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April 16, 2018Target Healthy Eating
1/4 cup flour
Pinch Kosher salt
Pinch black pepper
1 teaspoon dried thyme
2 Tablespoons butter
1.5 pounds choice lamb kabob pieces
4 slices Turkey bacon, well chopped
1 onion, chopped
2 scallions, chopped
3 shallots, chopped
1 green pepper, seeded and diced
1 yellow pepper, seeded, then diced
1 jalapeno, seeded, chopped well
1 cup fresh cilantro, well chopped
2 ribs celery, diced
25 fresh garlic cloves, sliced
2 Tablespoons curry powder
1 teaspoon coriander
3 Tablespoons golden raisins +extra for topping
1 28-ounce can Cento tomatoes chopped and their juices
3 Tablespoons slivered almonds, toasted +extra for topping
Serve over cooked quinoa, my carrot pancake recipe, noodles, my saffron rice recipe, regular basmati white rice, etc.
1. Do all the cutting, slicing chopping, before you do anything else.
2. Toast the almond slivers.
3. In a bowl, combine the flour, salt, black pepper and thyme
4. In a large skillet, melt the butter over medium-high heat until it foams.
5. Dredge the lamb pieces in the flour mixture, and fry, until browned on all sides, about 8 minutes. Put lamb on a plate and set aside.
6. Over medium heat, cook the turkey bacon, until it gets crisp and with a slotted spoon, remove turkey bacon to a plate and set aside.
7. Using the baking dish you plan to bring to the table later, add butter, then the onion, pepper, celery, garlic, curry powder and half the golden raisins, and saut? over medium heat until soft and fragrant, about 7 minutes.
8. Stir in the tomatoes and their juices, bring to a boil and simmer over medium-low heat for 10 minutes.
9. Preheat oven to 350 degrees.
10. Next, add the lamb on top of the tomatoes.
11. Cover and bake for 35 minutes. Remove the cover and cook for 15 minutes more.
12. Add the toasted slivered almonds and the golden raisins as garnish, just before serving. Have mango chutney on the table, or your choice.
Have a great week everyone!
From Our Table to Yours
April 16, 2018Regulatory
Next generation sequencing (NGS) works by looking at a person's DNA to detect genomic variations that may determine whether a person has or is at risk of developing a genetic disease and, in certain cases, may help to inform treatment decisions. Unlike traditional diagnostics that typically detect chemical changes associated with a single disease or condition, NGS can look at millions of DNA changes in a single test to help determine the cause of a person's disease or condition. Availability of these types of tests plays an important role in the advancement of the field of precision medicine.
The FDA has finalized two guidances to drive the efficient development of NGS technology. The guidances provide recommendations for designing, developing, and validating tests that use NGS, and will play an important role in the continued advancement of individualized, genetic-based medicine. The first guidance, Use of Public Human Genetic Variant Databases to Support Clinical Validity for Genetic and Genomic-Based In Vitro Diagnostics, describes an approach where test developers may rely on clinical evidence from FDA-recognized public databases to support clinical claims for their tests and help provide assurance of the accurate clinical evaluation of genomic test results. The guidance describes how product developers can use these databases to support the clinical validation of NGS tests that they are developing. These public databases may include resources like ClinGen, which is maintained by the National Institutes of Health (NIH). Using FDA-recognized databases will provide test developers with an efficient path for marketing clearance or approval of a new test.
The second guidance, "Considerations for Design, Development, and Analytical Validation of Next Generation Sequencing (NGS)-Based In Vitro Diagnostics (IVDs) Intended to Aid in the Diagnosis of Suspected Germline Diseases," provides recommendations for designing, developing, and validating NGS-based tests used to diagnose individuals with suspected genetic diseases. It describes what the FDA would look for in premarket submissions to determine a test's analytical validity, including how well the test detects the presence or absence of a particular genomic change. According to FDA, since information about genetic variants is generally stored in a manner that is not publicly accessible, the release of the FDA's final guidance should help to provide an even more efficient path to market by encouraging data sharing, as well as the accumulation in public databases of evidence supporting the clinical validity of genomic tests.
