Victoria Johnson joined MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and is now an assistant managing editor for HCPLive and RheumatologyLive. She doesn’t have enough time for her numerous hobbies. You can reach her at vjohnson@mjhlifesciences.com.

One year after the FDA approved brensocatib (Brinsupri) as the first disease-modifying therapy for 

, pulmonologists who prescribe it daily describe a field transformed from having no targeted treatment at all to weighing a genuine, if imperfect, first-in-class option.

The August 12, 2025 approval was built on the phase 3 ASPEN trial, the largest study ever conducted in bronchiectasis, enrolling 1721 patients.

 Brensocatib, an oral inhibitor of dipeptidyl peptidase-1 (DPP-1), works by dampening the neutrophil-driven inflammation that fuels the disease's cycle of infection and airway damage. In ASPEN, both the 10-mg and 25-mg doses reduced annualized exacerbation rates by 20% compared with placebo, and the 25-mg dose also slowed lung function decline by 38 mL over 12 months.

"It's been nice to have an option that we didn't have before, even from the perspective of the patients who are always looking for another possibility, another potential treatment to improve their lives," said Mark L. Metersky, MD, professor of medicine, chief of the Division of Pulmonary, Critical Care, and Sleep Medicine, and director of the Center for Bronchiectasis Care at UConn Health. "Many patients were aware of this drug that was being investigated and that it looked promising."

What is the 1-year real world experience?

Metersky said most patients on brensocatib report feeling better, even when they cannot fully explain why. Some describe less coughing or easier sputum clearance; a smaller subset describe the effect as transformative, with cough nearly resolved and quality of life meaningfully improved. 

"For most patients, I won't say it's life-changing, but they feel better, and that's important," he said. "We don't always expect a miracle, so we temper enthusiasm for anything because we generally don't get miracles, but this drug clearly has improved patients' lives."

Ashwin Basavaraj, MD, section chief of pulmonary, critical care, and sleep medicine, and director of the Bronchiectasis and NTM Program at NYC Health + Hospitals/Bellevue, described a similar sense of momentum. 

"We have patients with bronchiectasis that have suffered for years, not knowing what to do and not knowing where to turn in terms of improvement in their symptoms, and now there's an available therapy where we have seen in some patients a dramatic improvement in some of the symptoms," said Basavaraj, who is also associate director of the bronchiectasis program at NYU Langone Health. "Each patient is going to respond differently. But in general, I think it's exciting that there's a new therapy that patients can benefit from, and really exciting that there's a lot of research ongoing right now for additional therapies that has the potential to improve patients with bronchiectasis."

How do new guidelines reflect the change?

The 1-year mark coincided with another milestone: in 2026, the American College of Chest Physicians (CHEST) published its first North American clinical practice guideline for adult bronchiectasis, comprising 13 conditional recommendations.

"Between our new CHEST guidelines and other guidelines that have been released, I think there's been a pretty good job of covering most of the issues related to diagnosis and treatment," Metersky said. "The new aspect covered by the CHEST guidelines is brensocatib, obviously, because it wasn't available when the other guidelines were being produced. But the CHEST guidelines do recommend brensocatib, so that's something novel." Metersky noted the guideline also newly addresses hemoptysis management, recommending tranexamic acid, a long-available drug with emerging evidence of benefit in chronic lung disease, for a complication he said "may not be on the radar screen for many pulmonologists."

Metersky was careful to note that guideline coverage does not equal resolved unmet need. "Despite all of the treatments available, patients still have a large unmet need with chronic cough and sputum, frequent exacerbations, progression of disease," he said, adding that further gaps will only be closed by continued drug development.

Bronchiectasis is a heterogeneous disease, and both physicians pointed to unresolved questions about who benefits most. One is particularly relevant in the US: nontuberculous mycobacteria (NTM), an underlying chronic infection Basavaraj said affects roughly half of US patients with bronchiectasis, per the US Bronchiectasis and NTM Research Registry, a substantially higher proportion than seen in Europe.

"That question does exist: what potential impact do some of these therapies have on some of these chronic infections, such as NTM?" Basavaraj said. "I think there's more data that needs to be evaluated in outcomes of patients with NTM and DPP-1 inhibitors, and it's data that we are currently collecting right now." He added that NTM is not a contraindication to brensocatib use and that his practice has not observed worsening of chronic infection with the therapy, but he emphasized that dedicated outcomes data in this overlapping population remain limited.

