High-Risk Multiple Myeloma Redefined in Era of Modern Quadruplet Therapy

Advances in frontline treatment for multiple myeloma may prompt clinicians to rethink how they identify patients with the poorest prognosis, according to a study published in Cancer. Researchers report that patients whose disease progresses within 36 months—not the longstanding 18-month benchmark—should now be considered to have functional high-risk (FHR) multiple myeloma.

Functional high-risk disease has traditionally been defined as myeloma that progresses within 18 months of starting therapy, a group historically associated with survival of less than two years after relapse. However, widespread adoption of quadruplet therapy—combining an anti-CD38 antibody, a proteasome inhibitor, an immunomodulatory agent, and dexamethasone—followed by autologous stem cell transplantation (ASCT) has substantially prolonged remission for many patients.

To determine whether the historical definition remains appropriate, investigators from the University of Alabama at Birmingham and the CoMMiT consortium analyzed outcomes in 310 patients with newly diagnosed multiple myeloma treated with quadruplet therapy plus ASCT. Patients were followed for a median of 41.8 months (3.5 years), during which 66 experienced disease progression.

The researchers evaluated several definitions of FHR disease based on progression occurring within 12, 18, 24, or 36 months after treatment initiation. Their analysis found that progression within 36 months most accurately identified patients whose survival after relapse remained approximately two years or less despite current frontline therapy.

“The current analysis provides validation of FHR36 as the optimal classification for FHR multiple myeloma in the era of QUAD + ASCT, providing a benchmark for future therapies to be explored in this setting,” the authors conclude.

Using the new definition, 16.4% of patients were classified as having FHR36 disease. Many would not have been identified as high risk using conventional staging systems: more than one-third had lower-stage disease according to the second revised International Staging System, and more than half met standard-risk criteria under current International Myeloma Society guidelines.

The study also suggests that T-cell redirecting therapies (TCRTs), including CAR T-cell therapies and bispecific antibodies, may improve outcomes in this population. Although only 11 patients received TCRTs as second-line treatment, outcomes were markedly better than with conventional therapies.

The 12-month second progression-free survival rate was 80% for patients treated with TCRTs, compared with 23% for those receiving other therapies. Overall response rates were 91% versus 47%, respectively. Multivariable analysis showed that TCRT remained associated with improved progression-free survival after adjustment for FHR status.

The authors caution that these findings require confirmation because relatively few patients received TCRTs. Still, they argue that patients meeting the FHR36 definition should be prioritized for early T-cell redirecting therapy and enrollment in clinical trials evaluating novel agents.

“We believe FHR36 should be a standard definition for FHR going forward and that the safety and efficacy of experimental agents should be reported in this important subset of patients or dedicated trials should be designed and conducted for this special population,” the authors write.

 

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Patient-Derived Tumor Organoids Show Promise for Personalizing Cancer Treatment

The development of patient-derived tumor organoids for preclinical research will undoubtedly advance precision medicine research. Now, researchers have developed a pan-cancer patient-derived organoid (PDO) platform comprising 220 PDOs from 191 patients across 15 cancer types. This advance points to an increase in the use of tumor organoids as models for evaluating and optimizing cancer treatments.

This work is published in Science Advances in the paper, “Patient-derived organoids across cancers reveal conserved tumor heterogeneity and actionable therapeutic vulnerabilities.”

The team characterized the organoids extensively, showing that they retained key characteristics of the parent tumors over extended periods and showed promise in screens to identify unrecognized treatment candidates. The organoids had similarity to their parent tumors in terms of microscopic appearance, driver DNA mutations, gene expression patterns and other features.

More specifically, the comprehensive characterization demonstrated “high fidelity to parent tumors, with 93% histopathology concordance, 80% median genomic concordance for driver mutations, and a 0.85 median gene expression correlation.” Expression profiles remained largely stable over 10 passages, ensuring reproducibility for long-term screening. And clonality analysis, the authors note, showed that 85% of dominant tumor clones were preserved, with genomic concordance directly reflecting clonal similarity.

