Innovation at FDA – Accelerated Approval for Drugs and Biologics

 

In May 2014, FDA issued a Guidance for Industry – Expedited Programs for Serious Conditions – Drugs and Biologics. This Guidance codifies processes that FDA uses to get drugs and biologics to market sooner, when there is an unmet need to address serious conditions. Programs include:

 

Accelerated Approval Program: The FDA allows for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. Drug companies are still required to conduct studies to confirm the anticipated clinical benefit. These studies are known as phase 4 confirmatory trials. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug. If the confirmatory trial does not show that the drug provides clinical benefit, FDA has regulatory procedures in place that could lead to removing the drug from the market.

 

Breakthrough Therapy: Breakthrough designation is for a product that is intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. Breakthrough therapy designation will usually mean that the effect of the drug will be large compared with available therapies. In such cases, the development program for the breakthrough therapy could be considerably shorter than for other drugs intended to treat the disease being studied. However, FDA notes that a compressed drug development program still must generate adequate data to demonstrate that the drug is safe and effective to meet the statutory standard for approval. Omitting components of the drug development program that are necessary for such a determination can significantly delay, or even preclude, marketing approval. Sponsors can design efficient clinical trials in a number of ways. FDA will seek to ensure that a sponsor of a product designated as a breakthrough therapy receives timely advice and interactive communications to help the sponsor design and conduct a drug development program as efficiently as possible. During these interactions, the Agency may suggest, or a sponsor may propose, alternative clinical trial designs (e.g., adaptive designs, an enrichment strategy, crossover or N-of-1 design, use of historical controls) or use of an interim analysis by a data monitoring committee. These trial designs may result in smaller trials or more efficient trials that require less time to complete and may help minimize the number of patients exposed to a potentially less efficacious treatment (i.e., the control group treated with available therapy). Such approaches may be especially useful in studies in rare diseases. For example, single-arm trials may be an important option in rare diseases with well-understood pathophysiology and a well-defined disease course

 

Fast Track: Fast Track emphasizes the critical nature of close early communication between the FDA and sponsors and adds to existing programs, such as accelerated approval, the possibility of a “rolling review“ for an application.

 

Priority Review: A Priority Review designation means FDA’s goal is to take action on an application within 6 months (compared to 10 months) if the drug could provide significant advantages such as:

1. evidence of increased effectiveness in treatment, prevention, or diagnosis of condition;

2. elimination or substantial reduction of a treatment-limiting drug reaction

3. documented enhancement of patient compliance

4. evidence of safety and effectiveness in a new subpopulation

 

Edamame/Pepper/Corn/Bean Salad with Honey/Lemon/Soy Dressing, Cashews & More

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This salad is a tasty meal in itself. The white strips are jicama, which could be grated instead if you want smaller pieces. ©Joyce Hays, Target Health Inc.

 

Ingredients

 

2 cups shelled edamame beans

1 cup raw fresh yellow corn

1 or 2 diced avocados

1 cup toasted and salted (optional) cashews

1/2 cup sliced or diced roasted and peeled red bell peppers

1 1/2 cups fresh French green beans, blanched, shocked (ice water) and chopped

2 cups diced jicama

1/2 cup salad dressing, more as desired

1 cup daikon sprouts or substitute your favorite sprouts

1/4 cup plus 2 Tablespoons hemp seeds (or black sesame seeds)

 

 

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The variety of ingredients make this salad special. Take a deep breath of the fresh rosemary. The grated fresh ginger really adds a mysterious touch; you hardly know it’s there, but without it, you’d miss it. The jicama is healthy and gives a nice crunchy texture, not to mention the delicious cashews. Go with low sodium soy in the awesome dressing. ©Joyce Hays, Target Health Inc.

 

Directions

 

Make the Salad dressing First

1 1/2 teaspoons grated fresh ginger

1 1/2 teaspoons coarsely chopped shallot

1 1/2 teaspoons coarsely chopped fresh rosemary

2 Tablespoons soy sauce

2 Tablespoons honey

3 Tablespoons sweet rice vinegar (or white vinegar)

1 1/2 Tablespoons fresh lemon juice

1/2 cup canola oil

 

In a blender, grind the ginger, shallots and rosemary to a paste.

Add the soy sauce, honey, vinegar and lemon juice to the ginger mixture and blend until fairly smooth.

With the blender running, slowly add the oil. This makes about three-fourths cup dressing, more than is needed for the remainder of the recipe. The dressing will keep, up to 3 days, covered and refrigerated.

 

Complete the Salad

 

In a large bowl, combine the edamame, corn, avocado, cashews, peppers, green beans and jicama.

Add one-half cup of the dressing and toss to coat. Taste, and add additional dressing if needed.