Issuance of these final guidances is based on extensive feedback from the public and stakeholders who are developing NGS-based technologies, and the guidances serve as a continuation of the FDA's work creating regulatory efficiencies in the development and review of NGS tests. In 2017, the FDA took several actions to streamline the development and review of a variety of genetic-based tests - authorizing a third-party option for conducting reviews NGS tumor profiling tests and making clearance recommendations to FDA, as well as outlining standardized development criteria for carrier screening tests to allow for their marketing without prior agency review. FDA also established such criteria for genetic health risk tests and proposed to allow their marketing after a one-time agency review. As NGS technologies continue to evolve, the FDA remains dedicated to adapting our regulatory review capabilities and leveraging our authorities to the fullest extent in order to make innovative and accurate testing technologies available to patients as efficiently as possible.
April 16, 2018Rare Diseases
"Dripping candle wax" bone disease, is a very rare disorder known as melorheostosis, that causes excess bone formation that resembles dripping candle wax on x-rays. The condition causes pain and bone deformity, which can limit the function of bones. Although there are only about 400 known cases of this disorder worldwide, 15 unrelated adults with the condition from around the globe volunteered to come to the NIH Clinical Center to undergo biopsies of both affected and unaffected bones.in order to uncover a genetic basis of their bone disease. The results, appearing in Nature Communications (11 April 2018), offer potential treatment targets for this rare disease, provide important clues about bone development, and may lead to insights about fracture healing and osteoporosis.
The authors compared samples of healthy and affected bone from each participant to look for differences in the exome, the portion of the genome that codes for proteins. By comparing genetic information from both samples in each patient allowed the team to pinpoint even low levels of the mutations. The analysis revealed that 8 of the 15 participants had mutations in the MAP2K1 gene in the affected bone only. MAP2K1 produces the protein MEK1. The gene MAP2K1 has previously been linked to some types of cancerous growths as well as to conditions that lead to abnormal blood vessel formation in the head, face or neck. In melorheostosis, all the identified MAP2K1 mutations affected a region of the MEK1 protein that normally suppresses its activity, thus they cause MEK1 to become overactive. The bone growth is considered benign and does not spread to other parts of the body.
According to the authors, this is an exciting study of a very rare bone disorder that not only identified the responsible mutation in half of the patients, but uncovered fundamental information about the role of a cancer-related gene in the metabolic pathways of normal bone. The authors added that further studies on how this pathway works in both normal and mutant bone cells may have broad implications that could benefit a wider population. The reasoning is that while most adults have the problem of weakening bones as they grow older, these patients have the opposite problem as some of their bones are rock hard and still growing.
April 16, 2018Neurology
According to an article published online in the Proceedings of the National Academy of Sciences. (9 April 2018), losing just one night of sleep led to an immediate increase in beta-amyloid. The study is among the first to demonstrate that sleep may play an important role in human beta-amyloid clearance. Beta-amyloid is a metabolic waste product present in the fluid between brain cells. In Alzheimer's disease, beta-amyloid proteins clump together to form amyloid plaques, a hallmark of the disease, negatively impacting communication between neurons. While acute sleep deprivation is known to elevate brain beta-amyloid levels in mice, less is known about the impact of sleep deprivation on beta-amyloid accumulation in the human brain.
To understand the possible link between beta-amyloid accumulation and sleep, the authors used positron emission tomography (PET) to scan the brains of 20 healthy subjects, ranging in age from 22 to 72, after a night of rested sleep and after sleep deprivation (being awake for about 31 hours). Results showed that beta-amyloid increased about 5% after losing a night of sleep in brain regions including the thalamus and hippocampus, regions especially vulnerable to damage in the early stages of Alzheimer's disease.
In Alzheimer's disease, beta-amyloid is estimated to increase about 43% in affected individuals relative to healthy older adults. However, it is unknown whether the increase in beta-amyloid in the study participants would subside after a night of rest. Interestingly, the study also found that study participants with larger increases in beta-amyloid reported worse mood after sleep deprivation. According to the authors, even though the sample was small, the study demonstrated the negative effect of sleep deprivation on beta-amyloid burden in the human brain and that future studies are needed to assess the generalizability to a larger and more diverse population. It is also important to note that the link between sleep disorders and Alzheimer's risk is considered by many scientists to be "bidirectional," since elevated beta-amyloid may also lead to sleep disturbances.
