This is an impressive article and an impressive couple. From the site's about page:
My name is Eric Vallabh Minikel and I’m on a lifelong quest to develop a treatment or cure for human prion diseases. I originally trained as a city planner at M.I.T. and was working as a software engineer and data analyst in the transportation sector when, in December 2011, I got some bad news. My wife and the love of my life, Sonia Vallabh, tested positive for a mutation (PRNP D178N cis-129M) that causes genetic prion disease, and that had claimed her mother’s life one year earlier. Sonia was 27 at the time. The mean age of disease onset for her mutation is around 50, and the mutation is highly penetrant, meaning she is exceptionally likely to develop the disease unless a treatment or cure is found.
Sonia and I set out on a quest to re-train ourselves as scientists. We both started taking night classes, reading papers, calling up scientists, going to conferences. We left our old careers and found jobs in research labs, and eventually enrolled at Harvard Medical School, where we are now PhD students in biological and biomedical sciences.
One thing I've learned from smart family and friends about medical care is that you can sometimes improve outcomes quite a lot if you apply brains and effort. I have relatives who managed to convert their dad's brain cancer outcomes from a couple of months in palliative care (which the doctors were recommending) to five more mostly-good years, by finding better surgeons, signing him up for clinical trials, and getting to the cutting edge of the research and applying it. For example they switched him to a ketogenic diet back when this was just starting to show results for cancer. These guys are taking this approach to the extreme! I hope they get the result they're looking for.
> I have relatives who managed to convert their dad's brain cancer outcomes from a couple of months in palliative care (which the doctors were recommending) to five more mostly-good years
That's literally NOT how this works and this line of thought is VERY dangerous.
You have an N=1 sample size here.
Doctors use probability distributions when making prognosis and recommendations for treatment.
It's far more plausible that he was just an outlier.
By all means take care of your family members but you have NO idea that this behavior changed his outcome.
I would echo similar experiences. Healthcare is extremely bureaucratic, and there is an art to navigating it. While I have more than a sample size of one throughout my extended family, I can share my own experience when I had appendicitis right before my sister's wedding in my early 20's.
My initial action after I started having symptoms was to go to the ER. I waited five hours in the waiting room, and then decided to go home due to my pain and fever condition being intolerable. I then learned that I could have my primary care doctor call the ER to fast pass me from having to wait in feverish pain. Then, once my diagnosis was official, the resident on call recommended that I elect him to perform the surgery overnight. Since it was during my sister's wedding, my parents were able to come by after the rehearsal dinner, and insist that I wait until the morning for the doctor to perform the surgery. The doctor, who had a national reputation, came in at 6 AM, and with the confidence of Steph Curry lining up for a three point shot, changed the operating procedure due to specific circumstances. As a naive 20 year old, I would have just elected to do what the resident was recommending, and it probably would have been ok. Yet, I think I ended up better off with having family present, pressing back on the recommendation, and waiting for the doctor to arrive.
This was far more than just random chance. It's clear as day that quality of medical care makes a difference in outcomes, right? I have sent this discussion to the relatives in question so forgive me if I get the exact details wrong. But the median survival time for glioblastoma is fourteen months, and they were quite a while into that. Their original doctors were recommending palliative care (giving up on further medical treatment) with a couple of months expected survival.
My relatives live in a small city not known for its quality of medical care. They found the very best neurosurgeon in the country for glioblastoma and transferred him over with some difficulty. They learned that the initial surgeries were not done to a high quality or possibly even slightly botched - and the new surgeon was able to perform additional surgeries that were able to remove a significant amount of the tumour (and did follow-up surgeries later on). Of course this would extend a patient's lifespan!
The ketogenic diet now seems to be becoming a standard recommendation for glioblastoma treatment. There are TON of studies now. Here are a few more more. [1] [2] [3] Note the dates on these studies (2017-2018). Back when they looked at this, this was absolutely bleeding-edge stuff. Of course going to the cutting edge of the research, speaking to professors and using your brain to figure out what might help in your case will improve outcomes!
Regarding clinical trials, one can read up on / sign up for trials of treatments at different stages of the drug research pipeline. As the modelling spreadsheet in the link above shows, between 1-in-10 and 1-in-2 of these trials will result in FDA-approved treatments that will hit the market between 2 and 10 years from now. If your current expected survival time is in months, it is statistically worth implementing as many of these as possible, because at least one of them will probably work, and you don't have the time to wait for the FDA! You don't really care which one as, like you say, your sample size is N=1.
