The deadly condition known as pulmonary arterial hypertension (PAH), which afflicts up to 150,000 Americans each year, may be reversible by using an inhalable gene therapy
The deadly condition known as pulmonary arterial hypertension (PAH), which afflicts up to 150,000 Americans each year, may be reversible by using an inhalable gene therapy, report an international team of researchers led by investigators at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.
In their new study, reported in the July 30 issue of the journal Circulation, scientists demonstrated that gene therapy administered through a nebulizer-like inhalation device can completely reverse PAH in rat models of the disease. In the lab, researchers also showed in pulmonary artery PAH patient tissue samples reduced expression of the SERCA2a, an enzyme critical for proper pumping of calcium in calcium compartments within the cells. SERCA2a gene therapy could be sought as a promising therapeutic intervention in PAH.
“The gene therapy could be delivered very easily to patients through simple inhalation — just like the way nebulizers work to treat asthma,” says study co-senior investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center and the Arthur & Janet C. Ross Professor of Medicine and Professor of Gene & Cell at Icahn School of Medicine at Mount Sinai. “We are excited about testing this therapy in PAH patients who are in critical need of intervention.”
This same SERCA2a dysfunction also occurs in heart failure. This new study utilizes the same gene therapy currently being tested in patients to reverse congestive heart failure in a large phase III clinical trial in the United States and Europe.
“What we have shown is that gene therapy restores function of this crucial enzyme in diseased lungs,” says Dr. Hajjar. “We are delighted with these new findings because it suggests that a gene therapy that is already showing great benefit in congestive heart failure patients may be able to help PAH patients who currently have no good treatment options — and are in critical need of a life sustaining therapy.”
When SERCA2a is down-regulated, calcium stays longer in the cells than it should, and it induces pathways that lead to overgrowth of new and enlarged cells. According to researchers, the delivery of the SERCA2a gene produces SERCA2a enzymes, which helps both heart and lung cells restore their proper use of calcium.
“We are now on a path toward PAH patient clinical trials in the near future,” says Dr. Hajjar, who developed the gene therapy approach. Studies in large animal models are now underway. SERCA2a gene therapy has already been approved by the National Institutes of Health for human study.
A Simple Inhalation Corrects Deadly Dysfunction
PAH most commonly results from heart failure in the left side of the heart or from a pulmonary embolism, when clots in the legs travel to the lungs and cause blockages. When the lung is damaged from these conditions, the tissue starts to quickly produce new and enlarged cells, which narrows pulmonary arteries. This increases the pressure inside them. The high pressure in these arteries resists the heart’s effort to pump through them and the blood flow between the heart and lungs is reduced. The right side of the heart then must overcome the resistance and work harder to push the blood through the pulmonary arteries into the lungs. Over time, the right ventricle becomes thickened and enlarged and heart failure develops.
The gene therapy that Dr. Hajjar developed uses a modified adeno-associated viral-vector that is derived from a parvovirus. It works by introducing a healthy SERCA2a gene into cells, but this gene does not incorporate into a patient’s chromosome, according to the study’s lead author, Lahouaria Hadri, PhD, an Instructor of Medicine in Cardiology at Icahn School of Medicine at Mount Sinai.
“The clinical trials in congestive heart failure have shown already that the gene therapy is very safe,” says Dr. Hadri. Between 40-50 percent of individuals have antecedent antibodies to the adeno-associated vectors, so potential patients need to be screened before gene therapy to make sure they are eligible to receive the vectors. In patients without antibodies, the restorative enzyme gene therapy does not cause an immune response, according to Dr. Hadri.
The Latest Bing News on:
Inhalable Gene Therapy
- Electric pulses boost gene therapy uptake in liver cellson April 30, 2024 at 10:37 pm
In an effort to improve delivery of costly medical treatments, a team of researchers in electrical engineering at the University of Wisconsin–Madison has developed a stimulating method that could make ...
- Gene Therapy Is Halting Cancer. Can It Work Against Brain Tumors?on April 29, 2024 at 2:32 pm
Grant of up to $11 million will fund a clinical trial at UCSF that uses a smarter new CAR-T guided by precision technology.
- What Lies Ahead? Shaping the Future of Cell and Gene Therapyon April 29, 2024 at 1:24 pm
Sponsored content brought to you by The launch of Revvity, Inc. in May 2023 signaled the rebranding of an 85-year-old company with a dedicated focus on the diagnostics and life sciences sector.
- Coave Therapeutics showcases its Aligater platform for generating conjugated AAV vectors to deliver ocular gene therapy through suprachoroidal routeon April 29, 2024 at 6:44 am
The data will be presented in oral session at the 2024 American Society of Gene & Cell Therapy meeting May 7-11 in Baltimore, Maryland.
