New Insight with Dr. Veeral Sheth

Diabetes Dialogue

Don’t Miss a Beat

Crisis Point

A trio of stakeholders join Lungcast to discuss the ongoing, long-term assessment into the patterns and drivers of chronic lung disease in a real-world cohort.

Checking in on the Lung Health Cohort Trial

 Seeking “the cholesterol of lung disease”


 Imaging strategies for long-term lung assessment

The American Lung Association (ALA) Lung Health Cohort trial is aiming to provide pulmonary specialists what is available in spades across specialties like cardiovascular medicine: a robust data pool defining the progression of chronic disease in young adults.

The ongoing cohort trial aims to discern patterns in 

 progression as they relate to lifestyle choices and patient demographics; it could potentially highlight new biomarkers for COPD and lung cancer, or refine understanding of the impact of environmental or socioeconomic factors on how asthma worsens over time. As is often the case with ongoing, longterm analyses, the potential for impact on the field is high.

, a trio of expert stakeholders in the Lung Health Cohort join the show to discuss its design, progress and potential. The featured guests include:

Ravi Kalhan, MD, Louis A. Simpson Professor of Pulmonary Medicine at Northwestern University’s Feinberg School of Medicine

George Washko, MD, professor at Harvard Medical School and physician at Brigham and Women’s Hospital

Amy Olson, MD, MSPH, executive director of medical affairs for Boehringer Ingelheim (BI) Pharmaceuticals

Lungcast is a monthly respiratory health podcast series hosted by Albert Rizzo, MD, chief medical officer of the American Lung Association (ALA), and produced by 

Rizzo and the group discuss the parameters of the ongoing, 4000-participant cohort, including its initial observations, hurdles in recruitment and representation, the utility of assessments, and support from industry including BI.

Podcasts

The American Lung Association's Lung Health Cohort trial seeks to establish a comprehensive data pool for understanding chronic respiratory disease progression in young adults, akin to cardiovascular studies. The trial aims to identify patterns related to lifestyle, demographics, and potential biomarkers for COPD and lung cancer, while examining environmental and socioeconomic impacts on asthma. In a recent Lungcast episode, experts discussed the trial's design, progress, and challenges, including recruitment and industry support. The trial's potential impact on respiratory medicine is significant.

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

News

news page, resources on the topics of disease- and specialty-specific medical news and expert insight can be found. Content includes articles, interviews, videos, podcasts, and breaking news on health care research, treatment, and drug development.

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.

COPD

COPD condition center page, resources on the topics of medical news and expert insight into chronic obstructive pulmonary disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on COPD research, treatment, and drug development.

Asthma

The HCPLive asthma page is a comprehensive resource for clinical news and insights on asthmatic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for allergic and severe asthma, and more.

Lung Cancer

Pulmonary Fibrosis

Victoria Johnson joined the MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and now HCPLive. You can reach her at vjohnson@mjhlifesciences.com

Low dose, CD19-targeted chimeric antigen receptor (CAR) T-cells were well-tolerated and showed efficacy in pediatric patients with 

Data from a phase 1/2 study (NCT06222853) evaluating MC-1-50 CAR T cells were presented at 

The American Society of Nephrology (ASN) Kidney Week 2024

 in San Diego, California, held October 23-26, by Xue He, Children’s Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang Province, China.

“Recent advancements indicate that CAR T cell therapy shows promise for treating SLE, there remains a significant lack of data in pediatrics,” He and coinvestigators wrote in their abstract.1

He and colleagues manufactured MC-1-50 CAR T cells with the PRIMCAR manufacturing platform. Most participants received preconditioning with fludarabine (30 mg/m2) and cyclophosphamide (300 mg/m2) daily for 3 days. Investigators reduced dosages in some participants due to infections or intolerance.

As of August 2024, the investigators had infused 11 participants with MC-1-50. With doses ranging from 0.3×10^5/kg cells to 3×10^5/kg cells. Grade 1 cytokine release syndrome (CRS) occurred in 9 participants (81.8%) and grade 1 immune effector cell-associated neurotoxicity syndrome (ICANS) occurred in 2 participants (18.2%). No unexpected adverse events (AE) or serious AEs were reported.

Five participants have follow-up for over 3 months and 4 have achieved Systemic Lupus Erythematosus Responder Index (SRI-4) responses. The fifth participant had persistent proteinuria at 3 months but achieved normalization in all other active indicators, with her serum creatinine levels declining from a peak of 786 to 169μmol/L and her urine output increasing from 50-100ml/day to 1000mL/day.

