An introduction to CCSVI thanks to the Reformed Multiple Sclerosis Society

I first heard of the Reformed Multiple Sclerosis Society from this New York Times article back in June of last year.

The Reformed MS Society started much in the same way this Foundation started with one spouse fighting for the rights and treatments for another.

Mr. Steven Simonyi-Gindele began the Society after his wife, Ruth, who suffers from Multiple Sclerosis, had the treatment for CCSVI. It is theorized that MS may indeed be a vascular disease and that the resulting destruction of myelin comes from iron deposits in the brain (due to lack of blood flow) which triggers the autoimmune response.

CCSVI, or chronic cerebrospinal venous insufficiency, is treated with balloon angioplasty and stenting of the blocked veins.

The Reformed Multiple Sclerosis Society is your best source for information regarding the treatment for CCSVI. They are not beholden to donations from pharmaceutical companies. We will also start to keep our own section on CCSVI and update with information as it becomes available. I spoke with Elizabeth of the Reformed MS Society back in November of last year. They are a wonderful and accessible organization and if you have any questions, they will try and answer them. Their office number is 604-639-4405.

More about CCSVI here. And Wheelchair Kamikazee‘s articles on his own trials with CCSVI treatment. From Marc’s blog which reinforces the theory that MS may be a vascular disease:

In short, the procedure was a “successful failure,” in that we successfully determined that I do have significant abnormalities in the vascular system associated with my central nervous system (a very important discovery), but those abnormalities unfortunately could not be remedied during the procedure.

And with the pharmaceutical companies regurgitating cancer drug after cancer drug as the “newest therapy” in their MS arsenal–reformulated and slapped with a higher price tag–it’s time for some real research into the disease and not research into how more money can be made from the disease, year-after-year with no end in sight.

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The kind of Multiple Sclerosis research that should be well funded in this country, but isn’t.

José Antonio Lozano, Borja Calvo and Iñaki Inza, in the Computer Faculty of the UPV/EHU.

I found this article yesterday about some very important research that you may have never heard about. It is being done by the University of the Basque Country (UPV/EHU) located within Spain.

Before I get into trouble with the people of the Basque region, please know that pinpointing your location within Spain is just recognizable for those unfamiliar with the country, region or your really good food.

Okay, on to the research. The University’s computer researchers, specifically the Intelligent Systems Team (working with the use and application of algorithms), are working with BioDonostia to find the genetic markers for Multiple Sclerosis.

Finding the genetic markers would give researchers the ability to find a cure for MS and also, possibly, a host of other autoimmune diseases. Finding a cure does not sit too well with companies who make millions (or as Mr. Termeer of Genzyme has postulated, billions) from just maintaining the disease.

Algorithms can even help to better understand certain diseases, as well as to find biomarkers related to their diagnosis and prognosis. This is one of the key functions of bioinformatics. In fact, four members of the Intelligent Systems Team (José Antonio Lozano, who is the director of the team, and Borja Calvo, Iñaki Inza and Rubén Armañanzas, the latter currently at the Polytechnic University of Madrid), are working closely with researchers from Biodonostia, the first health research body within the Autonomous Community of the Basque

Who or what is BioDonostia?

The BioDonostia Institute was founded in December 2008 by the Osakidetza’s (Basque Public Health System) Management Board and the Basque Foundation for Health Innovation and Research (BIOEF), Board of Trustees, focusing on fostering biomedical research. Its headquarters are in the Donostia Hospital, located on the Paseo Dr. Beguiristain in Donostia-San Sebastian.

Based on the tools developed by the Bioinformatics engineers at the University of the Country of Basque, BioDonostia is using the tools for medical research. It’s a cooperative research system where the objective isn’t a higher stock price, but actually working out a solution to our most basic human problems.

They have worked together on studies linked to Parkinson’s, frontotemporal dementia and muscular dystrophy. Nevertheless, as Mr Inza explained, “at present the most visible fruit of our work is with multiple sclerosis. We have published an article in an international journal (in the US Public Library of Science, PLoS ONE, in 2009) and there is a patent pending between Osakidetza (the Basque National Health Service) and the UPV/EHU”.

