Cancer Horizons CURE Talks Cancer

Last week we saw some FDA approvals come through, as well as research that explored the psychosocial outcomes of individuals who survived pediatric rhabdosarcoma. And finally, we’ll discuss another cancer vaccine clinical trial that got the green light from the Food and Drug Administration. 

FDA Approved Besponsa for Children With Acute Lymphoblastic Leukemia

The first FDA approval of last week was one in the pediatric cancer space. The agency approved Besponsa for children who are at least 1 year old and have relapsed or refractory CD22-positive precursor acute lymphoblastic leukemia., also known as ALL. 

The approval is coming after findings from a single-arm study involving 53 children. Of which, 22 — that’s 42% — achieved a complete response from the therapy, with the median duration of complete response being 8.2 months. Additionally, the majority of patients who achieved a complete response also had minimal residual disease negativity, which indicates that there were 5% or less blasts found in the bone marrow and no cancer cells detected in the blood. 

Having a new treatment option for children with ALL is particularly exciting, as ALL is one of the most common pediatric cancers, according to the American Cancer Society. 


FDA Approves Opdivo Plus Chemo for Unresectable or Metastatic Bladder Cancer

Last week, the FDA also approved an immunotherapy/chemotherapy regimen for certain patients with bladder cancer. Specifically, the agency OKed Opdivo plus cisplatin and gemcitabine for the frontline treatment of adults with unresectable or metastatic urothelial carcinoma. 

This approval was backed by findings from the CheckMate-901 trial, which showed that the Opdivo-chemotherapy regimen improved overall survival (which is the time patients live before death of any cause) and progression-free survival (which is the time patients live without their disease worsening) compared to those who did not receive Opdivo. The median overall survival was 21.7 months for those who received Opdivo compared to 18.9 months for those who did not, while progression-free survival was 7.9 and 7.6 months, respectively. 


FDA Approves Brukinsa, Gazyva Combo for Relapsed, Refractory Follicular Lymphoma

Last week, the Food and Drug Administration also approved Brukinsa plus Gazyva for patients with relapsed or refractory follicular lymphoma after findings from the ROSEWOOD trial showed that not only did more patients respond to the two-drug treatment compared to Gazyva alone, but also at a median follow-up of 19 months, more patients were still responding compared to the Gazvya arm as well. 

Rhabdomyosarcoma Survivors May Have Poor Psychological Outcomes

A study published in the journal, Cancer, found that survivors of rhabdomyosarcoma — which is a rare cancer affecting soft tissues — may face increased risk of psychological challenges, especially if they were exposed to previous radiation therapy or have a history of smoking.

Researchers examined neurocognitive impairment, emotional distress and health-related quality of life in survivors compared to their siblings. Results showed higher rates of issues like memory impairment and emotional distress among survivors, with smoking linked to poorer outcomes. The CURE® team spoke with study author, Ellen van der Plas on the findings. Here is what she had to say. 


Ovarian Cancer Vaccine Trial Gets FDA Clearance to Proceed

The FDA has given the green light for a clinical trial of a vaccine designed to treat advanced ovarian cancer. Known as Innocell, this personalized therapy utilizes cells from the patient's own tumor which is inactivated via riboflavin and UV light. The drug is being manufactured at City of Hope in Los Angeles, and the trial aims to assess the vaccine's safety and effectiveness in stimulating immune response.

This is one of the many cancer vaccines being explored and developed in the oncology space. Check back on prior CURE® coverage for updates o

In addition to a breakthrough therapy designation for a lung cancer drug, this week we’ll be talking a lot about additional side effects and health conditions that may come with a cancer diagnosis, and how to manage them. 


We heard from an expert about using cannabis during cancer care, took a look at a patient population that may be more prone to cardiometabolic conditions after cancer treatment and we’ll highlight a study that’s looking at preventing infection and GVHD in patients with blood cancer who underwent a stem cell transplant. 






Patients Should ‘Have the Conversation’ About Cannabis With Care Teams


The use of cannabis seems to be growing when it comes to mitigating side effects from cancer treatment, though it is important that patients talk to their providers if they are using these products or have questions about them, explained Dr. Brooke Worster from Thomas Jefferson University. 


I recently spoke to Woster about the conversations patients with cancer should be having if they’re using or considering using cannabis. Namely, she discussed seeking guidance and having open discussions with the care team, but also remembering that cannabis is not a proven cure for any kinds of cancer. 






Drug Gets Breakthrough Therapy Designation for HER2-Mutant Lung Cancer

 
A novel drug, BAY 2927088 received a breakthrough therapy designation for treating HER2-mutant non-small cell lung cancer. This designation, granted by the FDA, signifies a potential advancement in treatment options for patients with this specific type of lung cancer, which happens in approximately 2% to 4% of advanced NSCLC cases. Now that the drug has a breakthrough therapy designation, its review will be fast tracked. 





