Off the (Tysabri) Rebound

Someone asked if there is anything I wished I'd known before doing the chemo/HSCT procedure.

Yes. I wish I'd had more comparative safety data regarding HSCT versus FDA-approved immune modulating drugs. Dr. Burt's research, and information provided by HSCT patient-activist George Goss, were of great help in providing counterpoints to the anti-HSCT scare tactics my neurologist threw at me. But the scare tactics were traumatizing nonetheless.  I steadied my resolve to fight to the death and updated my last will and testament before heading to Chicago for my chemo treatment.

It turned out the HSCT/chemo risk was overblown, and the risk from the Tysabri I'd been on had been downplayed.  The following article hit the financial press just before the crippling MS rebound attack that resulted from my neurologist taking me off of Tysabri. MS rebound from a pent-up overaggressive immune system often happens when MS patients are taken off Tysabri.  The rebound concept wasn't really addressed in this article, probably because Biogen is still figuring it out as neurologists continue to prescribe the drug. Though the article does contain an NIH link to "worsening symptoms" resulting from coming off Tysabri. The technical term is Immune Reconstitution Inflammatory Syndrome (IRIS).  The article headline speaks volumes.

MS Patients Choose Death Risk With Potent Drug Treatment

Australians demand HSCT for MS

Aussie MS patients are justifiably angry. Their misinformed MS Society has helped shut down HSCT in that country. The society refuses to acknowledge that the procedure can be used to help patients recover from the intense level of chemotherapy needed stop aggressive MS in its tracks, as it did for me.

What's troubling is that the same shutdown might happen here in the US if our own MS Society decides to push in that unfortunate direction. I'm resistant to entertaining conspiracy theories, but it is difficult to fully dismiss the idea that money from MS drug companies might have undue influence on the talking points and policies of national MS Societies.  When I go to www.nationalmssociety.org, an onsite search for "hsct" or "hematopoietic" turns up scant information. This seems very wrong on a website with a homepage that leads with "A World Free of MS" and "Research for a Cure."  At the moment, the US National MS Society's policy toward HSCT seems to be one of benign neglect. Too bad, considering chemo and HSCT is the closest thing we have to a proven MS cure.

In this video, the Australian doctor's comments mirror what Dr. Burt at Northwestern says.  HSCT is a proven procedure, not an experiment. And there is no moral dilemma, since the stem cells come from each patient's own body.

The video also includes an explanation of the procedure, and some interviews with patients who have done it by traveling overseas.

http://au.news.yahoo.com/sa/video/watch/21715059/stem-cell-therapy-push/

Changed to the core by HSCT?

Well, this could explain a lot.

An interesting study in the journal Immunology, Oct., 2013, looked at immune system T-cell response after HSCT treatment compared to patients treated with natalizumab (Tysabri).  The upshot seems to be that HSCT resets the immune system to be like a normal, healthy individual, while Tysabri-treated patients maintain imbalanced/sick immune systems. This may explain why I continued to lose mobility during the two years I was on Tysabri. Though the drug seemed to slow the development of MS lesions, something was obviously still wrong with me.

Per the study summary: HSCT-treated patients had similar levels of Treg cells and of Th1 and Th17 cells as healthy subjects, whereas natalizumab-treated patients had lower frequencies of Treg cells and higher frequencies of Th1 and Th17 cells.   My interpretation of this is that there are not enough regulator cells in the natalizumab-treated patients to prevent the attack cells from launching autoimmune assaults on the myelin that protects nerves in the central nervous system.

These cellular-level observations also seem to explain why so many (85%) of Dr. Burt's patients appear to go into complete MS remission after HSCT. Changed from the core of the bone marrow, the immune system becomes normal.  With my HSCT completed, I look forward to further improvement, without MS drugs.


http://www.ncbi.nlm.nih.gov/m/pubmed/23721329/?i=10&from=autoimmune+diseases+after+hematopoietic+stem+cell+transplantation

WBC = Happy Valentines Day

Now that I'm home, weekly blood tests monitor my cell counts to make sure recovery is staying on track.  I'm pleased to say so far, so good. White blood cell count (WBC), in particular, is in the "normal" range. I'm getting out in public more, and am thinking about going into the office next week.  They have much work for me, I miss the gang, and it's time to get my bank account moving in a positive direction again.

