Since those days of uncertainty, the disease has ravaged millions, though in its wake followed a continually growing understanding of the virus and how it works.  I remember talking about it in high school several times, mainly, I think, for the purpose of empowering us to make smart decisions as we awakened to new knowledge of ourselves and of our sexuality.  It seemed everyone agreed that the best way to defeat the disease was to be steadfast in our resolve to prevent its spread and continually educate people on how to accomplish this task.

Unfortunately, however, education didn’t always work, and for those that succumbed to the disease, medicine offered many ways to combat its effects.  People soon were able to live many more years than they ever had before, though the illness still lingered in a body that had to be maintained by a cocktail of drugs.  And while both education and drugs are available in abundance in the US, other countries, especially those in Africa, have been much worse off because of the lack of resources.

Perhaps we are finally approaching a time when the disease can be eradicated rather than just held at bay.  Boston.com reported on November 12th that German hematologists Eckhard Thiel and Gero Huetter of Berlin’s Charite Medical University may have developed a method for defeating the full-blown virus.

Dr. Huetter had been treating a 42-year-old American patient living in Berlin for leukemia, which was unrelated to the AIDS infection in his system.  When screening possible donors to treat the cancer, Dr. Huetter remembered a study he read in 1996 that posited that certain people carry a genetic mutation that makes them resistant to the HIV infection.  If a person inherits the mutation, called Delta 32, from both parents, it prevents HIV from attaching itself to cells and spreading throughout the body.  The doctors treating the patient screened 80 donors for the mutation, and found one that both matched the patient’s marrow type and carried the special mutation.

Prior to the transplant, the patient followed a radiation treatment to eradicate his own infected bone marrow cells and disable his immune system.  Because Huetter’s team feared that the drugs might interfere with the treatment, he was also weaned offf his AIDS drug cocktail.  After the transplant and in the 20 months since, the patient has shown no signs of the AIDS virus.  Though researchers and the doctors themselves admit that the results could be no more than a fluke, it does offer hope that a breakthrough has been made in the treatment of the disease.

The thought that we may be approaching a “cure” for AIDS is astonishing.  Have we really made such inroads in 30 or so short years that a treatment could destroy the disease instead of just treat its symptoms?  We haven’t been able to create a “cure” for the cold or the flu (which our bodies can thankfully handle if they’re strong enough), but we’re now approaching a treatment for something that to me seems infinitely more complex and deadly because of its aggressive nature.

Certainly, a better understanding of what happened needs to occur before the method can become an accepted way to treat AIDS, and more research needs to be done in the dangers of this treatment and how they can be negated in the future.  Destroying the patient’s immune system before initiating a transplant of new marrow cells, for example, carries with it a high risk of mortality by itself.  Coupled with the other parts of the treatment, it’s miraculous that this person even survived.

Then again, cures for what afflicts us never come without risks.  Countless cancer patients probably wouldn’t be around today were it not for experimental, risky procedures.  The first heart transplant, artificial heart, or even recent successful face transplant are all examples of risky medical procedures that yielded results that improved both quality and longevity of life for their patients.  It may take some time before the procedure is perfected, but there is no reason that with some genetic research, AIDS can’t be on that list of curable ailments.

Now, if we could only figure out a cure for that common cold.

I think the surprise must have registered pretty immediately on my face, because she seemed almost apologetic after that; not apologetic for her views as a republican, but for the shock she had just imparted on me. For me it was just more of a surprise that my assumption was incorrect.

I remember the first time I really cared about politics was the election of 1984, a landslide victory of Regan against Mondale.  I was around 10 years old at the time and remember arguing with my grandparents, whom I had discovered had voted republican, about why they were wrong to have voted the way they did.  I didn’t understand anything about politics and only had a vague understanding of the classic Democratic platform vs. the classic Republican platform, but I remember being so passionate that I actually tried to convince them that what they were doing was bad for the country.  Seriously?  What did I know?