BAY 2927088, an oral tyrosine kinase inhibitor, has shown promising results in a phase 1 trial, with a focus on safety, efficacy and patient outcomes. The drug works by blocking HER2, which can contribute to lung cancer proliferation. 






Hispanic/Latino Survivors May Be Higher Risk for Cardiometabolic Comorbidities


A recent study found that Hispanic/Latino cancer survivors have higher rates of cardiometabolic comorbidities — meaning health conditions that affect the heart and/or metabolic system — such as diabetes, hypertension and heart disease, which can complicate cancer treatment and post-treatment health management. 


The study showed that survivors with cardiometabolic conditions experienced lower health-related quality of life and had unmet supportive care needs, particularly in terms of emotional and physical well-being. The research also found that socioeconomic factors, such as income levels, were also linked to the prevalence of cardiometabolic conditions among Hispanic/Latino survivors, highlighting the importance of access to health care and healthy lifestyle behaviors in managing these health challenges. The study emphasized the need for holistic approaches to health that consider environmental influences and support policies promoting heart-healthy behaviors within communities.






Trial Evaluates Reduced Chemo Post-Stem Cell Transplant in Blood Cancers





Patients with blood cancers can talk to their cancer care team about possible enrollment in the OPTIMIZE trial, which is investigating a lower dose of post-transplant cyclophosphamide — also referred to as “PTCy” — to reduce infection risk post-stem cell transplant while preventing graft-versus-host disease in patients who underwent a stem cell transplant from a partially matched unrelated donor. 


This phase 2 trial aims to enroll 190 patients across cancer centers across the United States, and is expected to conclude in June 2026. By exploring reduced PTCy dosages, researchers hope to enhance patient survival and quality of life by minimizing toxicities associated with standard dosing.  







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Last week, the Food and Drug Administration (FDA) approved four different therapies in the oncology space — one of which, Amtagvi, marks the first cellular therapy for the treatment of solid cancers. 





The week’s first approval (an Onivyde regimen for metastatic pancreatic cancer) was covered in last week’s episode, but here’s a list of what has happened since that last recording. 







FDA Approves Tepmetko for Metastatic NSCLC Subtype





Patients with metastatic non-small cell lung cancer that has MET exon 14 skipping alterations may soon have a new treatment option, as the FDA approved Tepmetko in this indication. 





Notably, this full approval is coming three years after the agency’s accelerated approval of the agent back in February 2021. Follow-up clinical trial data showed that 57% of previously untreated patients responded to therapy with Tepmetko, with 40% having a duration of response that lasted a year or longer. 






FDA Approves Amtagvi for Pretreated, Advanced Melanoma 





On Feb. 16, the FDA approved Amtagvi for patients with advanced melanoma who had previously been treated with an immunotherapy or targeted therapy. Notably, Amtagvi is a cell-based therapy and is actually the first cell-based treatment to be approved in the solid tumor space. 





According to trial results that led to the approval, 31.5% of patients responded to therapy. Now this is a pretty exciting number, considering that this heavily pretreated population tends to have low response rates. Not to mention, TIL therapies like Amtagvi — while upfront they require about a three-week hospital stay — may set patients up for years without having to undergo more treatment, according to Dr. Rodabe Amaria from The University of Texas MD Anderson Cancer Center, who I spoke with after the approval.  







Tagrisso Plus Chemo Approved by FDA for EGFR-Mutated NSCLC





In the lung cancer space, we saw the approval of Tagrisso plus platinum-based chemotherapy for patients with locally advanced or metastatic non-small cell lung cancer whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations. 





Findings from the FLAURA 2 trial led to this approval, as data showed that progression-free survival was 25.5 months for patients who received Tagrisso plus chemotherapy, compared to 16.7 months for patients who received Tagrisso alone. Overall survival data is still immature at this point — meaning that the researchers just don’t have enough data to calculate averages — so stay tuned for more on that. 

For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.

Last week, we saw some FDA approvals for a new drug regimens, as well as some expert opinion about cancer vaccines. Additionally research touched upon the potential benefit of concurrent ctDNA and tumor testing, and physical activity for pain reduction in cancer survivors. 



FDA Approves Onivyde as First-Line Treatment of Metastatic Pancreatic Cancer

On Tuesday, the Food and Drug Administration approved Onivyde plus oxaliplatin, fluorouracil and leucovorin — a regimen referred to as NALIRIFOX — for the frontline treatment of patients with metastatic adenocarcinoma. The approval was based on findings from the NAPOLI 3 trial, which showed that the drug combination improved overall survival (which is the time after treatment patients live before death of any cause) and progression-free survival (time they live before their disease worsens) compared to a combination of gemcitabine plus nab-paclitaxel. 

