Research Update
Promising Futures
- The new drug denufosol addresses the underlying ion transport defect that exists in cystic fibrosis.
- Hypoglossal nerve stimulation devices show promise as part of an overall treatment for obstructive sleep apnea (OSA).
- The newly tested “triple therapy” technique appears to improve lung function for people with chronic obstructive pulmonary disease (COPD).
- Fiberoptic biopsy allows scientists to analyze the actual airway tissue of asthma patients.
- A vaccine for lung cancer patients is the first breakthrough in fighting the disease to come along in years.
The era of waiting years for the next scientific breakthrough is over. Nearly every day, researchers announce discoveries with the potential to change the lives of millions of people. Here is just a sampling of the promising progress that has been made in the world of medical science to combat COPD, asthma, obstructive sleep apnea, and other ailments that affect healthy respiratory and/or pulmonary function.
RESEARCH UPDATE:
Denufosol for the Treatment of Cystic Fibrosis
It is believed that 30,000 children and adults in the United States suffer from cystic fibrosis (CF). Characterized by thick, sticky mucus that clogs the lungs and obstructs the pancreas, CF cuts young lives short. Fortunately, tremendous progress has been made in CF research since the 1950s, when most children died of the disease before reaching school age. Today, the average CF patient lives into his or her mid-30s.
Treatment for CF involves a multi-tiered approach of nutrition, drug therapy and techniques for loosening airway-obstructing mucus. Until recently there have been no potential options that address the primary cause of the mucus buildup, which is a genetic deformation that prevents the body from achieving the needed balance of water and sodium. However, the new investigational therapy drug denufosol works on that causal factor to help prevent the mucus buildup that ultimately overtakes CF sufferers. According to the company, denufosol “addresses the underlying ion transport defect of the disease. It is a uniquely designed receptor-mediated chloride channel activator that increases chloride secretion, inhibits sodium absorption and increases ciliary beat frequency.”
The drug is manufactured by North Carolina-based biopharmaceutical manufacturer Inspire, and the clinical studies are partially funded by the Cystic Fibrosis Foundation (CFF). It is currently in its second Phase 3 clinical trials, which includes a 48-week trial comparing 60 mg of denufosol to a placebo in 450 CF patients, making it one of the largest and longest trials of CF patients ever conducted. Results are expected in the first quarter of 2011. The drug has fast-track status with the FDA and will hopefully become available later that year.
Researchers stress that this drug, which is unlike others currently in development in that it has the potential to benefit all CF patients, should be administered early in the disease’s progression. New data presented in October at the 23rd Annual North American Cystic Fibrosis Conference (NACFC) in Minneapolis, Minnesota, underscore the need for early intervention. Fortunately, early diagnosis is likely. By law, babies born in the United States must be tested for CF, and 70 percent of CF patients are diagnosed by the age of two. Denufosol is meant to be a lifelong treatment that can be used in conjunction with other drugs and treatment strategies.
RESEARCH UPDATE:
Hypoglossal Nerve Stimulation for the Treatment of Obstructive Sleep Apnea (OSA)
The hypoglossal nerve is one of the two cranial nerves that provide the muscles of the tongue with signals from the brain that allow the tongue to move. The hypoglossal nerves play a critical role in eating, swallowing, and talking. In 2001, Johns Hopkins did a pilot study to test the effectiveness of hypoglossal nerve stimulation in addressing the problem of obstructive sleep apnea (OSA), with some promising results.
Certainly the overall technology of nerve stimulation isn’t new. However, newly developed devices created specifically for hypoglossal nerve stimulation are currently undergoing clinical trials in Europe. The devices are designed to sense the movement of the rib cage and stimulate the tongue muscle, thereby preventing the collapse of throat tissues associated with OSA. So far, three companies have such devices: ImThera, Apnex and Inspire.
The hypoglossal nerve stimulation treatment, which involves an outpatient surgical procedure and regular monitoring, was presented and discussed last year at the Annual Meeting of the American Academy of Otolaryngology – Head & Neck Surgery in San Diego. Although it shows promise, many experts believe that it is best combined with other proven OSA treatments such as CPAP.