The fact is, that response may have been pre-programmed in me, according to a new study I just read about in the Boston Globe.  The article, by Eve LaPlante and published on November 2nd, argues that our reactions to politics may be more related to how we feel in our gut about an issue more than our rationalization of it.  This theory and the associated study, coauthored by several political scientists driving an emerging field called “political physiology,” challenges the notion that poticial views are nurtured rather than ingrained in our psyche based on our environment.  The hypothesis is that when we debate on issues, we’re asserting our individuality rather than trying to make a political point.

The scientists in the study say that people with different political views perceive the world differently:

Conservatives are more easily startled by threats, and when performing a habitual task they have more difficulty switching to a new response. Liberals, on the other hand, react less vigorously to threatening stimuli, and in performing a habitual task they are quicker to provide a new response.(1)

This theory that there could be some genetic basis to political views was first borne out in a study done with identical and fraternal twins at Rice University, published in 2005 in the American Political Science Review.  In their examination of thousands of sets of volunteers, identical twins were far more likely to share political views than their fraternal counterparts.  The conclusion was that 40-50% of political and social attitudes are heritable, and therefore significantly influenced by some genetic component.

The research team, using the Rice University study as a basis for their hypothesis, conducted experiments using images and sounds to evoke responses from their subjects and used their reaction as a marker for their political disposition.  The study found that test subjects’ responses to these stimuli, specifically what they felt threatened by, ended up being reliable predictors of their conservative or liberal values.

One of the most frustrating things as a liberal or as a conservative is not being able to understand why anyone would not think the same way you do.  Doubly frustrating is the inability to understand the opposing person’s thought process, and why they may choose a path that to you is so obviously wrong.  Perhaps this study shows us that our tendency towards one end of the spectrum or another is not only shaped by our experiences, but how we’re wired to perceive those experiences through time.

My friend did surprise me with her revelation that day, but I never felt like I would hold it against her.  I know many conservatives and liberals, and think no less of them if their views differ from mine.  Our forefathers wrote the Constitution well aware of the diversity of opinions that shape who we are, and seemed to understand that we could find strength in our diversity.  Though political rhetoric sometimes rules Washington, I am hopeful that as we understand what drives our political instinct we can more harmoniously determine a path for this country that will deliver us through the next 219 years.

What do you think?

(1) “Born to party“, Boston.com, Eve LaPlante, November 22nd, 2008, pg2.

The past few weeks have been rough.  I have a lot of project work going on, and I find myself staying up later and later to try to keep up with it all.  I guess it’s one of the consequences of trying to build a new company, grow a family, and keep in shape.  There just isn’t time to stare blankly into space for a few minutes and recharge the batteries.

I’ve considered trying to make a habit of drinking a glass of wine every night after dinner, but I just can’t get into it.  It is, believe it or not, because of my doctor that I’m even trying; my cholesterol is great (well, my good cholesterol is *too* low), but he feels that one glass of red wine per day is good for the heart and strikes a balance that may have escaped over the course of a hectic day.  What’s not to like about that kind of advice?

The only problem is, a recent study from Wellesley College and Boston University of more than 1800 people found that the more alcohol a person consumed the smaller their total brain volume.  Aging is another factor in decreasing a brain’s size, and it’s this shrinking that has been linked with the progression of dementia, trouble learning new things, memory and cognition.  Based on the information provided in the article published on Boston.com, this seems to be only a preliminary study with more in-depth investigation to follow, but why take a chance?

For some people it’s hard to give up that drink, but it’s even harder to give up smoking.  The same article indicates that giving up such a vice after a hospital stay is up to 65% more effective when patients are provided with anti-smoking counseling both before leaving the hospital and after they arrive home.  The biggest problem, apparently, are the cues of smoking that are much more prominent once someone returns home than while they’re in the hospital, and even those with the best of intentions are apt to fall into old habits once they’re back.  I don’t deny it’s a difficult habit to break, but if the help is there, why not use it?

