UPDATE:Glad I didn't wait for Lemtrada/Campath/Alemtuzumab

An earlier blog post, Follow the MS Money, explained my reasons for ignoring a neurologist's recommendation to not do HSCT and instead await hoped-for FDA approval of the drug alemtuzumab (Campath/Lemtrada) for treatment of MS sometime in 2014. 

A November 11 news item affirms that I made the correct decision to proceed with HSCT. The 2014 launch of Lemtrada is now in doubt because of an unacceptably high risk of thyroid cancer. FDA sorting out the risk/benefit equation for Lemtrada will take longer than expected. I can only imagine how many MS patients will get worse as they await a drug that trades mild improvement in MS symptoms for a high risk of cancer.

I'm glad I did not wait. Though HSCT is not without risk, the potential quality of life benefit is much greater. Whereas Lemtrada has shown a 1 point improvement on the MS disability scale of 1 to 10,  HSCT is showing greater than 1 point of improvement, with 2 to 3 points of improvement for highly inflammatory relapsing remitting MS cases like mine. 



TOPIC UPDATE:

In the report on Genzyme’s Lemtrada, FDA drug reviewer John Marler detailed a litany of conditions and complications that could be caused by the MS treatment. He also called into question the way clinical trials of the drug were conducted.

“The certainty of the risks of potentially lifelong hypothyroidism [a thyroid condition], serious infusion reactions, melanoma, and other malignancies, Grave’s ophthalmopathy [a thyroid-related eye disease] and other autoimmune disorders and prolonged increased susceptibility to infection may not be balanced by the uncertainty that exists in the limited evidence of potential clinical benefits from clinical trials that were not well-controlled,” Marler wrote.  Full article below.

Report slams Genzyme’s MS drug on eve of hearing

People of faith, adult stem cells, and science.

When I first launched this blog on October 27, 2013, many of my friends, colleagues and acquaintances learned for the first time that I have multiple sclerosis, a disease I have struggled with since 2007. That alone is a big piece of news to digest.

Bigger still, I piled on additional news with the word jumble “autologous hematopoieic stem cell transplant” (HSCT), a procedure I will soon undergo at Northwestern University in Chicago. Please allow me to define a couple of those words. Their definition in regard to the type of stem cells involved is important, and I should have done a better job of defining these words earlier to clarify that there are no embryonic stem cells involved in HSCT.

AUTOLOGOUS

1:  derived from the same individual (me)

2:  involving one individual as both donor and recipient <an autologous stem cell transfusion> 

Hematopoietic

refers to organ where blood is formed. ex. the spongy bone, " a site of the bone where blood is formed" (In my case, the immune system component of blood, antibodies, lymphocytes and white blood cells.)

Emotions and Facts

The media has done stems cell science no favors. In offline conversations, several of my friends, people of faith and science, used the word “controversy” when referring to stem cells. Online, I have learned that some people who have already done HSCT try to avoid saying the words “stem cells” at all, partly because it leads to difficult social situations. I can understand their reticence. But I believe patients like me must be part of the solution to an educational problem being grappled with by leaders of both science and faith institutions, including Northwestern University, the National Institutes of Health and the Vatican. 

Following are two links to demonstrate how doctors, patients and faith leaders are building bridges of understanding.

After you click the following link, either read the whole article, or scroll halfway down to read what Dr. Burt had to say at the 2013 Vatican Conference, as well as two of his patients. In December, I will be another one of Dr. Burt’s patients. Though I am not personally Catholic, I find it very encouraging that the Vatican is open to listening and learning about stem cell science.

Vatican Conference Hopes to Promote Truth on Adult Stem Cell Therapy

http://www.zenit.org/en/articles/vatican-conference-hopes-to-promote-truth-on-adult-stem-cell-therapy

Following is a video link to a remarkable young lady who spoke at the 2013 Vatican Conference. She and her mother describe how horrific disability was stopped and reversed by the same HSCT procedure I will soon undergo with Dr. Burt. Though her MS attack started much earlier in life than mine, her MS symptoms and frustrations with conventional MS drug treatment were very similar to me.

http://www.youtube.com/watch?v=J7B2KQIyrZA

The Purpose of All This for Me

Despite following standard of care MS drug treatments and suffering their side effects, my MS advanced from a slow relapsing remitting form to a more rapid aggressive form in June 2013. It put me in a wheelchair for awhile.  After some extreme temporary treatments with steroids and blood plasma exchanges, my vision and hand coordination has improved and I am just now starting to ambulate with a cane. This is a very positive indication that stopping the immune system attacks with chemo will put me on the road to recovery.

