• Education
    • Higher Education
    • Scholarships & Grants
    • Online Learning
    • School Reforms
    • Research & Innovation
  • Lifestyle
    • Travel
    • Food & Drink
    • Fashion & Beauty
    • Home & Living
    • Relationships & Family
  • Technology & Startups
    • Software & Apps
    • Startup Success Stories
    • Startups & Innovations
    • Tech Regulations
    • Venture Capital
    • Artificial Intelligence
    • Cybersecurity
    • Emerging Technologies
    • Gadgets & Devices
    • Industry Analysis
  • About us
  • Contact
  • Advertise with Us
  • Privacy & Policy
Today Headline
  • Home
  • World News
    • Us & Canada
    • Europe
    • Asia
    • Africa
    • Middle East
  • Politics
    • Elections
    • Political Parties
    • Government Policies
    • International Relations
    • Legislative News
  • Business & Finance
    • Market Trends
    • Stock Market
    • Entrepreneurship
    • Corporate News
    • Economic Policies
  • Science & Environment
    • Space Exploration
    • Climate Change
    • Wildlife & Conservation
    • Environmental Policies
    • Medical Research
  • Health
    • Public Health
    • Mental Health
    • Medical Breakthroughs
    • Fitness & Nutrition
    • Pandemic Updates
  • Sports
    • Football
    • Basketball
    • Tennis
    • Olympics
    • Motorsport
  • Entertainment
    • Movies
    • Music
    • TV & Streaming
    • Celebrity News
    • Awards & Festivals
  • Crime & Justice
    • Court Cases
    • Cybercrime
    • Policing
    • Criminal Investigations
    • Legal Reforms
No Result
View All Result
  • Home
  • World News
    • Us & Canada
    • Europe
    • Asia
    • Africa
    • Middle East
  • Politics
    • Elections
    • Political Parties
    • Government Policies
    • International Relations
    • Legislative News
  • Business & Finance
    • Market Trends
    • Stock Market
    • Entrepreneurship
    • Corporate News
    • Economic Policies
  • Science & Environment
    • Space Exploration
    • Climate Change
    • Wildlife & Conservation
    • Environmental Policies
    • Medical Research
  • Health
    • Public Health
    • Mental Health
    • Medical Breakthroughs
    • Fitness & Nutrition
    • Pandemic Updates
  • Sports
    • Football
    • Basketball
    • Tennis
    • Olympics
    • Motorsport
  • Entertainment
    • Movies
    • Music
    • TV & Streaming
    • Celebrity News
    • Awards & Festivals
  • Crime & Justice
    • Court Cases
    • Cybercrime
    • Policing
    • Criminal Investigations
    • Legal Reforms
No Result
View All Result
Today Headline
No Result
View All Result
Home Science & Environment Medical Research

Two decades of research lead to treatment for rare, painful skin condition

May 3, 2025
in Medical Research
Reading Time: 4 mins read
A A
0
wound
5
SHARES
10
VIEWS
Share on FacebookShare on Twitter


wound
Credit: Unsplash/CC0 Public Domain

Patients with severe dystrophic epidermolysis bullosa, or EB, have skin so fragile, the slightest touch can lead to blistering and, eventually, large, open wounds that never heal, causing immense pain.

A treatment developed at Stanford Medicine, skin grafts, can treat those large, open wounds. Genetically engineered from a patient’s own cells, the grafts were granted approval as an EB therapy on April 29 by the U.S. Food and Drug Administration.

“I’m super excited,” Jean Tang, MD, Ph.D., professor of dermatology said, noting that the grafts are the product of more than 20 years of Stanford Medicine research. Tang treats children with EB at Lucile Packard Children’s Hospital Stanford. “Who would have thought that an experiment in a Stanford lab would lead to a personalized therapy for EB patients?”

We spoke with Tang about the disease, the research that led to the treatment and hope for patients with the condition.

What is dystrophic epidermolysis bullosa?

Severe dystrophic epidermolysis bullosa is a rare genetic condition affecting 1 in every 500,000 people. Those with the disease have a defect in the gene for collagen VII, a protein that normally holds the skin together.

Collagen VII is like a staple that attaches the top layer to the bottom layer of your skin. Without this molecular “staple,” the layers of patients’ skin separate in response to slight friction, even a light touch. This causes wounds that can persist for years, along with extreme pain and itching.

