CURE Talks Cancer

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 nausea over the course of 120 hours. 

Notably, this lower dose can also lead to a decrease in side effects related to the drug, such as feeling of lethargy and drowsiness. 

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

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 endometrial, cervical, ovarian, bladder, biliary tract, pancreatic or other cancers that are HER2 positive. 

FDA Grants Fast Track Designation to Avutometinib-Lumakras Combo


Also in the regulatory space, the FDA granted a fast track designation to a two drug combination consisting of Lumakras and the novel agent, avutometinib for patients with KRAS G12C-mutant metastatic non-small cell lung cancer. The intended patient population for the regimen is those who have been treated with at least one systemic therapy and have not received a KRAS G12C inhibitor. 


The regimen is being investigated in the ongoing phase 1/2 RAMP 203 trial, which will analyze the effectiveness of the drug, as well as the overall response rate — which is the percentage of patients whose cancer decreases from the drug — and safety. Findings from the second phase of the trial, which is specifically looking at patients who have not received or did not respond to a KRAS G12-inhibitor are expected to be published some time in the first half of 2024. 

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

It’s been a busy few weeks here at CURE® and in the oncology space as a whole, as the last two weekends had back-to-back meetings: the American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium, and then their Genitourinary Cancers Symposium. 

Here are some highlights from the conference, but as always, you can find all of our coverage at curetoday.com. 

Gastrointestinal Cancers Symposium 

Imfinzi, Avastin, TACE May ‘Set a New Standard of Care’ in Liver Cancer

For patients with liver cancer whose disease is not eligible to be removed via surgery, adding Imfinzi and Avastin to transarterial chemoembolization — also known as TACE — tended to lengthen the time patients lived before their disease got worse, according to findings from the EMRALD-1 trial. These improvements in progression-free survival over TACE alone could lead to a new standard of care for this patient population, according to the lead study author, Dr. Riccardo Lencioni. 

More specifically, patients who received Imfinzi and Avastin plus TACE lived for a median of 15 months before death or disease worsening, compared to 8.2 months for patients who received TACE alone. This correlates to a 23% reduction in the risk of disease progression or death, and benefits were seen across different patient subgroups. 

Notably, the researchers on EMRALD-1 are still monitoring how the addition of the two drugs impacts overall survival. Once those data become more clear, it is possible that the drug manufacturers could submit this regimen to the FDA for approval, thereby officially shaking up the standard of care of TACE, which has remained the main treatment in this setting for about two decades. 

Cancer in Bloodstream May Predict CRC Outcomes

Circulating tumor DNA — also known as ctDNA — was another hot topic at the Gastrointestinal Cancers Symposium. So ctDNA measures little fragments of cancer that are found in the bloodstream after cancer treatment. 

Now, findings from the BESPOKE trial highlight the fact that ctDNA may offer insight into the recurrence risk in patients with stage 2/3 colorectal cancer who underwent surgery and then chemotherapy. The researchers used ctDNA to help determine minimal residual disease, or MRD, status. Essentially, patients with disease still detected in the blood stream were MRD positive, while those without detectable cancer were MRD negative. Findings showed that those with MRD negativity tended to live longer without experiencing relapse or death compared to patients with MRD positivity. 

Genitourinary Cancers Symposium

Survival Benefits with Keytruda and Padcev in Advanced Urothelial Cancer

Back in December, the Food and Drug Administration approved Padcev plus Keytruda for patients with previously treated locally advanced or metastatic bladder cancer. The approval was based on primary findings from the EV-302 trial. Now, updated findings from that trial are showing that the drug duo continues to outperform chemotherapy when it comes to progression-free survival — that’s the time patients live before their disease gets worse — as well as overall survival, which is the time patients live before death of any cause. 

Notably, these survival benefits were seen across patient subgroups, such as those with visceral metastases and lymph node-only disease. According to the lead study author, Dr. Michiel S. Van Der Heijden, this could result in a new standard of care in patients with locally advanced or metastatic urothelial carcinoma. 