Mr Inza stated that “whoever finds the biomarkers for multiple sclerosis will receive a Nobel Prize”, to underline the difficulty of the challenge. But at least they believe that are taking steps in the right direction. In the words of Mr Borja Calvo, the Biodonostia researchers suspect that some of the molecules known as micro RNA could be linked to multiple sclerosis, or act as biomarkers, which is why they have taken samples and analysed their levels of expression. This was when bioinformatics came into play: “They generated these data, they passed them on to us and we aimed to construct a classificatory model which, introducing levels of expression into it, was able to predict if there was a disease or not, or the state thereof”. The results were quite good: “The models predicted the disease quite well and, on this basis, a series of validation phases has been initiated”.

I just wanted to highlight the fact that this research is taking place–but not in this country where we have a lot of money being thrown at Multiple Sclerosis: a lot of fundraisers, a lot of 501(c)(3)’s raising millions to fund research but also to fund salaries, rent on high priced real estate, brochures touting their work, and the other expenses with running a high profile foundation.

See the Susan G. Komen Foundation and their work blocking the Patients Bill of Rights in 1999, 2000 and 2001, which certainly runs counter to their work to help women suffering from breast cancer, as a prime example of high profile foundations working for themselves and not for their original charity-driven mission.

Could it be that money isn’t given to fund research to find a cure when so money is to be made from not finding a cure?

My understanding of finding the genetic marker for any disease would be, as Mr. Inza puts it above, worthy of a Nobel Prize. The rest of the article is here, and I urge you to read it. It is incredibly interesting, especially for you nerdy people out there like me, how these researchers are converting the genes into numbers and then using a DNA chip (which fits in your hand) to store the information. Your genome on a chip!

Now research that used to take decades on a single gene can be sorted out much more quickly. With the use of the chip, a researcher can begin to look at the genetic make-up of someone with Multiple Sclerosis and compare that DNA with someone without the disease and see where the genetic differences may lie. We hope they succeed quickly.

“When the DNA from a sample of a person’s body is inserted behind the chip, each gene goes to its allotted slot, as it were”, stated Mr Inza. Then images of colours, partitioned into these slots, are obtained. These colours represent “levels of intensity and are proportional to the level of expression of each one of these genes. These are translated into numbers”. José Antonio Lozano adds, “The numbers express a level of fluorescence, the intensity of the signal.” With these numbers, computer models enter the scene.

Biogen reports 6 new cases of PML with Tysabri

The article below is taken directly from Reuters.

If you are taking Tysabri, made by Biogen, we urge you to please go and speak with your doctors as the total number of deaths reported is now at 16, while 69 people remain in varying states of disability from PML–on top of having Multiple Sclerosis. More information on what PML is here at WebMd and here at Wikipedia.

Good source for all MS drugs is: Drugcompare.destinationrx.com.

More Tysabri information herehere and here.

Everything below is taken directly from Reuters.

Biogen reports 6 new cases of PML with Tysabri

(Reuters) – Biogen Idec Inc said six patients taking its multiple sclerosis drug Tysabri developed a potentially fatal brain infection in December, bringing the total number of such cases associated with the drug to 85.

The biotechnology company said that as of January 7, the overall incidence of progressive multifocal leukoecephalopathy, or PML, was 1.06 per 1,000 patients, up from 1.0 in 1,000 last year.

Biogen, which makes the drug with Irish drugmaker Elan Corp Plc, temporarily withdrew Tysabri from the market in 2005 after it was first associated with the condition; it was brought back, with stricter safety warnings, in 2006.

The drug is widely considered the most effective on the market, but its sales have been crimped by concerns over PML. The risk of the condition increases with the length of treatment.

After two years of monthly infusions, the incidence of PML is now 2.13 per 1,000 patients.

The overall rate of PML in clinical trials was 1 in 1000, a benchmark that has now been exceeded.

Of the 85 cases of PML reported, 16 patients have died, while 69 are still alive with varying degrees of disability.

Biogen is developing a test that it hopes will allow doctors to screen patients to identify which might be more likely to develop PML.

Biogen shares were down $2.02 or 2.9 percent to $66.76 in afternoon New York trading, while Elan shares were down 10 cents or 1.5 percent to $6.37.

(Reporting by Toni Clarke, editing by Gerald E. McCormick)

What repeal of health care would mean, by congressional district.

Today Rep. Henry A. Waxman, Ranking Member of the Committee on Energy and Commerce, and Rep. Frank Pallone released, for each congressional district and the 30 largest metropolitan areas, an analysis of the impact of the repeal of patients’ rights, protections, and benefits contained in the historic health care reform law. Click on the highlighted text to be taken to the Committee’s website.

There you will find a map with all the states broken down into their districts with each representative’s name. The numbers of people the Affordable Care Act would help, and are helping, speak for themselves.