“Essentially, these organoids appear to be very good preclinical models of the parent tumor, and are practical models because they can be used long-term,” said Andrea Sboner, PhD, associate professor of pathology and laboratory medicine, director of informatics and computational biology in the Englander Institute for Precision Medicine and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell.

The team selected a subset representing patient tumors that, based on standard clinical criteria, had been deemed ineligible for treatment with a new class of drugs—PARP inhibitors. They then tested a PARP inhibitor, talazoparib, on the organoids, and found that more than half—58%—showed substantial sensitivity, implying that the current clinical criteria are excluding patients who could benefit from such drugs. The team characterized the mutational and other features that made these organoids susceptible—offering clues to how the clinical criteria might be expanded—and identified drugs that synergistically enhance talazoparib’s effects.

“You can use these organoids as patient ‘avatars’ during clinical trials of experimental therapies, for example, to get an early picture of treatment effects and side effects,” notes Juan Miguel Mosquera, MD, a professor of pathology and laboratory medicine and director of research pathology at the Englander Institute.

The scientists also envision the future use of tumor organoid technology in selecting treatments for individual patients—growing an organoid from a sample of the patient’s tumor and then testing it rapidly with different treatment regimens.

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New CVD Risk Tool Boosts Women’s Risk, Lowers Risk for Some Black Adults

A change in the guidelines used to judge a person’s cardiovascular risk in the U.S. means who is classed as ‘at risk’ or not depending on their lipid levels and other factors will change.

Men, non‑Hispanic Black adults, and current smokers are more often moved down, whereas women and people with diabetes would see their risk category upgraded with the new tool suggests research led by Allison Peng, MD, Johns Hopkins University School of Medicine, and colleagues.

The research is published in JAMA and compares risk classification with the new PREVENT-atherosclerotic cardiovascular disease (ASCVD) tool and the 2013 pooled cohort equations, which have been the standard tools for estimating 10-year atherosclerotic cardiovascular disease risk for a long time.

The team used data from the National Health and Nutrition Examination Survey, a large ongoing study that interviews and examines a sample of the U.S. population every year and focused on 24,403 adults aged 40 to 79 years who did not already have cardiovascular disease and did not have very high levels of low‑density lipoprotein (LDL) cholesterol.

For each person, the investigators estimated the chance of a heart attack or stroke in the next 10 years using both the old and new calculators, grouped them as low, borderline, intermediate, or high risk under each system, and then asked who moved up or down. They also examined whether this reshuffling would change who is advised to take cholesterol‑lowering medicines such as statins.

The new tool removes race as an input, adds more risk factors such as body mass index and markers of glycemia and kidney disease, and predicts both 10‑ and 30‑year cardiovascular risk, whereas the old tool focused on 10‑year ASCVD and treated race as Black vs. White.

The results show 22% of study participants changed risk category with the new calculator. Around 14% of all adults were placed into a lower‑risk group, while about 7% were moved up. Around 50% of the population continued to be classed as low risk, but the borderline group grew from about 13% to 18%, while the intermediate group shrank from about 25% to 20%, while the high‑risk group did not change much from about 12% to 11% with the new tool.

Key changes show women’s risk increasing and men’s decreasing, factors such as diabetes playing a bigger role in risk estimation and Black adults being generally classed as lower risk.

In the older framework, an intermediate or high risk often directly triggered a statin recommendation. With the new tool, more patients sit in the enlarged borderline group and would be offered additional tests like coronary artery calcium scoring and allowed shared decision‑making about medications and preventive treatment. Despite these shifts, the proportion of adults for whom statins would be recommended was almost unchanged at around 50% before and after.

“Overall, cardiovascular risk reclassification with the 2026…Dyslipidemia Guideline may result in opportunities for consideration of more personalized cardiovascular risk assessment with risk enhancers and coronary artery calcium scoring to guide lipid-lowering therapy and cardiovascular disease prevention,” write the authors.

