Two people shaking hands
Thalidomide was a drug developed in the late 1950s in West Germany and promoted as a sedative and later an antiemetic (helps with nausea and vomiting) to women who were pregnant. The company that developed and promoted this new drug was named Chemie Grunenthal GmbH. By the mid-1950 almost 14 pharmaceutical companies were making and marketing thalidomide in 46 countries under various trade names such as Distaval, Tensival and Asmaval. The original German name was Kevadon as it came to the US market in the early 1960s. A doctor in Australia by the name of Willian McBride wrote a paper in the Lancet in 1961 early on describing some birth defects with this drug when given to pregnant women. However, this was much ignored and the industry that manufactured this drug for those purposed knew of the problem but concealed this fact from the public.
It was not until a drug company called Merrell which obtained a license to make and distribute this drug decided to market it in the USA and was attempting to get it FDA approved. They placed the application on the desk of Dr. Frances O. Kelsey a Canadian-born and US-trained physician and pharmacologist new to the FDA (actually this was the first drug approval application she received at her new job). Dr. Kelsey was undaunted and did not bend to the political and industrial pressures to pass this drug on to the American public. She researched thalidomide and was aware of the thousands of “thalidomide babies” born with horrific birth defects and refused not once but four times to pass the drug and give FDA approval. This did not sit well with the leadership of Merrell. The pressures the drug company place on her trying to discredit her as a grumpy and petty bureaucrat did not sway her. They finally withdrew their application, and the drug never came to market in the USA at that time. Only a few samples were ever dispensed by unknowing doctors and there were some 17 or so cases of thalidomide deformities in the USA due to this.
The reason Thalidomide effects in utero development is because of the nature of the drug. It was found that it had antiangiogenetic properties and that is the reason it had teratogenic effects. While babies developed in the womb the drug would affect the progression of limb development by disrupting blood flow. In the mid-2010s research on this drug found that it had inhibition effects on cereblon, a ubiquitin ligase and inhibited the process of angiogenesis (bringing blood supply to cells). Therefore, this drug was repurposed for use in cancer treatments. It appears to be helpful with the treatment of a type of Leprosy (a condition called erythema nodosum leprosum (ENL)) first used in Israel on a very sick patient with good results. In Brazil in the 1960s it became a standard therapy for ENL. Shortly afterward it was used as an anti-cancer therapy. So, this is a tale of how a drug developed seventy years ago can have a new life with new disorders. This drug was terrible for pregnant women and resulted in 10,000 deformities in children just in West Germany alone back in the day. However, avoiding its use in gravid women it can be used successfully in the treatment of a rather resistant condition such as ENL and in some cancers.
It is sad to report that once the cat was out of the bag about thalidomide and court cases against the manufacturer were levied the courts did not rule in favor of those harmed to their satisfaction and the leadership of the drug companies were never fully held accountable.
Dr. Kelsey went on to work for the FDA for some 40+ years and earn the respect and admiration of many at the agency and in the public. She almost single-handedly averted a major medical disaster in the USA by holding her ground and refusing to bend to the wishes of a well-funded pharmaceutical company (a true David and Goliath story). She lived to be 101 years old and is celebrated as an American hero in the medical and pharmacological fields. She was awarded one of the highest honors a civilian can obtain. In 1962 President John F. Kennedy awarded her the Federal Civilian Service Award for her service to the American public. She was also appointed to the Order of Canada just before her death.
This story is mirrored today by the likes of Dr. Pierre Kory who is standing up against the evils of the medical-industrial-complex in the fight against COVID-19. He has been before a Senate committee to discuss what has gone wrong with early and in-patient treatments for those stricken with COVID-19 and the negligence of our world (WHO) federal agencies (FDA, CDC, NIH) in identifying and fixing this problem. Once again as seen time and again he has been ridiculed, threatened, and attempts to defame him are made by some rather nefarious individuals. He will stand in the history books alongside luminaries such as Dr. Kelsey as dedicated patient advocates doing the right thing and making much self-sacrifice.
This is also a story of how what was considered a dangerous and banned drug can reemerge with a new and beneficial journey. There are just some precautions that need to be taken to avoid its toxicities. This also parallels the drive to discover new and emerging uses on existing drug in our long list of FDA-approved medications to be repurposed for new uses. They have longer track records, much more in safety data, and tend to be much more affordable to the public and cost-effective to the system than new novel drugs under patents that allow pricing to become exorbitant. Ivermectin has emerged as a drug with a fifty-year history as being rediscovered as much safer and more effective than Remdesivir with regards to COVID treatment. Much more funding should be put towards the examination of existing drugs and chemicals for use in medical conditions.