April 16, 2018History of Medicine
Graphic credit: From People's Cyclopedia of Universal Knowledge (1883). Transferred from en.wikipedia Original uploader was Whbonney at en.wikipedia, Public Domain, https://commons.wikimedia.org/w/index.php?curid=6693422
Phrenology is a pseudo medicine primarily focused on measurements of the human skull, based on the concept that the brain is the organ of the mind, and that certain brain areas have localized, specific functions or modules. Although both of those ideas have a basis in reality, phrenology extrapolated beyond empirical knowledge in a way that departed from science. Developed by German physician Franz Joseph Gall in 1796, the discipline was very popular in the 19th century, especially from about 1810 until 1840. The principal British center for phrenology was Edinburgh, where the Edinburgh Phrenological Society was established in 1820. Although now regarded as an obsolete amalgamation of primitive neuroanatomy with moral philosophy, phrenological thinking was influential in 19th-century psychiatry. Gall's assumption that character, thoughts, and emotions are located in specific parts of the brain is considered an important historical advance toward neuropsychology.
Phrenologists believe that the human mind has a set of various mental faculties, each one represented in a different area of the brain. For example, the faculty of "philoprogenitiveness", from the Greek for love of offspring, was located centrally at the back of the head (see illustration of the chart from Webster's Academic Dictionary).
These areas were said to be proportional to a person's propensities. The importance of an organ was derived from relative size compared to other organs. It was believed that the cranial skull - like a glove on the hand -accommodates to the different sizes of these areas of the brain, so that a person's capacity for a given personality trait could be determined simply by measuring the area of the skull that overlies the corresponding area of the brain. Phrenology, which focuses on personality and character, is distinct from craniometry, which is the study of skull size, weight and shape, and physiognomy, the study of facial features. Phrenology is a process that involves observing and/or feeling the skull to determine an individual's psychological attributes. Franz Joseph Gall believed that the brain was made up of 27 individual organs that determined personality, the first 19 of these ?organs' he believed to exist in other animal species. Phrenologists would run their fingertips and palms over the skulls of their patients to feel for enlargements or indentations. The phrenologist would often take measurements with a tape measure of the overall head size and more rarely employ a craniometer, a special version of a caliper. In general, instruments to measure sizes of cranium continued to be used after the mainstream phrenology had ended. The phrenologists put emphasis on using drawings of individuals with particular traits, to determine the character of the person and thus many phrenology books show pictures of subjects. From absolute and relative sizes of the skull the phrenologist would assess the character and temperament of the patient.
Gall's list of the "brain organs" was specific. An enlarged organ meant that the patient used that particular "organ" extensively. The number - and more detailed meanings - of organs were added later by other phrenologists. The 27 areas varied in function, from sense of color, to religiosity, to being combative or destructive. Each of the 27 "brain organs" was located under a specific area of the skull. As a phrenologist felt the skull, he would use his knowledge of the shapes of heads and organ positions to determine the overall natural strengths and weaknesses of an individual. Phrenologists believed the head revealed natural tendencies but not absolute limitations or strengths of character. The first phrenological chart gave the names of the organs described by Gall; it was a single sheet, and sold for a cent. Later charts were more expansive.
Historically, among the first to identify the brain as the major controlling center for the body were Hippocrates and his followers, inaugurating a major change in thinking from Egyptian, biblical and early Greek views, which based bodily primacy of control on the heart. This belief was supported by the Greek physician Galen, who concluded that mental activity occurred in the brain rather than the heart, contending that the brain, a cold, moist organ formed of sperm, was the seat of the animal soul - one of three souls found in the body, each associated with a principal organ. The Swiss pastor Johann Kaspar Lavater (1741-1801) introduced the idea that physiognomy related to the specific character traits of individuals, rather than general types, in his Physiognomische Fragmente, published between 1775 and 1778. His work was translated into English and published in 1832 as The Pocket Lavater, or, The Science of Physiognomy. He believed that thoughts of the mind and passions of the soul were connected with an individual's external frame. Of the forehead, When the forehead is perfectly perpendicular, from the hair to the eyebrows, it denotes an utter deficiency of understanding.
