right, and 60 minutes w version now understand can have a site like the right to present data used on instagram. follow the take the measurements, choose the fabric, sketch cuts. so it's made to measure one of a kind 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 dw sign show. welcome

to the program. boy. raz is 6 years old. at 1st glance, he's a boy who's developed normally 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 since cause normal van, hey, what's going on? he was just like, there's $200.00 plus orthodontics like that. um, either shocked with the news and very upset actually. so doctors just told us that there is 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. our son 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 porras from head to toe and 0 in on his symptoms. so it's pointed on know, here most of the day on now he has expected his legs

are unstable, but something else is also striking. less one disease. what by so is that 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 anything. it's also very difficult for him to remain still in one place without making compensate to remove minutes. ringback well, now it's nice to see a neurologist mount, a synopsis, a member of the research team tracking poor arises. 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 kentucky. the doctors can to

report rise, all right, 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 poly rose's 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 mean 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 arises 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, v a 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 gonna book all the discover, the therapy that porter has received in tubing and is not approved, but it says only chance, this makes it all the more important to really measure test and compare everything, all of my, of my signal go back there stick it on and return on sweet. 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 and damage and these are 19, 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. 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 we know that this depends on the, on the various other diseases. so think that is a good chest, that it's kind of

a cute as well, and 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, boy reza cells are absorbing d r n a therapy. but the doctors here want to do even more than just health. their young patient. they already have more r n, a snippets in their collection that might help others with similar gene dfcs. the same or in a kit can be used to tailor drugs to specific patients. it's still, it's still a way off, but why we treat one patient. we're already developing 2nd and 3rd or any 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 point 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. i imagine says log is a disease and imagine this case is a cure opening to lock me is finding the right treatment. but here's the cash you're not faced with. just one key means yes of them.

now, you couldn't be trying one key of the time. just kind of when we search as of the down, up until now, or you can predict which works for like using i think the something that is a, i has the power to massively accelerate the discovery of a treatment, a drug discovery company di power tools to accelerate, drug discovery sciences 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. agent can be targeted, aging can be delayed and aging in certain ways can be stopped and reversed also. so what exactly is a drug included help us live longer on

this thing is utilizing a i, i said to the console in by a many problems with a lie and you can decrease the probability of success and also make of cheaper and faster drug discovery consists in finding new medications to treat diseases. in this case we're focusing on dizziness costs like proteins by goes to a tried is marcusson's or a timer disease. and because the process is partially repetitive, it can be partially ultimatum. the computer to agents of this quality process is slow noon. it's something that has to be try and for many decades before but a goes beyond automation. it predicts, it looks like good candidates, understand some of the features and the predicts either good candidates. you could also take it a step further. imagine that you can you 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 and 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 that most of us don't even consider a disease age. what is aging? why do we age the 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 the aging. businesses nearby is a lie, a groundbreaking research is in the science. so phones have it to one 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 cells that are already fusing only takes a 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 foster and they, vanessa a team and they come what may be the most promising similarly they've ever seen. so willie, 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 a lab chamberlain, we meet with david machine for a peek inside who's allowed him to let the honey seen walk us through the main stages of john x. and you don't know what most of monic yous are doing. yeah, maybe just a few falls of my, if you

a say actually know what they're doing and that's and on the human body. but there are many, many more money kits available. the 1st step is to decide which monitor is the best if this test many, many comb funds this much as possible to see if there would be access on 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 with ricky. now from sexual transfer. except this other machine uses a powerful microscope to capture the 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 they test the full series of images and the human couldn't possibly analyze all of this into the system. basically, where the machine on and can step in the network, scanned the images to find a good candidate for festival. here we have a lot of onto the 2 agents which checklist to grab a bite for another molecule where we don't know actually what kind of pharmacologic activity and see a test. and we see that it's really close to the 2 agents into space mcclendon the us, and maybe i think this is, that's what us and a s for an intimate to a tablet prediction a meg still needs to be tested. if anything, the tow tasks come from the predictions made by i, there are still many steps necessary to ensure that drug is safe and effective. then once we go to markets and extra sets of costs,

then you go to the kinetic $5.00 clinical trials. take a long time and there's a little way i can do to speed up that part of the process. the ai is a disruptive technology, not a functional fuse 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. we are also wanting to plan for set them to and due to related diseases, they're going to be sent on these dates available for patients to take in the next 10 years. they, 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 by malaria. an intensive care unit at a hospital in the switch city of them. 2 patients here are infected with antibiotic resistant bacteria, pathogens that coming onto politics and no longer able to kill the stuff where can protective clothing and keep the effect to patients isolated to prevent any further transmission. the head of the infectious diseases department, christina, too, and have discuss his treatment options with team. it does have a 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. then 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 from the bottom of the for to see what would that mean for us, but the patients would stop dying again from bacterial infections that to date have been considered treatable. x. but stroke of assigning pandemic one that's creeping up on us and growing big 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 direct to bob republic. since the issue is clear, the process of data fall, no question. we need to study flow of new and t bios. it says that vital and the problem is guessing why all the time. and it's not going to just go away it. i have a freedom, but even the trickle of new antibiotics hitting the market is threatening to dry up . most big pharmaceutical companies shut down their onto bios research is like i

hate public say and damage to your 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 positive into pharmacists. the solution lies in so called incentive systems. other members of the association agree fumey and companies must have the expectation the, the launch risky investments. they may well one, the payoff alone, and that's not the case at the moment, gave them one incentive model for c's. i kind of subscription plan. the firm a company would pay for research and development if that lead to a successful new anti biotech to state would pay the firm and annual premium for a guaranteed number of years. in return, the company would guarantee the availability of the antibiotic by the non whites as 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 marketing tool. 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 ones agreements that go beyond that countries. boat is even if they feel switzerland should lead the way pregnant if not, so that so and then who made and we have cussing at 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 and 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 made of n? where is it formed? every adult has between 5 and st. cleats as 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 ways to the lungs, to be excelled. the platelets on the other hand, play an important role in quilting and heating loons. and the white blood cells are

able to recognize and mode of pathogens the cells and platelets all suspended in what's called blood plasma. this clea, a yellowish fluid is made up of russia, 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 buildings. 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 loans like the ribs 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 and melissa 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 that 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. at 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 checkup every 4 months. back in 2016 when she was 16 to tell contract that the severe auto immune disease systemic lupus risk my toes this or s l e, and athletic teenager. at 1st, she mistook the symptoms for sore muscles to collections, doing main didn't get any better, it got less when and then in december i developed extreme fatigue. i got more and more tired with lupus be 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. 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, could hire 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 tell as a last resort, only has to be a month and 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, those kinds of like took off 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, but 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, because of the 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 and treatment of auto immune diseases.

and that's it for this week on tomorrow. today dw science show. thanks for watching and see you next time. bye for now, the, [000:00:00;00]

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