What have we learned this year about MS? Two new medicines. 20 years of modern care. MS is no longer a dread disease. The FDA is working against us.
20 Year Anniversary of Betaseron
The more mature among us will recall 1993 with the landmark release of the first disease modifying treatment for MS. Prior to this, doctors choose to use chemotherapy (cyclophosphamide and hair loss), or older immunosuppressive drugs (azathioprine, cyclosporine), or lots of steroids to treat MS. MS was rapidly disabling.
Twenty year follow up of the patients in this trial (98% ascertainment) showed an astonishing and still marvelous effect. Those people who waited 2-3 years to start treatment with Betaseron (the first interferon-beta for MS) are not only more disabled, but twice as likely TO DIE from MS. The government will not let this information be discussed by the pharma companies because they did not approve the study (more on FDA censorship below and at another time). So both patients and doctors are unaware of this important finding. An old dogma is that MS does not shorten lives (which is wrong). MS is more deadly than breast cancer. MS patients are three times as likely to die as their peers. Why? Disability kills. More respiratory infections.
So we can be thankful that interferon-beta makes MS better by 50%. In fact at first symptom the initiation treatment protects the majority of patients from a relapse of disease for over 7 years.
Why don’t you hear about this?
The FDA is censoring that from promotion by Pharma as well. So the mission of FDA is protect us. They are failing us by penalizing the dissemination of information by Pharma. Yes censorship exists in America, and the Department of Justice and Food and Drug Administration are in bed together to extract billions in fines from Pharma for overstepping “claims of efficacy”. This results in more government lawyers, less money for research and education, less dissemination of scientific knowledge, and less innovation and drug development by Pharma.
Early MS treatment is the standard
An enlightening study performed by a worldwide MS consortium demonstrated for the first time that in specialty practices, there is a group of first symptom patients who does far worse than the others. Who are these unfortunate souls? They are the ones who choose not to be treated at the first symptom. They fare even worse than the really severe MS patients on treatment. The moral of the story is hard to swallow for many newly diagnosed first symptom patients.
Choosing Wisely er Unwisely:
How The American Academy of Neurology (AAN) Betrayed People with MS and Their Doctors
A year long saga has been ongoing with a stealth attack of the insurance industry on MS treatment. MS has become the most expensive disease yet to treat (surpassing cancer), because of the nature of the medications, cost of the research and development, and the longer and more productive lives which people with MS are having due to favorable responses to treatment.
The American Board of Internal Medicine has an initiative called “Choosing Wisely” where committees try to identify cost-saving practices. Well when it came the time for AAN to offer suggestions the board empaneled a small committee of neurologists. This committee claimed to have used a Delphi process (expert input and development of opinion) to construct a series of recommendations to save money. The head of the committee was Annette Langer-Gould, a physician employed in a partially administrative capacity by the largest HMO in the world, Kaiser Permanente, widely known for it’s penny-pinching denials and heavy-handed healthcare policies. Without input of a single other specialist in her field, this doctor crafted a treatment guideline for MS that anyone with progressive features should not be treated. She claimed there was no evidence of benefit in treating people with progressive MS (with or without relapses) with interferon-beta or glatiramer acetate. There are in fact many trials showing the same benefits of treating higher disability people with MS as those with lower disability, on relapse and MRI. This astonishingly unscholarly work got published in a prestigious journal. Furthermore, she claimed to have consulted the largest community of neurologists in the world. She never did. When we cried foul, her defense was “you know I am right and nothing works.” We were astonished how our society could have put such an ignoramus into such an important role, and we complained both to AAN and the journal Neurology. Then the CYA-fest began. The Editor of Neurology claimed “she didn’t really commit fraud because she just made a mistake.” The other members of the committee defended themselves by saying “she was the expert.”
It will take years to repair the message “you shouldn’t treat MS” and opens the doors to insurance companies to increase the difficulty and expense of being treated, even though decades of solid scientific study show the benefits. I suspect in the next year your insurer will start to examine if you have disability and haven’t had new MRI and relapse (that is you are being treated effectively) that you should not be entitled to therapy because they want to call you progressive MS.
This is an antiquated and useless term other than for retrospective shorthand purposes. Hopefully, we can reverse the damage. I expect insurance companies will take liberties with MS patients, as they are taking people of one treatment to save a few hundred dollars a month, even though they are doing well to try to make them take another that is not as well supported or convenient because they can get it a little cheaper.