In an accompanying editorial in the same journal, Philip Greenland, MD, Northwestern University Feinberg School of Medicine, and Karen Lasser, MD, Boston Medical Center, Boston University, note that despite improving risk assessments, many people who are at risk still do not get treatment or reach suggested goals for healthy cholesterol levels.

“A large percentage of U.S. adults aged 30 years or older are candidates for guideline-directed lipid lowering therapy, and many who are currently taking lipid lowering therapy are not reaching new (or previous) low density lipoprotein cholesterol goals. This is a huge public health burden for the U.S. population for a medical condition that should be highly treatable.”

They conclude: “Given the large numbers of people who would be recommended for treatment following a risk-based discussion, these reports signify a major challenge, and so far, a missed opportunity, for the U.S. health care system.”

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STAT+: Pharmalittle: We’re reading about a telehealth firm and patient safety, PBMs overcharging, and more

Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and invigorating because that oh-too-familiar routine of meetings, deadlines, and the like has returned with a vengeance. You knew this would happen, yes? To cope, we are relying, as always, on cups of stimulation. Our choice today is laced with traces of cocoa. Feel free to join us. Remember, no prescription is required. Meanwhile, here are some tidbits to help you along. Best of luck accomplishing your goals today and, of course, do keep in touch. …

Novo Nordisk, maker of Ozempic and Wegovy, lists the telehealth company LifeMD on its website as a provider that offers “legitimate medicine sourcing and patient support” for people seeking GLP-1 drugs. But some former employees describe LifeMD differently, as a company that has sought to maximize the volume of prescriptions it doles out at the expense of patient safety, STAT reports. Former workers told STAT that providers were pressed to expedite their work to a pace that was not clinically responsible, with two of them saying providers at times were expected to review the cases of 25 people per hour based only on electronic forms the patients filled out — the equivalent of spending about two minutes on each case.

A recent audit of Iowa Medicaid records revealed how pharmacy benefit managers are using complicated and sophisticated approaches for handling prescription drug claims that ultimately overcharge taxpayers, a finding that underscores controversy surrounding these crucial middlemen in the pharmaceutical supply chain, STAT explains. The audit found that one large pharmacy benefit manager appeared to have made more than $100 million by adjusting the amount of money paid to pharmacies without passing some of the savings back to managed care plans working on behalf of the state, according to Iowa officials. The review scrutinized records from 2019 through 2021.

Continue to STAT+ to read the full story…

Patient journey mapping in an open-door psychiatric inpatient unit

BackgroundOpen-door policies in inpatient psychiatric care have been increasingly adopted to enhance patient autonomy and reduce coercive practices. However, systematic methods for evaluating patient experiences in these settings remain limited. This study applied Patient Journey Mapping in an acute psychiatric unit operating a structured, time-limited (6 hours/day) open-door policy to comprehensively analyze the hospitalization experience.MethodsAn exploratory sequential mixed-methods design was used. Qualitative data were first collected through focus groups with patients (n = 18), following GRAMMS guidelines, to identify critical touchpoints across the hospitalization journey. Quantitative data were then collected from patients (n = 32) using an adapted Likert-type ordinal scale to assess satisfaction with the identified touchpoints. Data were analyzed using content analysis methods.ResultsThe analysis delineated the main phases of hospitalization and identified key experiential touchpoints, including “Moments of Truth,” “Moments of Pain,” and “Wow Moments,” which substantially shaped patients’ perceptions of care quality. Open-door policies were perceived as supporting autonomy and reducing psychological distress. However, several challenges remained, particularly regarding the admission process, privacy during medication administration, and communication about structured activities.ConclusionPatient Journey Mapping provides a useful patient-centered approach for evaluating experiences in open-door psychiatric inpatient settings. The findings highlight opportunities to optimize service delivery, strengthen therapeutic environments, and support recovery-oriented practices in acute psychiatric care.

STAT+: Telemedicine company touted by Novo Nordisk stressed profits over patient safety, ex-workers say

Novo Nordisk, maker of Ozempic and Wegovy, lists the telehealth company LifeMD on its website as a provider that offers “legitimate medicine sourcing and patient support” for people seeking GLP-1 drugs. But some former employees describe LifeMD differently, as a company that has sought to maximize the volume of prescriptions it doles out at the expense of patient safety.