In 1796 the German physician Franz Joseph Gall (1758-1828) began lecturing on organology: the isolation of mental faculties and later cranioscopy which involved reading the skull's shape as it pertained to the individual. It was Gall's collaborator Johann Gaspar Spurzheim who would popularize the term phrenology. In 1809 Gall began writing his principal work, The Anatomy and Physiology of the Nervous System in General, and of the Brain in Particular, with Observations upon the possibility of ascertaining the several Intellectual and Moral Dispositions of Man and Animal, by the configuration of their Heads. It was not published until 1819. In the introduction to this main work, Gall makes the following statement in regard to his doctrinal principles, which comprise the intellectual basis of phrenology:
The Brain is the organ of the mind
1. The brain is not a homogenous unity, but an aggregate of mental organs with specific functions
2. The cerebral organs are topographically localized
3. Other things being equal, the relative size of any particular mental organ is indicative of the power or strength of that organ
4. Since the skull ossifies over the brain during infant development, external craniological means could be used to diagnose the internal states of the mental characters
Through careful observation and extensive experimentation, Gall believed he had established a relationship between aspects of character, called faculties, with precise organs in the brain. Johann Spurzheim was Gall's most important collaborator. He worked as Gall's anatomist until 1813 when for unknown reasons they had a permanent falling out. Publishing under his own name Spurzheim successfully disseminated phrenology throughout the United Kingdom during his lecture tours through 1814 and 1815 and the United States in 1832 where he would eventually die. Gall was more concerned with creating a physical science, so it was through Spurzheim that phrenology was first spread throughout Europe and America. Phrenology, while not universally accepted, was hardly a fringe phenomenon of the era. George Combe would become the chief promoter of phrenology throughout the English-speaking world after he viewed a brain dissection by Spurzheim, convincing him of phrenology's merits.
The popularization of phrenology in the middle and working classes was due in part to the idea that scientific knowledge was important and an indication of sophistication and modernity. Cheap and plentiful pamphlets, as well as the growing popularity of scientific lectures as entertainment, also helped spread phrenology to the masses. Combe created a system of philosophy of the human mind that became popular with the masses because of its simplified principles and wide range of social applications that were in harmony with the liberal Victorian world view. George Combe's book On the Constitution of Man and its Relationship to External Objects sold over 200, 000 copies through nine editions. Combe also devoted a large portion of his book to reconciling religion and phrenology, which had long been a sticking point. Another reason for its popularity was that phrenology balanced between free will and determinism. A person's inherent faculties were clear, and no faculty was viewed as evil, though the abuse of a faculty was. Phrenology allowed for self-improvement and upward mobility, while providing fodder for attacks on aristocratic privilege. Phrenology also had wide appeal because of its being a reformist philosophy not a radical one. Phrenology was not limited to the common people, and both Queen Victoria and Prince Albert invited George Combe to read the heads of their children.
Phrenology came about at a time when scientific procedures and standards for acceptable evidence were still being codified. In the context of Victorian society, phrenology was a respectable scientific theory. The Phrenological Society of Edinburgh founded by George and Andrew Combe was an example of the credibility of phrenology at the time and included a number of extremely influential social reformers and intellectuals, including the publisher Robert Chambers, the astronomer John Pringle Nichol, the evolutionary environmentalist Hewett Cottrell Watson, and asylum reformer William A.F. Browne. In 1826, out of the 120 members of the Edinburgh society an estimated one third were from a medical background. By the 1840s there were more than 28 phrenological societies in London with over 1000 members. Another important scholar was Luigi Ferrarese, the leading Italian phrenologist. He advocated that governments should embrace phrenology as a scientific means of conquering many social ills, and his Memorie Risguardanti La Dottrina Frenologica (1836), is considered "one of the fundamental 19th century works in the field".
Traditionally the mind had been studied through introspection. Phrenology provided an attractive, biological alternative that attempted to unite all mental phenomena using consistent biological terminology. Gall's approach prepared the way for studying the mind that would lead to the downfall of his own theories. Phrenology contributed to development of physical anthropology, forensic medicine, knowledge of the nervous system and brain anatomy as well as contributing to applied psychology. John Elliotson was a brilliant but erratic heart specialist who became a phrenologist in the 1840s. He was also a mesmerist and combined the two into something he called phrenomesmerism or phrenomagnatism. Changing behavior through mesmerism eventually won out in Elliotson's hospital, putting phrenology in a subordinate role. Others amalgamated phrenology and mesmerism as well, such as the practical phrenologists Collyer and Joseph R. Buchanan. The benefits of combining mesmerism and phrenology was that the trance the patient was placed in was supposed to allow for the manipulation of his/her penchants and qualities. For example, if the organ of self-esteem was touched, the subject would take on a haughty expression.