And it’s apparent that more and more people are refusing the help of vaccinations for their children, especially the flu vaccine.  Another article on Boston.com explores not only the medical implications of skipping out on your flu vaccine this year, but also the social implications.  Two families known to the author had children who died as a result of complications of the flu in their children, and 83 children nationwide died of the disease.  Of those 83 children, the Center for Disease Control and Prevention estimated only about 5 or 10 had actually been vaccinated.  Overall, 36,000 people die every year, close to the same number as those who die from breast cancer.

Author Carey Goldberg describes a concept of “herd immunity,” which essentially strengthens the effect of a vaccine by allowing a group of inoculated people to, in essence, protect one another by preventing an infection from creeping its way throughout each member of the group.  The fewer members of the group that are vaccinated, the weaker the chain, thus giving the flu (or any other virus) the opportunity to break through and begin infecting multiple members of the group.

Dr. Ken Mandl of Children’s Hospital in Boston is cited in the article as saying that children are essentially “little bioterrorists,” because they can incubate and so rapidly propagate a disease among peers, parents and grandparents.  In vaccinating them, you not only protect them, but you protect yourself, your parents and the community.

I thought it was a little harsh to think of my son as a “little bioterrorist,” but thinking back I realized that I have gotten sick much more often than I ever had before I had him…and he’s not even in school yet.  It’s gotten to the point that my wife and I can almost gauge when we’ll get sick based on how he’s feeling.  We’re all even getting over a sinus infection, which he absolutely started, as I write this post.

Unfortunately, I think that parents are paralyzed into thinking that the flu vaccine (and other vaccines, for that matter) could lead to autism in their children, and they therefore don’t take the measures recommended to prevent the infection and spread of these diseases.  I’ve written several posts on this blog about this very topic, the most recent of which is here.  The problem is that the possibility that vaccinations could cause autism has been so ingrained into our public psyche, we believe that the simplest answer, ceasing vaccinations, is the best one.

Jenny McCarthy exacerbates the problem by appearing on popular Hollywood magazines such as US Weekly and telling the story of her son, who suffered from autism but now is cured.  She believes her son’s suffering began when he received his MMR vaccination and had a seizure shortly thereafter.  I feel for McCarthy — I really do — but I don’t believe her belief in the cause of her son’s illness, without being substantiated by fact, should be spread to cause concern among parents already concerned about the best course of action for their children.  A private, informed conversation should be taking place between parents and their doctors, and proper vaccinations should be discussed in great length to determine their necessity and effectiveness.

Maybe I would be less apt to dismiss McCarthy’s conclusions if one of my children were suffering from the disorder.  But then again, maybe not.  I just feel that, given the severity of the diseases against which vaccinations protect us, we put our children in greater danger by not protecting them.  It is starting to bear out with a resurgence of Measles, and based on the information provided in Carey Goldberg’s article, I’m worried influenza will be next.

I know how I feel about it; what about you?  As always, I encourage you to talk to your doctor to make a decision that’s right for you, because it’s a personal decision that involves the saftey of your child.  Decisions should not be based on this new type of McCarthyism alone, with its finger pointed squarely at the vaccinations that were intended to help.

Unfortunately, the Pharmaceutical and Biotechnology industries have not been immune to the collapse.  Since this all began a couple of weeks ago, it seems that many companies have seen a downward trend in their publicly traded stocks.  Schering-Plough (SGP) has seen its price drop from $18.08 on September 15th to $16.05 as of yesterday.  Sanofi-Aventis (SNY) has dropped from $34.98 to $30.90 in the same time period.  And even though Pfizer (PFE)  seems to have traded relatively steadily over the same time period, it is trading in a range that its not seen since 1997.  And biotechs like Celgene (CELG) and Genentech (GENZ) plummeted 7.5% and 7.9% yesterday alone.

All of this obviously induces stress and sleeplessness, and what could be takes the toll on the owners of small businesses.  Never before have I wished so badly that I could go to sleep and wake up to find my portfolio back to its pre-crisis state.  And you’d think a bunch of other bleary-eyed people would love to join me, if only insomnia would not rear its ugly head and keep these people awake.

