trials for EGFR exon 20–positive NSCLC and improving access to novel therapy for patients.

Enrolling in Clinical Trials for EGFR Exon 20–Positive NSCLC

 I want to take you back to when your husband was getting treatment. Quality of life, for us, is really the reason why we take care of patients because lung cancer patients are living longer, but you have to have good quality of life. Everything that we add has to be something that is meaningful and adds good life to patients. How did the treatments he received affect his quality of life? What things did you and your husband have conversations about, and what priorities were you making to maintain that quality of life?

 Initially, when he was diagnosed, before we learned anything about the Exon 20 Group, before we learned about clinical trials, before we had access to the drugs that you’re speaking about, we started with chemotherapy, and it was difficult. It was very taxing on my very strong and healthy husband and it was crippling to the point that quality of life was decreasing. I remember my husband specifically asking friends and family to pray for him to feel like himself and not feel like a cancer patient. He didn’t want to lose his identity in the battle and just be about fighting and not about living. We experienced that. Once we were accepted on a clinical trial, once we got access to the medication, life began to feel normal for him again. He could attend our children’s activities and sporting events. He could—wow—remarkably, he could sing again. He could enjoy the things that brought him joy, like making his famous tacos for the family and going on boat rides with us. He learned to surf with the kids. He could be active and be out and make those beautiful memories with our family that we will always cherish. That became the highlight of every day in our journey, rather than fighting cancer being at the forefront.

 Thank you for sharing. I love how your face lit up when you were describing these memories. I really think quality of life is something that the patients need to lead, and why it’s so important to share these decisions. Especially now that we have different options for treatment for 

 20 insertion, the patient can take a lead in terms of how they prefer to get the drug, what the priorities are for the patient, if they favor going into the cancer center so they’re monitored and they make sure they get their drug because it’s IV [intravenous] and is being administered. If they have an infusion reaction that’s bad, they can deal with it, or they may want to have the freedom of having an oral pill that is once a day, which is mobocertinib [Exkivity], and maybe they can travel for the summer, taking this pill, and not have to come in as often. There are a lot of decisions that doctors can’t make for patients. Patients need to have this conversation together about how these drugs work, what potential side effects they have, what works for them, and what works for their quality of life.

I think that really would improve adherence, and ultimately, patients who are feeling better do better. I do think that making the right decision for each patient is a conversation. I wanted to highlight the toxicity because even the oral pills have some toxicity. Even with traditional chemotherapy, we have patients who are going to have hair loss, but they’d rather not. We may have a patient who is going to have a rash because they’d rather not get a drug. All these decisions may be different for each patient. I wonder, regarding how your husband dealt with the side effects and how he managed, what was more important at that point?

 My husband always wore his hair short. He had beautiful hair, but he always cut it off and wore his hair in a bald style. He had never truly been bald until he had gone through the regimen of the chemotherapy. He truly was bald. He had a really great sense of humor about it. He joked about it and talked about all of the time that he got back from not having to cut his hair. He made these beautiful videos of himself throughout his experience during treatment and shared that with family and friends so they were aware of his clinical trial experience, his side effects, his battles, challenges, and the victories that we had experienced. That was a beautiful piece of him that he left with us to still revisit and enjoy.

One of the things I wanted to make mention of, Dr Rodriguez, is you mentioned traveling on a patient’s own terms with their family, not necessarily having the burden of traveling to gain access to medication. One of the things that we are really pushing to highlight and pushing forward at the Exon 20 Group is the idea of decentralization of clinical trials, which means we don’t want the patient to have to travel unnecessarily just to gain access to their medication, especially if they’re seeing that the medication is working. We’re hoping to aim to empower the patients as well as their local community oncologist to run clinical trials. Those clinical trials become something that’s a lot more open to the public. There are a lot more of them in the community. Then, that particular local oncologist who let’s say that patient had been seeing for months or years, the patient can keep that particular oncologist and they themselves can submit a request for expanded use for their patients. The patient doesn’t have to travel here and there just to gain access to a medication. I think that begins to enable patients to advocate for themselves and be treated near home with their families so that they can spend more quality time again living, and if they choose to travel, that travel is going to be for and with family. It would be for enjoyment and vacation purposes and not necessarily the burden of traveling just to get to the much-needed medication.

 Thank you. I think that’s something that we definitely have to envision a new way of doing research for because right now there are a lot of barriers. Distance travel is one of them, but so is the time that it takes to get lab tests and come back for a routine exam and another visit for an EKG [electrocardiogram]. We need to rethink this. That’s part of the reason why our trials are not very representative of minorities and the population, because they put the burden on patients to participate and we have to envision a different way. I think that one thing we learned during the COVID-19 pandemic is that we can take care of very complex cases and seek patients virtually, which we never thought we could do safely. I have clinics in which 50% of the clinic is all telemedicine. In a way, having the option of doing virtual visits, you could see patients more often. You can talk to their family members and you can talk to their children. I think that’s something that we can all look ahead to, and I’m glad that your group is taking the lead on that because the patient advocates have a lot out of power to really demand that things change because the system can be very slow.

