Fenbendazole vs Cancer
Related: Mebendazole vs cancer
26 April 2017
Oklahoma grandfather who claims a drug for
DOGS cured him of 'head-to-toe' cancer is tumor-free two years
after doctors gave him three months to live
By Natalie Rahhal
Joe Tippens, of Oklahoma was diagnosed with
late-stage small cell lung cancer in 2016
In January 2017, Joe Tippens was certain that he would die of
small cell lung cancer.
By January 2017, it had spread throughout his body
Joe's life expectancy was three months, but doctors enrolled
him in a clinical trial that they hoped could give him up to a
A veterinarian suggested he try the dog de-worming drug,
fenbendazole, which has shown cancer-fighting properties in
By May 2017, all cancer had disappeared from Joe's scans
Now, two years later, he is still cancer free and Oklahoma
medical researchers plan to look into Joe's case
WARNING: There have been no trials of fenbendazole for
treating cancer, there may be risks involved and the
medication is not recommended by doctors
But then a veterinarian suggested he try something unconventional,
to say the least: a drug for dogs.
The medication, fenbendazole, is an anti-worm compound used to
treat hookworms, roundworms and other gut parasites in animals,
In recent years, studies suggesting anti-worm drugs might have
cancer-fighting properties have been cropping up in a growing
number of journals.
It's far from a proven treatment, but with three months to live
and nothing to lose, Joe decided to take a chance on it.
Joe was diagnosed with small cell lung cancer in 2016, turning his
plans upside down, just two days before he was set to move to
Switzerland from Oklahoma.
He kept up a fighting attitude, but in January 2017, he got the
news that no one is prepared to hear.
The aggressive cancer was everywhere. It had spread to his liver,
pancreas, bladder, stomach, neck and bones.
His PET scan 'lit up like a Christmas tree,' he says on his
At that late stage of small cell lung cancer, Joe's odds of
survival were less than one percent, and the average life
expectancy was three months.
He had a trans-Atlantic move planned. He was expecting a grandson.
And now everything had to come to a halt.
Doctors at MD Anderson Cancer Center in Texas told him they
wouldn't give up, and would put him in a clinical trial that
wouldn't save Joe, but might give him a year or so to live.
He might get to meet his grandson.
'A year (or so) sounds a lot better than 3 months, so I said
"let's go for it,"' Joe writes.
Browsing an online forum for his alma mater, Oklahoma State
University, Joe saw a post that caught his eye that same month:
'If you have cancer or know someone who does, give me a shout.'
He did, and from the poster, a veterinarian, he learned that
scientists had accidentally discovered that a dog de-worming drug
seemed to combat many cancers in mice.
The same scientist that had conducted that research, as it
happened, had stage 4 brain cancer, and the same prognosis Joe had
been given, according to the vet.
She started popping the dog pills, and within six weeks, as the
vet told it, the scientist's cancer was gone.
Joe, who was 'a skeleton with skin hanging off of it' at half his
previous weight, he told KOCO 5 News, placed an order of
His new dosage of dog pills cost just $5 a week. His insurance
company had spent '$1.2 million on me with traditional means,' he
According to a study published in Nature, the drug compound
essentially starves cancer cells and kills them.
It also is, of course, already in production, cheaper, and,
according to cell studies and reports from people who have tried
it, not very toxic, especially compared to chemo and
That said, it was a risk.
Joe stayed in the clinical trial (he does not disclose what
therapy he received) added vitamin E, CBD, bioavailable curcumin
and, of course, the dog medicine.
He didn't mention the de-worming drug to his doctors.
In May, Joe's first grandchild, Luke, was born. Joe was there to
Two-and-a-half weeks later, he had another PET scan.
'Three months earlier...There was cancer in my body from head to
toe. And it was a terrifyingly dangerous metastasis that leaves
virtually 100% of its victims dead within 3 months. Here I was 3
months later and the PET scan was completely dark......void of any
light.....anywhere,' Joe writes.
He was dumbfounded. His oncologist was dumbfounded, according to
Joe writes that his doctor told him, 'We don't quite know what to
make of this as you are the only patient on the clinical trial
with this kind of response.'
In September 2017, Joe went for yet another scan, and was still
cancer free. At last he told his doctor what he'd been doing
outside the hospital.
There was no way at that point to prove that it was the de-worming
drug that vanished Joe's cancer, but his doctor did tell him that
he was an 'outlier' of the trial, Joe writes.
Joe's final scan was taken in January of 2018, and when he had a
follow-up appointment that April, he writes that his oncologist
kicked him out of the cancer center - because Joe had no cancer to
His results seem too-good-to-be true, but Joe claims to have
collected over 40 examples of similar success stories.
And his results were good enough to pique the interest of the
president of the Oklahoma Medical Research Foundation, Dr Stephen
'I'm usually skeptical, and I was and maybe still am about this
one, but there's interesting background on this' he told KOCO.
Now, Dr Prescott and Joe are working on a case study report,
according to KOCO.
Joe is careful to note that he's not a doctor, and is 'only one
man with limited resources.'
'I am not prescribing medicine and I am not qualified to give
advice on medical treatments.
BUT.....I am qualified to tell my story to as many people as
A Drug Made for Animals and Taken by Humans
to Treat Cancer: Fenbendazole
From anti-worms to anti-cancer
Previously, we discussed on this website the anti-worm drug
Mebendazole (Ref.), which based on a good amount of scientific and
clinical evidence, shows relevant anti cancer potential. Indeed,
there are case reports published in peer review papers showing
that Mebendazole can induce anti-cancer response in some
In the same article (Ref.) we explored the mechanism behind the
anticancer action of Mebendazole, and found out that Mebendazole
acts in a similar way as a group of chemotherapies such as Taxol.
Yet, in contrast to chemotherapies, due to the way Mebendazole
works, its toxicity is incomparably lower. Because of its good
safety profile, the drug is an over the counter drug in most of
I specifically like the anti-worms, anti-parasites, antibiotics,
antiviral drugs, as a pattern start to emerge suggesting that the
origin of cancer may be related to such a trigger (e.g. viruses,
parasites, etc.) in much more cases than we currently are aware
of. Multiple findings and observations, that I will discuss in a
different post, indicate that such triggers may initiate cancer
when they land in a “fertile ground”, represented by specific
genetic weaknesses combined with a compromised immune system (due
to e.g. stress, lifestyle, medication, etc.). This is why, I would
seriously consider using anti-worms, anti-parasites, antibiotics,
antiviral drugs as a part of more comprehensive treatment
approaches that could also include conventional therapies. As long
as the toxicity is low, it could make sense to cycle various drugs
of this type.
The anti-worm drug Fenbendazole has anti-cancer potential
In the same group of drugs as Mebendazole, a group called
benzimidazoles, there is another anti-worm drug called
Fenbendazole. Fenbendazole, is a drug used typically not for
humans like Mebendazole, but for animals (including fish, birds
and mammals). It is labelled to kill worms such as roundworms,
hookworms, whipworms, and some tapeworms. Fenbendazole is found
under various brand names such as Panacur or Safe-Guard.
I did came across this drug some years ago during my research, but
only recently I was motivated to look closely at it following
several e-mails from friends who shared with me the blog of a man
with Small Cell Lung Cancer, who successfully treated his cancer
with Fenbendazole (Ref.). On his website, Joe Tippens, not
only reports his experience but also anecdotally reports being in
contact with more patients experiencing benefits while using
Fenbendazole, including two cases of 4th stage Pancreatic Cancer,
Prostrate Cancer, Colorectal Cancer, Non-Small Cell Lung Cancer,
Melanoma, Colon Cancer. This anecdotal report would not be
enough to trigger me writing this post, if I would not be
convinced by the existing scientific evidence indicating the anti
cancer potential connected with many of the benzimidazoles drugs.
