Category: Advocacy
Gene Therapy Approaches Milestone; Potential To Cure Diseases Caused By Inherited Gene Defect
You can expect to hear a lot more about gene therapy as a treatment for serious diseases in the years head. Gene therapy is still an experimental treatment in the United States, but holds the potential to cure genetic disorders by replacing a defective gene with a corrected copy of the gene. As The New York Times noted, gene therapy neared a milestone this week.
The European Medicine Agency’s Committee for Medicinal Products recommended approval of a gene therapy medicine to treat lipoprotein lipase (LPL), a rare inherited disorder that involves a defective gene, according to a press release posted Friday on its website. Patients with the disorder cannot produce enough of an enzyme that breaks down fat and may experience life-threatening pancreatitis attacks
If the European Medicine Agency follows the committee’s recommendation, as is typical, the medicine Glybera would be the first gene therapy medication approved for authorization in European countries. No gene therapy has been approved so far in the United States.
Jeffrey Ostrove, chief executive of Ceregene, a gene therapy company in San Diego told The New York Times, that pharmaceutical companies have been reluctant to invest in developing gene therapy drugs because there are no approved medicines. Ostrove said that approval of a gene therapy medicine in western Europe has the potential to change the way gene therapy is viewed.
Researchers have been studying the effectiveness of gene therapy in treating various diseases in clinical trials since a breakthrough discovery in 1989 that an abnormality in one gene on a specific chromosome caused the disease cystic fibrosis. That was a huge discovery. It led to the premise that doctors could cure a patient’s disease by identifying an abnormal gene mutation and replacing the defective gene with a corrected copy. The concept isn’t hard to understand, but making it work is complex. The initial applications of gene therapy are likely to involve rare diseases that may be cured by replacing a single defective gene. Lipoprotein lipase is a good example of that.
Researchers are investigating the applications of gene therapy for treatment of cancer including mesothelioma, a cancer of the lining of the lung caused by asbestos exposure. The University of Pennsylvania has an ongoing gene therapy clinical trial for patients who are newly diagnosed with mesothelioma and patients whose cancer has not responded to other treatments. Patients receive a combination of chemotherapy and a new type of gene therapy called immuno-gene therapy that uses a modified common cold virus to trigger the patient’s immune system to destroy cancer cells. Penn doctors have been encouraged by the response of mesothelioma patients receiving the treatment, Dr. Daniel Sterman an associate professor at the University of Pennsylvania said.
Approximately 2,500 to 3,000 people in the United States are diagnosed with mesothelioma each year with symptoms typically appearing 30 to 50 years after asbestos exposure. The disease is incurable, though there are treatments including chemotherapy, radiation and surgery.
Most people diagnosed with mesothelioma are older workers, retired workers or veterans who were exposed to asbestos fibers in the workplace or military service. Microscopic asbestos fibers when inhaled can lodge in the lungs and remain there a lifetime causing inflammation that eventually leads to asbestos related disease.
Mesothelioma Cases in UK Due to Asbestos Exposure in Workplaces Rises
A recent article published in the British Journal of Cancer discusses occupational cancers that are prevalent in Britain including lung cancer and mesothelioma.
Mesothelioma is a form of cancer that develops in the protective lining of the body’s organs known as the mesothelium. It is associated with exposure to asbestos, a mineral fiber used in many industries in the 20th century. Mesothelioma tumors most commonly appear in the lining of the chest cavity, but also may appear in the lining of the abdominal cavity or the lining around the heart.
According to the article, the number of mesothelioma deaths has increased markedly each year in Great Britain since the 1960s. Mesothelioma accounted for 2,046 cancer deaths in Great Britain in 2005 compared to 153 deaths in 1968, the year that Great Britain began a registry of mesothelioma deaths. The British Mesothelioma Register is the most comprehensive source of data on mesothelioma in the country.
Mesothelioma is more common in men who tend to have jobs in industries that had occupational risk of asbestos exposure. Among the industries that used asbestos and presented a higher risk of mesothelioma are mining and milling, insulating, shipyard work, sheet metal fabrication, asbestos by-products manufacturing, and the asbestos cement industry. Males account for about 85 percent of mesothelioma deaths each year.
