Dr. Jules Mitchel Again a PharmaVOICE 100 – This Time Commander & Chief
Dr. Jules Mitchel, President of Target Health was again chosen as a PharmaVOICE 100, this year as a Commander & Chief.The PharmaVOICE 100 was debuted in 2005 to recognize outstanding leaders across all sectors of healthcare who provide the guiding light of inspiration, motivation, and innovation to their teams, organizations, and communities. Over the past 10 years, the PharmaVOICE 100 has honored more than 1,000 of the industry’s most inspirational and innovative leaders, and this year. The Inaugural PharmaVOICE 100 Celebration, will be held on September 17, 2015 at the Alexandria Center for Life Science in NYC. The event is open to everyone in the industry and there is limited capacity. In keeping with the philanthropic spirit of the PharmaVOICE 100, please note that a portion of proceeds will be donated to charity.
Can You Find The Hat!!!
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, and if you like the weekly newsletter, ON TARGET, you’ll love the Blog.
Joyce Hays, Founder and Editor in Chief of On Target
Jules Mitchel, Editor
The Legacy of Civil War Medicine (test your knowledge of medical history)
Walt Whitman remarked on the plethora of hospitals around Washington D.C., calling them grim clusters. (Library of Congress)
During the American Civil War, both sides were devastated by battle and disease. Nurses, surgeons, and physicians rose to the challenge of healing a nation and advanced medicine into the modern age. From the stench of putrefying flesh wafting through unsanitary and crowded camps to the unglamorous illnesses of syphilis and dysentery, our modern disgust toward Civil War medical practices is generally justified. However, while advanced or hygienic may not be terms attributed to medicine in the 19th century, modern hospital practices and treatment methods owe much to the legacy of Civil War medicine. Of the approximately 620,000 soldiers who died in the war, two-thirds of these deaths were not the result of enemy fire, but of a force stronger than any army of men: 1) ___. Combating disease as well treating the legions of wounded soldiers pushed Americans to rethink their theories on health and develop efficient practices to care for the sick and wounded.
At the beginning of the Civil War, medical equipment and knowledge was hardly up to the challenges posed by the wounds, infections and diseases which plagued millions on both sides. Illnesses like dysentery, 2) ___ fever, pneumonia, mumps, measles and tuberculosis spread among the poorly sanitized camps, felling men already weakened by fierce fighting and meager diet. Additionally, armies initially struggled to efficiently tend to and transport their wounded, inadvertently sacrificing more lives to mere disorganization.
Wounded soldiers gather outside of a field hospital after Battle of the Wilderness in May of 1864. (Library of Congress)
For medical practitioners in the field during the Civil War, 3) ___ theory, antiseptic (clean) medical practices, advanced equipment, and organized hospitalization systems were virtually unknown. Medical training was just emerging out of the heroic era, a time where physicians advocated bloodletting, purging, blistering (or a combination of all three) to rebalance the humors of the body and remedy the sick. Physicians were also often encouraged to treat diseases like syphilis with 4) ___, a toxic treatment, to say the least. These aggressive remedies of the heroic era of medicine were often worse than patients’ diseases; those who overcame illness during the war owed their recoveries less to the ingenuity of contemporary medicine than to grit and chance. Luck was a rarity in camps where poor sanitation, bad hygiene and diet bred disease, infection, and death. The wounded and sick suffered from the haphazard hospitalization systems that existed at the start of the Civil War. As battles ended, the wounded were rushed down railroad lines to nearby cities and towns, where doctors and nurses coped with the onslaught of dying men in makeshift hospitals. These hospitals saw a great influx of wounded from both sides and the wounded and dying filled the available facilities. The Fairfax Seminary, for example, opened its doors twenty years prior to the war with only 14 students, but it housed an overwhelming 1,700 sick and wounded soldiers during the course of the war.