Therefore, I do believe that if Mebendazole could show relevant
anti-cancer effects in humans, which it did, Fenbendazole could do
it as well and hopefully even better.
In some diseases, it has been indeed shown that Fenbendazole can
be more effective than Mebendazole. For example, when tested
against Cryptococcus neoformans (an encapsulated fungal organism
that can cause disease such as meningoencephalitis in
immunocompromised hosts), it has been shown that Fenbendazole was
more active than Mebendazole or other drugs against this
opportunistic fungus (Ref.).
While there is more prior literature suggesting anti cancer
effectiveness related to Fenbendazole, the paper I found most
relevant to specifically cite here first is a paper that was just
published during 2018 in one of the most prestigious scientific
magazine, that is Nature, which adds a lot of weight to the
communicated message. This paper, entitled “Fenbendazole acts as a
moderate microtubule destabilizing agent and causes cancer cell
death by modulating multiple cellular pathways“, concludes the
“The results, in conjunction with our earlier data, suggest that
Fenbendazole is a new microtubule interfering agent that displays
anti-neoplastic activity and may be evaluated as a potential
therapeutic agent because of its effect on multiple cellular
pathways leading to effective elimination of cancer cells.”
In this paper, the authors cite potential anti cancer mechanisms
associated with Fenbendazole, including disruption of microtubule
function and proteasomal interference, but it was also associated
with blocking the glucose uptake by cancer cells (through reducing
the expression of Glut-4 transporter as well as hexokinase) and
thus starving cancer cells. This means Fenbendazole could also
work nicely in supporting chemotherapy and radiotherapy as well as
metabolic therapies. Because of the way it works (interacting with
a site on tubulin similar to colchicine but distinct from that of
Vinca alkaloids), Fenbendazole will not compete with Vinca
alkaloids (such as Taxol) but instead will add to the anti cancer
effect of these conventional treatments similar to other
Interestingly, when insulin stimulates glucose uptake in the
cells, glucose transporter isoform 4 (GLUT4) translocates from
intracellular vesicles to the plasma membrane ready to absorb
glucose. This movement of GLUT4 towards the plasma membrane takes
place via both rapid vibrations around a point and short linear
movements (generally less than 10 microm). The linear movement
seems to take place along microtubules. When disrupting the
microtubules with drugs such as Fenbendazole, GLUT4 movements are
disrupted as well strongly reducing insulin-stimulated glucose
While Fenbendazole could be relevant for many types of cancers (as
also suggested by the anecdotal reports listed above and by
literature on the anticancer effects of benzimidazoles drugs)
prior literature has so far indicated it’s anti cancer effects in
Non-small Cell Lung Cancer Cells (NSCLC)
Fenbendazole inhibits the cellular proteasome function dose- and
time-dependently and leads to accumulation of ubiquitylated
derivatives of various cellular proteins, including p53, which, in
turn, leads to apoptosis via the mitochondrial pathwaythe cells
first undergo G2/M arrest followed by apoptosis Fenbendazole
induced endoplasmic reticulum stress, reactive oxygen
species production, decreased mitochondrial membrane potential,
and cytochrome c release that eventually led to cancer cell death.