What barriers are there to brensocatib access?

Basavaraj oversees bronchiectasis care in 2 distinct New York City patient populations: an academic program at NYU Langone and a public-hospital program at NYC Health + Hospitals/Bellevue. The latter, he said, sees a different demographic profile than most bronchiectasis clinical trial cohorts, with a higher proportion of Asian and African American patients and fewer white patients than typically represented in trial populations.

"I think it's really important to study those patient populations and really try to include them into some of these clinical trials, so we can have a real-world representation in research for all patients with bronchiectasis," Basavaraj said. He reported that his public-hospital cohort has broad access to advanced bronchiectasis therapies, with no major barriers currently limiting uptake of new treatments including brensocatib. "We want to ensure that they get the same standard of care therapy that all patients with bronchiectasis deserve," he said.

What is brensocatib’s real world tolerability?

Brensocatib's adverse event profile includes findings less familiar to pulmonologists than typical respiratory drug effects: periodontal disease and localized skin thickening and sloughing. Metersky said neither has meaningfully affected patient acceptance of the drug in his practice. 

"In general, that's not been an issue that patients fear," he said. "I had one patient who asked if all her teeth were going to fall out if she started it, and of course I reassured her that periodontal disease doesn't work that way. In truth, we didn't really see a significant signal for increased risk of periodontal disease in the phase 3 study."

He advises patients to maintain routine dental hygiene and follow-up, noting that any periodontal changes would likely develop slowly and be caught early. Skin changes, involving thickening and sloughing behind the knees, elbows, feet, or hands, have occurred in only 1 of his patients to date. "Other than that, it's not been an issue," Metersky said.

Brensocatib's development history outside bronchiectasis has been mixed. A phase 2b trial in hidradenitis suppurativa failed to meet its primary and secondary efficacy endpoints in 2026, leading the manufacturer to discontinue that development program, a reminder that the drug's demonstrated benefit to date is specific to non-cystic fibrosis bronchiectasis.

One year in, both physicians describe a field that has gained a genuine first-in-class treatment and, now, a first national guideline to help direct its use, alongside real questions still being worked out about which patients benefit most, how the drug performs alongside common comorbid infections, and how its early real-world safety profile holds up over time.

Basavaraj and Metersky’s disclosures include Insmed and others.

FDA approves first bronchiectasis therapy, brensocatib, for ages 12 and up. HCPLive. 

https://www.hcplive.com/view/fda-approves-first-bronchiectasis-therapy-brensocatib-12-up

. Published August 12, 2025. Accessed August 13, 2026.

Redefining bronchiectasis care with brensocatib FDA approval, with James Chalmers, MBChB, PhD. HCPLive. 

https://www.hcplive.com/view/redefining-bronchiectasis-care-brensocatib-fda-approval-chalmers

. Published August 11, 2026. Accessed August 13, 2026.

CHEST guideline 2026: 13 recommendations for non-CF bronchiectasis. HCPLive. 

https://www.hcplive.com/view/chest-guideline-2026-13-recommendations-bronchiectasis

Brensocatib phase 2b study in HS does not meet endpoints, program discontinued. HCPLive. 

https://www.hcplive.com/view/brensocatib-phase-2b-study-hs-does-not-meet-endpoints-program-discontinued

. Published April 2026. Accessed August 13, 2026.

Articles

Brensocatib, the first disease-modifying therapy for non–cystic fibrosis bronchiectasis, has shifted practice from symptomatic management to targeted anti-neutrophil intervention. In the 1721-patient ASPEN phase 3 trial, 10 mg and 25 mg reduced annualized exacerbations by 20%, and 25 mg slowed FEV1 decline by 38 mL over 12 months. Early real-world use suggests modest-to-marked symptom improvement for many patients with acceptable tolerability. 2026 CHEST guidelines now include conditional recommendations incorporating brensocatib and hemoptysis management, while questions persist around optimal patient selection, NTM comorbidity, and long-term safety.

One year after brensocatib's FDA approval, 2 pulmonologists describe real-world response and a new CHEST guideline.

Pulmonology

Infectious Disease condition center page is a comprehensive resource for clinical news and insights on respiratory health. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for asthma, COPD, interstitial lung disease, COVID-19, and more.