Garnish with the daikon sprouts and hemp seeds. Serve immediately

 

 

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These 3 fresh fruits are reaching a peak. Now they’re 4 out of 5 stars and luscious together: peaches, strawberries and pixie tangerines. A wonderful dessert by themselves or add a little peach schnapps for added zip. ©Joyce Hays, Target Health Inc.

 

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Dekuyper Peachtree Schnapps

 

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Feeling adventurous, we tried a New Zealand sauvignon blanc, called Man O’ War. This is the first white wine that gave a real unexpected punch and a finish that doesn’t let go. Much earlier in the year, we recommended an Australian red called, Sexy Beast, that knocked our socks off (so to speak) with its robust nose, palette, finish, and a 93 point rating from Wine Spectator. On the white side, Man O’ War gives a similar knockout punch, if that’s what you like in your whites. Wines from this part of the world are delicious and well worth trying. Man O’ War gets a 91 point rating. Neither of these breaks the bank.

 

So, we began our meal with chilled Man O’ War and the delicious fresh veggie salad, with recipe, above, in this newsletter.  Although, we ate indoors, this salad is wonderful for meals out of doors on summer days & evenings, because with no mayo in it, it won’t spoil.  Next we had rare fillet mignon with a marsala mushroom sauce.  We only eat steak at home, because we have never had such a tender cut at a restaurant, including well known steak houses and premier restaurants.  Along with the delicious steak, so tender you could cut it with a fork, were baked mini red potatoes.  Each one is bite size. Before baking, I soak them in a bowl with olive oil and garlic, then just spread out on a baking sheet and bake for less time than a usual size baked potato.  We had crunchy broccoli saut?ed in the olive oil and garlic, I used for soaking the mini potatoes.  Dessert was the freshly cut up peaches, tangerines and sweet strawberries with peach schnapps which is lighter than most licquers.  This was a lovely meal.

 

Winter is gone in the Big Apple and New York is green and glorious with tree lined blocks and flowers everywhere.  We saw a play at the Lucille Lortel Theater on Christopher Street in the West Village, our old stomping grounds years ago.  Fun to be back in the Village, but the play, a dark comedy called Punishment,was bizarre and without a point, about husbands finding agreed upon reasons to spank their wives, I guess to give their lives more structure or more excitement or who knows?  On top of this odd focus, there was no point of view about masochism or victimhood.  The plot appeared to be an assortment of loosely formed ideas about marriage which never came together with any kind of resolution. One form of idiocy led to another, followed by an intermission and then back to the same old grind of stupid meaningless situations.  The first scene of a spanking bordered on the risque, but the playwright backed away a bit, which could/might have made the time spent in the audience, more interesting, but even the possibility, escaped the writer. Because we love New Yorktheater (and donate to many theater clubs), we give such a play a wide berth.  Off Broadway theaters are important laboratories for ideas and we accept this play in that vein.

 

Hope your Memorial Day (USA) weekend was relaxing and pleasurable.

 

 

From Our Table to Yours!

 

Bon Appetit!

 

Date:
May 21, 2015

Source:
University of Wyoming

Summary:
A certain type of soil bacteria can use their social behavior of outer membrane exchange to repair damaged cells and improve the fitness of the bacteria population as a whole, new research demonstrates. This is the first evidence that a bacterium can use cell-content sharing to repair damaged siblings, the authors say.

 

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Chris Vassallo, a University of Wyoming doctoral student in the Department of Molecular Biology, performs routine maintenance of bacterial cultures in the lab. Vassallo was lead author of a paper about cell rejuvenation that was published in PNAS.
Credit: University of Wyoming

 

 

A University of Wyoming faculty member led a research team that discovered a certain type of soil bacteria can use their social behavior of outer membrane exchange (OME) to repair damaged cells and improve the fitness of the bacteria population as a whole.

Daniel Wall, a UW associate professor in the Department of Molecular Biology, and others were able to show that damaged sustained by the outer membrane (OM) of a myxobacteria cell population was repaired by a healthy population using the process of OME. The research revealed that these social organisms benefit from group behavior that endows favorable fitness consequences among kin cells.

Wall says, to the research group’s knowledge, this is the first evidence that a bacterium can use cell-content sharing to repair damaged siblings.

“It is analogous to how a wound in your body can be healed,” Wall says. “When your body is wounded, your cells can coordinate their functions to heal the damaged tissue.”

Wall was the senior and corresponding author on a paper, titled “Cell Rejuvenation and Social Behaviors Promoted by LPS Exchange in Myxobacteria” that was published in the May 18 online issue of theProceedings of the National Academy of Sciences (PNAS).

Chris Vassallo, a UW doctoral student in molecular biology and originally from Cheyenne, was the paper’s lead author and conducted most of the lab experiments.

“During nutrient depletion, myxobacteria cooperate to build a macroscopic structure called a fruiting body,” Vassallo says. “The structure resembles a tree or mushroom in appearance.”