Fairfax Seminary, in Alexandria, Virginia served as a makeshift hospital for the Army of the Potomac. (Library of Congress)
On his many tours of these improvised hospitals, the great American poet and Civil War nurse Walt Whitman noted in his Memoranda during the War the disorderly death and waste of early Civil War medicine. At the camp hospital of the Army of the Potomac in Falmouth, Virginia in 1862, Whitman saw a heap of amputated feet, legs, arms, hands, &c, a full load for a one-horse cart and several dead bodies lying near. Of the hospital itself, which was a brick mansion before the battle of Fredericksburg changed its use, Whitman observed that it was quite crowded, upstairs and down, everything impromptu, no system, all bad enough, but I have no doubt the best that can be done; all the wounds pretty bad, some frightful, the men in their old clothes, unclean and bloody. Of the division hospitals, Whitman noted that these were merely tents, and sometimes very poor ones, the wounded lying on the ground, lucky if their blankets are spread on layers of pine or hemlock twigs or small leaves. However, the heavy and constant demands of the sick and wounded sped up the technological progression of medicine, wrenching American medical practices into the light of modernity. Field and pavilion hospitals replaced makeshift ones and efficient hospitalization systems encouraged the accumulation of 5) ___records and reports, which slowed bad practices as accessible knowledge spread the use of beneficial treatments.
A medical kit during the Civil War, with scissors, gauze and needles. (Tria Giovan/CORBIS)
Several key figures played a role in the progression of medicine at this time. Jonathan Letterman, the Medical Director of the Army of the Potomac, brought order and efficiency in to the Medical Service with a regulated ambulance system and evacuation plans for the wounded. As surgeon general of the Union army, William A. Hammond standardized, organized and designed new hospital layouts and inspection systems and literally wrote the book on hygiene for the army. Clara 6) ___, well-known humanitarian and founder of the American Red Cross, brought professional efficiency to soldiers in the field, especially at the Battle of Antietam in September of 1862 when she delivered much-needed medical supplies and administered relief and care for the wounded. Disease and illness took a heavy toll on soldiers, but as these historic characters show, every effort was made to prevent death caused by human error and ignorance through the development of organized and more advanced practices. The sheer quantity of those who suffered from disease and severe wounds during the Civil War forced the army and medical practitioners to develop new therapies, technologies and practices to combat 7) ___. Thanks to Hammond’s design of clean, well ventilated and large pavilion-style hospitals, suffering soldiers received care that was efficient and sanitary. In the later years of the war, these hospitals had a previously unheard of 8% mortality rate for their patients.
An ambulance drill at the Army of Potomac Headquarters near Brandy Station, Va., March, 1864. (Library of Congress)
Though the mortality rate was higher for soldiers wounded on the battlefield, field dressing stations and field hospitals administered care in increasingly advanced ways. Once a soldier was wounded, medical personnel on the battlefield bandaged the soldier as fast as they could, and gave him whiskey (to ease the shock) and 8) ___, if necessary, for pain. If his wounds demanded more attention, he was evacuated via Letterman’s ambulance and stretcher system to a nearby field hospital. Under Hammond and Letterman’s encouragement of triage organization that is still used today, field hospitals separated wounded soldiers into three categories: mortally wounded, slightly wounded and surgical cases. Most of the amputations performed at field hospitals were indeed horrible scenes, but the surgery itself was not as crude as popular memory makes it out to have been. Anesthetics were readily available to surgeons, who administered chloroform or 9) ___to patients before the procedure. Though gruesome, amputation was a life-saving procedure that swiftly halted the devastating effects of wounds from Minie balls.
By the end of the war, newly designed, well-ventilated hospitals decreased the mortality rate of the sick and wounded. (Library of Congress)
In field hospitals and pavilion-style hospitals, thousands of physicians received experience and training. As doctors and nurses became widely familiar with prevention and treatment of 10) ___ diseases, anesthetics, and best surgical practices, medicine was catapulted into the modern era of quality care. Organized relief agencies like the 1861 United States Sanitary Commission dovetailed doctors’ efforts to save wounded and ill soldiers and set the pattern for future organizations like the American Red Cross, founded in 1881. Death from wounds and disease was an additional burden of the war that took a toll on the hearts, minds, and bodies of all Americans, but it also sped up the progression of medicine and influenced practices the army and medical practitioners still use today. While the Union had the advantage of better medical supplies and manpower, both Rebels and Federals attempted to combat illness and improve medical care for their soldiers during the war. Many of America’s modern medical accomplishments have their roots in the legacy of America’s Civil War.Sources: Civil War.org; Wikipedia
ANSWERS: 1) disease; 2) typhoid; 3) germ; 4) mercury; 5) medical; 6) Barton; 7) death; 8) morphine; 9) ether; 10) infectious
Mary Edwards Walker MD (1832-1919)
Walker with Her Medal of Honor
Mary Edwards Walker was an American feminist, abolitionist, prohibitionist, alleged spy, prisoner of war and surgeon. As of 2015, she is the only woman ever to receive the Medal of Honor. In 1855, Walker earned her medical degree at Syracuse Medical College in New York, married and started a medical practice. The practice didn’t do well and she volunteered with the Union Army at the outbreak of the American Civil War and served as a surgeon at a temporary hospital inside the capitol. Women and sectarian physicians were not even considered for the Union Army Examining Board because they were considered unfit, let alone someone who met both of those qualifications. She was captured by Confederate forces after crossing enemy lines to treat wounded civilians and arrested as a spy. She was sent as a prisoner of war to Richmond, Virginia until released in a prisoner exchange.