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One Year of Brensocatib: How Bronchiectasis Care Has Changed

Jay Lieberman, MD, from University of Tennessee

Reframing when caregivers reach for epinephrine and expanding stock epinephrine access could meaningfully reduce delayed treatment in infants and toddlers with 

, according to Jay Lieberman, MD, professor at the University of Tennessee Health Science Center and program director of the Allergy/Immunology Fellowship Program at Le Bonheur Children's Hospital.

Underuse spans every age group, but infants and toddlers are treated less often than older children during confirmed reactions.

A recent review by Leeds and colleagues found 48% of caregivers withheld epinephrine because they did not believe symptoms were severe enough.²

"Infants shouldn't be treated any differently," Lieberman told 

 "If we think they're having anaphylaxis, they should get epinephrine."

Stock Epinephrine Could Close Anaphylaxis Care Gap, With Jay Lieberman, MD

Building Caregiver Confidence in Administering Epinephrine

Hands-on exposure appears to move the needle more than instruction alone, Lieberman said. Parents who watch a clinician administer epinephrine during a supervised food challenge report greater comfort using an autoinjector on their own later.

Education emphasizing that standard auto-injector doses are not harmful, paired with a written anaphylaxis action plan, was also associated with higher caregiver willingness to treat. However, Lieberman noted randomized evidence directly testing action plans remains limited.

Why do caregivers delay giving epinephrine?


Many underestimate symptom severities, and a review found 48% of caregivers withheld treatment for this reason.


Undesignated epinephrine kept on-site, at a school for example, that can be administered to any child during a suspected reaction regardless of a known allergy diagnosis.

Lieberman said community caregivers, including daycare staff, school nurses, and camp staff, represent a persistent education gap, with no single delivery method proven superior among clinician-led sessions, video training, and parent-volunteer programs. The intervention Lieberman flagged as highest yield is stock epinephrine, now reflected in allergy practice parameters, which allows a dose to be given to any child regardless of known allergy history.

“It's the same idea as having defibrillators in stadiums that anyone can use if someone has a heart attack,” Lieberman said.

Lieberman added that awareness should keep pace with early allergen introduction guidance, since earlier peanut and other allergenic food exposure may increase reaction frequency in the populations reviewed.

Reported disclosures for Lieberman include AQUESTIVE THERAPEUTICS, Genentech USA, kaleo, Bayer Healthcare, ABBVIE, BioCryst US Sales Co, and Amgen.

Pistiner M, et al. Recognizing and Treating Serious Allergic Reactions (Anaphylaxis) in Infants and Toddlers: A Plain Language Review of Four Studies. 

Leeds S, Anagnostou A, Pistiner M, et al. Epinephrine Underuse for Anaphylaxis in Infants and Toddlers: A Practical Review for Pediatricians. 

Gabryszewski S, Gupta R, and Hernandez-Trujillo V. Peanut Allergy Rates Plummet 10 Years After AAP Early Introduction Guidelines. HCPLive. Published October 30, 2025. Accessed August 13, 2026. 

https://www.hcplive.com/view/peanut-allergy-rates-plummet-10-years-aap-early-introduction-guidelines

Videos

In part 2, Lieberman discusses why caregivers hesitate to use epinephrine and what system-level changes could close the gap in infants and toddlers.

Allergy

Allergy condition center page is a comprehensive resource for clinical news and insights on allergic and inflammatory diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for food and seasonal allergies, as well as allergic asthma and related conditions.

Anaphylaxis

The HCPLive anaphylaxis page is a comprehensive resource for clinical news and insights on acute allergic reactions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for preventing anaphylaxis, and more.

Tiffany Mayo, MD, associate professor at the University of Alabama at Birmingham who will soon join UT Southwestern in Dallas, joined host Mona Shahriari, MD, associate clinical professor of 

 podcast to discuss what she called the pressure of representation that comes after becoming a leader in medicine.

, said she initially envisioned herself as a mentor for nontraditional medical students, but was surprised when established colleagues began reaching out to admire her work. She said the experience felt intimidating because she had not yet reached what she once imagined leadership should look like, before realizing that mentorship is rooted in the ongoing process of growth rather than a fixed destination.

The Medical Sisterhood: The Pressure of Representation, With Tiffany Mayo, MD

How Does Leadership Change the Way Physicians Present Themselves?

