One of my favorite shows right now is True Detective, an HBO show in which two cops pursue a serial killer over the course of over 17 years. Starring Woody Harrelson and Matthew McConaughey, it’s an amazingly creepy show, and McConaughey is amazing at playing his character, Rustin Cohle. I’m sad that the show will be ending tomorrow, but I really do want to see how it ends.

Unfortunately, as much as I like Matthew McConaughey as an actor, he is in part responsible for re-inspiring a movement that has the potential to do profound harm to patients and cancer research. That’s because his other big role over the last year has been in an Oscar-nominated movie, Dallas Buyers Club, where he plays Ron Woodroof, an early AIDS patient who in the 1980s smuggled unapproved pharmaceutical drugs into Texas when he thought he found them effective at alleviating his symptoms, distributing them to fellow sufferers by establishing the “Dallas Buyers Club” while battling the FDA. I haven’t seen the movie, and I really don’t want to, given that, from everything I’ve heard about it, it’s basically the story of a “brave maverick” who bucks the FDA, complete with all the tropes about indifferent bureaucrats who don’t care if these brave patients die. That might not be so bad if it weren’t also riddled with inaccuracies and misinterpretations of the AIDS crisis in the 1980s. Worse, the real Woodruff rejected the one truly promising drug at the time, AZT, as hopelessly toxic and instead smuggled drugs like Peptide T, which never panned out. Basically, what Woodruff appears to have smuggled as part of his activities for the “Dallas Buyers Club” was a mixture of useless supplements, experimental drugs that were never approved, and a handful of experimental drugs that showed promise. Meanwhile, the movie portrays the FDA as the implacable enemy of these sorts of activities, jackbooted thugs not unlike the stereotype promoted by “health freedom” quacks who don’t like the FDA preventing them from selling their quackery. As far as I can tell without actually seeing the movie, the overall message is a typical uplifting story of an underdog who fights the power and in doing so finds redemption.

Of course, Dallas Buyers Club is just a movie, no matter how good a movie it might be and no matter how much Matthew McConaughey might have deserved an Oscar for his performance. Unfortunately, it appears that legislators in several states seem to think that it should serve as a template for health policy. This public policy, which is extremely bad policy and is being considered and promoted in four different states, comes in the form of bills known as “right to try” bills. Basically, “right to try” bills, if passed into law, would grant terminally ill patients the right to have access to experimental therapies. According to these laws, the drugs need only have passed phase I trials. Now, remember, phase I trials do not demonstrate efficacy. They are only designed to test for safety and toxicity, determine the maximum tolerated dose, and provide an estimate for the dose to use in real clinical trials. The concept of “right to try” bills is highly popular, because if you don’t know a lot about medicine and how clinical trials work it sounds like a good idea What could be the possible harm, after all? A lot, it turns out, but I’ll get to that in a moment. First, let’s take a look at the rationale for Arizona’s “right to try” bill, which is being promoted by the Goldwater Institute:

Arizona legislators are poised to green-light legislation in committee Thursday that would pave the way for terminally ill patients to access experimental drugs not yet cleared for market. Known as the “Right to Try” Act, similar bills are currently being considered by lawmakers in Colorado, Louisiana and Missouri, and legislators in California and Massachusetts have expressed interest.

Designed by the Goldwater Institute, the Right to Try Act would enable terminally ill patients who have exhausted all of their available treatment options to access experimental drugs that have been deemed safe but whose efficacy has yet to be determined. Under the current system, even after an investigational drug has passed the Food and Drug Administration’s Phase I (the testing phase during which safety is established), it can take an additional six or more years for the drug to be approved for market–even if clinical trials are yielding promising results.

And while many patients facing terminal illness attempt to get into those clinical trials, the vast majority cannot, because they are too far along in their illnesses or because of other factors. 40% of cancer patients pursue admission into clinical trials, but only 3% succeed. Last year, more than 500,000 Americans died from cancer alone.

According to Christina Corieri, a health care policy analyst at the Goldwater Institute, the tragedy is that many of the drugs terminal patients can’t access today will be saving the lives of future patients just a few years from now.

“The sickest Americans don’t have the luxury of time to wait for these drugs to come to market through the traditional process,” said Corieri. “The Right to Try Act puts the decision about whether to try an experimental treatment back where it belongs: in the hands of patients and their doctors.”

This is, of course, partially true. There is always an inherent conflict between wanting to push for faster approval of drugs in order to treat patients who are dying now and the need for rigorous testing to assure safety, so that more patients aren’t harmed than helped. Patients and their families ask, “What’s the harm?” while advocates like Corieri sell their policy with the assumption that experimental drugs are highly likely to help these patients, or at least not so unlikely as not to be worth trying. Here’s the problem. Just because a drug has passed phase I trials does not mean that it is effective. It does, however, frequently mean that the drugs have significant side effects. Indeed, determining those adverse events is part of the entire reason that we do clinical trials in the first place.

“Right to try” laws versus science

The “right to try” bill currently being considered in Arizona follows the template for such bills that are metastasizing to other states, such as Colorado, so I’ll look at it first. The text of the bill is available online. The major provisions of the bill includes the following definition of an eligible patient:


The bill defines an “investigational” drug as a drug that’s passed phase I trials but is not yet FDA-approved. The bill doesn’t require manufacturers of an investigational drug to make it available, but basically allows them to do so if they so choose. Tellingly, the bill also doesn’t require that the manufacturer provide the drug for free, as it must to patients undergoing clinical trials in order to achieve FDA approval:



And, to top it all off, the bill prohibits the state medical board (or any other state regulatory board) from going after the license of any physician or health care practitioner who recommends and/or administers such investigational agents to patients while making any state official or employee who attempts to block access of an eligible investigational drug to an eligible terminally ill patient potentially guilty of a class 1 misdemeanor. If the bill passes both chambers in Arizona, it would next go to the voters in November. My guess is that if the bill makes it onto the ballot this fall, it will likely pass. I can picture the ads now. They’ll feature cute, terminally ill children and brave adults battling fatal diseases invoking their right to choose what goes into their body and begging voters to “give them a chance to live” and asking, “What’s the harm?” Against such images it will be hard for science-based medicine to prevail.

Currently, similar bills are wending their ways through the legislatures of multiple states, including Colorado (of course!), Missouri (where legislators recently listened to emotional testimony and the bill is sponsored by a legislator, Jim Neely, whose daughter suffers from stage IV colon cancer), and Louisiana, while states as disparate as Utah, Oklahoma, Massachusetts, and California are showing early interest in such laws. The bills under consideration are basically all based on the Goldwater Institute’s template (indeed, the Arizona bill is almost verbatim the same), which is based on this policy report available on the Goldwater Institute’s website, deceptively entitled Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment.

The report itself is loaded with emotionally charged language about the FDA and terminally ill patients and highly dubious statements. For example, it’s hard not to notice that every experimental drug is apparently “potentially life-saving,” at least the ones that made it through phase I trials. There’s so much loaded language, coupled with so many dubious assertions, that I can only hit the “high” points, such as they are. For instance, the Goldwater Institute bemoans the expansion of FDA authority in the 1960s by the Kefauver-Harris Amendments that required that the FDA not just require demonstration of safety but of efficacy as well before approving new drugs. This expansion of FDA power was in reaction to the thalidomide debacle, leading the Goldwater Institute to make the rather bizarre (OK, very bizarre) argument that because the issue with thalidomide was a safety problem, not an efficacy problem and because thalidomide was never approved in the US (mainly due to the FDA, let’s not forget), the expansion of FDA power in response to the thalidomide debacle was “unwarranted.”

Another highly dubious argument follows:

Phase I involves administering the investigational drug to a small group of 20 to 80 volunteers to test for toxicity and immediately observable side effects.30 The major emphasis of Phase I testing is safety. Over 60 percent of investigational drugs in Phase I testing are deemed safe enough to move on to Phase II.