Many Patients Miss Out on Testing to Guide Prostate Cancer Treatment

On the prostate cancer front, a study found that many people with metastatic castration-resistant prostate cancer are not undergoing germline or somatic testing. Now this is really important because back in 2020, two PARP inhibitors were approved in this setting. These are targeted drugs approved for patients whose cancers have certain characteristics, which can be determined by these types of tests. 

Rates of germline and somatic testing have increased since the FDA approvals, but according to the study — which looked at real-world evidence of patients being treated in community cancer and urology centers — about 40% of patients did not undergo standard-of-care testing. 

Study author, Dr. Neal Shore, said that this indicates the need for improved education on the importance of germline and somatic testing. 

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

Last week, we saw some big headlines in the oncology space, from Dexter Scott King’s death from prostate cancer and MLB Hall-of-Famer Ryne Sandberg announcing that he was diagnosed with the disease. 

The FDA also requested a label update for CAR-T cell therapies that would warn patients and providers about secondary malignancies that have been reported from the treatment. Also, we took a look at laughter therapy, and how it could help patients and caregivers. 

We’ve also been busy covering two conferences — ASCO’s Gastrointestinal Cancers Symposium, as well as their Genitourinary Cancers Symposium, so tune in later this week for a special podcast episode highlighting some major research from those events. 


Dexter Scott King Dies of Prostate Cancer, Ryne Sandberg Diagnosed With the Disease

Last Monday, Jan. 22, we saw two big stories in the prostate cancer space. First, Dexter Scott King, the son of the Civil Rights activist, Martin Luther King, Jr., died of prostate cancer. He was 62 years old. 

At the time of his death, King was the Chairman of the King Center, which is an organization focused on educating the world about the life and legacy of Dr. Martin Luther King Jr. Dexter Scott King was also the president of the King estate. 

In a statement announcing King’s death, his wife, Leah Weber said, “He transitioned peacefully in his sleep at home with me in Malibu. He gave it everything and battled this terrible disease until the end.” 

And on the same day Dexter Scott King died, Major League Baseball Hall-of-Famer, Ryne Sandberg, announced that he was diagnosed with metastatic prostate cancer. 

The 64-year-old — who was a 10-time All Star during his tenure for the Chicago Cubs, which ran from 1982 to 1997 — announced his diagnosis on Instagram. He said that received the diagnosis a week earlier and has started treatment. He asked that fans keep him in their thoughts and prayers. 


FDA Requests Warnings on CAR-T Cell Therapies, Citing Secondary Cancers

The investigation into CAR-T cell therapies continues. Recently, the Food and Drug Administration (FDA) requested that approved BCMA- or CD19-targeted CAR-T cell therapies update their labeling to include a warning of reports of T-cell malignancies, including CAR-positive lymphomas, which have been reported in patients who use this type of therapy. 

Back in November, the FDA announced that it was investigating reports of secondary diseases in patients who underwent CAR-T cell therapy. The available data shows that these diseases are extremely rare, and researchers are still looking into what, exactly, is causing them. 

Now, the FDA wrote letters to the manufacturers of five CAR-T cell therapies, requesting that they include a Boxed Warning — which is the highest safety-related warning for drugs — outlining the potential risks of CAR-T cell products. The companies must respond to the FDA within 30 days of receiving the letters, which were sent out on Jan. 19. 

Laughter Therapy May Improve Mood, Decrease Pain in Patients With Cancer

And on a much lighter note, we covered recent research showing that laughter therapy can decrease mood disturbances in patients receiving palliative care for late-stage cancer, as well as their loved ones. The findings, which were published in the journal, Cancer Nursing, also found that the laughter therapy reduced pain perception in patients and decreased levels of burnout in caregivers. 

Laughter therapy refers to alternative and complementary therapy using humor to help relieve stress and pain, in addition to potentially improving a patient’s sense of well-being, according to the National Cancer Institute. In this instance, it consisted of five 20- to 30-minute sessions held over five consecutive days. The participants introduced themselves using funny tools to relieve tension, and moved their bodies in laughing rhythms. 

