The following is a copy of part of an email I sent to a fellow autoimmune suffer. He has psoriasis like my son but not PsA or AS. I thought the email summed up a bunch of problems we in the chronically ill community suffer from. I decided to share part of the email with this blog audience. I ADDED SUBTITLES TO THE LETTER FOR THIS POST.
NO LUCK GETTING NEW REVOLUTIONARY PSORIASIS MED--USTEKINUMAB
No, Paul has not yet had the opportunity to try Ustekinumab despite my best attempts to get it for him. We almost got him into a clinical trial to test Usetkinumab on PsA but his psoriatic lesions were not large enough. It did not matter how bad and disabling his PsA was.
It’s sad that there are not better ways to measure the systemic effects of PsA then to physically look at the skin and joints. Wouldn’t it be nice if there were blood tests looking at gene expression to determine the status of the disease instead of subjective quality of life measurements and size of lesions?
IF FDA ALLOWED USTEKINUMAB LIKELY WOULD HELP RA, DIABETES I, AND MS
I also think it is absolutely unconscionable that a medication with such promise as Ustekinumab can be denied to people in need for so long. The FDA approval process is way too slow and based on archaic disease classification criteria. If Ustekinumab is FDA approved this month for use, it will only be approved for psoriasis. Yet its mechanism of action very likely would be of benefit for related inflammatory autoimmune diseases as well.
It should certainly work for PsA and ankylosing spondylitis. Every medication that helps PsA started as an RA medicine so shouldn’t CNTO 1275 be very likely to be useful for RA as well as PsA? I would think that diabetes type I and even MS might be helped. Too bad the FDA requires separate billion dollar clinical trials for each condition rather than allowing for the testing of many related conditions at once.
CIMIZIA (CROHN'S APPROVED) AS A POSSIBILITY?
As to the Cimizia and Paul, no we have not tried it. Because of Paul’s history of hives angioedema and breathing problems with medications and foods, a “humanized” monoclonal like Cimzia is not my first choice. Humanized monoclonal are up to ten percent mouse protein. Right now Paul is taking Remicade a chimeric monoclonal that is up to fifty percent mouse. Each infusion is scary like spinning the cylinder on a revolver in Russian roulette.
He and all other patients who are infused with Remicade must take massive doses of steroids and antihistamines before the Remicade to try to prevent reactions. Even so many patients especially those like Paul develop severe adverse reactions after a few to few dozen infusions.
Cimzia is not FDA approved for psoriasis, is it? I know it has been in clinical trials for psoriasis. I have not heard of it being approved for psoriasis yet only Crohn’s. So we would have to use it off label. Just googled it. Supposed to be less likely to cause reactions—less immunogenicity—don’t you love research talk? Hmmm. Maybe it would be worth looking into.
DOCTORS ARE UNWILLING TO GO 'OFF LABEL' CAREERS MORE IMPORTANT THAN SICK PATIENTS
Except we have had difficulty getting physicians to use medication “off label” even when clinical trials indicate it might be helpful. We have tried for years to get Rituxan* for Paul—no luck so far. We even tried to get him into a clinical trial for Rituxan with no success. Also my wife’s medical insurance refuses to cover off label use which if we could find a doctor to prescribe Cimzia, it would be an off label prescription. We would have to pay all of it out of pocket.
For Rituxan the cost of a complete course of treatment would be less then $10,000. Expensive but doable if we found a doctor willing. Cimzia, once a month subcutaneous, would cost what? $600 to $1000 I would guess based on what Enbrel and Humira cost. That would be in the $10,000 a year ball park. Same price. Still where could we find a doctor who would be willing to prescribe “off label” and risk his “career investment” as a physician?
REMICADE WITH MEDICAL INSURANCE COSTS $2000 EVERY SIX WEEKS--ALMOST TWENTY THOUSAND DOLLARS OUT OF POCKET A YEAR!!
We are spending around $2000 out of pocket every six weeks for Remicade infusions, currently—close to $18,000 a year. Maybe Cimzia would be worth asking about if Ustekinumab is not approved this month.
TNF ALPHA INHIBITORS KEEP PAUL BREATHING
Certainly TNF alpha inhibitors like Cimzia help Paul. They keep his chest flexible enough to allow him to breathe. When the Remicade starts to wear off he starts to have more difficulty breathing—not as bad yet as the two suffocating episodes in summer of 2007 when the Enbrel stopped working. (Paul says he feels like the Tin Man in the Wizard of Oz before Dorothy oiled the rusty joints in the week or so before he gets the next Remicade infusion as the benefits of the previous infusion wear off)
Thanks for the Cimzia idea. Being FDA approved and on the market it has to be easier to access than Ustekinumab has been.
PAUL NEEDS FULLY FUNCTIONING IMMUNE CELLS
My long term goal is to find some way to get fully function regulatory immune cells working in Paul’s body (and mine too.) The closest to that happening that I can find are the clinical trials using Prochymal from Osiris.