I still have a few restrictions:  I must  avoid sick people, sushi, Chinese buffets, salad bars, gardening, plant potting, moldy basements or fraternizing with feral animals. Sadly, the animal restriction includes my mother's indoor-outdoor cats.  Though she keeps her house very clean, there is just too much risk from whatever a cat might drag in from the wild. My immune system is still redeveloping, so a cat scratch, weird raccoon infection (or who knows what) could set me back.  I will try to visit mom and dad half-way at a non-cat location soon.

Here's a quirky photo of white blood cells for Valentine's Day.

Keep fighting, keep learning

Thanks to my friend Sharon for reminding me about David Bexfield.  Mr. Bexfield had the same form of aggressive MS I did. He was accepted for aHSCT treatment, which halted his MS in 2010.  My own aHSCT treatment concluded in February 2014. It is my hope that my results will be as good as, or even better than his.

Similarly to me, Dave had to pay for the treatment himself because his health insurance would not pay. Dave is still battling with his insurance company to try to get reimbursed for the cost of the treatment.  Here is Dave's video , which emphasizes that aHSCT treatment probably saved his life.

One of the last frames of Dave's video references a scholarly paper that outlines the benefits of aHSCT. That paper will now be added to my State of Minnesota appeal to overturn my insurance company's rejection decision. Thanks, Dave. Good luck to us all.

----------
The following excerpts include highlights from the scholarly paper, Autologous Hematopoietic Stem Cell Transplantation as a Treatment Option for Aggressive Multiple Sclerosis, Current Treatment Options in Neurology, June 2013. Nikolai Pfender, MS, Riccardo Sac card, MD, Roland Martin, MD

The full text of this paper may be purchased at www.springer.com


Opinion statement

Despite the development of several injectable or oral treatments for relapsing-remitting multiple sclerosis (RRMS), it remains difficult to treat patients with aggressive disease, and many of these continue to develop severe disability. During the last two decades autologous hematopoietic stem cell transplantation (aHSCT) has been explored with the goal to eliminate an aberrant immune system and then re-install a healthy and tolerant one from hematopoietic precursor cells that had been harvested from the patient prior to chemo- therapy. Clinical studies have shown that aHSCT is able to completely halt disease activity in the majority of patients with aggressive RRMS. Research on the mechanisms of action supports that aHSCT indeed leads to renewal of a healthy immune system. Below we will summarize important aspects of aHSCT and mention the currently best-examined regimen.

...Eight drugs are currently available and multiple others either already filed for approval or in late stage clinical development. However, due to the chronic nature of MS, all of these need to be given for long times or forever. Further, all of these have side effects and some of them very serious ones, and, depending on their route of administration, some treatments compromise quality of life. Also, most of these are very expensive, and lead to substantial socioeconomic burden...  

...in recent years, aHSCT has advanced substantially, and transplant-related mortality has been between 1 %–1.5 % since 2000 with the BEAM-ATG regimen. Through tight collaboration be tween hematologists/transplant specialists and neurolo-gists aHSCT is now more standardized, mortality is within the range of the most active approved therapy, ie, mitoxantrone, the efficacy is likely superior to all other available treatments, its mechanisms are better under stood, and aHSCT is probably the only treatment of MS that has to be applied only once with no further need for therapy in the majority of patients, provided that they have been selected carefully...

... Some of the MS drugs, eg, mitoxantrone or alemtuzumab, have long-lasting effects on the hematopoietic system or carry the risk of secondary malignancies and cardiac damage (mitoxantrone) or of secondary autoimmune diseases (alemtuzumab). Natalizumab (Tysabri) may lead to PML with increasing risk following 2 years of treatment or even higher risk following prior treatment with chemotherapeutic agents such as mitoxantrone...