Former workers told STAT that providers were pressed to expedite their work to a pace that was not clinically responsible, with two of them saying providers at times were expected to review the cases of 25 people per hour based only on electronic forms the patients filled out — the equivalent of spending about two minutes on each case.

The company also discouraged providers from asking what they felt were medically relevant questions to patients, so that they don’t “delay care,” former employees said.

Continue to STAT+ to read the full story…

STAT+: State audit of Medicaid records points to methods used by PBMs to obscure drug costs

A recent audit of state Medicaid records revealed how pharmacy benefit managers are using complicated and sophisticated approaches for handling prescription drug claims that ultimately overcharge taxpayers, a finding that underscores controversy surrounding these crucial middlemen in the pharmaceutical supply chain.

The audit of Iowa’s state Medicaid program found that one large pharmacy benefit manager appeared to have made more than $100 million by adjusting the amount of money paid to pharmacies without passing some of the savings back to managed care plans working on behalf of the state, according to Iowa officials. The review scrutinized records from 2019 through 2021.

The overall conclusion was similar to audit results conducted in a few other states, but in this instance, the auditors identified what amounted to creative accounting maneuvers, which not only made it possible to obscure the flow of money but evade prohibitions on a controversial pricing practice that is now outlawed in Iowa and some other states.

Continue to STAT+ to read the full story…

Organ Aging Linked to Breakdown in Immune Cell Interaction and Senescent Neutrophil Clearance

We may age at different rates, but none of us escapes aging. A study in mice and in human cells by Stanford Medicine researchers has linked organ aging to the increased inability—with advancing age—of tissue resident macrophage (TRM) immune cells to clear aged neutrophils, another type of immune cell.

The study found that these TRMs appear to be central coordinators of age-related organ decline. Blocking a single receptor, EP2, on these cells preserved the youthfulness of multiple organs in mice, including the brain, heart, skeletal and heart muscle, liver, spleen, bone marrow, kidney, and colon. The receptor binds specifically to a hormone, prostaglandin E2, which is known to cause inflammation and pain in humans as well as in mice.

The researchers found that in mice, selectively disabling this receptor exclusively on tissue-resident macrophages genetically, or using an experimental selective EP2 antagonist drug, prevented chronic-inflammation-driven disorders of age—including frailty, excessive fat accumulation, and heart trouble—and also substantially slowed cognitive decline.

Research lead Katrin Andreasson, MD, the Edward F. and Irene Thiel Pimley Professor in Neurology and Neurological Sciences, said, “We’ve shown that when tissue-resident macrophages don’t have EP2 on their surfaces anymore or when that receptor is plugged up by a drug, this decline doesn’t happen … We’ve been trying to figure out why we age. Now we know at least one big reason for it.”

The discoveries help to clarify systemic inflammation’s significant contribution to aging and the debilities that accompany it. The findings also point to a pharmaceutical approach that could restrain our organs’ unavoidable march toward senescence and so extend overall health span.

Senior author Andreasson, together with first author Jessy Tan, PhD, an instructor in neurology, and colleagues reported on their findings in Science, in a paper titled “Restored clearance of senescent neutrophils by tissue-resident macrophages limits organ aging.” In their research article summary, the team stated, “This work identifies EP2 signaling in TRMs as a central regulator of organ-wide aging through its control of senescent neutrophil clearance, reframing aging as a failure of active cellular clearance rather than passive degeneration.”

Aging is accompanied by parallel functional decline across organs, but the cellular drivers remain unclear, the authors wrote. “Although molecular hallmarks of aging have been identified, the cellular events that initiate and propel tissue decline remain poorly defined.”

The most abundant white blood cells in our immune system are neutrophils, which act as the body’s main first responders. Produced in bone marrow, new neutrophils are transferred to the bloodstream, where they circulate and attack bacterial, viral, or fungal pathogens that they encounter. Neutrophils are also extremely short-lived, surviving just 12–24 hours.