Phrenology has been psychology's great faux pas. - J.C. Flugel (1933)
Phrenology was mostly discredited as a scientific theory by the 1840s. This was due only in part to a growing amount of evidence against phrenology. Phrenologists had never been able to agree on the most basic mental organ numbers, going from 27 to over 40, and had difficulty locating the mental organs. Phrenologists relied on cranioscopic readings of the skull to find organ locations. Jean Pierre Flourens' experiments on the brains of pigeons indicated that the loss of parts of the brain either caused no loss of function, or the loss of a completely different function than what had been attributed to it by phrenology. Flourens' experiment, while not perfect, seemed to indicate that Gall's supposed organs were imaginary. Scientists had also become disillusioned with phrenology since its exploitation with the middle and working classes by entrepreneurs. The popularization had resulted in the simplification of phrenology and mixing in it of principles of physiognomy, which had from the start been rejected by Gall as an indicator of personality. Phrenology from its inception was tainted by accusations of promoting materialism and atheism, and being destructive of morality. These were all factors which led to the downfall of phrenology . Recent studies, using modern day technology like Magnetic Resonance Imaging have further disproven phrenology claims.
During the early 20th century, a revival of interest in phrenology occurred, partly because of studies of evolution, criminology and anthropology (as pursued by Cesare Lombroso). The most famous British phrenologist of the 20th century was the London psychiatrist Bernard Hollander (1864-1934). His main works, The Mental Function of the Brain (1901) and Scientific Phrenology (1902), are an appraisal of Gall's teachings. Hollander introduced a quantitative approach to the phrenological diagnosis, defining a method for measuring the skull, and comparing the measurements with statistical averages. In Belgium, Paul Bouts (1900-1999) began studying phrenology from a pedagogical background, using the phrenological analysis to define an individual pedagogy. Combining phrenology with typology and graphology, he coined a global approach known as psychognomy. Bouts, a Roman Catholic priest, became the main promoter of renewed 20th-century interest in phrenology and psychognomy in Belgium. He was also active in Brazil and Canada, where he founded institutes for characterology. His works Psychognomie and Les Grandioses Destinees individuelle et humaine dans la lumiere de la Caracterologie et de l'Evolution cerebro-cranienne are considered standard works in the field. In the latter work, which examines the subject of paleoanthropology, Bouts developed a teleological and orthogenetical view on a perfecting evolution, from the paleo-encephalical skull shapes of prehistoric man, which he considered still prevalent in criminals and savages, towards a higher form of mankind, thus perpetuating phrenology's problematic racializing of the human frame. Bouts died on March 7, 1999. His work has been continued by the Dutch foundation PPP (Per Pulchritudinem in Pulchritudine), operated by Anette Muller, one of Bouts' students. During the 1930's Belgian colonial authorities in Rwanda used phrenology to explain the so-called superiority of Tutsis over Hutus.
April 16, 2018What's New
Graphic credit: By Original concept by w:User:Washington Irving. Current shape by w:User:Mateuszica. Color modified by w:User:Hdante. Text labels by w:User:SAE1962. SVG by User:King of Hearts. - PNG on English Wikipedia, Public Domain, https://commons.wikimedia.org/w/index.php?curid=2221053
Researchers show for the first time that healthy older men and women can generate just as many new brain cells as 1) ___ people. There has been controversy over whether adult humans grow new neurons, and some research has previously suggested that the adult brain was hard-wired and that adults did not grow new 2) ___. This study, which appeared in the journal Cell Stem Cell on April 5, 2018, counters that notion. According to the authors the findings may suggest that many senior citizens remain more cognitively and emotionally intact than commonly believed. Results showed that older people have similar ability to make thousands of hippocampal new neurons from progenitor 3) ___ as younger people do. The study also found equivalent volumes of the hippocampus (a brain structure used for emotion and cognition) across ages. Nevertheless, older individuals had less vascularization and maybe less ability of new neurons to make connections.
For the study, the authors autopsied hippocampi from 28 previously healthy individuals aged 14-79 who had died suddenly. This is the first time it was possible to look at newly formed neurons and the state of blood vessels within the entire human hippocampus soon after 4) ___. The researchers had determined that study subjects were not cognitively impaired and had not suffered from depression or taken antidepressants, which the authors had previously found could impact the production of new brain cells. In rodents and primates, the ability to generate new hippocampal cells declines with 5) ___. Waning production of neurons and an overall shrinking of the dentate gyrus, part of the hippocampus thought to help form new episodic memories, was believed to occur in aging humans as well.
