Nothing affects families like serious illness. Life as we know it stops when a loved one gets cancer, Alzheimer’s disease or sickle cell anemia. But check this out, by the end of the century, experts are sure that disease as we know it will be either eliminated or just bothersome, like the common cold.
Investors from around the globe are so sure that the fight against disease will end in victory by the next century that they’re betting billions to see it happen. Why the sudden influx in confidence? Because in the last few years, medicine and technology have linked arms in unprecedented ways, and big things are happening. Amazing things that no one even thought was possible five years ago.
New discoveries in gene editing, cell manipulation and muscle regeneration are now proving themselves in clinical applications all around the world. In other words, humans are now being healed. Today’s most brilliant minds in technology, medicine, science and biology are zeroing in on how to outsmart disease.
The holy GRAIL for curing cancer
Right now, screening exists only for a few cancer types, and usually, it’s too late to ensure survival. But extensive research suggests that cancer may be directly detectable with DNA shed by tumors into the bloodstream. And GRAIL is on the forefront.
GRAIL stands for Gene Related to Anergy in Lymphocyte (immunology). They’re mastering the science of early cancer detection, and they’re doing it earlier than anyone ever thought possible. Just one drop of the patient’s blood will tell the tale.
Getting the blood is simple, but the computation is really intense. Each individual reading consumes over a terabyte of data and involves complex genome sequencing. But it’s totally worth it.
GRAIL Founding CEO Jeff Huber claims they are detecting cancer at the point where it is the most treatable. “We are taking on one of the biggest challenges of our lifetime, combining science, technology, and clinical studies to reveal cancer at its beginnings.”
Targeting the gene pool
Let’s move from the bloodstream into the actual gene itself. Jennifer Doudna and her colleague Emmanuelle Charpentier have amazed the clinical and research world by discovering a way to literally edit out disease genes, much like you would snip out a piece of audio or text on a computer.
Doudna is a biochemist at UC Berkeley. Her colleague Emmanuelle is the director of the Max Planck Institute of Infection Biology in Berlin. Together they’ve created an easy and cheap way to make precise changes in DNA in order to disable genes and correct genetic disorders.
The project is CRISPR-Cas9, a hybrid of protein and RNA – that’s the cousin to DNA – that functions as a sort of search-and-snip system in bacteria. At first, it was recognized as a way to hunt down and kill viruses in the food we eat.
But Doudna soon realized that it could also work well in other applications including human cells. She’s a hot item now, having been elected into the American Academy of Arts and Sciences, the National Academy of Sciences, and was recently awarded the Breakthrough Prize in the Life Sciences award.
Shedding light on stem cells
Let’s switch gears and move from genes to cells. Newsweek's October report caught my eye with exciting new results in non-neuronal Optogenetics and its role in cancer treatment. Basically, the effectiveness of treatment is sharply on the rise through the application of light.
Researchers use blue LEDs in their studies on mice, and flow cytometry to analyze their results. They proved the ability to control and stimulate intracellular Ca2+, which actually boost T cell immune responses in tumors. Nature Communications went on to describe how light stimulation may be able to set off anti-tumor responses outside of the light's illumination field, affecting the whole system.
In our PODCAST, Jared Toettcher, Assistant Professor of Molecular Biology at Princeton, told me that light delivery is getting easier and that actual clinical applications using Optogenetics is finally getting off the ground. Just in the past few years, his team has been able to develop photosensitive molecules for cell biology applications, light-controlled kinases, protein-protein interactions, and now even light modulation or control of different proteins.
What was once possible only on paper is now living proof. Researchers like Toettcher are confident that light-controlled cellular activity will continue to grow. Once they are able to activate a cell at a specific time and location, the ramifications will be revolutionary.
Taking it to the heart
Let’s move to the heart, and the use of stem cells to regenerate and repair. Eastern Newts and newborn mice are able to regenerate their legs, and even parts of their hearts. The key to their superpower is dedifferentiated cells. Researchers like Thomas Braun at the Max Planck Institute for Heart and Lung Research, Bad Nauheim are trying to figure out how to harness those same cells for humans by turning cells into a more primitive state. His research has revealed a signal substance response. Scientists are now hunting for a way to use it effectively.
Cardiologists, like Dr. Nabil Dib from ISCTR, are developing new technologies using stem cells. In addition, Dr. Dib has also developed a dissolvable heart stent, which dissolves into the patient after two years with no further need to take anticoagulants. To hear more from Dr. Nabil Dib, listen to my PODCAST.
These types of treatments are still limited in the U.S. and Canada, so heart patients like “Wayne” are traveling outside their country to receive care. There they find victory. Wayne traveled to the Regeneration Centre of Thailand for enhanced cardiac cell replacement therapy. He was 64 years old, a sedentary smoker, survivor of two heart attacks, both resulting in angioplasty. He was on nine different medications. One year after his Stem Cell Regenerative Transplants he has had zero incidents of chest pain, and NO visits to the ER. He reduced two meds and eliminated four altogether.
The ethical dilemma
In closing, I’m sure the prospect of fixing human disease brings up some mixed emotions. For me, the jury’s still out. But one thing’s for sure, I’m impressed by the technology and by the passion of the researchers. They truly do want to end human suffering in terms of disease. But at what lengths are they willing to take their methods?
Concerning ethics, Jennifer Doudna from CRISPR doesn’t feel that gene editing in embryos would be ethically correct, because the editing affects an entire genetic line. She recommends only editing genes in fully formed adults.
Jared Toettcher may have a different view. In this article, I only ask that you think critically for yourself and discuss it with your family. Because the question may come up one day if you or your kids have to make important medical decisions. Meanwhile, I’ll be sure and let you know when significant breakthroughs arise.
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