Radically different
Nature Medicine, Published online: 02 June 2026; doi:10.1038/s41591-026-04420-8
An ancient concept gathers scientific evidence as medically tailored meals show potential to lower healthcare utilization and costs — but larger studies are needed alongside policies to scale and sustain evidence-based approaches.
Nature Medicine, Published online: 02 June 2026; doi:10.1038/s41591-026-04407-5
Between 2020 and 2023, in Massachusetts, receiving medically tailored meals was associated with reductions in hospitalizations and emergency department visits and lower healthcare costs.
Nature Medicine, Published online: 02 June 2026; doi:10.1038/s41591-026-04442-2
A randomized, single-blind, parallel-group, phase 1b clinical trial compared fecal microbiota transplant or a 15-strain live biotherapeutic product (MTC01) derived from the same donor and found similar efficacy and engraftment between treatments.
The rapid rise of GLP-1 receptor agonists has transformed the treatment of obesity and type 2 diabetes. Now, a large observational study suggests these drugs may also play a role in cancer prevention.
Researchers from the Perelman School of Medicine reported that women taking GLP-1 medications were significantly less likely to develop breast cancer than comparable women who did not receive the drugs. The findings, presented at the 2026 American Society of Clinical Oncology Annual Meeting and simultaneously published in JCO Oncology Practice, add to a growing body of evidence linking GLP-1 therapy to reduced cancer risk.
While the study cannot establish causation, investigators say the results provide a strong rationale for prospective clinical trials evaluating whether GLP-1 drugs could become part of future breast cancer prevention strategies.
GLP-1 receptor agonists were originally developed to improve blood glucose control in patients with type 2 diabetes. More recently, agents such as semaglutide and tirzepatide have become widely used for weight management because of their ability to produce substantial and sustained weight loss.
Interest in the drugs has expanded beyond metabolic disease as researchers have begun exploring their broader biological effects. Obesity is a well-established risk factor for postmenopausal breast cancer, and GLP-1 therapies influence several pathways implicated in cancer development, including chronic inflammation, insulin signaling, and metabolic regulation.
“While our study was observational and does not definitively confirm an association between GLP-1 medications and reduced breast cancer incidence, it does add to the growing body of evidence suggesting that it’s worth investigating these weight-loss drugs as potential cancer prevention tools,” said Elizabeth McDonald, MD, PhD, professor of radiology at Penn Medicine and lead investigator of the study.
The researchers analyzed electronic health records from women who underwent breast imaging at Penn Medicine between January 2022 and June 2025.
The primary analysis included nearly 95,000 women between the ages of 45 and 80 who had a body mass index above 25, placing them in the overweight or obesity categories. Approximately 15,000 women had documented exposure to a GLP-1 medication before breast imaging, while nearly 80,000 had no recorded use of the drugs.
Breast cancer was diagnosed in 1.65% of women who had received a GLP-1 prescription compared with 2.6% of women who had not.
After statistical analysis, GLP-1 use was associated with a 37% reduction in the odds of developing breast cancer.
To address potential confounding factors, investigators also performed a propensity-matched analysis pairing GLP-1 users and non-users based on age, race, ethnicity, body mass index, breast density, and diabetes status.
The association remained significant in the matched cohort, where GLP-1 exposure was linked to approximately 25% lower odds of breast cancer diagnosis.
“These findings support the need for prospective trials investigating incretin medications for breast cancer prevention,” the authors concluded in their ASCO abstract.
The biological explanation remains uncertain, but several plausible mechanisms exist.
Weight loss itself is likely an important contributor. Excess adipose tissue promotes inflammation, alters hormone levels, and creates a metabolic environment associated with increased breast cancer risk, particularly after menopause.
However, researchers suspect the benefits may extend beyond weight reduction alone.
GLP-1 receptor agonists have been shown to reduce systemic inflammation, improve insulin sensitivity, and influence metabolic pathways that may contribute to tumor initiation and growth. Emerging laboratory studies have also suggested potential effects on cellular signaling and epigenetic regulation.
“GLP-1 medications are intriguing from a cancer research perspective because they weren’t designed for cancer therapy, but they do affect many different targets and pathways associated with cancer development, so we’re eager to study them in this context,” McDonald said.