Seriously? Phase I trials can be as few as 20 patients. That is not enough to determine safety, nor is it intended to. Phase I trials are designed primarily to identify major side effects and to use a process known as dose escalation to determine what is commonly referred to as the “maximum tolerated dose.” It is utterly impossible for such a small clinical trial to determine the safety of a drug, and even the Goldwater Institute inadvertently undermines its own argument. Note how the report says that over 60% of investigational drugs pass phase I testing and are determined to be “safe enough to move on to phase II.” That’s the standard: No unexpected major adverse events and a side effect profile that isn’t grossly more unsafe than the disease itself. Phase II and Phase III trials are needed to confirm safety. That’s why the premature diffusion of unapproved drugs has the potential to increase morbidity from adverse events and even hasten death. One example is amonifide for treating breast cancer. The drug made it through phase I trials, but serious life-threatening hematologic toxicity emerged during phase II trials.

Think of phase I trials as a screening test looking for the most obvious toxicities, with phase II and III studies confirming them. Indeed, even phase III trials can’t always adequately demonstrate that a drug is safe; it’s not uncommon for less common adverse effects not to show up until post-marketing surveillance, when much larger numbers of patients receive the drug. Moreover, only 5% of all cancer drugs that enter clinical testing are ultimately approved for patient use. Among drugs tested in phase II trials, only 30% go on to phase III.

The Goldwater Institute also doesn’t like the current expanded access programs, not because it doesn’t like expanded access programs, obviously. (After all, what are “right to try” laws other than much more liberal expanded access programs?) No, what the Goldwater Institute doesn’t like is the current system because of all those nasty regulations. Under current law, the Food and Drug Administration Modernization Act (FDAMA) of 1997, single patient INDs (we’re discussed these before in the context of Stanislaw Burzynski patients), which are basically compassionate use exemptions, can only be granted if:

  • The patient’s physician determines the patient has no comparable or satisfactory alternative therapy;
  • the FDA determines there is sufficient evidence of safety and effectiveness to support the use of the investigational drug;
  • the FDA determines that provision of the investigational drug will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval; and
  • the sponsor or clinical investigator submits information sufficient to satisfy the IND requirements.
  • the sponsor of the investigational drug must also be willing to supply the drug to the patient.

If all of these conditions are met, then the treating physician or drug sponsor submits an IND application, a proposed treatment plan, and a commitment to obtain informed consent from the patient and permission from the Institutional Review Board (IRB). No one denies that the process could be more streamlined. The report lists some of the burdens of the IND process and bemoans the FDA’s veto power over single patient INDs. What’s particularly disturbing about what the Goldwater Institute advocates, though, is its attack on the FDA’s requirement for a full IRB review of single patient IND applications as being a bad thing that keeps patients from accessing drugs. The language sounds as though it could have been written by Stanislaw Burzynski himself. Particularly Orwellian is the part of the report that demands that legislators must “act to protect patients.” I don’t know about you, but I’m not sure how, basically, stripping all restrictions on letting patients have access to drugs that have passed only phase I trials “protects patients,” but then I’m not a libertarian.

The Goldwater Institute justifies its proposal through still more dubious assertions. For instance, the report starts out with patient anecdotes in which patients with terminal cancers exhausted all conventional therapies and then applied for experimental therapies, which they either couldn’t get or had to wait too long to get. In each case, the stories are told in such a way as to suggest that “if only” these patients had had access to experimental drugs sooner they might have survived. This is, of course, nonsense. All these patients had stage IV cancer. Barring the incredibly unlikely possibility that an experimental drug is a miracle cure for advanced cancer even better than what the quacks claim in Tijuana for their treatments, these unfortunate patients would have died anyway. True, if the drug were effective, they might have gotten a few more weeks or months, but they would have died. Using such stories is nothing more than tugging at the reader’s heartstrings. It is not a good scientific argument.

