Thursday, November 26, 2009

Thanksgiving and hopefully some rest

First of all, Happy Thanksgiving!

My mom made Thanksgiving dinner today and we had our celebration tonight w/ me, my mom, stepdad, sister, and my three nephews who I adore....and oh yeah, can't forget the dogs! It was yummy and a really great dinner despite my 17year old nephew, Shawn, being really obnoxious today. I love him but sometimes I don't know what to do with when he says I am dumb just because my favorite team and his aren't the same. lol. The worst one though is when he or his 14year old brother get frustrated over me saying no to them about something.
They have had it tough at times I know. My sister has definitely had her issues and didn't really make much attempt at being a good parent until almost 3 years ago and their dad wasn't a whole lot better. My mom, along with my stepdad, has pretty much had them their whole life and I try to do as much as I can but it's hard. Justin, the 14year old, lives here but visits both his mom's apartment and his dad's house regularly. My sister has been making alot of positive changes in her life though, especially after having Michael (he'll be 20months next week) and Shawn lives with her now. Justin tried to do that but still has a lot of resentment towards her and never stays more than a couple nights when he goes to visit. The problem is he gets jealous over both of his brothers at times and won't admit it so instead he comes home in a foul mood and takes it out on somebody (or sometimes everybody) here. Often this person is me, generally after I tell him "no" about something he wants me to do for him. Like I said, I try to do as much as I can but sometimes I have to say no. Like this week w/ the fibro flaring and a ton of stuff I've been trying to finish up for school with finals only 2 weeks away for most of my courses (or next week for music appreciation). Almost all of my time right now is school, trying to de-stress as much as I can, and arranging myself as comfortably as I can to try and rest. I have tried to explain but he doesn't understand why I spend so much time studying or how bad I feel sometimes. When I remind him how tired and sore I feel he smarts off that I am faking it.

I wish I knew how to word what I say so that he does get it and realizes when he acts like that it just stresses me even more and that =more pain!


On the plus side I turned in my last research project today & got my grade back on another. So far the ones I have back in English have all been max points as was my phishing scam article for my office system course and I got an A (no % or points listed though???) so now it's just finishing up some databases & power-point for OST & getting ready for tests. I can see the light to our month long break and I am ready!

Tuesday, November 24, 2009

my final paper of the semester

My first semester back in school after too many years to count is almost over & for my writing class we had to choose a topic, localize it to ourselves or our community, and write an informative and persuasive or argumentative research paper on it. As a lupus patient and pharmacology major it was pretty easy for me to choose my topic. There hasn't been a single new treatment aproved for lupus in over 50 years. Lupus research is underfunded federally and this has to change. I wrote my paper on pharmaceutical research and why there needs to be more attention paid to diseases like lupus. I also wrote some about my experiences as a research patient and being part of the clinical trials for Benlysta, which could be the first new medicine aproved for lupus since Eisenhower was president. I may be wrong but I am 99.9% sure it's also the FIRST medication successfully developed specifically for lupus treatment.

I posted the paper a few minutes ago in a much longer entry if anybody wants to see what I have learned about lupus, how pharmaceutical research works, and what it's like being part of the clinical trials and being one of the first to recieve a new drug (I have been on BenLysta since phase 2 trials began and was one of the first about 500 people to recieve the drug).

Researching New Treatments

The United States spend around 130 billion dollars each year on medical and pharmaceutical research. Why then has it been over 50 years since a new treatment for lupus has been approved and brought to market?

Before we can begin to figure out what needs to be done and why researching and developing new treatments for lupus is important it helps to understand a bit about what the disease is and how it affects people. Lupus is an autoimmune disease in which your immune system attacks your own organs and tissue much the same way it would an infection- basically you are allergic to yourself. Lupus can affect any organ in the body and among the most commonly attacked organs are the skin, joints, lungs, and kidneys. 90% of lupus patients are female, most often diagnosed during their childbearing years. There is no known cure and the symptoms of the disease are often life diminishing and potentially life ending, especially when involved organs include the kidneys, lungs, heart, or liver.

There are only three medications currently approved for the treatment of lupus: aspirin, prednisone, and plaquenil. All of these have dangerous side effects which can be as problematic as the symptoms they are intended to treat. Aspirin is a common cause of stomach problems ranging from nausea to severe stomach ulcers and also acts as a blood thinner which can make it more difficult for blood to clot when someone has an injury such as a cut. It is also one of the most common drug allergies seen.

Plaquenil is an anti-malarial with common side effects such as Nausea, stomach cramps, decreased appetite, diarrhea, dizziness, and headache. Other side effects often seen with plaquenil include fast heartbeat, hair loss/color change, mental/mood changes, ringing in the ears/hearing loss. Among the most serious side effects though are blurred vision and glaucoma. Then there is the most commonly prescribed drug for lupus- prednisone. Prednisone is a steroid medication which often causes changes in mood, weight gain, difficulty fighting infections, and brittle bones. As you can see, these drugs cause as many problems as they solve.
We desperately need to find newer, safer, and less toxic treatments for lupus. In order to do this we need to increase funding for research into new treatments for this disease and others like it.

I am a lupus patient and also a research patient. The primary medications for controlling and managing my lupus are Imuran, an anti-rejection drug being used off label to help prevent further kidney involvement and what I think of as my miracle drug, Lymphostat-B. Lymphostat-B, also known as BenLysta is an investigational drug currently being studied for the treatment of lupus.

