Supreme Court Justice Ruth Bader Ginsburg died after five bouts with cancer.

Supreme Court Justice Ruth Bader Ginsburg Dies From Cancer

The addition of immunotherapy to radiotherapy increased the overall survival of patients with cancer who developed brain metastases, according to new research.

Adding Immunotherapy to Radiotherapy Increases Survival in Patients with Brain Metastases

CURE® compiled a roundup of five recent reports patients with skin cancer may have missed.

5 Reports on Skin Cancer Patients May Have Missed

Dr. Howard A. “Skip” Burris, III explains new advances in the Triple Negative Breast Cancer field.

An Expert Explains the Three Major Treatment Modalities in Triple Negative Breast Cancer

Articles

From a new study showing that the iCanQuit smoking cessation app is more likely to work than typical guidelines, to the House unanimously passing the Veteran’s Prostate Cancer Treatment and Research Act, here’s what’s happening in the cancer landscape this week. 

Friday Frontline: Smartphone app encourages smoking cessation, House passes Veteran’s Prostate Cancer Treatment and Research Act, and More

Finding the best care team can make all the difference for patients with diseases like multiple myeloma.

Program Leads Patients With Cancer on ‘The Right Track’

Some cancers and treatments can result in cognitive changes that affect thinking, learning, processing or remembering information. These changes can affect many aspects of life such as the ability to work or even to do everyday tasks. Find out whether you have an increased risk of cognitive changes.

Cognitive Changes After Cancer Treatment

Each person responds to treatment differently. No one knows in advance how effective cancer treatment will be. There's also no way to know how long anyone will live—with or without cancer.

Life Expectancy With Cancer

Mantle Cell Lymphoma (MCL), as described in this issue of CURE®, has an interesting and inspiring story. It was one of the first cancers known to have a genetic abnormality-chromosomal translocation t(11;14).

This mutation causes an over expression of the protein cyclin D1, which drives cells to grow, ultimately become malignant and then multiply. However, until recent years, t(11;14) and other mutations were only used to diagnose MCL, not as a target in treating the disease. MCL was treated like most other lymphomas, with chemotherapy and steroids and then eventually Rituxan (rituximab).

Like many times with cancer, scientific discoveries broke the stalemate of MCL that had become refractory, meaning it stopped responding to treatment. Additional cancer promoters were discovered, including Bruton tyrosine kinase (BTK) — an enzyme that promotes cancer proliferation. 

That eventually led to drugs that inhibited BTK to be tested — and effective in — refractory MCL. Continued study of the disease also found more targets, leading to other drugs like Venclexta (venetoclax). Most recently, Tecartux (brexucabtagene autoleucel), a genetically engineered T cell therapy was approved for MCL treatment. All the while, researchers are still studying cyclin D1 and the cell cycle pathways it controls, in a continued quest for more treatments for MCL.

Outcomes continue to improve for patients with MCL, as the disease has gone from being highly resistant and difficult to treat to one of the more curable types of blood cancers, thanks to newer and intelligently designed drugs — many that are the fruits of basic science research. 

This highlights the critical value of clinician expertise in diagnosis and treatment. In this day and age, we hope that telemedicine and simple awareness can bring these treatments to more patients who need them, improving outcomes and quality of life for all with the disease.

Mantle Cell Lymphoma (MCL), as described in this issue of CURE®, has an interesting and inspiring story. It was one of the first cancers known to have a genetic abnormality-chromosomal translocation t(11;14).


Closer to a Cure for Mantle Cell Lymphoma

Bonnie Annis is a breast cancer survivor, diagnosed in 2014 with stage 2b invasive ductal carcinoma with metastasis to the lymph nodes. She is an avid photographer, freelance writer/blogger, wife, mother and grandmother.

A cancer diagnosis can cause a person to feel a wide range of emotions like fear, anger, shock, dread, grief, or loss. Often those emotions come suddenly like a crazy whirlwind and other times, they sneak up quietly, one by one. No matter how they come, all of them are normal.

I remember sitting in the doctor’s office after my diagnosis hearing her speak but being unable to process her words. They came in cartoonish fashion and sounded like Charlie Brown’s teacher, from Charles Shultz classic comic series, “Peanuts.” All I could hear was, “Waa wa wa waa, wa waaa wa waa.” Nothing made sense.

The words, “You have breast cancer,” can feel like being handed an instant death sentence, but though it feels that way, it isn’t necessarily a fact. Breast cancer is a highly treatable disease.

When I was diagnosed with breast cancer six years ago, I learned a lot. And, I’d like to share some of the most practical information in hopes of helping others.

Breast cancer isn’t usually a medical emergency. That means there’s usually plenty of time to process information and emotions. Give yourself space and grace. There’s no reason to rush into making decisions. You have time to think.

It’s a good idea to enlist the help of a trusted family member or friend. You’ll be receiving a lot of medical information and it’s a good idea to have someone accompany you to appointments. The person who joins you can jot down notes and vital information. This will allow you to be present in the moment without worrying about details.

