in 45 minutes on d, w. as these categories are thoughts say, well, the take the measurements, choose the fabric sketch cut. so it's made to measure one of a kind of tailored. the idea of tailoring also works in medicine for the 1st time in europe and r n a therapy is being administered. that was made specifically for a particular patient, a huge step forward in the treatment of rare diseases. we'll look at that story and much more this week on tomorrow to day

d w. sign show. welcome to the program. boy rouse is 6 years old. at 1st glance, he's a boy who's developed normally. but when he was about a year and a half old, as parents realize that something was wrong, their son fidgeted constantly and stumbled more often than other kids. his age. the 1st symptoms of his illness. porras has an extremely rare genetic defect and effects about one and 100000 children. actually ever seen 1st normal van, hey, what's going on? he was just like those children. first, orthodontics make that either shocked with the news and very upset. actually, the doctors just told us that there was no treatment for this company. the and no treatment met. their child would soon end up in a wheelchair and would die. at

a young age. the doctors had diagnosed attacks he had tell inject taysia or a t for short virus and island, tried to find out more and found a needle in a haystack of research and to us another child with that he was receiving a novel r n, a therapy or some and the girl in the us have the same genetic mutation, so that the individual therapy that the girl takes into us was also suitable for us. we went to boston, we started with the treatment. and now it's continuing in keeping the doctors here in germany, tubing and university clinic center for rare diseases examined. boy rose from head to toe and 0 in on his symptoms. so it's pointing on now here almost every day on now,

he has expected his legs are unstable, but something else is also striking. last time because the unhappy what by so is it he wasn't just phoning the room right now. he also makes these jackie movements said he called control. well yeah, in order action is his thing is eyes on and seeing it's also very difficult for him to remain still in one place without making compensate to remove minutes. well no, it's nice to feed on neurologist mount a synopsis, a member of the research team tracking boy rises, treatment explains that the disease is already visible in the boy's brain to minutes, then slice get this. and there are already some very slight initial indications that the nerve cells are effected. you can see slight indentations here, for instance, indicating that nerve cells have already been lost in our goal now is to prevent further nerve cells from perishing assets and to quinn to keep the doctors can 2 or

4 raz outright, but they may be able to slow down the course of his disease, but how can they overcommit genetic mutation that keeps a specific important protein from forming in the boy cells that was put in guns 90 key. and we had to take a completely new approach and develop a therapy specifically for his genetic mutation i need to one that's precisely tate or like a key that fits the keyhole of piracy is gene modification. the key is an r n, a molecule developed in the lab. it's called a s o. it covers up the genetic defect, 60 gene, and don't within this m. as i can see that you 1st have to understand that his mutation creates a kind of incorrect reading pointing to gene. the therapy works by using an r n a messenger. that insures this incorrect reading site is covered uh and the normal correct reading site is used again. the parents lives alternate between home and clinic, highly and has given up her job as a clinical psychologist for rises,

disease demands a lot of time, energy and attention. every 3 months, the family travel from berlin to tubing and each time he undergoes multiple examinations and is given an injection in his spine when they were testing. uh, there's a very special medicine to submit this and it's for me though for to to, to chat, to chat and know about v believe in size. yes, yes. and if something is done over all the discover, the therapy that porter has received in tubing and is not approved, but it is only chance. this makes it all the more important to really measure test and compare everything, all of my and my signal. go back there, stick it on, and return on speed. okay. now go play roses, receiving what's called individualize therapy, which means the effectiveness of the approach hasn't been proven in trials. there

aren't any. he's the only participant in a trial with one subject. the only one who can deliver results that could advance research. would that make these exciting, these young children are pioneering the whole approach. that's why we're so glad that there are currently no side effects. because then we can say, okay, it seems to be going in the right direction. but, and we're learning a lot from the methodology and how to measure its effectiveness, whether the patients accept it and whether it could be used for other patients as well. and getting under the above, under percent, porras will need the therapy every 3 months for the rest of his life, the treatment cost. $70000.00 euro is a year. most of the work involved is performed at the clinic. but it's still too early to say for sure whether the r n a injection will work. and so you know the dispatch on that on the various other diseases. so think that is a good chest that it's kind of

a cute as well. now he's not getting worse and sick which point for us, his tables. and that's the good news. in the hospital microscope examination show point rises, cells are absorbing d r n a therapy. but the doctors here want to do even more than just help their young patient. they already have more r n, a snippets in their collection that might help others with similar gene defects. the same or in a kid can be used to tailor drugs to specific patients. if it's still, it's still a way off, but why we treat one patient we're already developing 2nd and 3rd r n a therapies. for other mutations, we can use the same principles of key development over and over again to produce all kinds of individual keys a genetic defect, a corresponding drug. that's the goal of the european research network called one mutation, one medicine. and boy raz is the 1st patient if it succeeds,

slowing down the advance of rare genetic conditions with r n a would be a giant step forward in medicine to develop a new medication, researchers have to determine exactly which compounds could play a healing role in the process, some estimates place the total number of possible drug candidates at around a november distilling molecules. that's a one followed by 60 zeroes, could artificial intelligence shorten the search for elusive therapies. imagine phase lock is a disease. and imagine this kid is a cure. open interlock means finding the right treatment. but here's the cash you're not faced with. just one key means yes of them.

now you could be trying one key of the time. that's kind of what research has of done up until now. or you can predict which works for like using i think the something that is a i, as the power to massively accelerate the discovery of a treatment, a drug discovery company di power tools to accelerate drug discovery. scientists around the world are using ai to discover new medicines and in the process, they could be unlocking the secret to a longer, healthier life. the agent can be targeted to the agent can be delayed. an aging in certain ways can be stopped and reversed also. so what exactly is a drug included have positive, longer on this thing is utilizing

a i also do the console in bio medical problems with a lie and you can decrease the probability of success and also make it cheaper and faster drug discovery consists in finding new medications to treat diseases. in this case we're focusing on diseases caused by proteins by goes to i've tried is marcusson's or else hymer disease. and because the process is partially repetitive, it can be partially ultimatum. the computer to a drug, this quality process is small noun. it's something that has to be try it for many decades before but a i, it goes beyond automation. it predicts, it looks like good candidates, understand some of the features and the predicts either good candidates. but you could also take it a step further. imagine that you keep from scratch with a design properties. so you create through a set of perfect use, the don't see the walk. some all the tools discovered we, they,

i have already entered clinical trials and the field is attracting billions in investments. so it's clear that the approach is very promising, but there's more, some research, those are looking for a cure to something the most of us don't even consider a disease age what he's aging, why do we age? do we have to age? the aging is a biological process. ok? usually team the editable. well we can not delay death, but we can do something about agent businesses nearby as a lie, a groundbreaking research is in the science. so from 71 of the things that happened with aging, we accumulate what's called zone b sales, or we call them cns themselves. that when they accumulate, they cause in environment to age and get diseases. we want something that keeps

only the deluxe, those that are very few. centrally dicks and known as a glass of monica into that can kill us anderson cells. so maybe i so we told why not utilize a i to die and find these drugs faster. and they did vanessa. a team and they come what may be the most promising single dictates, ever seen? so really i make us live longer. well, let's have a look finding the right molecule is only the 1st step of the druggist hubbard, process for this and how the whole process works, where he's using the lab chamberlain. we meet with david the ceiling for a peek. inside his lab internet. he's the walk us through the main stages of john x and you don't know what most of monic yous are doing, like the maybe just a few falls and stuff my fewest,

they actually know what they're doing. that's on the human body. but they have many, many more money kits available. the 1st step is to decide which model choose the best. if this test many, many column phones this much as possible to see if there would be active from this disease. so, so i can show it there's 170000 molecules in the freeze executive. okay. wow. and these little dots that we see each one of those contains a monitor executive vision machine moves the molecules on the proteins they want to target. so then that a come from flight, this is finished. it's ricky, now from sexual transfer. except this other machine uses a powerful microscope to capture their interaction. how it looks so weird, it looks like an aliens i come actually after this. so then, because if you do this kind of experiments, you might end up with

a lot of data in this left we uh, for expense the collecting, sometimes like test so far as of the images sent to human, couldn't possibly, and it has all of this into the system basically where the machine and then can step in the network, scanned the image, just a fine good candidate right here. we have a lot of onto pick 2 agents which i plus together like another molecule where we don't know actually what kind of pharmacologic a 2 minute see a test. and we see that it's really close to the 2 agents and dispatch mckenzie in the us. maybe i think this is that's what s and s for an intimate to a tablet for addiction, a meg still needs to be tested is anything they told us to go from the predictions made by? yeah, i, there are still many steps necessary to ensure that dr. is safe and effective. then

once we go to mock, it's an extra set of costs. then you go to the kinetic 5 clinical trials take a long time. and there's a little way i can do to speed up that part of the process. this, the ai is a disruptive technology, but not the functional fields at least for now, it can always be the 1st step of the job discovery process. i do not think that piece is on the corner something no disorder. researching goals are wanting to plan for set them to do to related diseases their boobies and all these things available for patients to take in the next 10 years. hey, i could also help us develop novel antibiotics and area of pharmaceutical research, where new drugs are urgently needed. antibiotic resistant bacteria are becoming

a major problem. worldwide in 2019, they are thought to have killed well over a 1000000 people to compare that's around twice as many killed in the same period. vimal area an intensive care unit at a hospital in the switch city of them. to patients here are infected with antibiotic resistant bacteria, pathogens that coming onto politics and no longer able to kill the stuff where i can protective clothing and keep the effective patients isolated to prevent any further transmission. the head of the infectious diseases department, christina, to and to discuss his treatment options with team the physics, the sofa. we've been able to control resistant pathogens to some extent. most of the time we still have compounds and reserve the work. but you can imagine that at some point that will no longer be the case. and that's a great film,

because that would put us back to the time before the antibiotics. and that would be a massive step backwards for both. right. and we'll see for, to see what would that mean? this book that patients would stop dying again, from bacterial infections, that to date of being considered treatable. x. but stroke of assigned and pandemic . one that's creeping up on this and growing big data by the year. it's an anomaly development for board members of switzerland's round table on antibiotics. a private association of experts from science, business and politics. managing director bob republic says the issue is clear of the fall, no question. we need a steady flow of new and t bio sick because that's vital. then the problem is guessing why all the time. and it's not going to just go away and see it. i have a freedom there. but even the trick to live new antibiotics hitting the market is threatening to dry up. most big pharmaceutical companies shut down there around

t by allstate. research is like i hate public saying that a steel companies that brought out new antibiotics and recent years went bankrupt. but why? because sales remains as low as new antibiotics have to be kept as back up probably . in to pharmacists, the solution lies in so called incentive systems. other members of the association agree, see me and companies must have the expectation the, the launch risky investments. they may well one day pay off, and that's not the case at the moment. they gave him one incentive model for sees that kind of subscription plan. the found a company would pay for research and development if that lead to a successful new anti biotech. the state would pay the firm an annual premium for a guaranteed number of views and return the company would guarantee the availability of the antibiotic by the non lights of these kinds of incentive systems are an intervention and existing incentives desktops. so they have to be

examined very carefully, and new antibiotics are developed for the global market. development is very expensive. so the approach both for the development and for the incentive systems needs to be coordinated international layouts and not quoting yet the onset to which is why the experts from the association wants agreements that go beyond that countries. boat is even if they feel switzerland should lead the way the train me to me is not switzerland then who me and we have causing a research here and a very large pharmaceutical industry. see, we also have many small and medium sized companies already involved in the field. we have all the prerequisites. so let's take the lead to hold for a in the intensive care unit spots at the hospital, doctors and this is fine for the lives of that patients. and hope that they'll be able to continue counting on life saving antibiotics in the future.

people who just finished the course of antibiotics should wait for several days before donating blood. one reason is to help prevent the possibility of even more microbial resistance this week. fewer question also about blood comes from mooney. couple stuff in colombia. what is our blood may don't, and where is it formed? every adult has between 5 and 6 fleets as of blood because it enables all of the other important bodily functions. blood is sometimes called a liquid organ. almost half of it though, is made up of solid components. red blood cells give it the characteristic color. they supply the body with oxygen and transport gas vs carbon dioxide waste to the lungs to be excelled. the platelets on the other hand, play an important role in quilting and heating wounds, and the white blood cells are able to recognize and mode of pathogens. the cells

and platelets all suspended in will exclude blood plasma. this clea, a yellowish fluid is made up of water, nutrients, whole moons, minerals, and proceedings through a brown ching circulatory system that is almost 100000 kilometers long. the blood is distributed to the farthest reaches of the bodies. besides oxygen and c o 2, blood carries and distributes humans nutrients and also heats itself form and the so called red bone marrow and then link tissue. that itself has a plentiful blood supply. and adults blood is mostly produced in short, flat phones like the rims and stun them in children. formation of codes and bones all over the body. the red bone marrow is home to special stem cells, but the able to produce all the different types of blood cells.

the many blood cells live only a few days. they have to be replenished constantly. and by the way, the full bus flood is produced mainly in the live and spleen. and these organs can resume the task even in adults. if for some reason the red bone marrow is damaged, the red. why are they at now over to you? do you have a science question, then send it to us as a video, text or voice mail? if we answer it on the air, we'll send you a little surprise as a thank you. the immune system can sometimes play havoc and begin attacking the body's own tissues,

causing what doctors call auto immune diseases. lupus is one example left unchecked . it can cause symptoms like massive inflammation of the kidneys. and one university in germany, doctors have now developed a new therapy for treating it to tell who is visiting this clinic in southern germany for a follow up examination. she has to have a complete check out every 4 months. back in 2016 when she was 16 to tell contract that the severe auto immune disease systemic loop as a risk. my toes this or s l e, an athletic teenager at 1st she mistook the symptoms for sore muscles. doing main didn't get any better. it got worse when and then in december i developed extreme fatigue. i got more and more tired. with lupus, the immune system begins finding the body's own cells and attacks internal organs.

none of the attempts to treat to tell this condition were successful. in early 2021 . all the treatment options had been exhausted. when the have you at that point i just had to cry. yes. the disease wasn't getting any better. it was getting less. all i could do was lie down at home. i was in pain. i had breathing difficulties, hot palpitations. i wondered if i was going to die because that just wasn't to kill, couldn't have big doctors from the fema tomochichi and immunology departments at the university hospital long and for already researching a treatment for lupus patients employing t cells from the patient that are genetically engineered in the lab, at that point, the cell therapy had only been used in cancer treatment. it was offered to to tow as a last resort was to get him to any he had to have to admit. quite honestly, i was very worried that it might not work at all because we had no experience and no one had done it before us. and there was also the risk that it might make things

worse. the discounts apply to the for, for the month. this is how the therapy works. t cells are isolated from the patient's blood and fitted with what's called a sion merrick adage and receptor or car. it enables these modified tea cells to recognize and destroy misdirected cells. in march 2021 to tell became the world's 1st patient treated with s l e car t cells. since then, her condition has improved in domestic during this semester break. i had a vacation job and they told me that, that it was actually a man's job. my parents thought that was great because it was the 1st sign for them that i was healthy again. just as little as 5 more patients with life threatening lupus have since been successfully treated with experimental car t cell therapy. the next step is a clinical trial with more subjects, it could mark a milestone in treatment of auto immune diseases. and

that's it for this week. on tomorrow today, d w sign show. thanks for watching and see you next time. bye for now the . the

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the w the . this is dw news light from the land produced a load book, locals to step in to help an influx of migrants. the u. n. is urging european countries today more of a $1012.00 have arrived on the italian island. plus libya struggles with thousands of bodies left by catastrophic flooding. they being pulled from the sea, just some of the victims swept away off the dams passed near the city of diona to bob as the left to pricing the 1000000 possible task of cleaning up the mess

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