He and colleagues noted that the other participants have had varying degrees of improvement and CAR T-cell expansion was observed in all dose levels. B cells were rapidly eliminated within 7 days after infusion and reconstituted, mostly as naive B-cells, in most participants by the second month.

The research adds important data on the use of CAR T-cells in pediatric patients with SLE. One of the pioneers in the space of CAR T-cells for autoimmune disease, Kyverna Therapeutics, recently presented data on its investigational KYV-101 CAR T-cell therapy across indications including SLE, lupus nephritis (LN), myasthenia gravis, multiple sclerosis, and IgG4-related disease.

The data were a mixed bag, with the patients with MG and the longest follow-up maintaining her durable immunomodulator-free response while another patient treated with LN experienced disease recurrence at 5 months posttreatment, despite having an initial disease response. KYV-101 continued to have a manageable safety profile, with 25 cases of CRS and 3 cases of ICANS, all below grade 3. The data need to mature in order to look closer into factors of durability in patients’ responses.

"CAR T-cell therapies are rapidly establishing themselves as the new North star in the autoimmune disease treatment universe," Peter Maag, PhD, the chief executive officer of Kyverna, said in a statement.

"We are seeing rising interests in unlocking the full potential of cell therapies in rheumatological and neurological B-cell driven autoimmune diseases."

REFERENCES
1. Liu Q, He X, Zhang Y, Liu F, Wang J, Mao J. Safety and Efficacy of Low-Dose CD19-Targeted CAR T Cells in Refractory Pediatric Systemic Lupus Erythematosus (SLE). Presented at: ASN Kidney Week 2024; San Diego, California; October 23-26. Abstract SA-OR100

2. Maag P. Anti-CD19 CAR T-cell therapy in rheumatologic autoimmune diseases and beyond. Presented at:EULAR European Congress of Rheumatology 2024, held June 12-15, in Vienna, Austria.

Articles

Four of 5 participants with follow-up reaching 3 months have achieved SRI-4 disease responses. 

Nephrology

The HCPLive nephrology page is a resource for medical news and expert insights on kidney disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments in nephrology, and more.

Conference

 conference coverage page features articles, videos, and expert-led live coverage from major medical meetings throughout the year.

Lupus

Clinical

clinical page is a resource for content on every specialty, disease, and topic available on our medical news site.

Low-Dose CAR-T Shows Efficacy in Pediatric Systemic Lupus Erythematosus 

 and joined the MJH Life Sciences team in April 2021. He graduated from The College of New Jersey with a degree in Journalism and Professional Writing. He enjoys listening to records, going to concerts, and playing with his cat Squish. You can reach him at 

Charles C. Wykoff, MD, PhD: Phase 1b/2a Results on Restoret for DME, nAMD

128th Annual American Academy of Ophthalmology (AAO) Meeting

, Charles C. Wykoff, MD, PhD, director of clinical research at Retina Consultants of Texas, presented first-time results on Restoret (EYE103) from the Phase 1b/2a AMARONE trial, targeting 

neovascular age-related macular degeneration (nAMD)

The innovative molecule, developed as a Wnt pathway agonist, aims to stabilize the blood-retinal barrier, offering an alternative to traditional anti-vascular endothelial growth factor (VEGF) therapies, which target vascular permeability. According to Wykoff, Restoret’s mechanism allows for enhanced retinal health through activation rather than inhibition, potentially requiring lower doses for efficacy.

“Across the Phase 1b/2a extension studies associated with AMARONE, we saw Restoret remained safe and well-tolerated across 123 injections,” Wykoff told 

. “Most notably, we saw these structural and functional improvements in a DME population treated without VEGF inhibitors, a very unique approach.” 

Restoret mimics Norin, a protein naturally present in the eye, to activate a Wnt pathway receptor, Frizzled-4, which maintains the structural integrity of retinal blood vessels. In AMARONE, which included patients with DME and nAMD, Restoret demonstrated strong safety outcomes, with no drug-related adverse effects or inflammation across 123 total injections. Patients with DME received Restoret alone, while patients with nAMD received it in combination with aflibercept.

Efficacy results were positive across both patient groups, with patients with DME receiving Restoret monotherapy experiencing an average visual acuity improvement of 11.2 letters and a reduction in central subfield thickness (CST) from approximately 500 to 350 µm by Week 12. In the nAMD cohort, Restoret and aflibercept combined therapy led to a mean visual acuity gain of 6.8 letters and substantial reductions in CST, achieving below 300 µm within one month.

Following these promising findings, Restoret is progressing to the pivotal Brunello trial comparing Restoret monotherapy against anti-VEGF treatments in DME patients with central vision impairment.

