Navigation Links
ASGCT: Gene Therapy Shows Promise for Patients with Pompe Disease
Date:1/30/2010

New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

Milwaukee (Vocus) January 30, 2010 -- New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

“Extraordinary Measures” is based on the true story of John Crowley, a father struggling to find treatment for his children with Pompe disease, a rare, and often fatal, inherited disease that severely weakens the heart and lungs. Currently, the only approved treatment for Pompe is the enzyme-replacement medication Myozyme, which was ultimately developed by Genzyme Corporation.

In a study released this week in Molecular Therapy, ASGCT’s official journal, researchers at the University of Florida reported that gene therapy targeted at the diaphragms of mice with Pompe disease improved the ability of the mice to breathe independently.

This summer, six infants with the disease will receive the treatment. Although the treatment is not a cure, when used in conjunction with other therapies, like the drugs portrayed in the film, it is another step toward improving the lives of patients with Pompe disease.

“While the availability of Myozyme has been a life-saving therapy and a breakthrough for those living with Pompe disease, it must serve as a catalyst for continued innovation,” said Robert Mattaliano, PhD, group vice president of protein research and development at Genzyme. “Gene and cell-based therapies are at the forefront of such endeavors and bring the potential go to beyond replacing a missing enzyme by further improving the muscle repair process in this disease and others.”

Barry Byrne, MD, PhD, director of the Powell Gene Therapy Center at the University of Florida, is one of multiple doctors on which Harrison Ford’s character, Robert Stonehill, is based. Byrne also served as a technical advisor on the film.

"The movie focuses on John (Crowley) and the scientists he worked with to develop a treatment," Byrne said. "The filmmakers strived to create a story the audience will understand. I think it will resonate with people to see how much a parent will go through for his kids."

“Extraordinary Measures” follows John Crowley through his battle to secure funding for research, which eventually leads him partner with William Canfield, PhD, and Canfield’s pharmaceutical company, Novazyme, to develop a treatment for Pompe disease. Harrison Ford’s character is also based on Canfield. Prior to Novazyme finding a treatment, Novazyme was purchased by Genzyme Corporation. Following the purchase, Genzyme remained committed to developing a treatment for Pompe disease; on April 28, 2006, Myozyme was approved by the U.S Food and Drug Administration.

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, www.asgct.org.

# # #

Read the full story at http://www.prweb.com/releases/gene_and_cell_therapy/Pompe_disease/prweb3539914.htm.


'/>"/>
Source: PRWeb
Copyright©2010 Vocus, Inc.
All rights reserved

Related medicine news :

1. AAPM statement on quality radiation therapy
2. Congress Urged to Take Immediate Action to Ensure Frail Patients Continue to Receive Critical Therapy Services
3. Intensive Insulin Therapy Wont Boost Septic Shock Survival
4. Patients with resectable esophageal adenocarcinoma benefit from neoadjuvant chemoradiotherapy
5. Therapy May Relieve Breast Cancer Surgery Complication
6. ALS Therapy Development Institute Receives $1.6 Million Grant From the Department of Defense for Lou Gehrigs Disease Research
7. MBM ScienceBridge GmbH Negotiates License Agreement Between the Georg-August-University Gottingen, Universitatsmedizin, the University of Regensburg, and the UKs Medical Research Council Technology for a New Therapy of Inflammatory and Immune Disea
8. New hope for therapy in heartburn-related cancer
9. Breast cancer multigene test helping patients avoid chemotherapy
10. Psychotherapy May Help Teen Girls Avoid Obesity
11. After Cochlear Implant, Music Therapy May Aid Speech
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/27/2016)... ... June 27, 2016 , ... ... health professionals, announced today its affiliation with Tennessee Counseling Association. This ... the network of the Tennessee Counseling Association, adding exclusive benefits and promotional offers. ...
(Date:6/27/2016)... ... June 27, 2016 , ... TopConsumerReviews.com recently awarded their highest five-star rating to ... of individuals in the United States and Canada wear eyeglasses. Once considered to be ... vision and make a fashion statement. Even celebrities use glasses as a way of ...
(Date:6/26/2016)... (PRWEB) , ... June 26, 2016 , ... Pixel Film ... Pro X. , "Film editors can give their videos a whole new perspective by ... Austin - CEO of Pixel Film Studios. , ProSlice Levels contains over 30 ...
(Date:6/26/2016)... ... June 26, 2016 , ... ... have been diagnosed with endometriosis. These women need a treatment plan to not ... comprehensive approach that can help for preservation of fertility and ultimately achieving a ...
(Date:6/25/2016)... ... , ... The temporary closing of Bruton Memorial Library on June 21 due to a possible ... often overlooked aspect of head lice: the parasite’s ability to live away from a human ... but a necessary one in the event that lice have simply gotten out of control. ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... , June 24, 2016 According ... by Type (Standard Pen Needles, Safety Pen Needles), Needle ... GLP-1, Growth Hormone), Mode of Purchase (Retail, Non-Retail) - ... This report studies the market for the forecast period ... reach USD 2.81 Billion by 2021 from USD 1.65 ...
(Date:6/24/2016)... Belgium , June 24, 2016 ... the appointment of Dr. Edward Futcher ... Non-Executive Director, effective June 23, 2016.Dr. Futcher was ... Nominations and Governance Committees.  As a non-executive member ... independent expertise and strategic counsel to VolitionRx in ...
(Date:6/23/2016)... 2016  MedSource announced today that it has ... solution of choice.  This latest decision demonstrates MedSource,s ... their clients by offering a state-of-the-art electronic data ... nowEDC as the EDC platform of choice in ... "nowEDC has long been a preferred EDC platform ...
Breaking Medicine Technology: