We’ve heard this story before: Raising loads of cash for unproven treatments

Being a cancer surgeon, I realize that my tendency is to view my blogging material through the prism of cancer, particularly breast cancer, my specialty. it’s easy to forget that there are diseases every bit as horrible, some arguably even more so than the worst cancer. When I think of such diseases, it’s not surprising that amyotropic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease after its most famous victim. It’s a progressive degenerative neurologic disease that affects the motor neurons, resulting in progressive muscle weakness throughout the body. Eventually, victims of this disease lose the ability to walk, use their arms, talk, and, near the end, breathe on their own. Most people with ALS die from respiratory failure within three to five years of diagnosis, with a median survival of around 39 months. While it’s true that Stephen Hawking, another famous victim of ALS, has lived with the disease more than 50 years, he is a very unusual case. only around 4% survive longer than ten years. If there’s a disease besides certain kinds of deadly cancer, like pancreatic or esophageal cancer, that scares the crap out of me, ALS is it.

Given the dire prognosis of ALS and the horrible way the disease relentlessly progresses, it just as unsurprising that its victims are desperate for anything that might slow the relentless deterioration of their muscle function caused by the disease. Unfortunately, that all too often means quackery. As of now, there’s only one drug (riluzole) that’s known to slow the progress of the disease and to prolong survival. Unfortunately, the effect is modest; so the search for better treatments goes on. While it goes on, there are victims of the disease like Lynne Grainger, who do what patients with with brain tumors who have become attracted to Stanislaw Burzynski do: Raise money to pursue unproven treatments. Unfortunately, that’s exactly what Lynne Grainger is doing. Diagnosed with ALS in 2012 and rapidly deteriorating, she is trying to take a “holistic” approach. Her website describes her undertaking craniosacral therapy. This article describes her quest for a cure:

In line with her desire to take a holistic approach, she consulted Dr. Ruan Jin Zhao of the Center for Traditional Chinese Medicine in Sarasota. Zhao began acupuncture treatments, but was frustrated by how little he could offer.

“As a doctor, a diagnosis like this is very painful,” says Zhao, who remembers the names of all seven patients with ALS he has treated, none still living. “And especially with her, because she is so sweet, so good, always so present, and has a great spirit for living. As soon as I met her I could feel that. For this kind of soul, I want to do everything I can.”

On the one hand, it’s tempting to feel a bit of schadenfreude for Ruan Jin Zhao. You mean there’s actually a disease that acupuncture can’t treat? I thought that acupuncture was good for everything that ails you, be it infertility, pain, menopausal symptoms, cancer, the need to quit smoking, and whatever. You mean it can’t treat ALS? Who knew? I mean, Zhao’s Center for Traditional Chinese Medicine claims to be able to treat arthritis, diabetes, cancers, asthma, hepatitis B and C, herpes, HPV, the flu, lupus, fibromyalgia, and many other diseases. Sadly, all Zhao can ofter is a kind word, because acupuncture is quackery. If that theatrical placebo brings Grainger a feeling of relief, I have a hard time being too critical.

Unfortunately, the search for a cure lead her to quackery:

Grainger also began alternative treatments — injections that had to be ordered from Switzerland, expensive supplements, aimed at cleansing her body of toxins and restoring her immune system, even a $5,000 machine (purchased by an ex-boyfriend) designed to tap into and heal the body’s “frequencies.”

I can’t be sure what that machine she got a hold of was, but my guess is that it was a Rife generator, which is based on the pseudoscience of radionics, which claims that diseases can be diagnosed and treated by tuning in on radio wave-like frequencies allegedly emitted by diseased organs and disease-causing agents. The ideas are, like most quack ideas, simple, persuasively compelling, and wrong. The claim is that all parts of the body emit electrical impulses with frequencies that vary with health and disease. Based on this, or so it was claimed, illnesses could be diagnosed by “tuning in” on a patient’s blood or handwriting and treated by feeding back the correct vibrations into the body with a similar device.

Vibrations. It had to be…vibrations.

