Dignitana AB Collaborates with Target Health Inc. to Conduct Pivotal Study Using Proprietary Clinical Trial Software

 

Dignitana AB Collaborates with Target Health Inc. to Conduct Pivotal Study Using Proprietary Clinical Trial Software

 

To the best of our knowledge, this announcement is for the FIRST submission, ever, that used eSource derived data fully integrated with an EDC system, where the study sites entered data at the time of the patient encounter. For this study, source data were entered directly into Target e*CRF® by the clinical site without having to write it down first on a piece of paper. eSource records were first captured in Target e*CTR (Target e*Clinical Trial Record)® before the data “hit” the Target e*CRF database. Thus, we were able to provide a system which complied with the creation of an “Independent Contemporaneous Independent Investigator Copy (CIIC)” of the source record, and where the investigational sites had full control of, and user access to, their eSource records. Not only was eSource used, but the benefits were huge. Here is what Jan Richardson, Dignitana CEO, stated: “The clinical trial was completed four months earlier than originally planned, thanks to the efforts of the investigators and their staff, and to the use of Target Health’s software solution for accelerating conduct of clinical trials.“

 

This project also hit The New York Times.

 

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Dignitana AB announced that the company has submitted for review an application to the US Food and Drug Administration (FDA) Center for Devices and Radiologic Health for approval of the DigniCap™ System. The DigniCap™ System is currently approved for marketing in Australia, Canada, China, Colombia, most European countries, Mexico, Russian Federation, Singapore and South Korea.

 

Dignitana engaged the Clinical Research Organization (CRO) Target Health Inc., with offices in New York and opening soon in New Jersey, in 2011, and Dignitana has been working in close collaboration with Target Health to interact with FDA, conduct the pivotal clinical trial and prepare the FDA application.

 

The pivotal clinical study was conducted at five clinical sites; University of California San Francisco, Wake Forest Baptist Medical Center, Weill Cornell Medical College, Beth Israel Medical Center, and UCLA Hematology Oncology Santa Monica. The first patient was enrolled in August, 2013 and the last patient was enrolled in October, 2014. A total of 101 evaluable patients were included in the treatment group and 16 patients were enrolled in the control group.

 

Dignitana would like to thank Target Health Inc., a Manhattan-based CRO, for their professionalism and expertise in interacting with the FDA, preparing and submitting the Investigational Device Exemption (IDE) and FDA application, as well as managing the clinical trial, performing data management, running the statistical analyses and writing the study report. Target Health’s novel approach to the paperless clinical trial included Target e*CTR (eClinical Trial Record®), a patented software that is fully integrated with Target e*CRF® (EDC). Use of this software resulted in an efficient and cost-effective clinical trial program, and allowed the clinical sites to enter the trial data directly into the study database at the time of the office visit.

 

Fixed in Ice – End of Winter in North Carolina

 

For this photo, our good friend and colleague, James Farley Director, Data Management and Programming at TransTech Pharma, “got down on the ground totally, focused and set my camera on the ice to get that ultra-low perspective; this made for more dramatic photos.

 

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Fixed in Ice ©JFarley Photography 2015

 

ON TARGET is the newsletter of Target Health Inc., a NYC-based, full-service, contract research organization (eCRO), providing strategic planning, regulatory affairs, clinical research, data management, biostatistics, medical writing and software services to the pharmaceutical and device industries, including the paperless clinical trial.

 

For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 104). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel or Ms. Joyce Hays. The Target Health software tools are designed to partner with both CROs and Sponsors. Please visit the Target Health Website.

 

Joyce Hays, Founder and Editor in Chief of On Target

Jules Mitchel, Editor

 

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Lacrimal Gland (tear ducts)

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As we get older, our eyes get dryer, our lacrimal (tear duct) glands have fewer tears. We thought our readers might be interested in this topic.

 

The lacrimal glands are paired almond-shaped glands, one for each 1) ___, that secrete the aqueous layer of the tear film. They are situated in the upper, outer portion of each orbit, in the lacrimal fossa of the orbit formed by the frontal bone. Inflammation of the lacrimal glands is called dacryoadenitis. The lacrimal gland produces tears which then flow into canals that lead to the lacrimal sac. From this sac, the tears drain through the lacrimal duct into the 2) ___. Anatomists divide the gland into two sections. The smaller palpebral portionlies close to the eye, along the inner surface of the eyelid; if the upper 3) ___is everted, the palpebral portion can be seen. Muscles in the upper eyelids were discovered by the first anatomist, Giovanni Battista Morgagni MD (1682-1771).