Some 90% of circulating neutrophils end up in the liver, spleen, and bone marrow, awaiting execution clearance by another type of immune cell. “Neutrophils are among the shortest-lived immune cells, aging within hours in the circulation and requiring continuous clearance,” the team noted.

This neutrophil clearance is critical. In aged animals, the vast bulk of neutrophils that never see combat undergo a fast transition to senescence, a zombie-like state in which they may injure, age, and inflame neighboring cells. And as we age, the neutrophil count rises, with senescent neutrophils constituting an ever higher percentage. “Senescent neutrophils are killing our tissues,” Andreasson said. “Clearance of these cells is essential for preventing chronic inflammation.”

That’s a job for macrophages. These cells comb the tissues for pathogens, signal other cells to lend a hand in the fight, and pump out growth factors that help repair damaged tissue. But first and foremost, Andreasson said, “They’re the body’s garbage collection crew. A lot of that garbage is defunct cells.” And a lot of those cells are neutrophils—to the tune of 100 billion a day.

Macrophages come in several subtypes. Tissue-resident macrophages are long-lived and ubiquitous. They take up residence in each of the body’s organs during fetal development and remain for their lifetimes in whatever organ they’ve inhabited, adapting their roles to fit that organ.

One of tissue-resident macrophages’ prime responsibilities is to swallow senescent cells. “A core TRM function is efferocytosis, the clearance of apoptotic, senescent, and damaged cells that is essential for preventing chronic inflammation,” they explained. Especially important targets for this operation, the study showed, are the potentially 100 billion neutrophils produced daily, which start showing signs of senescence within 8 to 12 hours after entering the bloodstream. (Neutrophils that haven’t arrived at senescence yet but have lived long enough and seen enough to put out “kill me now” flags of surrender on their cell surfaces are fair game.)

“Among primary TRM targets are neutrophils, the most abundantly produced immune cell, with more than 10 billion and 100 billion generated daily in mice and humans, respectively,” the investigator noted. “Uncleared aged neutrophils release proteases and extracellular traps that damage tissues, propagate inflammation, and promote aging, and are normally removed efficiently by TRMs in the liver, spleen, and bone marrow.”

But tissue-resident macrophages also grow old. As Andreasson and associates showed in a prior study, over the advancing years these long-lived cells become ever more prone to succumb to aging-associated inflammation and to propagate it. In their newly reported paper, they noted, “TRMs comprise 60–90% of macrophages in the brain, liver, lungs, heart, and kidneys, and their long lifespan makes them particularly vulnerable to aging, as they accumulate metabolic, oxidative, and inflammatory injury over years to decades.”

Immune cells produce hormones called prostaglandins. One of the five varieties of prostaglandin, called PGE2, can exert diverse effects on a cell, depending on which type of surface receptor is expressed on that cell’s surface. Of the various subtypes of receptors for PGE2, the EP2 receptor is highly pro-inflammatory. Tissue-resident macrophages are loaded with EP2.

Infection, injury, and toxic chemicals, including those produced by our aging bodies, increase PGE2 output. As the team’s prior work showed, that output grows substantially as we grow older. So does the concentration of EP2 on tissue-resident macrophages. “TRMs express the prostaglandin E2 (PGE2) receptor EP2, which suppresses macrophage metabolism and phagocytosis in aging,” the investigators noted.

This effectively creates a one-two punch. PGE2’s pro-inflammatory influence increases with age. The resulting unrelenting inflammatory PGE2 stimulation on tissue-resident macrophages, the new study showed, downshifts these cells’ ability to clear neutrophils. Senescent neutrophils then accumulate in tissues and blood.

Andreasson and her colleagues had previously shown that with aging, tissue-resident macrophages undergo a slow decay in their energy metabolism. “Once that starts, there’s a steady decline in a macrophage’s performance,” she said.

