CURE® spoke with an expert about Enhertu for HER2-positive solid tumors and how important this approval is for the patient population.

Treatment with Enhertu (fam-trastuzumab deruxtecan-nxki) “now provides an option for solid tumors,” for patients with HER2-positive solid tumors, an expert said.

The recent accelerated Food and Drug Administration (FDA) 

 is important for patients with HER2-positive solid tumors. Of note, Enhertu is the “first drug ever to be approved across solid tumors targeting HER2,” Dr. Sarah Sammons told CURE®.

The FDA’s announcement also stated that eligible patients previously did not have satisfactory alternatives for treatment.

“This is an unprecedented approval because Enhertu is the first drug ever to be approved across solid tumors for diverse HER2-positive cancer types,” Sammons explained. “It is the first tumor agnostic approval for HER2-positive solid tumors defined as having strong immunohistochemistry HER2 staining of three plus, which is really exciting.”

Sammons is a medical oncologist and associate director of the metastatic breast cancer program at Dana-Farber Cancer Institute in Boston.

Enhertu’s approval was based on three clinical trials, including DESTINY-PanTumor02, DESTINY-Lung01 and DESTINY-CRC02. In specific, the DESTINY-PanTumor02 included patients with various cancer types. Sammons stated that these cancer types included biliary tract, bladder, cervical, endometrial and ovarian, and pancreatic tumors.

Sammons explained that oncologists have “a lot of experience” using Enhertu. Notably, it was 

 for patients with HER2-positive breast cancer.

“The drug is highly potent and has excellent activity,” she said. “In the DESTINY-PanTumor02 clinical trial, over 50% of patients had actual tumor response or shrinkage of their lesions.”

With this approval of Enhertu, more development and unmet needs have been addressed, Sammons said. She noted that patients with HER2-positive solid tumors did not previously have drugs approved for them across all tumor types.

Expert Overview of HER2-Positive Breast Cancer Treatment

“Classically, we’ve studied HER2 in the context of breast cancer,” Sammons said. “More recently, HER2-positive gastrointestinal cancers such as colon or stomach have received attention.”

She added that HER2-positive solid tumors are rare in some cancer types, such as ovarian and endometrial cancers. Because of rarity, many HER2-positive solid tumor subtypes did not have any FDA-approved treatment options because the population is too small to study.

However, “because this is an approval for any tumor that is HER-2 positive (immunohistochemistry 3+), this now provides an option for solid tumors,” Sammons said.

She emphasized that metastatic tumors should now be tested for the HER protein by immunohistochemistry because testing was not standard before.

“So, this approval now makes that testing important to see if this drug could be an option for patients after standard options.”

Although this approval addresses several unmet needs, some still persist. Sammons explained that HER2 tumors have more of a “propensity to spread to the brain and cause brain metastasis.”

“Brain metastases are always an unmet need in solid tumors,” she said. “Now that we’ve been using Enhertu in breast cancer for quite some time, we know that it works well for HER2-positive breast cancers that have brain metastasis. We don’t know that yet for other HER2-positive solid tumors, but we know Enhertu gets into the brain. So, it would be very important for us to study if Enhertu could be an option for brain metastases from other solid tumors that are HER2-positive, and candidates for Enhertu.”

For respective patients, Sammons encouraged them to ask their doctors about testing to see if they could be eligible for Enhertu.

“For patients who are candidates for the drug that are going on it, I think it’s really important to know that nausea could be a problem,” she said. “Have anti-nausea medications at home and know that if they have a cough or shortness of breath, to let their treatment team know immediately, as inflammation of the lungs is a fairly common but serious side effect of the drug.”

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Treatment with Enhertu “is an unprecedented approval” for patients with HER2-positive solid tumors who did not have many options, an expert told CURE®.

Enhertu Marks First Therapy Approved Across HER2-Positive Solid Tumors

“That doesn't mean that the treatment that we were investigating wasn't effective, it just means it wasn't more effective than the current standard of care,” an expert explained to CURE®.

Patients with non-small cell lung cancer (NSCLC) who do not respond to immunotherapy or whose disease progressed after treatment via immunotherapy face an unmet need, as one expert explained.

