I’ve frequently called “right to try” laws that are popping up in various states like so much kudzu, to the point where 31 states have passed them in a little over two years, an amazing pace, a cruel sham, given how incredibly unlikely they are to help a single patient. Basically, state-level right-to-try laws are the brainchild of the libertarian Goldwater Institute and all based on a template that it produced. Their purpose is not, as the Goldwater Institute claims, to help patients, but rather to weaken and ultimately neuter the FDA’s power to regulate drug approval and thereby help to usher in a libertarian utopia in which drugs don’t need approval and the free market magically guarantees safety and efficacy through various “independent” testing labs. Unfortunately, in order to achieve its ultimate aim of neutering the FDA, the Goldwater Institute has made promises to patients that right-to-try can’t keep, enlisting them as their most effective foot soldiers to get such legislation passed. Unfortunately for patients, it’s been an incredibly effective tactic, because opposing right-to-try has successfully been framed as being heartless and taking away the last hope of desperate patients, all the while twirling one’s moustache, to the point where opposing right-to-try is viewed as being akin to being for burning the American flag while eviscerating puppies. No wonder politicians have, by and large, not even tried to stop right-to-try, other than Governor Jerry Brown.
One of the seemingly most compelling arguments in favor of right-to-try is that the FDA’s Expanded Access Program (a.k.a. Compassionate Use) is too cumbersome and doesn’t benefit very many people. I’ve addressed that trope before, pointing out that the FDA has make Expanded Access much easier and that it rarely rejects such applications. In comparison, I’ve thrown the question back at the Goldwater Institute: How many patients have benefited from right-to-try? Heck, I set an incredibly low bar and don’t even require that these patients have benefited. I ask how many patients have even received experimental treatments through a right-to-try law. Oddly enough, even though the Goldwater Institute claims it knows of 40 patients receiving experimental therapies through right-to-try (compare that to the 1200-1800 yearly who receive experimental therapeutics through Expanded Access), it never seems able to provide more details. Personally, I’ve called BS on its claims, but still periodically ask. I also note that at least one patient used right-to-try to access cancer quack Stanislaw Burzynski’s antineoplastons.
Frustrated by the professed lack of knowledge of patients who have benefited from right-to-try, even though it’s been well over two years since the first right-to-try law passed in Colorado, I decided to take matters into my own hands. Basically, the rationale for my approach was as follows. I did some searches on GoFundMe and Caring Bridge, two websites frequently used by patients to raise money for medical care. As ‘ve pointed out many times, if there were patients getting medications through right-to-try laws, given that most of these laws don’t require that drug companies provide right-to-try drugs for free, there would be some of those patients on GoFundMe and Caring Bridge asking for money. I must admit, the results were...disappointing.
First of all, a lot of what came up were comments about how various users of these two sites thought they were “right to try” to fund their efforts. In one example, a couple raising funds for in vitro fertilization stated that they thought they were “right to try.” In others, patients were raising funds for standard therapy but stating that they were advocating for right-to-try. This, of course, made finding true “hits” very difficult. Even so, basically I could find only two potential hits, and of those only one really seemed credible. For example, look at Patrick Sheridan, a man with pancreatic cancer:
I want to make something clear. This thing is only in me temporarily. It is not killing me. I am killing it. It picked the wrong person to mess with.
Traditional U.S. methods for for fighting cancer with chemo, radiation, and surgery aren't viable options for me at this time. My family and loved ones who have banded together to help me win this fight and I have already begun an aggressive mix of alternative and cutting edge therapies. I'll share more about the people and the therapies soon.
Unfortunately, many of the therapies that show the most promise (and in many countries, amazing results) are not covered by insurance. Some of these therapies may be available to me in the U.S. under Right to Try and Compassionate Use laws. Other, may only be available outside the U.S. We are pursuing them all. Most of you know how difficult it is for me to ask for help, but I'm asking for yours. A Go Fund Me page has been set up to help us cover some of the enormous costs it's going to take to beat this. Any help is greatly appreciated.
Unfortunately, Mr. Sheridan appears to be pursuing a mixture of pure quackery (like high dose vitamin C) along with treatments ranging from the questionable to possibly useful. Reading his sites, I could find no evidence that he accessed any experimental treatment through right-to-try.