“This indicates that our palliative care patients and family caregivers would have a positive view of the use of laughter or humor in their palliative circumstances,” the researchers wrote. 

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

Often, receiving a cancer diagnosis can require a crash course in oncology that few patients ever expected to take.

For colorectal cancer specialist Dr. Dustin Deming, the ACI/Schwenn Family associate professor in the division of hematology, medical oncology and palliative care at UW School of Medicine and Public Health, a diagnosis of rectal cancer two weeks after receiving his first faculty appointment required an education of a different sort.

Deming, a gastrointestinal oncologist and laboratory researcher for UW Health | Carbone Cancer Center, told CURE®, needed to learn how to be a patient.

“For me, the crash course was in being able to allow my medical friends to be my doctors,” said Deming. “So, that was the part that I really had to wrap my head around. I knew what we needed to do. I knew I knew what I was about to go through. But I hadn't sat in the patient chair before. And so, the crash course that I had to enter was the crash course in what it's like to be a patient.”

Deming was 31 at the time of his diagnosis in 2012, married and with a 12-week-old daughter who he’d brought to his colonoscopy appointment. Treatment with surgery, chemotherapy and radiation followed, with Deming working as he was able to do so.

While colorectal cancer is the fourth most common cancer diagnosis in the United States, only 2% of new cases occur in patients ages 20 to 34, with the majority of cases (25.5%) occurring in patients ages 65 to 74, according to the National Cancer Institute.

“It’s obviously extremely ironic, and (was) potentially life-shattering at the time,” Deming said. “You know, when I was diagnosed, I had a 12-week-old daughter who came to the colonoscopy with me. Getting that kind of news at 31, I don't think anybody's prepared for. Having been a colon cancer doctor and researcher was helpful in that it provided me in insight into what we needed to do. But it was also terrifying, in that I knew all the dirty secrets.”

After being cancer-free for eight and a half years, Deming experienced a recurrence in 2020. He received further chemotherapy, radiation and surgery, followed by a second recurrence about a year later that was treated with surgery and chemotherapy. Approximately 10 months after his latest surgery, he has no evidence of cancer.
Dustin spoke with CURE’s “Cancer Horizons” podcast about his cancer journey, the connections it’s created with his patients and his continuing dedication to treating others.

“Having been a patient myself, I feel like — now, I don't know how it feels for each individual patient, but I know how it feels for me — I know how it feels to hear the ‘cancer’ word, I know how it feels to have to go through chemo, radiation and multiple surgeries,” Deming said. “So, every time I meet a patient for the first time, I sit down and tell the patients kind of where I've been so that they know where I'm coming from and also (to) make sure that they know that I now get it and that I'm actually truly honored to be part of the team helping take care of them. 


“I know what it means to trust oncologists with your care, and I'm so glad that I'm here to be able to help more patients.”

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

It wasn’t even two weeks into the new year when the Food and Drug Administration made their first FDA approval. In this case, it was a Keytruda regimen for certain patients with gynecologic cancers. We at CURE® spoke with an the primary investigator on the study leading to the approval about what patients need to know about the latest new indication. 


Also last week, we highlighted the Lymphedema Treatment Act and spoke to an expert about the new law. 


Another story in the regulatory space: the FDA granted a Fast Track designation to speed up the review of a novel drug used to treat patients with relapsed or refractory CLL or SLL. 


Finally, research from the American Cancer Society showed that Medicaid expansion states tended to have improved post-surgical outcomes in patients with non-small cell lung cancer.  

Keytruda Plus CRT Offers ‘Better Chance of Cure’ in Advanced Cervical Cancer


The Food and Drug Administration has granted approval for Keytruda (pembrolizumab) in combination with chemoradiotherapy for the treatment of stages 3 to 4A cervical cancer, marking the first approval of an anti-PD-1 therapy with chemotherapy for this patient population and the third FDA approval for treating cervical cancer with Keytruda. 