A fruiting body is essentially a multicellular organism that produces dormant spores that are resistant to environmental stresses.

These myxobacterial cells, in their native environments, must cope with factors that compromise the integrity of the cell, Wall says. Rather than looking out only for themselves like other bacterial species, the individual myxobacteria cells band together as a social group to assist their kin that become damaged.

“Myxobacteria are unusual for bacteria in that they have a true multicellular life,” Wall says. “Researchers are interested in how the evolutionary transition occurred toward multi-cellularity; that is, how cooperation develops and single cells are not just interested in themselves. The Darwinian view is that each individual is out for themselves; ‘survival of the fittest.'”

“When environmental cells come together, they compete with each other,” Wall continues. “With OME, we think it allows myxobacteria cells to transition from a heterogeneous single cellular life to a more harmonious multicellular life.”

While the practical application of the science is to better understand how single cells can evolve to multicellular cells to be cooperative, Vassallo says the research could possibly have future real-world implications for treating infections in humans.

“The most direct applicability could be for antibiotic resistance,” Wall says. “Within the paper, Chris did an experiment where one strain of myxobacteria conferred antibiotic resistance to another strain. This works by the cells transferring their OM armor.

“The human skin protects the body and internal cells from environmental stresses. By analogy, bacteria protect themselves with their OM, and they are known to change their armor in response to stress. When they chemically change their armor, they can also change their antibiotic resistance profile.”


Story Source:

The above story is based on materials provided by University of Wyoming.Note: Materials may be edited for content and length.


Journal Reference:

  1. Christopher Vassallo, Darshankumar T. Pathak, Pengbo Cao, David M. Zuckerman, Egbert Hoiczyk, Daniel Wall. Cell rejuvenation and social behaviors promoted by LPS exchange in myxobacteria. Proceedings of the National Academy of Sciences, 2015; 201503553 DOI:10.1073/pnas.1503553112

 

Source: University of Wyoming. “Bacteria cooperate to repair damaged siblings.” ScienceDaily. ScienceDaily, 21 May 2015. <www.sciencedaily.com/releases/2015/05/150521133738.htm>.

Date:
May 20, 2015

Source:
The Lancet

Summary:
Cold weather kills 20 times as many people as hot weather, according to an international study analyzing over 74 million deaths in 384 locations across 13 countries. The findings also reveal that deaths due to moderately hot or cold weather substantially exceed those resulting from extreme heat waves or cold spells.

 

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Cold weather kills 20 times as many people as hot weather, according to an international study analyzing over 74 million deaths in 384 locations across 13 countries.
Credit: © ventura / Fotolia

 

 

Cold weather kills 20 times as many people as hot weather, according to an international study analyzing over 74 million deaths in 384 locations across 13 countries. The findings, published in The Lancet, also reveal that deaths due to moderately hot or cold weather substantially exceed those resulting from extreme heat waves or cold spells.

“It’s often assumed that extreme weather causes the majority of deaths, with most previous research focusing on the effects of extreme heat waves,” says lead author Dr Antonio Gasparrini from the London School of Hygiene & Tropical Medicine in the UK. “Our findings, from an analysis of the largest dataset of temperature-related deaths ever collected, show that the majority of these deaths actually happen on moderately hot and cold days, with most deaths caused by moderately cold temperatures.”

The study analysed over 74 million (74,225,200) deaths between 1985 and 2012 in 13 countries with a wide range of climates, from cold to subtropical. Data on daily average temperature, death rates, and confounding variables (eg, humidity and air pollution) were used to calculate the temperature of minimum mortality (the optimal temperature), and to quantify total deaths due to non-optimal ambient temperature in each location. The researchers then estimated the relative contributions of heat and cold, from moderate to extreme temperatures.

Around 7.71% of all deaths were caused by non-optimal temperatures, with substantial differences between countries, ranging from around 3% in Thailand, Brazil, and Sweden to about 11% in China, Italy, and Japan. Cold was responsible for the majority of these deaths (7.29% of all deaths), while just 0.42% of all deaths were attributable to heat.

The study also found that extreme temperatures were responsible for less than 1% of all deaths, while mildly sub-optimal temperatures accounted for around 7% of all deaths — with most (6.66% of all deaths) related to moderate cold.

According to Dr Gasparrini, “Current public-health policies focus almost exclusively on minimizing the health consequences of heat waves. Our findings suggest that these measures need to be refocused and extended to take account of a whole range of effects associated with temperature.”

Writing in a linked Comment, Keith Dear and Zhan Wang from Duke Kunshan University, Kunshan, Jiangsu, China say, “Factors such as susceptibility or resilience have not been included in the analysis, including socioeconomic status, age, and confounding air pollutants…Since high or low temperatures affect susceptible groups such as unwell, young, and elderly people the most, attempts to mitigate the risk associated with temperature would benefit from in-depth studies of the interaction between attributable mortality and socioeconomic factors, to avoid adverse policy outcomes and achieve effective adaptation.”