You’re not alone. While it may feel like you’ve been thrust into a battle you weren’t expecting to fight, you won’t be the only soldier on the field. Your medical team will want you to be well informed. Their goal is for a good outcome.

At some point in the early stages of your care, you’ll be assigned a nurse navigator. The nurse navigator can help provide answers to questions you may feel uncomfortable posing to your doctor. The nurse navigator will also help keep you stay on top of upcoming appointments. This person will become your advocate. Learn to trust and rely on them. No question or concern is too insignificant. They’ve heard it all before, so ask away.

You’ll also have a tumor board. A tumor board is a group of doctors who collaborate on your individual case. They work together to coordinate your treatment and care. It may feel odd knowing you’re being discussed on a regular basis, but by working together, they can ensure you receive the very best care.

It’s OK to cry. Cancer is a scary word, and everything associated with cancer carries a feeling of uncertainty. Give yourself permission to cry and don’t be afraid to let others see your emotions.

Hit something! Get angry if you must. Anger is a powerful emotion and one many try to control, but in situations like receiving devastating health news, it’s acceptable to express frustration and anger. Anger won’t solve any problems but sometimes, those feelings can allow a person to process information and move forward.

Tell others about your diagnosis when you’re ready. A cancer diagnosis is personal. The diagnosis will undoubtedly cause you to feel emotional pain and shock. It will more than likely have the same effect on your loved ones. Adjusting to the news takes time. Communicate the information to others when you’re strong enough and do it on your terms.

Find your people and build your tribe. You may or may not have an emotional support team in immediate family members. Sometimes it’s necessary to reach outside your family unit and rely on a trusted friend(s). Look for those who are attentive and optimistic. Positivity will be beneficial to your recovery.

You may find new friends through a breast cancer Facebook group or other online support groups. Community will be a vital part of your healing process.

Communication is key. Talking about cancer during the initial phase of diagnosis will be challenging, but necessary. You’ll need a confidant to share thoughts, feelings, and fears with, but you’ll also need someone who is non-judgmental and understanding. You have the power to choose when and if you want to share information. Some people find it helpful to set up a website for sharing information with multiple people easily. 

 is a free website many have found helpful. Don’t feel pressured to share publicly, but if you choose to do so, do it your way.

Don’t believe everything you read or hear. It’s easy to fall prey to information overload. Be careful! One of the first traps many newly diagnosed patients experience is a need to know. As soon as you find yourself near that slippery slope, pull back! There’s so much information on the internet, in books, and from survivors it’s easy to be overcome with details. Most doctors’ offices or treatment centers provide helpful, documented information to aid in guiding you through treatment options. If they don’t offer reading material, ask for it. Or check reputable websites.

There are also several very good books with helpful information: 

Remember, knowledge is power. The more information you have about your disease, the less power it has over you.

When in doubt, speak out! If information is unclear, ask questions. The doctor and medical team work for you. You have a right to clear, concise answers.

Remember, you’re only human. From the day of diagnosis forward, you’ll be required to make decision after decision. Some of them will be small and others large. Some will be required immediately, and others will not. All these decisions can bring about feelings of stress. An unknown future can be frightening, but there are more people surviving breast cancer today than in the past.

The best piece of advice I can offer is to cling to hope. That’s what’s helped me the most along with choosing to live one day at a time.

“Sometimes even to live is an act of courage.” ― Lucius Annaeus Seneca

Remember, knowledge is power. The more information you have about your cancer, the less power it has over you.

Practical Tips for People Newly Diagnosed with Cancer

As oncology nurses, we know that birthdays are a privilege denied to many. Our stem cell transplant unit has been fortunate to have had a great champion of birthdays in Courtney Ebbeskotte, RN.

On one occasion, she noted that one of our patients was about to turn 21 and discovered that on his birthday, he would be admitted to the hospital for his final round of chemotherapy. During his second-to-last hospital stay, Courtney made arrangements with his mother and her fellow staff members to throw a surprise party during his next admission. 

When he arrived, he was met with streamers, a cake and enthusiastic nurses and patient-care techs shouting “Surprise!” It made his birthday and final hospital admission an experience to remember.

Another time, she found that she and one of her patients shared a birthday the upcoming week. Although not scheduled to work on her birthday, Courtney came to the floor and delivered a bouquet of balloons to the patient. She has wholeheartedly celebrated patient milestones and was among our unit’s most enthusiastic participants whenever a patient completed the 5K, half- marathon or full marathon that are part of our unit’s walking program. If she wasn’t the nurse to send a group text arranging the finish-line celebration, she would most certainly be one of the first to respond and stand by, ready to cheer.

Courtney has been a champion not only of current birthdays but also of patients reaching more birthdays. Every shift, she would strive to motivate her patients to do what was in their best interest for recovery, whether that meant eating and drinking more, walking just a few laps in the halls or sitting up in a chair for at least one meal. She is particularly effective in motivating non- compliant patients. 