PROCHYMA FROM OSIRIS MY NEW GREATEST HOPE FOR THE CURE
Prochyma is a mixture of blood and immune forming cells from a lot of donors. It has had tremendous success in Graft versus Host Disease, Crohn’s and now diabetes type I.
Somehow getting these pre-immune and blood cells from lots and lots of donors overcomes the problem of the new immune cells attacking and destroying the recipients tissues (graft versus host) which has been one of the main problem with using bone marrow transplants as a way to cure autoimmune disease.
PROCHYMA DRAWBACK--IT'S PERMANENT
The drawback of Prochymal is that the infused cells cannot be tracked or eliminated once transfused and they can stay alive for decades even for entire life of the recipient. If something goes wrong, it stays wrong.
PAUL NEEDS FULL ACCESS NOW!!!!!!!!!!!!!!
Yet if Paul and I had free access to any experimental drug or therapy, I would grab Prochymal in a minute. I have read anecdotal stories of Crohn’s patients, with decades long disease who two weeks after having the infusion of Prochymal had no sign of Crohn’s in their bodies. Endoscopes of intestines were completely normal. Patients had complete relief from their disease for first time in their lives. If Paul had an infusion of Prochymal today, he could be running around and playing ping pong with me by Christmas. If I got it, I could go back to work in January. Just the possibility of that cure fills me with hope. I do not think it is false hope. The only thing separating Paul and I from living normal lives is properly functioning immune cells—the kind that normalize out of control immune reactions.
RADIO SHOW APPEARANCE
That hope is why I am going on the radio show. I want patients to have better access to clinical trial drugs.
WHERE ARE WASHINGTON LOBBYISTS WHEN YOU NEED THEM?
If I were a well placed Washington insider, I would advocate for the FDA to create a special classification for severely incapacitated or those in imminent danger of death as “accident victims,” then allow pharma companies to treat those patients without any danger of liability under some kind of “Good Samaritan” type umbrella of protection from legal liabilities and negative drug approval process consequences.
LET PAUL AND I BE "ACCIDENT VICTIMS"
With such a classification people like my son and I could have access to any experimental medication that might help. The Pharma companies would be able to test new drugs and therapies on a wide variety of human diseases without fear that a failure would doom approval of their medication. In fact they may serendipitously discover that their drug is even better for a related condition, then for the one they were going to clinical trials with.
GOOD SAMARITAN PHARMA COMPANIES WOULD PROFIT
Besides relieving the pharma companies of any worries of negative consequences if they allowed expanded compassionate use for “accident victims”, the willing companies the “Good Samaritans” should also be able to get all positive results on “accident victim” patients as officially approved FDA data to be used in the FDA approval process of their new drug. I bet pharma companies would jump at the chance for more humans with more conditions being tested especially if there was a chance that they could get their new drug to market faster with positive data they could get fast and cheap.
LAB BENCH TO PATIENT BEDSIDE NOW
With so many really amazing new therapies and drugs being developed, there is a huge need to get the research advances from lab bench to bed side quicker.
LET US BE HEROES, LET US EXPLORE THE UNKNOWN TO FIND CURES FOR OURSELVES AND OTHERS!
Paul and I would volunteer to be the “accident victims” in a heartbeat. Actually I would see us more like heroes or explorers willing to go out into the unknown at risk to ourselves to pave the way for better treatments for others.
I wonder if I can say all that in a ten minute segment on the radio?
Sorry to be long winded. I am so excited by the progress being made in the research community. Autoimmune disease is a fixable problem and the fix is at hand.
I asked for two hours segment and was offered ten minutes—better than nothing. I also asked if Frank Burroughs of Abigail Alliance can be included to talk about his Full Access legislation in the House and Senate. I hope to be on the radio show this weekend or next.
Hope all is well with you and your family.
Let’s make our generation the last one that ever has to suffer with autoimmune disease.
Father of Paul
RITUXAN AUTOIMMUNE REMISSION POSSIBILITY
*You probably know that Rituxan depletes B cells. A few “maladapted” B cell clones are responsible for AUTO-antibodies. Rituxan gets rid of all B cells including both the good ones (that make antibodies to childhood illnesses, etc.) and the “bad” AUTO-antibody producing ones.
Once the “bad” B cells are destroyed there are no AUTO-antibodies produced and the autoimmune disease goes into remission for weeks or even years.
DOWNSIDE OF RITUXAN--DEATH BY INFECTION
Down side risk of Rituxan is the chance of life ending infections picked up in the environment after B cell depletion or from existing hidden and suppressed but not dead viruses in the patient’s body.
PML is the worst of the lot that include various herpes viruses. PML is a potentially lethal brain virus that is hidden and suppressed in a large part of the American population. Perhaps one in 2000 patients who take Rituxan will die from PML. So Rituxan is also Russian roulette, but with the hope of a long term remission of the disease.
Even with the chance of death Paul would take Rituxan if he could. So far no doctor in our area will prescribe it for Paul. It is “off label” for PsA.