The first of two stages of the Phase I clinical trial show positive results for Kamada's Alpha-1 Antitrypsin (AAT) liquid drug candidate for inhalation
delivered with PARI's eFlow electronic nebulizer for the treatment of patients suffering from AAT deficiency. Dosage levels studied resulted in good safety profiles and patient tolerability, leading the way to continued drug development of aerosolized AAT delivered with an optimized eFlow.
The study, which was approved by the European Medicines Agency, involved 24 patients who received various doses of inhaled AAT. AAT, also known as Alpha-1 Proteinase Inhibitor (API), is used for chronic replacement therapy in individuals who lack AAT and have an inherited form of panacinar emphysema.
Lack of AAT leads to various health problems including significant reduction in lung function, lung inflammation, shortness of breath, and recurrent
exacerbations. This leads to emphysema, which is marked by damage to the walls of the air sacs in the lungs resulting in inefficient breathing and shortness of breath.
"In November 2006, we signed a strategic agreement with Kamada to develop inhaled AAT using our advanced aerosol delivery platform, eFlow. Today, we are pleased that this collaboration is showing good initial results and are optimistic that this partnership will benefit patients in the long term," said Dr. Martin Knoch, President of PARI Pharma GmbH. "By developing a targeted therapy that can be administered directly to the lungs, we believe this could be a substantial improvement and a great example of how the advancements in eFlow are helping to improve drug delivery to the lungs in the future."
AAT is currently used for replacement therapy in the form of weekly intravenous infusions that distribute the medication throughout the
bloodstream in order to reach the lungs. An inhaled treatment would offer a more targeted therapy by delivering medication directly to the lungs. Kamada
is developing inhaled AAT in partnership with PARI. Kamada also produces and markets an intravenous AAT in several countries and is undergoing Phase III trials in the United States with its intravenous AAT.
Inhaled AAT was designated, both in Europe and in the USA, as an orphan drugfor the treatment of congenital emphysema and cystic fibrosis.
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Common Cold Virus Leads to Death in Lung Transplant Patients
Date: December 15, 2006
Human rhinovirus (HRV), the leading cause of most common colds, struck two immunosuppressed lung transplant patients, leading to progressive respiratory failure, graft dysfunction and death. The two were part of a group of 11 transplant patients who suffered clinically significant respiratory infection from HRV in both the upper and lower airways, overturning the long-held belief that HRV affects only upper airway tissue.
The research appears in the second issue for December 2006 of the American Journal of Respiratory and Critical Care Medicine, published by the American Thoracic Society.
Laurent Kaiser, M.D., of the Central Laboratory of Virology at the University Hospital of Geneva, Switzerland, and 13 associates assessed the incidence of chronic rhinovirus infection and its potential clinical impact on 69 lung transplant recipients at two centers. Over a 19-month period, they screened
all lung transplant patients using molecular analysis to detect 13 different respiratory viruses. Human rhinovirus is a leading cause of respiratory infections in adults and children. Adults, on average, get infected with the virus once per year. Lung transplant patients, with impaired immune systems due to drugs to halt rejection, are at potentially higher risk from the virus.
“Our evidence demonstrates that rhinoviral disease is not exclusively limited to the upper respiratory tract,” said Dr. Kaiser. “It can also lead to lower respiratory complications, which immunosuppressed patients can be at higher risk of developing.”
Although eight lung-transplant patients had transient rhinoviral infections,
three showed a persistent infection. The others were able to clear the virus
from their system.
“We confirmed the persistence of a single strain in each of three lung
transplant recipients clinically infected by rhinovirus,” said Dr. Kaiser.
“Two of the three had chronic upper respiratory tract infections. All three
had relapsing lower respiratory infections, and two subsequently died with
graft dysfunction.” Dr. Kaiser noted that the persistent infection suggests that certain cases can
act as viral reservoirs to sustain transmission of rhinovirus.
