The ice bucket challenge in 2014 people all over the world were pouring ice water over their heads in a bid to focus more medical research on the rare motor neuron disease a less lethal they posted the videos on social networks and raised millions in private donations scientists took no theyd seen how their laboratories and the public could be just a click apart. What would you rather we did next tuesday contests i think we can now invent new drugs faster if in the kind i believe that they so much more than we can deal. With why public support medical researchers can tackle diseases at the pharmaceutical industry overlooks. Leap. Live. In the land of social media cats rule one of their stars is lil bub she looks a bit unique but she was exactly what 2 molecular biologist from berlin were searching for. Now for the 1st time will be able to understand all the secrets. Even you know what. Scientists daniel d. Bring him in a jar a look here at this work for the Max Planck Institute for molecular genetics. And in. One night i was on the internet on going this cat documentary came up in my recommendation and since one of the cats they showed was a little bug and they said she has 6 toes which was interesting to me but ive seen that before and then they said the cats also small because shes got weird bones which means this cat has both a limb and the bone malformation its i thought this cant be happening it doesnt come. This is precisely the 2 scientists specialty they deal with hereditary diseases. And its. The next day i went to daria and said you have to see this cat you wont believe what shes got of we knew that there are genetic mutations that cause limit from actions and also mutations that cause bone malformations but ive never seen a combination of the 2 so i figured the best thing to do was to sequence that which is our specialty it was totally amazing that something we normally do in our institute which makes most of our friends yawn was related to something that interests millions of people and you mentioned. They set out to decipher the famous catch genome but Laboratory Analysis is expensive so the 2 researchers moved to make use of little bubs high profile in the tiny town of bloomington indiana. With over 200000 you tube subscribers and 3000000 likes on facebook the feline reaches a vast number of people. Social celebrity we wanted to do a broad not only in both us but kind of get engaged also the fans or people like tom i mean that is the in understanding what is behind the said time features that neither of us. Will bubs own or microsofts he was thrilled for him his cat stardom was never an end in itself. When she started getting favors i mean its a pretty strict rules were hearing if were going to do this that there should be some sort of positive message attach where there is are you know reached out. As 3 exciting. The berlin researchers launched a Crowdfunding Campaign in 2015 crowdfunding draws on the vast number of people using the internet. The caps fans were able to donate small sums to the crowdfunding platform the scientists needed a total of 6500. 00 to cover part of the research costs. But people donated even more and in the end it brought in over 8200. 00. Now the Real Research could begin. So normally what happens you know because. You basically work in the lot for i think 2 or 3 years and then you keep your results and theyre not the same then you basically release all your results we can reform the 2nd belief in experience because 1st you can get engaged socially with the people that theme that actually your Research Interest and then doing the entire process you can communicate with them. The money for a little bubs genome decryption came together within a month mike sent a sample of the cats blood to berlin abraham and his colleagues captured the most. Meant for the donors. To find anywhere sequencing you know. Staying in touch with the public was a completely new task for the researchers suddenly they themselves were the focus of an audience but it also gave them an opportunity to make science more transparent. We are going to try and do on this done follow how they do you know functional and hollow and durations in the genome can lead to human disease. The analysis found that little club was suffering from an unusual accumulation of genetic mutations. Osteo patro cysts a rare bone disease where the bone tissue becomes considerably more dense was the most obvious she also had polly dr lee hands her 6 claws. As humans can be born with surplus fingers and toes as well little bugs genome could help understand these rare hereditary diseases better in the future. As it comes on crowd funding could be a good way to enable research into a particular rare disease because that doesnt happen a lot otherwise funding agencies give a lot of money to medical research but its often difficult to get money for Rare Diseases off me so im for. Crowd funding as a way of enabling research that might otherwise not happen. But ill show you that the bus house hes home edition that nobody has discovered before in the study of the said machine gun. Information that we had not seen previously before. All those disarranging. New forms of Research Funding are particularly needed when no one else is investing. Hardly anyone knows this better than the people that access the alliance of chronic Rare Diseases were meeting christina moment loss. With the voice of those affected by red diseases. That affect some 4000000 people in germany alone but few of them suffer from the same rare disease. This is in american terms its normally a few 1000 people but it can also be only a handful of patients worldwide get. Patients like josefina schultz whos 10 years old today she was born with a swellings and a heart defect. Her parents went from doctor to doctor but no one to give them an answer it took 18 months to get a diagnosis josefina has a rare genetic disorder williams syndrome but she still has a normal Life Expectancy for not is it if youre affected by red izzys it means 1st and foremost that you have to search parents tell us what an enormous burden it is for them to know that something is wrong with their child and not get an answer as to what it means and where it leads the way with words when. The pharmaceutical industry focuses on the development of therapies for common diseases secrets from the association of Research Based pharmaceutical Companies Says this is the right approach. For now nobody is responsible for basic research we have a total of 30000. 