 

4) ___ secreted collect in the fornix conjunctiva of the upper lid, and pass over the eye surface to the lacrimal puncta, which are small holes found at the inner corner of the eyelids. These pass the tears through the lacrimal canaliculi on to the lacrimal sac, in turn to the nasolacrimal duct, which dumps them out into the nose. The lacrimal gland is a compound tubuloacinar gland, which is made up of many lobules separated by connective 5) ___. Each lobule contains many acini. The acini contain only serous cells and produce a watery serous secretion. Each acinus consists of a grape-like mass of lacrimal gland cells with their apices pointed to a central lumen. The maxillary nerve, which travels through the inferior orbital fissure. Once it has traversed this opening, the parasympathetic secretomotor fibers branch off with the zygomatic nerve and then branch off again, joining with the lacrimal branch of the ophthalmic division of CN V, which supplies sensory innervation to the lacrimal 6) ___ along with the eyelid and conjunctiva. The lacrimal artery, derived from the ophthalmic artery supplies the lacrimal gland. Venous 7) ___ returns via the superior ophthalmic vein. The glands drain into the superficial parotid lymph 8) ___. The lacrimal nerve, derived from the ophthalmic nerve, supplies the sensory component of the lacrimal gland. The greater petrosal nerve, derived from the facial nerve, supplies the parasympathetic autonomic component of the lacrimal gland.

 

When you have chronic 9) ___ eye disease, the eye glands that make tears become inflamed. The inflammation affects the nerves in your eyes, leading to decreased tear production. In contrast to normal moisture of the eyes or even crying, there can be persistent dryness, scratching, and burning in the eyes, which are signs of dry eye syndrome (DES) or keratoconjunctivitis sicca (KCS). With this syndrome, the lacrimal glands produce less lacrimal fluid, which mainly occurs with 10) ___ or certain medications. A thin strip of filter paper placed at the edge of the eye, the Schirmer test, can determine the level of dryness of the eye. Many medications or diseases that cause dry eye syndrome can also cause hyposalivation with xerostomia. Treatment varies according to etiology and includes avoidance of exacerbating factors, tear stimulation and supplementation, increasing tear retention, eyelid cleansing, and treatment of eye inflammation.

 

ANSWERS: 1) eye; 2) nose; 3) eyelid; 4) Tears; 5) tissue; 6) gland; 7) blood; 8) nodes; 9) dry; 10) aging

 

https://www.youtube.com/watch?v=G-kJVmEWWV8

 

Lacrimosa – Mozart – Requiem – Karl Bohm – Sinfonica de Vienna

 

Giovanni Battista Morgagni MD (1682-1771)

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Careful, steady respected scholarship with long lasting contributions to medicine.

 

Giovanni Battista Morgagni was an Italian anatomist, celebrated as the father of modern anatomical pathology. His careful, steady respected scholarship has left a legacy of long lasting contributions to medicine. His parents were in comfortable circumstances, but not of the nobility. His wife was from nobility, which probably influenced his own rank. It appears from his letters to Giovanni Maria Lancisi, that Morgagni was ambitious, and it may be inferred that he succeeded from the fact that he is described on a memorial tablet at Padua as nobilis forolensis, “noble of Forli.“

 

At the age of sixteen Morgagni went to Bologna to study philosophy and medicine, and he graduated as doctor in both faculties three years later, in 1701. He acted as prosector to Antonio Maria Valsalva (one of the distinguished pupils of Malpighi), who held the office of demonstrator anatomicus in the Bologna school, and where he assisted more particularly in preparing his celebrated work on the Anatomy and Diseases of the Ear, which was published in 1704. In 1740, Morgagni edited a collected edition of Valsalva’s writings, with important additions to the treatise on the ear, and with a memoir of the author. When Valsalva was transferred to Parma, Morgagni succeeded to his anatomical “demonstratorship“. During this period, he enjoyed a high reputation in Bologna where, at 24, he was made president of the Academia Enquietorum. He was said to have signaled his tenure of the presidential chair by discouraging abstract speculations, and by setting the expectations towards exact anatomical observation and reasoning. Morgagni published the substance of his communications to the Academy in 1706 under the title of Adversaria anatomica, the first of a series by which he became known throughout Europe as an accurate anatomist. The book included Observations of the Larynx, the Lachrymal Apparatus, and the Pelvic Organs in the Female. After a time he gave up his post at Bologna, and occupied himself for the next two or three years at Padua, where he had a friend in Domenico Guglielmini (1655-1710), professor of medicine, but better-known as a writer on physics and mathematics. Guglielmini wanted to see Morgagni as a teacher at Padua, but the unexpected death of Guglielmini made this possible. Antonio Vallisneri (1661-1730) was then transferred to the vacant chair, and Morgagni succeeded to the chair of theoretical medicine. At 31, Morgagni came to Padua in the spring of 1712, and he taught medicine there with the most brilliant success until his death on 6 December 1771.