Story Source:

The above story is based on materials provided by The Lancet. Note: Materials may be edited for content and length.


Journal Reference:

  1. Antonio Gasparrini, Yuming Guo, Masahiro Hashizume, Eric Lavigne, Antonella Zanobetti, Joel Schwartz, Aurelio Tobias, Shilu Tong, Joacim Rocklöv, Bertil Forsberg, Michela Leone, Manuela De Sario, Michelle L Bell, Yue-Liang Leon Guo, Chang-fu Wu, Haidong Kan, Seung-Muk Yi, Micheline de Sousa Zanotti Stagliorio Coelho, Paulo Hilario Nascimento Saldiva, Yasushi Honda, Ho Kim, Ben Armstrong. Mortality risk attributable to high and low ambient temperature: a multicountry observational study. The Lancet, May 2015 DOI: 10.1016/S0140-6736(14)62114-0

 

Source: The Lancet. “International study reveals that cold weather kills far more people than hot weather.” ScienceDaily. ScienceDaily, 20 May 2015. <www.sciencedaily.com/releases/2015/05/150520193831.htm>.

Date:
May 19, 2015

Source:
Kansas State University

Summary:
A researcher has found that the dopamine transporter gene DAT1 can have both positive and negative effects on leadership in the workplace. The researchers found that people who had the 10-repeat allele in the dopamine transporter were most likely to engage in adolescent mild rule-breaking behavior, which is positively associated with leadership. Such mild rule-breaking behavior may include actions such as skipping class, but it is not serious deviant behavior such as shooting.

 

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To become a leader and be a good leader involves multiple factors — genes and the environment — working together.
Credit: © zinkevych / Fotolia

 

 

The right genes may help you become an organization’s next president or CEO. But the same genes may also hinder your leadership path, according to Kansas State University psychological sciences research.

Wendong Li, assistant professor of psychological sciences, and collaborators have found a “mixed blessing” for workers who hold workplace leadership positions, from the formal leader of a CEO to an informal group leader. Their study focused on the dopamine transporter gene DAT1, which can influence leadership and is important for reward and motivation systems in humans.

“It’s like a mixed blessing — this gene can have both positive and negative effects on leadership,” Li said. “An implication is that it really depends on environmental factors to determine if overall it is a positive or negative.”

On the positive side, the researchers found that people who had the 10-repeat allele in the dopamine transporter were most likely to engage in adolescent mild rule-breaking behavior, which is positively associated with leadership, Li said. Such mild rule-breaking behavior may include actions such as skipping class, but it is not serious deviant behavior such as shooting.

“Mild rule-breaking is actually positively correlated with the chance for you to become a leader in adulthood,” Li said. “These kinds of behaviors can provide you with an advantage because they allow adolescents to explore boundaries and learn something new.”

On the negative side, the researchers found that people with the dopamine transporter gene scored lower on proactive personality, which can lead to positive changes at work and is important for leadership emergence.

“These people were less likely to regulate their own behaviors to make a positive change,” Li said. “It can be very difficult to make a positive change because it involves mobilizing resources to overcome difficulties and obstacles so that the change can happen. These people were not good at regulating behaviors such as being persistent.”

The takeaway from the study? To become a leader and be a good leader involves multiple factors — genes and the environment — working together, Li said. Some influential environmental factors — though not studied in this research — can include democratic parenting, a supportive family, and a challenging and cultivating workplace.

Managers cannot assume that changing one aspect of the work environment will be beneficial for all individuals, Li said, because employees bring individual characteristics to the organization. Some individual differences can’t be ignored because they are rooted in genetic makeup and enhance the chance for individuals to engage in certain types of behaviors, either positive or negative.

“In the long run, we are advocating more individualized and customized management practices, which allow people to choose the type of work environment that fits their individual characteristics,” Li said. “Customizing workplace practices is good for employee learning, development and leadership potential. Ultimately, it is good for employee performance and well-being, which in turn may enhance organizational effectiveness.”

The researchers used two sets of data for the study: The National University of Singapore’s Strabismus, Amblyopia, and Refractive Error Study, or STARS, which includes 309 people, and the National Longitudinal Study of Adolescent Health, which includes more than 13,000 individuals. The researchers had similar results with both samples, Li said.


Story Source:

The above story is based on materials provided by Kansas State University. The original article was written by Jennifer Tidball. Note: Materials may be edited for content and length.


