Today in my email in box I received an email from Psoriasis Action Network, a fine organization I presume. I decided to write back with what I saw as the greatest need for action in the autoimmune community. The email below speaks for itself
Dear Alyssa Brown of Psoriasis Action Network
My son has P, PsA and AS. All are related inflammatory diseases. RA, Crohn's, diabetes type I are also related and in some of our relatives on both my wife's and my side. Similar meds and research innovation that stop immune system inflammation can help these and other autoimmune inflammatory conditions.
(1) Why don't advocacy groups work together? Wouldn't our voices be more powerful?
(2) Revolutionary therapies and medication are being delayed for decades by the current FDA clinical trial process. I have read that it cost more than a billion dollars for a single drug to go through trials for a single disease. Insane! In addition it takes a decade or more from research discovery to patient bedside. Double insane!! We need research centers, perhaps twenty or so, spread across US where new research can immediately be tried on patients hero volunteers--lawsuit free with IRBs who think first of patient and LAST about protecting their institution and jobs.
(3) There are over 80 named autoimmune diseases. The FDA requires each new therapy to be tested SEPARATELY on each of the named diseases. There are many underlying similarities in these diseases, why not a clinical trial with patients from many if not all autoimmune named conditions?
(4) The expense and lengthy time requirements to bring revolutionary cures to market mean that Big Pharma wins. They get to keep peddling less effective but very profitable 'blockbuster" drugs longer before better more effective therapies come and take away their market. The time and expense also forces small companies with innovative research to sell that research to Big Pharma or enter into onerous contracts in order to bring the research to market. Imagine if the electronics industry were forced to work that way. Today we would still be using crank handle phones!
(5) Only the suffering and deaths of a tiny handful of volunteers in clinical trial count when FDA makes a call on allowing medications to market. The massive scale of suffering and death by those who get no chance at these meds is not counted.
My son has been in wheel chair for four years. He cannot get his own food or water.
Ustekinumab was shown to have "stunning results" in clinical trials TWO YEARS AGO. It still is not approved today by the FDA rhymes with DELAY. My wife and I have gone through the Abigail Alliance and contacted Centocor senior executives asking for and being repeatedly DENIED ACCESS. Under current FDA compassionate use provisions Centocor is given the final and only say. There is NO APPEAL!
The Jacob Gunvalson court case means even the federal courts refuse to help alleviate the suffering and death of helpless patients. Do something! Why aren’t we blocking traffic and marching in the streets until treatment advances are released for patient use like the AIDS folks did in the eighties. It worked for them.
Why are we such wusses? Why don’t we fight and scream and riot? We are like abused spouses who sit passively back and re- take the abuse over and over and do nothing because they are “helpless” to change the situation. No we are not helpless. AND The situation needs changing now!
Ustekinumab beat Enbrel head to head clinical trials announced last September--still no approval. Do not accept FDA DELAY. Fight the monster. My son's suffering should count. His two brushes with death should count. Patients like him should not have to beg for access that is routinely denied.
(6) Stem cell research--Prochymal is a REVOLUTIONARY advance in treatment of ALL blood and immune cell conditions. The Osiris company has found the "magical" hematopoietic stem cells that can be infused into any human being, no matter blood or MHC type, without the stem cells attacking the patient (graft versus host) or the patient's immune system attacking the stem cells. Think what that means. If healthy immune cells that give rise to healthy immune regulatory cells, virtually all patients with all immune dysfunctions could be cured. All my son needs are healthy regulatory cells to bring balance to his immune system.
Prochymal is stuck in the FDA one named disease at a time nightmare. First they used it on GvHD and stopped it!!!! GVHD kills something like 50% of all patients who experience it in less than a year of onset. Prochymal stops it.
GvHD is like having every autoimmune disease at once so if it can be stopped autoimmune disease can be stopped. Osiris tried it first on Crohn's. It stopped ALL Crohn's symptoms in as little as two weeks. Now they are trying it on diabetes type I but it should be tried for ALL AUTOIMMUNE DISEASE NOW not decades from now.
What is wrong with FDA bureaucrats who cannot understand that if we can get working regulatory cells in our bodies then these cells can with exquisite precision control our immune dysfunctions. The control will be far better and far more targeted than the massive intermediate doses of monoclonal antibodies blocking one inflammatory cytokine at a time.. First too much blocked than as time goes on too little. Exquisite LIVING control is SO MUCH BETTER!
(7) Vaccines for autoimmune, where are they. Anti-TNF alpha, anti-maladapted dendritic and T effector vaccines--lets get them approved! Let HEROES volunteer in research centers today. Stop denying access. There are also vaccines that instead of alerting body to destroy maladapted cytokines and auto-immune cells instead promote tolerance of the proteins under autoimmune attack. These vaccines act like allergy shots. An MS vaccine along these lines has shown promise. Why aren't we finding the proteins being attacked for P, and PsA and creating these tolerance vaccines?
(8) B cell depletion therapies show tremendous promise. Why is the old mouse protein, Rituxan, still the only B cell therapy on the market? Why aren’t we getting HuMax CD 20 et al approved? What is wrong with the FDA? and what is wrong with NIH and research grants to find better B cell therapies that are more targeted to only kill the active autoimmune antibody producing B cells? instead of the "good guy" B cells that stand ready to protect us from various infective illnesses that we are immune to?
I have watched my son go from the editor of his college newspaper with a huge circle of friends to a hunched over, pain wracked, helpless disabled person. These past five years have been Hell for him.
When he first exhibited symptoms of his first autoimmune disease in his senior year of college in fall of 2003, I started reading on line everything I could. I found tremendous hope there that autoimmune disease could be cured and were being cured in the lab. We expected these cures to be immediately expedited and translated into patient cures. We waited eagerly. Then we waited some more and more and more.
Still we wait even for the simplest band-aid “bridge to future” treatment like Ustekinumab. It is should have been approved in summer of 2007 on some special provisional approval that allowed access for the sickest and those willing to give up legal rights to sue. Why wasn't it? Why do we wait?
Ustekinumab is a PRIMITIVE band aid to a problem that is could soon be curable with stem cell research. But it is a very helpful band aid, so lets get approved!
Sorry to ramble. Please forward this email to anyone you think can help get my son and other patients gain faster ACCESS to revolutionary cures currently in research labs and clinical trials.