As critical as I am of this sort of quackery, make no mistake, Grainger’s situation was even worse than just having ALS. She developed breast cancer. Although it was early stage and she didn’t require chemotherapy, she did opt for a double mastectomy with reconstruction. After her last reconstruction her speech and walking became more compromised and her fatigue worsened, which is not uncommon after major surgery in patients with ALS. Also, although the article doesn’t say this is the case for certain, it does state that Grainger believes that the reason she hasn’t been eligible for any clinical trials for ALS is because of her history of cancer.

It was at this point that, well, I’ll let the article tell it:

That left just one option: stem cell treatments available only outside the country.

But after spending than $30,000, she had used up all of her savings; she’d even moved in with Daudrumez for their first time in their relationship so she could rent out her own home for additional income.

So four months ago, she started a www.gofundme.com campaign aimed at raising the $45,000 she would need for a series of three treatments in Mexico, using stem cells grown from her own fat.

“People ask me, ‘Why are you doing this?’ ” she says. with halting, indistinct words. “But you have to navigate your own way. I know the body can heal, but sometimes, if it’s very far gone, it can take a lot of work.”

Here’s a good rule of thumb. If a clinic in Mexico, Central or South America, or Eastern Europe (among other places) is offering something that it calls “stem cell therapy” and is charging tens of thousands of dollars for it, chances are that what is being delivered contains neither stem cells nor effective therapy. Still, curious at what this therapy might be, I sought out Grainger’s GoFundMe page, where there’s a heartbreaking video of her asking for money with slurred speech juxtaposed with and older video of her bicycling. The procedure is described this way:

She is seeking stem cell treatment from a U.S. based lab, which does the actual procedure in Mexico. The whole thing costs $60,000. We did a fundraiser in March and April and now we are planning a cycling event. At this point, because of the constraints of time and organization, we can only do a “friendly ride” or “ride with friends” to benefit Lynne with donations. The thing is, we don’t have much time before summer and also the beginning of her treatment.

Here’s another hint. If the lab is in the US but the procedure is done in Mexico, you can be pretty sure it’s not scientifically supported. It turns out that the company is a company called CellTex in Houston, where the procedure was described by Grainger thusly:

Let the healing begin! I had my fat extracted from a local plastic surgeon (Dr. Scott Engel). It was shipped on ice (seriously) to CellTex in Houston. There the lab begins the process of growing/banking stem cells, which will be for my treatment. It’s a process, but I should get my first treatment in May. Still fundraising as each treatment is $13,500 plus travel, etc. Regenerative medicine comes with a price. So, watch for a music fundraising event April 30th or go online to donate.

I hadn’t heard of CellTex; so I checked out its web page. Basically, it’s a stem cell banking company that is very careful to state that it does not treat patients or provide any health care services. However, apparently, if you can find a willing surgeon to remove some fat from your abdomen, CellTex will isolate “stem cells” from that fat and then grow them up if you need them. To me this is basically semantics; in essence CellTex is providing a biological treatment, whether it is administering it or not. Thus, to me CellTex is a medical company. As for Dr. Engel, apparently he is a believer that stem cells from abdominal fat can be useful and that these stem cells “rejuvenate the skin” when fat grafting is used as a filler. Maybe they can be, but if they are no treatment has been validated for them yet. Certainly no treatment for ALS based on stem cells has as yet been validated in a clinical trial.

In any case, although CellTex is very careful to inoculate itself against charges of practicing medicine or using an unapproved biological to treat disease through its disclaimer on its website. It also basically admits that stem cell treatment is unproven for most claimed indications by emphasizing that their stem cells are safe, touting a study from 2011 performed in South Korea. Basically, it was an animal study in which these stem cells were injected into immunodeficient mice and then into eight patients with spinal cord injury. No specific toxicity was observed. CellTex also touts “dozens of other clinical trials” that have demonstrated safety and “hundreds” of pre-clinical animal studies. In other words, there are no studies suggesting efficacy—for anything. But, hey, CellTex is sponsoring clinical trials. It is very vague about what these trials will be testing, but, damn if it’s not sponsoring clinical trials. They’ve even been reviewed by an IRB! Too bad there isn’t any evidence of a clinical trial demonstrating that these mesenchymal stem cells can impact the course of ALS.