And what about those who need that little extra something to help them to nod off?  You may recall last year when I cited a Northwestern University study indicating that a lack of sleep has a cumulative negative effect on our bodies.  Sanofi-Aventis’ Ambien and Sepracor’s Lunesta were two drugs I mentioned that help people transition from a waking state to a sleeping one.  Though Lunesta has recently had some difficulties in a market becoming increasingly saturated with competitors (like Ambien and generic formulations of the same), they all seem to be viable sleep options for some people (provided you don’t use them as a cure for insomnia and a substitute for self-induced sleep; if that’s the case then you should see your doctor for other remedies).

Personally, I’ve found a good cup of warm chamomile or warm milk with a little bit of honey always helps to relax me.  I’ve also read that Cognitive Behavioral Therapy, Visualization and Progressive Muscle Relaxation can act as powerful and natural tools for those who don’t wish to turn to drugs as the answer for sleep deprivation.  Even a good book, after a couple of chapters, weighs my eyes and lulls me into sleep.

When one is stressed, as it seems is always the case for many people lately, it is clear that sleep is critical for keeping those stress levels at bay and keeping your mind as sharp as possible to face the challenges on the horizon.  If you’re not sleeping, you should; your productivity suffers and in general you are likely to be less content.  And though forcing yourself away from work, especially when you are in a groove, may seem like the worst thing you could do, the sleep you get in return will certainly make up for it.

What kinds of remedies do you use for getting yourself to sleep at night?  Do you use any home remedies, take advantage of pharmaceutical sleep aids, or just stare at the wall?

I look forward to hearing your responses!

Sequenom is one such company on the list that I found particularly interesting. 

As I approach my mid-thirties, along with many of my friends, I find myself still at the early stages of raising my own familial unit, with future children on my mind.  This is in sharp contrast to the experience of our parents, who most likely at this point were finished with that stage of their lives and had children many years older than ours.  We all have our reasons for starting our families at a time that would be considered “late” by our parents’ standards, but one of the downsides is increased (albeit still small) risk to the baby for Down Syndrome.  Amniocentesis is one of the best accepted methods to screen for this disorder, but it carries with it a risk of miscarriage and can only postiviely identify 70 to 90 percent of the cases prior to birth.  Sequenom, however, has developed a method that could trump amniocentesis at identifying Down Syndrome cases — so far with 100% accuracy in clinical trials — and do so with minimal risk using only a prenatal blood test.

Sequenom’s technology, dubbed SEQureDx(TM), targets fetal DNA circulating in the mother’s blood to examine the genetic status of the fetus.  The first application identified a fetus’ risk for RhD disease, which occurs when the blood of an expectant mother is incompatible with her unborn child.  Jaundice, anemia, brain damage, heart failure and even death can result from the incompatibility, so identifying it early is crucial to taking measures against it.  Showing that it can additionally be applied as a method for testing a fetus for Down Syndrome would be an added incentive for any potential buyers for the company.

I am encouraged by the company’s charter to make “safe, non-invasive prenatal testing available to all women, independent of age and other factors that may contribute to pregnancy complications,” and am hopeful that the knowledge gained in this kind of research could lead to an increased understanding of the kind of defects that could affect the normal development of an unborn fetus.  As parents, knowing in advance how we should be prepared for any difficulties an unborn little one might have is invaluable and gives us the opportunity to prepare for what might be.

The obvious win for any pharmaceutical company that entertains the idea of Sequenom as an acquisition is an increase in the number of parents willing to run the test.  Like many parents, I fear the risks of amniocentesis, despite the information it will give me.  If the risk is mitigated, then wouldn’t more parents do it?  Would it just become part of the standard barrage of tests that expectant mothers are put through?