This week, I spoke with Dr. Linda R. Duska, a gynecologic oncologist and principal investigator on the study leading to the approval, who discussed the KEYNOTE-A18 trial, which enrolled 1,060 patients and demonstrated a 30% decrease in progression for those receiving the combination of Keytruda and chemotherapy. The overall survival data is not yet mature, but the treatment showed a 41% reduction in the risk of disease progression or death. While Dr. Duska emphasized that cervical cancer is preventable thanks to a safe and effective vaccine, she said that for patients with the disease, this new regimen is particularly exciting. 


January is also Cervical Cancer Awareness month, so definitely stay tuned to curetoday.com for more of our coverage on the disease. 

Medicare Must Now Cover Lymphedema Treatment Garments


The Lymphedema Treatment Act, signed into federal law on Dec. 23, 2022, is now in effect as of Jan. 1, 2024, allowing Medicare coverage for doctor-prescribed compression supplies for patients experiencing. The bill encompasses standard and custom-fitted gradient compression garments and other approved items prescribed by healthcare professionals to treat lymphedema. Now basically what that means is that patients insured by Medicare Part B can now have their lymphedema garments covered by insurance. 


This coverage is expected to ease financial burdens on patients, especially as these garments can be expensive. 


I spoke with Joanna Fawzy Doran, CEO of Triage Cancer, who emphasized the importance of patient and provider awareness about coverage rights, appealing denials and navigating the process. Although the law does not mandate private insurers to cover compression sleeves for lymphedema patients, Doran noted that Medicare's coverage sets a precedent for broader access in the future.

FDA Grants NX-5948 Fast Track Designation for R/R CLL, SLL


The FDA has granted Fast Track designation to NX-5948, a novel drug, for adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma who have previously undergone two lines of therapy, including a BTK inhibitor and a BCL2 inhibitor. 


Fast Track designation aims to expedite the development and review of drugs addressing serious conditions with unmet medical needs. 


NX-5948 is currently in a Phase 1a/1b clinical trial, with initial findings presented at the 2023 American Society of Hematology (ASH) annual meeting indicating safety, tolerance, and clinical activity. The drug showed no dose-limiting toxicities or treatment-related side effects leading to discontinuation, and the most common side effects were purpura/contusion, nausea, and thrombocytopenia. 

Medicaid Expansion May Decrease Early Mortality in Some With NSCLC


States with Medicaid expansion tended to have a significant decrease in early, postoperative mortality from non-small cell lung cancer, according to recent research. 


The research focused on nearly 15,000 patients undergoing surgery for NSCLC, with 62.1% residing in states supporting Medicaid expansion. The study found notable reductions in 30-day and 90-day postoperative mortality in patients with stages 1, 2, or 3 NSCLC in expansion states. Patients in non-expansion states were found to be younger, non-Hispanic Black, uninsured, and with comorbidities. 


The study also evaluated changes in early mortality before and after the Affordable Care Act (ACA) implementation, showing a decrease in the 30-day mortality rate in expansion states from 0.97% to 0.26% after the ACA. Now, advocates, including the American Cancer Society, continue to emphasize the importance of expanding Medicaid eligibility to improve health outcomes.

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

This week in oncology news, we saw a few decisions from the Food and Drug Administration, ranging from the agency agreeing to review a drug and potentially grant it a full approval to their citing manufacturing concerns for a gastric cancer drug. 


Additionally, a small study showed 100% disease control rate in a subtype of non-Hodgkin lymphoma, and a clinical evaluation of a new prostate cancer drug will continue to enroll patients. 

FDA Accepts sBLA For Tivdak in Recurrent or Metastatic Cervical Cancer


The Food and Drug Administration has accepted a supplemental Biologics License Application (sBLA) to Tivdak for the treatment of patients with recurrent or metastatic cervical cancer that has progressed after first-line therapy. Tivdak, the first antibody-drug conjugate for this patient population, received accelerated approval in 2021. Now, the sBLA is for the continued approval, which is contingent upon clinical benefit verification in confirmatory trials. 


Results from the phase 3 innovaTV 301 trial presented at the 2023 ESMO Congress demonstrated that Tivdak reduced the risk of death by 30% compared to chemotherapy as a second- or third-line treatment for this patient population. The trial also showed improvements in overall survival, progression-free survival, and confirmed objective response rate. Treatment-related side effects were observed in a majority of patients, with anemia and nausea being the most common. 


