Is Thalidomide the Next Big Cancer Drug?

By Victoria Forster, Newcastle University | October 20, 2014 1:18 pm

pills-question cancer drug

This article was originally published on The Conversation.

Innovative new drugs to treat cancer frequently make the headlines, either due to great success or controversy, as pharmaceutical companies get lambasted for selling the drugs at too high a price for state systems to afford.

But alongside this high-budget pharmaceutical research is a different tactic being quietly waged in the background: investigating old, inexpensive drugs, originally designed for a variety of maladies, to see whether they might be able to treat cancer – essentially, repurposing old for new.

Repurposing Drugs in Oncology (ReDO), the international organization aimed at promoting work in this area, defines repurposing as “the use of existing and well-characterized non-cancer drugs as new treatments for cancer.” ReDO believes that such drugs “may represent an untapped source of novel therapies.” Current candidates include diclofenac, an anti-inflammatory pain relief medication; clarithromycin, an antibiotic; and cimetidine, an antacid prescribed for stomach ulcers.

Cancers are increasingly being treated on the basis of the mutations that cause them, rather than where they are located. Seemingly distinct and unrelated cancers can arise due to the same genetic defect. Developing new drugs that target these mutations in a way that largely spares healthy cells is far from serendipitous and involves complex mathematical modelling and tens of thousands of laboratory hours to achieve even a prototype drug. All of this costs time and money.

Some researchers are shunning this process and instead turning to well-established drugs to improve cancer treatment. And it is an approach that is paying dividends.

Rehabilitating Thalidomide

Infamous thalidomide, regrettably used to treat morning sickness in the late 1950s, was catastrophic for developing fetuses and responsible for the birth of thousands of disabled babies. But despite this dark past, the potentially beneficial properties of thalidomide are being re-explored and it is currently one of the most talked about drugs in cancer therapy. More than 700 thalidomide clinical trials are currently registered in almost every cancer type imaginable and it is currently approved to treat a blood cancer called myeloma.

Although it isn’t fully understood how thalidomide works in cancer treatment, it is known to prevent the growth of new blood vessels, which can “starve” tumors and potentially cause them to shrink. There are other drugs which have similar properties, but many are expensive and come with a range of side effects. Thalidomide on the other hand is off-patent, cheap and relatively well evaluated.

Aspirin, available commercially for more than a century, has also been investigated not only for its potential use in cancer treatment, but also in prevention. Jack Cuzick, director of the center for epidemiology at Queen Mary, University of London, led a research project which earlier this year convincingly showed that daily aspirin reduces the risk of developing a variety of cancers and reduces the risk of dying from colon cancer by 40%. Cuzick practices what he preaches and has been taking aspirin daily for years, stating that doctors “absolutely should recommend daily aspirin to all those over 50.”

These kinds of discoveries are not always made using educated guesses, and scientists often test thousands of drugs just to see if anything might work. Researchers at St Jude’s Children’s Hospital in the US recently tested 1,300 existing medicines on medulloblastoma cells (a type of childhood brain cancer) in the lab. Two drugs typically used to treat breast cancer – gemcitabine and pemetrexed – were found to effectively kill these cells. A clinical trial has now been set up and researchers hope to improve a dismal cure rate of 40% in children with hard-to-treat medulloblastoma.

Grunt Work Already Done

Alan Worsely, head of science communication for Cancer Research UK, said: “The advantage of re-using an existing drug is that much of the early work to find the correct dose of a treatment and discover any potential side effects is already done, so researchers can get on with finding out whether it can help cancer patients.”

Increasingly cheaper and quicker genetic testing methods mean that researchers are also now able to analyze the DNA of patients who respond atypically to normally successful treatments to figure out why. Childhood acute lymphoblastic leukemia is one of the success stories of modern cancer treatment – 90% of children now survive for the long term. However, this still leaves 10% of patients who still die from the disease, mainly due to a relapse, which is very hard to treat.