 

When Morgagni had been three years in Padua an opportunity he was promoted by the Venetian senate to the chair of anatomy, in which he became the successor of an illustrious line of scholars, including Vesalius, Gabriele Falloppio, Geronimo Fabrizio, Gasserius, and Adrianus Spigelius. Shortly after coming to Padua he married a noble lady of Forli, who bore him three sons and twelve daughters. Morgagni enjoyed an unequaled popularity among all classes. He was of tall and dignified figure, with blonde hair and lilac eyes, and with a frank and happy expression. His manners were polished, and he was noted for the elegance of his Latin style. He lived in harmony with his colleagues, who are said not even to have envied him his unprecedentedly large stipend. His house and lecture-theatre were frequented by students of all ages, attracted from all parts of Europe. Morgagni enjoyed the friendship and favor of distinguished Venetian senators and of cardinals, and successive popes conferred honors upon him. Before he had been long in Padua, the students of the German nation, of all the faculties there, elected him their patron. He then advised and assisted them in the purchase of a house to be a German library and club. Morgagni was elected into the imperial Caesareo-Leopoldina Academy in 1708 (originally located at Schweinfurth), and to a higher grade in 1732, into the Royal Society in 1724; into the Paris Academy of Sciences in 1731; the St. Petersburg Academy in 1735; and the Berlin Academy of Sciences in 1754. Among Morgagni’s more celebrated pupils were Antonio Scarpa who connected the school of Morgagni with the modern era, Domenico Cotugno, and Leopoldo Marco Antonio Caldani, the author of the magnificent atlas of anatomical plates published in 2 volumes at Venice in 1801-1814.

 

In his earlier years at Padua, Morgagni brought out five more series of the Adversaria anatomica (1717-1719). These strictly medical publications were few and casual (on gallstones, varices of the Venae cavae, cases of stone, and several memoranda on medico-legal points, drawn up at the request of the curia). Classical scholarship in those years occupied his pen more than anatomical observation. It was not until 1761, when he was in his eightieth year, that he brought out the great work which, once for all, made pathological anatomy a science, and diverted the course of medicine into new channels of exactness or precision – the De Sedibus et causis morborum per anatomem indagatis (Of the seats and causes of diseases investigated through anatomy), in five books printed as two folio volumes, which during the succeeding ten years, notwithstanding its bulk, was reprinted several times (thrice in four years) in its original Latin, and was translated into French (1765), English (1769), and German (1771). The only special treatise on pathological anatomy previous to that of Morgagni was the work of Theophile Bonet of Neuchatel, Sepulchretum: sive anatomia practica ex cadaveribus morbo denatis, “The Cemetery, or, anatomy practiced from corpses dead of disease“, first published in 1679, three years before Morgagni was born. It was republished in Geneva in 1700, and again at Leiden in 1709. Although the normal anatomy of the body had been comprehensively, and in some parts exhaustively, written by Vesalius and Fallopius, it had not occurred to anyone to examine and describe systematically the anatomy of diseased organs and parts. Harvey, a century after Vesalius, poignantly remarked that there is more to be learned from the dissection of one person who had died of tuberculosis or other chronic malady than from the bodies of ten persons who had been hanged. Francis Glisson (1597-1677) shows in a passage quoted by Bonet in the preface to the Sepulchretum, that he was familiar with the idea, at least, of systematically comparing the state of the organs in a series of bodies, and of noting those conditions which invariably accompanied a given set of symptoms. The work of Bonet was, however, the first attempt at a system of morbid anatomy, and, although it dwelt mostly upon curiosities and monstrosities, it enjoyed much repute in its day. Haller speaks of it as an immortal work, which may in itself serve for a pathological library. Morgagni, in the preface to his own work, discusses the defects and merits of the Sepulchrelum: which was largely a compilation of other men’s cases, some not well authenticated. Morgagni’s work could now be called objective impartiality, a quality which was introduced as decisively into morbid anatomy by Morgagni as it had been introduced two centuries earlier into normal human anatomy by Vesalius.