“This is a two-year multi-center randomized trial in which Restoret monotherapy is being compared directly to anti-VEGF therapy,” Wykoff told 

. “This trial is actively recruiting now, so I’m looking forward to seeing more data from Restoret in the years to come.”

Disclosures: Relevant disclosures for Wykoff include Annexon, Apellis, Eyebiotech, Genentech, Regeneron, and others.

Do DV. Restoret (EYE103) for the Treatment of Diabetic Macular Edema and Neovascular AMD: First-time Extended Results From the AMARONE Phase 1b/2a Clinical Trial. Presented at the American Academy of Ophthalmology (AAO) 2024 Meeting. Chicago, Illinois. October 18-21, 2024.

Videos

Restoret (EYE103), a Wnt pathway agonist, shows promise in treating diabetic macular edema (DME) and neovascular age-related macular degeneration (nAMD) by stabilizing the blood-retinal barrier. In the AMARONE trial, Restoret demonstrated safety and efficacy, with DME patients experiencing significant visual acuity improvements and reduced central subfield thickness without VEGF inhibitors. nAMD patients also benefited from combined therapy with aflibercept. Restoret is advancing to the Brunello trial, comparing its monotherapy to anti-VEGF treatments in DME patients with central vision impairment.

At AAO 2024, Wykoff discussed first-time extended results from AMARONE showing Restoret’s effectiveness in improving outcomes for patients with DME and nAMD.








Ophthalmology

Ophthalmology page, resources on the topics of medical news and expert insight into ophthalmic disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on eye disease research, treatment, and drug development.

Age-Related Macular Degeneration

age-related macular degeneration condition center page is a comprehensive resource for clinical news and insights on retina disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for patients with AMD.

Diabetic Macular Edema

The HCPLive diabetic macular edema page is a comprehensive resource for clinical news and insights on DME. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for the leading cause of blindness, and more.

Tim Smith joined the MJH Life Sciences team as an assistant editor for HCPLive in August 2022. He graduated from UC Berkeley with a degree in political science, working in multimedia journalism as a staff writer prior to joining MJH. In his spare time, he enjoys reading, watching TV, listening to podcasts, and rock climbing. You can contact him at 

In her Fall Clinical interview, Murina highlighted several key takeaways related to her talk titled ‘Managing Psoriasis with Oral Therapy: 2024 & Beyond.’

Methods to Manage Psoriasis Using Oral Therapies, with Andrea Murina, MD

A presentation, titled ‘Managing Psoriasis with Oral Therapy: 2024 & Beyond,’ was conducted at the 

44th Annual Fall Clinical Dermatology Conference

 in Las Vegas, Nevada. The talk was led by Andrea Murina, MD, associate professor of dermatology and program director at Tulane University School of Medicine.

In this conference interview with Murina, the 

 editorial team asked her about some of the major takeaways of her presentation regarding oral treatments for psoriasis management.

“Oral medicines are really important in the treatment of psoriasis,” Murina and colleagues wrote. “In fact, we have a lot of older systemic medicines that we've used for years for psoriasis, things like methotrexate, acitretin, and cyclosporine. It really wasn't until about 2014 we had a new agent called apremilast. So I was really excited to talk about the legacy of oral psoriasis therapies. But also more excitingly, we have on the horizon, beyond 2024, the TYK2-inhibitors. Those are tyrosine kinase 2 inhibitors.”

Murina noted that TYK2-inhibitors usually are administered once-per-day orally, allowing for convenience for patients. She highlighted the newest one, which is deucravacitinib, as well as a new TYK2 in development.

“What I like to think about deucravacitinib is that we're really affecting some of the drivers of psoriasis,” Murina said. “So when you look at the data, you can see that it has a higher efficacy than apremilast, which is great. It's once a day instead of twice a day, like apremilast…Newer agents that are coming out that block TYK2 seem to have a higher level of selectivity to the TYK2. And it's also important to note that there's differences in TYK2 versus the JAK family.”

Murina highlighted in her presentation that the TYK2 inhibitors are so specific that they do not block JAK1, 2 and 3. She pointed to data to indicate with hematologic parameters and lipid parameters that they are not hitting that JAK1, 2, or 3 target.

For additional information on these treatment options, view the full interview posted above. To find out more about conference presentations, view our 

The quotes used in this interview description were edited for clarity.