Julie Irving, a reader in experimental hematology at Newcastle University recently published research showing that a drug called selumetinib, originally designed for tackling a faulty cell growth controlling pathway in lung and colon cancer, could be used to help children who relapse after treatment for acute lymphoblastic leukemia but who also have the same faulty pathway.

The development of a new cancer therapy can cost upwards of $1.6 billion dollars and take up to 15 years from initial development to reach patients. Mining the resource of thousands of “old” off-patent drugs is proving to be an important avenue in cancer treatment – and one which must be fully embraced by doctors and scientists.

The Conversation

Image by / Shutterstock


CATEGORIZED UNDER: Health & Medicine, Top Posts
  • Goldpenny’s Graffix

    I’m not saying don’t because if I had cancer, I’d grasp at straws to. But here Google “children of thalidomide ‘ images. I know this article touched on it but until you’ve seen the effects and until those effects have been expressly addressed this is insane. Especially when we’ve heard talk of an electromagnetic machine that just being in the room with it for hours a day days at a time can completely negate and cure cancer. Now I love better Living through chemistry, but the least you could do is understand the effects of a drug on your body before jumping into “the next big thing”. Which I am also a huge fan of.

    • Avatar1337

      What? That only applies to pregnant women.

      • BlindBraille

        Lol yeah, because all the offspring of men who took thalidomide that were born deformed were just a coincidence? Smh moron. Silly little kids shouldn’t be commenting on threads when they have no idea of the topic or research.

    • Vicky

      Hello Goldpenny’s Graffix. As Avatar1337 says below, the birth defects experienced by the children of pregnant women in previous decades were an incredibly sad consequence of thalidomide treatment. However, the safety profile of treating non-pregnant individuals in a range of cancers, has been reasonably well evaluated. That is not to say of course that it does not have side effects – it does, as with all chemotherapy agents. Just because a compound causes severe effects in unborn foetuses, does not mean that it will have similarly devastating effects on the rest of the body thankfully. As for the electromagnetic machine you speak of, I haven’t heard anything about it and I suspect it might be a lot of woo. Thanks for your comment.

      • Goldpenny’s Graffix

        “a lot of woo”. I like that. You haven’t seen it in mainstream news ergo it MUST be fake. Well cigarettes have been linked to causing lung cancer, yes? But on every cigarette packet there is a warning that specifically says it may be harmful to women and their fetus’ who smoke during pregnancy. I am usually amazed when I see this level of stupidity. I’m not saying this drug can’t help cancer patients, I’m saying that based on actual factual information (which you obviously have none of because “sad” doesn’t cover the amount of devastation this drugged caused on unborn fetus’) I’d rather not see a drug proven to do horrible things be used on cancer patients who do in fact still have the ability to procreate. That being said my main problem is the news article and the way it is presented. I’m not a conspiracy nut, or rather I just don’t care if there are conspiracies and cover ups, I’m. Sure there are but personally, I don’t care of the twin towers was an inside job. It happened, this bad enough. But the machine I speak of had clinical trials which many records have been lost or redacted. The actual scientist who presented the material in the 80s died of an overdoses of blood pressure medicine having no history of high or low blood pressure. That being said I don’t care is its a conspiracy, ‘a lot of woo” completely fake or real but untested. No my problem is that instead of pursuing valid cures for cancer like certain glycerol protine bonding with cancer cells and destroying them (also research that has been halted in the name of big pharma) that people are even considering a drug, like thalidomide with KNOWN and QUANTIFIED horrific results as an Avenue for cancer treatment. Which can only benefit it’s manufactures , distributors and stockholders. There are avenues not homeopathic silliness, but actual avenues of cancer treatment that haven’t been given proper due because it would be to harmful to big pharma’s bottom line and I simply express my irritation that sheep minded people will eat up any headline they see because it is mainstream. As a previous pharmaceutical rep. I can tell you that the amount of money we offer doctors and innuendo insinuated to push a certain drug is often over the top and unreasonable. If you have any common sense you’ll Google the fall and decline of paxil. But since you immediately shrugged off the idea of an electromagnetic machine who’s results were nothing if not miraculous you are obviously to lazy or to stupid to do any actual research. But for your brains sake please Google how cancer is treated in animals. For now I’m done with your idiocy it makes me want to vomit.