...More than 500 MS patients have received aHSCT in Europe alone in the last 20 years, and followup for substantial fraction of these is longer than 10 years. A joint study of the European and American Bone Marrow Transplantation Societies on long-term outcomes after HSCT in MS is currently ongoing...

... existing data indicate that even the most effective available drugs need to be given continuously, that particularly the most effective therapies carry substantial risk, and that they are expected to be inferior to aHSCT regarding their efficacy. Further, aHSCT is a one time treatment with no need for continuing immunomodulation, and, when considering the cost of currently available treatments of up to 45,000 USD/year, will lead to substantial socioeconomic benefit in patients with aggressive MS... 

Doctors jumping on the bandwagon

It's very promising for patients that more researchers and doctors around the world are now inferring that they discovered the procedure similar to what I just did with Dr. Burt at Northwestern. This video released by Johns Hopkins seems to follow that pattern, trotting out their own MS patient success stories. That's a pretty good indication that something works, I think.

Dr. Burt started studying high-dose cyclophosphamide and HSCT in animals 30 years ago, and he is now into Phase 3 human studies. That probably puts him ahead of everyone else in this field. But frankly, I really don't care who gets the credit for being first.  Every good result reported by other patients and doctors is good news for me, and for many patients with aggressive MS and other autoimmune  disorders.

If you are an MS sufferer interested in treatment with Dr. Burt, I highly recommend that you review the below criteria for his current clinical trial.  If your treatment history and condition is a close match to those criteria, there's a good chance his procedure can help you.  For other autoimmune diseases, just use the search engine on the clinical trials site.

Stem Cell Therapy for Patients With Multiple Sclerosis Failing Alternate Approved Therapy- A Randomized Study

http://www.clinicaltrials.gov/ct2/show/NCT00273364?term=Autologous+hematopoietic+stem+cell+transplantation+for+multiple+sclerosis&rank=9

Insurance appeal during recovery

Just days after release from Northwestern Hospital, I'm home feeling pretty good about my decision to pursue HSCT.  I'm now walking short distances with no cane, driving my car and getting things done. This has included fun things like hosting a mini Superbowl party, and cooking up a batch of homemade chili. I'm standing, yes standing, at my kitchen counter and chopping up fresh vegetables. Before I had to lean on the counter and stove, which made chopping and cooking difficult.

I can visit with people if they aren't sick. Just no large groups yet. My immune system is still rebounding. Once a week, I get blood drawn to monitor my immune system's progress. The first blood test since I got home showed that I'm on track.

BCBS Minnesota has refused to pay for my HSCT treatment, even after three appeals. Their repeated reason for the denial is that they will not pay for an investigative treatment.

Job one for me now is crafting a written appeal to the State of Minnesota. If I make a convincing case, the State may compel my insurer to honor its contract and pay for my treatment. Since I paid up-front out of my life savings in order to secure a spot at Northwestern Hospital under Dr. Burt's care, that would mean I'd eventually get my $125,000 back.

My case will make the following key points:
Urgent Need
--I had been failed by multiple rounds of conventional MS drug treatments and procedures since my MS diagnosis in 2007.
--A rapid acceleration of the disease in June 2013 and six new brainstem lesions had put me in a wheelchair.
--Stopping the disease quickly was crucial to prevent permanent nerve damage and disability.
--High-dose chemotherapy followed by HSCT is the only proven way  to quickly and permanently stop the aggressive autoimmune attacks I was having.
--HSCT is more cost-effective than continued use of MS drugs, which average $55,000/year, have side-effects, and merely slow the disease.