For their newly reported study, Andreasson’s lab bioengineered a mouse in which, at a time of the scientists’ choosing, the EP2 gene gets deleted—but only in tissue-resident macrophages. The results of their experiments showed that disappearance of EP2 from these cells reinvigorated the neutrophil-clearance process that PGE2 undermines.

For their experiments, the Stanford Medicine researchers studied younger normal mice, aged 6–8 months, which corresponds to late adolescence or early adulthood in humans, and they also studied older normal mice, at 23 to 25 months of age, whose human counterparts would be in their 60s or 70s. They also looked at older mice whose EP2-encoding gene had been deleted at 4 to 6 months of age (equivalent to their “teenage” years).

The team’s analyses identified 71 proteins, found in blood, whose levels were significantly altered in older normal mice. Of those proteins, 59 stayed at youthful levels in older mice whose tissue-resident macrophages lacked EP2. Many of these proteins originated in the liver. “The liver is one of the body’s most tissue-resident-macrophage-enriched organs and a major contributor to aging-related changes in blood chemistry,” Andreasson said. “It’s the central organ determining the body’s metabolic rate.”

The study showed that in normal old mice, smoldering senescent neutrophils accumulated in the liver, spleen, and bone marrow and, to a lesser extent, in many other organs the researchers looked at.

But the organs of older mice lacking EP2 on their tissue-resident macrophages retained the lower neutrophil numbers of youth. These mice looked younger, leaner, and more physically fit compared with control littermates. They evidenced less visceral fat and greater muscle mass. Their performance on tests of multiple organs’ function equaled that of young mice.

EP2 deletion in addition reduced inflammation in the blood, liver, colon, heart, kidney, and hippocampus (a brain region tightly tied to memory and navigation ability) in the older mice. Their speed, balance, and forelimb grip strength resembled that of young animals.

Reducing EP2 activity in older mice also preserved their memory capabilities. These animals could thread their way through a maze or recall previously encountered objects almost as well as younger mice—and far better than similarly old mice with tissue-resident macrophages expressing functional EP2. “Reducing TRM EP2 signaling in aged mice preserved youthful mitochondrial fitness and prevented cognitive decline, frailty, sarcopenia, adiposity, cardiac impairment, and systemic inflammation,” they wrote in summary.

There are, today, no approved drugs that selectively shut down EP2 activity, although there are several that target PGE2. Non-steroidal anti-inflammatory painkillers work by blocking PGE2 production, Andreasson said. That’s how aspirin and similar drugs reduce pain, fever, swelling, and redness. But to greater or lesser degrees these drugs all block other vital prostaglandins. Even PGE2 has beneficial properties when it binds to receptors other than EP2, rather than the detrimental inflammatory one examined in this study.

As part of their study, the investigators treated otherwise normal 22-month-old mice for two months with an EP2-inhibiting experimental drug. The results showed that the treatment reduced total and senescent neutrophil counts in old mice toward youthful levels. In culture dishes, old age diminished—but the EP2-blocking drug likewise significantly restored—the mice’s tissue-resident macrophages’ ability to engulf and digest burnt-out neutrophils. “Together, these results demonstrate that pharmacologic EP2 inhibition partially reverses age-associated TRM dysfunction and senescent neutrophil accumulation, with strongest rescue in the liver,” they stated.

Finally, the team turned to a large human database characterizing different cell types in young, old, and diseased human livers. This database revealed the same age-related neutrophil buildup, increased neutrophil senescence, tissue-resident-macrophage decline, and heightened EP2 activity in older—and even more so, diseased—livers that the Stanford Medicine researchers had seen in mice. This was a first-time observation in human cells, according to Andreasson. “These human findings, while correlative, position the TRM EP2-efferocytosis axis as a candidate mechanism in human aging that warrants further functional testing,” they noted. “Specifically, future studies should assess whether the impaired clearance of senescent neutrophils also occurs in human TRMs and whether pharmacological EP2 blockade can restore this defect.”

Andreasson suggested that targeting neutrophil clearance may yield big therapeutic benefits. “We need to develop a safe drug that incapacitates EP2 without disrupting upstream events such as PGE2 production.”

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