Thalidomide: Return of an Infamous Pill

ARCHIVAL (HEARING ON PRESCRIPTION DRUG PRICING, C-SPAN, 5-8-19):
SENATOR RON WYDEN: Pharma executives, all of you that are here today are here because the way you’ve been doing business is unacceptable.

NARRATION: Drug company executives were recently called to testify about what has led to america’s sky-rocketing prescription drug costs.

ARCHIVAL (HEARING ON PRESCRIPTION DRUG PRICING, C-SPAN, 5-8-19):
SENATOR DEBBIE STABENOW: Let me just say, I think that you charge more here because you can.

NARRATION: One drug they focused on was Revlimid.

ARCHIVAL (HEARING ON PRESCRIPTION DRUG PRICING, C-SPAN, 5-8-19):
DAVID MITCHELL (PATIENTS FOR AFFORDABLE DRUGS, PRESIDENT & FOUNDER ): Today it sells for about $18,000 for a 21 day supply.

NARRATION: Revlimid is a derivative of a drug called thalidomide – long before it became a poster child for rising drug costs, thalidomide was a cautionary tale for drug safety.

That story begins in the 1960s with a warning from the president.

ARCHIVAL (PRESIDENTIAL NEWS CONFERENCE, 8-1-62):
PRESIDENT JOHN F. KENNEDY: Every doctor, every hospital, every nurse has been notified. Every woman in this country I think must be aware, that it’s most important that they check their medicine cabinet and that they do not take this drug.

NARRATION: Only two years earlier thalidomide — a sedative billed as the latest pharmaceutical marvel — had been set to arrive on American shores.

ARCHIVAL (CBC GLOBAL NEWS REPORT):
MORLEY SAFER: A hypnotic as the doctors called it, that was an answer to a prayer.

ROCK BRYNNER (CO-AUTHOR, “DARK REMEDY”): The hallmark, defining quality of thalidomide was its safety. So safe, that in Germany there was no prescription needed.

NARRATION: The German company that developed thalidomide, Chemi Grünenthal, claimed that even pregnant women could take it.

ROCK BRYNNER: The drug company had handed out samples of this drug all over the place, starting with employees of its own company. On Christmas Day in 1956, a baby girl was born in Germany without ears, and she was the daughter of an employee of the drug company Grünenthal.

NARRATION: No immediate connection was made to thalidomide – which soon sold almost as well as aspirin in some European countries.

ARCHIVAL (CBC BROKEN PROMISES):
DR. CLAUDE MURPHY (CANADIAN OBSTETRICIAN): We received it in quantities like 1000 pills. There was tremendous pressure all over the world to get this wonderful new drug on the market.

ROCK BRYNNER: They had two million tablets ready to go the moment the FDA approved the drug, which was almost a foregone conclusion, until one doctor came along and began working at the FDA.

ARCHIVAL (FDA INTERVIEW, 2-23-95):
DR. FRANCES OLDHAM KELSEY: It just so happens that my first application was for the drug thalidomide, I got this because I was new, and they thought I should have an easy one to start on.

NARRATION: But Dr. Kelsey was uneasy with what she saw as the lack of rigorous scientific studies.

ROCK BRYNNER: The best thing that could be said about thalidomide at the time was simply that you could not kill a rat, no matter how much thalidomide the rat ate.

NARRATION: With thalidomide being prescribed for morning sickness in other countries, Kelsey became particularly concerned with what effect it might have on a developing fetus. In June of 1961, an article appeared promoting its safety during late pregnancy.

ARCHIVAL (CBC BROKEN PROMISES):
ANCHOR: It was allegedly written by a Dr. Ray Nulsen. But, in fact, the article was written by the medical director of the drug company.

NARRATION: About six months later, long-ignored evidence became public in Germany linking thalidomide to a rash of birth defects.

Although hundreds of thousands of pre-market samples had been provided to American doctors, Dr. Kelsey’s stubborn delay of the drug’s approval for more than a year had prevented a similar scale of tragedy from unfolding in the United States.

ROCK BRYNNER: Dr. Kelsey was absolutely a unique hero in American history.

NARRATION: But thalidomide’s reach continued to be felt across the rest of the world, including in Trinidad and Tobago, where Giselle Cole was born.

GISELLE COLE (ADVOCATE FOR THALIDOMIDE VICTIMS’ RIGHTS): When I came along – I’m a first born – they were a young, married couple. I mean I was never unloved, or not wanted, or anything like that, but I would be foolish to think that it was easy for them. My disability is – the official term is “phocomelia,” coming from the Greek meaning “shorter arms,” or “flipper- like.” I think people always expect that I would’ve been angry. And I’m certainly not angry and never have been.

NARRATION: Long discussed but seldom implemented, major regulatory reforms were finally forced on the pharmaceutical industry following the thalidomide scandal.

ARCHIVAL (CBC, BROKEN PROMISES):
ANCHOR: For some time President Kennedy has tried to get congress to approve new controls but without much success – now with the thalidomide scare most of the opposition has melted.

ROCK BRYNNER: Largely, the same FDA guidelines that we live under today were created in the immediate wake.

NARRATION: These regulations were too late for thalidomide’s thousands of surviving victims across the world — who soon became the story.

ARCHIVAL (CBS NEWS, 12-26-75):
REPORTER: Phillipa Bradbourne is one example. Her mother rejected her.

ARCHIVAL (CBS NEWS, 12-14-72):
REPORTER: 10-year-old Carl Davies leads a relatively normal life for a boy without arms.

