
Parkinson's patients 'walk and talk again' after receiving cancer drug in trial - AndrewDucker
http://www.independent.co.uk/life-style/health-and-families/health-news/parkinsons-patients-given-new-lease-of-life-after-receiving-cancer-drug-in-trial-a6699031.html
======
medymed
Translational medicine is bench-to-bedside. This was indeed translational
research.

This kind of medicine _seems_ more like bedside-to-bedside, as in giving the
cancer guy's drug to the Parkinson's guy in the next bed over and seeing what
happens.

But actually there's some animal model research behind it from the same people
in 2013, so they definitely did their mouse-homework and hopefully it will
continue to show promise in larger trials. From their 2013 paper's abstract:
"Here, we show that lentiviral expression of α-synuclein in the mouse SN leads
to Abl activation (phosphorylation) and lentiviral Abl expression increases
α-synuclein levels, in agreement with Abl activation in PD brains.
Administration of the tyrosine kinase inhibitor nilotinib decreases Abl
activity and ameliorates autophagic clearance of α-synuclein in transgenic and
lentiviral gene transfer models."
[http://hmg.oxfordjournals.org/content/early/2013/05/09/hmg.d...](http://hmg.oxfordjournals.org/content/early/2013/05/09/hmg.ddt192.abstract)

------
kosievdmerwe
It reminds me of using Zolpidem to wake up vegetative people:
[http://www.nytimes.com/2011/12/04/magazine/can-ambien-
wake-m...](http://www.nytimes.com/2011/12/04/magazine/can-ambien-wake-
minimally-conscious.html)

------
contingencies
MDMA is supposed to reliably relieve the symptoms of Parkinson's. While it's a
temporary relief, I find it disconcerting this is rarely mentioned in the
media.

[http://mdma.net/misc/ecstasyantipark.html](http://mdma.net/misc/ecstasyantipark.html)
\+
[http://www.ecstasy.org/info/parkinsons.html](http://www.ecstasy.org/info/parkinsons.html)
\+ [http://www.bluelight.org/vb/threads/722158-Parkinsons-
Diseas...](http://www.bluelight.org/vb/threads/722158-Parkinsons-Disease-and-
MDMA) \+ [https://news.agedcareguide.com.au/2012/06/05/parkinsons-
pati...](https://news.agedcareguide.com.au/2012/06/05/parkinsons-patients-in-
ecstasy/)

------
rsync
Propecia was "designed" as a prostate drug, but didn't do so well ... but
mystery unexpected side effect! Kind-of-sort-of baldness cure! So yeah, that's
what that drug is for.

Nilotinib was "designed" as a leukemia treatment, but ... didn't go so
well.[1] Oops, just kidding - it's a parkinsons/dementia treatment!

(also, one of the erectile dysfunction drugs, I forget which one, was also
"designed" for something similarly unrelated...)

The real headline here is that these people have _no fucking idea_ what they
are doing. They are throwing shit at the wall to see what sticks and then
informing the marketing department as to which side effects were the most
interesting.

This dovetails nicely with their other favorite activity: finding efficacious
natural compounds and then tweaking them just enough into a synthetic compound
that can be patented.

[1] "Interim results showed Tasigna is unlikely to demonstrate superiority
compared to Novartis's Glivec® (imatinib)*, the current standard of care in
this setting." (wikipedia)

~~~
eurokc98
While this comment is funny, it's also arrogant and incorrect. It's not fair
to indict folks trying to change lives based on what's needed to drive
pageviews, ie attention grabbing headlines.

30 seconds of research provides a pretty obvious connection, regardless of
your "no fucking clue what they're doing" accusation...

Nilotinib is a Tyrosine Kinase Inhibitor
[https://en.wikipedia.org/wiki/Nilotinib](https://en.wikipedia.org/wiki/Nilotinib)

Tyrosine kinase inhibitors regulate serotonin uptake in platelets
[http://www.ncbi.nlm.nih.gov/pubmed/7589171](http://www.ncbi.nlm.nih.gov/pubmed/7589171)

Tyrosine kinase in serotonin induced cerebral constriction
[http://www.ncbi.nlm.nih.gov/pubmed/9472895](http://www.ncbi.nlm.nih.gov/pubmed/9472895)

It's apparent that Nilotinib plays a role with Serotonin, which is of course
implicated in Parkisons and many other neurological disorders, but I guess we
are hung up on cute headlines and not the real story.
[http://phys.org/news/2010-06-serotonin-decades-old-
mystery-p...](http://phys.org/news/2010-06-serotonin-decades-old-mystery-
parkinson-disease.html)

[edit:sp]