Prostate Cancer (Ref.) and taxane-resistant prostate
cancer cells Glioblastoma
The questions, is why I would consider using Fenbendazole, a drug
used for animals, when we already have Mebendazole made for use in
humans that is associated to similar anticancer mechanisms? There
are three major reasons for me to do that and consider trying
Fenbendazole as well:
First, as discussed above, in some diseases, Fenbendazole was more
effective than Mebendazole;
Second, it is known that this type of drugs is not very well
absorbed in the body and the absorption may differ from person to
person. Therefore switching between different drugs with similar
expected mechanisms may make sense as one of them may be better
absorbed in our specific case;
Third, there is a good chance that the underlying anti-cancer
mechanism is different for each of the drugs, even if the
scientific observations suggest similar mechanisms of action (we
should always remember that science represents not a complete
understanding of nature, but only steps towards a better
Fenbendazole is well tolerated in humans
Although a drug that is used for animals, according to a report
available at the European Medicine Agency “Fenbendazole seems to
be well tolerated in humans after oral exposure (single oral dose
up to 2,000 mg/per person; 500 mg/per person for 10 consecutive
What Type and how is Fenbendazole used
Taking Panacur C granules from Merck
There are people taking it for deworming and they seem to prefer
the Fenbendazole version that is meant to be used for fish (Ref.).
In this case, its is used in the range of 5mg/kg/day to
However, on his website, Joe Tippens, shows a picture of
Panacur C box from Merck, sold as Canine Dewormer, containing
Fenbendazole granules 22.2%. This means every gram of granules
contains 222mg of pure Fenbendazole.
Dose and treatment regime
In his treatment protocol, following a discussion with one
scientist from Merck animals who treated her brain cancer with
Fenbendazole, Joe Tippens uses 1g granules (containing 222mg pure
Fenbendazole) each day, and he is taking that 3 consecutive days.
He than stops taking Fenbendazole for the next 4 days. After that
he starts again, and he goes like this continuously during the
year. So the drug administration is 3 days ON and 4 days OFF.
If due to any reason taking Fenbendazole for ever it’s not an
option, I would at least consider taking it for 3 days, then
repeat a three day course at three weeks and again at three
months. This the minimum treatment schedule in my view and is
inline with the rule of 3’s used when treating whipworms
(Trichuris vulpis). The idea behind this treatment regime is that
some warms such as Whipworms take 3 months to mature from an egg
to an adult. If you kill adults at day 1, then three weeks later
there will be some immature adults which will have matured, but
you’ll still have eggs and larval worms present (Ref.).
Nevertheless, this is the minimum regime I would use but I would
probably better follow the treatment regime used by Joe Tippens
(continuous 3 days ON and 4 days OFF) for 2-3 months and check if
there is a response. If there is response, and tumors are
shrinking I would continue, if not I would stop. Joe Tippens said
he will take it for the rest of his life. He states there were no
side effects for him or for over 50 people he knows taking it.
Since it has been shown that 500 mg/per person for 10 consecutive
days is well tolerated in humans (Ref.), and since as discussed
below the absorption of Fenbendazole in humans is poor, it may
help to use from time to time a higher dose, such as 2g granules
(each gram containing 222 mg pure Fenbendazole) each day, which
would lead to a daily dose of 444 mg Fenbendazole.
Therefore, a suitable daily dose of Fenbendazole for longer term
use may be between 220 mg (the dose that was effective for Joe
Tippens) and 500mg (the dose shown tolerable in humans).
Since there are various produces for Fenbendazole under different
brands, in different forms (granules, solution, capsules) and for
different animals, it is best to check on the package to
understand how much active ingredient (Fenbendazole) is in one
gram (or ml) of the product we buy. And make sure that the daily
dose of active ingredient (Fenbendazole) is somewhere between 220
mg and 500 mg. Better to take it with or after food
Like many benzimidazoles, Fenbendazole is very poorly soluble in
aqueous systems, which are found in the gastrointestinal tract,
causing its low absorption to the bloodstream and thus very low
bioavailability. Maximal plasma concentration levels of
benzimidazoles in humans is known to be markedly increased if the
substance is used immediately after a meal (Ref.1, Ref.2).