So I moved on to the other potential hit, Gail Christopher, who has a GoFundMe page. Here is her story:
My cancer journey began September 30, 2008 after being diagnosed with Stage IV pancreatic neuroendocrine tumors, that had already spread to my spleen and liver. Fast forward 7 very tough years which have included a distal pancreatectomy, splenectomy, liver wedge resection with ablation, removal of gallbladder, another liver surgery to remove 2 large tumors, 2 liver embolizations and unfortunately breast cancer surgery with a partial mastectomy. During this time I was on 7 different chemotherapy treatments and chemo is no longer working for me as the tumors have gotten very large on my liver and are pressing against my lungs and have metastasized to my
Having this rare disease, means that a lot of already scarcely available drugs will not work for me, so my treatment options are extremely limited. This cancer has been resistant to standard chemotherapy, however, I continue treatment because I am determined to fight this terrible disease to ensure I am able to look at the beautiful sunrise from my bedroom window, snuggle with my precious dog Jack and be around to see my nephews and nieces get married.
Recently, I applied for a clinical trial at Memorial Sloan Kettering to participate in an exciting new therapy called PRRT, Peptide Receptor Radionuclide Therapy. Unfortunately, my hopes were dashed during the screening checks when they shared that my tumors are too large and what is termed "poorly differentiated."
I can’t help but note that Steve Jobs died of a pancreatic neuroendocrine tumor. These tumors tend to be indolent, slow-growing, but relentless. Thus, an eight year course after diagnosis is not at all unusual for this particular tumor type. Christopher notes:
PRRT will be a huge financial burden, and that is why I am reaching out for private funding. Any donation you could generously offer will be used to pay for my treatment which alone is approximately $38K, this does not include the additional expenses of traveling out of state and accomodations for multiple extended periods.
PRRT has been available as a life-saving treatment in Europe for 15 years but sadly is not FDA approved in the US. I will be undergoing 4 PRRT treatments at Excel Diagnostics in Houston, TX every 6-8 weeks. Excel is able to offer this treatment due to a "A Right to Try" act, which states terminal patients have a right to try any medicine to prolong their life whether FDA approved or not.
So what is PRRT? It’s a therapy that is simple in concept but not in execution. In the case of pancreatic neuroendocrine tumors, PRRT uses the fact that pancreatic neuroendocrine tumors express a lot of a protein, a receptor, that binds a molecule known as somatostatin. PRRT takes advantage of that by labeling somatostatin, although it uses another molecule that binds to the same receptor, octreotide. In PRRT, octreotide is combined with a therapeutic dose of radionuclides, with the most common ones being Yttrium 90 (Y-90) or Lutetium 177 (Lu-177). Basically, the idea is to target the cancer with a radioisotope by taking advantage of a specific molecule’s affinity for a receptor unique to the cancer. The radionuclide-conjugated molecule binds to the receptor on the tumor cell and kills it, thanks to the radiation from the radionuclide.
It turns out that Excel Diagnostics is based in Houston and uses Indium-111 or Lutetium-177 Octreotide therapy. There are several clinical trials on LRRT listed on PubMed, and Excel Diagnostics is listed as an investigator in several trials. Oddly enough, most of the trials have statuses listed as “Active, not recruiting” or “unknown.” In the meantime, I note that the Texas right-to-try law has a provision that is rare:
(c) If a manufacturer makes available an investigational drug, biological product, or device to an eligible patient under this subchapter,the manufacturer must provide the investigational drug, biological product, or device to the eligible patient without receiving compensation.
Yet, according to Gail Christopher, her treatment will cost $38,000 not counting travel and lodging expenses, of which she has raised $16,665 so far, according to the GoFundMe page. It is not at all clear to me whether these charges are for the whole treatment, or if Excel Diagnostics is providing its drug free of charge but that the ancillary medical expenses will be $38,000. I have to assume that the drug is being provided free of charge if Christopher is truly accessing radionuclide-labeled somatostatin analogue through right-to-try. Given that the company’s products are in active clinical trials as part of the process for FDA approval, one can’t help but wonder whether she’s actually receiving the drug under the FDA’s Expanded Access program, because I wonder whether Excel Diagnostics would be willing to take the sort of risk of administering the drug without the FDA’s blessing.
Whatever the case, at her latest update, which is from four months ago, Christopher states:
Well, I leave again for Houston in 2 weeks for PRRT treatment number 2. This treatment is WORKING!! When I last updated everyone my cancer specialist sent me to a bone surgeon to look at resecting cancer in my frontal bone on my skull. While waiting for that appointment with the bone specialist, the tumor disappeared!! I have to believe that it's working on other tumors in my body also. Thank God!!
I've applied for a small grant to assist me with these medical expenses and will hopefully hear good news on that in June. I'm still hoping to raise almost $25K to pay for treatments 2, 3 and 4...and I want to thank everyone who has donated so far. Some have gone over and beyond and have sent in a second donation for my second upcoming treatment.