“Unless we have special pill therapies for subtypes of lung cancer with particular mutations, the vast majority of patients will start treatment with chemotherapy and immunotherapy. Sometimes those are highly effective, but oftentimes the tumors progress at some point after those therapies,” said Dr. Joel Neal, associate professor of medicine, Division of Oncology at Stanford University.

“[In] the second line setting — we call it second line because patients have already gotten the first treatments of chemo and immunotherapy — there have been many clinical trials, but there's really an unmet need for something better,” said Neal. “The one drug that's approved in this setting is [a chemotherapy] called docetaxel. It has a lot of side effects. It has neuropathy, numbness and tingling, it has low blood counts. Sometimes infections as a result of that, oftentimes hair loss depending on how the dosing is given. But it remains the FDA-approved treatment in that second line setting for non-small cell lung cancer, sometimes in combination with another IV treatment called [Cyramza (ramucirumab)] [a monoclonal antibody].”

Neal was among the international lead investigators of a study comparing the monoclonal antibody Tecentriq (atezolizumab) plus Cabometyx (cabozantinib) versus docetaxel among patients with metastatic NSCLC whose disease progressed following concurrent or sequential treatment with anti–PD-L1/PD-1 and platinum-containing chemotherapy.

Tecentriq, according to the National Cancer Institute, prevents cancer cells from suppressing the immune system, clearing the way for the immune system to fight cancer, while Cabometyx prevents cancer cells from growing and spreading.

At a minimum follow-up of 10.9 months, the median overall survival (the time a patient lives following treatment, regardless of disease status) was 10.7 months for the 186 patients in the investigational arm and 10.5 months for the 180 patients who received the standard of care, according to study findings published in the 

. There was no statistically significant difference between these two median survival times.

“That doesn't mean that the treatment that we were investigating wasn't effective, it just means it wasn't more effective than the current standard of care,” explained Neal. “And that meant that it could not be approved.”

The median progression-free survival times (the time a patient lives without their disease spreading or worsening) were 4.6 months and four months, respectively.

“The progression-free survival was actually a little bit better and significantly different for the investigational therapy,” Neal said. “We also looked at subsets of patients. We found that women actually did a little better with docetaxel, which was surprising, it hadn't really been seen before. Whereas men, especially men with a smoking history and cancer that was called squamous non-small cell lung cancer did significantly better with the [Cabometyx]-containing treatment.”

Serious side effects occurred in 71 patients (38.4% of patients) and 58 patients (34.7% of patients), respectively, with grade 3/4 treatment-related side effects occurring in 73 patients (39.5% of patients) and 58 patients (34.7% of patients), respectively. Death occurred in 14 patients (7.6% of patients) and 10 patients (6% of patients), and was treatment-related in four patients (2.2% of patients) and one patient (0.6% of patients), respectively, according to study findings.

“I believe that [Cabometyx] is an effective drug in non-small cell lung cancer, [but] it's probably effective for a small population of patients though,” said Neal. “And when we do big, big studies like this in a large, unselected population of patients, it's really hard to see that benefit that we see in small populations. So research is still ongoing with [Cabometyx] and related molecules to see exactly what that niche population is.”

Tecentriq plus Cabometyx conferred no overall survival benefit when compared with chemotherapy, a study showed. 

Tecentriq, Cabometyx May Not Improve Survival Over Chemo in NSCLC

The Food and Drug Administration (FDA) has granted accelerated approval to Ojemda (tovorafenib) for the treatment of patients 6 months old and older with relapsed or refractory pediatric low-grade glioma (LGG) with a BRAF fusion or rearrangement, or BRAF V600 mutation.

According to the FDA, this is the agency’s first approval of a systemic therapy for the treatment of patients with pediatric LGG with BRAF rearrangements, including fusions.

findings from the FIREFLY-1 clinical trial

 of patients with relapsed or refractory pediatric LGG with an activating BRAF alteration following at least one line of prior systemic therapy.

The overall response rate (patients whose disease responded partially or completely to treatment) was 51%, with a median duration of response of 13.8 months.

