The Woman Who Changed Drug Development

[Music] back in 1944 scientists were only just beginning to suspect that DNA was our genetic material that’s the Year Gertrude Elian first started studying nucleotides the chemical building blocks that form DNA over the next five decades Elian became a leading expert on nucleotides and her outside-the-box thinking led to methods that totally transformed the world of drug development including life-saving therapies we still use today Gertrude elion was born in New York City in 1918 the daughter of Eastern European immigrants the year she finished high school she watched her grandfather’s to come painfully to cancer his death inspired her to study chemistry in college and eventually earn a master’s degree there weren’t a ton of research jobs available during the Great Depression particularly for a woman but after World War Two broke out she scored a position in the lab of a guy named George hitching Hitchings had this idea that if you could understand a biological process you should be able to use that information to design chemicals to disrupt that process this idea might seem obvious now but that’s just because it’s how we like to develop drugs today at the time this rational approach to drug design as it came to be called was new and pretty radical see in the mid 1940s most of the drugs that were available were either based on plant compounds that people have been using for millennia like aspirin or they’ve been discovered by accident like penicillin but Elian and Hitchings wanted to take a more deliberate approach and there were two key reasons why nucleotides seemed like a good place to start one all cells need them to divide since dividing means doubling your DNA and DNA needs nucleotides and too certain bad cells like cancer parasites and bacteria divide way faster than healthy cells this makes them especially hungry for nucleotides if you had a way to exploit this hunger you might be able to fight all those things the problem was nobody knew much of anything about how cells make or use nucleotides one of elion first assignments that her new job was to start figuring all of this out so she synthesized a bunch of chemical analogs that were similar to nucleotides or things that cells needed for making nucleotide the idea was to see what cells would do with these imposter compounds some of the analogs had key chemical differences that made cells unable to use them like normal nucleotides they were a biochemical dead-end and they would gum up the works essentially blocking a cell’s ability to make DNA or RNA which is exactly what Elian and Hitchings were looking for in a drug but they had to make sure it wasn’t too toxic to people the first breakthrough came in 1951 when Elian synthesized six mercaptan urine or 6mp it’s one of those dead end molecules and is especially good for stopping out-of-control immune cells it was a huge step forward in treating childhood leukemia and it opened the door to new ideas for treating cancer in general in testing 6mp and related molecules elion started to piece together that different cell types and cells from different species responded differently to some analogs and that’s the key to making drugs like these work once she found a promising lead she would design slightly different compounds to try to exploit some of those differences her approach to synthesizing them was novel as well on top of that she was among the first to follow what happened to drugs in the body and used that information to design drugs that were more specific less toxic and more effective all of these approaches in combination led to lots of new discoveries about nucleotide metabolism and they led Elian to treatments for an incredibly diverse set of problems including malaria gout tissue rejection and autoimmune diseases later in her career she showed that it was possible to develop highly effective drugs for viral diseases in the 1960s most of the research world including Hitchings her partner believed that since viruses use human cells to replicate it would be impossible to develop a drug that would disrupt a viral replication without harming the human host but Eliam proved them wrong in a big way she connected the dots between several studies some hers some from another lab and realized that modified nucleotides could block viral replication she followed the lead and developed a cyclo veer it’s a compound that interferes specifically with a nucleotide making enzyme from the herpes virus but not the human version of that same enzyme and this work paved the way for AZT the first effective antiretroviral drug in the fight against HIV and AIDS in fact it was scientists from aliens research team who developed a CT after her official retirement not only Raz t 6mp and other drugs Elian help develop still in use today the World Health Organization counts them among the safest most effective drugs available but beyond the drugs themselves it was Elian and Hitchings contributions to the process of drug discovery that had the greatest impacts once they showed that their process worked that you could rationally build drugs from other drug companies around the world started using it and that’s what earned them a Nobel Prize Elliot and Hitchings along with Co awardee James Black received the Nobel Prize in medicine in 1988 for their contributions to the field of drug development it’s rare that a half-century of toil earns such recognition but no one can argue that it’s not well deserved and as long as we continue to use the drugs she designed elion contributions will continue to improve the lives of people everywhere thanks for watching this episode of scishow if you like learning about awesome women in science and want to help us make more episodes like this one consider supporting us on patreon you learn neat perks and you’ll help us make great free videos for everyone to enjoy check it out at patreon.com/scishow [Music]

