>> this weekend on "reel a 1960's special new series on the warren report anchored by walter concrete, investigating unanswered questions into john f. kennedy's assassination. 24, the mobovember scene continues as osborne is brought into the jail. televisionl sight of users, jack ruby searches through the crowd and shoots oswald dead. >> watch tonight on c-span3. joining us from boston this morning is antonio regalado, who is science editor for m.i.t.'s "technology review." thank you for joining us.

i went to start with this photo "peopleseen an open wil magazine" and elsewhere, the lanza family. are a familysmans in brooklyn. both of their sons were diagnosed with a rare disease, a ve disease,e ner ultimately fatal. they have one son diagnosed and also a newborn. theincredible thing about a fundingstarted campaign to get a new treatment for their family. they found scientists willing to get a shot, to get treatment for the family as soon as possible. quite aey have raised

bit of money so far, antonio, that we know. here is a portion of the appeal through gofundme. here is a brief look. [video clip] >> we are here in moraine park, brooklyn. we are overwhelmed because so much money has been raised on behalf of our twins. they had a rare disease, a degenerative disease where the children lose abilities that normal people should have come at the abilities to breathe, the ability to swallow, the ability to speak, any kind of motor function. >> we decided we were not going to just take what do doctors said, that we should go home and love them and make them, that we are going to find something, and when we searched, we found there was so much science and research

done already. because it is a new disease, there is just not enough money. to landsman: we are here bring about a cure for the disease, because when they hear canavan disease, it will be the first securing als. guest: they approached researchers working on the disease, will this work for our was yes, the answer but it would have to pay for it. gene therapy is arriving, and people, because they have big accounts or fundraising success

like the landsmans, are able to reach into the future and get a little bit about their kids. host: here is the front page of the m.i.t. "technology review" and your piece -- $2 million to save their life. could you pay? should you? medicine is becoming hyper personalized, hyper accurate, and hyper unequal. concept?xplain that where are we headed with this medicine? guest: the hyper personalized part is there comes a point in science and technology where someone with a rare disease, you know, they have a mutation in a gene that is causing a problem. it is not quite easy to diagnose those problems with gene sequencing, and the technology, gene therapy and other technologies, means you know the genetic error, there is no possibility of actually repairing it. specific.that is van is rare, but the way

science is going as hyper personalization, so in principle, they come payments, even for a signal -- single individual. is just the part incredible cost. not everybody is able to raise scientists to develop a treatment for their kids. for every landsman family, their there are 10 families, 20 families that are suffering and do not see a path for any kind of hope. i mean, they cannot gather the money selling cupcakes in big sales. part.s the when the drugs hit the market, they have a high cost, $1 million, $4 million for gene therapy, savannah question is how society will pay for it.

-- so then the question is how society will pay for it. folks kencan call in. (202) 748-8000 and (202) 748-8001. touch onegalado, you this in your piece, but let's expand on it. the problem is that who will pay? underlying the unheard of prices because of human resource, human research to get the fda to sign growing gapult is a that could be treated as gene therapy and those that actually are. guest: these are inherited

diseases, muscular dystrophy, can advanceerosis, canavan's- disease. a drug tot to bring market only treats 50 kids, the price will be really high. host: is the federal government active in terms of funding, research, moving this process along? jean barely a basic research has been funded by the government all along. national institute of health steps away once it comes to market. a companies are starting to invest. nobody has a solution really for this issue that the diseases are just really, really rare. represents a new

form boutique medicine, a way to get those with bad checkbooks or those with vital fundraising campaign special access to cutting edge, it is a preview of the personalized medicine that will be increasingly available more generally. tell us about what the future holds in this area, mr. regalado. that wasere was a case even more extreme here in boston, it was a family whose a disease called athens disease, and it truly was an individualized medicine. it was kind of a gene repair technology during that was a case where again the $1 million a drug for alop single child, that is where we are with the hyper or solicitation -- hyper personalization.

it is possible these treatments will be tailored for one person. what we're missing is a system to deliver the kind of medicine it cannot cost $1 million, $2 million, so the question going forward is how this new form of medicine is going to be organized. host: who is addressing that question? who is answering it? how are they addressing it at this point? guest: in our story, we interviewed a scientist named stephen gray at the university of texas southwestern, and he is one of the people addressing it. he has about 23 gene therapies in the pipeline. families come to him all the time with children, and he often takes on their case. so he has developed a kind of a pipeline of gene therapies to help these parents access the technology. but i talked to him, and he is not hopeful that a solution is in hand. again, just the people who can

pay and that can raise $2 million, $3 million are getting access to the treatments. as yet, there is no sort of solution to how to deliver these types of treatments more generally. host: we are looking to line up calls now for antonio regalado, writing in the m.i.t. "technology review." he writes that gene therapy is sort of in a golden age right now. explain the process of getting a gene therapy developed and approved by the fda. how long does it take typically? how much does it cost? guest: gene therapies are moving pretty quickly. the reason is that sometimes they can be extremely effective. a child can get a dose of a gene therapy, there is a disease called spinal muscular atrophy, and it is really kind of speeding up the approval process. in that case, they only treated about 15 children before they were almost ready to go to the