After the war, she was approved for the highest United States Armed Forces decoration for bravery, the Medal of Honor, for her efforts during the Civil War. She is the only woman to receive the medal and one of only eight civilians to receive it. Her name was deleted from the Army Medal of Honor Roll in 1917 and restored in 1977. After the war, she was a writer and lecturer supporting the women’s suffrage movement until her death in 1919.
Walker was born in the Town of Oswego, New York, on November 26, 1832, the daughter of Alvah (father) and Vesta (mother) Walker. She was the youngest of seven children. She had five sisters and one brother. Walker worked on her family farm as a child. She did not wear women’s clothing during farm labor, because she considered them too restricting. Her mother reinforced her view on corsets and tight lacings as being unhealthy. Her elementary education consisted of going to the local school where her mother taught. As a young woman, she taught at the school to earn enough money to pay her way through Geneva Medical College (now State University of New York Upstate Medical University), where she graduated as a medical doctor in 1855 as the only woman in her class.
Walker married a fellow medical school student, Albert Miller, and they set up a joint practice in Rome, New York. The practice did not flourish, as female physicians were generally not trusted or respected at that time. They later divorced. Walker briefly attended Bowen Collegiate Institute (later named Lenox College) in Hopkinton, Iowa in 1860, until she was suspended after refusing to quit the school debating society, which had previously been all male.
At the beginning of the American Civil War, she volunteered for the Union Army as a civilian. At first, she was only allowed to practice as a nurse, as the U.S. Army had no female surgeons. During this period, she served at the First Battle of Bull Run (Manassas), July 21, 1861 and at the Patent Office Hospital in Washington, D.C. She worked as an unpaid field surgeon near the Union front lines, including the Battle of Fredericksburg and in Chattanooga after the Battle of Chickamauga. As a suffragette, she was happy to see women serving as soldiers and alerted the press to the case of Frances Hook in Ward 2 of the Chattanooga hospital. In September 1862, Walker wrote to the War Department requesting employment on Secret Service to spy on the enemy, but the offer was declined. Finally, she was employed as a Contract Acting Assistant Surgeon (civilian) by the Army of the Cumberland in September 1863, becoming the first-ever female surgeon employed by the U.S. Army Surgeon. Walker was later appointed assistant surgeon of the 52nd Ohio Infantry. During her service, she frequently crossed battle lines, treating civilians. On April 10, 1864, Walker was captured by Confederate troops and arrested as a spy, just after she finished helping a Confederate doctor perform an amputation. She was sent to Castle Thunder in Richmond, Virginia and remained there until August 12, 1864, when she was released as part of a prisoner exchange. While she was imprisoned, she refused to wear the clothes provided because they were more becoming of her gender. Walker was exchanged for a Confederate surgeon from Tennessee on August 12, 1864. She then went on to serve during the Battle of Atlanta and later as supervisor of a female prison in Louisville, Kentucky, and head of an orphanage in Tennessee. After serving in the war, Walker became disabled. Walker was awarded a disability pension for partial muscular atrophy while she was imprisoned during the war. She was given $8.50 a month beginning June 13, 1865, but in 1899 it was raised to $20 per month.
Walker became a writer and lecturer, supporting such issues as health care, temperance, women’s rights and dress reform for women. She was frequently arrested for wearing masculine styled clothing and insisted on her right to wear clothing that she thought appropriate. She wrote two books that discussed women’s rights and dress. Mary Edwards Walker was a supporter of the women’s suffrage movement. She was a member of the central woman’s suffrage Bureau in Washington. During her time as a member, she solicited funds to endow a chair in the medical school at Howard University to be filled by a woman professor. Walker attempted to register to vote in 1871, but was turned away. The initial stance of the movement, taking Dr. Walker’s lead, was to claim that women already had the right to vote, and Congress need only enact enabling legislation. After a number of fruitless years taking this stance, the movement took the new tack of working for a constitutional amendment. This was diametrically opposed to Mary Walker’s position, and she fell out of favor with the movement. She continued to attend conventions of the suffrage movement and distribute her own brand of literature, but was virtually ignored by the rest of the movement. Her penchant for wearing male-style clothing, including a top hat, only exacerbated the situation. She received a more positive reception in England than in the United States. In 1907, Walker published a work on Crowning Constitutional Argument to state her views. Walker argued that some states, as well as the Constitution, had already granted women the right to vote. She testified on women’s suffrage in 1912 and 1914 before the U.S. House of Representatives.
