“Right to try”: A miserable failure thus far

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
heart.

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.