 

Morgagni narrated the circumstances under which the De Sedibus took origin. Having finished his edition of Valsalva in 1740, he was taking a holiday in the country, spending much of his time in the company, of a young friend who was curious in many branches of knowledge. The conversation turned upon the Sepulchretum of Bonet, and it was suggested to Morgagni by his dilettante friend that he should put on record his own observations. It was agreed that letters on the anatomy of diseased, organs and parts should be written for the perusal of this favored youth; and they were continued from time to time until they numbered seventy. Those seventy letters constitute the De sedibus et causis morborum, which was given to the world as a systematic treatise in 1761, twenty years after the task of epistolary instruction was begun. The letters are arranged in five books, treating of the morbid conditions of the body a capite ad calcem, and together containing the records of some 646 dissections. Some of these are given at great length, and with a precision of statement and exhaustiveness of detail hardly surpassed in the so-called protocols of the German pathological institutes of the present time. Others, again, are fragments brought in to elucidate some question that had arisen. The symptoms during the course of the malady and other antecedent circumstances are always prefixed with more or less fullness, and discussed from the point of view of the conditions found after death. Subjects in all ranks of life, including several cardinals, figure in this remarkable gallery of the dead. Many of the cases are taken from Morgagni’s early experiences at Bologna, and from the records of his teachers Valsalva and I.F. Albertini (1662-1738) not published anywhere else. They are selected and arranged with method and purpose, and they often consist of a long excursus on general pathology and medicine.

 

The range of Morgagni’s scholarship, as evidenced by his references to early and contemporary literature, is astonishing. His works differ from treatises of his time insofar as to his approach that symptoms determine the order and manner of presenting the anatomical facts. His 1769 work described the post mortem findings of air in cerebral circulation and surmised this was the cause of death. Although Morgagni’s cases resulted from gas embolism due to damage to the bowel, the same pathology is seen in decompression illness. Although Morgagni was the first to understand and to demonstrate the absolute necessity of basing diagnosis, prognosis, and treatment on an exact and comprehensive knowledge of anatomical conditions, he made no attempt (like that of the Vienna school sixty years later) to exalt pathological anatomy into a science disconnected from clinical medicine and remote from practical experience with the scalpel. His precision, his exhaustiveness, and his freedom from bias are his essentially modern or scientific qualities. His scholarship and high consideration for classical and foreign work, his sense of practical ends (or his common sense), and the breadth of his intellectual horizon prove him to have lived before medical science had become largely technical or mechanical. His treatise was the commencement of the era of steady, or cumulative progress in pathology and in practical medicine. From that time on, symptoms ceased to be made up into more or less conventional groups, each of which was a disease On the other hand, they began to be viewed as the cry of the suffering organs, and it became possible to develop Thomas Sydenham’s grand conception of a natural history of disease in a catholic or scientific spirit.

 

How Genetic Changes Lead to Familial Alzheimer’s Disease

 

Mutations in the presenilin-1 gene are the most common cause of inherited, early-onset forms of Alzheimer’s disease (AD). Presenilin is a component of an important enzyme, gamma secretase, which cuts up amyloid precursor protein into two protein fragments, Abeta40 and Abeta42. Abeta42 is found in plaques, the abnormal accumulations of protein in the brain which are a hallmark of AD. Numerous studies suggested that presenilin-1 mutations increased activity of gamma-secretase. However, it has been unclear exactly how the presenilin mutations cause AD. Drugs have been developed that block gamma-secretase, but they have so far failed in clinical trials to halt the disease.

 

Results from an NIH-funded study, published online in Neuron (4 March 2015),  may transform the way scientists design drugs that target these mutations to treat inherited or familial AD, a rare form of the disease that affects approximately 1% of people with the disorder.

 

The study led by Raymond Kelleher, M.D., Ph.D. and Jie Shen, Ph.D., professors of neurology at Harvard Medical School, Boston, provides a plot twist in the association of presenilin-1 mutations and inherited Alzheimer’s disease. Using mice with altered forms of the presenilin gene, the authors discovered that the mutations may cause the disease by decreasing, rather than increasing, the activity of gamma-secretase. One of the presenilin mutations also caused impairment of memory circuits in the mouse brain and age-dependent death of neurons.