00 diseases 628000 of them are rare in view of that i think its obvious that the pharmaceutical industry cannot tackle all diseases at the same time. Con Research Spending by governments in the pharmaceutical sector has been rising for years there are a must to achieve the e. U. s lisbon target of 3 percent of Gross Domestic Product germany austria and switzerland together and. Asked about 120000000000 euros in research and development every year. However those affected by Rare Diseases do not automatically benefit the pharmaceutical industry applies a simple formula. You know if you history class how common or how rare is the disease. Is there any basic research that shows how it could be treated. Or can and can we expect research into it to pay off at some point. But i think. I was taught. Many people affected by Rare Diseases the hope for new drugs for gene therapy yes but its a complex process scientists must 1st find and develop an active substance then they tested on animals so as not to endanger humans. These studies alone often take years. And then when all the trials have gone well if you go into Clinical Trials that is into research with actual patients initially Healthy Volunteers will be thinking. Of around 10000. 00 active substances evaluated in Preclinical Research only about 5 actually get as far as Clinical Trials active ingredients are tested in 3 phases often on many thousands of patients and the drug will only be approved if the Clinical Trial goes off without a hitch despite the whole thing takes about 10 to 13 years and cost several 100000000. 00 you want to know pharmaceutical Companies Prefer to invest in projects with high profit expectations Rare Diseases are not included theyre often complex and unprofitable christian among us see the challenge it isnt the same congo we know that red is easons can only be treated in an interdisciplinary and cross sectoral way. That means networking is crucial on this the digital revenue. Of course supports us on this. And digital techniques are incredibly good tools for his school to see his middle. But where the market shows no interest the state and our society can step in with donations if necessary. It worked with the ice bucket challenge at least 140000000. 00 in donations were collected worldwide and a year later the researchers could actually report a success they were able to replace the damaged protein ls with a synthetic one in the laboratory thus preventing the death of affected cells but still dozens of the new cars on the we have to be aware that this probably wont happen again in that way and we also have to be aware that the people who made the donations didnt necessarily learn a lot about the disease but learned carved by only enthusiastic celebrities tipping ice cold water over their heads and the following it attracted you. Is i. Wasnt to follow what. How big does the crowd have to be. Many followers does it take to tackle the thousands of Rare Diseases why does the money. When we have the tools to help. Romania or tease founded the global aids organization rare for nomics in 2012. It helps patients with Rare Diseases to finance genome analysis. Yes mean fun for a queen and her brother are 2 of them. D. N. A. Analysis showed that the siblings had different gene defects that required completely different therapies. It is very important to people i mean really has over 80 percent agree diseases are known to be genetic in origin. Comprehensive Health Insurance is a luxury in the us some families have gone bankrupt trying to treat Rare Diseases. I work directly with families to actually talk to the Insurance Companies to act ask for Insurance Coverage and every time youre early on by. Oh its experimental this experimental test so we cant cover it and we started helping any families that we would offer that as an option if they could not for the test so we theyre also trying to do Insurance Coverage and that didnt work we say well well help you have funding youre willing to do that. Mix a companys patients from the Crowdfunding Campaign through analysis of the results to possible therapies. Ive seen just in the past i would say for years alone there has been a big increase in interest in Rare Diseases and in pharma the more research that is done the more findings you know whole new mutations associated with disease the more people will be interested in. Networking and crowdfunding are useful tools for people suffering from Rare Diseases. But theres also a need for political oversight. And its incentives have now been created at the e. U. Level to encourage the pharmaceutical industry to tackle red diseases and also to see the appeal in developing drugs for them to come and its because. These incentives for the pharmaceutical industry include simplified approval procedures and better patent protection at the same time the costs of d. N. A. Analysis have fallen dramatically when the human genome project was launched in 1900 it was the 1st time that all 3000000000 base pairs of the human genome have been identified and mapped. The project took 13 hears and cost 3000000000. 