71 comments

  1. I've never been involved in drug discovery but as far as I know it often costs roughly 1 billion dollars to discover and get a single drug on the market (I recently made a video about this topic). However, 86% of drugs still fail in this process. There are many reasons why this is the case, one of which is that mice are often used during pre-clinical phases. Although the physiologies between mice and humans are often very similar, there are slight differences on a molecular level. As a consequence, drugs, which might work in mice do not work in humans. Some years ago, however, we started to develop organoids. Those are structures and tissues which are made from human cells in the laboratory (cerebral organoids, for example, mimic the brain) and they are very exciting as they might partially replace mice in drug discovery leading to better outcomes! Feel free to ask me anything about the human body or stem cells (since I am conducting research in this field)!

  2. That (bit at the end) wasn't fair. We will continue to benefit from her work even after every drug she conceived is replaced by a more effective drug, because those drugs will likely de developed using her rational methodology.

  3. thise drugs also cause many cancers. Ive has AZT for Crohn’s. Didnt help my symptoms and made me sick as well as catching illnesses.

  4. AZT safe? What about the facial wasting that occured in large amounts of the population that took the drug. What is this a commercial?

  5. I had no idea this video was going to be so relevant to me when I clicked on it. My gastroenterologist is wanting to prescribe me 6MP for my ulcerative colitis, as the first phase of treatment has failed. This video got me slightly emotional and thankful to know that a woman was the one who designed this life saving drug. I’m so thankful she got the recognition she deserved in the end 😊. SciShow, thank you for continually making amazing content

  6. So how about how insulin was discovered? It was discovered before this, and it was neither an accident like penicillin, nor a plant compound.

  7. Acyclovir saved my Mom's life when she had mengioencephalitis from shingles. I'm genuinely happy to learn more about the woman that made that drug possible.

  8. 0:27 The year of the pandemic that killed around 2% of human population.
    In some countries, as much as 10% of national population.
    The Spanish flu.

  9. Thank you Gertrude for helping to develop Acyclovir so I could get over shingles faster so I wouldn't need to be quarantined! Very intelligent woman and a visionary that helped so many people!

  10. Incredible how far we've come. It's hard to imagine there was a time when the idea "what if we actually think and use logic instead of just making up random stuff that feels right?" was revolutionary and brilliant. Actually, now that I think about it, we may not have come as far as I'd like to think. Too often these teachings are still ignored in other fields to this day.

  11. I was so afraid that because she was a woman in the 60s she would get the Rosalind Franklin treatment and never get the Nobel Prize. Glad to see she got the recognition she deserved!

  12. How many unknown male scientists who did far more did you gloss over to arrive at Elion? And where's your video discussing how women are overrepresented in STEMM, where the 2nd M stands for Medicine? –When can we expect that?

  13. I recall the steps achieved before AZT's invention. Sorry Scishow but AZT was a dangerous and harmful drug that for a time was the sole HIV/AIDS drug considered effective. It was as deadly as the disease. The side effects made people wish for death. You should know better-or at least ask someone who does. Perhaps you can do a video on the development of antiretrovirals after AZT.

  14. So, I suppose most people think this is pretty cool, but as someone who is now doing a master in drug development this seems incredible. Those drugs are (1) really cool and (2) rational drug design has had an incredible impact on the farmaceutical world

    In my book, these people are genuinely genius

  15. The revolutionary process that Elion developed is, for all practical purposes, known as tweaking. That's what most drugs on the market do. They don't correct the underlying malfunction, but rather tweak the incredibly complex processes so lab outcomes look more favorable. A simplified version of this process in the computer industry would be called a "work around". This approach hasn't been very productive in helping sick people function better, but it has been incredibly profitable for drug companies.

  16. Can't find any streets (or libraries or anything) named after fer on Google maps. And no mentions of anything such on her wiki page either.
    Let's make it happen, people!

    if/when a new street or hospital or school is planned in your area/city, suggest they name it after her 🙂

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