Journal Reference:

  1. Wen-Dong Li, Nan Wang, Richard D. Arvey, Richie Soong, Seang Mei Saw, Zhaoli Song. A mixed blessing? Dual mediating mechanisms in the relationship between dopamine transporter gene DAT1 and leadership role occupancy. The Leadership Quarterly, 2015; DOI:10.1016/j.leaqua.2014.12.005

 

Source: Kansas State University. “Genes may influence leadership in the workplace.” ScienceDaily. ScienceDaily, 19 May 2015. <www.sciencedaily.com/releases/2015/05/150519122305.htm>.

Date:
May 18, 2015

Source:
University of Utah

Summary:
Engineers have developed an ultracompact beamsplitter — the smallest on record — for dividing light waves into two separate channels of information. The device brings researchers closer to producing silicon photonic chips that compute and shuttle data with light instead of electrons.

 

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The overhead view of a new beamsplitter for silicon photonics chips that is the size of one-fiftieth the width of a human hair.
Credit: Dan Hixson/University of Utah College of Engineering

 

 

University of Utah engineers have taken a step forward in creating the next generation of computers and mobile devices capable of speeds millions of times faster than current machines.

The Utah engineers have developed an ultracompact beamsplitter — the smallest on record — for dividing light waves into two separate channels of information. The device brings researchers closer to producing silicon photonic chips that compute and shuttle data with light instead of electrons. Electrical and computer engineering associate professor Rajesh Menon and colleagues describe their invention today in the journal Nature Photonics.

Silicon photonics could significantly increase the power and speed of machines such as supercomputers, data center servers and the specialized computers that direct autonomous cars and drones with collision detection. Eventually, the technology could reach home computers and mobile devices and improve applications from gaming to video streaming.

“Light is the fastest thing you can use to transmit information,” says Menon. “But that information has to be converted to electrons when it comes into your laptop. In that conversion, you’re slowing things down. The vision is to do everything in light.” Photons of light carry information over the Internet through fiber-optic networks. But once a data stream reaches a home or office destination, the photons of light must be converted to electrons before a router or computer can handle the information. That bottleneck could be eliminated if the data stream remained as light within computer processors.

“With all light, computing can eventually be millions of times faster,” says Menon.

To help do that, the U engineers created a much smaller form of a polarization beamsplitter (which looks somewhat like a barcode) on top of a silicon chip that can split guided incoming light into its two components. Before, such a beamsplitter was over 100 by 100 microns. Thanks to a new algorithm for designing the splitter, Menon’s team has shrunk it to 2.4 by 2.4 microns, or one-fiftieth the width of a human hair and close to the limit of what is physically possible.

The beamsplitter would be just one of a multitude of passive devices placed on a silicon chip to direct light waves in different ways. By shrinking them down in size, researchers will be able to cram millions of these devices on a single chip.

Potential advantages go beyond processing speed. The Utah team’s design would be cheap to produce because it uses existing fabrication techniques for creating silicon chips. And because photonic chips shuttle photons instead of electrons, mobile devices such as smartphones or tablets built with this technology would consume less power, have longer battery life and generate less heat than existing mobile devices.

The first supercomputers using silicon photonics — already under development at companies such as Intel and IBM — will use hybrid processors that remain partly electronic. Menon believes his beamsplitter could be used in those computers in about three years. Data centers that require faster connections between computers also could implement the technology soon, he says.

Co-authors on the paper include research associate Randy Polson and doctoral students Bing Shen and Peng Wang.


Story Source:

The above story is based on materials provided by University of Utah. Note: Materials may be edited for content and length.


Journal Reference:

  1. Bing Shen, Peng Wang, Randy Polson, Rajesh Menon. An integrated-nanophotonics polarization beamsplitter with 2.4 × 2.4 μm2 footprint. Nature Photonics, 2015; DOI: 10.1038/nphoton.2015.80

 

Source: University of Utah. “Computing at the speed of light with ultracompact beamsplitter.” ScienceDaily. ScienceDaily, 18 May 2015. <www.sciencedaily.com/releases/2015/05/150518121153.htm>.

Symposium on Risk-based Monitoring and eSource – DIA Meeting, 2015

 

On June 16, 2015, at the DIA Annual Meeting in Washington, DC, between 3:30- 5:00, Dr. Jules T. Mitchel, PhD, MBA, President of Target Health Inc., will be chairing a symposium on How Risk-Based Monitoring and eSource Methodologies Are Impacting Clinical Sites, Patients, Regulators and Sponsors. This symposium will show how risk-based monitoring and eSource methodologies are impacting the way clinical trials are being conducted and managed. Specific topics include:

 

The Time Is Now for Risk-Based Monitoring: Frances E. Nolan, Medidata Solutions Worldwide,

Overcoming Clinical Trial Data Collection Challenges with eSource Solutions and Leveraging Mobile Technologies: Avik Kumar Pal, CliniOps

Using eSource to Maximize Clinical Development Productivity and Efficiency: Edward Stephen Seguine, Clinical Ink

Please let us know if you will be attending.