When she served as our unit representative on the pressure ulcer prevention team, she strove to motivate her fellow workers in an effort to help us help them. She accounted for the patients needing frequent repositioning and placed reminders, which she designed and redesigned, on the patient doors and inside the rooms. She would hold us accountable and inspire us to be better healers.

Many patients owe their birthdays, both the ones celebrated now and those they can celebrate in the future, to the compassion, work ethic and healing nature of Courtney Ebbeskotte.

Oncology nurses are there for many moments on the cancer journey and can help champion important milestones that might otherwise go by unnocticed. 

Oncology Nurses Help Champion Patient Milestones

Clinical trials are essential in the development of new cancer therapies, yet many patients have misconceptions about clinical trials that may make them apprehensive to enroll in one.

, CURE® spoke with Dr. Susan M. O’Brien, a hematology oncologist at UCI Health in Orange, California, who is actively involved in clinical trials as director of the UCI Health Sue and Ralph Stern Center for Cancer Clinical Trials and Research. O’Brien discusses the importance of clinical trials for advancing the field of chronic lymphocytic leukemia (CLL) and offers advice for patients who may be interested in a trial but don’t know where to start.

What are the different kinds of clinical trials, and why are they important?

: Clinical trials come in a lot of different flavors. They can be trials of new agents, completely novel agents that don’t yet have FDA (Food and Drug Administration) approval. And they could be drugs that are next generation. So for example, we have a BTK (Bruton tyrosine kinase) inhibitor, but ... some company might think that they have a better BTK inhibitor. There can be trials looking at treatments for relapse disease; there (can) be trials looking at initial frontline (treatments).

I think a lot of people sometimes have the misconception and anxiety of enrolling in a clinical trial because they might be treated with a placebo. That’s very rare to see in a cancer trial. I don’t think I’ve ever seen (a CLL clinical trial) in patients who need treatment where there’s any kind of assignment to a placebo.

I think it’s important for a patient to consider a clinical trial, particularly with the novel agents if they’re available. ... Think about the fact that Imbruvica (ibrutinib) completely changed the landscape for CLL. It’s really been a total game changer. Consider how long it takes to get a drug FDA approved, and that one, by the way, went really fast. What that means is that people who are on the clinical trials actually have access to the drug years before that drug becomes FDA approved. So it can really get you access to novel, great drugs before they’re even out there for the rest of the community to use.

We also have to keep in mind that clinical trials are very important because (they’re) the only way we get new drugs. Every drug that is approved by the FDA had to be tested in usually more than one clinical trial. (The phase 1 trial determines) the appropriate dose, and the second trial looks at how well people respond. And then when we go to what we call phase 3; those are the randomized trials where a patient is randomly assigned to what’s usually considered the standard of care or to the newer drug. 

There are different places along the way where we have to accumulate that trial data before it goes to the FDA for approval. That’s why people who happen to get on a great drug can have years of early access to it before it even gets approved.

Can you talk a bit more about the misconceptions that people have about clinical trials that may make them apprehensive?

I think some people have anxiety (and think), “You’re experimenting on me.” We don’t like to call it experimenting. But yes, you can think of it as the fact that you are testing out a drug that maybe has not been given to a lot of people.

If you’re (on a phase 1 trial), then yes, you are one of the first people ever to get that drug. If you’re on a phase 2 trial, you’re looking at response (to the drug): We’ve already defined the dose, we know what the dose is, we know what the (side) effects are. People did get (the drug) before you, and (it was deemed promising enough to move) forward to phase 2. By the time it gets to phase 3 — where you’re randomly comparing it to standard of care — there’s plenty of knowledge. (There’s often already publicly) presented data or published data from the prior clinical trials, so you are going into a trial to get ... a drug or combination that you (might not be able to) get outside the trial. But hopefully that’s something that will benefit you and then benefit society as a whole if (it) leads to subsequent approval of that drug.

How can patients learn more about clinical trials?

Of course the Leukemia & Lymphoma Society is a great (resource) that really helps to match up patients with trials in their area. This is one of the issues about clinical trials: It’s probably easier for patients in larger metropolitan areas to participate than it might be for someone who lives in a really rural area where they’re not that close to their medical center. They have to be able to get there.

There are certain trials where the larger centers will partner with smaller practitioners. ... In some of the cooperative groups, that hub-and-spoke model is how they operate. So for example, there are major academic centers, but then there are affiliated, much smaller centers that participate in those trials too. So cooperative group trials are sometimes easier for patients to have access to because of their design. And you don’t have to (go) to one of, say, the four centers that’s doing that trial. So that can be really helpful.

