In the 1960s, a drug called Thalidomide was given to pregnant women around the world as a treatment for morning sickness. But as a result of this treatment, more than 10,000 babies were born with severe defects such as malformation of the limbs, eyes, ears, face, genitals, and cardiovascular system. Many babies died within the first year of life and there were reports of increased miscarriage rates. Survivors also suffered from dysfunction of internal organs and nerve damage.
Thalidomide was licensed as a safe treatment in more than 46 countries for only a few years but the ‘Thalidomide Disaster’ was deemed the worst medical atrocity of the 20th century. For many years, survivors have lived with severe physical disabilities, discrimination, and early onset of old-age related disorders, with many families sacrificing normal lives to provide necessary care.
Identifying the cause
Despite the known risks, Thalidomide is still prescribed successfully today for conditions including multiple myeloma and complications of leprosy. Careful preventative schemes are in operation in most countries to prevent more children being born with Thalidomide damage. However, sadly, a new generation of Thalidomide survivors has been seen in Brazil, where the drug is used to treat complications of leprosy. To gain a better understanding of the risks and the mechanisms of the drug, and to determine if new forms of Thalidomide can be made which retain the clinical benefit without causing birth defects, Thalidomide has been researched extensively by academics at the University of Aberdeen.
Professor Neil Vargesson, Chair in Developmental Biology, leads a team using chicken and zebrafish embryos to explore how and when the drug disrupts the formation of limbs and the range of other forms of damage that are caused by Thalidomide.
The team’s research has shown that chicken and zebrafish embryos that are exposed to Thalidomide suffer the loss of newly formed and forming blood vessels, which then results in a lack of further limb growth and prevents limbs from fully forming.
Understanding limb malformation
The research also demonstrated that variation and severity of malformation could arise depending on the timing of Thalidomide exposure.
By being able to identify the time-sensitive nature of the effects of Thalidomide, the team’s discovery has become a cornerstone in the understanding of the drug.
Thalidomide caused what is arguably the single biggest disaster in medical history, but when used safely it can be used to effectively treat a number of diseases such as leprosy and multiple myeloma. Our initial research uncovered how and when the drug can cause malformation and is helping to enhance understanding of Thalidomide use around the world.
Our most recent research has unravelled the mechanisms of the drug to help us identify and understand safe forms of Thalidomide that can potentially be used for clinical benefit, such as treatment of inflammatory disorders in adults, but without causing embryonic damage. This work has resulted in several granted patents.
For decades, the legacy of the Thalidomide tragedy has severely affected the lives of survivors and their families who have suffered difficulties, pain, and discrimination, but Professor Vargesson’s expertise is playing a key role in the recognition, support and compensation of Thalidomide survivors.
Professor Vargesson said: “Our research findings have led to a number of impacts including helping to identify and compensate Thalidomide survivors and influencing governmental responses by informing guidance around the effects of Thalidomide and demonstrating how embryo abnormalities caused by the drug can be identified.”
In 2014, Professor Vargesson’s expert testimony led to a collective settlement of more than AUS$100 million for 107 disabled claimants in Australia and New Zealand, following a successful landmark class action lawsuit.
In the same year, the World Health Organisation (WHO) brought together an expert team on Thalidomide embryopathy, in response to a lack of medicine regulation, distribution and safety in South America, where Thalidomide was still used for complications of leprosy. Professor Vargesson was invited to be part of the team and contributed to a report that assisted in diagnosing drug related embryopathy and offered revised diagnostic criteria for identifying victims of Thalidomide embryopathy.
Additionally, Professor Vargesson’s research has influenced international government policy to support Thalidomide survivors and identification of those affected by the drug in the UK, Canada, Australia, New Zealand, Italy, and the USA.
- By confirming that Thalidomide’s ability to inhibit blood vessels is the basis of how the drug causes birth defects, the research has enhanced understanding of Thalidomide around the world
- Research findings have led to support and compensation for hundreds of Thalidomide survivors and their families
- Professor Vargesson’s advice and recommendations were adopted in the Canadian Thalidomide Survivors Support Programme (CTSSP) which launched in 2019 and he will serve on the CTSSP committee to assess applications for recognition and compensation
- In 2019, Professor Vargesson was appointed by the Royal Australasian College of Physicians as an Advisor and Subject Matter Expert to contribute to the development of eLearning resources that support the education of clinicians and medical professionals
- The New York Times called upon Professor Vargesson’s expertise for a series of articles highlighting the ‘forgotten survivors’ of Thalidomide in the USA, which led to an online seminar to the US Survivors Group
- Most recently, Professor Vargesson gave advice to the Italian government and the Italian Thalidomide Network Group on supporting claimants who have non-classical damage. A book outlining the group’s work is being produced to highlight the support needed for Thalidomide survivors.
- Patents have been granted covering new forms of Thalidomide which retain clinical benefits in inflammatory disorders, but don’t cause embryonic damage in model systems.