They say knowledge is power.  Today, we may be having our children later, but we know a lot more about them before they are born than our parents ever did.  Gender, heartbeat, skeletal development, and soon even certain genetic diseases can be safely screened well before the baby is born and steps can be taken to prepare for or even prevent hardship for the child later in life.  But does this take any of the excitement or anticipation out of child birth?  Does knowing any of this information change the way you feel about a child growing inside your wife (or you)?  Or does knowing the information help you prepare to be a better parent?

Your thoughts, as always, are welcome.

On September 3rd, CNN posted an article which casts further doubt on Wakefield’s theory, punctuated by the fact that his co-author, Irish pathologist John O’Leary, is also a co-author on the new study. Published in the peer-reviewed journal of the Public Library of Science, PLoS ONE, the new study concludes that the MMR vaccine causes neither autism nor gastrointestinal disorders, adding to a growing body of evidence against the claim.

Wakefield’s original theory attempted to establish that in some children, the measles virus used in the MMR vaccine would grow in the intestinal tract of some children, leading to inflammation that would make the bowel porus. Toxic material would then seep from the bowel into the body, affecting the nervous system and causing symptoms of autism. John O’Leary and the rest of the new study’s team of investigators replicated Wakefield’s experiments in the same lab that was used for the original analysis, using samples from 38 children with bowel disorders, 25 of whom had autism. They found that only one child in each group had trace amounts of the measles virus in their samples.

Further, there was no evidence that there was a relationship between the children showing symptoms of autism or GI disorders and timing of the vaccine; the three events appeared to be independent of one another.

For the researchers involved in the study, along with many who have read the study itself, the evidence appears to be conclusive. Even the vice president for scientific affairs of “Autism Speaks,” an advocacy group, indicated he believed the results were indisputable. However, other organizations such as the Autism Society of America and the National Autism Association feel that further study is needed and that the PLoS ONE study may even be flawed.

What is more frightening to me, however, is the fact that all of this is causing a decline in the number of parents opting for vaccinations for their children. While I can understand one’s trepidation (just the thought of managing a child with autism is something that unnerves me) the fact is that this kind of decision is now starting to play out in statistics within the US. Measles levels are at their highest in more than a decade, with nearly half of those involving children whose parents rejected vaccinations, according to another CNN report on August 21st.

The number of cases reported this year is still relatively small at 131 through July, but doctors are worried because this is already over triple the 42 cases that were reported for all of last year. The disease is no longer endemic to the US, but is brought in from people visiting from other countries or students studying abroad. Once it gets here though, proliferation of the disease in children has been held in check, widely credited to the aggressive childhood vaccination rates.

But what happens when those vaccinations are undermined by information linking them to autism? You put parents in a very precarious situation. A decision as to whether they should defend their children against diseases that can kill by using vaccinations, or defend their children against what can be a debilitating social disorder that is autism. Unfortunately, because we’re dealing with the health and safety of children, any study is discredited by its detractors and the result is almost a political debate where the burden of proof falls not on the person presenting the evidence, but on the person listening, having to waddle through technical and medicinal jargon that they may not understand.

Ultimately, I believe it comes down to the trust that you have in your doctor, and the trust that you have in yourself as a parent. Being able to prevent your child from having to live with autism would be a wonderful thing, but I don’t personally believe that there’s enough evidence to indicate there is a way to prevent it. Vaccinations, I believe, are a red herring in this case and divert our attention from the real cause of the problem. But before we can move forward, we’ll need to find certainty in a study that will help us all move on and fish for something else.

I was packaged April 28, 2008, in Puerto Rico. My Batch Number is AA6-73P008. I will expire on January 15, 2012. I am associated with 2-D barcode KBDCHDH1528. I was packaged for distribution in Canada. I can tell you as much information as you design me to tell. My codes are undetectable to the human eye so no one can copy me. I am traceable anywhere in the world. I will tell you and only you this information. And I can tell you all of this for less than a penny. (Image published with permission)

So read the ad for NanoGuardian, a state-of-the-art protection system against those who would hope to counterfeit real drugs for a quick profit, or divert real drugs from their intended destination. I found it in the August 2008 issue of Pharmaceutical Executive magazine and was so intrigued by the concept that I had to find out more. So I took note of the web site and immediately started an investigation.

The technology that makes tagging this pill possible, NanoEncryption(TM), was developed by a company named “NanoGuardian (a division of NanoInk).” Using this method, as the ad implies, pharmaceutical companies can add an invisible layer of encrypted data to each dosage of their medications, linking it to an authentication at every stage of their supply chain. Only special tools can actually decipher this code, which links the dose to the package using a proprietary nanolithographic encryption on the dose itself, and Radio Frequency ID tags (RFID) and bar codes on the package. Therefore, if the pill is separated from the package before it reaches the patient, there’s a way to find out.

The technology probably couldn’t have come soon enough, given the growing counterfeit market out there. Email marketing from dubious online pharmacies, which I explored on this blog before, is just one of the many ways these groups can hawk their bogus wares. To me, the biggest concern isn’t even the lost revenue that counterfeiters cause the pharmaceutical companies in creating these fake pills, which NanoInk estimates around $35 billion worldwide. The biggest problem are the patients who are getting their hands on these drugs and endangering their health by taking them.

The process does not chemically alter the drug, and implementing the technology in the manufacturing process requires minimal changes. In fact, a client of NanoGuardian has already submitted a plan to the FDA to implement brand protection through use of the product and has been approved. It wouldn’t surprise me, if this first implementation can be executed successfully, to see other companies start publicly touting the added protection they’re giving patients who rely on their products.

The whole thing actually reminds me a little of a story that came out earlier in the year, where a Silicon Valley company, Gemory, had developed a process for permanently inscribing microscopic versions of photos on a diamond. But while their process may be a novelty and something that is a luxury for people with the money to do it, NanoGuardian’s product seems to be almost a necessity.

The one thing that’s unclear to me is exactly how the patient ends up being protected in this process. The web site for the company is clearly designed to “sell” the product to the industry, which it completely should be. But how do I know as a consumer that what I’m buying is a legitimate drug? The NanoGuardian site indicates that the encryption can only be detected using specialized equipment at the NanoGuardian Authentication Centers…but does that mean that as a consumer I have to send my drugs there before using them? Do pharmacies need to authenticate their medications before stocking them on their shelves? And if it’s not a requirement, what is the impetus for sending the drug for authentication in the first place? If counterfeiters are really good enough to make drugs that look nearly identical to the product they’re duplicating, how would I know that I even need to authenticate? And what’s the cost of authentication? Putting the information on the pill may not cost much…but ensuring a pill has the right information before it reaches the consumer? What does that cost?

I’m sure some of these questions are obvious to some that are entrenched in the industry and familiar with the supply chain. And maybe my brief exposure to the process is naive in that I’m thinking the authentication will be applied in a singular place in the process. I’d love to understand this process more, and would welcome comments from anyone who understands its application better than me. In the meantime, I look forward to seeing the first real products that can ensure their quality because of the tiny writing on the pill.

That said, no other exercise gets me into shape more quickly. It gives me reason to hate it even more. I suppose it’s one of life’s cruel ironies that something I hate doing so much could be so good for me. I often wonder to myself (or aloud when I’m running, much to the dismay of passers-by), if there was some way I could get the benefit from my workout with less effort on my part. It is a paradoxical and unrealistic request given everything we know about exercising, but, believe it or not, there may soon be a pill for it.

A California research team doing tests on mice may have identified a method to activate compounds in the body that would increase fat burning ability and dramatically increase endurance, according to Boston.com’s White Coat Notes from Thursday, July 31st. The study, published on the same day in the journal “Cell,” shows that the PPAR-delta master gene, responsible for controlling metabolism and muscle fibers, can be reprogrammed to burn fat and increase exercise endurance using only drugs.