HSCT should no longer be considered "investigative."
--More than 100 MS patients have been treated with HSCT by Dr. Burt since 1996 with no mortality.
--Thousands of cancer patients have been treated with HSCT around the world. The language often used to describe this is bone marrow transplant.
--BCBS Minnesota refused to speak with Dr. Burt about the merits and safety of HSCT for my case.
--Many other insurance companies have paid for HSCT treatment.

I'm not the only Dr. Burt patient who has had trouble with BCBS Minnesota. Fortunately, I have received guidance from a previous Dr. Burt patient whose BCBS rejection was overturned at the state level. I hope to repeat his success. Also, my old friend and colleague Bernie has offered to review my submission and offer advice before I send it. He served as Minnesota Commerce Commissioner under the Ventura administration. Among other things, he was a champion for health insurance consumers. His advice will be invaluable.

This TV News feature covers a mother with MS who is trying to shame BCBS into paying. I hope it works for her. The video also includes an interview with astute comments by Dr. Burt.

https://www.youtube.com/watch?v=i6ngmOpzQ2I

Transition out

After weeks of isolation in a hospital, life in the outside world seemed farther and farther away. The nurses at Northwestern were beyond competent. They were also kind enough to chat with me and listen to stories about my life as well as share some of their own. They made me feel less alone.

More importantly, they did their jobs so well, that I had no complications. Smooth sailing. No infections. Successful engraftment. This all led to me being released by Dr. Burt on day 10 after stem cell infusion.  This best-case scenario saved me thousands of dollars in hospital time. Since I paid up-front, I should get a bit of a refund.

But with hsct,  even smooth sailing is no picnic. I experienced the initial nausea/barfing of the chemo,  then, the discomforts of getting my stem cells back.

As is typical of this procedure, I had extreme deep bone pain as the stem cells started to engraft back into my bone marrow. My engraftment occurred quickly, so I had fewer days than others in that painful state. I gutted it out without narcotics. Meditation, tyelenol and my own unusually high pain threshold got me through without crying.  I've been following the blog of a gentleman who went to India to have a similar hsct procedure done. He paid a fraction of what I did, but he's had problems, including a full-body skin rash. He's now into day 12 of engraftment with continuing bone pain. He is crying, and I wish him well. Hang in there, brother.

When Dr. Burt came into my room and said I could leave on day 10, it was a day earlier than I expected. Good news, except that I wasn't yet emotionally ready to leave.  The nurses were tolerant of me muddling around, delaying departure from a hospital room that seemed like my home.  I finally got packed, left and checked-in at a nearby hotel.

I think it was a combination of the bone pain and the emotional stress of leaving the hospital that brought on a pretty severe MS-like attack, locking up my legs again in spasms over the past 14 hours. Presumably, my frazzled nerves are no longer being attacked by  a bad immune system, but there is significant healing ahead of me now. Improvement can be expected over time.

In the meantime, it seems my leg spasms are abating. I should be OK for flying back to Minneapolis today.

WOOHOO! White count 1.5!

This morning came at 6 am from a nurse with very good news. Over the course of 24 hours, my white blood cell count has jumped from 0.1 (virtually nil) to 1.5 (reduced but good enough to enter the world with precautions).

From here, WBC counts should improve even more. It seems certain I will be flying home on Sunday, I booked a flight. Splurged and got a first-class seat. This extravagance is as much a celebretory treat to myself as it is an additional precaution against contracting a flu or something else that could set back my progress. No poopy-colic croup-crying baby section for me!

Anna will be bouncing over from her dorm at Loyola to escort me from the hospital to the airport. I have several friends on standby to pick me up at MSP.  I'll pick the one least likely to have the flu.

Bone pain from the neupogen? Yes, it's still there. I'm glorying in it.

Addendum: Dr. Burt just breezed into my room and congratulated me. He's kicking me out of the hospital today.

Hmmm, it's snowing outside, my flight for Sunday is already booked, and Anna has a busy day on campus. Rather than try to change to a flight that might end up snowbound, I'll grab a Marriott room at the Northwestern patient discount rate and just fly home tomorrow as planned.