Before discussing BenLysta and how it affects me specifically, it might be helpful to know a little about how pharmaceutical research and development works. There are many different things that go into developing new treatments and many different people involved at each stage from the conception of the idea to it becoming available for use. It starts with the scientists and pharmacists who develop an idea or model for a new treatment for a disease or class of diseases and ends finally with the patients who receive the drug during clinical trials that test for safety and efficacy of the treatment before the Food and Drug Administration will consider it for approval.
I interviewed Lu Anne Novello, the Director of Clinical Operations for Human Genome Sciences. Human Genome Sciences is the research group developing BenLysta in partnership with the pharmaceutical company Glaxo-Smith Kline. She has worked in pharmaceutical research for 27 years after graduating from the University Of Connecticut School Of Pharmacy in 1982 with her Doctorate of Pharmacy. Ms. Novello explained how research projects are chosen:
"Drug or biologics can be specifically engineered based on a model or an existing protein and then tested on a specific target. Or a compound could theoretically have more than one therapeutic target. In that instance preclinical studies (work in tissue or animal models) are conducted to help define what indications may have the greatest chance for success in humans. The identified disease areas are also evaluated to determine medical need. The following are considered: how many patients have the disease, how many other products are currently on the market and might your drug have a benefit (greater safety or better efficacy) than what is currently available. Often multiple indications for a product are pursued in parallel. The first one that completes the required testing will be submitted to the regulatory agencies as the initial NDA (New Drug Application) or BLA (Biologic License Application). Other indications are then submitted later in supplemental filings. She also explained to me that all treatments, both chemical and biologic, are subject to many levels of testing before ever being tested in human subjects during clinical trials. All new drugs are put through a series of preclinical experiments testing both the drugs potency and how it acts against different sets of cells. This is done first in a dish, then in rodents, and finally in primates before ever being tested in humans. At each stage of this process different doses are tested for safety and whenever possible for efficacy."

After the preclinical trials are completed the new treatment is ready for tests in humans. There are four different phases of clinical trials which are conducted in humans.

Phase one trials are done in a very small number of, usually healthy, people in order to test for safety (side effects and toxicity) and pharmacokinetics (level of drug in the bloodstream). At this stage there are generally a couple of different dosing levels evaluated in different subject groups. If these tests are successful the drug may move on to phase two studies.

During the second phase the drug is tested in patients with the target indication. This is the first time that the drug is used in patients who have the condition the drug is being developed to treat. Like in phase one, different dosages are looked at for safety but now it is being looked at for efficacy in humans as well. The researchers want to see if the drug behaves as predicted and is safe for patients who are afflicted with a given condition. Because phase two is in large part evaluating safety it is conducted in a small number of patients studies during this phase are considered “underpowered” meaning there is still a risk of statistical error. A drug could possibly be effective but appear not to be due to the small sample size, or vice versa- it may appear effective but turn out not to be any more effective than a placebo or a current standard of care.

Next, if phase two has been successful, is phase three which the new drug will be tested in a larger number of patients. This round of trials examines the drug’s efficacy against current standard of care as well as providing further data on the safety of the drug in a larger number of its’ intended recipients. There are generally at least two different phase three studies and all of these should show comparable results. If these trials show the drug to be safe, effective, and have the potential for improvement in the standard of care for patients then the data is submitted to the FDA and other regulatory authorities for approval.

In addition to Lu Anne Novello, I interviewed Kathy Benish. Ms Benish is the Research Administrator for Kentucky Research. Kentucky Research is a research department involved with conducting clinical trials and is a part of the Kentuckiana Center for Better Bone & Joint Health run by Dr. Steven Stern. She is also the nurse and coordinator overseeing the study I am in and explained the role of the doctor’s offices or clinics in the research process, “The Dr.'s office/clinic is where the trials are being conducted. We are pivotal in getting new drugs to market or approved drugs for a new indication to market, new treatments for patients, and offer treatment options to patients that otherwise would not be available”.

Kentucky Research is currently involved in several clinical trials for lupus and rheumatoid arthritis and in response to being asked what she saw as being most important to help increase the development of new, more effective, and safer treatments for diseases such as lupus Ms. Benish said patient education and involvement is important along and funding for research. “Cost is a huge factor in developing new drugs” then added, “Insurance companies are reluctant to cover patients if they are in a trial. This is a big factor in patient recruitment.” Insurance companies’ refusal to cover patients in studies adds to a patient’s out of pocket medical costs and acts as a deterrent to potential recipients sometimes making it harder to recruit participants for clinical trials. This is despite the fact the group developing the treatment covers the costs for treatment, labs, and visits to the study doctor.

Funding the research for new treatments is one of the biggest problems and something that needs to change. The National Institute of Health publishes its’ research expenditures on its' website each year. It lists what it actually spent on lupus research in 2008 as 126million dollars and includes a projection of 129million in 2009 and 130million in 2010. In contrast, the same page lists 245million in actual spending on herpes and related conditions in 2008 with spending predictions of 251million in 2009 and 255million in 2010.

I stated before that I am both a lupus patient and a research patient. I have been part of a phase four or post-market study for Imitrex and phase three studies for Relpax and Amerge. These are three different migraine medications. These have all come out in the past 20 years along with other medications such as Zolmig, Treximet, and several others. While I am grateful to have these drugs available it perplexes me as to why we have made so many advances in how to treat a migraine yet there has been nothing new for lupus?

Thankfully that may change soon. The study drug I am on for my lupus has successfully completed phase three testing and is expected to be submitted to the FDA and European regulating agencies in the first half of 2010 and may be available for doctors to use in the treatment of lupus patients later in the year.