In Chicago, the plan is for my harvested adult stem cells to be transplanted back to me after chemo wipes out the defective immune system that is currently attacking my brainstem. In short, MS is trying to cripple and kill me, so I am turning to fight back and kill it with chemo first. If all goes well, my stem cells will be transplanted back to quickly rebuild a new immune system that will no longer attack my central nervous system.

I could not do this without remarkable advances in technology that will surely become safer and better over time for future patients. But for me, the time is now. I am putting my life in the hands of Dr. Burt and his competent staff. I am being afraid and doing it anyway, with great confidence and faith that this medical adventure will work and give me my life back.

Thank you all for your support, well wishes and prayers.

Follow the MS Money

As described in my previous post, I am moving ahead with HSCT, an experimental procedure that uses chemo and stem cells to halt MS.  This attempt to stop and perhaps cure my MS will cost me significant time, suffering and treasure. It would be easy to chicken out of what I'm about to do.

Except... In addition to maxing out my health insurance deductible every year since my 2007 MS diagnosis, I've been keeping notes and saving an inch-thick stack of medical records to remind myself of some things that that have hurt my health and just really angered me. To sum up what's wrong with health care, and MS treatment in particular, I defer to the old journalist axiom; follow the money. My story is surely a microcosm of a larger problem. My abbreviated MS treatment 1-year diary follows.

March 2013: How Teva, Biogen and FDA probably put me in a wheelchair
The oral drug BG-12 was supposed to be FDA-approved and ready for me to use by March 2013. I needed BG-12 to help me transition off Biogen Idec's infusion drug, Tysabri. Tysabri had stabilized my MS for two years, but a blood test in August 2012 had showed the drug was seriously threatening to give me a deadly brain infection called PML. My neurologist's plan was to safely wash me out of Tysabri and start BG-12 as a replacement. His logic seemed prudent, so I agreed.

But then Teva Pharmaceuticals gamed the FDA to delay BG-12 approval. 
http://www.bloomberg.com/news/2013-01-10/teva-cites-bg-12-safety-in-asking-fda-to-add-ms-reviews.html

The FDA delay preserved another quarter of market share for Teva's old first-line MS drug, Copaxone. Teva made millions of dollars off this gambit. I'm sure they did not care even a little bit that their brilliantly orchestrated delay tactic caused thousands of patients to suffer longer with painful Copaxone injections, or me to become wheelchair-bound from a vicious MS attack.

June 2013: An expensive emergency
The resulting MS attack manifested in my legs and torso being locked up in excruciating spasms. Vision and hand coordination also suffered severely. Ataxia and incontinence became an issue. It was hard to do my job as a freelance PR writer and I lost many billable hours, not to mention an entire summer of Minnesota sailing, camping and summer festivals with friends.

The initial rescue treatment to release the spasms was a drug called Acthar. But why $28,000 for ONE week of what is basically hog adrenaline injections? I needed two weeks of Acthar to start recovering from  my MS attack. Wait until you see how much the same drug used to cost.

http://www.fiercepharma.com/story/135m-rights-deal-questcor-locks-rival-its-pricey-acthar-drug/2013-06-17

September 2013: The outrageous remarketing of Campath -- Why I'm not waiting for Lemtrada
Instead of doing HSCT, my neurologist recommended I wait for FDA approval of a "new" MS drug called Lemtrada, expected sometime in 2014. Hmm, wait for FDA approval... where had I heard that before? Then I learned about the drug side effects and where Lemtrada came from and I got angry again. Sanofi thinks it can hold MS patients and their insurers over a barrel for a huge markup of an old drug. And they'll probably get away with it.
http://www.fiercepharma.com/story/sanofi-pulls-campath-clear-way-higher-priced-lemtrada/2012-08-21

Overview: MS drugs cost $40,000 to $60,000 a year

http://www.healthline.com/health-news/ms-why-are-ms-drug-prices-so-high-071913

MS is hellishly expensive for patients and very profitable for drug companies. With billions at stake, how hard do you think these companies will actually work for a cure? How hard might they scheme and lobby the FDA to delay a cure?  I'm not a crazy conspiracy theorist. I've just watched and experienced MS market dynamics in action, and know that I'm very tired of being a cash cow. Bring on the  HSCT chemo.