I hope that Christopher continues to do well, but the lack of an update in four months and a seemingly dormant GoFundMe page is certainly cause for concern, at least to me.
Curious, I looked up what Excel Diagnostics is offering and found this:
Excel Diagnostics & Nuclear Oncology Center is pleased to announce that after careful review the Federal Drug Administration (FDA) has approved Investigational New Drug Clinical Trial by LU-177 Octreotate for patients with Neuroendocrine cancers. Excel Diagnostics & Nuclear Oncology Center is the first research facility in North America to receive authorization to initiate this much needed cancer therapy.
This therapy can be applied to the category of neuroendocrine tumors which include Carcinoid, Islet Cell Carcinoma of the Pancreas, Oat Cell Carcinoma of the Lung, Pheochromocytoma, and Iodine refractory or Medullary Thyroid Carcinoma”.
On ClinicalTrials.gov, I found two potential trials of PRRT in neuroendocrine tumors with which Excel Diagnostics is involved: 177Lutetium-DOTA-Octreotate Therapy in Somatostatin Receptor-Expressing Neuroendocrine Neoplasms and A Study Comparing Treatment With 177Lu-DOTA0-Tyr3-Octreotate to Octreotide LAR in Patients With Inoperable, Progressive, Somatostatin Receptor Positive Midgut Carcinoid Tumours (NETTER-1). According to the Society of Nuclear Medicine and Molecular Imaging’s patient guide, PRRT is palliative, not curative, and it can be effective at slowing the progression of advanced neuroendocrine tumors and in relieving symptoms. Evidence suggests that at least one form of PRRT is most effective against well- and moderately differentiated somatostatin receptor-expressing neuroendocrine tumors (which Christopher did not have, which is why she was not eligible for the existing clinical trial).
As I said before, however Christopher received PRRT, through Expanded Access or the Texas right-to-try law, I hope she is doing well, despite the lack of recent updates on her GoFundMe page. From what I can tell of her story, she certainly looked like a good candidate for expanded access, given her history, but obviously I can’t know the details of her case other than what she has chosen to reveal. I do know, having known a friend of a friend who suffered for several years with a neuroendocrine tumor and the symptoms it caused, that, even though such tumors are usually slow-growing, they are nonetheless a nasty set of tumors because of the symptoms they cause.
I’ve said before that, if lots of patients were benefiting from right-to-try or even if it is only the 40-60 patients that the Goldwater Institute claims to know about, their social media footprint would be visible and unmistakable, given that in most states right-to-try does not require the company manufacturing the experimental therapeutic to provide it free of charge. I would expect to see patients on sites like GoFundMe asking for money to help with the expense. I’d expect to see patients who succeed in accessing drugs under right-to-try to be trumpeting their fortune on Twitter, Facebook, and other social media. What is remarkable about right-to-try is how, even more than two years after the first such law passed, there is so little evidence of such activity. Gail Christopher is the only such patient I could find so far, and she appears to have been a good candidate for Expanded Access. I hope she is still alive and still having good results with PRRT.
The Goldwater Institute and other supporters promised in their pitch for right-to-try that it would provide access to experimental therapeutics to far more patients than the FDA’s Expanded Access Program, which it consistently derided as ineffective, too slow, too bureaucratic, and, at times, literally killing people. Two and a half years later, we see that for the lie that it was. That’s because the purpose of right-to-try was never to help patients (although that’s how it was sold and if it did that would be an ancillary benefit). It was to attack the FDA.
My condolences to the family.
Oh, geez. How did I not find that? I probably should have searched for her name + "obituary" but I didn't want to think that way and go too far down that path. I guess I was just hoping she was still alive.
Damn. So sad.
@Orac, alas, that was the search terms that I used, hoping to not find her name.
So sad to hear that. I was thinking this morning of my sister-in-law who died of cancer 6 years ago, so I truly sympathize with her family.
To be honest, I felt like a shitheel to even report her death, but, well, it had to be done.
Ask our intrepid leader on giving bad news. It has to be done.
I lack the practice on gently administering it.
Nor would I ever want to have to learn that practice.
I'm still realizing that I entirely refrained from reading DICOM imagery that I'm perfectly capable of reading of my wife's liver, after learning of her bilary cirrhosis.
I realized that late last evening, while on shift, where a DICOM viewer isn't available.
I returned home at 07:00, got distracted, it's nearly 13:00 local, still haven't reviewed the imagery.
Yeah, massive psychological block present. As I briefly reviewed the imagery for spinal issues, I suspect something is present that I'm not wanting to consciously review.
For, I know two people intimately well, to the lowest level and highest level, my wife and myself.
I've scheduled it for tomorrow to review the imagery, with intent to review the liver imagery. Complete with computer annoying me until I do, lest protective routines try to interfere.
I learned a long time ago how to overcome the natural things that one's mind puts barriers in front of.
No, it did have to be done. Actually, I screwed up in not finding that obituary when the way to find it was so obvious.
Eh, we all have an off day and miss something obvious.
I've wasted hours looking at something, but seeing what I was expecting, rather than a mistyped bit of code.
The "stories" of "right to try" web page ( http://righttotry.org/patient-stories/ ) by the Goldwater Institute is less than inspirational to me. I'm sorry if I sound like some hard-hearted bastard, but these "stories" (including a 5 y/o boy with DMD who even with current therapies should have another 15 years at least ahead of him but parents want to experiment on him anyhow) don't tell the whole story, and you're right, Orac--after 2+ years you think they'd have more to trumpet.
Resonating well with me, while I eagerly await my next echo of my abdominal aorta, which was 2.2 cm enlarged. Hit around 3 cm, things start to get interesting, hit 3.5 cm. Erm, Google Dead.
None of these stories actually used right-to-try, as far as I can tell. Some of them used FDA Expanded Access. One told of parents moving overseas to access a drug approved in another country but not the US. One told of a patient recently diagnosed with colon cancer with no mention of experimental therapeutics. Basically, this is some really thin gruel, given that it's been almost two and a half years since the first right-to-try law passed in Colorado.
I'll give them one thing, Orac.
No chance, vs slim chance.
Seriously, no chance or try, which would you try?
I'd honestly try a chance. Well, save when someone's trying to make serious money.
I'm also a realist.
An even money chance, I'd take that chance.
Not a good chance, but it's a chanceish.
popping up in various states like so much kudzu
But I'm sure you're not advocating stamping out kudzu since it shows promise as good medicine?
Alcoholism. Early research suggests that heavy drinkers who take kudzu extract for 7 days consume less beer when given a chance to drink. ...
Heart attack (myocardial infarction). Early research suggests that injecting puerarin, a chemical in kudzu, intravenously (by IV) along with usual treatment might help some people after a heart attack. Puerarin injection products are not available in North America.
I'd think the true intent of 'right to try' may not apply so much for pharma experimentals as it does for (herbal) treatments not available in the US.
@Gli, considering my own ethanol intake each week, perhaps a comparative survey should be in order.
I will admit to an ethanol intake that is quite literally legendary, which is obviously unhealthy. I'm astonished that my wife has a liver problem long before mine started to flag!
Although, now, I have a physician who's willing to prescribe for pain. I'm still adjusting.
Yes, dead serious. As in, 3.5+ liters of distilled spirits each and every week kind of serious.
*Finally*, pain is being addressed, but not the ethanol, I'm working that issue. Although, I can "cold turkey" it and not have anything beyond sleep disturbances, that's worrisome enough.
Or more simply, I drink more than quite a few alcoholics, habituated to it even, interrupt it, I have problems sleeping for a while.
Food is far more important. :)
Pain relief, less important than food, albeit, I'd eat a fair amount less.
Treat pain, zero high, I'd be the happiest creature on this planet. Fix the problem, I'd be in Xanadu.
Alas, insurance company regulations create problems that make me want to send a B-52 after...
My wife was just turned down for Osteoporosis injections, as there's an "oral substitute" that's far less effective.
She currently is healing from several vertebral fractures and has a pair of new fractures.
Pissed off is mild in my emotional chain right now, I'm thinking city erasure rage.
Sorry, but this just, quite literally came in.
Angry?! No, I want to vivisect someone. For anyone not knowing the term, it's dissecting an individual alive.
Which also makes me want to vomit.
I passed rage on the left, accelerated hard and found nothing.
I apologize, but, I'm at a loss, both for words and a plan.
Her spinal issues are severe, even life threatening at the beginning edge.
This crap ads in additional risk of terminal problems, due to delay of treatment.
Add in a flagging liver, secondary to an ignored gallstone condition until it became a massive problem, plus decades of APAP in every goddamned medicine, think that I'm enraged?!
I'd beat the dogcrap out of the Incredible Hulk right now.
While my mind whirls with zero ideas!
Welcome to Doctor's land. Bad news, no idea for good news, try for the best.
The reality of it is, I still am considering, hours later, how best to taste a bullet if I lose her after 34+ years.
Welcome to reality, not something you hear about, very, very real world.