The study obviously has several moral and ethical implications, so much so that in anticipation of the Olympics, scientists in conjunction with the World Anti-Doping Agency have concocted a test for blood and urine that can test for even the tiniest traces of the two substances that make up the drug cocktail. The suspicion is that a lab somewhere may be able to reproduce the chemicals for competitive athletes, giving them an unfair advantage in competition. Any broken record is reason enough for suspicion, as Dara Torres is quickly discovering after she broke a US swimming record recently.

The other downside to the pill is getting the dosage right. According to the study, the month-long trial in the mice would translate to about a three-year regimen in humans. In treating diseases such as obesity, it might be too long a wait.

The process is activated through use of two drugs, AICR and GW1516, the former of which is currently licensed by Schering-Plough to help control bleeding in the human heart. It will probably be some time, however, before clinical trials of the two drugs in tandem can commence, and even longer before scientists understand the implications of changing how muscles function in the body. And I don’t know about you, but the thought of taking one or more pills for the rest of my life to maintain a svelte figure and a higher level of endurance isn’t appealing to me. Perhaps, if proven to be safe, whatever company decides to produce this drug can dangle it as a carrot to Mike Adams, sponsor of the $10,000 health challenge to big pharma.

The fundamental philosophical question is how far we’re willing to push to have something else do the work for us? We’re developing pills to make us younger, pills to make us healthier, pills to put us in better shape. What happened to eating right? Exercising? Scheduling enough time in what seems to be our infinitely busy lives to use the tools that nature gave us rather than looking for the quick fix? As time goes on, it seems more choices are developed by the pharmaceutical industry to circumvent a natural process to give us more time. As we get it, however, we lose sight the one basic principal through which appreciation of that time is developed: our patience.

I think I’ll take that run now.

According to the FDA, in order to be considered a “botanical” product, a drug must have the following characteristics:

• A botanical drug product consists of vegetable materials, which may include plant materials, algae, macroscopic fungi, or combinations thereof.
• A botanical drug product may be available as (but not limited to) a solution (e.g., tea), powder, tablet, capsule, elixir, topical, or injection.
• Botanical drug products often have unique features, for example, complex mixtures, lack of a distinct active ingredient, and substantial prior human use. Fermentation products and highly purified or chemically modified botanical substances are not considered botanical drug product. (1)

Headquartered in Jamesburg, NJ, Phytomedics was launched in 1996 to focus on such botanical drug products and has partnered with the Biotech Center at Rutgers University for its research and development, allowing it to function as a private company with access to cutting-edge research facilities while keeping its costs low. Those costs are being covered by several investment firms including Inventages Venture Capital GmbH (life ventures by Nestle), Burrill & Company, Polar Investments Ltd and Biotech M.A.H. Plant Genomic Fund.

The company currently has 2 drugs in development. The first, which has already commenced its Phase III clicnical trial development, is a new, oral drug for treatment of auto-immune diseases. In its Phase II, double-blind clinical trial, PMI-001 was given to 120 patients with moderate to severe rheumatoid arthritis. The results showed significant efficacy of the drug, with rapid pain reduction in as little as 2 weeks and a halt in joint erosion in 6 months. PMI-002 is the second botanical drug under development and shows promise in the treatment of cancerous cells as well as the possible prevention of neurological and opthalamic degenerative disorders.

Other drugs are also in their pipeline, but have not yet received the same focus as their other products (it seems due to lack of funding). These products include:

• A botanical bioactive that acts as a COX-2 inhibitor but with less risk than a Vioxx, Celebrex or other synthetic chemical entity.
• A botanical appetite suppressant that could be leveraged for weight loss, and a counterpart derived from an agricultural waste processing stream that could block fat absorption.
• A plant-derived agent that could be used to limit the impact of aging on muscle cells.

It seems that these drugs, along with the proprietary methodologies the company is using to develop them, could usher in a new wave of drugs that will be prescribed by doctors in the future. As you may know if you’ve read a few of my posts, I’m very interested in keeping an excessive amount of “artifical” stuff out of my body, and the research here shows promise for drugs that may be headed in that direction. I am hopeful that companies like Wellgen and Phytomedics can provide breakthroughs in this area of research and demonstrate the safety and efficacy of these drugs because of their origins as completely natural substances.