Lifestyle is a big thing
I touched on this in the last blog. Vitamin D supplements, stop salty and greasy food, stop tobacco. Eat tuna and salmon. Exercise.
Aubagio (teriflunomide) for MS
This new pill came to market a year ago. It is very similar to an existing medication for rheumatoid arthritis. I wasn’t sure if it would be better. I have become convinced it has less blood and liver abnormalities than it’s parent drug leflunomide. We are learning how to use it. While the company made a bold move initially of pricing it less than the other drugs, insurance companies were not kind to them. So they then raised the price back up to where other drugs were and offered discounts in negotiation. The biggest obstacle to it’s use is that it has a high liability for birth defects if a developing baby is exposed, and this is even more difficult, because the medication lasts for many many months after stopping it, unless a complicated regimen to extract the medication from the body is done. This has limited the use of this medication also to essentially infertile women (and couples) or post-menopausal couples, or those with absolute bullet proof contraceptive use. I cannot take the time to educate for hours a fertile woman on all the issues to assure that they understanding the risks to a baby and how to use. Concern for minor transient hair thinning is present, and most young women do not view this enthusiastically. It is a well tolerated medication, but does not distinguish itself in efficacy, although it works for patients with progressive MS and is simple after monthly blood and liver tests the first six months.
Tecfidera (BG-12, dimethylfumarate)
This medication was long awaited and proved even more popular than the “Wow” it was given a couple years ago when the medication’s efficacy data was presented. Tecfidera was released in April and quickly became a market leader despite some major missteps in handling it by the most experienced MS company in the world BiogenIdec. Biogen changed the manner in which the drug is started by trying to economize on the smaller capsules and make people take bigger capsules right away. This resulted in a much greater rate of serious side effects (and hospitalizations and serious nausea and vomiting). There was even a death caused by it. Furthermore, Biogen published a disingenuous response to a scientific report demonstrating that dimethylfumarate causes PML, a devastating brain infection caused usually by Tysabri. They went around trying to convince physicians it was different from the form of the drug which caused PML. This was all scientific disinformation designed to obfuscate. The FDA failed to properly disclose to physicians and patients when approving the drug that this, as well as other serious infections (one called Kaposi’s sarcoma) had occurred with dimethylfumarate. Also, no mention was made of the rare cancers that occurred. These risks are fortunately small, one in many thousands. However, all in all this is a good drug, and will likely continue to be used in MS in people who tolerate it. I am thankful that Biogen has brought it to market.
Will we get Lemtrada (CAMPATH, alemtuzumab) or not?
So we end up in December. The FDA is on the “Naughty” list this year yet again for it’s handling of the biggest game-changing medication ever for MS. They managed to find a panel of physicians who were unbiased to review the research data to decide about approval for MS. Unfortunately, these were the most ignorant group of physicians ever assembled to review an MS drug application. 20 years of research data. Thousands of patient years. Absolute unbelievable ignorance. They voted the trials were inadequate and poorly designed (not a single expert believes this). The medication was twice as effective as Rebif, a standard of care medication. It was so much better than Rebif anyway you would have thought that Rebif was an inactive medication. Yet they voted it should be approved (they were not following the rules). We’ll see if there is someone wise enough at FDA to clean this mess up. This after the poor company waited TWO YEARS for the FDA to address the application. Another reason drugs cost too much…the FDA is slow. Not understaffed, just really really slow and lazy about following its own rules.
We really really need alemtuzumab. Just like you need a chainsaw when a big tree falls in your yard. This is the drug for the heavy lifting in MS which we have needed for years. It ACTUALLY MAKES PEOPLE BETTER FOR YEARS. More about this when the drug nears approval.
Well I wish you a very happy 2014. I am dreading January and the abuse which insurers heap on us physicians with unnecessary paperwork, trying to kick patients off of treatment a few weeks so they can make more money off of sick people.
Dr. Samuel F. Hunter, M.D., Ph.D. practices at Brain and Nerve Neurology in Franklin, Tennessee, and directs the advanced Neurosciences Institute (http://neurosci.us) and the NeuroNexus Neurology Education and Research Center (http://neuronexus.org). Follow him on twitter at NeuroNexus1. He conducts research in part sponsored by pharmaceutical companies, of which he is a great fan, as well as critic and iconoclast.