Friday, December 19, 2008

Indecent FDA delays, Shades of Dr. Mengele

The issue of clinical trial access is far bigger than most Americans suspect. I will be on the Dr. Ken Kornhaus radio show tomorrow (Saturday) at 8am EST with Frank Burroughs of Abigail Alliance to discuss the phony promise of Compassionate Use under current FDA regs, the pointless bureaucratic delays of revolutionary new meds and therapies, and the increasing difficulty of getting into clinical trials due to inclusionary and exclusionary criteria that eliminate the sickest patients.

I urge all of you to listen to the show and call in with comments. You and your family may be in great health now but in a moment that can change. Once you get the “horror” diagnosis any extra time or finances you and your family have will likely be consumed. There will be precious little extra left to fight the FDA and big pharma. Get involved now. Look up Abigail Alliance and find out how.

There is a revolution in research going on in America right now. The medications and the therapies were undreamt up a decade ago. But this revolution is not getting to patients. The journey from lab bench discoveries to patient bedside therapies has become an ever lengthening process often taking decades. More awful still is that dozens of discoveries never get to patients because of the paperwork, time and especially cost of this process. These discoveries never advance. No one ever gets help. Something needs to be done. One start is a reform of the FDA clinical trial procedures to increase access and efficiency.

New research knowledge offers the possibility of the end of some of the most horrible chronic diseases in history of humanity. Researchers can manipulate cells inside the body with drugs like the PTC 124, so that slight errors in genetic instructions can be ignored by the cells gene reading machinery allowing the gene to function properly and the disease process to stop. There are meds called HDAC inhibitors which can turn on incorrectly turned off genes. Cancer in many patients is caused by p53 cancer suppressing gene being wrongly shut down. Turn on the p53 cancer suppressor with HDAC inhibitors or other similar drugs and the cancer melts away.

Read more about the denial of access to PTC124 in this article:

Other techniques allow immune cells to be manipulated inside the patients body with the result that "tolerance" cells can be exponentially increased. These “tolerance” cells normalize the immune system and turn off immune dysfunctions that cause various autoimmune disease and possibly even asthma and allergy. Or the immune cells can be “revved up” and targeted to kill particular cancers or infectious pathogens. All of this and far more is possible and it is being done today in labs and clinical trials. Far too many of us are dying while we wait for these advances.

New ways to kill resistant and increasingly virulent bacterial infections like MRSA, C diff, Iraqi bacter and others are held back from dying patients.

A father of three children at my wife's K-5 elementary school got a small cut on his hand on the job as a plumber. A resistant bacterial infection set in. Three days later he was dead! The brother of one of the teachers at the school was burned in a garage fire. He could have survived the burns, but he did not survive the infection. A perfectly healthy12 year old boy at a nearby middle school (Ramona) got the flu last winter ten days later he was dead from a bacterial pneumonia.

Three cases, three different doctors (at least), not one doctor asked for a compassionate use exemption for clinical trial antibiotics that might have saved all three lives.

Why? Not because they were lazy. No, because the compassionate use process is a dinosaur. It is incredibly time consuming and difficult even for emergencies. When doctors do try the companies invariably deny the request. NO is always the easy and safest answer. A “yes” might result in some unexpected outcome that would have to be reported to the FDA which could delay the already snail like pace of the FDA approval process even further. Each delay costs million maybe 100s of millions in lost sales and additional tests.

Recently an FDA approved cancer drug (Rituxan) showed remarkable efficacy in stopping Multiple Sclerosis. One of the researcher team members did not want the results shared. He said he was afraid too many MS patients would obtain Rituxan “off label” and there would not be enough MS patients available for his clinical trials to be completed. Shades of Josef Mengele! This researcher’s need for fame and compensation was far more important to him than the suffering of his fellow American citizens. We, the chronically ill, are more than clinical trial fodder. We are suffering human beings.

Worse, the FDA approval process is ever longer and ever slower. Fewer new drugs are approved each year. Bush budget cuts did not help. There are constant delays in the process. Bureaucratic black holes which shut down the approval process suddenly appear for who knows why? or for how long? It is always safer for FDA bureaucrats to say "no, let’s wait" then, "yes let’s go".

Clinical trials cost a billion dollars each and not just for each new drug, but worse for additional testing of existing FDA approved drugs for similar disease. For example Rheumatoid Arthritis (RA) drugs must be tested and FDA approved again before being used for Psoriatic Arthritis (PsA)—a nearly identical disease. Every PsA med was first tested for RA. There is not one RA drug tested on PsA patients that did not also work in PsA, yet PsA patients must wait years more for FDA approval while RA patients have the access to drugs.

PTC124 could save the lives of half of all the cystic fibrosis patients in Israel and up to 10% of cystic fibrosis patients in US. PTC 124 and similar meds have the potential to end any chronic disease that is caused by a tiny error in the DNA that causes the gene not to function. PTC 124 should work for some patients with autoimmune diseases and cancers. HDAC inhibitors hold the similar promise for a colossal range of chronic diseases.

Decency demands that these drugs be rushed to approval and allowed for every possible disease they might help.

Chinese medical tradition requires that the physician try to help the patient. Giving up is not an option. Western medical tradition says, “First do no harm.” Doing nothing may have been a good philosophy 3000 years ago when there was little knowledge and lots of bad out comes. Today it is a terrible philosophy. Doing nothing condemns thousands of Americans to death every year—that just those who die of out of control antibiotic resistant bacterial infections. Doing nothing is doing harm. How many more die of chronic conditions that could be helped and helped fast if the FDA clinical trial procedures were streamlined.

My son nearly died a year and a half ago of suffocation as his ribcage froze while a medication that could save his life and turn off his three related autoimmune diseases was denied to him. I could not get approval for Compassionate Use from the company even though phase II and III clinical trials results released months before my son’s near death were called "stunning" by outside observers. This medication that he was denied beat the most prescribed FDA approved med for his condition in head to head clinical trials. He was using that FDA approved medication and he was dying. How much worse could he get on a clinical trial med with such promise? Yet he was denied access.

In June of 2008 and FDA expert panel UNANIMOUSLY recommended approval of that medication. Wouldn't you expect the FDA to immediately approve it for marketing? No, of course not. As of today more than six months later, the FDA rhymes with DELAY still has not approved the medication. By the way, the name of that medication is Ustekinumab (CNTO 1275)—a FULLY human monoclonal that targets DIFFERENT immune inflammatory signals (IL12/23) than any other medication on the market. It is unique or would be if the FDA would stop stalling.

My son is still alive today only because another older and FAR more dangerous med has allowed enough reduction in inflammation for his ribcage to work again. Each six week infusion of this very dangerous medication made with NON-human mouse proteins is a spin of a medical Russian roulette. At any infusion his body could have a fatal adverse reaction to the FOREIGN mouse proteins.

What would you do if your child was dying and the medication that could save his life was locked up and denied to him? What value is human life in America today? What is more important antiquated FDA protocols or human life?

No comments: