A discovery by Washington State University scientist Dan Rodgers and collaborator Paul Gregorevic could save millions of people suffering from muscle wasting disease.
The result of the team’s four-year project is a novel gene therapeutic approach. The work was published (http://stm.sciencemag.org/content/8/348/348ra98) July 20 in Science Translational Medicine, a journal of the American Association for the Advancement of Science.
“Chronic disease affects more than half of the world’s population,” said Rodgers, professor of animal sciences (https://ansci.wsu.edu/people/faculty/dan-rodgers/) and director of the Washington Center for Muscle Biology (http://wcmb.wsu.edu/). “Most of those diseases are accompanied by muscle wasting.
“It occurs with chronic infection, muscular dystrophy, malnutrition and old age,” he said. “About half the people who die from cancer are actually dying from muscle wasting and there’s not one single therapy out there that addresses it.
Family history inspires search for treatment
“I have a strong motivation to do something about this, to do more than simply publish results,” said Rodgers, who teamed with Gregorevic of Baker IDI Heart and Diabetes Institute in Australia (https://www.bakeridi.edu.au/). “My father died from cachexia,” the wasting disease caused by cancer, “and my nephew has Duchenne muscular dystrophy, an incurable, fatal disease that could claim his life in his teens.
“Others have tried and failed to develop treatments for muscle wasting,” Rodgers said, “and some drugs have even caused serious safety problems. Our targeted approach only affects muscle and completely avoids these problems, which is why we think we have a solution.”
In the paper, lead author Catherine Winbanks, a postdoctoral fellow of Gregorevic, details how researchers built muscle in healthy mice and prevented the loss of skeletal and heart muscle in mice with tumors.
Hormone’s muscle-wasting effect blocked
In cachexia, tumors secrete hormones that cause muscle deterioration; in effect, the body eats its own muscles, causing weakness, frailty and fatigue.
“What kills a lot of people isn’t the loss of skeletal muscle but heart muscle,” said Rodgers. “The heart literally shrinks, causing heart failure.”
Researchers have long sought to stop this process, but failed to find a safe way. That’s because the hormones that cause wasting – in particular, a naturally occurring hormone called myostatin – play important roles elsewhere in the body.
Rodgers and Gregorevic needed a way to stop myostatin, but only in muscles. Their solution: an adeno-associated virus – a benign virus that specifically targets heart and skeletal muscle.
The virus delivers a small piece of DNA – a signaling protein called Smad7 – into muscle cells. Smad7 then blocks two signaling proteins called Smad2 and Smad3, which are activated by myostatin and other muscle-wasting hormones. By blocking those signals, Smad7 stops the breakdown of muscles.
“Smad7 is the body’s natural break and, by inhibiting the inhibitor, you build muscle,” Rodgers said.
For cachexia patients, such a therapy could massively increase their chances of survival.
“Instead of having one year to fight cancer, you’d have 10 or 15,” Rodgers said.
Startup works to develop commercial drug
In 2015, Rodgers launched AAVogen, a company that will develop this discovery into a commercial drug, AVGN7.
He has been working with Norman Ong, a technology licensing associate at WSU’s Office of Commercialization, on patents, startup funding and recruitment for AAVogen. Using the funds from WSU’s commercial gap fund award, Rodgers’ lab will determine the minimum effective dose for AVGN7.
“We want to turn WSU discoveries into real-world uses that benefit the public,” said Ong. “Dan is a very busy scientist, so we’re proud to help him and AAVogen connect with the right people.”
“I formed this company for one purpose: to move the science into society, to see it applied,” Rodgers said. “WSU’s Office of Commercialization has been instrumental and invaluable to this endeavor.
“Now we have a company with the potential to save a lot of lives,” he said.
Learn more: Scientist develops gene therapy for muscle wasting
The Latest on: Muscle wasting
[google_news title=”” keyword=”Muscle wasting” num_posts=”10″ blurb_length=”0″ show_thumb=”left”]
via Google News
The Latest on: Muscle wasting
- U.S Rep. Marjorie Taylor Greene Says the U.S Should Have Deported Murdered Student Laken Rileyon April 28, 2024 at 6:38 am
Congresswoman Marjorie Taylor Greene made an unexpected statement on Laken Riley’s terrible murder. The controversial lawmaker made the comment during a recent Homeland Security Committee budget ...
- Minnesota DNR “debunks” fear among deer hunters about transfer of Chronic Wasting Disease to humanson April 27, 2024 at 7:02 am
ST. PAUL, Minn. (KFGO/WCCO) – A recent article published by Field and Stream has sparked unnecessary fear and panic among Minnesota deer hunters according to the Minnesota DNR. Chronic Wasting Disease ...
- Changing lives, one arm at a time - The Funny Business of Parentingon April 26, 2024 at 2:09 am
One of the things you should not do is offer to arm wrestle a child. If you arm wrestle a child and lose, you will be reminded of it every day for the rest of your life. I don’t know how it happened, ...
- So THAT'S Why Your Voice Sounds Older When You Ageon April 25, 2024 at 11:59 pm
Presbyphonia can start as early as your 50s, but grows in prevalence as people age, said otolaryngologist Dr. Dehra McGuire, known as “voicedocok” on Instagram. “Most of the patients I see are around ...
- Veru to Present at the American Association of Clinical Endocrinology (AACE) 2024 Annual Meeting on May 9-11on April 25, 2024 at 7:08 am
About Veru Inc. Veru is a late clinical stage biopharmaceutical company focused on developing novel medicines for the treatment of metabolic diseases, oncology, and ARDS. The Company’s drug ...
- 3 Speculative Weight Loss Drug Stocks to Bet On for Big Gainson April 24, 2024 at 3:33 am
InvestorPlace - Stock Market News, Stock Advice & Trading Tips Investors continue to search for the next big weight loss drug stocks ...
- Woman with incurable muscle-wasting disease becomes Peru's first to die by euthanasiaon April 23, 2024 at 9:29 am
Ana Estrada, 47, has become the first person to die by euthanasia in Peru after suffering from an incurable disease for several years and campaigning for the right to die ...
- Best SARMS Stack for Bulking and Cutting 2024 - Top SARM Stacks for Muscle Growth and Fat Losson April 23, 2024 at 12:02 am
Although it's possible to use a single SARM on its own, it's more common to combine two or more SARMs and use them together. The best SARMs stack for bulking consists of RAD 140, Ligandrol, and ...
- Visiting Speaker to Discuss Impacts of Treatment and Tumor on Wasting During Lung Canceron April 22, 2024 at 10:24 pm
Deena Snoke, an up-and-coming leader in the study of cancer-induced cachexia, will speak at the U of A at 2 p.m. on Friday, April 26, in HPER 311.
- I’m 25 & my muscles are wasting away fatally but I can't afford S$375,000/year treatmenton April 19, 2024 at 4:45 am
"It’s a devastating, cruel irony, to know that there is a drug that can stop my body from wasting away and help me maintain my quality of life, yet it’s financially inaccessible." ...
via Bing News