Mesothelioma symptoms typically appear 30 to 40 years after exposure to asbestos, meaning that most people who develop the disease are older. They are often retired workers or veterans. About two-thirds of the people diagnosed with mesothelioma are between the ages of 60 and 80 years old, according to the article.
In addition to mesothelioma, the researchers at the British Occupational Cancer Burden Study Group say that a large number of studies report increased lung cancer among workers exposed to asbestos. All forms of asbestos cause cancer, according to the International Agency for Research on Cancer. Lung cancer ranks among the cancer with the highest number of cases associated with occupational exposures. The recent article cites a 1996 study from the American Journal of Occupational Medicine. The earlier article, by researchers at the National Institute for Occupational Safety and Health in the United States, estimated that 9,900 to 11.900 develop lung cancer from occupational exposure to carcinogens and approximately half of the cases involve asbestos.
In the United States, approximately 2,500 to 3,000 people a year are diagnosed with mesothelioma. Most people were exposed to asbestos decades ago in a workplace or during military service such as while serving in the Navy. Mesothelioma is an incurable disease, but there are treatment options to manage the cancer. Treatments are more effective if the cancer is detected before it has reached an advanced stage.
Protein Linked to Tissue Inflammation Suggests New Approach To Treating Mesothelioma, University of Hawaii Researchers Say
Asbestos was used extensively at the Pearl Harbor shipyards during World War II and the decades afterward. Today, Hawaii has some of the highest rates of mesothelioma in the nation, according to cancer researchers at the University of Hawaii.
For more than a decade, researchers at the University of Hawaii Cancer Center have been deciphering the molecular processes that cause normal cells to tranform into malignant mesothelioma cells. In a study in the July issue of the scientific journal Cancer Research, they report that malignant mesothelioma relies on a particular protein known as HMGB1 to fuel the growth of tumors. Suppressing the protein may be key to a new approach for treating mesothelioma, which is notoriously difficult for doctors to manage.
Mesothelioma is a cancer of the lining of the chest and abdominal cavities. It develops from inhaling microscopic asbestos fibers that lodge in the thin tissue lining the body cavity causing inflammation that leads to cancer. People in jobs in which asbestos exposure is an occupational hazard such as mining, shipbuilding, maintenance, plumbing and electrical work have a higher incidence of asbestos-related disease.
Medical researcher Haining Yang, an assistant professor at the University of Hawaii, and colleagues have studied asbestos-related disease for more than a decade. In a series of research papers, the Hawaii cancer researchers have detailed how asbestos damages and kills cells through a process called programmed cell necrosis that leads to the release of a protein molecule called HMGB1.
Patients with mesothelioma have elevated levels of the protein in their blood. The researchers suggest the protein may play a critical role in transforming healthy mesothelial cells into cancer cells and fueling the growth of malignant tumors.
In the most recently published study, the researchers treated mice with malignant mesothelioma with antibodies aimed at suppressing the protein HMGB1. They observed that inhibiting HMGB1 reduced the growth of cancer cells and extended the lives of the mice. Their findings suggest that mesothelioma cells rely on HMGB1 and that removal of the protein may produce a therapeutic response in mesothelioma patients, suggesting a new approach for malignant mesothelioma treatment.
Mesothelioma takes the lives of about 3,000 people a year in the U.S. People typically develop mesothelioma symptoms 20 years to 50 years after exposure to asbestos. The incidence of mesothelioma has risen steadily in the last decade in some parts of the world, including Europe and China.
Work at Shipyards Raises Risk of Mesothelioma
For decades, medical social worker Abby Shulman Palmer was perplexed by what caused the death of her father, a physicist who worked for many years at the Brooklyn Navy Yard. Lester Shulman died of cancer in 1985 at age 69, but doctors never pinpointed where the cancer originated.
After Palmer took a job with the Mesothelioma Alliance as a social worker advocate providing information to mesothelioma patients about treatments and clinical trials, she educated herself about asbestos and gained new insights into her father’s illness.