Actually, there isn’t a single good scientific argument in the entire report. There’s a survey of orthopedic surgeons cited in which a majority of them have said they thought that the slow FDA approval process had hurt patients. This is well-nigh meaningless for a discussion like this one because (1) most orthopedic surgeons don’t take care of cancer patients (orthopedic oncology is a subspecialty, and, fortunately, orthopedic malignancies are rare) and (2) orthopedic surgeons are much bigger into medical devices than drugs. Moreover this survey is bundled in a table with other with results of other surveys that range from 1995 to 2007. Finally, the reference cited is from the Competitive Enterprise Institute, a free market “non-profit public policy organization dedicated to advancing the principles of limited government, free enterprise, and individual liberty.” I will admit that I’m a bit disappointed that between 68% and 73% of physicians would support a proposal to change FDA law so that unapproved drugs or medical devices could be made available to physicians as long as they carried a warning label about their unapproved status. Apparently, the physicians polled didn’t realize what a boon to pharmaceutical companies this would be! Why bother with those pesky and expensive phase II and phase III trials if you could market your unproven drugs or medical devices direct to doctors to use on patients after nothing more than a favorable phase I?

Unfortunately, the “right to try” movement is not limited just to the US. A reader made me aware of a similar bill being promoted in the UK right now. Known as the Medical Innovation Bill, this legislation was introduced into the House of Lords by Lord Maurice Saatchi, whose wife Josephine Hart died of cancer in 2011. While it’s easy to feel sympathy for Lord Saatchi, that doesn’t change how misguided the Medical Innovation Bill is. The bill is similar to “right to try” laws in that its ostensible purpose is to allow patients with terminal diseases to undergo treatments that are experimental or unorthodox. It’s different in the sort of tack it takes. First, Lord Saatchi seems obsessively focused on shielding doctors who use unconventional treatments from liability for doing so, even though malpractice suits are not nearly as big a concern for physicians in the UK as they are in the US. (One UK physician described them to me as being virtually “not an issue” for most docs in the UK.) That’s one difference.

Another difference is that Lord Saatchi’s bill doesn’t so much allow the use of experimental drugs outside of a clinical trial as to claim both to support “innovation” and to “expose the maverick.” Supporting the bill, Lord Saatchi claims that the “NHS predicts it will face a bill of up to £24 billion for legal cases in the coming years, and the figure is rising,” but doesn’t say how many years or how much of that is due to doctors being sued for using unapproved treatments for patients who are dying. My guess, but I don’t have the data, is that that fraction is very, very small. Even in the US it’s pretty rare for dying patients (or their families) to sue doctors who try unconventional therapies on them. The other aspect of the bill is this:

Doctors will not be protected by the Saatchi Bill unless they go through a rigorous and specific process to ensure that the attempted innovation is the right course of action for the patient.

As the draft Bill states in paragraph 2 (3a) a doctor wishing to try a new treatment – for example in the case where standard treatments aren’t working – must consult a body of senior and relevant medical experts and get their consent.

The doctor must also record their opinion, including and dissenting voices.

The decision of the panel of experts must then be presented to the patient – including any contrary opinions if there are any – and the patient must of course also agree to go through with the innovative treatment.

Finally, the note of the opinions must then be attached to the patient’s consent form as a permanent record.

Only then will the doctor be legally covered by the Bill.

The Bill imposes a much higher standard of consent than other health legislation.

If that’s truly the case, one wonders why the alternative medicine community has rallied behind this bill. If, as Lord Saatchi claims, this bill would tighten up considerably regulations over “brave maverick doctors,” why does one of the biggest organizations promoting quackery in the world, the Alliance for Natural Health, so strongly support it, the way it has supported, for example, Stanislaw Burzynski, arguably one of the most famous “brave maverick doctors” in the world? My guess is that it all hinges on the definition of “medical experts” in the bill. After all, for “integrative medicine,” who will be the “experts”? Other “integrative medicine” docs, of course! Meanwhile, in the comments we see stories like this from Dr W. Witvliet, who linked to this website on his name (I’ll let the website speak for itself but cite his comment):

my patient, was threatened with loss of his NHS contract just as I was if I refused to stop advising patients that arterial disease is reversible even though I had the photographic evidence from the retinal arteries to prove it.
The new science which I named CardioRetinometry not only eliminates

coronary heart disease and heart bypass operations but also reduces the incidence of cancer and diabetes.