I shared this in the hope that some of the smart people on this forum would read this and know that this is something you may be able to do. We are engineers and scientists. We can read studies and think critically. We can spend a ton of time absorbing specialised information about the N=1 case that matters to us. Death comes for everyone and the above may not make a difference in many cases. But it turns out that sometimes it does! Don't treat medical care like a black box.
I get that people would try anything to help their loved ones. This in itself is admirable and goes to show the guy was truly loved.
We are beyond discussing the scientific value of your approach, so I'll try to communicate what you say conveys for a professional.
This is the typical american cancer story. The guy did not want to die, and his family and himself forced him through loads of invasive/experimental procedures because the success of treatment was viewed as a meritocratic achievement, which it is within the bounds of established treatment. I've seen this too many times to count and it saddens me greatly that already sick people get a substantially added dose of suffering on the basis of wishful thinking.
If the guy wanted to help OTHERS through his participation in trials, then that's really nice. His family, although very nice people I am sure, should not have had anything to do with his decision.
PS: the "best" surgeon wanting to operate on someone most of his colleagues would not touch with a 10-foot pole should have you start running.
I understand what you mean by the typical American cancer story. But I do get the sense you have pattern-matched this story for what I shared and may have missed the value of it.
We're not talking about quack treatments and unnecessary procedures here. Being intelligent about diving into the medical literature for a specific cancer, and consulting with academics and professionals in the field, in order to correctly identify treatments that may be effective for this case at the cutting-edge years before they enter the mainstream - this seems like an eminently correct thing to do. A simple Google search of "glioblastoma ketogenic diet" should show you the growth of research and good results now emerging in this area. They were ten years ahead of the curve on this.
And it's a fact that some medical professionals are better than others and this makes a quantifiable difference. There are bad doctors. And this is reflected in the studies. Doctors fall behind on recent developments, or have different levels of experience with specialised procedures (say, due to being in lower-population rural areas, as in this case), or just plain have worse manual dexterity. [1] [2] The original hospital did a bunch of damage, and I believe they even tried to sweep things under the rug in some way (I don't remember the exact details unfortunately).
What I am describing is not something the average person can do. Which is why it is not a mainstream recommendation on how to interface with the medical system. The average person will probably end up suckered into magic cancer beads, quack doctors, and then into the story you describe. This takes intelligence, effort and time. And risk-reward calculations. My relatives are quantifiably in the top one percent on measures of intelligence and education. As mentioned I chose to share this here specifically because many of us here fit the same kind of demographic.
> One thing I've learned from smart family and friends about medical care is that you can sometimes improve outcomes quite a lot if you apply brains and effort.
I'd be very careful with this sentiment though, because it is easy to turn it into the toxic notion that if a loved one dies from some disease (which is pretty inevitable), you didn't put enough brains or effort in it.
Unfortunately, every medical "support group" I have ever belonged to bent over so far backwards to not "blame the victim" that you absolutely couldn't talk about what worked because, inevitably, the sickest people with the worst war stories were the same ones who smoked, actively dismissed the idea that replacing carpeting with wood and tile floors might make a difference, etc. No, it absolutely wasn't socially acceptable to suggest that there was some correlation between the folks being proactive and having positive outcomes and the people shit-talking such approaches and their horrendous prognoses.
It creates an atmosphere where we can't realistically even try to investigate or develop a mental model for what actually works. So we just pretend that success or failure is some random number generator outcome. That always has me wanting to pointedly ask "So, why go to a doctor at all if it is basically totally random?" But I know how well that will go over, so I usually bite my tongue.
What's sure is that you do not understand the medical system at all. I hope this poor man's family did not force him through too much suffering.
FYI clinical trials, despite what some would like you to think, are absolutely not designed to help those participating in it.
FYI 2: "average doctors" have to follow guidelines like everyone else. The "exceptional doctors" proposing "advanced new cures" are either dishonest or failed to communicate properly with their patient. It's always astonishing to me when I realize people think that practionners have such freedom in deciding their treatment plan. This is not how it works.
>For example they switched him to a ketogenic diet back when this was just starting to show results for cancer
I'm curious as to how this would have an impact. Is there any research demonstrating that keto slows the development of cancer? Or hypotheses on why that would be the case?
Though it's incredibly dependent on the type of cancer, it's history, it's environment, and it's genome, it is related to the metabolic pathway that you shut off.