Palmer, a native of Rockaway and graduate of Rutgers University, learned that the Brooklyn Navy Yard was a hotspot for asbestos exposure during World War II and afterward. Asbestos was widely used by the military in construction of ships because it was heat resistant, tough and inexpensive.
During World War II, Lester Shulman worked on a team that was developing degaussing procedures for U.S military ships to protect them from mines in the water. Degaussing is the process of eliminating unwanted magnetic fields. Asbestos dust was everywhere at Brooklyn Navy Yard, creating an occupational hazard to all workers. Shulman would have been regularly exposed to asbestos.
Inhaling asbestos causes serious respiratory disease including lung cancer and mesothelioma, an aggressive cancer of the lining of the lung. Navy veterans and workers in shipyards have an elevated risk of developing mesothelioma because of the prevalence of asbestos, though disease symptoms take decades to show.
“When my dad died, all we knew was it was cancer, and that it had spread, but the doctors never figured out the primary site,” said Palmer, who is profiled in June issue of the Rutgers alumni magazine. “When I talked with my mother and sister about the possibility that it might be mesothelioma, they said, ‘Wow, that certainly is a possibility.’”
Approximately 2,500 to 3,000 people are diagnosed with mesothelioma each year in the United States. Most are older workers, retired workers and veterans (primarily Navy veterans and yardbirds) who were exposed to asbestos in a workplace.
The symptoms of asbestos disease such as chest pain, fatigue and difficulty breathing typically take 20 years to 50 years to appear. But once the disease appears, it advances aggressively. Mesothelioma is incurable, but there are treatments to control the disease if it is diagnosed at an early stage.
British Study Finds Many Workplace Cancer Cases Involve Asbestos Exposure
About 13,600 new cases of cancer and 8,000 cancer deaths in Great Britain each year are linked to workplace exposures, particularly jobs involving exposure to asbestos or diesel engine fumes, a new study shows.
The study, funded by the British Health and Safety Executive, a government work safety agency, found that nearly half of the cancer deaths were among male construction workers who are most likely to encounter asbestos, a known carcinogen and other carcinogens such as silica and diesel exhaust. Breathing asbestos is associated with serious respiratory diseases including lung cancer, malignant mesothelioma, a cancer of the lining of the lung, and asbestosis, a chronic scarring of the lung.
The study, published in the British Journal of Cancer, indicated that four in 10 work-related cancer cases and nearly half the occupation-related deaths in Britain involved construction workers. Around 70 percent of the occupation-related deaths in construction workers were linked to asbestos.
Even though asbestos is no long used in new construction, remodeling and maintenance on older buildings containing asbestos materials can put workers at risk of exposure to the asbestos fibers.
“This study gives us a clear insight into how the jobs people do affect their risk of cancer,” Dr. Lesley Rushton, an occupational epidemiologist at Imperial College London said in British Journal of Cancer press release. “We hope these findings will help develop ways of reducing health risks caused by exposure to carcinogens in the workplace.”
The researchers cautioned that the estimates of cancer cases and deaths related to occupational exposure are conservative and could be high as new work-related risk facts are identified.
Asbestos remains the most important occupational risk factor.
Dame Helena Shovelton, chief executive of the British Lung Foundation, a non-profit group, said in a BBC news report that asbestos-related diseases kill more people in Great Britain than traffic accidents and the number of deaths is projected to continue increasing in Britain until 2016.
Millions of houses and building were built in Britain and the United States during the decades when asbestos was a widely used building from World War II to about 1980. As long as people are living or working in the buildings, they are at risk of exposure to asbestos if the material is disturbed.
When inhaled, microscopic asbestos fibers typically lodge in the lungs, causing inflammation that can eventually lead to malignancy. Symptoms of mesothelioma and other asbestos diseases typically take 20 years to 40 years to appear. People recently diagnosed with mesothelioma may have been exposed to asbestos in the 1960s or 1970s.
Approximately, 3,000 people are diagnosed with malignant mesothelioma each year in the United States. Most are older workers, retired workers and veterans who were exposed to asbestos in a workplace such as a factory, shipyard or construction site. Construction workers and demolition workers are among the occupations most at risk today of asbestos exposure.
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