The findings arrive at a time when breast cancer prevention options remain limited.
For women at very high genetic risk, preventive mastectomy can dramatically reduce cancer incidence but is an irreversible surgical intervention. Anti-estrogen therapies such as tamoxifen can also reduce risk substantially, yet uptake has historically been low because of concerns about side effects.
In contrast, GLP-1 medications are already being used by millions of patients worldwide and have established safety profiles in obesity and diabetes management.
If future prospective studies confirm a protective effect, these agents could potentially represent a new category of pharmacologic prevention for women at elevated breast cancer risk.
Researchers at Penn are already planning a multisite clinical trial that will evaluate whether GLP-1 therapy can reduce breast cancer incidence among high-risk women, including breast cancer survivors.
Despite the encouraging findings, the study has several limitations typical of retrospective analyses.
The investigators did not evaluate individual GLP-1 agents, duration of treatment, genetic predisposition, breast cancer subtype, or stage at diagnosis. Residual confounding also remains possible despite matching analyses.
Because the study was observational, it cannot determine whether GLP-1 therapy directly lowers breast cancer risk or whether other factors associated with medication use contributed to the observed differences.
Nevertheless, the consistency of the signal across multiple analyses strengthens the case for prospective investigation.
“Ultimately, we want to find better options to prevent breast cancer,” McDonald said. “It’s been encouraging to see the survival rates for breast cancer improve over recent decades, and we’d love to see the same gains in prevention.”
As GLP-1 therapies continue to reshape obesity treatment, researchers are increasingly asking whether their benefits extend far beyond weight management. This study suggests breast cancer prevention may be one of the most important questions to answer next.
The post GLP-1 Weight Loss Drugs Linked to Lower Breast Cancer Risk appeared first on Inside Precision Medicine.
The word patient comes from the Latin patiens — an adjective meaning enduring, suffering. In medicine, that endurance has long meant waiting: waiting to be seen, to be diagnosed, to be treated. Over the past two decades of practicing emergency medicine, my shifts have begun the same way — walking past a room full of people waiting for care.
That room is not called a lobby or a reception area. It is called a waiting room, because the expectation of waiting is built into the architecture and culture of medicine. Triage — the systematic process of prioritizing patients by the severity of their condition — determines the length of the delay. The sickest are seen first; everyone else bears both their illness and the constraints of the system they have turned to for help.
In less than a decade, telehealth has expanded from a sideshow of health care to an industry worth tens of billions of dollars. Companies like Hims & Hers and Teladoc have become household names, their ads interrupting streaming TV and flooding social media feeds with the promise of quick, convenient care.
Despite their popularity, few patients understand who’s actually taking care of them when they click through a telehealth site.
National telehealth brands present a unified front to patients across the country. But in more than 30 states, it’s illegal for corporations to practice medicine. So behind the scenes, telehealth companies work with distinct, independent medical groups. Owned by physicians — who often hold 50 state licenses at once — those practices are meant to act as a firewall, making sure that clinical decisions are driven by patients’ needs, not the profits of the corporations they deal with.
No one wants to pay for genetic medicines, especially gene editing therapies. This is a painful truth that patient advocates and startup founders alike have to face.
For years, leaders, even pioneers of CRISPR gene editing technologies, have passionately pleaded with government, banks, and the pharmaceutical industry for support. It only gets harder to hear when patients and their families are begging and praying, their fates blown one way or another by the actions of massive entities that shape the development, funding, and regulation of these potentially life-saving drugs. On the patient side, parents have been on the fence about research-as-care gene editing therapies because of the (rightly) perceived cost, millions of dollars.
Most gene-editing medicines target tiny patient populations, often numbering in the thousands worldwide, and can cost millions of dollars per patient. Manufacturing and delivery are far from solved. And despite years of excitement around CRISPR and related technologies, gene editing has yet to reshape mainstream medicine.
So, most parents don’t even get to choose whether to sell their house to save their child—they just watch and wait. For the thousands of monogenic inherited diseases, gene editing is a mirage, seemingly forming on the horizon only for it to vanish when reached.