 

Although plaques are the main biological indicator of AD, neurodegenerative changes are also an important feature of the disease. These changes include loss of brain cells, cognitive deficits such as problems with memory, changes in the brain’s electrical activity and inflammation. Commonly used mouse models of the disease exhibit excessive plaque deposition, but do not show symptoms of neurodegeneration. According to the authors, this may be one reason that treatments developed in mice have not been successful in patients.

 

Previous research demonstrated that presenilins and gamma-secretase play an important role in learning and memory, communication between brain cells and neuronal survival, and cautioned against the use of gamma-secretase inhibitors for Alzheimer’s disease therapy. Later, a large phase III trial was stopped because treatment with a gamma-secretase inhibitor worsened the cognitive ability of patients.

 

Although the majority of cases are not inherited, familial AD is associated with early onset of the disorder, with symptoms often appearing before age 60. The authors hope that the mechanisms uncovered in this study may provide insight into the common forms of the disorder that affect more than five million people in the United States.

 

Unexpected New Indications for Marketed Drugs – A Must Read

 

This article describes 2 new findings that marketed products may have more than one indication for use and that mechanisms of actions of diseases may overlap and may be more important than having limited diagnoses.

 

ANTI-HERPES DRUG MAY HELP CONTROL HIV

Typically, HIV patients take a cocktail of several anti-HIV drugs because a single drug is not enough to suppress the virus. Multiple HIV medications also hinder the virus’ ability to develop resistance to the drugs.

 

According to a study published online in Clinical Infectious Diseases (13 March 2015), Valacyclovir, a drug commonly used to control the virus that causes genital herpes, appears to reduce the levels of HIV in patients who do not have genital herpes. The study of 18 patients is the first to show that the drug does not require the presence of herpes simplex virus 2 (HSV-2) to suppress HIV in patients. According to the authors, if valacyclovir’s effectiveness against HIV can be confirmed in a larger cohort, it could be added to the mix of drugs used to suppress the virus, and might prove especially helpful in cases in which HIV has developed resistance to other drugs.

 

These results follow a 2008 study by the same research team, which showed that acyclovir suppresses HIV in laboratory cultures of human tissues that were infected with various kinds of herpes viruses. Valacyclovir is referred to as a prodrug for acyclovir because it’s structurally similar to acyclovir, and is converted to acyclovir in the body. For the current study, the researchers used valacyclovir because it remains in the blood longer than acyclovir and so would not need to be taken as often. Earlier studies have shown that acyclovir reduces HIV levels in patients coinfected with HIV and HSV-2, the virus that causes genital herpes. However, this effect has been attributed to the drug’s anti-HSV-2 activity. The decrease in immune activity results in fewer active immune cells for HIV to infect. In contrast, results of the current study indicated that the drug likely reduced HIV levels by interfering directly with HIV’s reproductive machinery and did not require the presence of HSV-2. HSV-2 chemically alters acyclovir, by attaching chemical groups known as phosphates to it. It is this altered form of the drug that suppresses HSV-2. The authors believe this form also interferes with HIV’s ability to reproduce. In their earlier study, it was found that many other kinds of herpes viruses can also attach phosphate groups to acyclovir. It was noted that these other herpes viruses are widespread and that most people harbor at least one of them.

 

For the study, 18 HIV-infected patients were enrolled, none of whom were infected with HSV-2. Half of the enrolled patients took valacyclovir twice a day while the other half received a placebo. After two weeks, the placebo group received valacyclovir, while the group originally treated with the drug switched to the placebo. Results showed that when the patients took valacyclovir, their blood HIV levels declined significantly. In addition, when a genetic analysis was conducted it was found that the HIV in the study volunteers did not develop resistance to valacyclovir. But because HIV has a history of becoming resistant to the drugs used to treat it, the authors do not discount the possibility that the virus could develop resistance to valacyclovir with longer treatment. Given the ability of the drug to lower HIV levels, however, it was suggested that valacyclovir could one day be added to the cocktail of drugs given to HIV-infected people. Clearly, larger randomized trials and cost effectiveness analyses are warranted to further explore the potential of valacyclovir in the context of HIV-1 infection, in particular in combination with other antivirals.