Mayo and Shahriari discussed how visibility changes self-presentation, describing different professional versions of themselves that emerge depending on the setting. Mayo said these versions only become unsustainable when they stop feeling authentic, and that staying grounded in core values across situations prevents leadership from feeling like a performance.

Why Do Physician Leaders Struggle to Set Boundaries?

The conversation turned to boundaries, with both physicians acknowledging the discomfort of saying no as expectations grow alongside visibility. Mayo said protecting personal time is an ongoing challenge, particularly when mentees treat their own urgency as universal, and stressed the importance of communicating response-time expectations clearly.

How Does Representation Pressure Differ for Women of Color in Medicine?

As women of color active in the Skin of Color Society (SOCS), Mayo and Shahriari discussed the added pressure of representing broader communities, not just themselves, in professional spaces. Mayo said she has learned to view her presence at decision-making tables as an opportunity to offer a distinct perspective rather than conform, adding that authenticity, including physical self-presentation, becomes a marker of genuine comfort in leadership.

Editor’s note: Neither speaker had any relevant disclosures of note. This episode was summarized with the help of AI tools.

Want to join The Medical Sisterhood community? Click here to join our LinkedIn Group!

Mayo T, et al. Skin of Color Savvy: HS Management in Skin of Color, with Tiffany Mayo, MD, Steven Daveluy, MD, Jasmine Ivanna Espy. HCPLive. May 12, 2025. Accessed August 13, 2026. 

https://www.hcplive.com/view/skin-of-color-savvy-hs-management-skin-of-color-tiffany-mayo-md-steven-daveluy-md-jasmine-ivanna-espy

Podcasts

Tiffany Mayo, MD, discusses the pressure of representation in medical leadership, setting boundaries, and staying authentic as a woman of color.

The Medical Sisterhood, with Mona Shahriari, MD

The Medical Sisterhood, hosted by Mona Shahriari, MD, is a podcast dedicated to empowering female physicians by providing a platform for shared experiences, mentorship, and support. 

Each episode delves into the unique challenges women in medicine face, including work-life balance, leadership roles, and overcoming gender biases. Shahriari aims to inspire women to lead fulfilling professional and personal lives on their own terms through candid discussions and expert interviews. 

e as we foster a community where women can uplift and support each other in achieving their goals!

podcasts page features a feed of medical news and interview podcast series, including DocTalk, Lungcast, Derm Discussions, and Heart Trials.

Dermatology

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Rima Rachid, MD, from Boston Children’s Hospital.

 remains difficult to manage, with no approved therapy offering durable disease modification once treatment stops. Earlier work identified dysbiosis in the gut microbiome of food-allergic infants, along with specific Clostridiales and Bacteroidales bacteria capable of protecting against anaphylaxis in mouse models.¹ These findings drove the design of clinical trials evaluating fecal microbiota transplantation (FMT) as a treatment for peanut allergy.

A phase 1 trial of oral FMT showed durable effects, with RORγt+ regulatory T cell expansion persisting up to 12 months in responders, an effect linked to bacterial bile acid metabolism and Bacteroides ovatus colonization.² Antibiotic pretreatment before FMT also raised the response rate from 30% to 60% in this cohort.²

A second-generation, purified formulation known as microbiota transplantation therapy (MTT) was developed in collaboration with the microbiota therapeutic programs at the University of Minnesota. MTT is > 99% microorganism by content, requires only 5 capsules compared with 36 in the original protocol,² and remains stable at standard refrigerator temperature for approximately 6 months. A phase 2 trial is now randomizing 24 patients to antibiotic pretreatment or placebo before MTT dosing, with reactivity assessed through food challenges at 1 and 4 months.³

Rima Rachid, MD, co-director of the Food Allergy Program at Boston Children's Hospital and associate professor of pediatrics at Harvard Medical School, is leading this research. In the following Q&A, Rachid discusses the new MTT formulation, the design of the ongoing phase 2 trial, and the mechanistic findings shaping future donor selection strategies.

Q&A: Microbiota Transplantation Therapy Simplifies Dosing for Peanut Allergy

: Can you walk through the new peanut allergy study and the MTT formulation developed with the University of Minnesota?