A number of different sites can be really helpful when trying to identify trials, particularly in a certain region, (because you probably don’t want) a patient who lives in Arizona (to have) to fly to New York on a weekly basis. I mean, that’s realistically not going to work. There are other trials at major centers where they have to go to the trial to enroll, but some of the treatment — particularly if it’s an oral agent that the patient takes themselves — can actually be administered at home.

So those kinds of trials, the patients do have some back and forth to the trial center, but much less than if it’s a drug that actually has to be delivered at the trial center. Some do, some don’t. There are a variety of possibilities, even for people who don’t live in highly populated metropolitan areas, to potentially (participate) in a clinical trial.

Clinical trials lead to new cancer treatments — and can give a patient access to effective drugs years before they’re made available to the public.

Clinical Trials are Fueling Medical Breakthroughs in Cancer

Barbara Tako is a breast cancer survivor (2010), melanoma survivor (2014) and author of Cancer Survivorship Coping Tools—We'll Get You Through This. She is a cancer coping advocate, speaker and published writer for television, radio and other venues across the country. She lives, survives, and thrives in Minnesota with her husband, children and dog. See more at www.cancersurvivorshipcopingtools.com,or www.clutterclearingchoices.com.

It is a delightful, small treat. Here I am in my comfy spot with my nail file and wooden nail stick. I am a long-term cancer survivor stealing a precious moment to tidy up my ragged fingernails. Is that sweet but a little sad? That’s what I was going for.

What I mean is — in the months and years since a cancer diagnosis, remember to practice self-care. Some of us tend to forget about self-case as we get pulled back into our busy lives and routines.

Maybe it really isn’t so bad, and maybe this is just a shoutout to the sweetness of the simple things. You know, like enjoying a sunset or watching the birds fly across the sky. Or, how about the not-so-simple (or inexpensive) things, like a mani-pedi or a two-hour massage or a long observant walk?

Why is self-care so difficult? This is about more than money. Sometimes I do make selfish choices, usually purchases to feather our nest or my wardrobe or to work on a home improvement project, but the precious gift of time for self-care? That can often be a different story for cancer survivors who are filled with anxiety and fear of recurrence. Here is how this 10-year cancer survivor would do it differently if she could go back in time:

It is OK not to be OK. After my double mastectomy, I was back at the doctor’s office a couple weeks later, not understanding why it was taking so long to heal. I think there are quite a few survivors who try to get back on their feet too quickly and then we wonder why we crash and burn or become exhausted and resentful. Sometimes it is our own fault — we quickly get back up to full speed and rush around trying to take care of everyone and everything else before we are quite healed or ready.

You know how precious it is. Please give yourself time to heal. After getting to still be here 10 years after my first cancer diagnosis, my main regret is not giving myself the gift of time. No one could have done this for me. Treat yourself to the little things and the big things. Sometimes “things” aren’t really things but intangibles that can improve our happiness — like connecting with nature or having meaningful conversations.

Time after time I have rediscovered that this just works. Even glass-is-half-empty people like me benefit from focusing on the good rather than the bad daily. Hey, you are still here. Most days I try to mentally list three things that make me feel grateful. Sometimes I put them in my journal and other times, I just think it through without writing anything down.

Even as an introvert, I think many of us have had too much alone time due to the pandemic. Lengthy alone time can get me all twisted and wrapped up in my own thoughts. Instead, helping someone else can help me heal too. Helping someone else can be more fulfilling than lengthy navel gazing. It can be a form of self-care too. Give it a try.

Finally, consider unplugging a little from our amazing electronic devices. Yes, it can be handy and distracting to have instant access to news, shows, Facebook, and more, however sometimes unplugging can be more soul healing. You know yourself best, so try and figure out what works best for you and do not be afraid to give yourself the precious gift of self-care during and after your cancer journey. You deserve this.

You know yourself best, so try and figure out what works best for you and do not be afraid to give yourself the precious gift of self-care during and after your cancer journey. You deserve this.

Cancer Survivors Must Remember to Practice Self-Care

September was named Childhood Cancer Awareness Month to draw attention to the leading cause of death by disease for children under the age of 14.

® Talks Cancer podcast, we chat with Dr. Tanja Gruber, the new chief of pediatric hematology, oncology and stem cell transplantation at Stanford Children’s Health, about how the field of pediatric cancer has evolved over the last decade and where the landscape is headed in the future, thanks to advances in treatments like immunotherapy.

Gruber also discusses the idea that late effects of treatment need to be taken into consideration when creating a therapeutic plan, and offers some advice to newly diagnosed patients and their families.

Podcasts

In this week’s episode of the CURE® Talks Cancer podcast, we chat with Dr. Tanja Gruber, the chief of pediatric hematology, oncology and stem cell transplantation at Stanford Children’s Health, about how pediatric cancer treatment is evolving thanks to advances in treatments like immunotherapy, and what parents should do after their child receives a diagnosis.

The ABC’s of Pediatric Cancer

As the options for cancer treatment continue to expand, patients and their caregivers should be aware of the various clinical trials currently being conducted — including studies they can possibly join.

