The ice bucket challenge in 2014 people all over the world record ice water over their heads in a bid to focus more medical research on the rare motor neuron disease a unless. They posted the videos on social networks and raised millions in private donations scientists took note theyve seen how their laboratories and the public could be just a click apart. What would you rather we didnt just think of us i think we can now invent new drugs faster i thin than kind i believe that for much more than we can deal. With why public support medical researchers can tackle diseases at the pharmaceutical industry overlooks. In the land of social media cats rule one of their stars is little but 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. The. Scientists daniel to bring him in a jar look here at this work for the Max Planck Institute for molecular genetics. And in. One night i was on the internet and this cat documentary came up in my recommendations and 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 limited formation and a 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 going to 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 but 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 the yellow. Lab. 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 broadway that not only us but kind of get engaged also. Or people a common interest in understanding what is behind the certain features that neither of us. Will bubs own or microdots 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. 3 x. The berlin researchers launched a Crowdfunding Campaign in 2015 crowdfunding draws on the vast number of people using the internet. The cats 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 to bring in. So normally what happens you know because scientific. You basically work in the lot for i dont know 2 or 3 years and then you keep your results on theyre not the same then you basically release all your results we can from these are completely different 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 downers. To find anywhere secret thing that you know you know about. Staying in touch with the public was a completely new task for the researchers who suddenly they themselves were the focus of an audience but it also gave them an opportunity to make science more transparent. We are basically trying to understand how how did you know function on and how. The regime did you know can lead to human disease. The analysis found that little bug was suffering from an unusual accumulation of genetic mutations. Osteo which rose its a rare bone disease where the bone tissue becomes considerably more dense was the most obvious she also had poly dr lee hence 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. Because this comes on crowdfunding 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 of me so im. Crowdfunding as a way of enabling research that might otherwise not happen. But actually the bus husky some addition that nobody has discovered before in the study of the machine gun. On information that we have not seen previously before in military mother moved to change. New forms of Research Funding are particularly needed when no one else is investing. Hardly anyone knows this better than the people at the alliance of chronic Rare Diseases were meeting christine im going to 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. Doesnt in american terms thats 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 swellings and a heart defect. Her parents want from doctor to doctor but no one can 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 when i visit 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 to i didnt want him here at. The pharmaceutical industry focuses on the development of therapies for common diseases secrets home 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. On Research Spending by governments in the pharmaceutical sector has been rising for years there aim is to achieve the 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. View i think 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 a hope for new drugs or 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. The 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 5 if you can. Of around 10000. 00 active substances evaluated increase Clinical Research only about 5 actually get as far as Clinical Trials active ingredients are tested in 3 faces off and on many thousands of patients and the drug will only be approved if the Clinical Trial goes off without a hitch thats got the whole thing takes about 10 to 13 years and cost several 100000000. 00 a year one dollar off the pharmaceutical Companies Prefer to invest in projects with high profit expectations Rare Diseases are not included theyre often complex and unprofitable christian among us sees the challenge of this in the same Congress Know that red is easons can only be treated in an interdisciplinary and cross sectoral way. That means networking is crucial unless the digital revenue. Of course supports us in this. Digital techniques are incredibly good tools and his crew does his middle. But where the market shows no interest in the state and now 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 no sense of the new clothes on the sofa 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 learn carved by only enthusiastic celebrities tipping ice cold water over their heads and the following it attract it. Is i suppose when it comes to that time of the so listen to follow what. How big does the crowd have to be. How many followers is it take to tackle the thousands of Rare Diseases why does the money. When we have the tool to help. Romania or tease founded the global aids organization rares or nomics in 2012. It helps patients with Rare Diseases to finance genome analysis. Yes mean fun brooklyn 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. I mean reagan has over 80 percent of gray 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 Insurance Companies to act ask for Insurance Coverage and every time here 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 if thats not work we say well well help you how 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 hes 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 ranges he says and also to see the appeal in developing drugs for them so you can. 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. And the prize the drop in the cost of sequencing means that you can suddenly do anything you can think to do. Im going to force an income. But what impact this 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 Crowdfunding Campaign brought in more than twice as much. I guys. David knott is a psychiatrist at Imperial College in london by scanning the 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 into germany because the clintons are 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 we its now up to us as scientists and technicians to take up talent. Not is focusing his research on psychoactive substances its a difficult but he thinks promising field of research. Classic antidepressants like prozac they increase 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 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 can help 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 saying 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 its stuff 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 2 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. 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 there are harms that drugs can do to society as a result of people using it. When you look at societal harms way way above Everything Else at the top there and then. Its just so psychedelics have no society may even have benefits 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 dangers to the test subjects but david not thinks crowdfunding is also important for another reason. Just 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 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. Communications strategy where we get people to understand that their families are being disadvantaged because the law doesnt allow the use of the phone. If they stayed in the pharmaceutical industry dont support it in the crowd really step in and even Impact Health policy will medical research become like grassroots democracy in the future. But it seems to be more easier to mobilize people for the other Rights Research was a bit different in my case. Were visiting christopher rom in stockholm hes also been trying to obtain funding for his Research Topic 1st from conventional bodies then through crowd funding hes a psychiatrist at the carolyn institute and wants to study the treatment of pedophilia. Tried the traditional way. Many many times but i was to fly in all the time the 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 start this indicate. Adult population one to 3 percent have sexual fantasies and more than children almost one in 10 girls have been sexually abused 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. Wants to find out whether a Prostat