ARCHIVAL (CBC BROKEN PROMISES):
ANCHOR: Another young mother, her husband, his sister and her doctor are charged with the mercy killing of her deformed infant.

GISELLE COLE: I’m one of the lucky ones in that my parents were adamant that I was their daughter, and their daughter first before anything else. And I was treated as such. Many were put in homes, because they just didn’t know what to do, some families battled with doctors to have amputation of fingers and toes and what not to accommodate these prosthetics. Many families were broken irrevocably.

NARRATION: Instead of quickly settling, the drug companies dug in, with Grünenthal originally arguing that the children’s deformities were caused by everything from nuclear fallout to botched home abortions – anything but thalidomide.

GISELLE COLE: It was a very long and difficult process.

ARCHIVAL (NBC NEWS, 12-25-72):
REPORTER: British thalidomide children so far have not received any compensation from the rich company that made the drug which crippled them so brutally.

NARRATION: Most cases were eventually settled, but litigation continues, with some survivors saying the original settlements cannot cover the cost of their specialized care. Grünenthal didn’t apologize to its victims until 2012 – 50 years after the tragedy unfolded.

GISELLE COLE: They issued a statement saying that it has taken them the 50 years to come forward, to say anything, because they were shocked. They don’t have a right to be shocked. The shock doesn’t belong to them.

NARRATION: Despite all that thalidomide’s victims endured over the decades, they could long take solace in one simple fact.

ARCHIVAL (NBC NEWS, 12-25-72):
JOHN CHANCELLOR: Thalidomide is now banned everywhere.

ARCHIVAL (CBS NEWS, 7-30-73):
ANCHOR: The now banned thalidomide…

ARCHIVAL (CNN, 7-18-98):
REPORTER: The drug was banned in 1962.

GISELLE COLE: And I would have liked to have seen it never used again.

DR. ROBERT D’AMATO (BOSTON CHILDREN’S HOSPITAL): There’s a tremendous amount of luck in science. It’s almost like an Easter egg hunt.

NARRATION: In 1992, while conducting research into macular degeneration, ophthalmologist Robert D’Amato began his own hunt — this one to find a pill that might restrict blood vessel growth.

DR. ROBERT D’AMATO (BOSTON CHILDREN’S HOSPITAL): I started searching for drugs that caused some sort of damage to a fetus, a birth defect and at the top of this list was thalidomide.

NARRATION: After much trial and error, Dr. D’Amato finally had a breakthrough when he demonstrated that thalidomide could starve blood flow to cells — a discovery that held the potential for treating cancerous tumors.

DR. ROBERT D’AMATO: The people that really understood the results were excited, because we had a tool that we didn’t have before. But the knee-jerk reaction was this is a dangerous drug, no one will ever want to use it. There was thousands of victims that still remain. There had been a promise that thalidomide would never be developed again.

GISELLE COLE: We had to make a decision, what position we were going to take. And many would’ve thought and expected that we would’ve been screaming, “No, no, no, no, no!”

NARRATION: But following a surge of reports about promising studies, that’s not what happened.

ARCHIVAL (CBC GLOBAL NEWS, 3-10-97):
GISELLE COLE: We are nervous. We are of course frightened and dismayed. But we realize that this is not a perfect world.

ARCHIVAL (NBC NEWS, 9-23-97):
ROBERT HAGER: Thalidomide is back.

ARCHIVAL (CNN, 7-18-98):
ANCHOR: The Food and Drug Administration says thalidomide will be the most restricted drug ever distributed.

ARCHIVAL (CBS NEWS, 7-16-98):
ELIZABETH KALEDIN: Female patients taking the drug will even have to submit to pregnancy tests.

NARRATION: Since then, thalidomide has gone on to combat a surprising variety of diseases, from tuberculosis and Crohn’s disease, to multiple sclerosis and leprosy. And D’Amato’s hunch about the drug’s effect on cancerous tumors paid off in a groundbreaking treatment for certain types of the disease.

DR. ROBERT D’AMATO: Thalidomide and its derivatives have become the primary treatment for multiple myeloma.

GISELLE COLE: It’s a very fine line that I walk, and many of us walk, when we think about thalidomide in today’s world. Clearly, you can see what it has done to the thousands of us who are still on this Earth, but if it provides some kind of assistance, help, relief of suffering, then I cannot in good conscience be opposed.

NARRATION: Brynner, who saw thalidomide’s curative powers firsthand, after being prescribed it for a deadly skin disease, says that the drug’s circuitous history tells us a great deal about the accidental nature of drug discovery.

ROCK BRYNNER: It’s kind of surprising, I think, to most people to learn that drugs are, in fact, developed and then become, as they’re called, “a drug in search of a disease.”

NARRATION: But, for all its benefits, the scientific rehabilitation of thalidomide has come with a heavy cost.

ARCHIVAL (BBC NEWS, 7-23-13):
NEWS REPORT: We were told this could never happen again.

NARRATION: In Brazil, where the drug is used extensively to treat leprosy, researchers have documented that new thalidomide children have been born.

ARCHIVAL (BBC NEWS, 7-23-13):
NEWS REPORT: Alan is eight years old. He was damaged by thalidomide, which his mother took by accident when she was pregnant.

GISELLE COLE: Fifty years down the road, I would like to think that there’s no such thing as thalidomide, that we have created something and we have developed something that would allow us to bury thalidomide – literally. Destroy it. Get rid of it so that there are no further discussions, other than a note in history.

(END)