That the rate-limiting factor is not in the science is clear as day. For nearly a decade, gene editing has carried the aura of scientific revolution while remaining, in practical terms, a niche corner of medicine. But the economics and scale of those therapies have remained limited.
Pharma behemoth Lilly appears to have picked up an ax, struck into this frozen wall, and made a sizeable crack with their experimental in vivo base-editing therapy VERVE-102. Originally developed by Verve Therapeutics, a recent Lilly acquisition, VERVE-102 is designed to permanently reduce cholesterol by switching off the PCSK9 gene inside liver cells. In adults with atherosclerotic cardiovascular disease (ASCVD), one dose of VERVE-102 should reduce PCSK9 and LDL-C levels safely, significantly, and sustainably.
Early clinical results, published in the New England Journal of Medicine, from 35 participants with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) showed just that. This ongoing Heart-2 Phase Ib study could lead to the approval of a one-time, possibly best-in-class treatment for a condition that is a leading cause of death of adults worldwide. If ultimately validated, VERVE-102 could reshape cardiovascular medicine.
The trial is also one of the clearest signs that the biotech industry is progressing in transforming gene editing from a boutique technology for rare disease into a scalable platform for chronic illness.
Gene-editing wizard and base editor inventor David Liu, PhD, told Inside Precision Medicine, “I consider VERVE-102 to be important in several ways—as the first example of clinical in vivo base editing (note that now the majority of the 23+ clinical trials that use base or prime editing are in vivo!); as an effective, one-time treatment for a serious genetic disease (FH); and as a key demonstration of the use of precision gene editing for disease PREVENTION, not just for the correction of pathogenic mutations. In this case, lowering LDL reduces the risk of the #1 killer of humans around the world, so the potential implications of using precision gene editing to lower disease risk are vast.”
Uli Stilz, PhD, senior advisor at Flagship Pioneering, board member to biotech companies and venture capital funds, and advocate for using genetic editing approaches to treat complex diseases, echoed Liu’s statements. “VERVE-102 is important not simply because of the clinical results, but because it continues to test a much larger hypothesis: can one-time genetic medicines become practical therapies for common chronic diseases?” Stilz told Inside Precision Medicine. “The field is moving beyond proof of concept. The next challenge is demonstrating the combination of safety, durability, scalability, and clinical utility required for broad patient populations. If successful, this could represent a fundamental shift in how we think about the prevention and treatment of cardiovascular disease.”
Two significant events from ten years ago underpinned the VERVE-102 results released this week. In April 2016, Alexis Komor, PhD, and Liu published the first base editor— programmable editing of a genomic DNA target base without double-stranded DNA cleavage— in Nature, rocking the gene editing world. The other, the idea behind Verve Therapeutics, was kept under wraps and thus did not cause nearly as much of a stir.
From its conception to its official founding in 2018, Verve Therapeutics co-founders Sekar Kathiresan, MD, and Kiran Musunuru, MD, PhD, anticipated that delivering gene-editing therapies safely into the body would be its biggest challenge. Kathiresan and Musunuru, one half of the clinical duo at the heart of the baby KJ story, intentionally developed multiple product candidates with different lipid nanoparticle (LNP) delivery systems to reduce that risk.
This approach became particularly important after Verve halted its Heart-1 trial of VERVE-101 in April 2024 due to a severe but reversible adverse event in one patient, which Verve attributed to the LNP delivery shell rather than the gene-editing machinery itself. While VERVE-101 used an Acuitas-developed LNP, Verve’s newer candidates, VERVE-102 and VERVE-201, rely on a second-generation GalNAc-enhanced LNP designed to improve both safety and targeting efficiency.
Despite the trial setback and stock decline, Verve showed the first human proof of concept for in vivo base editing, with permanent PCSK9 gene editing reducing LDL-C in early patients. A bit over a year later, in June 2025, Lilly made a prophetic acquiring Verve for north of $1 billion in all-cash. About two years after VERVE-101 and the Heart-1 trial were paused, VERVE-102’s historic results were announced and published.