Italian pharmaceutical company Menarini Ricerche announced that the first patient has been treated in Europe in the expansion cohort phase of the DIAMOND-01 clinical trial of the investigational drug SEL24/MEN1703 to treat acute myeloid leukemia (AML).

The phase 1/2 dose escalation and cohort expansion trial is designed to evaluate the activity and safety of SEL24/MEN1703, an oral dual PIM/FLT3 inhibitor, in patients with relapsed/refractory AML and patients who have not yet received therapy and aren’t eligible for chemotherapy.

As of July, the first patient selected within the United States to participate in the expansion cohort phase of the trial began receiving treatment.

The first patient has been dosed in a phase 2 trial of patritumab deruxtecan to treat patients with advanced or metastatic colorectal cancer who did not respond to, or were unable to receive, at least two prior lines of systemic therapy, according to the agent’s manufacturer, Daiichi Sankyo.

Eighty patients across the U.S., Europe and Japan are expected to be enrolled on the two-cohort, two-part open-label trial.

“In this study, we are exploring whether the targeted delivery of cytotoxic chemotherapy with patritumab deruxtecan to cancer cells with varying levels of HER3 expression may be a potential treatment option for previously treated advanced or metastatic colorectal cancer,” said Gilles Gallant, of Daiichi Sankyo, in a company-issued press release.

Partner Therapeutics announced it has resumed enrolling patients in the National Cancer Institute-sponsored EA6141 study of Leukine (sargramostim, yeast derived rhu-GM-CSF) plus Yervoy (ipilimumab) and Opdivo (nivolumab) to treat melanoma in the frontline, or initial-treatment, setting.

The phase 2/3 trial was initially launched in September 2015. Patients with stage 3/4 inoperable melanoma were randomized to receive immunotherapy with either Yervoy and Opdivo with or without Leukine, a colony stimulating factor. Enrollment was paused in June 2017 after 240 patients were enrolled into the interim analysis. Based on results of the interim analysis, enrollment into the phase 3 portion of the study was greenlit. It is expected that 600 patients will be enrolled into the trial.

“The prior data with sargramostim (Leukine) supporting improvement in survival and reduction in immune-related toxicity, as observed in the E1608 study, highlights the importance of further clinical evaluation in combination with checkpoint inhibitors,” said study co-chair Dr. Ahmad Tarhini, director of cutaneous and clinical translational research at H. Lee Moffitt Cancer Center and Research Institute, in a company-issued press release. “EA6141 is a very important study in frontline melanoma with the possibility of changing the standard of care, and I expect a rapid enrollment across a large number of centers throughout the U.S.”

Nanospectra Biosciences announced that four clinical trial sites have been added to its ongoing open-label study of AuroLase Therapy for the focal ablation of prostate tissue via nanoparticle-directed near-infrared irradiation.

The extension phase of the trial is designed to assess the efficacy of using MRI/ultrasound technology to direct focal ablation of tissue affected by prostate cancer using nanoparticle-directed laser ablation.

The new sites added to the trial include: Duke University, Mayo Clinic, Northwell Health and Huntsman Cancer Institute at the University of Utah.

“End-of-study clinical and functional outcomes of the initial subset of subjects in the feasibility study were very encouraging, with 13 of the first 15 prostate cancer patients treated showing no detectable signs of cancer upon biopsy a year after treatment," said Dr. Ardeshir Rastinehad, vice chair of urology at Lenox Hill Hospital within Northwell Health System, system director of prostate cancer for Northwell and the national principal investigator for the overall clinical program, in a company-issued press release. 

“This is an exciting and intrinsically novel technology that now has the ability to reach its full potential due to advancements in visualization and targeting and unlock some of the promise underlying nanomedicine.”

Here is a list of some recent trials that launched within the cancer space in September.

CURE’s Clinical Trial Corner: September 2020

The experimental drug sotorasib (AMG510) demonstrated promising antitumor activity in patients with non-small cell lung cancer (NSCLC) that harbors the KRAS p.G12C mutation, an aberration for which no targeted treatment has yet been approved, according to findings presented at the 2020 ESMO Virtual Congress and published simultaneously in the 

In a subgroup of the multicenter, phase I CodeBreaK100 trial that included 59 heavily pretreated patients with KRAS p.G12C-positive NSCLC, researchers found that 32.2% experienced a confirmed objective response, meaning disease that shrank or disappeared, and 88.1% experienced disease control, meaning they achieved either objective response or stable disease. The drug also had a favorable safety profile, Dr. David S. Hong, deputy chair of investigational cancer therapeutics at The University of Texas MD Anderson Cancer Center in Houston, said. Hong, the study’s lead author, shared the data in an oral presentation at the congress.