 

Tetanus Shot Improves Patient Survival With Brain Tumor Immunotherapy

According to an article published online in the journal Nature (11 March 2015), an innovative approach using a tetanus booster to prime the immune system was shown to enhance the effect of a vaccine therapy for lethal brain tumors, dramatically improving patient survival. The study not only presented survival data for a small, randomized and blinded patient trial, also presented details on how the tetanus pre-conditioning technique works, providing a roadmap for enhancing dendritic cell immunotherapies that have shown promise treating the most lethal form of brain cancer.

 

The researchers built the study on earlier findings that glioblastoma tumors harbor a strain of cytomegalovirus (CMV) that is not present in the surrounding brain tissue, creating a natural target for an immune therapy. One such targeted approach uses dendritic cells, which train the immune system to respond to specific pathogens. The research team developed a process to extract white blood cells, coax the growth of dendritic cells and load them with the viral antigens. Armed with these marching orders, the dendritic cells are injected back into the cancer patients, where they head to the lymph nodes and signal the immune fighters to search and attack the CMV-laden tumor. While this immunotherapy worked well, the authors sought additional approaches to boost the immune system. They chose to use a shot of tetanus/diphtheria toxoid — which is widely available and safe as a clinically approved vaccine – to incite the troops of lymphocytes in the lymph nodes.

 

In a small human study, 12 brain tumor patients were randomly assigned to receive a tetanus booster or a placebo injection. The next day, patients in both groups were then given the dendritic cell immunotherapy. Researchers did not know which therapies the patients received. Results showed that patients randomized to get a tetanus shot showed a significant increase in survival from the time of pre-conditioning compared to patients receiving just the dendritic cell therapy, with half living from 51 to 101 months, compared to 11.6 months for the comparison group. One patient from the tetanus group continues to have no tumor growth and is still alive at eight years after the treatment.

 

The authors then used mouse studies to track how the immunotherapy worked. They identified a new role for an immune signaling protein called CCL3, which had previously been known for mediating other immune activities, but had not been associated with creating increased migration of dendritic cells to the lymph nodes. The protein was found to work systemically, not just at the injection site. According to the authors, the fact that both mice given tetanus and the patients had elevated CCL3 prompted the investigation of the role of this protein in the mechanism, which in turn revealed that both CCL3 and the recall responses from tetanus boosting needed to work together to increase the migration of the vaccines. The authors added that while dendritic cell vaccines have shown some promise in the treatment of patients with advanced cancers, including glioblastoma, the dynamics of this process have not been well understood. The authors plan a new study to determine whether successful dendritic cell migration could be used as a prognostic indicator of patient survival since the current\ study indicated that dendritic cell migration to the lymph nodes can be improved significantly by pre-conditioning the vaccine site with a tetanus boost, and this appears to improve antitumor responses and prolonged survival.

 

FDA Approves Drug for High-Risk Neuroblastoma

 

Neuroblastoma is a rare cancer that forms from immature nerve cells. It usually begins in the adrenal glands but may also develop in the abdomen, chest or in nerve tissue near the spine. Neuroblastoma typically occurs in children younger than five years of age. According to the National Cancer Institute, neuroblastoma occurs in approximately one out of 100,000 children and is slightly more common in boys. There are an estimated 650 new cases of neuroblastoma diagnosed in the United States each year. Patients with high-risk neuroblastoma have a 40 to 50% chance of long term survival despite aggressive therapy.

 

The U.S. FDA has approved Unituxin (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma. Unituxin is an antibody that binds to the surface of neuroblastoma cells. Unituxin is being approved for use as part of a multimodality regimen, including surgery, chemotherapy and radiation therapy for patients who achieved at least a partial response to prior first-line multiagent, multimodality therapy.

 

The FDA granted Unituxin priority review and orphan product designation. Priority review shortens the timeframe for review of drug applications by four months, compared to standard reviews, and is granted to drugs that, if approved, will provide a significant improvement in safety or effectiveness in the treatment of a serious condition. Orphan product designation is given to drugs intended to treat rare diseases. With this approval, the FDA also issued a rare pediatric disease priority review voucher to United Therapeutics, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This is the second rare pediatric disease priority review voucher granted by the FDA since inception of the rare pediatric disease review voucher program, which is designed to encourage development of new therapies for prevention and treatment of certain rare pediatric diseases.

