Back in the 1950s, a German drug company came up with a drug that was supposed to be ideal for helping pregnant women fight off the nausea of morning sickness.
Unfortunately, it turned out to be a disaster for babies and their mothers, one that resulted in the babies being born with horribly deformed arms and legs.
But eventually, through a series of strange twists and turns, the drug has now become an important platform for designing new ways to fight cancer.
Thalidomide as a cancer treatment? Yes. Keep reading. . .
Continued below. . .
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Big Pharma blunder
Contrary to what many people think, the drug thalidomide, was never approved for sale in the United States when it was introduced in 1957. For once, the FDA’s endless foot-dragging served a good purpose.
But in Europe and in other parts of the world, it was tragically discovered – too late – that the drug was linked to serious birth defects. Unfortunately, that was only after many pregnant women had taken it. It is estimated that before the sales of the drug were halted, up to 10,000 babies were born with serious birth defects because their mothers had taken this drug.
Thalidomide was banned before the damage could get even worse.
The disaster was an excellent lesson (still true today) of the dangers of taking a newly introduced drug. The possibility of unforeseen side effects – as happened with thalidomide – is a frightening reality.
That's why you should wait a considerable time to see if a new, highly touted drug works as advertised. Otherwise the consequences can be deadly.
What to do with the leftovers?
In the early 1960s, even after distribution of thalidomide was halted, the widespread use of the pharmaceutical meant there were a lot of leftover bottles in pharmacies around the world.
So in Israel, in 1964, Dr. Jacob Sheskin, searching for a way to treat people who were suffering from leprosy, gathered up a dusty bottle of thalidomide and began giving it to one of his desperately ill patients.
The leprosy victim saw an almost immediate dramatic improvement after only four doses. And that success set off a series of studies that eventually resulted in thalidomide’s being used to treat other victims of leprosy.
Soon after Dr. Sheskin's experience with the drug, thalidomide was put into use for other diseases that were similar to leprosy.
Fast forward 30 years. . .
Its success with leprosy put thalidomide back into production. But the drug’s potential link to cancer treatment wasn't evident until more than 30 years later.
Which takes us to 1998 and Beth Wolmer, a New York City lawyer who was frantically looking for a new treatment for her husband's multiple myeloma – a type of cancer that affects immune cells called plasma cells that are located in bone.
Mrs. Wolmer had heard that researchers were analyzing several drugs for their potential anti-cancer benefits.
One of the drugs under study was thalidomide. The researchers were conducting lab experiments to see if the drug could be useful for leukemia.
Mrs. Wolmer, whose husband was near death, decided it was worth trying thalidomide to see if it could help his multiple myeloma.
She must have been a very persuasive lawyer. She convinced the researchers to let doctors give thalidomide to her husband even though no one had ever taken it for cancer.
But the drug failed and didn't save his life. Frankly, I wouldn’t have done tried this. I regard it as a crazy gamble. There are plenty of proven alternative treatments. But obviously Mrs. Wolmer had a different take. And in the long run it turned out her intuition, or whatever it was, was sound.
Even though it didn't work for Mr. Wolmer, his wife's persistence put the idea into the researchers' heads that they should give it to other people with multiple myeloma.
As a result, tests at the University of Arkansas showed, in fact, that thalidomide helped improve one out of three people who had this deadly type of cancer. In the initial study, along with improvements in the conditions of two-thirds of the subjects, two out of the 84 patients who took thalidomide went from being hopelessly sick into what was apparently total remission.1
Which was a much better result than any other conventional therapy that had been tried out for multiple myeloma.
Treatment for cancer
After that study, the drug became a central part of the standard treatment for multiple myeloma – even though no one understood why the drug helps fight the disease.
But recently researchers are finally starting to understand how thalidomide affects cancer cells.
“What makes the story (of thalidomide) really bizarre, at least from a chemical biologist’s perspective, is that this is a compound that was made (by the original drug company) in the sense of ‘let’s put this in the body and maybe it’ll do something,’” says Joshiawa Paulk, who is with the Dana Farber Cancer Institute in Boston. “But it ends up being this huge platform for designing, and it’s actually changing the way that we think about how to target things in cancer.”2
Protein elimination
Paulk's work on thalidomide is one of a number of studies demonstrating particular actions of the drug that helps it act as an anti-cancer drug.
Research now shows that thalidomide affects the behavior of cereblon, a substance in human cells that acts as a type of garbage disposal, chewing up and eliminating unwanted proteins. Only, in the case of mutliple myeloma, when cereblon encounters thalidomide, it can target and destroy proteins that the cancer cells must have in order to multiply and reproduce.3
The studies at Dana Farber are now looking to combine thalidomide (and thalidomide derivatives) with substances that are effective at destroying other types of cancer other than multiple myeloma.4
An advantage of thalidomide, according to researchers, is that by allowing anti-cancer drugs to totally eliminate (not just incapacitate) certain proteins needed by tumor cells, it drastically reduces the possibility that the cancers can become drug resistant.
Consequently, tests of thalidomide are now focused on its use against fight lung cancer.
Going for the gold
So, despite the tragedy that accompanied thalidomide's introduction in the 1950s, it has helped many multiple myeloma victims during the past decade. From what I can learn, it’s pretty safe – certainly compared to chemotherapy drugs. Apparently the most dangerous side effect is the formation of blood clots in some people.
Meanwhile, the lesson for Big Pharma is that you should be careful when you accept ideas from lawyers.
Beth Wolmer, who is now Beth Jacobson, has decided that since using thalidomide to fight cancer was her idea, the drug company Calgene, which today sells versions of thalidomide for treating multiple myeloma, should pay her a hefty royalty.
Turns out that sales of the drugs are now a $2 billion a year business. And Jacobson is suing for upwards of $300 million.
Calgene, of course, doesn't want to share the money and is fighting her in court. On the face of it, I don’t think she has a case. But you never know what the courts will do.
Meanwhile, research into thalidomide goes on – Scientists at the Feinstein Institute for Medical Research in New York have found that the drug may also help treat sickle cell anemia, which affects millions of people, by improving the hemoglobin in the blood.5
Kindest regards,
Lee Euler,
Publisher