While this approval provides a new, promising treatment for this patient population, Dr. Anthony Shields of the Karmanos Cancer Center in Detroit mentioned that the Onivyde-containing regimen is not a cure. 

“In our patients with advanced disease, this is not a curative therapy at this point,” Shields said in an interview with CURE®. “It clearly improves survival. It's still got its share of toxicities, though. … We need better drugs, despite the improvements. If patients get this regimen is the first line, inevitably if they're doing OK we will give gemcitabine/nab-paclitaxel (combination) as the second-line regimen. But we really don't have a third line regimen.”


Cancer Vaccine Could Go ‘Above and Beyond Standard of Care’ For Patients


The oncology community is on the cusp of a sea change regarding cancer vaccines, as one expert told us.

“Current vaccines have a dismal record, and minimal evidence of efficacy,” said Dr. Jeffrey S. Weber, deputy director of the NYU Langone Perlmutter Cancer Center and Laura and Isaac Perlmutter Professor of Oncology at NYU Grossman School of Medicine, via email.

Weber was among the researchers on recent KEYNOTE-942 study investigating mRNA vaccine mRNA-4157 (V940) and Keytruda versus standalone Keytruda for the treatment of patients with advanced-stage melanoma.With a median follow-up of 23 and 24 months, the recurrence or death rates were 22% and 40% and the 18-month recurrence-free survival rates were 79% and 62%, respectively.

The Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to the combination for the post-surgical treatment of patients with high-risk melanoma in 2023 based on the results of KEYNOTE-942.

“This mRNA vaccine would be the first approved cancer vaccine with clear cut evidence of efficacy in a well-done phase 3 trial [which was recently initiated],” Weber said of mRNA-4157.

Pharmaceutical companies Moderna and Merck have initiated V940-001, a phase 3 study evaluating mRNA-4157 and Keytruda as postsurgical treatment for stage 2B to 4 melanoma, announcing in June of 2023 that global patient recruitment had begun following primary analysis of the findings of KEYNOTE-942.


Tumor Testing, ctDNA Finds More Patients Eligible for Personalized Drugs

Circulating tumor DNA and tumor tissue-based testing can both help identify cancer characteristics that may point a patient toward a more targeted treatment regimen. Oftentimes, patients undergo only one of these two tests, but recent research showed that undergoing both of these tests may improve patients’ chance of identifying targetable mutations. 

Now, some patient populations — such as those with non-small cell lung cancer — may already be undergoing both tests in accordance with NCCN guidelines. The findings support that other groups in particular, such as those with breast cancer, may benefit from the dual testing modality. 

In an interview with CURE, one of the study authors noted that the two tests can be “highly complementary,” and patients shoul

Last week, we saw a few moving parts in the regulatory space, from new NCCN guidelines for pediatric neuroblastoma treatment to FDA Fast Tracks and Priority Reviews. Also last week, we covered research showing that a lower dose of an anti-emetic drug could have similar efficacy — and fewer side effects — than the standard, higher dose. 

NCCN Guidelines Give Framework for Childhood Neuroblastoma Treatment

The National Comprehensive Cancer Network recently published guidelines for the treatment of pediatric patients with neuroblastoma. This resource is geared toward mitigating unnecessary side effects and over treatment in patients with low-risk disease, while also developing the best treatment plans for high-risk patients. 

CURE® spoke with Dr. Rochelle Bagatell, professor of Pediatrics and Solid Tumor Section Chief at Children's Hospital of Philadelphia, and Chair of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Panel for Neuroblastoma, who emphasized that while these guidelines can influence treatment strategies, conversations between patient families and clinicians and even insurance coverage, each patient’s care should be as personalized as possible. 

“There may be specific cases where the nuances of a particular patient's case means that you have to adjust your thinking from what's written on those nice, clear lines,” Bagatell said. “But the general guidance about how to think about the risk of recurrence, what general type of therapy would be appropriate, how much chemotherapy when to do surgery. Those are the kinds of things that patients and families can look at and bring to their doctor and discuss.”

FDA Fast Tracks ARV-471 for Metastatic Breast Cancer

Last week the Food and Drug Administration (FDA) granted a Fast Track designation to ARV-471, a novel drug being studied for the treatment of patients with ER-positive, HER2-negative locally advanced or metastatic breast cancer. Specifically, this indication of ARV-471 is for patients who previously underwent endocrine therapy. 

Fast Track designations are given to drugs that show promise in treating serious conditions and fill an unmet need. The goal is to speed up the review and potential approval of these therapies. 