 

View From the Triboro Bridge at 6 am (NYC)

 

The Triboro Bridge, now officially known as the Robert F. Kennedy Bridge since 2008, connects Manhattan, Queens and The Bronx. Here is the view from the bridge, when Dr. Mitchel landed at 5 am at JFK, after returning from a conference and meetings with clients in Israel.The bridge you are looking at is the one you use when taking Amtrak to Boston. By the way, “real New Yorkers“ still call it the TriboroBridge.

 

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View from the Triboro Bridge at 6am ©Target Health Inc. 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

 

QUIZ

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Preventative Medicine

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Asclepius, the god of medicine and healing in Greek mythology. Depictions of the serpent-entwined rod of Asclepius are frequently used to represent medicine.

 

Preventive medicine or healthcare consists of measures taken for disease prevention, as opposed to disease 1) ___. Disease and disability are affected by environmental factors, genetic predisposition, disease agents, and lifestyle choices. Health, disease, and disability are dynamic processes which begin before individuals realize they are affected. Disease prevention relies on anticipatory actions that can be categorized as primary, secondary, and tertiary prevention. Each year, millions of people die preventable deaths. Studies show that about half of all 2) ___ in the United States are due to preventable behaviors and exposures. Leading causes included cardiovascular disease, chronic respiratory disease, unintentional injuries like falls especially in older people, diabetes, and certain infectious diseases. One study estimates that 400,000 people die each year in the United States due to poor diet and a sedentary lifestyle. According to estimates made by the World Health Organization (WHO), about 55 million people died worldwide in 2011, two thirds of this group from non-communicable diseases, including cancer, diabetes, and chronic cardiovascular and lung diseases.

 

A 2010 study reported that in the United States, vaccinating children, cessation of smoking, daily prophylactic use of aspirin, and screening of breast and colorectal cancers had the most potential to prevent premature death. Preventive health measures that resulted in savings included vaccinating children and adults, smoking cessation, daily use of aspirin, and screening for issues with alcoholism, obesity, and vision failure. There are many methods for prevention of disease. It is recommended that adults and children aim to visit their doctor for regular check-ups, even if they feel 3) ___, to perform disease screening, identify risk factors for disease, discuss tips for a healthy and balanced lifestyle, stay up to date with immunizations and boosters, and maintain a good relationship with a healthcare provider. Some common disease screenings include checking for hypertension (high blood pressure), hyperglycemia (high blood sugar, a risk factor for diabetes mellitus), hypercholesterolemia (high blood cholesterol), screening for colon cancer, depression, HIV and other common types of sexually transmitted disease such as chlamydia, syphilis, and gonorrhea, mammography, colorectal cancer screening, a pap test (to check for cervical cancer), and screening for osteoporosis. Genetic testing can also be performed to screen for mutations that cause genetic disorders or predisposition to certain diseases such as breast or ovarian cancer. However, these measures are not affordable for every individual and the cost effectiveness of preventive healthcare is still a topic of debate.

 

Primary prevention consists of “health promotion“ and “specific protection.“ Health promotion activities are non-clinical life choices, for example, eating nutritious meals and exercising daily, that both prevent disease and create a sense of overall well-being. Preventing disease and creating overall well-being, prolongs our 4) ___ expectancy. Health-promotional activities do not target a specific disease or condition but rather promote health and well-being on a very general level. On the other hand, specific protection targets a type or group of diseases and complements the goals of health promotion. In the case of a sexually transmitted disease such as syphilis health promotion activities would include avoiding microorganisms by maintaining personal hygiene, routine check-up appointments with the doctor, general sex education, etc. whereas specific protective measures would be using prophylactics (such as 5) ___) during sex and avoiding sexual promiscuity.

 

Food is very much the most basic tool in preventive health care. The 2011 National Health Interview Survey performed by the Centers for Disease Control was the first national survey to include questions about ability to pay for food. Difficulty with paying for food, medicine, or both is a problem facing 1 out of 3 Americans. If better food options were available through food banks, soup kitchens, and other resources for low-income people, obesity and the chronic conditions that come along with it would be better controlled. A “food 6) ___“ is an area with restricted access to healthy foods due to a lack of supermarkets within a reasonable distance. These are often low-income neighborhoods with the majority of residents lacking transportation. There have been several grassroots movements in the past 20 years to encourage urban gardening, such as the GreenThumb organization in New York City and the Brooklyn Grange. Urban gardening uses vacant lots to grow food for a neighborhood and is cultivated by the local residents. Brooklyn Grange grows crops on rooftops. Mobile fresh markets are another resource for residents in a “food desert“, which are specially outfitted buses bringing affordable fresh fruits and vegetables to low-income neighborhoods. These programs often hold educational events as well such as cooking and nutrition guidance. Programs such as these are helping to provide healthy, affordable foods to the people who need them the most.