Walker died on February 21, 1919, from natural causes at the age of 86 and is buried in Rural Cemetery Oswego, New York. She had a plain funeral, but an American flag was draped over her casket and she was buried in her black suit instead of a dress. Her death in 1919 came one year before the passage of the Nineteenth Amendment to the United States Constitution, which guaranteed women the right to vote.
Dr. Mary Edwards Walker, around 1911
After the war, Walker was recommended for the Medal of Honor by Generals William Tecumseh Sherman and George Henry Thomas. On November 11, 1865, President Andrew Johnson signed a bill to present her the medal. In 1917, the U.S. Congress created a pension act for Medal of Honor recipients and in doing so created separate Army and Navy Medal of Honor Rolls. Only the Army decided to review eligibility for inclusion on the Army Medal of Honor Roll. The 1917 Medal of Honor Board deleted 911 names from the Army Medal of Honor Roll, including those of Dr. Mary Edwards Walker and William F. Buffalo Bill Cody. None of the 911 recipients were ordered to return their medals, although on the question of whether the recipients could continue to wear their medals, the Judge Advocate General advised the Medal of Honor Board that there was no obligation on the Army to police the matter. Walker continued to wear her medal until her death. President Jimmy Carter restored her medal posthumously in 1977. She was one of six people to regain the award.
Walker felt like she was awarded the Medal of Honor because she went into enemy territory to care for the suffering inhabitants when no man had the courage to respond in fear of being imprisoned. She had no fear of being imprisoned; resulting in her doing what her calling was, which was a doctor.
A List of Dr. Walker’s Awards
Rank and organization: Contract Acting Assistant Surgeon (civilian), U. S. Army. Places and dates: Battle of Bull Run, July 21, 1861; Patent Office Hospital, Washington, D.C., October 1861; Chattanooga, Tenn., following Battle of Chickamauga, September 1863; Prisoner of War, April 10, 1864 – August 12, 1864, Richmond, Va.; Battle of Atlanta, September 1864. Entered service at: Louisville, Ky. Born: 26 November 1832, Oswego County, N.Y.
Citation: Whereas it appears from official reports that Dr. Mary E. Walker, a graduate of medicine, has rendered valuable service to the Government, and her efforts have been earnest and untiring in a variety of ways, and that she was assigned to duty and served as an assistant surgeon in charge of female prisoners at Louisville, Ky., upon the recommendation of Major-Generals Sherman and Thomas, and faithfully served as contract surgeon in the service of the United States, and has devoted herself with much patriotic zeal to the sick and wounded soldiers, both in the field and hospitals, to the detriment of her own health, and has also endured hardships as a prisoner of war four months in a Southern prison while acting as contract surgeon; and Whereas by reason of her not being a commissioned officer in the military service, a brevet or honorary rank cannot, under existing laws, be conferred upon her; and Whereas in the opinion of the President an honorable recognition of her services and sufferings should be made. It is ordered, That a testimonial thereof shall be hereby made and given to the said Dr. Mary E. Walker, and that the usual medal of honor for meritorious services be given her.
Dr. Walker was inducted into the National Women’s Hall of Fame in 2000.
1. During World War II, a Liberty ship, the SS Mary Walker, was named for her.
2. In 1982, the U.S. Postal Service issued a 20-cent stamp in her honor marking the anniversary of her birth.
3. The medical facilities at SUNY Oswego are named in her honor (Mary Walker Health Center). On the same grounds a plaque explains her importance in the Oswego community.
4. There is a United States Army Reserve center named for her in Walker, Michigan.
The Whitman-Walker Clinic in Washington, D.C. is named in honor of Dr. Walker and the poet Walt Whitman who was a nurse in D.C. during the Civil War.