“Therefore, in lung transplant recipients with severe immunosuppression,
clinical rhinovirus infection needs to be considered,” said Dr. Kaiser. “"This
point might have substantial implications in terms of diagnostic procedures,
clinical management, and anti-viral use, if available.”
In an editorial on the research in the same issue of the journal, Marc B.
Hershenson, M.D., of the University of Michigan, Ann Arbor, and Sebastian L.
Johnston, M.D., Ph.D., of the National Heart and Lung Institute at Imperial
College in London, wrote: “"The report by Drs. Kaiser and colleagues ends once
and for all the argument that rhinovirus cannot infect the lower airways.
Although interesting new data suggest that rhinoviruses may induce
proinflammatory responses in lung cells independent of viral replication,
replication is almost certainly required for a maximal response. However,
until the present report, which includes positive bronchoalveolar cultures and
lung immunochemistry, incontrovertible evidence of rhinoviral replication in
the lung in the setting of spontaneous infection has been lacking. This report
informs our understanding of the mechanisms underlying rhinovirus-induced
exacerbations of asthma and COPD.”
They continued: “These exciting new data raise the possibility that patients
with asthma and other patients with chronic airway disease are unusually
susceptible to rhinovirus infection leading to increased rates of
exacerbation. These results may also help explained the increased
susceptibility of children to rhinovirus infections. Further studies on
susceptibility to rhinovirus infection in the population are now required.”
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A Statement from the NIH Director, Elias A. Zerhouni, M.D.,
Regarding the National Institutes of Health Reform Act of 2006
Date: December 11, 2006
“I commend Congress for its overwhelming bipartisan show of support and confidence in the National Institutes of Health. The passage of the 2006 NIH
reauthorization bill is an affirmation of the importance of NIH and its vital role in advancing biomedical research to improve the health of the Nation.
The legislation preserves the core authorities of NIH, while adding new tools to maximize NIH’s effectiveness. Congress has taken an important step towards
modernizing the operation of NIH, in conformance with a new era of science. It brings greater hope for the many people across the Nation and around the world suffering from disease and disability. It also increases our future potential for pre-empting disease before it strikes and improving people’s health.
This support from Congress could not have come at a better moment. We are at a pivotal point in the history of medical research–now is the time to take
full advantage of the tremendous momentum in science to help revolutionize medicine and health in this country.”
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Talecris Continues Global Expansion into Europe
Date: December 1, 2006
Talecris Biotherapeutics GmbH established as a wholly-owned sales and
service organization to serve more than 15 European countries; represents
second expansion in past nine months
RESEARCH TRIANGLE PARK, N.C. & FRANKFURT, GERMANY:
Talecris Biotherapeutics Inc. today announced the establishment of Talecris
Biotherapeutics GmbH, its European headquarters in Frankfurt, Germany. Europe
represents the second expansion in 2006 for the North Carolina-based
biotherapeutics manufacturer, completing the next regional phase in the
company's ongoing global expansion plan.
“Our expansion into Europe signifies another key milestone demonstrating
the strategic growth we have planned for our company,” said Alberto Martinez,
M.D., CEO and President, Talecris Biotherapeutics. “We est ablished Talecris
Europe to deliver tailored sales and support services to our European
customers, and our European leadership team brings the knowledge, experience
and dedication to do exactly that.”
Building on significant success and milestones achieved since its launch,
Talecris has grown rapidly over the last 20 months and is well-positioned for
continued success and growth, while remaining committed to its patients,
employees, customers, and continuing product pipeline innovations.
Commenting on Talecris' establishment of a European presence, Professor
Claus Vogelmeier, M.D., Division of Pulmonary Diseases, University Hospital
Marburg, Germany, said “We are pleased to continue working with Talecris, now
in Europe, to help physicians identify and treat patients with alpha-1
antitrypsin deficiency, also known as genetic emphysema. Their commitment to
invest in research projects and patient and professional support prog rams will
continue to strengthen our ability to improve patient outcomes.”