This is known as the Warberg Effect [0]. Essentially, for some reason, some cancers will start eating only via glycolysis and not via oxidative phosphorylation. Glycolysis requires glucose (sugars, like carbohydrates), so if you just stop eating foods that turn into glucose, those types of cancers will starve. Low sugar diets are ketogenic diets.
I'll be clear here, this is super speculative. Cancer isn't something like measles, each one is super specific to the organism and evolves over time. Keto diets may work for a while to harm cancers, but may also select for more 'hearty' cancers over time. It's not very well understood, even still.
Each cancer is unique to the organism. Not all cancers are death sentences. Nearly every sunburn you or I have had has been cancerous to some degree; the inflammation and reddening are your body fighting those cancers (among many other issues). Some cancers can be benign, some can be handled quite easily, some can be treated in other ways. Again, each is unique.
The data on Ketogenic diets and cancer are showing a lot of promise, but you should follow the advice of you oncologist/s. They have dedicated some portion of their lives to helping you out with your cancer. You do not want to end up in a situation where you have convinced yourself that kale is better than chemo. Again, each cancer is unique though.
Palliative care is an admission that chemo won't help and pretty much all options have been tried. At that point one can sit back and die, or try something.
Again, it depends on the situation at hand. Though ketogenic diets are showing promise, it depends on the cancer, as such diets may aggravate the cancer. One should follow the advice of the oncologist/s as well as research on their own.
If you google for studies, at this point you'll see an abundance of research on this with positive results, which apparently was just getting started when they dug into it and perhaps not well known to the average doctor. My memory plus a quick look into one [1] suggests it's because brain tumor cells largely feed on glucose, whereas normal brain cells can also metabolise ketone bodies for energy. So you're starving the cancer of resources.
[1] was a mouse study. Do you have any examples of human studies? I have this picture in my mind of someone showing up to a doctors office with a thumb drive of 30 PDFs of nonclinical studies they found through a Google search and demanding treatment x, diet y, and so on.
Another factor is that cancer cells act more aberrantly under stress. Fasting (not sure if this also applies to keto) creates resources competition by which cancer cells neither compete as effectively and make theme-selves more apparent to the body’s immune system. Lot’s of anecdotal information here with some evidence but as a hypothesis with reasonable with reasonable biological justification it makes enough sense to me that I’d be personally comfortable taking some of these risks for my self or my family.
I wouldn't expose my family to my own pet theories. I'd choose to listen to the oncologist. There is a downside to fasting as well. Removing the pleasure and normal lifestyle may isolate the patient from social interactions that involve food. Those social interactions could be really important to personal well being and staving off depression.
It's instances like these that I believe why the medical-pharma industrial complex isn't necessary, and likely more inefficient and soaking up more money - directing them towards profits, or other less efficiently than individuals who are passionate for a solution; yes, we need the institutions like Harvard Medical School to support these people - however the capitalism that then takes advantage of innovation through rent-seeking behaviour for cures is questionable.
My name is Eric Vallabh Minikel and I’m on a lifelong quest to develop a treatment or cure for human prion diseases. I originally trained as a city planner at M.I.T. and was working as a software engineer and data analyst in the transportation sector when, in December 2011, I got some bad news. My wife and the love of my life, Sonia Vallabh, tested positive for a mutation (PRNP D178N cis-129M) that causes genetic prion disease, and that had claimed her mother’s life one year earlier. Sonia was 27 at the time. The mean age of disease onset for her mutation is around 50, and the mutation is highly penetrant, meaning she is exceptionally likely to develop the disease unless a treatment or cure is found.
Sonia and I set out on a quest to re-train ourselves as scientists. We both started taking night classes, reading papers, calling up scientists, going to conferences. We left our old careers and found jobs in research labs, and eventually enrolled at Harvard Medical School, where we are now PhD students in biological and biomedical sciences.
One thing I've learned from smart family and friends about medical care is that you can sometimes improve outcomes quite a lot if you apply brains and effort. I have relatives who managed to convert their dad's brain cancer outcomes from a couple of months in palliative care (which the doctors were recommending) to five more mostly-good years, by finding better surgeons, signing him up for clinical trials, and getting to the cutting edge of the research and applying it. For example they switched him to a ketogenic diet back when this was just starting to show results for cancer. These guys are taking this approach to the extreme! I hope they get the result they're looking for.