00 today you can have your genome sequenced in a single day for about 1000. 00 and the price continues to fall. The highest the drop in the cost of sequencing means that you can suddenly do anything you can think to do. The fortunate come. But what impact does having to 1st court the broad public via the internet have on research. What sort of projects stand a chance. Were flying to london where british researchers were the 1st to lay bare the impact of l. S. D. On the brain using modern scans. The proposal was an online crowd pleaser instead of the target of 25000. 00 pounds the Crowdfunding Campaign brought in more than twice as much. I guys you know. David knott is a psychiatrist at Imperial College in london by steady brain out of the influence of l. S. D. He wanted to create the 1st precise imaging study of the drugs effects for medical purposes his priority is the treatment of depression. Depression is the largest schools of disability in the western world including germany because the clintons not very Good Condition starts young continues all ones life and so its very expensive but weve made so little progress in the last 20 years that most pharmaceutical companies have given up and that means weve is now up to us the scientists and technicians to take up the challenge. Not is focusing his research on psychoactive substances its a difficult but he thinks promising field of research. Classic antidepressants like prozac they increase a return in the brain and they protection from stress they dampen down the stress responses so eventually you know you can cope with the stress of life and the psychedelic play kind of reset the brain so that you can overcome the stress rather than protect yourself against. The w. H. O. Classified l. S. D. Is highly dangerous which means no public funding for research into the substance is available. It is not a medicine theres only one class and that class is the most harmful and once youre in that class its very hard to work with. The money from the crowd funding camp david complete his imaging study subjects were given l. S. D. And put into the brain scanner with unexpected results. We found exactly the opposite of what we predicted we thought people having a trip on magic mushrooms or as the seeing all sorts of wonderful lights images you know theyre traveling through space and going to magic mountains and things we thought their brain will be activated its not switched off. Because these drugs disconnect you from the outside world youre seeing the inner workings of your mind rather than the constraints is put on your mind. And thats why they work in depression because they switch off the circuit of depression to people write to me every day almost saying can i have the treatment i say no because were not allowed to give you the treatment because its illegal to. Further studies are needed but the British Medical Research council which is responsible for Research Funding is still skeptical money from the state for research into class a drugs its unthinkable. There are 2 kinds of harms a drug can do theres a harm the drug can do to the people who use it and the harm that drugs can do to society as a result of people using it. And when you look at societal harms way way above Everything Else at the top there and then. Psychedelics have no societal harm they may even have benefit because they have different. Research against the mainstream is exhausting because state support programs deliberately and quite rightly contain control mechanisms ethics committees constantly reassess the benefits of the general public and the dangerous of the test subjects but david not thinks crowdfunding is also important for another reason. Hes part of a bigger profile of communicating science and also communicating the obstructions to science thats actually critical because were in this stupid position of having banned drugs for 50 years because the traditional media refuse to think differently in social media youve got much more opportunities to communicate the truth to people so so i see crowdfunding as part of a larger public. Communication strategy where we get people to understand that their families are being disadvantaged because the law doesnt allow the use of it but. If the state of the pharmaceutical industry dont support it can the crowd really step in and even Impact Health policy will medical research become like grassroots democracy in the future. Where it seems to be more easy to mobilize people for the Rights Research was a bit different in my case. Were visiting christopher rom and stockholm hes also been trying to obtain funding for his Research Topic 1st from conventional bodies then through crowd funding hes a psychiatrist at carolyns institute and wants to study the treatment of pedophilia. Tried the traditional way. Many many times but i was declined all the time there are people reading this application maybe some of them have personal opinions on the topic there is not in my backyard syndrome around this. There are issues nobody wants to know about although theres a real need for action. The overarching aim of the study is to find ways of reducing the number of child sexual abuse is by establishing an evidence based scientifically proved. Method to treat adult men with pedophilia disorder some studies indicate. Adult population one to 3 percent have sexual fantasies a moment children almost one in 10 girls. Sexual abuse during childhood and one in 20 boys and in almost 50 percent of the cases it is done by someone with a pedophile it is a wonder. Around wants to find out whether a Prostate Cancer drug can also preventative lee help pedophiles by inhibiting the bodys own to start their own production. Looked on the br