The KRAS p.G12C mutation, which fuels cancer, occurs in 13% of NSCLCs, 3% to 5% of colorectal cancers and 1% to 3% of other cancers, Hong noted. Sotorasib, he said, is a first-in-class small molecule. It targets the mutated gene and prevents it from sending signals that would generate cancer-causing cell activity.

CodeBreaK100 included 129 patients: 59 with NSCLC, 42 with colorectal cancer and 28 with any of 11 other solid tumor types. Hong’s presentation at ESMO focused on the patients with NSCLC.

Those patients were divided into four groups, each of which received a different dose of the daily oral medication: 180 mg (three patients), 360 mg (16 patients), 720 mg (6 patients) and 960 mg (34 patients). Although tumor reduction was seen across all dose levels, 960 mg was determined to be tolerable and the most effective, and thus was studied in the trial’s expansion phase.

The main goal of the study was to test safety, with secondary goals including objective response rate (ORR), duration of response (DOR), disease control rate (DCR) and progression-free survival (PFS), meaning the length of time from the start of treatment until disease worsening. At a median follow-up of 11.7 months, 14 patients were still receiving treatment. Most of the 45 patients who discontinued the drug did so due to disease progression.

Study participants were a median 68 years of age, 59.3% female and 89.8% current or former smokers. All had previously received platinum-based chemotherapy, and most had also taken checkpoint inhibitor immunotherapy targeting the proteins PD-1 or PD-L1. In all but two cases, the patients were in good physical health except for their cancer. Although eligibility criteria disqualified patients with brain metastases, 18 developed the condition, and Hong shared a case study demonstrating that one of them experienced response of brain lesions to sotorasib.

At the first of a series of scans conducted every six weeks, researchers observed tumor shrinkage, of any magnitude, in 42 patients (71.2%). In the 34 patients who took the 960 mg dose, Hong said, the confirmed ORR was 35.3%, or 12 patients, and the median DOR was 10.9 months, with 10 of 19 responders still in response when researchers stopped collecting data on June 1, 2020. The DCR was 91.2%; the median duration of stable disease was four months.

There were no complete responses. The rate of stable disease was 55.9% in both the 960 mg cohort (19 patients) and across the whole study population (33 patients). The median time until response was 1.4 months.

The median PFS for all patients with NSCLC in the study was 6.3 months.

There were no dose-limiting side effects, nor were there any treatment-related deaths. Serious or severe treatment-related side effects occurred in 18.6% of patients. This included a single severe event, increased alanine transaminase (ALT), which is an indicator of liver damage.

The most common serious side effects were increased ALT in six patients and diarrhea and increased aspartate aminotransferase (AST), another indicator of liver problems, in three patients each. The most frequent side effects of any grade were diarrhea in 15 patients and increased ALT and AST in 12 patients each.

Hong said that sotorasib demonstrated activity across NSCLCs with a range of different mutations, with no particular mutation associated with a heightened response.

He added that “additional CodeBreaK trials evaluating sotorasib as a monotherapy or in combination with other anticancer agents are currently underway.”

Specifically, Hong said, it’s likely that sotorasib can be combined with checkpoint inhibitor immunotherapy because it “can have an immunogenic effect on the tumor microenvironment, so in combination will likely be synergistic.”

“This drug is overall relatively well tolerated and works across different dose levels,” he concluded, so “I’m confident we’ll be able to find a therapeutic window.”

There is no approved treatment that targets the KRAS p.G12C mutation that drives some cancers like non-small cell lung cancer. But a recent study shows how the experimental drug sotorasib sparked responses in some patients with advanced non-small cell lung cancer that harbored the mutation.

Experimental Treatment Shows Promise for Certain Cases of Advanced NSCLC

Join us online for our next Chronic Lymphocytic Leukemia webinar where experts discuss CLL information highly relevant to patients, caregivers and advocates right now. Participants will have the opportunity to submit questions to be answered live by our expert panel. 

Treatment Options: Oral agents and drug adherence

Patient-Physician Conversations: How to decide treatment

Psychosocial: stress, anxiety between treatments/tests

®’s community of leukemia and lymphoma experts.

Get to hear from professionals in the CLL space including:

Join us for the full webinar and complete a survey the next day to be entered to win one of 3 gift cards valued at $100, $200, and $300!

On Thursday, September 30, 2020, from 5:30 – 6:30 p.m. ET join CURE® for the next EDUCATED PATIENT® CLL Webinar. 

Register For the Next EDUCATED PATIENT® CLL Webinar 

Over Mother’s Day weekend 2011, Kimberly Schuetz took her 2-year-old son on a road trip from their hometown of Fall River, Wisconsin, to visit family in Illinois. Leading up to the journey, Austin had been his normal, energetic self.

“He was eating like a horse and being very active, very playful. No sicknesses, fevers, or anything like that,” Schuetz says.

On Saturday, Schuetz’s father-in-law noticed something alarming while tickling his grandson. One of Austin’s lymph nodes on his neck had swollen to the size of a golf ball. As an oncology nurse, Schuetz immediately had a sick feeling in her stomach. Her mind went straight to thoughts of cancer.