With the expansion to Europe, Talecris also is now positioned to work
closely with physicians and their patients to improve clinical care of primary
immune deficiency patients. According to Volker Wahn, M.D., of the
Immunodeficiency Center of the Charite (IDCC), Berlin, Germany, “Talecris has
been a valuable industry partner in efforts to educate and inform physicians
about PID. Now patients in Europe may benefit from a stronger Talecris
presence.”
Talecris has experienced tremendous success in its first 20 months of
business. By the end of June 2006, as one of the leading biological products
manufacturers, Talecris achieved sales of more than $1 billion, and has grown
to employ more than 3,000 talented employees. The company supplemented its
business with the acquisitions of Precision Pharma Services, which provides
additional fractionation cap acity, and again in 2006 by acquiring 58 plasma
collection centers from International BioResources to complete the vertical
integration of the business.
For further information: Talecris Biotherapeutics Lacy McMahon,
919-316-6316 Fax: 919-316-6673 E-mail: lacy.mcmahon@talecris.com or
Fleishman-Hillard Uta Schwuchow, +49.(0)89.230 316 35 E-Mail:
uta.schwuchow@fleishmaneurope.com
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“Three Things COPD Sufferers Should Know During the Cold Season.”
Date: November 22, 2006
Holland, MI: If you have emphysema, chronic bronchitis or asthmatic bronchitis (the group of diseases that make up Chronic Obstructive Pulmonary Disease or COPD), you have a few good reasons to keep your teeth clean. There are over 300 species of bacteria that live in your mouth. Periodontal disease is caused by the plaque producing bacteria. Studies suggest that periodontal disease may promote the progression of COPD. Bacteria in the mouth may infect the body either through saliva or from breathing into the lungs. Cytokines are released by the body in defense of periodontal disease. These cytokines tax the body’s immune system.
COPD sufferers know an ordinary cold or flu can be destructive. Coughs linger and flu turns into pneumonia. The bacteria that cause periodontal disease and are taxing your immune system are breeding right now in the cozy, moist, acidic environment of your mouth. The areas between the teeth are particularly good breeding grounds because the bacteria thrive in the absence of oxygen. Symptoms of periodontal disease are often not noticeable until the disease is advanced. A dentist can diagnose the disease in the early stages, prior to individuals realizing they have it.
Periodontal disease is prevented by thoroughly cleaning your teeth. Professional cleanings at a dentist office every six months, brushing teeth twice a day and flossing once a day are recommended. Because it is a laborious task to floss, most people don’t. Yet, to prevent and control periodontal disease, flossing is extremely important. Unfortunately, The Journal of Clinical Periodontology reported that for those that do floss, only 18–35% of the plaque between teeth is removed. And oral irrigators can’t cut through plaque’s sticky biofilm.
More tools are available to keep teeth and gums healthier than in the past. Electric toothbrushes, oral irrigators, tongue scrapers, oral disinfectants and a new device—Dental Air Force® that combines brushing and flossing—are available. The Dental Air Force® (www.dentalairforce.com) also has an added benefit of aerating the sites between teeth, changing the environment and making it difficult for the bacteria to grow.
Studies show that oral health is critical to total health. The National Center for Health Statistics reports that there are over 16 million Americans with COPD and it is the fourth leading cause of death. So, keeping your teeth clean is good “health sense.”
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“Gene Therapy Shows Promise against Hereditary Lung Disease.”
Date: November 21, 2006
GAINESVILLE, Fla.—An experimental gene therapy to combat alpha-1 antitrypsin deficiency, a common hereditary disorder that causes lung and liver disease, has caused no harmful effects in patients and shows signs of being effective, University of Florida researchers say. In a clinical trial, researchers evaluated the safety of using a so-called gene vector—in this case an adeno-associated virus—to deliver a corrective gene to 12 patients who are unable to produce a protein essential for health called alpha-1 antitrypsin.