These kids are wrapped in wound dressings almost from head to toe, just to protect their delicate skin. They’re known as butterfly-skin children because their skin is as fragile as butterfly wings.

The wounds are prone to infection, and even bathing is painful. Because of the unhealed wounds and inflammation, EB patients are at high risk for skin cancer.

Other parts of the body are also affected, as collagen VII helps hold layers of the digestive tract and eye together, but the skin problems are the most difficult aspect of the disease to live with.

Describe the research that has led to the skin grafts receiving approval by the FDA.

In 2003, Paul Khavari, MD, Ph.D., the Carl J. Herzog Professorship in Dermatology in the School of Medicine, and his team developed a safe and effective way to genetically engineer EB skin cells with a corrected gene. The team showed that the resulting skin could be grown into small sheets that had functional collagen VII and could be safely grafted to mice.

This work led, over the next two decades, to Stanford Medicine studies that developed gene therapy skin grafts for people, including a phase 1 clinical trial led by Alfred Lane, MD, now emeritus professor of dermatology, and Peter Marinkovich, MD, associate professor of dermatology, which showed early signs of safety and effectiveness of the gene-therapy grafts and was published in 2016.

Our team also played a leading role in the last phase of clinical trials, with results that we are looking forward to sharing when they are published.

The treatment was then licensed from Stanford University by Abeona Therapeutics Inc., which will manufacture grafts for patients. The grafts will be available at five hospitals across the country, including Packard Children’s.

How do the grafts work?

To make the skin sheets, which are grown individually for each patient, a physician collects a small biopsy of the patient’s un-wounded skin. The biopsy is taken to a lab, where a retrovirus is used to introduce a corrected version of the collagen VII gene, COL7AI, to the skin cells. The genetically engineered cells are grown into sheets of skin that are about the size of a credit card. The process of preparing the grafts takes about 25 days, after which patients go to the hospital and our plastic surgeons suture the genetically engineered skin onto a wound.

Patients stay in the hospital for about a week of recovery. Because each graft is created from the patient’s own skin, the treatment provides healthy skin that matches the patients’ own immune markers, preventing immune rejection of the grafts.

What are people saying about the grafts?

Participants in our clinical trials have told me just how much of their life and attention span had been focused on these painful wounds. To not have that is very freeing. Their wounds stay healed, and they don’t have to wear as many bandages. They can attend school and do other everyday activities more easily.

If these patients are diagnosed as infants and start another gene therapy product, a gene therapy gel that was approved in 2023, maybe they won’t develop big wounds. But if the gels don’t work and a wound does expand, the skin graft therapy is the right treatment. The arc of their disease will, I hope, be modified, with less suffering.

Provided by
Stanford University


Citation:
Q&A: Two decades of research lead to treatment for rare, painful skin condition (2025, May 3)
retrieved 3 May 2025
from https://medicalxpress.com/news/2025-05-qa-decades-treatment-rare-painful.html

This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no
part may be reproduced without the written permission. The content is provided for information purposes only.



wound
Credit: Unsplash/CC0 Public Domain

Patients with severe dystrophic epidermolysis bullosa, or EB, have skin so fragile, the slightest touch can lead to blistering and, eventually, large, open wounds that never heal, causing immense pain.

A treatment developed at Stanford Medicine, skin grafts, can treat those large, open wounds. Genetically engineered from a patient’s own cells, the grafts were granted approval as an EB therapy on April 29 by the U.S. Food and Drug Administration.

“I’m super excited,” Jean Tang, MD, Ph.D., professor of dermatology said, noting that the grafts are the product of more than 20 years of Stanford Medicine research. Tang treats children with EB at Lucile Packard Children’s Hospital Stanford. “Who would have thought that an experiment in a Stanford lab would lead to a personalized therapy for EB patients?”

We spoke with Tang about the disease, the research that led to the treatment and hope for patients with the condition.

What is dystrophic epidermolysis bullosa?

Severe dystrophic epidermolysis bullosa is a rare genetic condition affecting 1 in every 500,000 people. Those with the disease have a defect in the gene for collagen VII, a protein that normally holds the skin together.

Collagen VII is like a staple that attaches the top layer to the bottom layer of your skin. Without this molecular “staple,” the layers of patients’ skin separate in response to slight friction, even a light touch. This causes wounds that can persist for years, along with extreme pain and itching.

These kids are wrapped in wound dressings almost from head to toe, just to protect their delicate skin. They’re known as butterfly-skin children because their skin is as fragile as butterfly wings.