While her gut reaction told her to take Austin to the emergency department, Schuetz decided to wait until Monday to visit his pediatrician back home. After all, her son was acting fine.

A week later, after visits to two pediatricians, the emergency department at their local hospital and pediatric hematologists at a children’s hospital, a bone marrow biopsy confirmed that Austin had acute lymphoblastic leukemia (ALL). A cancer of the blood and bone marrow, ALL is the most common type of cancer that occurs in children.

“I literally felt like I was living somebody else’s life, like I was watching a movie. I remember sitting there and listening to the doctor, but I also felt like I was floating above myself,” Schuetz says. “I was numb and couldn’t cry. Your brain just goes into this state of shock.”

Omar Durani, a family physician in Dallas, Texas, went through similar feelings when his 9-month-old daughter Kenza received an acute myeloid leukemia (AML) diagnosis. In 2016, her swollen lymph nodes signaled to Durani and his wife that something wasn’t right.

“As a physician and dad, it’s kind of difficult to figure out what hat to wear when, and how much to worry about things like that,” he says. “When the swelling worsened, we started advocating for blood work, but all the doctors we visited only kept giving her antibiotics.”

Finally, an emergency department physician who Durani knew personally listened to their concerns and performed blood testing. The results came back with white cell counts through the roof, confirming the family’s worst fears: Kenza had cancer.

Both ALL and AML are treated initially with chemotherapy, and for the vast majority of children, it works. More than 95% of children with ALL and about 85% to 90% of those with AML enter remission after the first phase of chemotherapy. However, remission does not necessarily mean a cure.

Austin relapsed in October 2012 after two phases of chemotherapy, and despite receiving a bone marrow transplant, his ALL came back a third time in May 2013. About 10% to 20% of children with ALL have a disease that relapses or is resistant to treatment. At that point, with no treatment options left, Austin’s parents were told he had three to six months to live.

“We live 45 minutes away from the hospital, and our doctor ended up driving to our house, not to offer any solutions, because we didn’t have a plan, but just to sit and cry with us, because she was so heartbroken and, in such disbelief,” Schuetz says.

Some cancer in children does not respond to chemotherapy. After three rounds of chemotherapy, doctors told Durani that his daughter’s only hope would be a bone marrow transplant. But as a child of Asian descent and mixed ethnic background, Kenza had a less than 2% chance of finding a bone marrow donor match.

While research in adult blood cancers has led to an explosion of new targeted therapies and immunotherapies, such progress has been slower for younger patients. This can leave children such as Austin and Kenza without a path forward. Even for patients who do achieve long-term survival, the lingering toxicity of chemotherapy can lower their quality of life as adults.

“Only four cancer treatments have been approved for first use in children in the last 40 years, compared with hundreds approved for treating adults during this same time frame,” said Dr. Gwen Nichols, chief medical officer of the 

“Out of all the federal funding that goes into cancer research, only 4% goes toward pediatric cancer research,” said Dr. Deepa Bhojwani, director of the Leukemia and Lymphoma Program

at Children’s Hospital Los Angeles in California. “That being said, I would say within the last five to 10 years, there has been a lot more progress in terms of targeted treatment and immunotherapy than there was in the past.”

But the tide may be turning for this young, vulnerable population with new research initiatives dedicated to applying precision medicine to pediatric blood cancers. Much of today’s research in pediatric blood cancers aims to find tailored alternatives to chemotherapy that could prove just as or more effective with fewer harmful side effects.

Cancer is still the leading cause of death from disease among children and adolescents in the United States. From 2012 to 2016, leukemia and lymphoma accounted for 38.7% of all cancer types for patients under age 20. Approximately 40% of children with AML and 20% of those with ALL experience a relapse of their disease. Non-Hodgkin lymphoma, the most common type of lymphoma in children under age 14, that is resistant to first-line therapy has a very poor prognosis.

Before joining LLS, Nichols worked for a pharmaceutical company. She says that getting pediatric drugs tested and approved may be difficult because they typically do not bring in as much revenue as adult therapies.

As a result, upon arriving at LLS she was determined to figure out a faster, more efficient way to bring new agents and therapies to children with blood cancers.

In 2016, LLS launched the Beat AML Master Trial for adults with AML. It represented a paradigm shift in the format of clinical trials by testing several different targeted therapies in individual study arms instead of only one drug or combination of drugs at a time. Patients had a genomic screening to determine whether they had one of the genetic mutations that would make them eligible for one of the study arms to test a particular targeted therapy.

“It’s about 50% less expensive to do a master trial than it is to do one drug at a time. So, we needed to do something similar in pediatrics to make it more cost-efficient. But even more than that, if you want to get targeted therapies to kids, you have to have the right kids to test the drug,” Nichols said. “If you’re doing one drug at a time, you have to screen thousands of kids to find the five that fit your drug, which costs time and money.”