In the interim Heart-2 analysis, Verve researchers treated 35 participants with HeFH or premature CAD across six escalating dose cohorts ranging from 0.3 mg/kg to 1.0 mg/kg. The results showed clear dose-dependent effects: mean reductions in circulating PCSK9 protein ranged from 51% at the lowest dose to 88% at the highest, and LDL-C reductions ranged from 9% to 62%, with participants in the highest-dose group seeing an average absolute LDL reduction of 78 mg/dL. Perhaps most striking was VERVE-102’s durability. Some participants have now been followed for up to 18 months, and cholesterol lowering has thus far persisted throughout follow-up.
Marc S. Sabatine, MD, chairman of the Thrombolysis in Myocardial Infarction (TIMI) Study Group, Endowed Chair in Cardiovascular Medicine at Brigham and Women’s Hospital (BWH), and professor of medicine at Harvard, put the efficacy findings into context with existing cardiovascular medications. In speaking with Inside Precision Medicine, Sabatine said, “The reductions in LDL-C are on par with what has been seen for the monoclonal antibody PCSK9 inhibitors and, more recently, the oral PCSK9 inhibitor enlicitide. Moreover, the effect appears durable, with data out to one year.”
Researchers reported no dose-limiting toxicities. The most common side effects reported from the Heart-2 trial were mild-to-moderate infusion-related reactions and temporary elevations in liver enzymes. One participant developed aspiration pneumonitis associated with pre-existing gastroesophageal reflux disease.
According to Lilly, with no unanticipated safety findings observed, the overall safety profile remains encouraging. Sabatine added, “In this small number of patients, the safety profile appears good so far, with only mild to moderate infusion reactions and only transient, mild elevations in levels of aminotransferases.”
All of the participants are expected to enroll in a long-term follow-up study for up to 15 years. The long-term follow-up requirement reflects both the promise and the tension of permanent gene editing. Unlike conventional drugs, DNA edits may persist for decades—potentially for life. That raises the possibility of lifelong benefit from a single treatment but also creates unusually high regulatory scrutiny around safety and durability.
“These data, although very preliminary, are exciting,” Sabatine said. “If these observations are confirmed in larger and longer studies, VERVE-102 would be another powerful tool in our PCSK9 inhibitor armamentarium, which might someday allow patients to choose between a daily pill, periodic injections, or a once-in-a-lifetime infusion.”
According to a Lilly spokesperson, enrollment in the Heart-2 study is ongoing, together with continued participant follow-up. Together with the safety findings, these data will help inform dose selection for Phase II of the study, which is expected to begin before year-end.
Lilly isn’t the only horse in the race, which began to heat up in March 2026 when Chinese researchers published a study in Nature using their own in vivo PCSK9 base editor delivered via GalNAc-modified LNPs YOLT-101 to patients with HeFH. In a Phase I study with Shanghai Jiao Tong University School of Medicine and the Shanghai Key Laboratory of Precision Gene Editing and Clinical Translation, YolTech Therapeutics reported that YOLT-101 reduced LDL-C by 52.3% and circulating PCSK9 protein by 74.4% at 24 weeks in patients receiving the highest dose.
Lilly appears to be making a calculated bet that gene editing can evolve into a commercially viable platform for chronic disease—not just scientifically, but economically. That would represent a dramatic shift for the field. Rare disease therapies often justify high prices because they target extremely small patient populations. But cardiovascular disease operates on an entirely different scale. Elevated LDL-C affects hundreds of millions of people worldwide. Even a fraction of that market would dwarf nearly every current gene therapy indication.
The challenge is that gene-editing medicines have historically struggled with scalability, manufacturing costs, and delivery efficiency. To succeed commercially in chronic disease, companies will likely need therapies that are easier to manufacture, simpler to administer, safer in broader populations, and capable of producing durable benefit from a single intervention.
At the same time, Lilly is positioning genetic medicine not as a niche business unit but as a central long-term pillar of the company’s research strategy. According to Lilly, VERVE-102 is a key part of Lilly’s growing genetic medicines portfolio, which now accounts for more than one-third of the company’s R&D portfolio.
Since the Verve acquisition, Lilly has made several acquisitions that echo Kathiresan and Musunuru’s concern with genetic medicine delivery. The acquisitions of Kelonia Therapeutics and EngageBio expand Lilly’s genetic medicine capabilities with novel viral and non-viral in vivo gene delivery and integration technology, respectively, that has potential for broad applicability. Since these acquisitions are only applicable in oncology, Lilly will face a major challenge in expanding its targeting from the liver, the default homing site for many LNPs, to the rest of the body.