However, until an actual product has been released and clinical trials can demonstrate the safety of the product and its interaction in the human body can be better understood, I remain cautious of drugs developed within this emerging field.

Your thoughts?

(1) See “What is a Botanical Drug?“, FDA web site.

She holds an M.S. in Statistics from Rutgers University and a B.A. from Cornell University. She is a member of the American Statistical Association and the New York Area SAS Users Group. Her contributed article and contact information follow.

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For many in businesses that rely on clinical research, the ‘sizzle’ of a headline or claim seems to be enough. But imagine the following scenario:

A prospectus sits on your desk. There is a sentence “The formulation shows a p-value of .023, significance at 95%, at a dose of 10 mg. Sounds good…its statistically significant…move on. But then you get a call six months later. A regulatory reviewer questions the randomization technique. But you thought everything was okay at 95% confidence. They did the statistics.

Statistics are the language for reporting results of efficacy and safety trials. We report significance, confidence and p-values, the probability of an event under certain conditions. These statements make their way into press releases and business documents.

In truth, statisticians play a role before the study is started. They advise on the study design, data collection and implementation. While the study is in progress, they bridge communication between the computer professionals such as programmers and data managers and the medical professionals such as clinical investigators and research associates.

In the jargon of the industry, the research on human subjects is referred to as Phase 1 through Phase 4 clinical while other activities such as drug discovery, manufacturing and toxicology get lumped into “non-clinical”. Clinical statisticians do work in a structure of tables, listings and graphs that adheres to the requirements of the FDA and various other national and international organizations. The roles vary for non-clinical statisticians. There is more interest in applications of statistics in early stage research and manufacturing processes.

One can find various definitions of the word ‘statistics,’ including the vernacular usage as any display of numbers. The American Statistical Association in its career section website (www.amstat.org/careers) answers the question “What is statistics?” as follows–Statistics is the scientific application of mathematical principles to the collection, analysis, and presentation of numerical data. Statisticians talk in the language of science-observation, estimation, hypotheses, generalization and modeling. Unlike other approaches to data analysis that aim only for a mathematical optimization, statistical analysis concerns itself with the scientific context of the data collection such as the measurement ranges and precision of lab equipment. It is not black-box computation.

In the early 20th century a mathematical framework for statistics emerged built on the backbone of probability theory, an older branch of mathematics. The expansion of this mathematical framework continues in universities around the world. While clinical research sometimes uses new techniques, it relies on basic ideas like distributions of averages and randomization. Randomization is a plan for treatment assignment that is independent of human choice. We know the probability that a subject is placed in a treatment group. This assignment lets the researcher use probability-based statistics to assess the effect of a treatment and whether it is just a result of ‘chance.’

As a professional statistician, most people find what I do mysterious. They remember a dreaded required course. If older they think of grayish books with grids and curves. If younger they think of the horrible software they were forced to use. Fortunately these classes, the books and the software are now a lot more applicable, even fun.

But you don’t have to take a new-fangled level 200 class to bring make clinical statistics more relevant. A good start is thinking about the numbers that express the result. This will make your questions more effective. A desired clinical change can be expressed as a change in a decimal number, a count, or a percent. A ‘20% drop’ can explain going from ‘200 to 160′ or ‘15 to 12′. This is where the ‘scientific context’ becomes important. The expression of a clinical result is the end-product of the clinical practices, the regulatory guidelines, previous company studies, and the judgment of the current statistician, and perhaps a previous statistician.

My ending comment is stay aware. A short phrase in a business or legal document is a small blip in the thousands of pages in a clinical trial submission. The minds of regulatory reviewers are at once specialized and focused but tuned into current trends. Clinical statistics can become pivotal in the approval process.

Georgette Asherman
Applied Statistician
Direct Effects, LLC.
www.directeffects.net
201 673-4301