This study has been very rewarding to be a part of in a couple of ways. First, it actually helps my lupus. I have been on this drug since before it actually had a name. It was simply LBSL-99 when I began receiving it during the first phase two trials over six years ago and was also referred to as Lymphostat-B, probably because it acts by inhibits the biological activity of B-lymphocyte stimulator in order to suppress the immune system. Before receiving the drug I had many days where I was in so much pain and so ill I could not even walk down the stairs at home, much less work or think about finishing my degree. After the first few treatments I began to feel an improvement in my pain and energy levels and my labs started to improve. After six months I tried returning to work and was able to survive the Christmas season in retail. I still have bad days or weeks at times but thanks to BenLysta, my miracle drug, those days are fewer and more manageable.

The real reason why this study has been so rewarding for me is knowing that by participating in this trial and acting as a “human guinea pig”, as my friends have referred to me before, I am helping other lupus patients. I get asked quite often by my fellow “lupies” what the medication is like and if it really helps. Without a doubt it has helped me and when one of my friends who also has lupus thanked me for trying the drug out first it made me smile because I realized that I wasn’t just helping myself, I was helping them too. One of the comments made by Kathy Benish in her interview was, “without patients, we could not do the trials and new drugs would not come to fruition”.

Participating in the trial for BenLysta has taught me many things and has motivated me to return to school and pursue a degree in pharmacology. Even more, it has shown me that there is something that can be done to improve the quality of life for the millions of people worldwide who are living with lupus. We just need to prioritize how we’re spending precious research dollars and pay a little more attention to a disease that is life diminishing, and in about 15% of cases life ending.

Sunday, November 22, 2009

Pit Bulls

This is another paper I wrote for my writing class, and it's about a topic very dear to my heart. I hope maybe that I can help open a few eyes to what wonderful dogs pits can be if cared for properly and if they have people who are willing to take the time to learn about these wonderful dogs!

About Pit Bulls & Why They Shouldn't Be Banned

Before anybody seeks to outlaw or ban something, shouldn't they first be able to define it? Then, if they can define it, shouldn't they seek to understand it?

There are many people, on both sides of the debate surrounding "pit bulls" and the issue of whether or not they should be banned. A large percentage of those people can't accurately define a pit bull or don't even realize that the term "pit bull" doesn't refer to a specific breed but rather a loosely defined group of breeds.

According to a February 2007 article titled "Is This Dog Dangerous" written by Cameron Lawrence for Louisville Magazine, "The pit bull is not recognized as a breed per se but rather as a type - a descriptor of several breeds of dogs with similar physical characteristics." The article goes on to say this commonly includes the American Pit Bull Terrier, the Staffordshire bull terrier, and the American Staffordshire terrier among others.

Generally pit bulls are described as "a medium sized, squarish dog usually weighing 40 to 60 pounds with a short coat revealing their strength. They are sometimes referred to as 'dog body builders' or more commonly 'the bully breeds". These dogs also tend to be "intelligent, confident, and loyal" though their critics say they are "hyper-aggressive and unpredictable". (Lawrence)

My experience, as someone who has had dogs her entire life including three American Staffordshire Terriers in addition to a Basset Hound, Maltese, Cairn Terrier, Beagle, and Dobermans to name just a few, is that these dogs are extremely loyal and intelligent dogs that will do anything to please their owner.

As stated by Lawrence, this group of dogs descended from breeding a mix of various bulldogs and terriers. Several of the resulting breeds were recognized by the American Kennel Club (AKC), begin with the American Staffordshire Terrier in 1936, and have set and recognized breed standards. The breed standards define the ideal build, color or markings, and temperament for the breeds.

The so called "bully breeds" fall in the terrier group which includes dogs such as Yorkies and Scotties. The terriers, as a group, require training, discipline, and an owner who knows how to handle them since, again as a group, terriers are generally described as being "strong willed".

Marcy Setter is the director of education and public relations for a large rescue group known as Pit Bull Rescue Control which is based in Missouri. Lawrence spoke with her during research for his article. She points out that pit bulls are merely the latest dogs to be targeted as dangerous or vicious and that the fear of aggression toward people is overblown, "This is really easy. It rotates every 10 years or so. Back in the 70's it was the German shepherds. In the 80's it was the Dobermans. In the 90's it was the Rottweillers. Now it's the pit bulls." She pointed out that while pits may have some tendencies toward dog aggression other breeds, including the Great Pyrenese and Australian Cattle Dog, have been bred for that same tendency. She insists "pits were never bred for human aggression". (Lawrence)

My personal experiences as a dog owner, and specifically a "pit bull owner" back up this assertion as well as the description of them being intelligent and loyal. For example our first "pit" was an American Staffordshire Terrier named Caesar. We had Caesar when I was in middle school. He was a very intelligent and well behaved dog who went a lot of places with me including walks to the corner store after school most days. The owner even allowed him into the store while I shopped because he was so well behaved. One such afternoon, after completing our shopping, we were headed back home when a clearly intoxicated man bumped into me. Caesar was, of course, protective of me. When the man bumped me, Caesar rose up on his hind legs and used his front paws to push the man away from me but didn't attack or present any vicious tendencies whatsoever, just a message to "back off and leave her alone". The man was a bit startled by this but did continue on his way leaving us to continue on home with no further incident.

Lawrence interviewed Linda Laun, a local dog trainer, who had this to say "some breeds have a heightened alert mechanism" and "these dogs are a bit weary of unusual circumstances, less accepting of new experiences." This trait, she says, can make them useful as guard dogs. She includes "German Shepherds, Dobermans, and some types of pits" as being examples of such breeds. However she adds "these breeds have no more propensity for causing harm, or likelihood of causing harm than a Golden Retriever, a Yorkie or any other."

I can attest to that as out of all the dogs I have ever owned or known only one has ever been determined by animal control authorities to be a "dangerous dog". The dog wasn't a pit, a Doberman, or any other medium to large dog. It was my dad's seven pound miniature pinscher, Bud who bit, of all people, the homeowner's insurance adjuster. Yet, nobody is seeking to ban miniature pinschers or any other small breed.