ARV-471 is being studied in the phase 3 VERITAC-2 clinical trial, which is comparing ARV-471 to Faslodex in this patient population. Preclinical studies showed that the drug induced tumor shrinkage and degradation. 






FDA Grants Priority Review for Alecensa in Some ALK-Positive NSCLC

Also in FDA news from last week, the agency granted a priority review to Alecensa as a postsurgical treatment for patients with early-stage ALK-positive non-small cell lung cancer. 

The priority review is based off findings from the phase 3 ALINA trial, which showed that the drug led to a 76% reduction in the risk of disease recurrence or death compared with chemotherapy treatment. Findings from this study also showed that at two years, 93.8% of patients taking Alecensa experienced disease-free survival (which is the time after treatment when patients do not have symptoms of complications from their cancer), compared with 63% in the chemotherapy group. At three years, disease-free survival rates were 88.3% and 53.3%, respectively. 

With the priority review, the FDA said that they plan on making an approval decision on Alecensa on or by May 22, 2023, though those dates can always change. 






Lower Dose of Nausea, Vomiting Drug Controls Chemo Symptoms

Finally, research showed that a lower dose of a nausea and vomiting drug could be just as effective as the higher, standard dose when it comes to controlling chemotherapy-induced nausea and vomiting. 

A study published in The Lancet Oncology found that a 2.5-milligram dose of olanzapine is not inferior (meaning it is no less effective) than a 10-milligram dose. Specifically, the researchers looked at the use of rescue medications, vomiting episodes and mild na

Last week, we saw some research regarding how a popular tool used to plan breast cancer treatment may be misguiding therapy for Black women, as well as an update on when we can expect to see a new cancer vaccine be readily available for patients. 





And on the FDA front, we’ll discuss a priority review for Enhertu for patients with HER2-positive solid cancers, as well as a fast track designation for a new drug duo in the lung cancer space. 






Cancer Vaccine Likely ‘Several Years’ Away From Wide Availability





ELI-002 is a vaccine being investigated for the treatment of patients with KRAS-mutant pancreatic or colorectal cancers. While cancer vaccines have been in the headlines a lot in recent months, this one, at least, is still a ways away from being readily available for patients across the United States. 





Findings from a phase 1 trial showed that the vaccine could be beneficial for this patient population, and now, a phase 2 trial recently started that will evaluate the efficacy of an injection version of ELI-002, compared to observation. The first patient was dosed in the trial in January 2024, so it could still be several more years until the drug is available, Dr. Christopher Haqq, chief medical officer and vice president, head of research and development at Elicio Therapeutics, said in an interview with CURE®. 





“We'll be talking to the regulators like the US Food and Drug Administration and others around the world to align on the data that we'll need to provide for a marketing application. And so, we haven't had that input yet. So I can't give an exact answer for you (on when the vaccine will be commercially available). But we'll work as fast as possible. It's even possible that the type of evidence that we gather in this randomized study could serve that purpose. But we won't know until we have further discussion,” he said. 







Test May Be ‘Misguiding’ Breast Cancer Treatment for Black Women 





A recent study showed that the 21-gene breast recurrence score may lead clinicians away from prescribing chemotherapy to Black women who may benefit from the treatment. 





The 21-gene breast recurrence score is the standard test to help guide treatment decisions for patients with estrogen receptor-positive (also known as ER-positive) disease. Most patients with ER-positive cancer undergo hormone therapy, but the outcomes for this test may help decide if a patient would benefit from additional chemotherapy, too. Now, findings from a recent study discovered that Black women — and younger Black women, in particular — may be missing out on chemotherapy that they could potentially benefit from. 





Now, this research team is conducting further research looking at potential molecular differences in breast cancer in Black women, as well as how other social disparities could be playing into an increased risk of breast cancer death in these women. 






FDA Grants Priority Review to Enhertu for HER2-Positive Solid Cancers 





The Food and Drug Administration granted a priority review for a supplemental biologics license to Enhertu for the treatment of patients with previously treated metastatic HER2-positive solid tumors that cannot be removed via surgery. Basically what that means is that the drug showed promise in a clinical trial, and now the FDA will work with the pharmaceutical company developing the drug to expedite the review and potential approval of the agent. The agency plans on making its decision on whether or not Enertu will be approved some time in the second quarter of this year. 





Enertu is an antibody drug conjugate, which is a type of drug that works by finding and binding to certain proteins found on cancer cells — in this case, the HER2 protein. The drug was previously approved for patients with lung cancer and metastatic breast cancer, and now, the phase 2 DESTINY-PanTumo02 trial will help determine if it will be approved for patients with endom