It is so suppressed by the NHS that they were forced to get me struck off the register on a technical misconduct charge, for defending public health, despite my having 200 written testimonials and no lay complaint against me.

Quacks seem very enthusiastic about this bill. I wonder why. I also can’t help but note that supporters of Stanislaw Burzynski are enthusiastic supporters of “right to try” laws in the U.S. Indeed, Steve and Melissa Heil, whose daughter has a brain tumor and is being treated by Stanislaw Burzynski, were prominent among the patients and patients’ families testifying in favor of the Missouri “right to try” bill. Again, I wonder why. Parents like the Heils, I can understand. They honestly, albeit almost certainly mistakenly, believe that antineoplastons are the only thing keeping their daughter alive. Other than with family members who watched loved ones die and have been mistakenly led to believe that “right to try” bills might have saved their loved ones, I find it hard to be charitable with most supporters of “right to try” bills, as they appear to be mostly ideologically motivated, like the Goldwater Institute, or motivated by support for quackery, like the ANH.

I will likely have to look into Lord Saatchi’s bill in more detail, but it sure does smell, as one Twitter user described “right to try” laws, like a “Trojan duck,” a wonderful term that I will have to appropriate forthwith for legislation that appears reasonable on the surface but upon closer examination will promote the wider use of quackery. It is also based on the same magical thinking that underlies “right to try” laws.

The magical thinking behind “right to try” laws…

Given how bad an idea “right to try” laws are, here’s the funny thing: They almost certainly won’t do what they are designed to do. The reason is simple. They are nothing but state laws, and the FDA controls drug approval. Arizona can say as much as it wants that patients can get any investigational drug they want, but what the state of Arizona says is meaningless as long as the FDA says that you have to have a single-patient IND approved for patients to have access to an investigational agent outside the auspices of a clinical trial being undertaken to win approval for the drug. Because the FDA exercises a lot of its power through the federal government’s power to regulate interstate commerce, perhaps the only way I can imagine for a state to get around this would be if the pharmaceutical company is in the same state as the patient. If the company ships an investigational drug across state lines in violation of federal law, it’s screwed. Of course, even this might not be enough, because a lot of states have a “mini-FDA” act, which bans the use of drugs that haven’t been FDA-approved. Indeed, the only reason Stanislaw Burzynski managed to get away with administering antineoplastons to patients early in his career is because at the time Texas didn’t have a mini-FDA act, and Burzynski was very careful never to ship antineoplastons across state lines.

Even more pertinent, drug companies need their drugs to be FDA approved to recoup their investment in drug development and make a profit, because without FDA approval they can’t sell the drug. The Arizona “right to try” law requires that the “investigational drug” (1) have passed phase I trials and (2) still be in the clinical trial process. There’s only one reason for a drug to be still in clinical trials after phase I trials, and that’s because the drug company still wants FDA approval for its drug; i.e., that it hasn’t abandoned development. That means the investigational drug is still under FDA regulation. A single patient IND would still be required. Pharmaceutical companies providing drugs to patients in “right to try” states without proper INDs would be violating FDA regulations and would thus endanger their chances of ultimate FDA approval. In other words, these “right to try” laws are nothing but feel-good placebos. They have no real effect because reputable pharmaceutical companies will not cross the FDA by providing an investigational drug to patients without a proper IND. That means that the only companies that might take advantage of “right to try” laws would be disreputable companies like Stanislaw Burzynski’s institute. Like Burzynski, “right to try” laws offer nothing to cancer patients but false hope. In fact, I’m hard-pressed to think of anyone whom these laws would benefit other than Burzynski, which makes me wonder if his minions have anything to do with promoting them.