The presentation at the 44th Annual Fall Clinical Dermatology Conference focused on advancements in oral therapies for psoriasis, emphasizing the significance of TYK2 inhibitors. Andrea Murina, MD, highlighted the evolution from traditional systemic treatments like methotrexate to newer agents such as apremilast and deucravacitinib. TYK2 inhibitors, including deucravacitinib, offer improved efficacy and convenience with once-daily dosing. These inhibitors are highly selective, targeting TYK2 without affecting JAK1, 2, or 3, thus minimizing potential side effects related to hematologic and lipid parameters.

Plaque Psoriasis

The HCPLive plaque psoriasis page is a resource for medical news and expert insights on psoriasis. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for psoriatic disease, and more.

Dermatology

The HCPLive dermatology page is a resource for medical news and expert insights on skin disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for dermatologic conditions, and more.

Obeticholic acid (Ocaliva) is being infrequently used as a second-line therapy in eligible patients with 

, according to new findings—and even among those that it’s being used, a considerable delay to initiating it may result in progression to poor outcomes including cirrhosis.

American College of Gastroenterology (ACG) 2024 Scientific Sessions

 in Philadelphia, PA, this week, a team of investigators supported by Intercept Pharmaceuticals found that fewer than 8% of patients with PBC eligible for second-line therapy actually initiated obeticholic acid care; the mean time from initiating said care since becoming eligible for it was more than 1 year. The findings highlight a persistent gap in timely, effective care for patients with worsening state from the cholestatic disease.

The research team led by Sonal Kumar, MD, MPH, of Weill Cornell Medical College in New York, sought to estimate the time from second-line therapy eligibility to the initiation of obeticholic acid in patients with PBC. Obeticholic acid is currently the only second-line therapy approved by the US Food and Drug Administration (FDA) to treat PBC, originally indicated for the use under an accelerated approval 2016. However, it remains without a full approval for the indication as of October 2024.

Investigators identified patients with PBC via Komodo Health database. Eligible patients had 1 inpatient or ≥2 outpatient claims for their condition and were eligible for second-line PBC therapy, defined as an inadequate response to ursodeoxycholic acid, between June 2016 and February 2022.

The team used first date of second-line therapy eligibility as index; patients were enrolled for ≥12 months pre-index and were ≥18 years old at index. Patients were followed until the first to occur between obeticholic acid initiation; end of insurance enrollment; longterm care admission; death; or end of study in May 2023.

The final assessment included 3880 adult patients with PBC who were defined as eligible for second-line therapy between 2016 – 2022. Mean patient age was 61.0 years old; 90.8% were female and approximately half (50.5%) were White.

Among the eligible patients, only 306 (7.9%) initiated obeticholic acid second-line therapy. Mean time to treatment from index was 14.2 months, or a median of 10.2 months (IQR, 2.8 – 18.1). Patients who did initiate obeticholic acid therapy were slightly younger (mean age, 57.2 years vs 61.3 years), and were similar proportions of female (90.7%) and White (42.1%).

In the 12 months prior to and including index date, a greater rate of patients with PBC who initiated obeticholic acid had cirrhosis than those who did not initiate (17.6% vs 12.4%). They were also more likely to have an autoimmune disease (29.3% vs 26.3%). The proportion of patients with cirrhosis was nearly doubled at the time of second-line therapy initiation versus the index of when they first became eligible for it (31.4% vs 17.6%).

Kumar and colleagues additionally observed that patients initiating obeticholic acid had higher mean baseline alkaline phosphatase (ALP) levels than those who did not, and that two-thirds (65.3%) of patients had ALP levels >1.67 times the upper limit of normal by the time of initiated second-line therapy.

Investigators concluded that, among eligible adult patients with PBC since 2016, approximately 1 in 12 actually initiated second-line obeticholic acid therapy, “with a considerable delay observed.”

“This delay suggests patients may experience disease progression, including cirrhosis, before starting second-therapy therapy,” they wrote. “Timely initiation of obeticholic acid in appropriate patients could enhance its clinical benefit on long-term outcomes.”

Kumar S, MacEwan JP, Levine A, Bessonova L, et al. Time to Initiation of the Second-Line Therapy Obeticholic Acid in Patients With Primary Biliary Cholangitis. Abstract presented at: American College of Gastroenterology (ACG) 2024 Scientific Sessions. Philadelphia, PA. October 25 – 30, 2024.

Brooks A. FDA Extends Review of Obeticholic Acid (Ocaliva) sNDA for Primary Biliary Cholangitis. 

. Published online October 17, 2024. https://www.hcplive.com/view/fda-extends-review-obeticholic-acid-ocaliva-snda-primary-biliary-cholangitis

Obeticholic acid is underutilized as a second-line therapy for primary biliary cholangitis (PBC), with less than 8% of eligible patients initiating treatment. The delay in starting therapy, averaging over a year, may lead to disease progression, including cirrhosis. The study, presented at the ACG 2024 Scientific Sessions, highlights a significant gap in timely care for PBC patients. Despite being the only FDA-approved second-line therapy since 2016, obeticholic acid remains without full approval. Timely initiation could improve long-term outcomes for patients with PBC.

Data from ACG 2024 show only 1 in 12 patients eligible for second-line PBC therapy are actually doing receiving it—and it may take up to a year to initiate.

Hepatology

Cholangitis

cholangitis page, resources on the topics of medical news and expert insight into cholangitis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on research, treatment, and drug development in PBC, PSC, and more!

Second-Line Obeticholic Acid for PBC Used Infrequently and with Delays

Abigail Brooks is an assistant editor for 

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.

Jacqueline Khalil, DO, reviews findings from 2 studies about the association between hormone replacement therapy with IBS and gastric motility disorders.

Gastroenterology

Gastroenterology condition center page is a comprehensive resource for clinical news and insights on digestive and GI conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for 

, IBS and IBD, Crohn's disease, and more.

Irritable Bowel Syndrome

On the HCPLive irritable bowel syndrome page, resources on the topics of medical news and expert insight into IBS can be found. Content includes articles, interviews, videos, podcasts, and breaking news on IBS, IBD, and more.

Hormone Replacement Therapy Linked to Greater IBS, Gastric Motility Disorder Risk

Video Series

Peer Exchange

HCPLive® Peer Exchange panel discussions feature authoritative insights, opinions, and perspectives on important issues facing today's care professionals.

UPCOMING SERIES: Advancing IgAN Care: Exploring the Next Wave of Treatments

ChatGPT may provide helpful information around gastroparesis, IBS and celiac disease diets, according to findings from a new study.

ChatGPT Reliable for Gastrointestinal Disease-Based Diet Advice

Braden Kuo, MD, explains how GLP-1 RA use increases residual gastric content but does not significantly impact negative endoscopy outcomes.

GLP-1 RAs Increase Residual Gastric Content But Pose Low Medical Risk During Endoscopy

This interview from Fall Clinical covers 2 additional tips for clinical practice as well as unmet needs among patients.

2 Additional Clinical Pearls for Dermatologists, with Eingun James Song, MD

Katherine Tuttle, MD, joins the podcast for a deep dive into the latest FLOW data from Kidney Week 2024. 

Type 2 Diabetes

Chronic Kidney Disease

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...

Kidney Compass: Navigating Clinical Trials

Hosted by Brendon Neuen, MBBS, PhD, and Shikha Wadhwani, MD, MS, Kidney Compass: Navigating Clinical Trials is a regular podcast exploring and breaking down the latest updates and nuances of clinical trials in nephrology. By listening to Kidney Compass, you'll get the guidance you need to navigate the changing landscape of clinical trials across the spectrum of kidney disease. 

Kidney Compass: FLOW Insights from Kidney Week 2024, with Katherine Tuttle, MD

Abdellatif discussed the importance of screening for osteopenia in patients with severe kidney disease. 

Gout

On the HCPLive gout page, resources on the topics of medical news and expert insight into gouty arthritis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on gout, and more.

Abdul Abdellatif, MD: Early Osteopenia Interventions Needed for Patients With Kidney Disease and Gout 

Lacy explains the potential value of an ambulatory wireless patch test for identifying unique gastric activity patterns and individualizing patient care.

Brian Lacy, MD, PhD: Profiling Gastric Activity Variations with Multiday Testing

At AAO 2024, Kay presented an overview of gildeuretinol, an oral vitamin A derivative offering hope for patients with Stargardt disease.

RDR Alert®

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

Christine N. Kay, MD: TEASE Trials Show Promise of Gildeuretinol for Stargardt

Fass reviews findings from a post hoc analysis of a phase 2 trial suggesting the value of vonoprazan as an on-demand NERD treatment.

Gastroesophageal Reflux Disease (GERD)

The HCPLive gastroesophageal reflux disease page is a comprehensive resource for clinical news and insights on GERD. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for reflux disease, and more.

Latest News

Checking in on the Lung Health Cohort Trial

Low-Dose CAR-T Shows Efficacy in Pediatric Systemic Lupus Erythematosus

Charles C. Wykoff, MD, PhD: Phase 1b/2a Results on Restoret for DME, nAMD

Methods to Manage Psoriasis Using Oral Therapies, with Andrea Murina, MD

Second-Line Obeticholic Acid for PBC Used Infrequently and with Delays