I apologize, but news came in earlier today. I'm usually good at hiding such things, but, screw that.
For the third time in my life, I'm crying, the other two times was for the death of each parent.
So, please, out of some modicum of decency, take you politics out, then insert it up your ass sideways. Twice.
While I try to figure out how to move onward for our grandchildren.
I think I would gave gone with right to try when my wife was diagnosed with advanced MBC. However, after watching my sister in law struggle with a Phase I clinical trial (the drugs almost killed her; a lower dose has allowed her to return to work) as well as a friend who has survived PNT for over 10 years dealing with extremely harsh side effects of his regimen, RTT, in my opinion, would lead to premature death for most patients. Not to mention quality of life. I've certainly changed my stance on this issue.
I'm not surprised you had such a low signal-to-noise ratio looking for RTT cases on GoFundMe. Many quacks charge their patients/clients hefty sums of money for unproven treatments, too, and there are some (*cough* Stanislaw Burzynski *cough*) who deliberately try to blur the line between genuine experimental treatments and woo. Somebody has to support Stan in the lifestyle to which he has become accustomed.
Looking at the right-to-try website stories, it seems more like they are stories of people who right to try could maybe, possibly help. Maybe. Almost all of them could be equally assisted by increased FDA funding earmarked for hiring more people to handle expanded access requests so the process can be speeded up.
Add in a flagging liver, secondary to an ignored gallstone condition until it became a massive problem
P-5-P (a form of b6)
**alcohol doesn't damage the liver, malabsorption of choline caused by alcohol does --
Endurance athletes and people who drink a lot of alcohol may be at risk for choline deficiency and may benefit from choline supplements. Studies on a number of different populations have found that the average intake of choline was below the adequate intake...
When deprived of choline in their diets, 73% of postmenopausal women given a placebo developed liver or muscle damage
Actually, I suspect that my hyperthyroidism is responsible for my lack of liver injury. Choline isn't the only pathway that can potentially cause problem, fat processing as well can create problems.
Still, I dealt with bones and plumbing in emergency situations, for the most part. Endocrinology lacks both. ;)
fat processing as well can create problems.
That's what the inositol is for.
Choline and myo-inositol have been shown to prevent abnormal or excessive liver accumulation of cholesterol and triglycerides in choline and myoinositol deficient rats.
NAC is availible over the counter and is what they give in the hospital for acetaminophen toxicity -- The number one cause of acute liver failure in the US. Like so much denatured alcohol, it's in every damn thing.
Indeed, APAP is in so damned many things and has caused severe injuries in the US.
Considering how narrow its therapeutic window is and how hepatotoxic it is, honestly, I'm for banning the crap.
That said, my liver function is still excellent. Surprisingly so.
I'll share this little bit of good news I noticed in the morning's articles here since it's the latest cancer related blog.
There are still more new cases of leukemia each year than new cases of brain cancer, but it no longer accounts for the most deaths. That's due to advances in leukemia treatment over the past few decades and because leukemia is easier than brain cancer to treat, experts said.
"Some types of leukemia that a generation ago were almost universally fatal are now almost universally treatable," said Curtin, a statistician who worked on the report.
But the rate of death from brain cancer for children has held at about the same level for at least 15 years, according to the CDC report.
The trends are similar for adults, too, according to the American Cancer Society.
Leukemia is a type of cancer that affects the blood. That makes it easier for doctors to get to it and fight it with treatments like chemotherapy.
The brain is protected by a barrier which helps keeps many dangerous chemicals—including many cancer drugs—from getting to brain tissue or brain tumors. Surgery is difficult and sometimes impossible, depending on where the tumor is located in the brain. Radiation treatment can damage the development of a child's brain.
"There's survival, and then there's survival at a price," said Dr. Katherine Warren, an expert in pediatric brain tumor research at the National Cancer Institute.
Another factor is that scientists have only recently begun to understand that pediatric brain cancers may be biologically different from adult versions, and could require different approaches to treatment.
In 2014, the brain cancer death rate was about 0.7 per 100,000 children ages 1 through 19. The leukemia death rate was about 0.6. The overall pediatric cancer death rate dropped by about a fifth between 1999 and 2014, the CDC reported, helped by the reduction in leukemia deaths.
Progress is slow but it's real.
w/r/t my post (#10), this just popped up in my email today regarding accelerated FDA approval of the first medication approved to treat DMD (Duchenne Muscular Dystrophy) : http://www.managedhealthcareconnect.com/content/fda-grants-accelerated-…
Good news (and really argues strongly against RTT)!