        • Vicky

          Dear Goldpenny’s Grafix,

          As a cancer research scientist, I spend my working life in the lab doing ‘actual research’ hence am neither lazy or stupid. I’m confused as to why you want me to ‘google’ things to find them out when there is so much rubbish surrounding issues such as cancer treatment on the internet. How do you decide which sources are reliable? Surely you must filter out most of what you ‘google’. Most of your post confuses me, but one thing that I can clarify is that because thalidomide is old, very old – ‘Big Pharma’ are unlikely to make much money from it. Also, cancer patients who cannot be successfully treated do not have the ability to procreate, as their disease progression will eventually result in their deaths. Seems an obvious point, but one you may have missed it would seem.

          • Goldpenny’s Graffix

            Methadone is older than thalidomide, and big pharma makes billions off its sale and distribution every year. It doesn’t matter if terminal cancer patients can’t procreate since few forms of cancer are genetic. And besides, that being the most idiotic thing I’ve ever read, “cancer patients can’t procreate”. If you’re a woman you’re at a predisposition for beast cancer and as far as I knew women are needed to procreate. I know you’re not a scientist and you don’t work in a lab, you couldn’t even pass a high school equivalency test, the idea that people like you would be the people trying to help people like my aunt who has beast cancer is a scary thought.

          • Vicky

            Dear Goldpenny’s
            I fear you aren’t understanding my comments correctly. I suggest you re-read them and amend your response. Also, all cancers are genetic in basis…as they are all caused by DNA mutations. I think you might be suggesting that few forms of cancer are ‘heritable’ meaning they are passed down from parents to children?

          • Goldpenny’s Graffix

            No I wasn’t suggesting that, because no forms of cancer can be inherited. You’re hereditary lineage can only put you at a genetic predisposition towards them. If you truly are a cancer researcher then you know about lineage and risk factors. Which side of who’s family has a history and such. But the fact of the matter is, big pharma makes more money off treatment than curing a disease. All forms of cancer can be treated, even if the treatment doesn’t work they still make money off it.

          • BlindBraille

            Wow you’re an idiot. First you say they can’t make money off a drug because it’s “old” which is obviously one of the most assinine comments on this thread. Then you imply cancer can be inherited, you then forget that you implied that, forgot you ever said it and then told someone they don’t understand what you were saying… You need to reread your own comments and then amend your brain.

    • rrocklin

      Scientifically illiterate.

      • BlindBraille

        Where as you’re just plain old illiterate.

  • Uncle Al

    Only one thalidomide enantiomer is teratogentic (e.g., phocomelia). However, resolved thalidomide epimerizes under physiological conditions. One therefore wonders whether locking the chiral center by substituting methyl or fluorine for hydrogen solves the problem without degrading the answer.

    Analogs lenalidomide and pomalidomide addressed potency and side effect issues but not epimerization.

  • Mike Menkhaus

    Thalidomide may be cheap to manufacturer, but it is anything but cheap at retail. Due to the ferocious potential liabilities associated with birth defects, a month of therapy runs over $10,000 per month. That was a year or so ago, before it was removed from retail pharmacies.

    Now it is only available In specialty pharmacies and I would expect that the retail cost has risen. – Mike, RPh

  • Mike Menkhaus

    Thalidomide may be cheap to manufacturer, but it is anything but cheap at retail. Due to the ferocious potential liabilities associated with birth defects, a month of therapy runs over $10,000 per month. That was a year or so ago, before it was removed from retail pharmacies.

    Now it is only available In specialty pharmacies and I would expect that the retail cost has risen. – Mike, RPh

  • Marriage Inn


  • Marriage Inn

    Velcade a great discovery

  • Episolve GI Natural_Antacid

    Drugs are drugs that have side effects and beneficial effects. We need to know when and where to use them and when to stop using them.


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