~~~
epistasis
Also, drug repurposing is often a highly data-driven field. So it may not
involve predictions where there's a high degree of certainty, but data-driven
methods can shift a 1 in a million (or more) chance of success into a 1 in 3
or 1 in 5. Such wins are absolutely huge.

------
qap
Honestly, this article exemplifies what is really wrong with science these
days. First of all, this wasn't science, because it wasn't a controlled
experiment, or peer-reviewed, before being broadcast to the global media.

Parkinson's disease is known to be particularly responsive to the placebo
effect
([http://www.pdf.org/summer12_placebo](http://www.pdf.org/summer12_placebo)).
Since the "experioment" (big air quotes) had no controls, we simply do not
know whether chemotherapy helped them or not.

If we want people to trust the results of science when it comes to
vaccination, global warming, etc, we need to ensure that overblown claims are
not made in the name of science, as when they are debunked it will inevitably
eat away at the public's confidence in science as an institution and as a
source of justified claims.

------
jjudge
This was covered on NPR's Morning Edition too
[http://www.npr.org/sections/health-
shots/2015/10/17/44832391...](http://www.npr.org/sections/health-
shots/2015/10/17/448323916/can-a-cancer-drug-reverse-parkinsons-disease-and-
dementia)

------
fallingfrog
"people with Parkinson’s disease or a similar condition called “dementia with
Lewy bodies” were given small doses of nilotinib for a six-month period with
startling results." Does that mean that if Robin Williams had just held on for
another year, he could have been cured? Wow..

------
msie
This is great news!

 _But despite the apparent striking effects, doctors have cautioned against
great expectations for the drug at this stage as there was no control group or
placebo used in the study for comparison._

Heh, of course. But right now it could be used as an off-label treatment?

~~~
cpncrunch
>Heh, of course. But right now it could be used as an off-label treatment?

Why bother? There's still fuck all evidence that it works. (Bear in mind the
placebo effect is a large factor in all Parkinson's trials).

This is yet another example of overhyped crap science being posted on HN.

~~~
msie
For the desperate, why not? Of course weigh the side-effects against the
benefits. First try placebo, if it doesn't work then try the real thing.

~~~
cpncrunch
Because there's an almost infinite supply of unproven quacky treatments for
every illness. I could sell you one if you really want.

~~~
manarth
So balance it out.

Potential benefit? Improved health/physical condition.

Potential cost? 1. Financial cost of treatment. 2. Side-effects of treatment.

Number 1 is an important question: if it's a few dollars a month, it's on a
par with a placebo. If it's a hundred dollars a month, I would hope it behaves
better than a placebo (although a saline injection has a larger placebo effect
than a sugar pill, and a $40 bottle of wine tastes better than a $10 bottle of
wine, so there's a potential placebo multiplier just in the pricing). If it's
tens of thousands of dollars a month, I'd expect some pretty dramatic results.
So pricing is a factor, and I haven't seen that mentioned.

The second issue is side-effects, but this is a drug that's already passed
clinical trials, so hopefully these are known and recognised.

It'd be great to see this experiment rolled out to double-blind placebo-
controlled clinical trials, but until that happens, I'd still be willing to
give it a try.

~~~
cpncrunch
>It'd be great to see this experiment rolled out to double-blind placebo-
controlled clinical trials, but until that happens, I'd still be willing to
give it a try.

Google "liberation therapy" for an example. This bad science really is
damaging, as it costs everyone a lot of money, hooks in vulnerable patients,
and doesn't provide any benefit over the placebo effect.

The placebo effect can be strong, certainly, but if you're relying on that you
are much better investigating the psychological factors that underpins it,
rather than hoodwinking patients (whether deliberately or due to ignorance).