Dangerous dog ordinances have been passed in cities across the country. Some locales, such as Denver, Colorado and Bracken County, Kentucky have instituted breed-specific laws or bans that are aimed at "pit bull dogs". (Lawrence) These laws fail to recognize that any breed or individual dog has the potential to be dangerous and cause harm to a person or another animal.

PETA (People for Ethical Treatment of Animals) is a large and well known animal rights organization that supports a ban on ownership and breeding of pit bulls "as long as they include a grandfather clause allowing all living dogs who are already in good homes and well cared for to live the remainder of their lives safely and peacefully". According to PETA pit bulls are the most abused group of dogs in the United States. They use that statement to justify their support of banning these dogs. They also state that a large portion of people who seek to own pit bulls do so because they "are attracted to the 'macho' image of the breed as a living weapon". These people then encourage aggression in the dogs by abusing them in various ways including physically restraining the dogs with heavy chains in all weather extremes as well as kicking or beating them to make them tougher and thus more aggressive. They are basically stating they want to ban pits to protect them from cruelty but that would be punishing the dogs for the bad behavior of people and is not the way to solve the problem.

Despite their support of laws banning them, PETA does do some admirable work with pit bulls such as rescuing them for abusive or negligent owners in addition to speaking out against dog fighting, and subsidizing spay and neuter programs. They admit that pits can be loving companions and the problem is with or caused by cruel and irresponsible owners. (PETA)

Why, then, punish the dogs? Shouldn't we instead punish the people who are mistreating them and encouraging this aggressive and overly territorial behavior?

PETA contacts "cruelty case prosecutors" when they rescue abused dogs, which staffers and volunteers do everyday. They have personally witness thousands of pit bulls who have been treated reprehensibly by their owners. Dogs like "Bear who was permanently chained outside and suffering from several painful conditions, including flystrike and a skin condition his owners tried to 'cure' by pouring motor oil all over him".(PETA) Bear wasn't the problem here, his owners were. They were the ones who were cruel and caused harm, not the dog. Again, why punish the dogs for the actions of people?

Perhaps, instead of supporting laws banning pit bulls, it would make more sense if PETA and others worked to strengthen laws that protect dogs from abuse and promote harsher penalties on irresponsible owners who subject the animals to the abuse and neglect often witnessed by shelter and rescue group volunteers.

Here in Louisville there have been proposals to outlaw pit bulls and in January of 2007 the city council instituted re-vamped animal control laws. Due to protests and outcry from owners, breeders, and the local branch of the AKC the breed specific wording was removed and the law applies to all dogs and their owners. It restricts the selling of "dangerous" and "potentially dangerous" dogs as well as defining what constitutes a "dangerous dog". The law defines a dangerous dog as "any dog that has been used in dog fighting, injured or killed a person in an unprovoked attack, a dog that attacks or injures other pets or livestock, or is used in the commission of a crime. It defines a potentially dangerous dog as one that "bites or harms someone in an aggressive manner or attacks and injures another pet or livestock."(Lawrence)

Again, I've had dogs all my life and the only one I've ever known that has been declared by animal control to be "a dangerous dog" is a tiny, seven pound miniature pinscher.

As the International Association of Canine Professionals (IACP) says, "breed specific legislation does not protect communities nor create a more responsible dog owner" and "limiting the risk of dog bites should be the legal responsibility of the dog owner". They point out that it is more than the breed or physical appearance of a dog that determines its disposition and personality. The factors involved include, but are not limited to, genetics, training, socialization, health, supervision, adequate shelter, and general care a dog receives.

Thus, it is logical to say, as stated in the IACP's position statement, "The vast majority of dogs typically affected by breed-specific legislation are not 'dangerous" by any standard" and should not be judged by phenotype or physical appearance. Laws directed at any specific breed unfairly punish not only the dogs but also responsible dog owners which includes the majority of people who own any dog. This is a point worth repeating. Don't punish a dog just because of how it looks. In my experience, just as with people, a dog's appearance can be quite deceiving.

At the same time we had Caesar we had a small dog too. Peppy was a Cairn Terrier and maybe about 6 inches tall. (For those who aren't familiar with them, Cairns look similar to a Scottie). Caesar, by contrast was 22inches at the shoulders and his jaw muscles made it look like he had baseballs in his mouth. Peppy, however, was the "mean one" and was much more likely to snap or bite. Peppy actually did bite my sister twice, Caesar never so much as looked at one of us the wrong way. This, I think, helps to demonstrate why you should never judge by appearances.

There is no such thing as a bad breed but there are many bad owners. It is up to people to be a responsible owner and take proper care of their dogs. This includes obvious things as providing proper shelter, food and water but it also includes less obvious things such as training, socialization, and supervision of all pets. It also includes strengthening (and enforcing) "dangerous dog" laws which are not breed specific and animal cruelty laws that help to protect dogs from the harm they suffer at the hands of people.

We have four dogs in our house right now. Among them we have Stacey, a Border Collie mix, and Dillon, my American Staffordshire Terrier (one of the "pit bull types"). Stacey absolutely bullies Dillon. If he walks past her dog bowl she stands guard over it and lets out a low growl, if he gets near a treat or toy she thinks is hers, she snaps or growls at him. (Actually she does the same thing with the other dogs in the house too, she is definitely the "alpha dog" in the group). Dillon is, by far, bigger and stronger than she is yet he is cowered by her and will tuck his tail and run away if she even looks at him sideways.

Stacey and Dillon were playing in the back yard one afternoon, chasing two tennis balls I was throwing for them and having a lot of fun. I needed a quick break so I tossed the balls to them and went in the house to get something to drink. I was inside for about five or ten minutes then went back outside where I found Stacey had decided both balls were hers. She had one tennis ball in her mouth and the other between her front paws while she stared Dillon down, watching every move he made. When he would come near, or when she thought he was trying to come near and take one of the tennis balls Stacey would let out a low growl that steadily got louder. Dillon's reaction to this was to look at her like he was pouting, tuck his tail, and slink up onto the deck where he lay down at my feet not daring to go back out into the grass and try to play any longer.

My big "bully" is definitely not the bully in this house and despite what many claim, I have never witnessed any of the pits I have known be the bullies they are made out to be. That title has always fallen to the dogs who are supposed to be "harmless", not the big, bad "pit bulls".

Banning pit bulls is not the answer. Teaching and enforcing responsibility and accountability in the people who raise not only pits, but all dogs, is the only answer there can be.

Works Cited

Lawrence, Cameron. "Is This Dog Dangerous?" Louisville Magazine. February 2007.

Web. 24 August 2009

"Position Statement on Breed Specific Legislation"

International Association of Canine Proffessionals. IACP, 2006. PDF file.

"PETA: Position On Pit Bulls" PETA.
About PETA. Web. 25 August 2009.

my interview w/ a research nurse

This is the second interview I conducted while working on my project about pharmaceutical research. This time I interviewed Kathy Benish who runs the Kentuckiana Research Center with Dr. Steven Stern. She is a nurse, holding her masters of nursing as well as being a registered nurse, and has worked both in bedside nursing and clinical research.

1) What is the most rewarding part of your job? The most challenging? One of the most rewarding parts of my job is that due to my involvement with Research, I can be a part of changing the way medicine is practiced in the future. When I was bedside nursing, I felt my major impact was with the patient and their families. While I feel that this is very important, what we do affects people on a global scale. Some other rewarding benefits is getting to see the progression of the patients in the trials & being on the cutting edge of your specialty.

Most challenging parts are being the mediator & contact person between the patient & the Dr., the Dr. & the Sponsor of the trial, the CRO (Contract Research Organization) and the Dr., the IRB (Internal Review Board), and following the protocol (how the study is to be conducted). Sometimes I feel I am being pulled in a hundred directions, all for the same cause. Everyone wants things their way and sometimes that is just not possible. Then you have to remember that you are dealing with patients who don't always follow the rules for whatever reason(because they forget, they don't want to or they get sick & can't). Patient recruitment can also be very challenging.

2) How are research studies chosen? Clinical Trials are sponsored/funded by a variety of organizations or individuals such as: Physicians, Medical Institutions, Foundations, Voluntary Groups and Pharmaceutical Companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD) & the Department of Veteran's Affairs (VA). In our office, we choose trials that look at the disease processes that are pertinent to our practice.

3) Who plays a bigger role in funding research, the government or the pharmaceutical companies? This is a toss up. In our world, it is the Pharmaceutical Companies.

4) What part does the research center or Dr's office play in treatment development? The Dr.'s office/clinic is where the trials are being conducted. We are pivotal in getting new drugs to market or approved drugs for a new indication to market, new treatments for patients, and offer treatment options to patients that otherwise would not be available.

5) How would you explain the difference in phase1, 2, or 3 trials to somebody who had never participated in a clinical trial? Phase I: is the initial introduction of an investigational new drug into humans. First time tried in human beings, closely monitored, and designed to determine the metabolism and actions of the drugs in humans. Phase II: evaluates the effectiveness of the drug for a particular indication (like SLE or RA) to determine the common short-term side effects & risk associated with the drug. Phase III: are intended to gather additional information about the effectiveness & safety that is needed to evaluate the overall benefit-risk relationship of the drug and to provide an adequate basis for physician labeling (dose, route, etc). Usually done on a larger number of patients than the Phase I & II trials & monitors side effects closely. Phase IV: these are post marketing studies (drug has been approved) and are done to gather additional information on risks, benefits and optimal use.

6)What do you see as the most important thing that can be done to help increase the development of new, safer drug therapies for chronic illness? Patient education about trial involvement is important. Without patients, we could not do the trials and new drugs would not come to fruition. Money for research. Cost is a huge factor in developing new drugs. Insurance companies are reluctant to cover patients if they are in a trial. This is a big factor in patient recruitment. Security is also crucial. Researchers spend millions of dollars developing a drug only to have it stolen or sold to other companies & countries. Punishment for this needs to be swift and firm. I am sure there are many other factors beyond my comprehension but these are a few that come to mind.

7)What new treatments are you involved with studying and what phase are the trials in? We are studying several new drugs for Rheumatoid Arthritis, Lupus and getting ready to start a Gout trial and possibly Osteoarthritis Trial. We do not do Phase I trials in the office. Our trials are Phase II & III. We have a Phase IV trial that we have been doing for approx. 5 yrs that is also being used as a Registry for several disease states that allows Dr.s to see how others in their fields are doing things.

My argument for & research on healthcare reform

This is a paper I wrote on healthcare reform and what I think needs to be done.

Healthcare Reform in America and Why We Need It

We have all heard the rhetoric on the evening news about healthcare reform and we have all seen the commercials promoting different viewpoints on the issue. What exactly are they trying to change though and how do the people (i.e. the patients and healthcare providers) feel about what needs to be done?

Healthcare is one of our biggest national expenses yet millions of Americans are without easy access (or in some cases any access) to quality healthcare. Health insurance companies make billions of dollars in profit each year according to the stories we see on TV or in the newspaper while denying coverage to millions, like me, because they either have a pre-existing condition or are seen as "high risk" due to things such as age, family background, past illness, or family history of particular illnesses. In essence saying "you can't have health insurance because you might actually need to use it". There is no perfect solution to our healthcare crisis but something must be done to allow more Americans access to quality medical care- both treatment for illnesses and preventive care to minimize the risk that a person will suffer from a catastrophic illness.