fda and ask permission to market it, something they have now done. so kind of comparison, treat 15 gives with gene therapy versus a save forlinical trial, a cancer drug, where you may see hundreds of thousands of people in a really long development time. it can be faster, but it is complex science. the gene therapies are delivered with science, bio particles, but the whole way to produce the gene therapy, sort cown bags of cowboy, is -- of located, but you make millions of particles, and if viruses go to the sales and basically deliver the new dna. we take a look at the calculus for the biotech companies and deciding which phases or which diseases are too rare to care, as you have written in your piece, and which

diseases are worth taking on. 4, 5 years ago, people looking at a disease like canavan's would just have ignored it because it was just too rare. if you have cancer, you can look online and find many studies of experimental drugs, but when you look at canavan disease, there is not a single one. that is how rare it is. no drug company would be interested in working on treatment in general because, as someone else in the article says, you and us in a company with a stack of 100 people that exists to treat 100 kids. the math does not add up. i mentioned the treatment is effective. this company that was developing the treatment for spinal muscular atrophy was acquired by novartis for $8 billion. so that open people's eyes.

maybe these diseases are not too rare. if we can find something that really works well, there might be a market for it. but the price is going to be superhigh come in the millions. host: with that background for our guest, antonio regalado, mic "technology review," we have todd on the line in kentucky. good morning. caller: good morning. i wanted to get your comment about the fact that people are willing to give $10 or $20 to help a couple with a child that has a unique genetic disease as a mechanism for funding the development of these treatments. it seems to me may be better than a government process. what do you think? guest: there is a lot to be said for it. in the case of the landsmans, they live in a tightknit jewish community in brooklyn. they really have a perfect storm of donors, people in their neighborhood, you know, the butcher, baker, that all came together to help them.

does not always work, so there will be another person, you can look online, there is a site told gofundme, and you can see lots of appeals for gene therapy, and someone raises $35. they will not get anywhere. i think it is true that when families involved, when they drive scientists forward, things can happen more quickly than under government funding. the families put i a fire really under researchers. they have a deadline. the clock is ticking. thanks can move a lot faster when families are involved. it is a model we definitely see. we found six cases of families that have actually gotten their children treated this wa, and then dozens more where the process is underway. host: there is the landsmans' page, the gofundme page, two children, they were looking for $1.5 million.

$1.6 million,out so they -- $1.26 million, so they are well on their way. joe is: from eastpointe, michigan. good morning. caller: good morning. box of pancake mix that i've been using for a while, and i was reading the ingredient list the other day, which i've never done. producedpartially through genetic engineering." what the hell are they doing to my flower, to my thinkin pancake mix genetically? i am too young to be poisoned. i am only 83. i want to get another 200, 300 years out of this. thank you. guest: it is a great question. 20 first century will be a century of genetic engineering. it is already in your pancakes, gmo's, biotech corn is

probably what you are talking about. it is well established. host: you talk about the ing for a lot of money on the website, but that is an unusual case, as he pointed out, a lot of people get money.rably less other people, you can imagine, might be able to flyfor this research if they are wealthy. besides a web, appeal or being flat out wealthy, our people raising money for this kind of treatment? guest: if you are going to wait for the government to get grants or scientists to be interested, you can be waiting a long time. even at the pace of sort of approval by the fda, it is going to take, like, 1000 years together all the rare diseases. people are definitely looking for ways to hurry it up. one of the parents told me, she kind of dream about asking bill

gates to help out. bill gates works on diseases that are problematic in malaria,g countries, public health questions, but this particular mother thought, if someone like bill gates would be able to set up an institute to develop the treatments, that can make a huge difference. we might see something like that pretty soon. host: let's hear from john in new jersey for antonio regalado. good morning. caller: good morning. i have been thinking about that issue for a long time. it is like defibrillators, they want to put on everybody's body and that sign of thing. i think it will end up on the courts, low income, immigrants, prisoners, they have a right to it, and the very rich will be able to get access with their money, movie stars and the billionaires like gates. it is just not enough money to extend everybody's life. i have never made a study, but

it does not seem reasonable to raise that much money for a tax system. i hope it is not that way, but i think that is the way it is going to end up. thank you. guest: i think it is a good point. you think about other countries. here is the united states, some of the treatments have been developed. they are very high-tech, available, paid for by insurance. the diseases affect people all over the world. in india, malaysia, and china. how are those countries going to pay for it? are they going to get the kind of cures, life i extensions that we have here? the caller raises another point of understanding people's lives. in the future, gene therapies might be used to help treat diseases, aging might be extended, the lifespan. that will be a whole new debate about who gets the. host: ethel is calling from

california. good morning. caller: good morning. i was told gmo is a typical products to digest in the digestive system, so how does that really affect? what are the long-term effects of that particular problem? back toou are getting the food questions, genetically modified food, not really the subject here, but we are talking about genetically modified people, which is quite interesting. therapy trials in the u.s., so you have people walking around whose genes have been modified. if we are ok with people getting their genes modified, you may not worry about so much what is in the pancakes. host: we have cody calling from la jolla, california. good morning. caller: good morning. thanks for hearing me. the idea that i'm having right now, i wonder if it is feasible