The most common side effects occurring in at least 30% of patients were rash, hair color changes, fatigue, viral infection, vomiting, headache, hemorrhage, fever, dry skin, constipation, nausea, dermatitis acneiform and upper respiratory tract infection.

The most common moderate or severe (grade 3 or 4) laboratory abnormalities in more than 2% of patients were decreased phosphate, decreased hemoglobin, increased creatinine phosphokinase, increased alanine aminotransferase, decreased albumin, decreased lymphocytes, decreased leukocytes, increased aspartate aminotransferase, decreased potassium and decreased sodium, the agency reported.

According to the FDA, the recommended dose of Ojemda based on body surface area is 380 milligrams/m2 orally once weekly up to 600 milligrams orally once a week with or without food until disease progression or intolerable toxicity.

The FDA has granted accelerated approval to Ojemda for patients 6 months and older with relapsed or refractory pediatric low-grade glioma with a BRAF fusion or rearrangement, or BRAF V600 mutation.

FDA Approves Ojemda for Pediatric Low-Grade Glioma

On my morning walk today, I encountered a pileated woodpecker religiously hammering its beak against a tree. As I observed this diligent bird, I thought about my emotional state a month ago and couldn't help but laugh. Its persistent pecking reminded me of the anxiety I felt a month ago before my Lynch syndrome cancer screenings.

Scanxiety often resembles the futile act of repeatedly striking one's head against a tree, sans the woodpecker's remarkable physical adaptations. The bird's hyoid bone, tightly enveloping its skull with each peck, serves as a protective shield, protecting its delicate brain from harm.

As I watched the woodpecker's relentless endeavor, an analogy unfolded. Every strike of its beak resonated with the familiar rhythm of scanxiety—a ruthless, unyielding beat. For me, anxiety transcends fleeting worry or momentary discomfort; it's an enduring force that can feel as fruitless as pounding one's head against a solid surface.The woodpecker's behavior reflected the familiar feeling of futility that often accompanies anxiety, where despite tireless efforts, the desired outcome seems just out of reach.

Still, amidst this shared struggle, solace emerged as I contemplated the woodpecker's remarkable adaptation: its hyoid bone. This seemingly simple anatomical feature assumed profound significance as I pondered its role as a protective barrier for the woodpecker's brain. The hyoid functions as a guardian with each peck, ensuring the bird's safety and wellbeing despite repeated head trauma.

Three weeks have elapsed since my return from Mayo. Since my last check-up a year ago, two benign polyps have surfaced—one in my stomach, the other in my duodenum. It is a firm reminder of the imperative and meticulous regular screenings — a necessary defense against Lynch syndrome's elusive nature.

To complicate matters, inflammation was discovered in my duodenum, and in turn, this has caused gastritis, potentially exacerbated by the daily aspirin intake aimed at preventing colorectal cancer. Navigating the management of one condition while inadvertently aggravating another one highlights the intricate balancing act inherent in life with Lynch syndrome— the juggling of priorities packed with potential consequences. I learned this lesson the hard way when I opted for the prophylactic removal of my ovaries to prevent ovarian cancer. Most medical decisions entail unsavory trade-offs.

Thinking of my family's medical history and the looming shadow of cancer, I'm reminded of the daunting prospect of facing similar health battles to my brothers. Preserving my physical integrity remains my priority, focusing on quality of life over mere longevity. The thought of undergoing treatments that might diminish my physical wellbeing is met with understandable hesitation.

I recounted the woodpecker encounter to my best friend this morning. She was well aware of my vulnerability and fragility before my scans, yet she laughed and remarked that I possess a spine of steel, unlike anyone else she knows. 

Following my morning stroll through the woods, I realized that akin to the woodpecker's protective adaptations and my moments of vulnerability, I possess an inner strength adept at confronting anxiety head-on. The woodpecker's presence evolved into a poignant symbol, serving as a reminder of resilience, fortitude amid vulnerability, and the importance of facing life's obstacles with determination and grace, even during the most challenging times.