The website includes a page outlining President Obama's goals on this matter. It lists quite a few but chief among them are:

  • Ending discrimination by insurance companies against people because of age, gender, or preexisting conditions.
  • Capping out of pocket expenses so people don't go broke because they get sick
  • Provide tax credits to help people buy insurance
  • Immediately offer new, low cost coverage through a national "high risk" pool to protect people with preexisting conditions from financial ruin
  • Require large corporations to cover their employees as well as requiring people who can afford to do so to buy insurance so that everyone shares in the responsibility of reform
  • Order immediate malpractice reform projects that could help doctors focus on putting their patients first, not on practicing defensive medicine.

The first health insurance plans resulted in a downturn in the U.S. economy in the 1920s leading up to the Great Depression. During the 1920s and the decade prior medical knowledge and technology had increased rapidly and for the first time the requirements for being a physician had become stricter requiring more formal education and training. These were, of course, important developments in human welfare but also added to the expense of treating illness and by the '20s, for the first time, a week's hospital stay became more expensive than the average American family's monthly income. People couldn't afford to get sick. Economist Lou Reed said at the time, "Very few of these families are indigent in the accepted meaning of that word. They have a home, they buy their own food and clothing and pay their doctor's bills in ordinary illness but when serious illness occurs, these families are unable to pay their way." (Cohn. 2008. p.6.) A blue ribbon commission, after spending five years conducting the first national census on healthcare, recommended that Americans share collective responsibility for medical costs. "In other words, it recommended the creation of insurance for medical care". (Cohn. 2008. p.6)

Industrial countries, other than the U.S., were beginning to guarantee the availability of medical care to their citizens either through government sponsored organizations or directly through the government itself. They were making healthcare a right rather than a luxury. In the United States this was strictly opposed by physicians worried about outside interference in how medicine was practiced and also by large corporations which feared government managed medical care would lead to interference elsewhere in the private sector. (Cohn 2008)

Patients weren't the only ones suffering though. Hospitals were also having financial trouble. Baylor Hospital in Dallas, Texas was one such hospital and had a mounting debt. They also had a new and quite innovative administrator, Justin Kimball. Kimball had come to the hospital from the Dallas public school system and decided to approach his former co-workers with an offer. He proposed that any teacher who agreed to a donation of fifty cents per month would be guaranteed coverage for a hospital stay of up to 20 days so long as a minimum of three quarters of the teachers agreed to join. Kimball had no problem recruiting teachers to join in his plan and the first health insurance plan was born. (Cohn. 2008)

The original intent of health insurance plans was simple, to help people be able to seek medical treatment without going broke and assist the hospitals being viable and able to stay open and available to care for people when they got sick. Everyone involved benefited it seems. What happened to change insurance from a mutually beneficial arrangement then, and why did it happen?

By the early 1980s most American families were covered by employer sponsored health insurance plans and the benefits had become more comprehensive in the extended years of prosperity following the end of World War 2. As the benefits expanded they grew to include wellness and preventative screenings and the premiums had grown to reflect the expanded role insurance was playing in healthcare. (Cohn. 2008) Given the sustained prosperity over the past several decades why would either the employers or beneficiaries mind an increase in cost given that there was also a continued growth in income? During the '80s though the U.S. economy stalled and manufacturing jobs began to disappear. Businesses began to look for ways to cut costs- one of those costs being how much they spent on healthcare coverage. (BNet. 2009.)

By the 1990s the majority of companies seeing economic success were companies like Wal-Mart which had reduced their employee benefits to the bare bones and enacted policies that limited access to what benefits they did offer. Sometimes these limits were so severe as to require an employee be a full time employee of a company for a period of up to two years before qualifying for medical coverage! (BNET. 2009) Despite having a job my entire adult life I never had insurance coverage from 1991-2000 so can remember these policies all too well.

Companies, such as General Motors, which were bound by agreements with their employees' union to provide health insurance for their workers paid a very high price which was usually passed on to their customers. For example in 1993 GM said, "health insurance for its' employees alone added more than seven hundred dollars to the price of every car and truck". By 2004 that number had grown to $1400.00 per vehicle. (Cohn. 2008) While these costs were being absorbed by businesses and consumers however health insurance was growing to a multibillion dollar industry with steep profits and a reputation for denying benefits to the people it was purporting to protect.

Insurance had changed. It had begun as just what its' name implies- insurance that people could get sick without going broke and insurance that hospitals could afford to operate and care for those same people. It had grown it would seem, to be in the business of insuring its own financial health rather than insuring people's physical health.

Today the increased cost of medical coverage has left millions of Americans without coverage and at risk. The risks for people, like myself, without coverage are many but topping the list would have to be increased risk of complications from illness coupled with financial ruin. The premiums for individual health coverage are high and unaffordable for many people- if they can find coverage at all. As we see on the evening news, millions are simply denied coverage due to having a preexisting illness or being seen as being at risk for developing a serious illness in the future.

As a lupus patient, I am one of the millions of Americans who are seen as "uninsurable" but at least in my case they can give me a reason. It doesn't make the lack of coverage any easier, nor do I think it's fair that I am denied coverage over a condition I had no control over developing. At least I know why they won't sell me a policy though. Millions are turned down simply because insurers think they could become sick later thus actually using the policy they are paying for. Among the reasons I have heard people being considered as "high risk" are age, income level, and family background along with their personal medical history.