The authors from Columbia University and New York State Psychiatric Institute found that even the oldest brains they studied produced new brain cells. While they found similar numbers of intermediate neural progenitors and thousands of immature neurons, older individuals form fewer new 6) ___ vessels within brain structures and possess a smaller pool of progenitor cells -- descendants of stem cells that are more constrained in their capacity to differentiate and self-renew. The authors surmised that reduced cognitive-emotional resilience in old age may be caused by this smaller pool of neural stem cells, the decline in vascularization, and reduced cell-to-cell connectivity within the hippocampus. The authors hypothesized that it is possible that ongoing hippocampal neurogenesis sustains human-specific cognitive function throughout 7) ___ and that declines may be linked to compromised cognitive-emotional resilience.
The authors feel future research on the aging brain will continue to explore how neural cell proliferation, maturation, and survival are regulated by hormones, transcription factors, and other inter-cellular pathways. Other researchers are focusing on brain evolution and have suggested that there are specific genes that control the size of the human brain. These genes continue to play a role in brain evolution, implying that the brain is continuing to evolve.
The study began with the researchers assessing 214 genes that are involved in brain development. These genes were obtained from humans, macaques, rats and mice. The authors noted points in the DNA sequences that caused protein alterations. These DNA changes were then scaled to the evolutionary time that it took for those changes to occur. The data showed the genes in the human brain evolved much faster than those of the other species. Once this genomic evidence was acquired, the authors decided to find the specific gene or genes that allowed for or even controlled this rapid 8) ___. Two genes were found to control the size of the human brain as it develops. These genes are Microcephalin and Abnormal Spindle-like Microcephaly (ASPM). The researchers at the University of Chicago were able to determine that under the pressures of selection, both of these genes showed significant DNA sequence changes.
Earlier studies displayed that Microcephalin experienced rapid evolution along the primate lineage which eventually led to the emergence of Homo sapiens. After the emergence of humans, Microcephalin seems to have shown a slower evolution rate. On the contrary, ASPM showed its most rapid evolution in the later years of human evolution once the divergence between chimpanzees and humans had already occurred. Each of the gene sequences went through specific changes that led to the evolution of 9) ___ from ancestral relatives. In order to determine these alterations, the authors used DNA sequences from multiple primates then compared and contrasted the sequences with those of humans. Following this step, the researchers statistically analyzed the key differences between the primate and human DNA to come to the conclusion, that the differences were due to natural selection. The changes in DNA sequences of these 10) ___ accumulated to bring about a competitive advantage and higher fitness that humans possess in relation to other primates. This comparative advantage is coupled with a larger brain size which ultimately allows the human mind to have a higher cognitive awareness.
Sources and Researchers: Maura Boldrini, Camille A. Fulmore, Alexandria N. Tartt, Laika R. Simeon, Ina Pavlova, Verica Poposka, Gorazd B. Rosoklija, Aleksandar Stankov, Victoria Arango, Andrew J. Dwork, Ren? Hen, J. John Mann. Human Hippocampal Neurogenesis Persists throughout Aging. Cell Stem Cell, 2018; 22 (4): 589 DOI: 10.1016/j.stem.2018.03.015. Cell Press. "Older adults grow just as many new brain cells as young people." ScienceDaily. ScienceDaily, 5 April 2018. www.sciencedaily.com/releases/2018/04/180405223413.htm; Wikipedia
ANSWERS: 1) younger; 2) neurons; 3) cells; 4) death; 5) age; 6) blood; 7) life; 8) evolution; 9) humans; 10) genes
April 16, 2018What's New
Target Health has extensive experience in Orphan and Rare Diseases including FDA approvals and multiple Orphan Drug Designations.
For those attending the World Orphan Drug Congress, April 25-27 at the Gaylord National Harbor Hotel in Oxon Hill, MD, please reach out to Warren Pearlson, Target's Director of Business Development who will be attending. Warren can meet with you to discuss our Regulatory Strategy and Services in the Orphan space, and delineate how our Full-Service eCRO, supported by Target Health's paperless eSource EDC software platform, enables efficient and cost-effective Clinical Trials.
For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 165). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel. The Target Health software tools are designed to partner with both CROs and Sponsors.