Chemo, Stem Cells, MS and Me

In December, 2013, I will become one of the relatively few people with multiple sclerosis to undergo an exciting and unconventional autoimmune disease treatment called autologous hematopoieic stem cell transplant (HSCT) at Northwestern University in Chicago. Out of the roughly 2 million people globally who have MS, just over 100 patients have received this treatment under the supervision of Dr. Richard Burt, one of the world's top experts in the field of immunology.

MS is an autoimmune disease where the immune system mistakenly attacks the myelin sheath that insulates and protects nerves in the spinal cord and brain. As MS attacks continue, the body's central nervous system starts to short out and bad things happen to coordination, mobility, vision and other basic body functions. Early research trials have shown great promise in HSCT stopping this immune system attack. Because research data continues to be collected, Dr. Burt will not yet use the word "cure" to describe HSCT. However, results so far indicate that HSCT is the closest thing to a cure that exists today. I am very excited to be part of this research.

The HSCT process, which requires chemotherapy and stem cell harvest over two months, is NOT quick or easy. You can see a complete overview of the procedure, here. The transplant roadmap at that link outlines what will be my time commitment to medical procedures in Chicago. I am thankful for my supportive family as well as my my friends Anna, Tony, +Doug Rohde  and +Sylvia Bachmann who will be accompanying me as caregivers and sources of sanity during parts of my treatment journey.

Why me, why now?

No doctor directed me to seek HSCT. In fact, my neurologist tried to discourage me when I sought the procedure on my own. Seven years of faithfully following his FDA-approved standard of care with MS drugs had failed me.  I was in pain, crippled, scared and mad as hell. I couldn't legally punch him in the nose or key his Porsche, so I turned my anger into motivation to find better options. After screening out much online quackery and unhelpful advice to live on a paleo diet of free-range yak cheese, Tibetan prayer flags, quinoa and kale, I found legitimate technical white papers like this one and this one. I also started finding amazing stories like the young man in this video who was part of an early HSCT research program. The video includes an excellent description of the HSCT procedure. These findings directed my successful letter-writing campaign to Dr. Burt and his staff

In June 2013, months before my first scheduled face-to-face meeting with Dr. Burt in Chicago, a vicious MS attack put me in a wheelchair and caused other severe neurological problems. But I did make it to Chicago in a borrowed wheelchair. Thanks to Goodwill for the wheelchair and Delta Airlines for accommodating the accessibly needs of a disabled solo traveler who did not know how to be disabled. Also thanks to Doug for straining his back getting my spasm-crippled body in and out of his truck. Every hope I had was hinged on making it to that meeting. The meeting was a success, perhaps partly because it was clear to Dr. Burt that I would have crawled to get there.

When I got back to Minneapolis, I faced the daunting prospect of stabilizing my condition until my HSCT treatment could begin. The best my neurologist had to offer was plasmapheresis, a dialysis plasma exchange process that temporarily removes the nerve-attacking antibodies in my blood plasma. It sort of works, so I guess I'm glad I left his Porsche alone. Though plasma exchange is now preventing nerve damage and has helped me start tenuously walking again, it is basically like bailing out a boat with  a hole in it – new defective antibodies just keep flowing in from my defective, hyperactive immune system. Until I reset my immunity with HSCT, I am stuck with a semi-permanent catheter in my chest and weekly trips to a dialysis center. Through summer and fall, I have seen the dialysis nurses so much that they're starting to seem like family. Perhaps I should marry one of them... Wait, no, then she would not have to be nice to me.

Sobering realities and hope
MS severity varies widely from one person to the next. Correct treatment for one person is not necessarily right for another. Aggressive MS like mine often ends with the person permanently disabled and stuck in a wheelchair until they die. Though I pray for and admire the people I know who have come to terms with that fate, I will still fight to the last to try and avoid it myself. I am fortunate to have an audacious plan and something more than hope allowing me to think this is possible. And I hope this research will somehow help the larger MS population too.

