A science bomb. DeepMind, a subsidiary of Google, succeeded at the end of 2020 in determining the 3D structure of almost all the proteins made by the human body. And announced, a few months ago, the free availability of its results to all researchers. This discovery is poised to accelerate medical research for decades to come. And for good reason: proteins are essential for life. They form antibodies to defend us against infections, hormones and enzymes to regulate digestion, breathing… All it takes is for one of them to malfunction for you to fall ill.
However, to open the way to healing, you have to design a key (the medicine) that fits perfectly into the lock (the protein). Until then, to know the complex shape of this lock, composed of amino acids folded on themselves, it could take years… and still. Today, in a few clicks, it’s done!
Artificial intelligence at the controls
At the heart of the success of DeepMind and its Alphafold software, artificial intelligence (AI), which succeeds in predicting, from a list of amino acids, the three-dimensional shape of the protein it composes. When the machine overtakes the human being… AI appears to be a tremendous opportunity to improve the diagnosis of a disease as well as its management. Understand that from a health database collected at the national level (frequency of such pathology in such region) and individual (genetic profile, lifestyle, biological results, etc.), a computer can predict the percentage of risks of developing a disease and specify the appropriate treatment.
Illustration through the story of Marie, 55, who complains of extreme fatigue and loss of appetite. His complete data is compared with that of other patients who have had the same symptoms. Thus, his doctor can move towards a benign or malignant cause. If the final diagnosis is a liver tumour, again, by specifying the genetic map of this cancer in computer software, the oncologist will be able to indicate whether Marie will react – or not – to such chemotherapy. Personalized medicine is therefore only in its infancy.
Consultations beyond reality
We produce more and more health data! If only the number of daily steps that appears on our smartphone. We have scales, blood pressure monitors, glucometers, or even watches connected to an application. And the sale of a “smart ring” is announced for this summer. This will continuously measure our vital parameters (temperature, blood oxygen level, etc.) and monitor our sleep. Still, the challenge for the years to come is to host all these health markers on secure sites. Insurance companies, or even employers, should not have access to it! Reassuring signals already exist.
Let us quote the creation, last February, of “ My health space “, a site set up by Health Insurance, which brings together all our medical information, which can be shared with doctors. A step forward in favor of remote consultations, which, according to e-health experts, will represent, in five to ten years, 70% of medical appointments. Everyone will be able to have the opinion of specialists, regardless of where they live. By 2040, telemedicine promises to be more efficient, with the creation of our digital twin, a virtual reproduction of our organs and/or all our health indicators. And this is no longer science fiction!
After telemedicine, let’s talk about nanomedicine – on the scale of a billionth of a meter! It is thanks to her that anti-Covid-19 vaccines based on messenger RNA have been able to exist. In principle, mRNA does not enter cells. But by integrating it into nano-drops of lipid particles, that changes everything. We are now witnessing a boom in research on these nano-vectors, which can transport all types of drugs directly to their site of action, as long as specific receptors are added to them. Imagine an anticancer brought in this way on such and such a tumour. Once injected, it will precisely treat the “diseased” cells and not attack the others.
The advantage: a drastic reduction in the doses administered, with a tenfold therapeutic effect (the active ingredient goes directly where it is needed) and, ultimately, a marked reduction in side effects. And nano-medicine can go even further. Welcome to the heralded era of theranostics, which combines therapy and diagnosis. The idea? Use nanoparticles to transport both markers, visible on imaging, which identify the areas to be treated, and a targeted drug. This will make it possible to treat incipient tumors or advanced metastases that conventional chemotherapy and/or radiotherapy cannot currently reach.
Our refurbished organs
Long live regenerative medicine! The principle is simple: reconstitute healthy tissue from so-called “pluripotent” stem cells. Either cells taken from the embryo, or even from the adult, which are capable of giving rise to all types of cells: skin, nerves, heart… Stem cells are currently the subject of around ten trials clinics around the world. Cultivated in laboratories by directing their transformation, they are deposited on a membrane to form a patch, like a piece of wedding veil which will then be “glued” to the tissue or the deficient organ.
On the retina, for example, a study on humans consists of “grafting” epithelial cells that feed the photoreceptors that are lacking in AMD. Tomorrow, the challenge will consist in “patching” both epithelial cells and photoreceptor cells, and thus treating this ocular pathology, even in its advanced form. Another test: the injection, directly into the brain, of dopaminergic neurons, obtained from stem cells, to replace those that no longer work in patients with Parkinson’s. To be continued…
Our DNA reviewed and corrected
Did you know ? Genetic scissors, composed of a fragment of RNA and an enzyme, already exist! This discovery earned Emmanuelle Charpentier the Nobel Prize in Chemistry in 2020. Cock-a-doodle Doo ! With CRISPR-Cas9, the “Swiss army knife” of medicine, we can cut one or more genes and overcome a disease, as soon as it is caused by a faulty gene. Research is now applied to refining the technique to target cutting without causing collateral damage. Future gene therapy does not stop there. It is based on what we currently master: injecting genetic material to replace a defective gene. With the hope of thus curing 80% of rare diseases of genetic origin. That’s not all. The dream of being able to provide a cure for all types of cancer is about to become a reality.
It is always a question of providing genes, this time not to repair but to give a super power to immune cells (lymphocytes). These then know how to specifically recognize tumor cells and destroy them. It remains to decode the genetic profile of all tumors. Gene therapy has not finished surprising us. Did you know that Alzheimer’s disease could be cured by injecting genes into the brain that block the degeneration of certain areas? A huge hope in perspective…
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Three questions for: Valérie Lemarchandel, Scientific Director of the Foundation for Medical Research (FRM)
How would you define the medicine of tomorrow?
More personalized, more precise, and above all more preventive than today. The FRM is taking this turn. Thus, the study of the impact of environmental problems on our health is one of our priority areas of research. The challenge ? Understand why and how environmental factors (pollution, climate change) increase the risk of cancers, respiratory diseases… We have the same thinking for neurodegenerative diseases. We cannot treat Alzheimer’s disease without having elucidated the processes. We are thus supporting a project on the mechanisms of propagation of abnormal proteins in the brain.
What are your other priorities?
We are working on restorative medicine using stem cells to replace an organ (the artificial esophagus, for example) or to help it regenerate. We are also investing in the field of Covid-19, in particular to study the problem of the long Covid.
What is your dearest wish?
This would be to promote bridges between research and industry, to increase the chances of developing treatments. A discovery must not remain in the state of a simple idea! Let’s not forget that it is from research that therapeutic innovations will be born, hence the importance of supporting it.
*Thanks to Amanda Silva Brun and Nathalie Mignet, CNRS researchers and board members of the French Nanomedicine Society; to Marco Fiorini, General Delegate of the Alliance for Research and Innovation in Health Industries (ARIIS); to Pr Boris Hansel, endocrinologist, director of the connected health DU at Paris Diderot University and manager of the youtube channel Pums (for better health); to Christelle Monville, professor of biology at the University of Evry and researcher at I-Stem; and to Frédéric Revah, director of the Généthon.
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This is what medicine might look like in 20 years
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