In 2019, LLS announced a collaboration with the National Cancer Institute and the Children’s Oncology Group called LLS PedAL (Pediatric Acute Leukemia), a global precision medicine master clinical trial in the same vein as the Beat AML trial. Children with relapsed acute leukemia will undergo genomic screening to match the specific abnormalities driving their cancer with one of several targeted therapies.

As part of The LLS Children’s Initiative: Cures and Care for Children, a $100 million, multiyear effort to take on pediatric blood cancers, LLS PedAL will test multiple agents simultaneously at up to 200 sites worldwide. Trials will follow the same format so that data from multiple institutions can be shared and consolidated.

Nichols and her colleagues hoped to begin the trial this year, but with the coronavirus disease 2019 pandemic, LLS PedAL’s start date has been pushed back to early 2021.

Another inspiring research project is the Pediatric Cancer Genome Project (PCGP), a joint initiative from St. Jude Children’s Research Hospital and Washington University School of Medicine. Since 2010, the two insti- tutions have pioneered efforts to define the genomic landscape of pediatric cancer by sequencing the genomes of normal and cancer cells from 800 patients.

“That work really filled in much of the landscape of mutations that drive ALL, AML and many of the other kinds of pediatric cancer,” says Dr. James R. Downing, president and CEO of St. Jude, who was instrumental in launching the PCGP. “In particular, important fundamental discoveries came out for ALL in terms of new understandings related to the identification of new genetic subtypes, amplifications, fusions and chromosomal translocations of the disease.”

For instance, the PCGP pinpointed genes linked to an ALL subtype with a poor prognosis known as Philadelphia chromosome-like ALL (Ph-like ALL). In 2009, St. Jude scientists were among the first to describe Ph-like ALL in children. Five years later, next-generation sequencing revealed the genetic alterations that give rise to Ph-like ALL, which could be targeted with drugs widely used to treat other types of leukemia more common in adults.

A 2017 study from the PCGP identified potential targets for immunotherapies in pediatric cancers, finding that 88% of leukemias have at least one that could be exploited. A 2018 pan-cancer analysis that examined commonalities and differences among various cancer types, including ALL and AML, suggest that half of the cancers studied may have a targeted treatment available or under development.

“The second phase of the genome project is moving all that technology into frontline diagnostics,” Downing says. “So now we’re getting the whole genome, whole exome and RNA sequencing of every patient, and we gain insight that can be funneled into a protocol that allows them to be treated.”

St. Jude’s frontline ALL protocol includes an unparalleled level of genomic sequencing on the patient, on both cancer and normal cells, which allows physicians to make personalized therapeutic decisions. Downing hopes that collecting such a wealth of data will help usher in new therapies and lead to a further improvement in outcomes.

“So the momentum is there, the studies are ongoing, and hopefully five years from now, we will have a huge variety of therapies to offer our kids instead of just one,” Bhojwani says.

Bhojwani participates in the Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) consortium, which has its headquarters at Children’s Hospital Los Angeles. TACL brings together children’s hospitals and universities to accelerate the progress of new therapies for pediatric leukemia and lymphoma. The consortium includes 31 institutions in the United States and Australia that collaborate on clinical trials of new drugs and novel therapy combinations in the hopes of expanding the number of treatments available.

Both Kenza and Austin were saved by an experimental treatment called chimeric antigen receptor (CAR)-T cell therapy administered in a clinical trial. It involves engineering the body’s T cells, a type of white blood cell, to produce receptors on their surface called chimeric antigen receptors (CARs). The engineered cells are infused back into the patient, where they can recognize and kill cancer cells.

The Duranis couldn’t find a bone marrow donor match for Kenza, despite having more than 60 donor drives inspired by her diagnosis that registered more than 5,000 new potential bone marrow donors around the country. At that point, they decided to move forward with CAR-T cell therapy coupled with a haploidentical, or partially matched, stem cell transplant in August 2016.

“It was a no-brainer for us since there were no other therapies available besides a standard bone marrow transplant,” Durani says. “Now Kenza’s been in remission for four years, and she’s doing fantastic. She’s a thriving, spunky 5-year-old.”

After his third relapse, Austin’s parents enrolled him in a clinical trial to receive CAR-T cell therapy as a last- ditch effort to save their son. He received the treatment at the beginning of October, and on Halloween – which also happens to be his mother’s birthday – they received the good news that his biopsy showed no traces of cancer.

“I immediately started screaming and crying. I was just elated. It was the best birthday present that I could ever, ever have. Nothing will ever top that,” Schuetz says.

Today, Austin is a typical 12-year-old boy who enjoys video games, swimming with his friends and complaining about household chores. He and Kenza are living proof that innovations such as CAR-T cell therapy give children who have run out of options another chance at life.

For years, childhood blood cancers were understudied forcing patients’ treatments into a one size-fits-all approach. But new efforts are changing that.

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