The company’s strategy reveals a broader industry transition now underway: using the massive profits generated by today’s blockbuster chronic-disease drugs to finance the development of entirely new therapeutic paradigms. Lilly’s current growth has heavily relied on incretin-based obesity and diabetes drugs, medicines that typically require continuous use. Gene editing, by contrast, could theoretically reduce or eliminate recurring treatment altogether. The Lilly spokesperson added, “Incretin-based therapies are transforming care for many people today, while genetic medicines represent a longer-term investment in approaches that could potentially shift some diseases from chronic management toward more durable intervention.”
These complementary approaches appear to be sufficient in making the risky and costly field of gene editing a strategic bet for Lilly. It may even be necessary. For pharmaceutical companies, that creates both a challenge and an opportunity: replacing recurring chronic revenue models with potentially curative interventions that may command large upfront prices but fewer repeat prescriptions.
The VERVE-102 data also highlight how rapidly genetic medicine is expanding beyond its original targets. “Genetic medicines have historically focused on rare diseases, and we believe they continue to hold significant potential for areas that have received less attention due to smaller patient populations and limited commercial incentives,” said a Lilly spokesperson. The company is exploring opportunities to apply these platforms across a range of conditions, “including inherited sensory disorders such as genetic hearing and vision loss, as well as chronic diseases where the underlying biology is well understood.”
What the early results from Heart-2 show is that gene-editing drugs need not only be reactive. Rather, if used preventively, drugs like VERVE-102 may ultimately prove the most disruptive aspect of gene editing in common disease. Lilly says that the early results from the Heart-2 study offer “an encouraging example of the potential in chronic diseases—treating disease at its source but also potentially shifting care from reactive treatment to earlier intervention and, in some cases, prevention.”
Historically, cardiovascular medicine has focused on lowering risk after damage has already begun accumulating. Patients develop plaque over decades, then receive progressively more intensive therapies as disease advances. A one-time gene-editing intervention administered earlier in life could theoretically alter that trajectory before severe disease develops.
Lilly and Verve researchers are already exploring adjacent approaches. In addition to the PCSK9 program, the Pulse-1 Phase Ib trial for VERVE-201 is testing another in vivo gene-editing therapy targeting ANGPTL3, another important regulator of lipid metabolism.
Together, the programs suggest a future in which cardiovascular disease may eventually be treated not with escalating layers of medication, but with molecular interventions designed to reset risk factors permanently at their genetic source. That future remains far from certain. Researchers still need to prove long-term safety, establish cardiovascular outcome benefits, and demonstrate that permanent editing can be deployed reliably across broad populations.
But the direction of the gene editing field is clearly no longer confined to the rarest diseases in medicine. And if therapies like VERVE-102 ultimately succeed, the age of in vivo gene editing may no longer be defined by rare disease at all, but by the much larger ambition of rewriting the treatment model for chronic illness itself.
The post Reporter’s Notebook: Are One-Shot Gene Editors Ready to Tackle Chronic Illness? appeared first on Inside Precision Medicine.
Researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Italy have developed a gene editing platform they say improves the precision and safety of CRISPR-Cas9 gene editing in human hematopoietic stem and progenitor cells (HSPCs), a development that addresses the long-standing difficulty of ensuring that edited stem cells carry only the intended genetic change without unintended genomic alterations. Details of the new platform, called SMArT (Selection by Means of Artificial Transactivators), are published in Nature Biotechnology.
“These unintended outcomes, such as large deletions of DNA sequences, have emerged as one of the most important limitations to the broader application of gene editing, especially in stem cells intended for transplantation,” said senior author Luigi Naldini, MD, PhD, director of SR-Tiget. “With SMArT, we aimed to create an intelligent selection system capable of identifying and enriching only those cells that achieved the desired genetic correction while excluding cells carrying potentially dangerous alterations.”