 

Scientific advancements in genetics have significantly contributed to the knowledge of hereditary diseases and have facilitated great progress in specific protective measures in individuals who are carriers of a disease gene or have an increased predisposition to a specific disease. Genetic testing has allowed physicians to make quicker and more accurate diagnoses and has allowed for tailored treatments or personalized 7) ___. Personalized medicine will be an important part of the future of preventive healthcare. Each individual will be required to obtain a greater knowledge of healthcare, and to maintain knowledge with ongoing self-education. Humans in the 21stCentury are going to have to be much better educated about everything. Similarly, specific protective measures such as water purification, sewage treatment, and the development of personal hygienic routines (such as regular hand-washing) became mainstream upon the discovery of infectious disease agents such as bacteria. These discoveries have been instrumental in decreasing the rates of communicable diseases that are often spread in unsanitary conditions.

 

Finally, a separate category of health promotion has been propounded, based on the ?new knowledge’ in molecular biology – in particular epigenetics – which points to how much physical as well as affective environments during fetal and newborn life may determine 8) ___health. This is commonly called primal prevention. It involves providing future parents with pertinent, unbiased information on primal health and supporting them during their child’s primal life (i.e., “from conception to first anniversary“). This includes adequate parental leave – ideally for both parents – with kin care-giving and financial help if needed.

 

Colorectal cancer (also called bowel cancer, colon cancer, or rectal cancer) is globally the second most common cancer in women and the third-most common in men, and the fourth most common cause of cancer death after lung, stomach, and liver cancer. It is also highly preventable; about 80 percent of colorectal cancers begin as benign growths, commonly called 9) ___, which can be easily detected and removed during a colonoscopy. Other methods of screening for polyps and cancers include fecal occult blood testing. Lifestyle changes that may reduce the risk of colorectal cancer include increasing consumption of whole grains, fruits and vegetables, and reducing consumption of red meat. Given that high quality screening and follow-up care has been shown to reduce cervical cancer rates by up to 80%, most developed countries now encourage sexually active women to undergo a pap test every 3-5 years.

 

Skin cancer is the most common cancer in the United States. The most lethal form of skin cancer, melanoma, leads to over 50,000 annual deaths in the United States. Childhood prevention is particularly important because a significant portion of ultraviolet radiation exposure from the sun occurs during childhood and adolescence and can subsequently lead to skin cancer in adulthood. Furthermore, childhood prevention can lead to the development of healthy habits that continue to prevent cancer for a lifetime. The Centers for Disease Control and Prevention (CDC) recommends several primary prevention methods including: limiting sun exposure between 10 AM and 4 PM, when the sun is strongest. 10) ___ cancer is very preventable.

 

ANSWERS: 1) treatment; 2) deaths; 3) healthy; 4) life; 5) condoms; 6) desert; 7) medicine; 8) adult; 9) polyps; 10) Skin

 

The Tragic Death of Dave Goldberg (1967-2015)

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Dave Goldberg in the offices of SurveyMonkey, where he was chief executive, in 2013. Credit Jim Wilson/The New York Times

 

David Goldberg Silicon Valley executive, who was CEO of SurveyMonkey, died of severe head trauma in an exercise accident at a resort in Punta Mita near Puerto Vallarta on Mexico’s Pacific coast. David Goldberg, husband of Facebook executive Sheryl Sandberg, was found lying next to a treadmill. It was reported that he left his room at about 4pm to exercise, and family members went to look for him after he didn’t return. He was found at about 6:30pm in a gymnasium lying by a treadmill in a pool of blood, with a blow to the lower back of his head. He apparently had slipped on the treadmill and hit the machine.

 

Goldberg was born in Minneapolis, Minnesota on October 2, 1967. His mother Paula Goldberg is co-founder and executive director of the Pacer Center and his father, Mel Goldberg (1942-1998) was associate dean and professor at the William Mitchell College of Law. He interned at the Minneapolis Star Tribune newspaper while in high school. He graduated from Blake School in Minneapolis in 1985, and graduated magna cum laude from Harvard University in 1989, majoring in history and government. Goldberg worked for Bain & Company for two years after graduating from college. He had planned to attend law school but instead joined Capitol Records, where he served as director of marketing strategy and new business development. He founded LAUNCH Media in 1994, and led it through its acquisition by Yahoo Inc. in 2001. He quit Yahoo in 2007 and joined Benchmark Capital. He then joined SurveyMonkey in 2009.

 

Goldberg married Cheryl Sandberg in 2004, and they had two children. They met when both worked for Yahoo. David was a Silicon Valley executive whose company runs an online polling and questionnaire service. In the early 1990s he maxed out his credit cards to fund his first Internet venture, and then went on to work at other tech companies including Yahoo. Under Goldberg’s leadership, SurveyMonkey grew from 14 to 500 employees, with a 2014 market cap of $2 billion and 25 million customers.