The FDA plans on making a decision on the approval by May 9, 2024.

FDA Cites Manufacturing Concerns in Missed Gastric Cancer Drug Deadline


Also in FDA news from the week, the agency will not meet the Jan. 12, 2024, deadline for approving zolbetuximab, a drug designed for the treatment of unresectable or metastatic HER2-negative, CLDN 18.2-positive gastric or gastroesophageal junction adenocarcinoma. 


The FDA attributed the delay to "unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility" and issued a complete response letter to Astellas, clarifying that no clinical concerns, including safety and efficacy, were identified. 


Zolbetuximab, a monoclonal antibody targeting the CLDN 18.2 protein overexpressed in certain gastric cancers, has shown promise in preclinical studies by inducing cell death in cancer cells. The drug had received a priority review from the FDA based on positive outcomes in the phase 3 SPOTLIGHT clinical trial and the GLOW clinical trial. Astellas, the manufacturer of the drug, is now collaborating with the FDA and the third-party manufacturer to address the identified issues and establish a timeline for resolving them. 

Enrollment Continues in Study Evaluating ONCT-534 For Advanced Prostate Cancer


Patients are still being enrolled in an assessment of the novel drug, ONCT-534, for the treatment of advanced prostate cancer that has relapsed or is refractory to approved androgen receptor pathway inhibitors (ARPIs). 


The study's first two cohorts received daily oral doses of 40 mg and 80 mg of ONCT-534, while the third and fourth patients, part of the third cohort, will be administered a 160 mg daily oral dose. 


The ongoing phase 1/2 ONCT-534-101 study aims to determine the drug's safety, tolerability and preliminary antitumor activity, with a total of 27 patients in the first phase and two cohorts of 16 patients each in the second phase. 


The study is anticipated to conclude in January 2028.

Novel Drug Shows 100% Disease Control Rate in WM


And finally, a novel drug, iopofosine I 131, showed a 100% disease control rate in patients with Waldenstrom’s macroglobulinemia, according to topline findings from the CLOVER WaM study. 


The CLOVER WaM trial investigated iopofosine I 131 in 50 WM patients who had undergone at least two prior lines of therapy, including a BTK inhibitor. In an efficacy evaluable population of 41 patients who received a total dose of 60 mCi 60 or more days ago, the trial met its primary endpoint with a major response rate of 61%. The overall response rate in evaluable patients was 75.6%, with 100% experiencing disease control and 76% not experiencing disease progression at an average follow-up of eight months. Notably, 8% of patients achieved a stringent complete remission. 


Now we should note, however, that this is a small study sample size of only 50 patients, so we would expect to see future studies evaluating this drug in a larger patient population. 

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

Kate Rice is proof of the power of persistence and positivity while navigating a cancer journey.

Rice, an award-winning journalist, received a diagnosis of stage 4 anaplastic thyroid cancer in October 2021 — and quickly applied the same dogged dedication that had served her reporting in support of her own survival.

“When I was a reporter, none of my sources or desired sources could escape me. Sooner or later, they were going to have to talk to me, I just have that kind of determination,” Rice said. “And it wasn't so much that I wanted to find out about the cancer I got diagnosed with; I wanted to find out who could cure it.”

Years before receiving her thyroid cancer diagnosis, Rice had learned there were what doctors described as “indeterminant” nodules on her thyroid and was told to monitor them. She noticed small lumps on her neck in June 2021 and was told she would have to wait six months or so to be examined. 

Rice, who had previously had a benign tumor removed from her neck and a case of melanoma, searched for doctors who could see her sooner — and, upon finally receiving her diagnosis, was told “I’ll pray for you” by a surgeon.

Anaplastic thyroid cancer, according to the American Thyroid Association, occurs in less than 2% of patients with thyroid cancers, but it is one of the fastest-growing and most aggressive of all cancers overall. The disease’s average survival rate is six months, and just approximately one-fifth of patients live longer than a year after receiving a diagnosis.