As for the case of Mebendazole, I expect another way to increase
its absorption in the body is by combining it with Cimetidine
Add Vitamin E to possibly enable Fenbendazole effectiveness
During 2008, at the School of Medicine from Johns Hopkins
University, it has been found that Fenbendazole could affect the
growth of a human lymphoma cell line only when combined with
vitamins (Ref.). Supplemented vitamins included B, D, K, E,
Indeed, in his treatment regime, Joe Tippens also included
the following: Tocotrienol form of Vitamin E (800IU per day, 7
days a week); Bio-Available Curcumin (600mg per day, 7 days a
week), and CBD (25mg per day, 7 days a week) oil (Ref.).
The common vitamin that is both present in the supplement vitamins
in the article cited above and Joe’s treatment regime is Vitamin E
(800IU per day, 7 days a week). He uses a VITAMIN E (AS D-ALPHA
TOCOPHEROL) complex of 400IU called Perfect E, containing:
MIXED TOCOPHEROLS (~D-GAMMA TOCOPHEROL 400 mg; ~D-DELTA
TOCOPHEROL 9 mg; ~D-BETA TOCOPHEROL 7 mg) TOCOTRIENOLS (FROM PALM)
(~GAMMA TOCOTRIENOL 19 mg; ~DELTA TOCOTRIENOL 6 mg; ~ALPHA
TOCOTRIENOL 12 mg; ~BETA TOCOTRIENOL 1 mg)
Given that each capsule has 400UI, 2 capusles/day should match
Indeed, other studies have also shown that the combination of
Fenbendazole and Vitamin E can be effective against cancer.
Where to buy
Panacure C can be found all over the world at online shops. It can
be found in packages of 3 packets of 1g granules (or 222mg
Fenbendazole, for small dogs) or 3 packets of 2g granules (or
444mg Fenbendazole, for adult dogs). Fenbendazole should not be
confused with Flubendazole.
In vitro anti-tubulin effects of
mebendazole and fenbendazole on canine glioma cells.
Benzimidazole anthelmintics have reported anti-neoplastic effects
both in vitro and in vivo. The purpose of this study was to
evaluate the in vitro chemosensitivity of three canine glioma cell
lines to mebendazole and fenbendazole. The mean inhibitory
concentration (IC50 ) (±SD) obtained from performing the MTT
assay after treating J3T, G06-A, and SDT-3G cells for 72 h with
mebendazole were 0.030?±?0.003, 0.080?±?0.015 and 0.030?±?0.006 µM
respectively, while those for fenbendazole were 0.550 ?±?0.015,
1.530?±?0.159 and 0.690?±?0.095 µM; treatment of primary canine
fibroblasts for 72?h at IC50 showed no significant effect.
Immunofluorescence studies showed disruption of tubulin after
treatment. Mebendazole and fenbendazole are cytotoxic in canine
glioma cell lines in vitro and may be good candidates for
treatment of canine gliomas. Further in vivo studies are required.
Impairment of the Ubiquitin-Proteasome
Pathway by Methyl
N-(6-Phenylsulfanyl-1H-benzimidazol-2-yl)carbamate Leads to a
Potent Cytotoxic Effect in Tumor Cells
In recent years, there has been a great deal of interest in
proteasome inhibitors as a novel class of anticancer drugs. We
report that fenbendazole (FZ) (methyl
N-(6-phenylsulfanyl-1H-benzimidazol-2-yl)carbamate) exhibits a
potent growth-inhibitory activity against cancer cell lines but
not normal cells. We show here, using fluorogenic substrates, that
FZ treatment leads to the inhibition of proteasomal activity in
the cells. Succinyl-Leu-Leu-Val-Tyr-methylcoumarinamide (MCA),
t-butoxycarbonyl-Gln-Ala-Arg-7-amido-4-MCA fluorescent derivatives
were used to assess chymotrypsin-like, post-glutamyl
peptidyl-hydrolyzing, and trypsin-like protease activities,
respectively. Non-small cell lung cancer cells transiently
transfected with an expression plasmid encoding pd1EGFP and
treated with FZ showed an accumulation of the green fluorescent
protein in the cells due to an increase in its half-life. A number
of apoptosis regulatory proteins that are normally degraded by the
ubiquitin-proteasome pathway like cyclins, p53, and I?Ba were
found to be accumulated in FZ-treated cells. In addition, FZ
induced distinct ER stress-associated genes like GRP78, GADD153,
ATF3, IRE1a, and NOXA in these cells. Thus, treatment of human
NSCLC cells with fenbendazole induced endoplasmic reticulum
stress, reactive oxygen species production, decreased
mitochondrial membrane potential, and cytochrome c release that
eventually led to cancer cell death. This is the first report to
demonstrate the inhibition of proteasome function and induction of
endoplasmic reticulum stress/reactive oxygen species-dependent
apoptosis in human lung cancer cell lines by fenbendazole, which
may represent a new class of anticancer agents showing selective
toxicity against cancer cells.