Joyce Hays, Founder and Editor in Chief of On Target
Jules Mitchel, Editor
April 9, 2018Target Healthy Eating
Pancake mix, follow directions on box
5 or 6 carrots, grated
1 teaspoon cinnamon
1 Tablespoon honey
1 Tablespoon tahini (stir the tahini very well before using)
1 Tablespoon vanilla halva
Pinch of nutmeg, freshly grated
1/4 teaspoon apple pie spice
1/4 teaspoon pumpkin pie spice
1. Shred the carrots
2. Mix the batter first
3. Add the shredded carrots and stir into the batter
4. Slowly, add all the other ingredients, stirring as you add
5. Fry the pancakes
Topping for Carrot Pancakes (optional)
1 cup sour cream
2 cooked carrots (save 1/2 cooked carrot for garnish)
1 fresh garlic clove
1 cup plain Greek yogurt
1 and 1/2 Tablespoon golden raisins, mixed
1 and 1/2 Tablespoon golden raisins, well chopped for garnish
3 Tablespoons almond slivers, toasted (1/2 for mixture, 1/2 for garnish)
1/2 teaspoon cardamom
1 teaspoon black sesame seeds, toasted with the almonds
1/2 teaspoon pumpkin spice
2 or 3 Tablespoons honey (add honey last, & taste to see if more honey is needed; if so, add more)
1. First toast the black sesame seeds alone, .and remove from pan. Put all into food processor.
2. Next, toast all the almond slivers, remove from heat when done and divide in half. Set aside half the toasted almonds (for garnish( and put the other half in the food processor.
3. Chop well, 1.5 Tablespoons golden raisins and set aside for garnish.
4. Put the other 1.5 Tablespoons golden raisins into the food processor.
5. Put all ingredients in food processor and pulse until everything is combined well.
6. With a spatula, scrape every bit of this topping out of the food processor and into a small bowl, to be used for the carrot pancakes.
This weekend we took our guests out to the theater and then to The Minetta Tavern, in the Village, for dinner.
Have a great week everyone!
From Our Table to Yours
April 9, 2018Regulatory
The following is based on a press release from Dr. Scott Gottlieb, FDA Commissioner.
Benefit-risk assessment is at the heart of what FDA does to ensure that Americans have access to medical products that are safe, effective and meet their needs. But FDA is also deeply aware that serious chronic illnesses aren't monolithic. Patient perception of the benefits and risks of different treatment options can vary based on the stage of the disease, the age of onset, alternative therapies available to treat the disease (if any) and whether a novel therapy improves a patient's ability to function normally, slows the rate of disease progression or impacts other aspects of a patient's quality of life.
A 45-year-old father of two who is diagnosed with aggressive prostate cancer may have very different goals than an 80-year-old man diagnosed with the same disease. To address these realities, FDA will continue to work in close partnership with patients to incorporate their experience into FDA's benefit-risk assessments. First-hand knowledge of living with a serious illness - communicated in science-based terms that patients value and understand - is integral to facilitating the successful development of safe and effective products that can deliver meaningful benefits in each disease, or disease state.
Today there are many more tools to measure these patient benefits - including wearable devices, medical apps and even machine-learning programs. These tools can bring a better understanding of how patients experience their illness, including how it affects their day-to-day feeling or functioning and how a given treatment may impact the course of that illness. Tools for capturing the patient experience may be quantitative or qualitative, but they are transforming nearly every aspect of medical product development. Patients can teach all of us about the benefits that matter most to them and the risks that they are most concerned about. Patients are, rightly so, becoming the driving force of the medical research enterprise.
Structured and Transparent Benefit-Risk Assessments
FDA's ongoing work to enhance their benefit-risk assessment and communication in the human drug review process began in 2013 as part of the Prescription Drug User Fee Act (PDUFA) V. The priority to enhance benefit-risk assessment has continued with new efforts begun in 2017, as part of PDUFA VI and further expanded under 21st Century Cures. Implementing these key pieces of legislation is improving clarity and consistency in communicating the reasoning behind the FDA's drug regulatory decisions. It's also helping integrate the patient's perspective into drug development and regulatory decision-making. FDA has issued an update to their implementation plan, titled Benefit-Risk Assessment in Drug Regulatory Decision-Making. This document provides an overview of the steps the FDA has taken since 2013 to enhance benefit-risk assessment in human drug review, which included implementation of the FDA's Benefit-Risk Framework into our drug regulatory review processes and documentation. This document also provides a roadmap for enhancing the Benefit-Risk Framework, working toward a goal of providing guidance by June 2020 that articulates the FDA's decision-making context and framework for benefit-risk assessment. This forthcoming guidance will also outline how patient experience data and related information can be used to inform benefit-risk assessment.