The very high expectation Genzyme has for Campath, now known as Lemtrada.

We have been posting (see here and here) on Genzyme’s possible treatment for Multiple Sclerosis, Campath, or as it is now being marketed, Lemtrada. Campath is a drug used to treat certain types of cancer but now Genzyme is looking to sell it for the treatment of Multiple Sclerosis. They think they can reach around $3 billion in sales doing so as well.

To get an idea of how much money is already involved in the marketing of this drug, which still has not received FDA approval, I found an article at PR Week where it was reported, back in December of last year, that Genzyme had already engaged a PR firm, Cohn & Wolfe, to help in their “global pre-launch communications,” of the drug.

Genzyme has said that the drug could generate between $3 and $3.5 billion in annual sales by 2017. That is, simply, a lot of money for one drug. But, considering what the CEO of Genzyme, Henri Termeer said of the market for Multiple Sclerosis, they obviously have very high expectations for their drug:

Termeer also noted that the overall market for multiple sclerosis is $14 billion, and Genzyme executives said their research showed there is significant demand for new treatment options. Alemtuzumab, they said, is more effective, convenient, and easier for patients to tolerate than drugs now on the market.

We agree with Mr. Termeer that there is a very significant demand for new treatment options, especially ones that don’t just manage the disease (or cause other debilitating diseases in the process, see Tysabri and brain infection, PML), but ones that could actually cure the disease. It’s why so many people with MS are looking at the new liberation treatment for CCSVI with great hope. 

To get an idea, a glimpse if you will, of how much Genzyme (or Sanofi, if the take-over does succeed) will charge for their still unapproved Multiple Sclerosis treatment of Campath/Lemtrada, a little mathematics and some fact gathering is in order.

Remember, Campath/Lemtrada is still a relatively inexpensive drug, especially for the treatment of Multiple Sclerosis, since the amount of the drug used is much smaller than for the treatment of cancer. So for Genzyme to be touting the market in the billions, we wanted to get an idea of how much they think they can charge.

We’ll start here with the population of the United States. It is, roughly, 310 million people. One report has it at 307 million, while this from the Census Bureau has it at 311 million. We’ll use 310 million as a mid-range, easy-to-calculate number.

Of that number, roughly 400,000 people have been diagnosed with MS. No one really knows how many people have it, according to the National Institutes of Health. My number is being a bit generous, by about 50,000, to play it safe.

What I found is, based on my numbers, roughly, .1% (that is point one percent) of the population of the United States has Multiple Sclerosis.

Based on Mr. Termeer’s number of the Multiple Sclerosis market being around $14 billion globally (I am assuming globally here) then Genzyme hopes that this one drug will corner around 21% of the global Multiple Sclerosis market–$3 to $3.5 billion of a $14 billion market.

And it still has not been approved. And they’ve hired a PR firm to help in pre-launch communications. Are they this certain? There are high expectations for this one drug and it seems there are even higher expectations on the pricing of this drug.

How much does Genzyme have to charge to gain 21% of the market?

Genzyme will either have to corner a very large share of the Multiple Sclerosis market by 2017, unless a cure is found before then, or raise the sales price of the drug for Multiple Sclerosis patients to hit those numbers.

Campath now generates roughly $112 million in sales for Genzyme* (2008 sales figures) so to get to $3 billion, that would have to be, roughly, a 97% increase in price combined with a deep market reach.

But how many people have Multiple Sclerosis and more importantly how many people globally with MS have access to adequate health care? Who can afford this?

The numbers on those questions remain elusive. We think that Genzyme sees its billions coming from raising the price of this relatively inexpensive drug to the price shared by the drug, Gilenya (which was first synthesized in 1992 for use in organ transplants),which is the most expensive Multiple Sclerosis treatment available at $48,000 per year.

Already MS patients are seeing issues with getting their supply of this expensive drug. See the MS World.org forums here and here.

It would be more humane (if this drug does get FDA approval) for Genzyme to keep their expectations of achieving a $3 to $3.5 billion share of a market more in line with reality and with what people with Multiple Sclerosis have to go through–from a body that is leaving its sufferers frustrated and debilitated, to insurance issues like the ones you see here.

After all, spin and PR it any way you want, Genzyme (and Novartis, since Gilenya or Fingolimod is relatively cheap to synthesize), its investors and executives are hoping to make money as the Multiple Sclerosis market grows–betting more will be diagnosed and suffer its effects–and then more money will be made from a drug that is, after all, relatively inexpensive for the treatment of MS in the first place.