The wounds are prone to infection, and even bathing is painful. Because of the unhealed wounds and inflammation, EB patients are at high risk for skin cancer.

Other parts of the body are also affected, as collagen VII helps hold layers of the digestive tract and eye together, but the skin problems are the most difficult aspect of the disease to live with.

Describe the research that has led to the skin grafts receiving approval by the FDA.

In 2003, Paul Khavari, MD, Ph.D., the Carl J. Herzog Professorship in Dermatology in the School of Medicine, and his team developed a safe and effective way to genetically engineer EB skin cells with a corrected gene. The team showed that the resulting skin could be grown into small sheets that had functional collagen VII and could be safely grafted to mice.

This work led, over the next two decades, to Stanford Medicine studies that developed gene therapy skin grafts for people, including a phase 1 clinical trial led by Alfred Lane, MD, now emeritus professor of dermatology, and Peter Marinkovich, MD, associate professor of dermatology, which showed early signs of safety and effectiveness of the gene-therapy grafts and was published in 2016.

Our team also played a leading role in the last phase of clinical trials, with results that we are looking forward to sharing when they are published.

The treatment was then licensed from Stanford University by Abeona Therapeutics Inc., which will manufacture grafts for patients. The grafts will be available at five hospitals across the country, including Packard Children’s.

How do the grafts work?

To make the skin sheets, which are grown individually for each patient, a physician collects a small biopsy of the patient’s un-wounded skin. The biopsy is taken to a lab, where a retrovirus is used to introduce a corrected version of the collagen VII gene, COL7AI, to the skin cells. The genetically engineered cells are grown into sheets of skin that are about the size of a credit card. The process of preparing the grafts takes about 25 days, after which patients go to the hospital and our plastic surgeons suture the genetically engineered skin onto a wound.

Patients stay in the hospital for about a week of recovery. Because each graft is created from the patient’s own skin, the treatment provides healthy skin that matches the patients’ own immune markers, preventing immune rejection of the grafts.

What are people saying about the grafts?

Participants in our clinical trials have told me just how much of their life and attention span had been focused on these painful wounds. To not have that is very freeing. Their wounds stay healed, and they don’t have to wear as many bandages. They can attend school and do other everyday activities more easily.

If these patients are diagnosed as infants and start another gene therapy product, a gene therapy gel that was approved in 2023, maybe they won’t develop big wounds. But if the gels don’t work and a wound does expand, the skin graft therapy is the right treatment. The arc of their disease will, I hope, be modified, with less suffering.

Provided by
Stanford University


Citation:
Q&A: Two decades of research lead to treatment for rare, painful skin condition (2025, May 3)
retrieved 3 May 2025
from https://medicalxpress.com/news/2025-05-qa-decades-treatment-rare-painful.html

This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no
part may be reproduced without the written permission. The content is provided for information purposes only.


Tags: Health ResearchHealth Research NewsHealth ScienceMedicine ResearchMedicine Research NewsMedicine Science
Previous Post

Which horse is the favorite to win the 2025 Kentucky Derby?

Next Post

SpaceX to launch 29 Starlink satellites on Falcon 9 rocket from NASA’s Kennedy Space Center – Spaceflight Now

Related Posts

cold sore

Could cold sores increase the risk of Alzheimer’s disease? A new study is no cause for panic

May 23, 2025
4
Infographic: The Rising Prevalence of Autism | Statista

‘Has to Be Something on the Outside’

May 22, 2025
4
Next Post
SpaceX to launch 23 Starlink satellites on Falcon 9 rocket from the Kennedy Space Center – Spaceflight Now

SpaceX to launch 29 Starlink satellites on Falcon 9 rocket from NASA’s Kennedy Space Center – Spaceflight Now

  • Trending
  • Comments
  • Latest
Family calls for change after B.C. nurse dies by suicide after attacks on the job

Family calls for change after B.C. nurse dies by suicide after attacks on the job

April 2, 2025
Pioneering 3D printing project shares successes

Product reduces TPH levels to non-hazardous status

November 27, 2024

Hospital Mergers Fail to Deliver Better Care or Lower Costs, Study Finds todayheadline

December 31, 2024

Police ID man who died after Corso Italia fight

December 23, 2024
Harris tells supporters 'never give up' and urges peaceful transfer of power

