1959/1989 "Game-Changer"- Cystic Fibrosis/CML – 30/40 year journeys of hard work to solve how the body fails.
Doug Hohulin
To Save 1 Billion Lives with AI, Exponential Blueprint Consulting LLC, President/Founder, When the AI System Has to Be Right: Healthcare, AV, Policy, Energy. Co-Author of 2030: A Blueprint for Humanity's Exponential Leap
New Cystic Fibrosis Treatment a "Game-Changer"
"30 years and two months ago—on September 8, 1989—researchers published three papers describing the gene responsible for cystic fibrosis. It was a huge breakthrough, and one they hoped would lead to a reliable treatment in no time. Well, it wasn’t “no time”, but the discovery has finally led to a drug that dramatically improves the lives of most people with cystic fibrosis.
A new three-drug combination called Trikafta was unveiled last week in a pair of papers in The Lancet and The New England Journal of Medicine. And it’s already being called a “game-changer” by medical professionals who don’t use that kind of language lightly."
I have a friend who should have died 15 years ago but because of the drug Gleevec, my friend's life expectancy is no different than the average person.
In the book, the Philadelphia Chromosome: A Genetic Mystery, a Lethal Cancer, and the Improbable Invention of a Life-Saving Treatment by Jessica Wapner, tells the story of how it took HI (human intelligence) ~40 years of discovery to find a cure to one way the human body fails. The disease, Chronic myelogenous leukemia (CML), that would kill a person in 5 years. Now with just a pill – Gleevec (Imatinib), there is a complete cure.?This is the type of wonder drug that is needed to be duplicated for other ways the body can fail.?The last chapter gives a great summary of key steps in the process of dedicated hard working doctors, nurses, researchers, patients, caregivers and people who care.??“The Philadelphia chromosome was discovered in 1959 when David A. Hungerford, a graduate student at Fox Chase Cancer Center, and Peter C. Nowell, a pathologist at the University of Pennsylvania School of Medicine, detected an abnormality on chromosome 22 in cells taken from patients with chronic myeloid leukemia (CML).”??https://www.foxchase.org/news/2010-08-23-chromosome-symposium
see this video and slide from the talk related to CML?NURSING EDITION: Great and Crazy things we do in Oncology | Lecture to Nurses & Staff (youtube.com)
The chart from Vinay Prasad MD MPH’s talk illustrates the transformation in the treatment of chronic myelogenous leukemia (CML), thanks to the drug Gleevec (imatinib).
In the 1970s, patients diagnosed with CML faced a grim prognosis: life expectancy was less than five years after diagnosis. At that time, there were few treatment options, and the disease was almost always fatal. As shown in the chart, life expectancy for patients with CML at age 55 was dramatically lower than that of the general population.
However, the introduction of Gleevec in 2001 marked a revolutionary shift in cancer treatment. The drug, which is a tyrosine kinase inhibitor (TKI), targets the BCR-ABL fusion protein responsible for the uncontrolled cell division in CML. Gleevec’s precision and effectiveness turned what was once a fatal disease into a manageable chronic condition.
The chart reflects the incredible success of this drug:
This chart exemplifies how targeted cancer therapies like Gleevec have changed the landscape of oncology. What was once a death sentence is now a manageable condition, illustrating the profound impact that scientific innovation and personalized medicine can have on patient outcomes. This transformation also provides hope for future breakthroughs, where AI and new treatments may continue to close the gap between diagnosis and life expectancy for other cancers and diseases.
See this blog: ML/AI, HI (human intelligence) IA (Intelligence Augmentation) to Solve how the body fails
“In modern medicine, Dr. Atul Gawande said, there are 13,600 diagnoses, or ways in which the human body can fail, and no patient comes in with just one diagnosis at a time. Now more than 6,000 drugs can be prescribed, and 4,000 medical and surgical procedures can be performed.”
This paper in nature highlights:
From study design to patient recruitment, researchers are investigating ways that technology could speed up the process.
It shows these important charts:
领英推荐
This quote highlights how AI can help with drug discovery and "Patient maintenance
The hurdles in clinical trials don’t end once patients enrol. Drop-out rates are high. In one analysis of 95 clinical trials, nearly 40% of patients stopped taking the prescribed medication in the first year. In a recent review article3, researchers at Novartis mentioned ways that AI can help. These include using past data to predict who is most likely to drop out so that clinicians can intervene, or using AI to analyse videos of patients taking their medication to ensure that doses are not missed.
Chatbots can answer patients’ questions, whether during a study or in normal clinical practice. One study4 took questions and answers from Reddit’s AskDocs forum and gave the questions to ChatGPT. Health-care professionals preferred ChatGPT’s answers to the doctors’ answers nearly 80% of the time. In another study5, researchers created a tool called ChatDoctor by fine-tuning a large language model (Meta’s LLaMA-7B) on patient-doctor dialogues and giving it real-time access to online sources. ChatDoctor could answer questions about medical information that was more recent than ChatGPT’s training data."
Here's to curing all the ways the body can fail.
If you are interested in learning more about and Exponential Medicine?
See this event: https://exponential.singularityu.org/medicine/
Exponential Medicine is a unique and intensive four-day experience that gathers world-class faculty, innovators and organizations from across the biomedical and technology spectrum to explore and leverage the convergence of fast moving technologies in the reinvention of health and medicine.
I really liked?Session 16 Neuromedicine At The Frontier
Our understanding of the brain and mind is accelerating. This session will explore the cutting edge, from consciousness, to preventing and treating neuro pathologies.
Rudiolph Tanzi PhD, Professor of Neurology, Harvard Medical School. Co-Director Center for Brain Health at Massachusetts General Hospital.
His talk starts
"Dr. Rudolph Tanzi, professor of neurology at Harvard Medical School and the director of the Alzheimer’s Genome Project?, recommends following the SHIELD plan, which emphasizes a multi-faceted approach to avoid disease. The six components of the plan include sleeping, handling stress, interacting with others, exercising, learning new things, and following a healthy diet."
Divya Chander MD PhD, Chair for Neuroscience, Singularity University
Here are links to all the talks each day
Exponential Medicine 2019 Day 1 https://www.youtube.com/watch?v=pGSXM1-r6dk
Exponential Medicine 2019 Day 2 https://www.youtube.com/watch?v=9TsYJq3rBV8
Exponential Medicine 2019 Day 3 https://www.youtube.com/watch?v=7eFkT-wtznk
Exponential Medicine 2019 Day 4 https://www.youtube.com/watch?v=gj1SSl25Sng