While strolling through the woods, my eyes landed on a woodpecker, whose actions sparked a cancer analogy in my mind.

Woodpecker Wisdom Unfolds a Cancer Analogy 

The Food and Drug Administration (FDA) approved Lutathera (lutetium Lu 177) for the treatment of children aged 12 years and older with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut and hindgut neuroendocrine tumors, the agency announced in a press release.

This is the first FDA approval of a radioactive drug for this patient population, the release noted.

The approval is based off findings from the ongoing phase 2 NETTER-P clinical trial evaluating Lutathera in patients between the ages of 12 and 17 with locally advanced or inoperable or metastatic SSTR-positive GEP-NETs or pheochromocytoma/paraganglioma (PPGL). Outcomes from 

, which included 229 patients with locally advanced or inoperable or metastatic SSTR-positive midgut carcinoid tumors, were also considered by the FDA in the approval process.

Lutathera Plus Sandostatin Boosts Outcomes in Advanced NETs

NETTER-P included nine children, four of whom had GEP-NETS. The main goal of the trial was to measure absorbed radiation doses in certain organs, as well as the incidence of side effects after the first treatment cycle. Researchers also looked at short-term side effects.

According to the study’s listing on ClinicalTrials.gov, the estimated study completion date is May 2029.

Lutathera was approved for patients 12 or older with SSTR-positive gastroenteropancreatic neuroendocrine tumors, the FDA announced. 

FDA Approves Lutathera for Pediatric GEP-NETs

A novel treatment for certain patients with relapsed or refractory acute myeloid leukemia has received a Breakthrough Therapy Designation by the FDA.

The Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to 

 for the treatment of patients with relapsed or refractory (R/R) NPM1-mutant acute myeloid leukemia (AML), according to an announcement from ziftomenib manufacturer Kura Oncology, Inc.

Breakthrough Therapy Designation, as explained on the FDA’s website, “is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).”

According to the announcement from Kura Oncology, ziftomenib is the first investigational treatment to receive Breakthrough Therapy Designation for the treatment of NPM1-mutant AML. The Breakthrough Therapy Designation comes nearly five years after the agency granted ziftomenib an Orphan Drug Designation for the treatment of AML.

“We are highly encouraged by FDA’s decision to grant Breakthrough Therapy Designation to ziftomenib, recognizing its potential as an innovative medicine for patients with relapsed/refractory NPM1-mutant AML,” stated Troy Wilson, president and chief executive officer of Kura Oncology, in the company’s news release. “NPM1-mutant AML represents approximately 30% of new AML cases annually, and this designation reflects that NPM1-mutant AML is a disease of significant unmet need for which there is no approved targeted therapy as well as the fact that ziftomenib offers potential to demonstrate substantial improvement over available therapies. We remain committed to bringing ziftomenib to the market as quickly as possible and look forward to working more closely with FDA to bring our ziftomenib program to patients in urgent need of effective treatments.”

The American Cancer Society reported that approximately 62,770 new cases and 23,670 deaths from leukemia will arise in 2024. Approximately 20,800 will be cases of AML, with about 11,220 deaths from AML, the majority being adults.

A Look at the Nutritional Impacts of an AML or MDS Diagnosis

The FDA, according to Kura Oncology, granted Breakthrough Therapy Designation to ziftomenib based on results from 

, with the manufacturer reporting that treatment with a 600-milligram oral daily dose of standalone ziftomenib resulted in a 35% complete remission rate (the disappearance of all signs of cancer) among 20 patients with NPM1-mutant AML.

With approximately 199 participants, the study is currently recruiting for its second phase, according to its listing on clinicaltrials.gov. The company stated that it expects to complete enrollment by mid-2024.

Vitamin C/D Supplements Decrease Complications in Patients with AML

Ziftomenib prevents the interaction of two proteins, menin and KMT2A/MLL. If joined, they are responsible for the survival and proliferation of some leukemia cells, according to Kura Oncology’s website. The site notes that blocking this process allows cancerous juvenile cells to mature into healthy white blood cells.