5. The Mary Walker Clinic at the National Training Center and Fort Irwin, California is named in honor of Dr. Walker.
6. The Mary E. Walker House is a 30-bed transitional residence run by the Philadelphia Veterans Multi-Service & 7. Education Center for women veterans who have found themselves in difficult life situations and without a home.
8. With the Civil War anniversary (2011-2015), a number of historians have had a renewed interest in Walker, especially historian Mercedes Graf, who wrote the introduction to Walker’s Hit for the 2003 edition.
Calorie Restriction May Lower Risk Factors for Age-Related Diseases
Calorie restriction is a reduction in calorie intake without deprivation of essential nutrients. It has been shown to increase longevity and delay the progression of a number of age-related diseases in multiple animal studies. Called Comprehensive Assessment of Long-term Effects of Reducing Intake of Energy (CALERIE), this randomized trial provides some of the first clues about the impact of sustained calorie restriction in adults. Results from a two-year clinical trial show calorie restriction in normal-weight and moderately overweight people failed to have some metabolic effects found in laboratory animal studies. However, the study found calorie restriction modified risk factors for age-related diseases and influenced indicators associated with longer life span, such as blood pressure, cholesterol, and insulin resistance. The study was reported online in the September, 2015 issue of the Journal of Gerontology Medical Sciences V.70:1097-1104).
In laboratory animals, calorie restriction’s favorable effects on life span have generally been found when it is begun in youth or early middle age. An equivalent trial in humans would take decades. However, shorter trials can determine feasibility, safety and effects on quality of life, disease risk factors, predictors of life span and effects on mechanisms influenced by calorie restriction in laboratory animal studies. CALERIE was a two-year randomized controlled trial in 218 young and middle-aged healthy normal-weight and moderately overweight men and women to measure these outcomes in a CR group, compared with a control group who maintained their regular diets. For the study, the calorie restriction participants were given weight targets of 15.5% weight loss in the first year, followed by weight stability over the second year. This target was the weight loss expected to be achieved by reducing calorie intake by 25% below one’s regular intake at the start of the study. The calorie restriction group lost an average of 10% of their body weight in the first year, and maintained this weight over the second year. While the weight loss fell short of the target, it is the largest sustained weight loss reported in any dietary trial in non-obese people. The participants achieved substantially less calorie restriction (12%) than the trial’s 25% goal, but maintained calorie restriction over the entire two-year period. The control group’s weight and calorie intake were stable over the period.
The study was designed to test the effects of calorie restriction on resting metabolic rate (after adjusting for weight loss) and body temperature, which are diminished in many laboratory animal studies and have been proposed to contribute to its effects on longevity. The study found a temporary effect on resting metabolic rate, which was not significant at the end of the study, and no effect on body temperature. Although the expected metabolic effects were not found, calorie restriction significantly lowered several predictors of cardiovascular disease compared to the control group, decreasing average blood pressure by 4% and total cholesterol by 6%. Levels of HDL (good) cholesterol were increased. Calorie restriction caused a 47% reduction in levels of C-reactive protein, an inflammatory factor linked to cardiovascular disease. It also markedly decreased insulin resistance, which is an indicator of diabetes risk. T3, a marker of thyroid hormone activity, decreased in the calorie restriction group by more than 20%, while remaining within the normal range. This is of interest since some studies suggest that lower thyroid activity may be associated with longer life span.
The study also assessed calorie restriction’s effects on mood (particularly hunger-related symptoms) and found no adverse effects. No increased risk of serious adverse clinical events was reported. However, a few participants developed transient anemia and greater-than-expected decreases in bone density given their degree of weight loss, reinforcing the importance of clinical monitoring during calorie restriction.
In Uveitis, Bacteria in the Gut May Instruct Immune Cells to Attack the Eye
The eye is one of the places in the body that has immune privilege meaning it is protected by a blood-tissue barrier that physically separates it from the rest of the body and minimizes the exchange of substances and blood-borne cells going in and out of the eye.
Uveitis is swelling and irritation of the uvea, the middle layer of the eye. The uvea provides most of the blood supply to the retina. Uveitis can be caused by autoimmune disorders, including rheumatoid arthritis or ankylosing spondylitis. It can also be caused by infection or exposure to toxins. In many cases, the cause is unknown. The most common form of uveitis is anterior uveitis. This involves inflammation in the front part of the eye. It is often called iritis because it usually only affects the iris, the colored part of the eye. The inflammation may be linked with autoimmune diseases, but most cases occur in healthy people. The disorder may affect only one eye. It is most common in young and middle-aged people. Autoimmune uveitis accounts for more than 10% of severe visual disability in the United States. Corticosteroids provide a blanket approach to the disorder by quelling inflammation, but their long-term use can lead to adverse side effects.
