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:6/26/2016)... ... June 26, 2016 , ... PawPaws brand pet supplements ... that was developed to enhance the health of felines. The formula is all-natural and ... main herbs in the PawPaws Cat Kidney Support Supplement Soft Chews are ...
(Date:6/25/2016)... ... , ... Austin residents seeking Mohs surgery services, can now turn to Dr. ... Russell Peckham for medical and surgical dermatology. , Dr. Dorsey brings specialization to include ... in Mohs Micrographic Surgery completed by Dr. Dorsey was under the direction of Glenn ...
(Date:6/25/2016)... CA (PRWEB) , ... June 25, 2016 , ... As ... with Magna Cum Laude and his M.D from the David Geffen School of Medicine ... and returned to Los Angeles to complete his fellowship in hematology/oncology at the UCLA-Olive ...
(Date:6/24/2016)... ... June 24, 2016 , ... A recent article published June 14 ... The article goes on to state that individuals are now more comfortable seeking to ... operations such as calf and cheek reduction. The Los Angeles area medical group, Beverly ...
(Date:6/24/2016)... ... June 24, 2016 , ... The Pulmonary Hypertension Association (PHA) ... will receive two significant new grants to support its work to advance research ... anniversary by recognizing patients, medical professionals and scientists for their work in fighting ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... , June 24, 2016  Collagen Matrix, ... design, development and manufacturing of collagen and mineral ... announced today that Bill Messer has ... Marketing to further leverage the growing portfolio of ... devices. Bill joins the Collagen Matrix ...
(Date:6/24/2016)... -- The Academy of Managed Care Pharmacy (AMCP) today ... allow biopharmaceutical companies to more easily share health care ... coverage decisions, a move that addresses the growing need ... The recommendations address restrictions in the sharing of product ... a prohibition that hinders decision makers from accessing HCEI ...
(Date:6/24/2016)... 2016   Pulmatrix, Inc ., (NASDAQ: ... drugs, announced today that it was added to the ... its comprehensive set of U.S. and global equity indexes ... important milestone for Pulmatrix," said Chief Executive Officer ... our progress in developing drugs for crucial unmet medical ...
Breaking Medicine Technology: