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The new miracles of medicine

As biotechnology begins to heal deadly diseases and treat incurable health problems, medicine is entering a new biotherapeutic era. Cell therapy, gene therapy and immunotherapy, researchers are developing new ways to heal. The first treatments finally arrive on the market.

Repaired retina, cured leukemia, improved immune system ... after years of pledges, biotechnology is starting to mature. And these are new miracles that are starting to become possible. Like the Kymriah of Novartis whose US Food and Drug Administration (FDA) has just approved the first gene immunotherapy treatment for leukemia. Or GSK's Strimvelis, which heals the immune deficiency of "bubble" babies, while Pfizer launches a clinical study against Duchenne muscular dystrophy (causing progressive degeneration of all muscles). Or even, in another genre, erectile dysfunction that Danish researchers today claim to cure, while increasing the size of the erect penis, thanks to the injection of stem cells ... Anecdotal or beginning of a long a series of miracles within the reach of medicine?

If the health giants have long shunned this DIY live cells, called biotechnology, some now take their tickets. Novartis will acquire American gene therapy specialist AveXis for $ 8.7 billion, while Merck, Roche and AstraZeneca are banking heavily on immunotherapy, while Bayer has created BlueRock Therapeutics for stem cell therapy. Mergers are also accelerating around immunotherapy nuggets, the most promising route in the short term:

Gilead Sciences paid $ 11.9 billion for Kite Pharma and Celgene was awarded Juno Therapeutics. In the redemption race, however, not all health techs are so close to the market. After huge ads without follow-up, "big pharma" have also learned to be wary of miracle sellers.

Gene therapy takes its time
While the first telethons of the 1990s were looking for solutions for children with myopathy, it took twenty-five years for Pfizer to announce a candidate for Duchenne muscular dystrophy. But there we are: six drugs have already been allowed (1), although, for now, only a few areas of the body, such as the retina, are easily accessible to genetic modification. They have the advantage of not provoking too aggressive rejection reactions, which confirms the director of the Evry Genopole, Jean-Marc Grognet:

"We begin to treat diseases of the eye, brain, immune system and blood, because the necessary doses are reasonable compared to those needed to treat muscles and other organs. But in the world, many trials are underway and we get first results on other tissues, such as the liver. "

Stem cells not yet ready
Another promise is regeneration by so-called pluripotent stem cells, that is to say, capable of transforming into muscle or skin, depending on where they are. These cells are those of embryos that multiply very quickly to form a baby in nine months. In 2012, Japanese and British researchers Shinya Yamanaka and John Gurdon were awarded the Nobel Prize for Medicine for developing a genetic modification that transforms our "old" adult cells into pluripotent young strains (called IPS). As a result, biotechs began to dream of regenerating damaged organs and making livers or kidneys with 3D cellular printers. The first results finally come with embryonic cells (see "Render the view", page 8), even if it is necessary to wait a little longer before knowing how to make skin per kilometer. According to Marc Peschanski, scientific director of the I-Stem research institute (AFM Telethon / Inserm), "the first big pharmas start investing in stem cells and there are more than twenty trials in the world on the retina, the muscle heart disease, insulin production in the context of diabetes ... "

Being a pharmacist is not just about being a drug salesman

The best-known role of the pharmacist is to deliver medications prescribed by a physician or other health professional who is authorized to do so. Beyond this essential role, it should be known that the pharmacist assumes full responsibility for the verification of prescriptions and what he delivers. If he considers that the prescribed treatment is not suitable or if he has doubts, he may refuse to issue the medicine concerned. He must therefore contact your doctor to make sure there is no error and possibly ask him to change the prescription. It is imperative that you check that all the medicines on your prescription are compatible with each other and that they do not present any interaction that may reduce the effectiveness of the treatment or that may cause problems of tolerance such as intestinal disorders or allergy.

Your pharmacist also makes sure that you understand your doctor's instructions regarding the frequency, dosage and timing of the shots. If necessary, he can add some useful tips so that you follow your treatment.

Your pharmacist must record the medications you are taking in your "Pharmaceutical Record". If you have a chronic disease or allergies to certain products, he can also note with you in this file. It thus ensures your security and allows a faster analysis in case of problems.

This is also true for drugs and other over-the-counter products that you buy on your own or on your own: these can also influence the effects and effectiveness of the treatment you have been prescribed. For your safety, It is also important that you report any consumption of food supplements or herbal products that you would have purchased outside of your pharmacy.

Your "pharmaceutical file" is at your disposal, you can consult it at your pharmacist if you wish or ask for a copy.

The pharmacist and his staff are bound by professional secrecy. So you have no fear to have, your data is kept completely secure.