 

Sports medicine retired physician, Gabe Mirkin MD says in an article on his website that it is very unlikely that he died just from falling off the treadmill and banging his head. Mirkin says that “the only thing I know about his medical history is that he reportedly had high blood pressure. The most common cause of high blood pressure is high blood sugar, and from looking at his pictures I am almost certain that he was diabetic or pre-diabetic. Almost all people with a fat belly and relatively small buttocks have excess fat stored in the liver, which increases risk for a heart attack and sudden death.“ According to Mirkin, newspaper reports claim that his death was caused by loss of a huge amount of blood from the cut on his head, which is almost impossible. When most people cut themselves, they form clots and bleed only a little. However, people with a large belly and high blood pressure are usually told to take aspirin or other anti-clotting medications that can cause massive bleeding into the brain after a head injury, which can stop breathing and cause death. People who store fat primarily in their bellies store excess fat in their livers, and excess liver fat can lead to diabetes. A high rise in blood sugar damages every cell in your body. To prevent your blood sugar level from rising too high after you eat, your pancreas releases insulin that drives sugar from the bloodstream into your liver. However, if your liver is full of fat, it will not accept the sugar from blood. Instead your liver releases its stored sugar into the bloodstream to drive your blood sugar level even higher. If you have a large belly and small hips, the odds are overwhelming that you are already diabetic and are headed for a heart attack. Furthermore, if you have diabetes or other heart attack risk factors, your doctor is most likely to put you on aspirin and other anti-clotting medications that will increase your chances of bleeding into your brain with any trauma to your head. Mirkin advises that if you and your doctor agree that you should take medication and/or supplements, make sure you are aware of their potential side effects. People who take aspirin or other anti-clotting medications would be wise to have another person present when they exercise or do anything that has a risk of falling.

 

Here at Target Health Inc., we feel that no matter what the sport or exercise is, you should do it with a partner. Who knows, perhaps Dave Goldberg’s life could have been saved if he had not exercised alone. We are truly sorry for the loss of this wonderful person. Sources: The New York Times; Gabe Mirkin MD; Wikipedia

 

NIH Study Solves the Mystery of the Tubulin Code

 

Microtubules are cylindrical structures that provide shape to cells and act as conveyor belts, ferrying molecular cargo throughout cells. Although all microtubules have the same basic appearance, they are marked on their outside surface with a variety of chemical groups. These markers impact a cell’s activity by changing the stability of microtubules, thus affecting cell shape, or by repositioning molecular cargo traveling on the microtubules. The microtubule markers are constantly being added and removed, depending on the local needs of the cell. Think about a highway system where street signs are constantly changing and roads are quickly built or torn apart.

 

Driving down the highway, you encounter ever-changing signs — speed limits, exits, food and gas options. Seeing these roadside markers may cause you to slow down, change lanes or start thinking about lunch. In a similar way, cellular structures called microtubules are tagged with a variety of chemical markers that can influence cell functions. The pattern of these markers makes up the “tubulin code“ and according to a paper published online in Cell, May 7, 2015, the mechanism behind one of the main writers of this code is tubulin tyrosine ligase-7 (TTLL7). According to the authors, understanding the structural characteristics of this specific molecule opens the door to learning how elaborate patterns of chemical markers are laid down on microtubules. In addition, deciphering the tubulin code could reveal how the markers affect normal cellular function as well as what happens when they are damaged, leading to neurodegenerative disorders.

 

TTLL7 is a protein that adds glutamate tags onto microtubules. Using a number of advanced imaging and biochemical techniques, the authors have revealed the 3-D structure of TTLL7 bound to the microtubule. There are nine proteins that make up the TTLL family, but TTLL7 is the most abundant in the brain and one of the main tubulin code writers. These results represent the first atomic structure of any member of the TTLL family. The findings define how TTLL7 interacts with microtubules and how members of the TTLL family use common strategies to mark microtubules with glutamate tags. The authors were able to see how TTLL7 positions itself on the microtubule by grabbing onto the microtubule tails.

 

The most common microtubule marker in the brain is glutamate. The addition of glutamate markers to microtubules plays important roles in brain development and brain cell repair following injury. For example, one of the signatures of damaged cells in cancer or blunt trauma is a change in the pattern of these microtubule markers. In addition, mutations in TTLL genes have been linked with several neurodegenerative disorders. The authors plan to extend their research by investigating interactions between members of this family of proteins. They want to mix and match the TTLL proteins to see how they can control patterns of microtubule tagging. From that, it may be possible to learn how the cell is making those patterns and what happens during cellular damage, as in cancers or neurodegeneration, when these patterns are disrupted.

 

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