Having an insurance policy doesn't guarantee anything though. Insurance companies routinely deny payment for medications and treatments. I spoke with a fellow patient in my rheumatologist's waiting room who was waiting to pick up paper work she needed to submit to her insurance company. We'll call her Mandy. Mandy has lupus with severe organ involvement and the Dr had recommended Cytoxan to try and slow the damage to her lungs and kidneys. Cytoxan is primarily a cancer drug though and its use in lupus patients is considered "off label". (Off label is a term used when a medicine is being used to treat a different condition than what it was developed and approved by the Federal drug Administration.) Most drugs currently used to treat lupus are "off label" (primarily chemotherapy drugs or anti-rejection medications) but are used because they have proven beneficial in lupus for many of the same reasons they are beneficial in treating a disease such as cancer or helping to prevent organ rejection, they suppress the immune system and in lupus (and all autoimmune diseases) by suppressing the immune system they slow the progression of and damage from the disease. Well, Mandy's insurance company had denied coverage for the Cytoxan because they said it was experimental despite having been used successfully in thousands of patients to treat lupus. She and the doctor had been fighting with the insurance company to approve the treatment for six months and had yet to succeed. (personal communication. October 2009) Cytoxan is an expensive course of treatment to be sure, but surely less costly than what further damage to a person's kidneys or lungs would seem.

In addition to speaking with Mandy informally while we were waiting at the doctor's office I also conducted two more formal surveys over the past few weeks, one of patients in my online lupus support group and in an online support group for fibromyalgia and the second of healthcare providers including two doctors here in Louisville, a nurse practitioner, and a pharmacist in Lexington, KY. I asked both groups what they see as the biggest obstacle in the U.S. healthcare system. Everyone agreed that it was the insurance companies. Patients said they have had to appeal decisions to deny payment for hospital stays and tests ordered by their doctors as well as the myriad of paperwork required to get approval for scheduled procedures recommended by their doctor or nurse practitioner (ARNP). Providers said they spend countless hours that should be spent caring for their patients dealing with an archaic amount of paperwork required to both receive payment for their work as well as to convince an insurance company of what treatment is best for their patient. One doctor said " the red tape involved and stonewalling by the insurance companies in receiving approval for newer, more effective treatments to help my patients often causes more stress for my patients and a higher long term cost because delay in treatment often leads to a longer and more complicated recovery". Again, it seems to me as if it would be more cost effective to approve more effective treatments and do so faster rather than prolonging the illness and thus requiring a longer (and often costlier) course of treatment. (personal communication. October 22, 2009)

Patients other major concerns with our healthcare system were fear of losing access to treatment and fear of not being able to afford getting sick. The group included patients with private or employer sponsored insurance, patients with government sponsored care (Medicare/Medicaid), and patients with no coverage. The patients without coverage admitted to going longer than recommended between visits to the doctor, putting off lab tests, and either not following their treatment plan or requesting a less expensive treatment even though it could be less effective (I have done this myself as well). The patients with insurance were a bit better about following their doctor's orders but feared what would happen if they lost coverage and both sets of patients were afraid of what would happen if they were to have a serious injury or complication as most insurance plans have high deductibles, maximum allowed benefits, or both. (personal communication. October 22, 2009) In addition to being sick they feared they'd also be bankrupt, the very thing that was behind the beginning of modern health insurance.

I asked providers what they would recommend if they were asked by Congress or President Obama to consult on healthcare reform. There were two answers that really stood out to me. The first was the subject of tort reform and malpractice laws. All agreed that there need to be protections and limits passed on malpractice lawsuits. They said that fraudulent malpractice suits cost both them and their patients more because of the rising malpractice insurance rates and all agreed that even were it not required by law they would still maintain malpractice insurance to protect themselves and their families. (personal communication. October 22, 2009)

I interviewed Dr Tad Seifert, a neurologist here in Louisville who pointed out that the tort reform laws in Texas has greatly reduced the number of frivolous lawsuits while still allowing the legitimate cases to be heard and in doing so has brought down the cost of malpractice insurance and increased the number of healthcare providers per capita in the state. The other thing I heard from them was a recommendation to expanding the qualifications for Medicaid. Specifically one doctor said he supported in raising the allowable income for families and individuals to allow more people to qualify for Medicaid. According to the information he provided currently a family of four is only eligible if they have less than $21,000.00 per year in income. Dr Seifert recommended raising that to $35,000 as well as saying he would support expanding the Medicaid system to allow uninsured Americans with chronic illnesses to be included even if they aren't disabled and even if they are single with no children. (personal communication. October 22, 2009). I agree with this as it would expand coverage and the current limits on both income and disability status seem to encourage people not to work. For example, because I am single and want to do basic things like go to school and have a job for as long as I am physically able I don't qualify for any type of medical assistance but under his idea I would be able to qualify for Medicaid without also having to be on disability (everyone who is on state or federal disability is automatically covered by Medicaid or Medicare). I would no longer be punished for wanting to be as responsible and productive a citizen of my community as I am capable of being.

The U.S. is the only large, industrialized nation in the world that does not guarantee all citizens access to medical care. Canada has found a way to do so, as have Britain and France. Even Cuba has found a way to do it. Why then can't (or won't) the U.S. do the same? Do we value corporate profits above the health of our citizens?

BNET. (2009) The Rising Cost of Healthcare: Strategic and Societal Consideration For Employers. Retrieved from

Cohn, J. (2007) Sick. (2nd ed). New York. Harper Collins

Organizing For America. (2009) The Obama Plan. Retrieved from

Interview With Lu Anne Novello of HGS

As part of my coursework this semester we were asked to research a topic of our choice and write an informative and argumentative/persuasive paper on the topic as the main part of our final assignment for our writing class. As a lupus patient and someone who has been a patient in clinical trials of new medications and treatments for lupus and migraines as well as a pharmacology major I chose pharmaceutical research for my topic. I intend to explain the basics of how the research process works and argue for why there needs to be improved funding for researching treatments for diseases such as lupus, a disease that has not had a new treatment aproved in over 50 years despite affecting more people than MS or AIDS.