I don’t know for sure what quack clinic in Mexico has enticed this desperate woman to spend tens of thousands of dollars, but I rather suspect I know what clinic it is. Because I can’t be sure, however, I won’t name the company. I will, however, point out just how incredibly dubious CellTex is. The reason that CellTex is sending its patients to Mexico to receive stem cells is simple: It got busted by the FDA for administering stem cells to patients on US soil at its facility in Houston when a patient named Debbie Bertrand blogged about her experiences during a five-injection treatment with cells prepared at CellTex. The FDA was not amused. It also found significant deviations from current good manufacturing practice during an inspection in 2012. Nor were legitimate stem cell researchers amused:

Stem-cell scientists contacted by Nature are concerned by Celltex’s activity. “Because we know so little about mesenchymal stem cells and whether they are indeed effective for treating any condition, I’d be very wary of how they are being infused into patients, and certainly concerned if practitioners are charging patients for medical procedures that haven’t been proven to work and could in fact be harmful,” says George Daley, director of the Stem Cell Transplantation Program at Harvard Medical School in Boston, Massachusetts, who helped the ISSCR to draft its guidelines. In the opinion of Arnold Kriegstein, director of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at the University of California, San Francisco, “the very nature of Celltex’s business plan, which involves charging patients considerable fees for so-called treatments for diseases and disorders for which there is no good clinical evidence of efficacy, crosses an ethical line”.

So, I would argue, is charging a dying woman desperate to live longer tens of thousands of dollars for a procedure that has not been demonstrated to have any efficacy against her disease. It gets even worse, though. CellTex hired bioethicist Glenn McGee to try to show the world that it was abiding by ethical standards in human research. He resigned after only three months on the job. Of course, one of the company’s founders trots out the same old excuse used by unscientific practitioners the world over for why he can’t do clinical trials:

Lotfi says that most of his patients claim to get better after the treatment, but he admits that there is no scientific evidence that the cells are effective. “The scientific mind is not convinced by anecdotal evidence,” he acknowledges. “You need a controlled, double-blind study. But for many treatments, that’s not possible. It would take years, and some patients don’t have years.”

“The worst-case scenario is that it won’t work,” he adds. “But it could be a panacea, from cosmetics to cancer.” He says that Celltex is conducting a trial in which patients “will be their own control”. “If you can compare before and after and show improvement, there’s no need for a placebo,” he explains. “How can you charge people, and then give them a placebo?”

I am still flabbergasted that anyone could have said something revealing such ignorance of placebo effects, the clinical trial process, and why doing placebo-controlled trials is important for many outcomes with a subjective component. While it is possible to do trials in which patients serve as their own control, it’s not really an appropriate trial design, however, to demonstrate conclusively the efficacy of a treatment intervention like stem cell therapy. Moreover, what Lotfi said about there not being time for a trial is utter nonsense, given the near-miraculous improvements claimed. If the improvement is that dramatic, it wouldn’t take long to do a clinical trial at all.

So how is CellTex getting away with it? Come on? Do you have to ask? It’s Texas, home of some of the laxest (read: close to nonexistent) regulation of medicine in the country, home to Stanislaw Burzynski. Moreover, CellTex appears to be connected, counting Governor Rick Perry as one of its enthusiastic clients.

Which brings us back to Lynne Grainger. To me, she’s no different than any patient of Stanislaw Burzynski I’ve discussed or any Burzynski patient whose story has been documented at The Other Burzynski Patient Group, and the promises made, either by CellTex or whatever quack clinic in Tijuana is using the stem cells that CellTex banks, are no different than Burzynski’s claims. No, I take that back. Burzynski has actually done some clinical trials. True, he’s never published a completed trial and what he has published is unimpressive, but he’s one up on CellTex in that department.

My heart goes out to Lynne Grainger every bit as much as it has gone out to every Burzynski patient I’ve ever blogged about. She is a woman in the prime of her life facing a fatal illness and a horrible death. It’s just depressing how there always seems to be someone willing to take advantage of the desperation of people like her who only want to live.