CRISPR-based therapies such as exagamglogene autotemcel (Casgevy) have already reached regulatory approval for hemoglobinopathies, the vanguard of the expected wave of CRISPR-based gene therapies. But drugs like Casgevy use CRISPR to knock out the disease causing gene, while approaches aimed at creating homology-directed repair (HDR), whereby a gene is inserted have been plagued by unintended genetic changes at the editing site. To date, HDR has been constrained by cell cycle limitations and competing DNA repair pathways that can generate deletions, rearrangements, and other structural changes at the target location.
SMArT was developed to address both low HDR efficiency and the unintended heterogeneity of the gene editing approaches. To do this the investigators created a method whereby transient synthetic “AND-gate” circuits are activated only when two conditions are met: successful targeted integration of a gene-sized cassette and preservation of correct genomic architecture at the edited locus. Only these cells transiently express a selectable marker, enabling their enrichment while removing cells carrying aberrant edits.
“HDR-edited HSPCs were enriched to 80–100% purity through transient selector expression, whereas cells carrying undesired and potentially genotoxic on-target edits were preferentially depleted,” the researchers wrote. They also showed that after transplantation into immunodeficient mice, the enriched cells sustained long-term human hematopoiesis, and the selector system was no longer detectable.
SMArT was developed in three configurations SMArT-1, SMArT-2, and SMArT-3. The most advanced version, SMArT-3, employs a CRISPR-based regulatory architecture that can detect correct integration of the edit at the intended site while also transiently activating genes to support stem cell engraftment. One version of this system uses a single programmable CRISPR-based regulatory system to reduce the need for multiple components.
In preclinical models, SMArT was tested in human HSPCs targeting loci relevant to inherited immune disorders, including X-linked severe combined immunodeficiency (SCID-X1) and Hyper-IgM syndrome. The system successfully enriched for correctly edited cells while reducing large deletions and other structural abnormalities associated with CRISPR-induced DNA repair. The selected cells demonstrated multilineage differentiation capacity after engraftment in mice, exhibiting preserved stem cell function.
“Our goal was not simply to improve editing efficiency, but to fundamentally rethink how to control the quality of edited cell products,” said co-senior author Samuele Ferrari, PhD, a project leader at SR-Tiget. “SMArT introduces a programmable framework that can simultaneously increase precision, reduce genotoxic burden, and preserve the functional potential of stem cells.”
The researcher noted that the SMArT platform is designed to be usable across loci and compatible with other genome engineering technologies beyond CRISPR-Cas9, including emerging targeted integration systems. The also believe it can be used to address multiple genetic diseases, particularly those requiring durable correction of blood or immune cell lineages.
Next steps include improving manufacturing efficiency, preserving clonal diversity during selection, and optimizing delivery methods for HDR templates. SMArT will also need evaluation to determine how it performs across different disease contexts and stem cell sources, as well as how it may integrate with non-myeloablative conditioning approaches to therapeutic development.
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Revolution Medicines reports that its investigational drug daraxonrasib doubled the overall survival of patients with metastatic pancreatic cancer compared to standard chemotherapy, according to results from the Phase III RASolute 302 trial. These findings were published yesterday in The New England Journal of Medicine and presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting.
“In this trial, daraxonrasib redefined treatment expectations in previously treated metastatic pancreatic cancer by reducing the risk of death by 60% and increasing median overall survival to more than one year, a result not previously reported in any Phase III clinical trial in any line of therapy for this disease,” said Mark A. Goldsmith, MD, PhD, chief executive officer and chairman of Revolution Medicines.
Pancreatic ductal adenocarcinoma (PDAC) is one of the deadliest cancer diagnoses, with a five-year survival rate of 3%. Currently available therapies offer very limited benefits for these patients, especially for those who have already been treated with a first line of chemotherapy and continued to see tumor progression.
In over 90% of pancreatic tumors, mutations that result in an overactivation of RAS(ON) signaling are a major driver of cancer growth. Daraxonrasib is an oral drug designed to block a broad spectrum of wild type and mutant RAS variants. The RASolute 302 trial set out to compare the effects of a daily dose of daraxonrasib to a standard chemotherapy course in 500 patients with metastatic pancreatic cancer who had previously received a first line of chemotherapy.