Indeed, making essentially untested drugs more widely available, as these “right to try” laws propose to do, will be far more likely to harm the individual patient than to help him. Advocates of these bills ask, “What’s the harm?” I’ll tell you: If there’s anything worse than dying of a terminal illness, it’s dying of a terminal illness and suffering unnecessary complications or pain for no benefit and having to pay for the medications causing the complications yourself. Remember, these laws allow the pharmaceutical companies to charge for their investigational medications. They don’t all necessarily require the company to provide the drug for free, as current law does in the case of single patient INDs. For example, as discussed before, the Arizona law doesn’t require drug companies to provide the investigational agent for free; the Colorado bill is being amended to have that requirement by its cosponsor Janak Joshi.

Worse, Dallas Buyers Club-style “right to try” laws risk undermining our entire clinical trial enterprise, which is a major part of the scientific basis for evidence-based medicine. After all, if early-stage experimental drugs were made widely available outside of clinical trials and taken for a wide variety of cancers, the signal-to-noise ratio would become very low. It would become very difficult to tell which drugs were working and for which cancers (and which were not), particularly since it would be reasonable to expect that such a policy would result in enrollments in clinical trials plummeting. And what would be the potential payoff for the shredding of patient protections proposed in these bills, even if the FDA didn’t put the kibosh on widely providing experimental drugs early in clinical testing (and, make no mistake, only having passed a phase I trial is early in clinical testing)? Very little at best, if even any at all. In reality, the likelihood of saving the lives of even a handful cancer patients by giving them access to early-stage investigational agents is quite low and hard to justify on a moral and practical basis, given the high likelihood of potential harm or premature death to so many other patients through the damage or destruction of a system that has been built up at such cost over several decades.

The entire justification for “right to try” laws also seems to rest on a misperception that there are “miracle drugs” out there that we will have to wait years for because the FDA is too slow to approve them. However, if there really were such a “miracle drug” that was amazingly effective compared to anything we have now, a large randomized phase III trial would not be necessary to detect its efficacy. Indeed, its efficacy could show up in even a small phase I trial or, at the latest, in phase II trials. There’d be examples of clinical trial subjects demonstrating amazing tumor shrinkage or even outright cures. In reality, we don’t see these things in phase I trials, because there are no miracle drugs, at least not yet (if there ever will be). Dallas Buyers Club-style “right to try” bills rest on a fantasy, and it’s a fantasy of false hope. Indeed, these bills serve an ideological purpose rooted in libertarian politics far more than they serve patients. It’s not coincidental that virtually the only sign of opposition to the Missouri “right to try” bill was from lobbyists representing the hospice community, who warned lawmakers that such legislation raises false hopes for patients when further treatment is likely futile.

The same is clearly true for Lord Saatchi’s Medical Innovation Bill, which seems to rest on an underlying assumption that there is a cornucopia of “unconventional” treatments out there waiting that could save thousands of patients with terminal cases of cancer and other diseases, if only doctors were allowed to use them more widely. Never mind that pesky scientific and clinical trial process that’s usually needed to demonstrate whether a treatment works or not. Given Lord Saatchi’s experience watching his wife die of cancer, one can understand his sentiments. However, I will point out that I watched my mother-in-law die what was undoubtedly an equally unpleasant death from metastatic breast cancer, and I know that no treatment, conventional or “unconventional,” could have saved her life. And, yes, I saw to it that she was evaluated by one of the best, if not the best, phase I oncologist in the world, an oncologist who is currently part of a Stand Up To Cancer Dream Team of researchers. My wife and I learned the pain and frustration of being informed that that she wasn’t eligible for any phase I trials. It is painful for me to admit this publicly, but I think it is important for you (and Lord Saatchi, if he sees this) to know that I have a personal understanding of the pain of watching a loved one die of cancer after having been told there’s nothing more that can be done for her. I still conclude that “right to try” bills and Lord Saatchi’s Medical Innovation Bill are profoundly misguided and based on magical thinking that I myself was tempted by.