 This is in collaboration with Alex Khoruts from the microbiota therapeutic programs. He is the director of the microbiota therapeutic programs at the University of Minnesota. Alex and his team have developed a second-generation fecal transplantation formulation where the fecal material is highly purified, so you end up with what we call microbiota transplantation therapy, or MTT. It is highly concentrated in microorganisms and contains less than 1% fecal non-microorganism material, so 99% of it is microorganisms coming from a human gut.

In the first study, the proof-of-concept phase 1, we… [used capsules] kept at minus 80 degrees in a research freezer, and when the patient comes, you have to administer it within 90 minutes of thawing.

MTT [is] also encapsulated, double encapsulated, [and] there [are] absolutely no complaints about taste or smell. It can also stay at 4 degrees Celsius, the temperature of a home fridge, for about 6 months [and remain] stable.

MTT cuts dosing from 36 capsules to 5 and is refrigerator-stable for about 6 months, replacing the -80°C storage the original FMT protocol required.

A phase 2 trial is randomizing 24 patients to antibiotic pretreatment or placebo before MTT dosing, with food challenges at 1 and 4 months.

A 1000-fold rise in Bacteroidales abundance was linked to transplant efficacy, a signal that may guide future donor selection.

How does the capsule dose and storage of MTT compare with the original fecal microbiota transplant protocol?

 The dose required is 5 capsules, as opposed to 36 capsules in the first trial. You are able now to [administer] to younger patients because it's a very small dose, relatively speaking.

What is the design of the current phase 2 peanut allergy clinical trial using MTT?

 We're no longer just administering FMT under medical supervision. We're bringing the patient in after they react to a trace of peanut, then randomizing them to receive either antibiotics or placebo. Those who receive antibiotics come in and receive MTT: 5 capsules under medical supervision, followed by another 5 capsules the next day, and then 2 capsules daily for a total of 28 days.

Then, they’ll undergo a food challenge at 1 month and 4 months post-MTT. Those on placebo receive placebo capsules. This study will enroll 24 patients, [with] 1-to-1 randomization, so 12 will get MTT and 12 will get placebo. We are very close to closing enrollment.

What are the next steps for larger MTT trials and the powder formulation for younger patients?

: What we're hoping for is to do larger studies. We will need larger studies to evaluate the donor-patient effect and see if particular patients might respond better to particular donors. We are aiming for 50, hopefully 100, patients… [who will receive] MTT from different donors… and hope to administer it for a period of at least 6 months to see the effect.

Another good news about MTT is that there is now a powder formulation that is in progress, and that powder formulation could potentially be evaluated in patients who cannot swallow capsules, so we’re talking about younger patients. There is a lot of evidence in food allergy that the sooner you intervene, the better, so we're hoping to take it down to ages 4 to 11 years and see the effect of this powder formulation.

What role do Bacteroidales and Clostridiales gut bacteria play in protecting against food allergy and anaphylaxis?

 What I think is very interesting is the Bacteroidales part. We saw the same type of bacteria that protects from anaphylaxis in a study where, initially, in the Nature Medicine paper we published back in 2019, we enrolled 156 babies. There were 54 who were food allergic, and the rest were controls.

We continued following them every few months up until 13 months of age, and when we analyzed the microbiome, we found there was continuous dysbiosis, an abnormal microbiome, in patients who were food allergic. But there [were] some carefully selected bacteria, Clostridiales species, and separately a few Bacteroidales species. This was in collaboration with our colleagues at Brigham, Lynn Bry and Georg Gerber.

We took those bacteria [and] put them into a highly allergic mouse model, and there was complete protection from anaphylaxis. That experiment was repeated multiple times. There was an increase in IL-10-positive T-regulatory-like cells in the mice, [a] decrease in atopic cells, and a decrease in ovalbumin IgE, the egg protein we challenged the mice with.

For the first time, we found bacterial species that protect from anaphylaxis, because a lot of focus had been on the Clostridiale species. In this particular study, [we found] that in the mice that responded after getting fecal material from patients who showed efficacy after transplantation, there was a 1000-fold increase in that bacterial species in the mice. That's a signal we would like to evaluate further.

We're hoping to evaluate Bacteroidales species alone as a consortium, but we might also be able to select some donors [based on that] We're not sure yet. We'd like to think more about it, but I think it's important to [consider] now there is a role for these bacteria in food allergy.