Unexpected antitumorigenic effect of
fenbendazole when combined with supplementary vitamins.
Diet containing the anthelminthic fenbendazole is used often to
treat rodent pinworm infections because it is easy to use and has
few reported adverse effects on research. However, during
fenbendazole treatment at our institution, an established human
lymphoma xenograft model in C.B-17/Icr-prkdcscid/Crl (SCID) mice
failed to grow. Further investigation revealed that the
fenbendazole had been incorporated into a sterilizable diet
supplemented with additional vitamins to compensate for loss
during autoclaving, but the diet had not been autoclaved. To
assess the role of fenbendazole and supplementary vitamins on
tumor suppression, 20 vendor-supplied 4-wk-old SCID mice were
assigned to 4 treatment groups: standard diet, diet plus
fenbendazole, diet plus vitamins, and diet plus both vitamins and
fenbendazole. Diet treatment was initiated 2 wk before
subcutaneous flank implantation with 3 x 107 lymphoma cells. Tumor
size was measured by caliper at 4-d intervals until the largest
tumors reached a calculated volume of 1500 mm3. Neither diet
supplemented with vitamins alone nor fenbendazole alone caused
altered tumor growth as compared with that of controls. However,
the group supplemented with both vitamins and
fenbendazoleexhibited significant inhibition of tumor growth. The
mechanism for this synergy is unknown and deserves further
investigation. Fenbendazoleshould be used with caution during
tumor studies because it may interact with other treatments and
confound research results.
Novel benzimidazole (thio)carbamates with antiparasitic
activity and the synthesis thereof
The present invention is concerned with novel benzimidazole
(thio)carbamates with antiparasitic activity. The present
invention provides compounds of the following general formula:
Formula I, wherein X1 and X2 are O or S, wherein at least one of
X1 and X2 is O, Y1 and Y2 are O or S, wherein at least one of Y1
and Y2 is O, R1 is alkyl of 1-4 carbon atoms, R2, R3 and R4 are
independently of each other hydrogen, or a cation, R5 and R6 may
both independently be hydrogen or halogen or alkyl having from 1-8
carbon atoms, or -OR7, wherein R7 is alkyl having from 1-8 carbon
atoms, or -SR8, wherein R8 may be alkyl having from 1-8 carbon
atoms, or aryl, or -CO-R9, wherein R9 is alkyl having from 1-8
carbon atoms, cycloalkyl having from 3-6 carbon atoms, or R9 is
aryl, or -OSO2-Ar, wherein Ar is aryl, or -S(O)R10, wherein R10 is
alkyl having from 1-8 carbon atoms, or wherein R10 is aryl. The
compounds of the invention are highly soluble and stable in water.
Moreover, it has been found that the compounds according to the
invention are stable for over 8 hours at pH 5 and at pH 9, which
are the lower and upper pH limits at which compounds should be
stable for over 8 hours in order to be suitable for drinking water
application. The compounds of the present invention have excellent
antiparasitic, and especially anthelmintic activity in vivo, which
is comparable to the state of the art, water insoluble,
benzimidazole carbamates such as albendazole and fenbendazole.