Autoimmune uveitis occurs when a person’s immune system goes awry, attacking proteins in the eye. What spurs this response is a mystery, but now a study on mice suggests that bacteria in the gut may provide a kind of training ground for immune cells to attack the eye. Evidence increasingly suggests that there is an association between the microbiota in the gut – bacteria, fungi and viruses – and the development of autoimmune disorders. Findings from this study suggest how that association may be made and therefore have implications about the origins of autoimmune diseases not only in the eye, but also elsewhere in the body.
In the case of autoimmune uveitis, immune cells (T cells) are thought to penetrate through this blood-ocular barrier. But first, they must become activated, which occurs when they come in contact with the protein that they are pre-programmed to recognize. This is how T cells fight an infection and some types of cancer — by targeting proteins on bacteria, viruses and cells. And herein lies a paradox that’s been puzzling uveitis researchers. The proteins believed to be targeted in autoimmune uveitis are sequestered in the eye; they don’t exist elsewhere in the body. So what activates the T cells and allows them to cross the blood-ocular barrier?
The authors asked this question by studying mice genetically engineered to develop autoimmune uveitis, due to a high level of retina-reactive T cells in their bodies. Before the mice had developed signs of the disease, the authors searched their bodies for activated T cells and made an interesting discovery that levels of activated T cells were not elevated in the lymph nodes (the glands that tend to swell during infections), but they were abundant in the intestines. What’s more, the T cells in the gut produced a protein shown in previous studies by the authors to augment the damage in autoimmune uveitis.
According to the authors, these discoveries support the idea that activation of T cells in the gut may actually precede the first signs of the disease. To test that idea, the authors gave the mice an antibiotic cocktail designed to wipe out a broad spectrum of bacteria in the gut and by rearing them in a germ-free environment. When this was done it was found that mice without gut bacteria developed autoimmune uveitis much later, and with less severity, compared to control mice with normal gut flora. There was also a similar delay in uveitis and decline in its severity when the uveitis-prone mice were raised in an environment free of bacteria and other germs. But when the same mice were later moved into normal housing, where they acquired normal gut bacteria, the uveitis roared in at full strength.
So how do bacteria in the gut activate T cells against cells in the eye? The authors hypothesized that bacteria in the gut produce a molecule that, to T cells, looks similar to a protein in the retina. This gives the T cells marching orders to look for that retinal protein and attack it. Consistent with this idea, the authors found that they could activate retina-specific T cells by exposing them to a soup of bacterial proteins extracted from mouse intestines. When those activated T cells were injected into normal mice (not prone to uveitis), the mice developed uveitis. The results don’t have immediate implications for patients, but will help inform further research to understand the disease and help to develop new therapies. Eliminating bacteria from our bodies isn’t a treatment option, and a bacteria-free state would not be feasible. However, if scientists could one day identify the bacteria specifically involved in promoting autoimmune uveitis, it might be possible to target only those of interest.
New Orphan Drug Approved to Treat Rare Autosomal Recessive Disorder
Target Health is very active in the Orphan Drug Space with FDA marketing approvals.
The U.S. Food and Drug Administration has approved Xuriden (uridine triacetate), the first FDA-approved treatment for patients with hereditary orotic aciduria.
Hereditary orotic aciduria is inherited from a recessive gene. The disease is due to a defective or deficient enzyme, which results in the body being unable to normally synthesize uridine, a necessary component of ribonucleic acid (RNA). Signs and symptoms of the disease include blood abnormalities (anemia, decreased white blood cell count, decreased neutrophil count), urinary tract obstruction due to the formation of orotic acid crystals in the urinary tract, failure to thrive, and developmental delays. Hereditary orotic aciduria is a rare metabolic disorder, which has been reported in approximately 20 patients worldwide.
The FDA granted Xuriden orphan drug designation because it treats a rare disease. Orphan drug designation provides financial incentives, like clinical trial tax credits, user fee waivers, and eligibility for market exclusivity to promote rare disease drug development. Xuriden was also granted priority review. An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment. The manufacturer of Xuriden was granted a rare pediatric disease priority review voucher – a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
Xuriden is an orally administered product intended to replace uridine. Xuriden is approved as oral granules that can be mixed with food or in milk or infant formula, and is administered once daily.