Results show that daraxonrasib increased the median overall survival from 6.6 to 13.2 months, while boosting progression-free survival from 3.5 to 7.3 months. Notably, these effects were consistent regardless of whether the tumors carried RAS mutations. Serious side effects were reported less frequently by patients who received daraxonrasib, reducing the likelihood of a patient having to discontinue treatment due to side effects by 10 times.
“Daraxonrasib significantly elevates the survival bar in the treatment of one of the deadliest human cancers, while better preserving quality of life compared to chemotherapy,” said Goldsmith. “These striking results firmly support daraxonrasib as the new standard of care for patients with previously treated metastatic pancreatic cancer, and usher in a new era of RAS-targeted therapy for patients living with this disease.”
Revolution Medicines has stated its intention to submit this data to the FDA and other regulators to seek approval of daraxonrasib in this patient population. Three other Phase III trials are currently underway, evaluating the drug in patients with PDAC and metastatic non-small cell lung cancer.
“These results from the Phase III RASolute 302 trial of daraxonrasib represent a major milestone for patients facing metastatic pancreatic cancer,” said Brian M. Wolpin, MD, director of the Hale Family Center for Pancreatic Cancer Research at the Dana-Farber Cancer Institute, professor of medicine at Harvard Medical School, and principal investigator for the RASolute 302 trial.
“For many patients, second line chemotherapy provides modest benefits, and new treatments delivering more durable tumor control have been urgently needed. These results will change how scientists, clinicians, and patients think about treatment for pancreatic cancer, and support a new paradigm where RAS(ON) inhibition enters standard of care for patients with previously treated metastatic pancreatic adenocarcinoma.”
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Results from the Phase IIb KEYNOTE-942 trial reveal that adding a personalized mRNA vaccine to standard immunotherapy after surgery can reduce the five-year risk of recurrence and death by 49% among patients with advanced melanoma. These findings were presented today at the annual meeting of the American Society of Clinical Oncology and published in the Journal of Clinical Oncology.
“Our study offers strong evidence to melanoma patients that intismeran vaccine therapy, when used in combination with immunotherapy, can demonstrably reduce their risk of having their cancer return and improve clinical outcomes,” said Janice Mehnert, MD, professor in the department of medicine at NYU Grossman School of Medicine and associate director of clinical research at Perlmutter Cancer Center.
Melanoma is one of the most common forms of cancer. Although immunotherapies like pembrolizumab (Keytruda) have significantly improved outcomes for melanoma patients, this cancer is still known for its ability to evade the immune system and become resistant to treatment.
Developed by Moderna, intismeran autogene is an mRNA cancer vaccine made specifically for each patient. The bespoke therapy is created by screening tumor samples for 34 neoantigens that can be leveraged to strengthen the immune system’s response against tumor cells and enhance the efficacy of immunotherapy.
The KEYNOTE-942 trial recruited 157 patients with advanced stages of melanoma and at high risk of recurrence who were randomized to either receive standard pembrolizumab immunotherapy, or a combination of pembrolizumab and intismeran. Since the treatment was administered after surgery, intismeran was individually manufactured for each patient based on an analysis of their resected tumor.
After five years, 68.8% of patients treated with the combination therapy remained alive and cancer-free, compared to 49.1% for those who received standard treatment. The addition of personalized vaccines also reduced the risk of developing a distant metastasis by 59%.
“Now with five years of follow-up data, today’s results highlight the potential of a prolonged benefit of the intismeran autogene and Keytruda combination in patients with resected high-risk melanoma,” said Kyle Holen, MD, senior vice president and head of development, oncology and therapeutics at Moderna. “We continue to invest in our platform in oncology because of encouraging outcomes like these, which illustrate mRNA’s potential in cancer care.”
A Phase III clinical trial is already underway to confirm the efficacy of intismeran as a first line therapy for melanoma in combination with pembrolizumab. Additional studies are looking into the effects of the cancer vaccine in other types of cancer, including non-small cell lung cancer, bladder cancer, and renal cell carcinoma.
“Our findings also serve as encouragement to cancer researchers globally that mRNA vaccines like intismeran could work well in combination with immunotherapy for other cancers whose high rates of mutations have proven difficult to target,” said Mehnert.
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