Harris tells supporters ‘never give up’ and urges peaceful transfer of power

0
Des Moines Man Accused Of Shooting Ex-Girlfriend's Mother

Des Moines Man Accused Of Shooting Ex-Girlfriend’s Mother

0

Trump ‘looks forward’ to White House meeting with Biden

0
Catholic voters were critical to Donald Trump’s blowout victory: ‘Harris snubbed us’

Catholic voters were critical to Donald Trump’s blowout victory: ‘Harris snubbed us’

0

Asia stocks rise on softer yields, Japan firms past strong inflation data todayheadline

May 23, 2025

Sri Lanka expects current account surplus in 2025 for third year: CB Governor todayheadline

May 23, 2025

Why Trump’s ‘Golden Dome’ Won’t Shield the U.S. from Nuclear Strikes todayheadline

May 23, 2025
Donald Trump

RFK Jr. on chemicals, sick kids and that swim in contaminated water

May 23, 2025

Recent News

Asia stocks rise on softer yields, Japan firms past strong inflation data todayheadline

May 23, 2025
4

Sri Lanka expects current account surplus in 2025 for third year: CB Governor todayheadline

May 23, 2025
4

Why Trump’s ‘Golden Dome’ Won’t Shield the U.S. from Nuclear Strikes todayheadline

May 23, 2025
3
Donald Trump

RFK Jr. on chemicals, sick kids and that swim in contaminated water

May 23, 2025
6

TodayHeadline is a dynamic news website dedicated to delivering up-to-date and comprehensive news coverage from around the globe.

Follow Us

Browse by Category

  • Africa
  • Asia
  • Basketball
  • Business & Finance
  • Climate Change
  • Crime & Justice
  • Economic Policies
  • Elections
  • Entertainment
  • Entrepreneurship
  • Environmental Policies
  • Europe
  • Football
  • Gadgets & Devices
  • Health
  • Medical Research
  • Mental Health
  • Middle East
  • Motorsport
  • Olympics
  • Politics
  • Public Health
  • Relationships & Family
  • Science & Environment
  • Software & Apps
  • Space Exploration
  • Sports
  • Stock Market
  • Technology & Startups
  • Tennis
  • Travel
  • Uncategorized
  • Us & Canada
  • Wildlife & Conservation
  • World News

Recent News

Asia stocks rise on softer yields, Japan firms past strong inflation data todayheadline

May 23, 2025

Sri Lanka expects current account surplus in 2025 for third year: CB Governor todayheadline

May 23, 2025
  • Education
  • Lifestyle
  • Technology & Startups
  • About us
  • Contact
  • Advertise with Us
  • Privacy & Policy

© 2024 Todayheadline.co

Welcome Back!

OR

Login to your account below

Forgotten Password?

Retrieve your password

Please enter your username or email address to reset your password.

Log In
No Result
View All Result
  • Business & Finance
  • Corporate News
  • Economic Policies
  • Entrepreneurship
  • Market Trends
  • Crime & Justice
  • Court Cases
  • Criminal Investigations
  • Cybercrime
  • Legal Reforms
  • Policing
  • Education
  • Higher Education
  • Online Learning
  • Entertainment
  • Awards & Festivals
  • Celebrity News
  • Movies
  • Music
  • Health
  • Fitness & Nutrition
  • Medical Breakthroughs
  • Mental Health
  • Pandemic Updates
  • Lifestyle
  • Fashion & Beauty
  • Food & Drink
  • Home & Living
  • Politics
  • Elections
  • Government Policies
  • International Relations
  • Legislative News
  • Political Parties
  • Africa
  • Asia
  • Europe
  • Middle East
  • Artificial Intelligence
  • Cybersecurity
  • Emerging Technologies
  • Gadgets & Devices
  • Industry Analysis
  • Basketball
  • Football
  • Motorsport
  • Olympics
  • Climate Change
  • Environmental Policies
  • Medical Research
  • Science & Environment
  • Space Exploration
  • Wildlife & Conservation
  • Sports
  • Tennis
  • Technology & Startups
  • Software & Apps
  • Startup Success Stories
  • Startups & Innovations
  • Tech Regulations
  • Venture Capital
  • Uncategorized
  • World News
  • Us & Canada
  • Public Health
  • Relationships & Family
  • Travel
  • Research & Innovation
  • Scholarships & Grants
  • School Reforms
  • Stock Market
  • TV & Streaming
  • Advertise with Us
  • Privacy & Policy
  • About us
  • Contact

© 2024 Todayheadline.co