According to the manufacturer, ziftomenib is also currently being evaluated for administration in combination with current standards of care. These include Venclexta (venetoclax)/azacitidine or cytarabine plus daunorubicin for patients with NPM1-mutant and KMT2A-rearranged AML in the KOMET-007 trial as well as with Xospata (gilteritinib), FLAG-IDA or LDAC among patients with NPM1-mutant and KMT2A-rearranged AML in the KOMET-008 clinical trial.

Researchers in KOMET-007 began dosing patients in 2023, while 

it was reported that the first patient was dosed in KOMET-008 in March

The investigational drug ziftomenib is currently being evaluated for patients with relapsed/refractory NPM1-mutant acute myeloid leukemia.

Ziftomenib Granted Breakthrough Therapy Designation by FDA in R/R AML

'Twas love I'd wager placed the defect there

within the wrinkle of your smile concealed.

The surgeon's blade our woe away might pare

its poison'd root and grew into your face.

it was removed. Does there remain a trace?

and need now but recall our pledge of troth —

For we are two in faith each with our part

but for all days to come we share one heart.

Some may ask as have I from sleepless bed

Not why, to question why leaves just because,

On earth might one snatch hope from cancer's jaws

The grip of dark that daylight might reveal

Of tears, a raft of hope, with steady keel.

Love's last trial waits, not while we are yet here

Atop the dune hills dreaming hand-in-hand

One set of footprints only press the sand

But whose? It matters not ('tis my belief)

My hope — that waves may wash away some grief

My wife Debra was diagnosed with stage 3B melanoma on her face and in a nearby sentinel lymph node in March 2021. Last February, we discovered that it migrated to the lymph nodes in her neck (although the doctors still maintain that it is not stage 4). She has been a member of the Facebook "Melahomies" site since that time.

During the first few months after Deb's diagnosis, while she was undergoing immunotherapy, we started having regular, honest and often difficult, conversations about the uncertainty of our future together because of this insidious disease. We continue to do so and are able to speak openly about our fears. We use the comfort and strength of our relationship to find a way forward.

This post was written and submitted by Joe Guastella. The article reflects the views of Guastella and not of CURE®. This is also not supposed to be intended as medical advice.

When my wife was diagnosed with stage 3B melanoma, we decided to openly speak about our fears.

Speaking Openly About Cancer-Related Fears

Anktiva plus BCG is used to treat certain patients with BCG-unresponsive non-muscle-invasive bladder cancer.

The Food and Drug Administration (FDA) has approved Anktiva (nogapendekin alfa inbakicept-pmln) with Bacillus Calmette-Guérin (BCG) for adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors, the agency announced.

approval was based on the QUILT-3.032 trial

 of 77 patients with BCG-unresponsive, high-risk NMIBC with CIS with or without Ta/T1 papillary disease who had undergone transurethral resection, with patients receiving Anktiva with BCG followed by maintenance therapy for up to 37 months.

The complete response rate (CR; the disappearance of cancer) was 62%, with 58% of patients having a CR duration of response of at least 12 months and 40% having a duration of response of at least 24 months.

The most common side effects occurring in at least 15% of patients, including laboratory test abnormalities, included increased creatinine, dysuria, hematuria, urinary frequency, micturition urgency, urinary tract infection, increased potassium, musculoskeletal pain, chills and pyrexia.

According to the FDA, the recommended dosage of Anktiva is 400 micrograms administered intravesically with BCG weekly for six weeks as induction therapy and a second induction course may be administered if patients do not experience complete response at month 3. Then, the recommended maintenance dose is 400 micrograms administered intravesically with BCG once a week for three weeks at months 4, 7, 10, 13 and 19, totaling 15 doses. Maintenance instillations with BCG may be administered for patients with an ongoing complete response at month 25 or later once a week for three weeks at months 25, 31 and 37 for up to nine additional instillations.

The agency advised that treatment be discontinued if the disease persists after the second induction, recurs or progresses, or if patients experience unacceptable toxicity, with a maximum treatment duration of 37 months.

A treatment combination of Anktiva and Bacillus Calmette-Guérin was approved by the FDA for certain patients with non-muscle-invasive bladder cancer.

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