One key to success with HSCT is not waiting until the disease progresses past the point of causing permanent nerve damage. Though my MS is severe and apparently drug-resistant, the good news is that my disease is still in an active inflammatory stage and has not yet progressed to an irreversible degenerative condition. This puts me at a balance point where the risk of treatment with HSCT is offset favorably by the odds that the procedure will halt the disease and improve my condition, perhaps to the point of total remission. My window of opportunity and time to act is now. Waiting helplessly for another year, hoping for the next MS drug that may or may not work, is no longer a forced option for me.

Chemo
Though "stem cells" is the trendy term that jumps out in the title of this treatment, high-dose chemotherapy, similar to what cancer patients receive, is actually the thing that will be used to stop my aggressive, nerve-flaring MS autoimmune attacks.

The high-dose, punctuated chemo used in HSCT is somewhat risky, but ultimately more effective and  less damaging to the human body than the extended periods of low-dose chemo that neurologists sometimes prescribe for aggressive hard-to-treat MS like mine. The higher-dose chemo with HSCT is made possible by first harvesting adult stem cells from the patient's own bone marrow. Infection is a danger after chemo wipes out the immune system, so patient isolation and antibiotics are key to survival until the stem cells can be transplanted to rapidly build a new, properly functioning immune system.

Adult stem cells

The term "stem cells" tends to freak some people out, but before engaging me in a Bible verse duel, you should know that the HSCT procedure uses adult stem cells, not embryonic stem cells. Really, check out the link. The science is fascinating and there is no ethical controversy surrounding adult stem cells.

HSCT also has nothing to do with the fraudulent easy-peasy stem cell mills in Latin America that have been exposed as snake oil ripoffs by investigative journalists. Contrary to claims from the unregulated banana republic clinics, stem cells won't produce lasting autoimmune disease remission without first stopping the autoimmune response with chemo.  

Cure me, kill me, or somewhere in-between
What, me worry? I could die from this procedure, but probably won't. As of yet, HSCT patient numbers represent a small sample size to base any medical statistics on. But I am an optimistic lab rat. So far, about 85% of people who have done HSCT had their autoimmune diseases halted, and, like the young man in the earlier video, many regained functional mobility to the point of feeling they are cured.  Odds of mortality from HSCT are debatable; different experts with different agendas say anywhere from 0.5% to 5% mortality. Putting it into perspective, MS itself could kill me if it's allowed to keep attacking my brainstem.

Pay it forward

Despite MS,  I've been fortunate to have the determination, energy, social support and available resources to pursue HSCT treatment. Though I'm still battling with my insurance provider and may have to pay for the entire treatment with my own money, I do not need or desire a fundraiser for myself.

However, I have been so surprised and delighted by the many friends, colleagues and family members who have reached out and offered to help me during these past few very difficult months. I feel it would be a shame to waste all the goodwill and awareness of so many remarkable people, some of whom have family or friends also suffering from MS and other autoimmune diseases. So,  please, please consider making a tax deductible donation to HSCT research.

Donating directly to the HSCT program is important because most existing MS Society fundraisers are sponsored by large pharmaceutical companies with an agenda to sell very expensive MS drugs. Moreover, the vast majority of MS research is funded by pharmaceutical companies. HSCT is a procedure, not a drug, so it does not benefit from drug company research funding. HSCT research at Northwestern is funded primarily by grants from the National Institutes of Health, patients and donations. Donations lower the cost of treatment for future patients.

Thanks!
Wayne

*DISCLAIMER: Nothing in this blog constitutes medical advice. Nor should any of it be considered a credible source of facts or attribution for journalists. I am merely a self-educated patient pursuing MS treatment and sharing some of what rolls around in my addled brain along the way. The blogosphere is populated by lunatics, sages and fools. I may be one of them or all three. Believe and proceed at your own risk.

**My thanks to George Goss, Barb Yoder Coppins and all the participants in their moderated discussion groups for sharing information resources, encouragement, experiences and knowledge about HSCT.