Fecal Transplant Sustains Peanut Allergy Protection to 12 Months

Antibiotic Pretreatment Improves FMT Response in Peanut Allergy

Abdel-Gadir A, Stephen-Victor E, Gerber GK, et al. Microbiota therapy acts via a regulatory T cell MyD88/RORγt pathway to suppress food allergy. 

Rachid R, et al. Fecal microbiome transplant in food allergy in humans and mice identifies a role for bile acid metabolites in oral tolerance. 

ClinicalTrials.gov. Evaluating the Safety and Efficacy of Oral Encapsulated Microbiota Transplantation Therapy in Peanut Allergic Patients. NCT05695261. Accessed August 12, 2026. 

https://clinicaltrials.gov/study/NCT05695261

Peanut allergy lacks therapies that sustain disease modification after discontinuation, prompting evaluation of microbiome-based approaches. Prior infant microbiome studies identified persistent dysbiosis and protective Clostridiales and Bacteroidales taxa that prevented anaphylaxis in murine models via increased IL-10–associated regulatory responses and reduced atopic parameters. Oral FMT in a phase 1 trial produced durable immunologic changes, including prolonged RORγt+ Treg expansion linked to bile acid metabolism and Bacteroides ovatus colonization, with antibiotic pretreatment improving response rates. A purified, refrigerator-stable, low-capsule MTT formulation is advancing into a randomized phase 2 trial.

Rima Rachid, MD, discusses the purified MTT formulation, phase 2 trial design, and the gut bacteria driving protection against peanut allergy.

Food Allergy

Germline genetic variants may help explain why CAR T cell therapy produces such variable toxicity across patients with aggressive lymphoma, according to findings published in Science Immunology.1 Investigators identified a specific gene, STXBP2, whose variants were enriched among patients who developed severe toxicity after treatment with axicabtagene ciloleucel.

"CAR T cells are different than every other form of therapy that came before," said Mark B. Leick, MD, medical oncologist and physician-scientist at Massachusetts General Hospital and the study's corresponding author. Unlike a standardized drug, Leick noted, CAR T cells are manufactured from a patient's own T cells, carrying all of that patient's inherited genetic variation into the final product.

A Genetic Signature May Predict Who Gets Sick From CAR T Cell Therapy

A Rare Disease Pointed Investigators Toward STXBP2

Investigators performed whole genome sequencing on patients with aggressive lymphoma treated with axicabtagene ciloleucel in the ZUMA-1 (n = 86 evaluable) and ZUMA-7 (n = 150 evaluable) clinical trials, in partnership with Kite Pharma.1 The hypothesis was rooted in hemophagocytic lymphohistiocytosis (HLH), a rare inherited hyperinflammatory syndrome that shares key biochemical features with cytokine release syndrome (CRS), the most common toxicity of CAR T cell therapy.1 Leick and colleagues reasoned that milder, adult-onset forms of the same genetic defects that cause HLH might also be driving toxicity in some CAR T cell recipients.

Among the ZUMA-1 toxicity cohort (n = 39), 6 patients (15.4%; 95% CI, 5.9%-30.1%) carried putative deleterious variants in STXBP2, a gene involved in lymphocyte degranulation, compared with none in the 39-patient control cohort (OR, >1.44; nominal P = .025).1 The finding was not replicated in ZUMA-7, which investigators attributed to that cohort's lower baseline inflammation and fewer prior treatments relative to the more heavily pretreated ZUMA-1 population.

CRISPR Experiments Confirmed a Mechanistic Role

To confirm a mechanistic role for the gene, investigators used CRISPR-Cas9 to knock out STXBP2 in CAR T cells manufactured from healthy donors.

The STXBP2-deficient cells showed impaired degranulation and cytotoxicity, along with increased inflammatory cytokine production when cocultured with tumor targets, mirroring the cytokine profile seen in variant carriers in the clinical cohort.

Investigators noted that the ZUMA-1 and ZUMA-7 cohorts were predominantly of European ancestry, limiting conclusions in non-European populations, and that the number of STXBP2 variant carriers identified was small, warranting confirmation in larger, more diverse cohorts.

Editor’s Note: Leick reports relevant disclosures with BioNTech, Cabaletta Bio, and Adaptimmune.

Leick MB, Sun B, Birocchi F, et al. Genomic correlates of clinical CAR T cell activity. Sci Immunol. 2026;11(121). doi:10.1126/sciimmunol.aef4134

Neelapu SS, Locke FL, Bartlett NL, et al. Axicabtagene ciloleucel CAR T-cell therapy in refractory large B-cell lymphoma. N Engl J Med. 2017;377(26):2531-2544.