A focused conversation on how a patient can maintain and improve quality of life while receiving therapy for non–small cell lung cancer.

Improving Quality of Life While Receiving Treatment for NSCLC

 I wanted to ask you, because your husband was treated with a similar oral drug that also causes some of these traditional side effects, how did you manage? What helped? What can you share with us?

 We had an amazing medical staff that came alongside and partnered with us to manage that. We had a nutritionist who would manage exactly his intake of food. We had a dietitian on board. We had several specialists giving ideas that we hadn’t thought about with regard to how to bulk up things and slow things down and get things back on track, as well as protecting him and hanging on to the very important nutrients that we were able to get inside him. We had a great team of professionals—as well as, of course, our advocates at the Exon 20 Group—assisting us and tirelessly working with us to try to overcome side effects and something else that we couldn’t see while we were down in the trenches every day battling cancer.

 You make a great point. It takes a village to treat patients dealing with lung cancer because the potential toxicities of the treatment can be ameliorated if you are managed appropriately and have resources to counteract the side effects. When patients have drugs that cause diarrhea, you need to have medications on hand to stop the diarrhea, and you need to be well hydrated. You need to find a way to get the nutrients in. A lot of cancer centers have these resources. I always find that we don’t have enough psychologists and nutritionists for every patient, but now, virtually, we’re able to meet more of our patients. There are a lot of resources online. Where else do you think patients can go if they’re not getting in an appointment? Where can they get more information about the side effects?

 I welcome them to come to our website, exon20group.org. There’s plenty of information there. Within our Exon 20 Group, we pride ourselves on being a bridge and a support to the community. That would be our patients, our care partners, family and clinicians. We have over 10 social media sites where patients can join a community of fellow patients. That’s an opportunity to discover and share insights on side effects and personal experiences. Let’s say a patient says, “I’m struggling with this. How are you guys handling or treating that?” That’s an awesome opportunity and platform for them to share those stories. We have a private Facebook as well as inspire social media groups, and every day we’re posting news, journal articles about patients who are similarly diagnosed and have had NGS [next-generation sequencing] testing. It’s a wonderful opportunity to bring together those in the community for moral support and encouragement, so they don’t feel alone and isolated.

One thing you mentioned is that once you get this diagnosis, it’s very daunting. In some cases, you feel alone and unprepared for the fight. There’s a lot to learn, and there’s a lot to equip yourself for, so you become an empowered advocate and aren’t passive. You’re pushing the envelope and learning and preparing yourself to fight, and fight for those around you and encourage them as well. This also enhances your quality of life, so you’re in community going through this with someone. We provide one-on-one patient and family consultations within the Exon 20 Leadership Group.

 That’s wonderful. Thank you for what you do, because patients—especially a lot of our patients with EGFR 20 insertion—are younger and connected to social media, and they connect to these networks. Having the support of other patients, who are going exactly through what they’re going through, is incredibly therapeutic. You can get resources, and you can learn how to manage the drugs.

One thing I’ll share with you is that these mutations are rare. I only see patients with lung cancer, and I’ve treated four or five patients who have this specific mutation. On average, community doctors see one every couple of years if they’re looking closely, or one every year if they’re looking more closely. The resources the patients have and the access you give is more experience than the average doctor in terms of understanding how to manage the drug. You being part of the conversation and what you bring into the equation is critical. We see patients in isolation, and you’re sharing information, sometimes internationally. I know some of the advocacy groups are international groups.

 When our patients join the Exon 20 Group, another support we offer them is the opportunity to have an angel buddy. Our beloved angel buddies are fellow patients in the Exon 20 Group who partner to provide peer-to-peer support with patients and their families. It’s been incredible, the impact that our angel buddies have had on other families and patients. We’ve experienced that with our beloved couple, who came along and walked with us on our journey, experiencing what we were experiencing about our cancer diagnosis and giving us insights. We knew that at any time, day or night, we could pick up the phone and call to see how they were experiencing what they were experiencing and if that resonated with my husband, so we could get tips from them. It’s been a tremendous support.

 That’s very valuable. It’s not for everyone. Some patients are private and don’t want to have their experience mirror someone else’s experience. But having access and information to someone who’s going through the same experience is very valuable.

Comprehensive discussion on support for patients receiving therapy for NSCLC, ranging from care team members to education materials to support groups.

Estelamari Rodriguez, MD, MPH

Unmet Needs in the Management of EGFR Exon 20–Positive NSCLC

Enrolling in Clinical Trials for EGFR Exon 20–Positive NSCLC

Improving Quality of Life While Receiving Treatment for NSCLC

Support for Patients Undergoing Treatment for NSCLC

Latest News