- Walgreens’ Next Frontier: Complex Conditions & Cell And Gene Therapyon April 29, 2024 at 4:31 am
Walgreens, one of the largest retail pharmacy companies in the world, announced last week that it plans to launch a variety of new services as a part of its newly integrated specialty pharmacy ...
- Cell and gene therapy companies trip at scalability hurdleon April 29, 2024 at 3:09 am
Experts hold scalability challenges and high costs accountable for market failures within the cell and gene therapy landscape.
- FDA Approves Pfizer’s One-Time Gene Therapy BEQVEZ for Hemophilia Bon April 28, 2024 at 2:23 pm
The BEQVEZ gene therapy inserts a working copy of the Factor IX (FIX) gene that codes for a high-activity FIX variant.
- US approves gene therapy treatment for hemophiliaon April 27, 2024 at 5:16 am
Pharmaceutical giant Pfizer has received US approval for a gene therapy against a form of hemophilia, a rare and inherited blood clotting disorder, the company said Friday.
- US FDA approves Pfizer's gene therapy for rare bleeding disorderon April 26, 2024 at 11:57 am
The U.S. Food and Drug Administration approved Pfizer's gene therapy for hemophilia B on Friday, the second such therapy for the rare bleeding disorder that typically requires regular infusions of a ...
- FDA Approves Second Gene Therapy for Hemophilia Bon April 26, 2024 at 7:38 am
Pfizer reported a list price of $3.5 million for its new gene therapy, Beqvez, for adults with this rare bleeding disorder.
The Latest Google Headlines on:
Inhalable Gene Therapy
[google_news title=”” keyword=”Inhalable Gene Therapy” num_posts=”10″ blurb_length=”0″ show_thumb=”left”]
The Latest Bing News on:
Pulmonary arterial hypertension
- Young Children in Medicaid have More Diabetes Complicationson May 1, 2024 at 5:02 am
The rate of ketoacidosis among children younger than 5 years of age with type 1 diabetes is higher in Medicaid than commercial plans, finds new analysis.
- United Therapeutics Surpasses Analyst Revenue Forecasts with Strong Q1 2024 Performanceon May 1, 2024 at 4:51 am
In a strategic move to bolster shareholder value, United Therapeutics implemented a $1 billion accelerated share repurchase program. This initiative reflects the company's confidence in its long-term ...
- Anger's role in heart attack risk may start in the arterieson April 30, 2024 at 5:00 pm
Short bursts of anger may temporarily damage the ability of blood vessels to properly dilate, a function believed to be pivotal in preventing arteries from hardening, new research suggests. The ...
- Global Eisenmenger Complex Management Industryon April 30, 2024 at 5:23 am
Global Eisenmenger Complex Management Industry is expected to reach US$ 12.9 Billion at a CAGR of 5.3% by 2033 | FMI Reveals ...
- Saudi Arabia Pulmonary Arterial Hypertension Market Size, Share, Growth Statistics, Leading Players and Forecast to 2024 to 2032on April 29, 2024 at 4:11 pm
Anticipated growth for ‘Saudi Arabia Pulmonary Arterial Hypertension Market’ is significant from 2024 to 2032. The market showed steady growth in 2024 and is expected to continue rising due to ...
- Merck highlights progress on HIV, hypertension, cervical cancer drugson April 25, 2024 at 10:15 am
- This May 1, 2018, shows Merck corporate headquarters in Kenilworth, N.J. RAHWAY, N.J. – Merck got off to an excellent start in the first quarter of 2024. Compared to the first quarter of 2023, ...
- Merck beats earnings forecasts as cancer drug sales boomon April 25, 2024 at 9:40 am
Merck’s stocks saw a bump on Thursday after the pharmaceutical giant reported a boom in first-quarter sales of its blockbuster cancer drug Keytruda, according to the firm’s latest earnings report.
- Merck to Report Earnings One Month After Major Drug Approval. What Investors Are Watching.on April 25, 2024 at 6:15 am
Merck is set to announce its first quarter financial results on Thursday, a month after the pharmaceutical company secured the approval of Winrevair, a new medicine that analysts expect to be a ...
- Merck’s first-quarter results beat expectations amid healthy vaccine-sales growthon April 25, 2024 at 3:31 am
Merck on Thursday reported first-quarter results that beat analysts’ expectations amid strong sales of vaccines and cancer drug Keytruda.
- Vietnam Pulmonary Arterial Hypertension Market Trendsetters of Tomorrow Market’s Dazzling Latest Trendson April 24, 2024 at 2:20 pm
Report Ocean’s latest analysis delves into the “Vietnam Pulmonary Arterial Hypertension Market” Report from 2024 to 2032, covering an array of market facets such as characteristics, size, growth, ...
The Latest Google Headlines on:
Pulmonary arterial hypertension
[google_news title=”” keyword=”pulmonary arterial hypertension” num_posts=”10″ blurb_length=”0″ show_thumb=”left”]