Immediately after receiving her diagnosis, Rice began sourcing for potential solutions, starting with a group text to her inner circle of loved ones.

“My cousin, who had been one of the first people I'd sent (a message to when I) cast that wide net out to try to get information, had promptly gone online and found that (The University of Texas) MD Anderson Cancer Center in Houston had a clinic that specialized in this very rare (cancer) … and actually cured people with it. So, I got my diagnosis, I think, at two o'clock Friday afternoon, I was walking home up Columbus Avenue, I guess, and on the phone with MD Anderson, to get in there because I can jump on the phone with both feet.”

Within days of receiving her diagnosis, Rice left New York City for Houston, Texas, seeking treatment from the Facilitating Anaplastic Thyroid Cancer Specialized Treatment Team at MD Anderson Cancer Center.

Such determined self-advocacy, Rice said, is “absolutely essential.”

“You have to stand up for yourself, you have to recognize that your doctors are specialists in whatever it is they're specialists in, but you're the specialist in your body,” she said. “And I knew something was going on. I mean, something was happening with my thyroid. Thyroid cancers, in general, are not the scariest cancers out there. They’re serious cancers, and the treatment for a whole bunch of different thyroid cancers is not fun. But I knew this was something that was potentially a very big deal. 

“And so when the first doctors I saw in New York were very relaxed and I couldn't get in to see a doctor I've been seeing for years because I had what are called indeterminate nodules on my thyroid — (which are) not malignant, but (they’re) not benign, either — they were like, ‘Yeah, well, we can't get you in for six months, but that's OK,’ I'm like, ‘No, not OK.’ Even before I got the diagnosis, I was a pushy patient and we all have to do that. And the thing is, you're like, ‘Oh, I don't want to be much trouble. These guys are the pros, they know.’ But really, you've got to listen to your body and stand up for yourself.”

Following treatment at MD Anderson Cancer Center, Rice said she’s “fine.” Now a radio disc jockey and ski instructor in Park City, Utah, she returns to Houston every six weeks for immunotherapy treatments at MD Anderson. She also documented her cancer journey in the 2023 book “Cured: A Tale of Badassery.”

She has advice for fellow patients facing the long haul of a stage 4 cancer diagnosis.

“Stay positive. Realize we all have to fight this cancer trauma that understandably, many people in this country, in this in the world, carry because we've seen terrible things happen to people we love, when my dad died of prostate cancer,” Rice said. “But, the thing to remember is so many cancers now are either curable or treatable and manageable. The whole thing is getting to the right place in time, which is a challenge with our healthcare system. But you really have to remember there is no such thing as false hope there is only hope.”

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

The year 2024 is now underway. After a busy 2023, we’re now looking back at some of the oncology headlines from the new year. 

Notably, we have a clinical trial hold for a lung cancer drug, a trial that showed that a CAR-T cell therapy is cost-effective — though far from cheap — for lymphoma treatment, and a novel drug that’s showing promise for the treatment of myelofibrosis. 


FDA Places Hold on Trial Evaluating TIL Therapy in NSCLC


The Food and Drug Administration placed a clinical hold on the ongoing IOV-LUN-202 trial, evaluating LN-145 TIL for patients with non-small cell lung cancer. With the clinical hold, new patients will not be able to enroll on the trial, and those who were previously treated will continue to be monitored. 

The FDA placed the hold on the trial after the death of a patient. According to the FDA, the agency has the power to request that a trial be stopped, and then the sponsor — that’s the organization or company running the study — can reply via a response letter addressing the concerns. The FDA then has 30 days to respond, and potentially allow for the trial to resume. 


Breyanzi Cost Effective As Second-Line R/R DLBCL Treatment
Research published in the journal, Blood Advances, found that the CAR-T cell therapy, Breyanzi, was found to be a promising and cost-effective treatment option for patients with relapsed or refractory diffuse large B-cell lymphoma — also known as DLBCL. 