“The primary end point in the trial was to see whether it was safe to give patients this gene transfer vector and then to try to begin to see if we could get the dose into a range where we would begin to replace the missing protein in the blood,” said Dr. Terence Flotte, a pediatrician, geneticist and microbiologist with UF’s College of Medicine and a member of the Powell Gene Therapy Center and the UF Genetics Institute. “We found that we can use this agent safely and we also saw evidence in the patients’ blood that the higher doses successfully introduced the vector DNA. In one patient we saw evidence for a very brief period that some of the alpha-1 protein was being produced, but not at a high enough level to be beneficial.”
The findings appeared online today (Nov. 21) in the journal Human Gene Therapy. Physicians injected doses of the virus containing copies of the gene for alpha-1 antitrypsin into the patients’ upper arms. Essentially, the virus is intended to “infect” patients’ cells with replacement genes that will do the necessary work to produce alpha-1 protein. UF scientists have successfully developed the technique in animal models. The next step is to test the therapy with a different version of the adeno-associated virus; about 200 variations of the virus exist in nature.
“We have another version of the virus that appears in animal studies to be close to a thousandfold more potent at making protein,” Flotte said. “That’s very encouraging to us. So the next trial, which has already begun, is to use the new version of the virus and take patients through a similar range of doses, in a very similar scheme, and see if we can maintain the safety while pumping up the efficiency of the protein production.”
In most people, alpha-1 antitrypsin is made in the liver and protects the lungs by counteracting inflammatory products that destroy lung tissue. But about 100,000 Americans have alpha-1 antitrypsin deficiency, according to the Miami-based Alpha-1 Foundation, a national not-for-profit organization devoted to finding a cure. In addition, medical authorities suspect less than 5 percent of affected individuals are diagnosed, often not until they are in their mid- to late-30s, after extensive lung damage occurs. Shortness of breath, wheezing, chronic cough and recurring chest colds are signs of the disease.
It is important that alpha-1 patients avoid cigarette smoke, said Dr. Mark Brantly, a professor of medicine and molecular genetics and microbiology at UF’s College of Medicine who develops clinical research programs aimed at developing therapies for alpha-1 patients. Alpha-1 deficiency can in some patients lead to emphysema and cirrhosis, both progressive diseases that can be fatal.
Alpha-1 patients with symptoms of emphysema can be treated through weekly intravenous injections of alpha-1 protein derived from human plasma. The injections must continue throughout a patient’s life, according to the American Lung Association. It does not cure, but it does appear to slow the progression of this disease.
Patients in the clinical trial—10 men and two women who ranged from 42 to 69—were asked to discontinue their replacement therapy 28 days before receiving the gene therapy. One volunteer who had not been on protein replacement therapy exhibited low-level expression of alpha-1 antitrypsin, which was detectable 30 days after receiving an injection. However, residual levels of alpha-1 antitrypsin from the replacement therapy in the other patients obscured whether the alpha-1 gene had begun to express protein.
“As the authors conclude, the results set up the more interesting approach of using other AAV serotypes more suited for muscle delivery as an alternative with the same transgene in the next trial,” said Richard J. Samulski, a professor of pharmacology and director of the University of North Carolina’s Gene Therapy Center. “These studies are important milestones that allow the potential for gene correction of AAT to advance, as well as the (gene therapy) field in general. They also represent the step-by-step process established by the FDA and research community to ensure that safe and good clinical studies are employed in these early days, and I applaud Terry Flotte and his group for being cautious and thorough in their clinical design.”
The trial is funded by a National Institutes of Health grant, and the Alpha-1 Foundation played a crucial role in helping to build the infrastructure to support the research, Flotte said. UF holds an equity interest in Applied Genetic Technologies Corp., a company formed by UF researchers to develop gene therapies.
Credits: Contact John Pastor, jpastor@vpha.health.ufl.edu.
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