The safety and effectiveness of Xuriden were evaluated over six-weeks in an open-label trial in four patients with hereditary orotic aciduria, ranging in age from three to 19 years of age, and in a six-month extension phase of the trial. The study assessed changes in the patients’ pre-specified hematologic parameters during the trial period. At both the six-week and six-month assessments, Xuriden treatment resulted in stability of the hematologic parameters in all four clinical trial patients. The safety and effectiveness of uridine replacement therapy were further supported by case reports from the published literature. No side effects were observed in patients treated with Xuriden for up to nine months.
Xuriden is marketed by Wellstat Therapeutics Corporation, based in Gaithersburg, Maryland.
Strawberries & Cream Jello Cake
A luscious dessert ©Joyce Hays, Target Health Inc.
All summer long we had this cool, very tasty dessert which we slathered with no-fat Cool Whip. The simple ingredients are shown in the photo below.
Simple ingredients and very few. ©Joyce Hays, Target Health Inc.
1 or 2 containers of Tofutti, depending on how many calories you want to eat
2 boxes sugar-free strawberry jello
1 pint fresh blueberries
15 to 20 fresh strawberries
12 ladyfingers, or biscotti or more to your taste, crumbled
Cool whip to add to your taste, as topping
1 springform cake pan
Into your springform cake pan crumble all the ladyfingers or biscotti. They are to be the bottom layer
In the process of crumbling ladyfingers ©Joyce Hays, Target Health Inc.
Fresh strawberries were wonderful this summer and fresh blueberries were spectacular ©Joyce Hays, Target Health Inc.
Follow the directions on one box of strawberry jello, boil water, stir in the powdered jello, etc. Then pour it into a measuring cup.
Jello in measuring cup ©Joyce Hays, Target Health Inc.
Next, add all of the Tofutti into a mixing bowl and use a spatula to scrape it all out
With the beaters on, slowly add the liquid jello until you get Tofutti and jello well combined
Slowly pouring jello into Tofutti on with beaters on. ©Joyce Hays, Target Health Inc.
Remove the beaters. Add most of the blueberries and stir into the Tofutti mixture.
Next, with spatula, scrape all of the Tofutti mixture with blueberries, into the springform pan. Once in the pan, don’t stir or the crumbs will not remain as the first layer.
Now, put the pan into the refrigerator for two hours.
Wash the strawberries, remove any leaves, and cut in half. Don’t use any strawberry that’s discolored, just eat it on the spot. You’ll see that using a discolored berry will ruin the presentation.
Pour the Tofutti and blueberry mixture into pan. ©Joyce Hays, Target Health Inc.
A 1/2 hour before you take the pan out of the refrigerator, make the second box of strawberry jello and pour into a measuring cup. Let it sit and cool down.
After two hours in the refrigerator, take the pan out and to the springform cake pan with the Tofutti mixture now solid, add the measuring cup of cooled liquid jello
Getting ready for final step ©Joyce Hays, Target Health Inc.
Pour the jello over the solid layer of Tofutti and of course, don’t mix.
I’ve just poured the second cup of jello over the Tofutti mixture. ©Joyce Hays, Target Health Inc.
Arranging the strawberries ©Joyce Hays, Target Health Inc.
Right after pouring the second cup of jello over the Tofutti mixture, carefully arrange the strawberry halves around the edge. They will be slightly underneath the jello, and that’s exactly what you want.
Add four strawberry halves in the center, as you see. ©Joyce Hays, Target Health Inc.
Add four strawberry halves in the center and then fill in the rest of the space with blueberries or all strawberries
Put the pan back in the refrigerator for another two hours before serving.
Only remove the side ring from the springform pan, after the last two hours, just before serving
Serve, adding cool whip as topping, on each individual serving
This cake had 2 containers of Tofutti and all strawberries on top©Joyce Hays, Target Health Inc.
Here is bottom layer with Amaretti cookies crumbled. ©Joyce Hays, Target Health Inc.
About to remove side ring of pan after last two hours ©Joyce Hays, Target Health Inc.
This had only one container of Tofutti. ©Joyce Hays, Target Health Inc.
This cake had only 1 container of Tofutti. ©Joyce Hays, Target Health Inc.
We started September with Sexy Beast, a Cab with a punch ©Joyce Hays, Target Health Inc.
From Our Table to Yours!