 

The safety and efficacy of Unituxin were evaluated in a clinical trial of 226 pediatric participants with high-risk neuroblastoma whose tumors shrunk or disappeared after treatment with multiple-drug chemotherapy and surgery followed by additional intensive chemotherapy and who subsequently received bone marrow transplantation support and radiation therapy. Participants were randomly assigned to receive either an oral retinoid drug, isotretinoin (RA), or Unituxin in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor, which are thought to enhance the activity of Unituxin by stimulating the immune system, and RA. Three years after treatment assignment, 63% of participants receiving the Unituxin combination were alive and free of tumor growth or recurrence, compared to 46% of participants treated with RA alone. In an updated analysis of survival, 73% of participants who received the Unituxin combination were alive compared with 58% of those receiving RA alone.

 

Unituxin carries a Boxed Warning alerting patients and health care professionals that Unituxin irritates nerve cells, causing severe pain that requires treatment with intravenous narcotics and can also cause nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure, during or shortly following completion of the infusion. Unituxin may also cause other serious side effects including infections, eye problems, electrolyte abnormalities and bone marrow suppression. The most common side effects of Unituxin were severe pain, fever, low platelet counts, infusion reactions, low blood pressure, low levels of salt in the blood (hyponatremia), elevated liver enzymes, anemia, vomiting, diarrhea, low potassium levels in the blood, capillary leak syndrome (which is characterized by a massive leakage of plasma and other blood components from blood vessels into neighboring body cavities and muscles), low numbers of infection-fighting white blood cells (neutropenia and lymphopenia), hives, and low blood calcium levels.

 

Unituxin is marketed by Silver Spring, Maryland-based United Therapeutics.

 

World’s Easiest Recipe: Roasted Beets with Melted Cheese(s)

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We started with red wine and a variety of melted cheeses on roasted beets ©Joyce Hays, Target Health Inc.

 

Ingredients

 

1 bunch yellow beets

1 bunch red beets

Assorted cheeses like, port salut, cotswald, feta cheese, mozzarella, boursin, goat cheese with fig, goat cheese with pear, Taleggio, or non-dairy tofutti (soy)

 

 

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Assorted beets, red and yellow ©Joyce Hays, Target Health Inc.

 

Directions

 

1. Preheat oven to 375

2. Cut the greens off and clean the beets

3. Now, roast the beets, whole. You can wrap them in foil if you wish and roast them that way for about 20-30 minutes or whole on a cookie sheet.

4. When soft, but not too soft, take out of oven and take foil off and let them cool.

5. When cool enough, take a paring knife and peel the skin off

6. Now slice the beets and either overlap them in a baking dish and sprinkle with goat cheese or your favorite cheese; or lay the slices out separately in a large baking dish and top with a variety of your favorite cheeses.

7. Put back in the oven, just before you plan to eat them and warm long enough to melt the cheese.

8. Take out and serve immediately

 

 

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Roast whole or wrap in foil ©Joyce Hays, Target Health Inc.

 

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Take out of oven and cool. When cool enough, peel, then slice ©Joyce Hays, Target Health Inc.

 

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Cotswold is an English cheese made with fresh chives

 

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Port Salut is a delicious mild French cheese made from cow’s milk

 

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Italian Taleggio, a creamy cow’s milk cheese

 

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You can overlap the beets in a baking dish and then sprinkle with goat or feta cheese. ©Joyce Hays, Target Health Inc.

 

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Serve right out of the oven, while the cheese is warm and gooey ©Joyce Hays, Target Health Inc.

 

This was a wild week, busy and exhausting. Jules was traveling but catching a plane timed to be home for my birthday dinner. I wanted easy things to make for dinner and had noticed red as well as yellow beets for sale. I loved the color and wondered how I could do something different with the beets. An easy appetizer was what I came up with. Because this was a birthday dinner, we opened a bottle of Stag’s Leap cabernet. Artemis is one of their special wines.

 

We also had a simple salad of tomatoes, avocados and cucumbers and then filet mignon more tender and better than any restaurant with a marsala and mushroom sauce. Tiny roasted micro red potatoes went with it.

 

For dessert we went way, way off our usually sensible diet with one mini red velvet cake and one mini chocolate cake. The slices were tiny but we had a slice from each cake and gave the rest away to our concierge on duty then. We had so-o much fun!

 

But, you ask, “Why did you do everything on your birthday?“ She smiles and simply shows off her fabulous necklace.

 

What can I say; life is good!

 

 

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These beautiful flowers are from our daughter, Vanessa.

 

From Our Table to Yours!

 

Bon Appetit!