In order for a drug or biologic product to be approved, the FDA conducts a comprehensive analysis of all available data to determine if the drug is effective and that its expected benefits outweigh its potential risks. This assessment is fundamental to our regulatory process. The goal of the FDA's Benefit-Risk Framework is to improve the clarity and consistency in communicating the reasoning behind drug regulatory decisions, and ensure that the FDA reviewers' detailed assessments can be readily understood in the broader context of patient care and public health. The structured framework also helps drug sponsors and other external stakeholders better understand the factors that contribute to the FDA's decision-making process when evaluating new drugs, including drugs under development. A standard Benefit-Risk Framework will also better ensure that the patient community can continue to engage effectively with the agency, and help FDA improve how it evaluates benefits and risks from the patient's perspective. The Benefit-Risk Framework has been applied in FDA reviews of novel drugs and biologics over the past few years, and FDA is now using it more broadly.
Incorporating Patient Voice into Benefit-Risk Assessments
The Benefit-Risk Framework recognizes that when FDA reviewers conduct a benefit-risk assessment, they consider not only the submitted evidence related to the benefit and risk and effects reported in clinical studies, but also, importantly, the clinical context of the disease. This context encompasses two major considerations: 1) an analysis of the disease condition, including the severity of the condition; and 2) the degree of unmet medical need. As part of this work, the FDA recognizes a need to learn about the clinical context more comprehensively and directly from the perspective of the patients who live with the disease and their caregivers. After conducting patient-focused drug development meetings in over 20 disease areas, the FDA has concluded that patient input can: 1) inform the clinical context and provide insights to frame the assessment of benefits and risk; and 2) provide a direct source of evidence regarding the benefits and risks, if methodologically-sound data collection tools could be developed and used within clinical studies of an investigational therapy. The FDA is now developing guidance to enable more widespread development of such patient experience data to inform regulatory decision-making, as part of our implementation of PDUFA VI and 21st Century Cures. Other efforts to more systematically incorporate patients' experiences and perspectives include:
1. Hosting patient-focused drug development public meetings to advance a more systematic way of gathering patients' perspectives on their conditions and available treatments;
2. Encouraging patient stakeholders and others to conduct their own externally-led, patient-focused drug development meetings;
3. Providing patients, caregivers, advocates and others with more channels to provide meaningful input into drug development and regulatory decision-making, and to more easily access information provided by others; and
4. Launching pilot programs - and advancing policies, in collaboration with the medical community - that help foster the design of clinical trials that place less burden on patients.
The benefit-risk implementation plan issued today is part of the FDA's ongoing commitment to advancing its mission of protecting and promoting public health. It marks another important step forward in increasing transparency of FDA decisions as well as streamlining the process by which FDA obtains input from the patient and stakeholder communities. In the battle against disease, engaged and informed patients are our best allies and one of the greatest resources.
April 9, 2018Women's Health
According to a study published in the journal Hypertension (2 April 2018), elevated blood pressure before conception may increase the chances for pregnancy loss. According to the authors, lifestyle changes to keep blood pressure under control could potentially reduce the risk of loss. T. The analysis found that for every 10 mmHg increase in diastolic blood pressure (pressure when the heart is resting between beats), there was an 18% higher risk for pregnancy loss among the study population. Millimeter of mercury, or mmHg, is the unit of measure used for blood pressure. The authors also found a 17% increase in pregnancy loss for every 10 mmHg increase in mean arterial pressure, a measure of the average pressure in the arteries during full heart beat cycles.
The authors analyzed data collected as part of the Effects of Aspirin in Gestation and Reproduction (EAGeR) trial, which sought to determine if daily low-dose aspirin (81 milligrams) could prevent miscarriage in women who had a history of pregnancy loss. The trial enrolled more than 1,200 women ages 18 to 40 years and took blood pressure readings before the women were pregnant and again in the fourth week of pregnancy. Average diastolic blood pressure for the women in the study was 72.5 mmHg; normal blood pressure in adults is a diastolic reading of below 80 mmHg. The authors began to see an increase in pregnancy loss among women who had a diastolic reading above 80 mmHg (approximately 25% of the participants). None of the women in the study had stage II high blood pressure (above 90 mmHg in diastolic high blood pressure or above 140 mmHg in systolic blood pressure).
The authors cautioned that the study does not prove that elevated blood pressure causes pregnancy loss. It is possible that another, yet-to-be identified factor could account for the findings. They added, however, that the relationship between preconception blood pressure and pregnancy loss remained the same when they statistically accounted for other factors that could increase pregnancy loss, such as increasing maternal age, higher body mass index or smoking.