Germline STXBP2 variants may predict severe CAR T cell toxicity, Mark B. Leick, MD, and colleagues find

Hematology

Tim Smith joined the MJH Life Sciences team in 2022 and is currently an associate editor for 

. He is also the executive producer of two podcasts: 

. Previously, he was the producer of the Lungcast podcast. He graduated from UC Berkeley and worked in multimedia journalism as a staff writer prior to joining MJH. You can contact him at tsmith@mjhlifesciences.com.

In the UP-AA trials, both upadacitinib doses outperformed placebo on SALT score ≤20 at week 24 in adults and adolescents with severe alopecia areata.

Alopecia

UP-AA: Upadacitinib Attains Primary Endpoint in New Alopecia Areata Data

Diana Isaacs, PharmD, is an endocrine clinical pharmacist, director of Education and Training in Diabetes Technology, and codirector of Endocrine Disorders in Pregnancy at the Cleveland Clinic.

In this segment from the latest episode of Diabetes Dialogue, Diana Isaacs, PharmD, and Natalie Bellini, DNP, discuss switching patients from basal insulin to Awiqli.

Endocrinology

The HCPLive endocrinology page is a comprehensive resource for clinical news and insights on endocrine system conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for diabetes, hormonal disease, and more.

This page also hosts content previously published by EndocrinologyNetwork. 


Type 1 Diabetes

Converting Basal Insulin Patients to Insulin Icodec

Saakshi Khattri, MD, is a board certified Dermatologist, Rheumatologist and Internist and one of only a handful of triple board-certified physicians across the US.

Saakshi Khattri, MD, outlines her key practice-changing takeaways for dermatologists and rheumatologists.

Video Series

Practice-Changing Perspectives on IL-23 Inhibition and Early Intervention in Psoriatic Disease

New research links germline genetic variants to CAR T cell toxicity risk and expansion in lymphoma patients treated with axi-cel.

Germline Variants May Predict CAR T Cell Toxicity, With Mark Leick, MD

Robert Sarkany, MD, FRCP, is a consultant dermatologist at St John's Institute of Dermatology at Guy's and St Thomas' Hospital in London and at the Lister Hospital.

Recent phase 2a findings on PORT-77 show rapid, dose-dependent plasma PPIX reductions in erythropoietic protoporphyria and X-linked protoporphyria.

Q&A: PORT-77 Phase 2a Results in Adults with EPP and XLP

Mark Metersky, MD, and Ashwin Basavaraj, MD, discuss novel therapy brensocatib on the 1-year anniversary of its FDA approval.

One Year Later: How Brensocatib Has Changed Bronchiectasis Care

Rima Rachid, MD, explains how bile acid metabolism and Bacteroides colonization drive durable Treg cell induction after FMT for peanut allergy.

Abigail Brooks is a strategic content lead overseeing 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

Experts discussed FCS diagnostic scoring tools, diagnostic delays, and new data on olezarsen and plozasiran for lowering triglycerides in a recent clinical forum.

Cardiology

Cardiology condition center page is a comprehensive resource for clinical news and insights on cardiovascular and cardiometabolic diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for heart disease and cardiovascular events, as well as associated diabetes, renal failure, and more...

Dyslipidemia

FCS Diagnosis and Management Explained: Clinical Forum Insights

In this segment from the latest episode of Diabetes Dialogue, Diana Isaacs, PharmD, and Natalie Bellini, DNP, discuss the commercial release of insulin icodec.

Type 2 Diabetes

Insulin Icodec Dosing Strategies for Type 2 Diabetes

In this segment from the latest episode of Diabetes Dialogue, Diana Isaacs, PharmD, and Natalie Bellini, DNP, discuss the promise of continuous ketone monitoring in T1D.

Latest News

One Year of Brensocatib: How Bronchiectasis Care Has Changed

Stock Epinephrine Could Close Anaphylaxis Care Gap, With Jay Lieberman, MD

The Medical Sisterhood: The Pressure of Representation, With Tiffany Mayo, MD

Q&A: Microbiota Transplantation Therapy Simplifies Dosing for Peanut Allergy

A Genetic Signature May Predict Who Gets Sick From CAR T Cell Therapy