The study found that the incremental cost-effective ratio of the drug was just over $99,000 per quality-adjusted life-year from a health care perspective, and just over $68,000 from a societal perspective. Both these numbers are lower than the assumed social willingness to pay up to $100,000 per quality-adjusted life-year gained, as established by the American Society of Hematology. 


While Breyanzi was deemed to be cost effective, CAR-T cell therapies are still far from inexpensive. In fact, the drug cost rose by 9% since its initial approval for DLBCL in 2022. In a CURE® Speaking Out® video series, Lee Greenberger, the chief scientific officer of the Leukemia & Lymphoma Society, expressed concern about the price of these drugs. 

 
“Some of that the government is going to have to pay, some insurance is going to have to pay, but some of it the patients are going to have to pay as well. And the price tags are going to be significant. How are we going to manage that?

Phase 2 Trial Shows Fibrosis Reduction in Some With Myelofibrosis


Also in the blood cancer space, findings from the phase 2a trial found that a novel drug, GB2064, reduced fibrosis in the bone marrow, thereby slowing cancer progression in patients with myelofibrosis who were previously treated with Jakafi. 


Myelofibrosis is a disease that affects the body’s production of blood cells in the bone marrow. The disease causes scar tissue to grow there — a process called fibrosis. By decreasing the amount of fibrosis that occurs, GB2064 has the potential to improve outcomes. For this patient population.
However, we should note that this trial was completed in only a small number of patients — 10, to be exact. Six patients had a decrease of fibrosis after receiving GB2064 for six months. 

The year 2023 has come and gone, and we figured that now is the perfect time to look back on some of the top cancer-related stories from the year. 

Vitamin D May Impact Colorectal Cancer Outcomes


In June, we spoke to an expert about research that analyzed 14 studies on vitamin D and colorectal cancer outcomes. Findings showed that people who had a vitamin D deficiency tended to have poorer mortality outcomes than those who supplemented with vitamin D. 


Laura Bolte, of the department of gastroenterology and hepatology at the University of Groningen and University Medical Center Groningen, explained that the use of corticosteroids and being underweight or malnourished — which are all things that can happen during cancer treatment — can increase the risk of vitamin D deficiency, so it is essential that patients speak with their health care team to determine if a vitamin D supplement will be appropriate and beneficial to them. 

Jimmy Buffett Dies of Cancer at 76


Every now and then, major celebrity news makes cancer headlines. On Sept. 1, “Margaritaville” singer, Jimmy Buffett died of cancer at the age of 76. 


Buffett had a rare and aggressive type of skin cancer called Merkel cell carcinoma, which is much more common to spread to other parts of the body, and can be very difficult to treat if it spreads, according to the American Cancer Society. 


Shortly after Buffett’s death, we spoke to Dr. Manisha Thakuria, who is the director or Merkel cell carcinoma at the Dana-Farber Cancer Institute. She said, “It's always hard to see any silver linings in losses. I am glad to see Merkel cell having a little bit of a spotlight on it, and I hope that (the media interest) will increase research funding for Merkel cell carcinoma and help more patients,” said Thakuria.

Chemo Drug Shortage Requires a ‘Holistic Solution’ 


Perhaps one of the biggest headlines in the cancer space from 2023 was the chemotherapy shortage. The shortage of cisplatin and carboplatin required a “holistic solution,” according to Dr. Anjan J. Patel. 


In a June article, I spoke with Dr. Patel and other oncology experts about the shortage, what it meant for patients taking these drugs and what was needed from a systemic level to ensure that these types of shortages never happened again. 

Patients Using Cannabis Experienced Worse Symptoms, Perceive Less Harm


Patients with cancer who use cannabis reported more severe symptoms and perceived less potential harm from cannabis than patients with cancer who did not use the drug within the last 30 days, according to study findings published this summer. 


Findings showed that study participants who used cannabis experienced significantly poorer physical and social functioning as well as higher pain intensity, pain interference, fatigue and sleep disturbances. 


“People are using it, we should study it, and we need to know more to be able to guide physicians and cancer patients and survivors as well,” Gregory Giordano of the department of psychology and neuroscience at the University of Colorado Boulder told CURE®

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