RESEARCH UPDATE:
Triple Therapy for the Treatment of Chronic Obstructive Pulmonary Disease (COPD)
Approximately 300,000 people die in the United States every year from COPD-related disease. As respiratory therapists and providers know, treatment of COPD is aimed at limiting exacerbations to prolong and improve the quality of life for COPD patients. A recent study conducted in Germany shows that patients with moderate to severe COPD can benefit from a “triple therapy cocktail” of a longacting a-agonist(LABA), an inhaled corticosteroid (ICS) and an anti-muscarinic agent.
The study, which appeared in an October issue of the American Thoracic Society’s American Journal of Respiratory and Critical Care Medicine, involved more than 600 with moderate to severe COPD who were asked to stop their use of LABA and ICS and use only a tiotropium (anti-muscarinic) inhaler. Participants were then randomized to use either the tiotropium plus LABA and ICS, or the tiotropium plus placebo daily.
After assessing the patients at weeks zero, one, six, and 12, researchers determined that the patients using the triple therapy fared significantly better than those using the placebo. Results included better lung function for the triple therapy group, as well as quicker symptom relief, more than 50% fewer exacerbations, and overall improvement in activity.
The study’s lead author, Prof. Tobias Welte, M.D., head of the Department of Respiratory Medicine of the Hannover Medical School in Germany, notes,“This [triple therapy] approach is of interest because the goal of COPD management is to achieve optimal control.” Until now, the benefits of using all three of these agents had never been demonstrated in a randomized controlled trial.
Researchers say they will investigate the differences between triple and double (ICS plus tiotropium) in future research, as well as the reasons for the seemingly unique effectiveness of triple therapy in treating COPD.
RESEARCH UPDATE:
Fiberoptic Biopsy for the Advancement of Treatments for Asthma
Asthma researchers have been making tremendous strides in investigating both causes and treatments for the disease. One obstacle to research has been obtaining the human airway tissue samples necessary to conduct the needed experiments and analysis. However, the process of minimally invasive fiberoptic biopsy is enabling today’s asthma researchers to obtain those samples more readily.
Asthma researchers previously only used resected human lung tissue or animals to conduct clinical work. “Research is this area has historically been limited by the relative lack of clinical materials, which have to come from asthmatic patients undergoing thoracic surgery for unrelated conditions (such as cancer) or post mortem,” explains Dr. Tak Lee, Head of the Division of Asthma and Allergy Research at King’s College London, and leader of the research on fiberoptic biopsy at that institution. “Then, a few years ago, we successfully developed techniques to obtain bronchial smooth muscle from asthmatic subjects using fiberoptic bronchoscopy. This is safe and can be done relatively easily. The smooth muscle cells can then be isolated from the biopsies and studied in detail.”
Since being able to obtain the needed tissue, scientists have learned that people with asthma have abnormal levels of calcium and reduced levels of SERCA2, which helps to control calcium levels in the cells. This is a potential key to developing new treatments and methods for treating or even preventing asthma.
RESEARCH UPDATE:
The Lucanix Vaccine for the Potential Treatment of Lung Cancer
Today’s prognosis for patients with lung cancer is grim. This common form of cancer results in death for about 85 percent of those diagnosed, partially due to the advanced age of the average lung cancer patient, which is 69. There are drugs to help extend life somewhat, but by and large, there has been little progress in the search for an effective treatment. However, a new vaccine currently in Phase 3 of clinical trials may be the breakthrough that so many people are hoping for.
The vaccine, called Lucanix, is currently being tested in patients who have already completed several rounds of chemotherapy. Lucanix contains lung cancer cells that have been genetically modified in such a way that they won’t suppress the body’s immune system. When the body recognizes the protein called TGF beta (which the genetically modified cells do not have), the cancer is then revealed to the body’s immune system, which in turn goes to work. The desired result: the body becomes programmed to fight off the remaining cancer cells.
“This is kind of more of [what] I would call the designer drug, designed to specifically target those cancer cells and not really hit those healthy cells,” says Lyudmila Bazhenova, M.D., a medical oncologist and hematologist at the University of California, San Diego Morse Cancer Center.
Results of Phase 2 of the study were encouraging. Whereas normally 30 percent of patients survive past the first year following chemotherapy, 61 percent of those who received the vaccine lived at least another year, and 41 percent survived for two years. The current Phase 3 involves 700 patients in 90 locations around the world.
This article originally appeared in the Respiratory & Sleep Management January 2010 issue of HME Business.