How much money is too much?

*I could only reach the cached version of the article I quoted. Here is the original link:
http://www.bioworld.com/servlet/com.accumedia.web.Dispatcher?next=bioWorldHeadlines_article&forceid=50472 It is a snapshot of the page as it appeared on Dec 29, 2010 23:18:07 GMT. The current page could have changed in the meantime

Campath by any other name–including Lemtrada–is still Campath, price included!

They’re still at it, Genzyme and Sanofi are still in talks about a takeover by Sanofi and Genzyme is still touting their as-yet-to-be-approved treatment for MS, now openly being called by the name “Lemtrada,” as a $3 billion per year seller.

Campath (Lemtrada) was approved in 2001 for the treatment of lymphocytic leukemia, so for it to be used as a possible treatment for MS, and for Genzyme to be using this as leverage for a higher asking price, is well, in our opinion, shameful considering the price of Campath, aka Lemtrada.

Campath now sells for $30,000 for leukemia treatment. But MS patients need only a fraction of the dose used in cancer patients. At the current price, MS patients could get Campath for $7,000 or so. That’s far less than other MS treatments; just consider Novartis’ new drug Gilenya, priced at $48,000 a year.

So, Genzyme has a dilemma. To get market prices for Campath use in MS, it would have to price the drug differently for each use. But what would stop doctors from using cancer-priced vials rather than MS vials? After all, off-label use is perfectly legal.

What would stop doctors from using the cancer-priced vials rather than the far more expensive MS priced vials?

Could this have anything to do with the name, Lemtrada? A small reformulation tied to a higher price? Rebranding to make us think it isn’t a cheaper drug? We think it’s one of those and we are awaiting more information as it becomes available and will post it up here on our site.

From Bloomberg.com:

Sanofi-Aventis SA and Genzyme Corp. said takeover talks now involve executives from both companies as the two sides discuss extra payments tied to Lemtrada, an experimental multiple sclerosis drug.

Genzyme’s Lemtrada, sold under the name Campath as a treatment for blood cancer, may have sales of $3 billion to $3.5 billion by 2017 if approved for multiple sclerosis, according to the Cambridge, Massachusetts-based company. Sanofi and Genzyme said they are discussing a “contingent value right,” under which the French drugmaker would make additional payments for regulatory approval of the treatment and the achievement of certain sales thresholds.

You can search for clinical trials of Campath here at ClinicalTrials.gov. More on what we wrote about Genzyme and Campath here.

Keeping an eye on Tovaxin from Opexa Therapeutics.

Why are we keeping an eye on Tovaxin? Because Opexa seems to understand that not killing the patient is the best way to go about trying to treat Multiple Sclerosis. The only adverse effects reported in the clinical trials so far have been mild to moderate reactions at the site of injection. No one has died!

Although there was a random glitch with their trials in 2009, where the control group had a very low relapse rate and the treatment group a high relapse rate, the newest trials have been very positive:

The analysis produced encouraging results which showed that these patients, when treated with Tovaxin, had a 64% reduction in annualized relapse rate versus placebo (p=0.046, n=70). This statistically significant efficacy result, coupled with the superior safety profile of Tovaxin, was highlighted during the FDA meeting to emphasize what the Company believes to be a promising benefit-to-risk profile for Tovaxin.

Opexa Therapeutics announced that they are preparing to start the late-stage clinical trials for Tovaxin, a new MS drug that uses your own T-cells to make a “personalized T-cell vaccination for the treatment of MS.”

Let’s break this down into easy to understand terms about how a “personalized T-cell vaccination” would work. Taken directly from Opexa’s website is this graphic that explains the process:

The treatment will consist of donating blood and creating a vaccine using the patient’s own cells. The vaccine cells will be irradiated to render them unable to divide, but able to evoke an immune response. The vaccines will be administered in the doctor’s office as a subcutaneous injection in the arm given five times a year.

No brain melt as can happen with Tysabri. No compromised immune system, bradycardia or macular edema as can happen with Gilenya. Campath, not as yet approved by the FDA, has had deaths in their clinical trials.

What remains to be seen is the cost. Will Opexa go the way Novartis did and charge $48,000 per year? (and Gilenya is a pill) or will they find a way to satisfy themselves and their shareholders and charge a reasonable fee for what looks to be a very promising therapy? Time will tell.

You can read more useful information from those who were in the Tovaxin trials here, at the This Is MS Forums.