not, some kind of app or almost union, anyent's' range from $100,000 to $3 million to the lumped into a group and offered in the marketplace. is happening. a lot of these diseases have were numerous patients, numerous families come together to fund the research. what we are seeing are a family-led or individual-led attempts. there is a large tradition of large foundations of affected people funding research and diseases. the cystic fibrosis foundation has been very successful, you know. host: anything else? caller: that is it, thank you. guest: thank you. host: we will hear from barbara

from harrisburg, pennsylvania for antonio regalado. hi, barbara. hi.er: i wonder, because i have a son with schizophrenia, do i have any hope in thinking that perhaps gene therapy might be effective for that mental disorder? thank you very much. guest: it is a question. schizophrenia is what they call a complex disease. it does not have a unique genetic cause. in the case of gene therapy, they are aiming at diseases called matt mainly in diseases -- matt mainly in diseases. is one disease with an error. they are using gene therapy to replace after something like schizophrenia is much more organic. the cause is not known, there is no singular cause, and so that

makes it unlikely to be addressed by gene therapy in the near future. maybe in the distant future, it could be. talking with antonio regalado, biomedicine science editor at mic "technology review." -- at m.i.t. technology review. hear about this particular case we are talking about out of new york and others? what brought it to your attention? i heard the case, must the idea that it exist, because we have been writing about gene therapy and it's kind of scenario that i am talking about, the idea that you can go directly from a genetic cause to a genetic solution seems like something that has to be out there, and furthermore, i had questions about in the larger gene therapy trials, i have questions about who was getting in them, which patients were being selected, and i had the idea that patients were kind theired in, but had done research on google or the web,

they had diseases that had previously been untreatable, but if you are well-informed, you might vehicle to get into the trial. i am interested into the information arbitrage, so to speak, where people who put their feelers out can sort of get this advanced medicine before other people can. and then it became a matter of looking for a case that was kind of dramatic and showed how all of this was working, so i found the landsmans. i found a lot of other families doing the same thing. host: you write in the piece that some families, other families are avoiding the rigors of formal studies and trying to secure untested gene therapy as emergency treatment. you write about florida, a little boy with canavan was treated after his parents had an emergency treatment.

for patientsp whose life is immediately threatened. can you speak to? guest: when you think about the fda process for my new person with a new drug that has never been tested, you need the approval of the fda, and if it works, years later, they might ultimately let you market it. but first, you have to get approval for the study, and the drug testing and tends to treat 10 people or 30 people, 100 people. there is an exemption in the allowsules that individual doctors to try hail marys, you might call them, try another drug with someone who has no other option. it is used commonly in. gene therapy there are about 700 gene therapy trials in the u.s. andagency told me about 700 them are expanded access trials that can involve a single

person. that is a method people are using to pay to getting therapy now. host: let's get to daniel in pennsylvania. hey, daniel. caller: good morning. saying that we are actually socializing the development of these treatments, and then prioritizing the distribution. natural resourcesona where we use blood and treasure to get access and then privatizers distribution. am i correct in that assumption? thank you. host: thank you, daniel. guest: i thank you are. aul mentions the case in florida. there was one boy treated for canavan disease, due to extended access protocol, so the family paid more than $1.5 million.

it seems to have gone well. not cause any ill effects are suddenly, biotech companies are very interested in that and have formed a company to advance it. the national institutes of health, the government, families, pay to get this to the research where principles improve, and then private industry picks it up. some of the risk is taken out, vacated the rest of the way to the market, and deliver it to us once again with very high prices. the possibility in my mind in a totally different system where these drugs would be delivered more like a hospital service. you go to the hospital, the replace your hip, they do something like that, the costs are not in the millions usually. in the future, it could be that hospitals ought to be the ones

providing the therapy instead of the drug companies. host: we have one last call for our guests, joseph in willow springs, north carolina. hey, joseph. us?ph, are you with guest,houghts from our antonio regalado. what are you looking for next in this story and where it is headed? guest: i think the next step is to really dig into the cost. people are trying to model what some of the treatments will cost and reach the market. i mentioned one gene therapy that cost $4 million. a lot of people are working on that question, the economics of that. tackle aresue i will just the prices there are some promising gene therapies out there. muscular dystrophy is a pretty common disease. it is fatal. if it were cured with gene therapy, i think society would then struggle with the true

cost. if it ends up costing $2 million, $3 million, it will add up. host: antonio regalado is the biomedicine senior editor at m.i.t. "technology >> "washington journal" live every day with news and policy issues that impact you. sunday morning we will talk about the agenda for democrats when they take over in january and potential democratic challengers to president trump in 2020. joining us as a guest from the center for american progress and the national reviews columnists discusses the republican party. be sure to watch c-span "washington journal" live at 7 :00 eastern sunday morning. join the discussion. >> the