After some preliminary/background reading on the topic, I contacted people working in the pharmaceutical research industry. The first Interview I am publishing was conducted by email and is with Lu Anne Novello Phd who is the Director of Clinical Operations for Human Genome Sciences, the research group responsible for developing BenLysta. Benlysta is the first treatment designed for lupus to success fully complete phase3 clinical trials in 5o years and will be submitted to the FDA for aproval in the first half of 2010.

How long have you worked in pharmaceutical research? I graduated from University of Connecticut School of Pharmacy in '82 . I had been lucky enough to have two internships in industry while I was in school and one of them was in clinical research. I loved it and I knew right then that I didn't want to work in a hospital or retail pharmacy. I went straight from school to a job in I guess that makes it over 27 years now!

What is the most rewarding part of your job? Being part of a team which brings an important new drug successfully through the research, development and regulatory approval process in the most rewarding part of my job When I hear first hand that a drug I helped to develop has made a difference in someone’s life it is a great feeling.

The most challenging? The most challenging part of the job is how long it takes to bring a new drug from the discovery stage to the point of being available to patients. The number of drugs that fail along the way is far greater than the number which are eventually approved. You may work on a product for years only to find it fails in the later stages of testing.

How are research topics (drugs or diseases) chosen? Drug or biologics can be specifically engineered based on a model or an existing protein and then tested on a specific target. Or a compound could theoretically have more than one therapeutic target. In that instance preclinical studies (work in tissue or animal models) are conducted to help define what indications may have the greatest chance for success in humans. The identified disease areas are also evaluated to determine medical need. The following are considered: how many patients have the disease, how many other products are currently on the market and might your drug have a benefit (greater safety or better efficacy) than what is currently available. Often multiple indications for a product are pursued in parallel. The first one that completes the required testing will be submitted to the regulatory agencies as the initial NDA (New Drug Application) or BLA (Biologic License Application). Other indications are then submitted later in a supplemental filings.

Who decides what to fund and who actually plays a bigger role in funding, the government or the pharmaceutical companies? Typically pharmaceutical/biotech companies make the decisions and fund their own research. In some special instances drug are developed in conjunction with the government and the government will fund the project. This may happen when there is a need for a drug that may not be commercially viable. An example is a drug to protect against a biological attack such as anthrax.

What is the process in developing a new drug and getting the drug to a stage where it's ready for trials in humans? New drugs and biologics are subjected to preclinical testing first. This is where you get the first indication of a drugs potency and activity. A drug can be tested against certain cell lines and tissues in a dish to see if the predicted activity is observed. From there the compound is usually tested in rodents to determine what dose is safe and if the predicted activity is maintained. Often an animal model representative of a certain disease state is used to better understand what effect might be seen in humans. If the drug is determined to be safe and have the predicted activity in rodent studies, the compound will next be tested in primates. Once again different doses will be evaluated for safety and if possible, a determination of potential efficacy. If preclinical studies done in primates are successful the drug is ready to test in humans.

How would you explain the difference in phase1, 2, or 3 trials to somebody who had never participated in a clinical trial? Phase 1 is the first time the drug is tested in people. Phase 1 trials are usually conducted in healthy volunteers that do not have the disease which the drug is intended to treat. In Phase 1 trials the focus is on safety (what side effects does the drug produce and pharmacokinetics (after the drug is administered what levels does it reach in the subject’s blood. Based on the preclinical studies different doses (lower and higher) are evaluated. If the drug has an acceptable profile in Phase 1 studies, the next step (Phase 2) is to evaluate the safety of the drug in patients with the target indication. Efficacy of the drug to treat the indication is also evaluated in Phase 2. Patients enrolled in the trial will be divided into groups and treated with either the investigational drug or a placebo/current standard of care. Phase 2 trials are smaller studies. Large numbers of patients will not be exposed to an investigational drug until safety and efficacy have been shown in a small number of patients. Because of this, Phase 2 studies are “underpowered” which means there is a statistical risk of arriving at the wrong answer at the end of the trial. The drug may actually be effective, but due to the small number of patients tested the effect may not be seen in a phase 2 trial. Or the drug may appear to be effective and in reality it may not be statistically different than placebo or the current standard of care. Any significant safety concerns seen in a phase 2 trial may limit the potential for a drug to go on to Phase 3 testing. The safety risk must be weighted against the medical benefit of the drug to the patient. In Phase 3 trials large numbers of patients will be treated with the investigational drug. As in Phase 2 patients enrolled in the trial will be divided into groups and treated with either the investigational drug or a placebo/current standard of care. Usually two Phase 3 trials must be conducted with the drug and both trials should have similar results for efficacy and safety. If both studies show the drug is effective and the safety profile is acceptable, a licensing application is prepared and filed with regulatory agencies (like the FDA) for review. The regulatory agencies review the data generated through all phase of the drug’s development and if they agree the drug can be cleared and made available to patients

What do you see as the most important thing that can be done to help increase the development of new, safer drug therapies for chronic illness? A balance must be maintained between the regulatory process necessary to protect the health and welfare of patients and the development path for new medical therapies. Clear communication between regulators and industry is needed to avoid costly (both time and money) diversions. Regulatory agencies must be adequately staffed to insure complete and timely review of data submitted by industry. Government and Industry must work together to achieve cost efficient solutions which move development forward while maintaining the responsibility of protecting human life.