Preparation method of fenbendazole
The invention discloses a preparation method of fenbendazole and
provides a brand-new synthesis route of the fenbendazole. The
fenbendazole is prepared from m-dichlorobenzene as a starting
material through the steps of nitrification, condensation,
amination, reduction and cyclization. The preparation method is
characterized in that the starting material m-chloroaniline in the
existing industrial route is changed to the cheap
m-dichlorobenzene; the existing reduction technology with sodium
sulfide dihydrate is changed to the clean and high-efficiency
reduction technology; and the new synthesis technology is concise
and simple, high in efficiency, slight in pollution, high in
quality, and suitable to industrial production.
UCSF, March 29, 2017
Deworming Pill May Be Effective in Treating
UCSF Researchers Use Computational Tools to Quickly Screen,
By Laura Kurtzman
Hepatocellular carcinoma (HCC), a cancer associated with
underlying liver disease and cirrhosis that often only becomes
symptomatic when it is very advanced, is the second leading cause
of cancer deaths around the world, and yet it has no effective
As with other conditions without treatments, the data that
scientists need to understand and treat the disease may be sitting
in plain view in databases that have barely been analyzed, says
Atul Butte, MD, PhD, director of the Institute for Computational
Health Sciences at UC San Francisco.
Bin Chen, PhD, a former postdoctoral scholar in Butte’s lab and
now a faculty member in Pediatrics in the Institute for
Computational Health Sciences, recently published a paper in
Gastroenterology about using data-mining computational tools to
identify a treatment for HCC.
Gene Expression and Drug Targets
Taking advantage of publicly available gene expression data, he
first derived a molecular disease signature for HCC – looking at
274 genes that are either up or down regulated in cancerous liver
tissues, but not in normal liver tissues.
Then, he looked for drugs that were known to target those genes
and found, to his surprise, that a close cousin of a deworming
pill, when used in combination with the standard care drug, was
highly effective at killing cancerous liver tissue that had been
engrafted into experimental mice.
“We found these disease genes were reversed after six weeks of
treatment in a patient-derived tissue in mouse model,” Chen said,
adding that the advantage of the approach he developed is that it
targets a host of genes at the same time, rather than simply
targeting a single mutation.
Chen said finding molecular signatures for diseases then looking
for drugs that work against those signatures is a promising way of
treating patients who may each have a different set of cancer
mutations and might not respond to drugs that just targeted them
one at a time.
“In this study, patients had similar gene expression profiles, but
not identical,” Chen said. “In most cases, cancers have many
mutations, and patients will relapse. Our approach might be used
to control the disease, to make it a chronic condition rather than
a lethal one.”
Head Start to a New Drug Candidate
Only a small number of drugs have been analyzed for their gene
expression profiles, and so it was somewhat lucky that Chen found
a hit with niclosamide. But even after discovering a match between
the drug and the disease, his team of UCSF and Stanford University
researchers was stymied. The drug, which was designed to kill
parasites, did not work well in the mouse model, because it was
It was only after Chen’s team stumbled upon a paper that described
its close cousin, NEN – an ethanolamine salt that is soluble in
human cells – that they made it work. When they combined NEN with
sorafenib, the standard of care for advanced HCC, they found that
the tumors stopped growing.
While NEN has been found safe to use against tapeworms in dogs and
cats, scientists do not yet know how it would affect humans, or
whether it would stop cancerous liver tissue from growing in
But Chen said it is a promising drug candidate that could be
developed at half or less than half the cost of a typical drug
because of the head start he got by using advanced computational
techniques to mine freely available data.
“Because our method is literally virtual, we can evaluate hundreds
of drug candidates very quickly,” Chen said. “We looked at more
than 1,000 drugs before discovering that the deworming pills were
effective. This is a very efficient way to do drug discovery.”