These bills seem new, but they’re just the latest wrinkle on an old story. We’ve been down this road before, just without an Oscar-nominated movie to add glitz to the campaign. For example, the Abigail Alliance has been lobbying for similar “right to try” laws for a decade now. Libertarians love these bad ideas because, you know, the market cures all and people should be allowed to sell and buy pretty much anything they want. So does the press because of the human interest stories coupled with the “little guy” battling the FDA. Even though expanded access programs could definitely use some tweaking, never forget that the reason the laws we have exist is to protect the public against drugs that don’t work or are too toxic and, just as importantly, from companies that would sell such drugs with no evidence of efficacy or safety and from “investigators” like Stanislaw Burzynski.

Supporters often point to the example of Gleevec (imatinib) for chronic myelogenous leukemia (CML), and, indeed, Gleevec did show spectacular results in its first phase I trial. What advocates gloss over is that results like what were seen in three early stage phase I/II trials for Gleevec in CML are incredibly rare. In fact, before Gleevec, such results were virtually unprecedented:

Two years later, this chemical, which was called ST1571 and eventually renamed Gleevec, entered its first clinical trial: a small phase I trial involving just 31 patients. Remarkably, all 31 individuals experienced complete remission; in other words, their blood counts returned to normal. In some of these patients, there was also cytogenetic remission, meaning that the Philadelphia chromosome was no longer found in their blood cells. As Druker noted, “That was virtually unheard of in a phase I clinical trial. Usually in a phase I clinical trial, if you see a 20% response rate, that’s remarkable. We had a drug that was extremely well-tolerated and had a 100% response rate. It was absolutely incredible to see this unfold” (Taubes, 2003). Subsequent clinical trials produced results just as astonishing.

Also, Gleevec was an unusual drug from a mechanistic standpoint as well because CML depends upon a single mutation resulting in a single aberrant protein whose targeting can completely reverse the cancer:

In a way, Gleevec is an exceptional case, and the same success is not likely to be achieved with other cancers any time soon. Unlike most other cancers, which are caused by a multitude of complex interacting genetic and environmental factors and therefore have many targets, CML is caused by a single aberrant protein related to a consistent chromosomal translocation. Scientists were thus able to focus all of their efforts on this single target. Nonetheless, the Gleevec story is no less an excellent and, some would say, beautiful example of how knowledge of the biological functioning of a cell can lead to life-saving medical treatment.

If there’s a drug that’s shown as incredible a result in an early phase I trial as Gleevec, I am unaware of it. Again, that’s the point. Gleevecs of the world are rare, but Dallas Buyers Club “right to try” legislation rests on the assumption that they are much more common than they actually are and that thousands of patients with terminal cancer would be saved if only the FDA weren’t such prigs about insisting on evidence of efficacy and safety. It’s a fantasy, not reality.

Don’t forget: Dallas Buyers Club is just a movie, nothing more. It might be a really great movie (given its Oscar nominations and McConaughey’s winning Best Actor, it almost certainly is), but it’s not fact. It’s a fictionalized account of man’s battle with AIDS that is designed to entertain and uplift more than to teach about history and science. As such, it’s a terrible model for health policy. Widespread adoption of Dallas Buyers Club-inspired “right to try” laws is far more likely to harm rather than help patients with cancer and terminal diseases.



Posted by David Gorski

Dr. Gorski's full information can be found here, along with information for patients. David H. Gorski, MD, PhD, FACS is a surgical oncologist at the Barbara Ann Karmanos Cancer Institute specializing in breast cancer surgery, where he also serves as the American College of Surgeons Committee on Cancer Liaison Physician as well as an Associate Professor of Surgery and member of the faculty of the Graduate Program in Cancer Biology at Wayne State University. If you are a potential patient and found this page through a Google search, please check out Dr. Gorski's biographical information, disclaimers regarding his writings, and notice to patients here.