" One orange." " One orange." "Chungwa moja." "Chungwa moja." "Two oranges." "Two oranges." "Machungwa mawili." "Machungwa mawili." "One pineapple." "One pineapple." "Mananasi moja." "[Speaking African Language]" "Mm-hmm." "[Speaking African Language, Loud Chatter]" "Omouri!" "Omouri!" "[Laughing and Cheering]" "[Omouri Laughing]" "Here he is, the cleverest boy in the world." "[Laughing] Hi, Mom." "And what's this?" "It's, uh, it's a picture." "Oh, my goodness." "And, uh, there's the Comoros." "Of course, it is." "Mrs. Odone, may I please have a word?" "Very skillful, sweetheart." "And, uh, Jupiter." "Jupiter, next to the Comoros?" "I'll be right there, Lorenzo." "Yes?" "Okay, Mommy." "Is Lorenzo having problems at home?" "Not that I know of." "He suddenly started throwing paints around." "He went wild destroying other children's paintings." "Surely he must have been provoked." "Came out of the blue." "He's normally a terrific kid." "How were they annoying you?" "Were they teasing you?" "No." "Were they touching your paints?" "No." "How were they annoying you?" "They just were." "Doing what?" "Things." "What kind of things?" "Things that make me feel annoyed." "[School Bell Rings]" "Mrs. Odone." "Another incident?" "Yes." "Today it was much worse." "Mrs. Odone, is there some trouble at home?" "Why do you assume the trouble's at home?" "There's nothing here... that explains his behavior." "It's... disturbed." "[Italian Opera] [Singing Along]" "[Skipped item nr. 43]" "[Muttering] [Screaming]" "[Aggravated Screaming]" "[Child Yelling]" "[Screaming]" "He does present as a hyperactive." "Hyperactives repeat tasks endlessly, don't they?" "They perseverate, yes." "Lorenzo's activities all have a beginning, middle and end." "He's learned three languages." "How could he be hyperactive?" "[Laughing] [Indistinct Conversation]" "Gifted children can also be disturbed." "Lorenzo needs to be referred to our I DP committee." "What's that?" "For what?" "Individual disability placement." "Lorenzo needs to be in a Special Ed class." "Any Special Ed our son needs will be provided at home." "[Mrs. Odone] Anyhow, it was snowing... and he began to rub my hands to warm them." "Uh-oh." "No, no." "He then produced photos of his wife and children." "Oh, that old game." "Oh, Deirdre." "Had I known this was the man I was going to wait 10 years for," "I'd have let you pick up the check." "[Mr. Odone] I did pick it up." "[Indistinct Chatter]" "[Bell Ringing] [Man] Please keep your seats." "[Mrs. Odone] I'm sorry, but I..." "I even left the tip." "[Mrs. Odone Laughing] Look at him, Mother." "[Woman] That's not fair." "[Laughter]" "[Door Opens]" "Lorenzo's fallen off his bike... and he is really bleeding." "Shh." "That's a boy." "Oh, such a brave boy." "[Woman] Doin'just fine here." "[Mr. Odone] You're going to be okay." "It's Christmas day, you know." "All across the country, little boys are trying out their new bikes." "Hey, champ, you better tell your folks to lighten up." "What are they gonna do when you're playing nose tackle for the Redskins?" "[Church Choir Music]" "[Skipped item nr. 86]" "[Panting]" "[Skipped item nr. 88]" "The E.E.G. Is normal, the skull X-ray is normal... and the CT scan is normal." "I don't know what to tell you." "This boy is neurologically intact." "Doctor, we spent three years in East Africa..." "In the Comoros." "We were wondering i..." "If he might have picked up some rare parasite." "It's possible." "[Loud Classical Music]" "Lorenzo, what are you doing?" "Lorenzo!" "[Music Stops]" "Sweetie?" "Mm-hmm?" "Can you hear Mommy?" "This, uh, may be an auditory processing difficulty." "The ears hear, all right, but the brain has trouble listening." "So... this means something is affecting his brain;" "but what, a tumor?" "Multiple sclerosis?" "It could be any one of a dozen things." "Listen, my friend," "I'd like you to stay in the hospital for three days." "Your mom and dad can come too." "We're gonna run some tests and get to the bottom of this." "[Doctor] Can you hear this sound?" "Focus right on that spot for me." "That helps me out." "All right, Lorenzo, close your eyes." "[Woman] Keep your eyes closed, buddy." "Stay nice and still." "[Opera Music]" "[Mrs. Odone] Easter festivals existed long before BabyJesus, back to pagan times when they marked the beginning of spring." "And the egg tells us... that the earth is gonna be reborn, like a baby chick." "Mrs. Odone, Mr. Odone," "Dr. Judalon is ready now." "Hey, Lorenzo, would you like to go feed the goldfish?" "It's all right, sweetheart, go ahead." "We'll be right back in a few minutes." "Go ahead." "[Doctor] This way, please." "Please have a seat." "[Air Conditioner Rattling]" "Well, we certainly don't need that noise, do we?" "So." "Please, Doctor, without equivocation." "There is a family of diseases that's quite rare. ;" "the leukodystrophies." "Lorenzo has one of them." "It's called A.L.D." "What A.L.D. Is is an inborn error of metabolism... that causes a degeneration of the brain." "It only affects males, usually between the ages of five and ten." "Its progress is relentless." "The end is inevitable." "All boys with A.L.D. Die, usually within two years of diagnosis." "And there are no exceptions?" "I am so sorry." "Are you absolutely sure?" "Yes." "Lorenzo shows the definitive sign." "He has an abnormally high level of fat in his blood, certain very long-chain saturated fats." "Uh... these fats, um, destroy his brain?" "But, but how?" "Well, there is an enzyme that should metabolize these fats, but in A.L.D. Boys, it's defective;" "so, they collect in the nerve cells, a little bit like cholesterol in the arteries." "And... in some way, this liquefies the white matter of the brain." ""Some way liquefies"?" "Could you be more specific?" "Well, to be honest, we're not quite certain just how it works." "You know what myelin is?" "Myelin is the fatty sheath that insulates the nerves." "It's a little like plastic around electrical wires." "Without it, the nerves cannot conduct an impulse." "What A.L.D. Does is strip away the myelin." "It corrodes it, if you like." "This causes degeneration of the brain." "As the brain degenerates, the body loses its functions." "But surely someone somewhere must be working on this, no?" "We would go anywhere, mm-hmm." "Yes." "The problem is ten years ago this disease hadn't even been identified." "We're still trying to understand just what it is." "I would like to offer you some hope, but..." "Then there..." "there's absolutely no treatment?" "Normally, at this point, we try to be constructive, we try to focus on what can be done, but in this case..." "[Child Speaking and Laughing]" "When we get home, can we read a story?" "Of course, my darling." "Lorenzino, let's go home." "[Quiet Sobbing]" "[Moaning]" "[Crying]" "[Groaning]" "[Phone Rings]" "[Woman on Intercom] Mr. Odone, I have a Dr. Judalon on line three." "Good." "Put him through." "They're waiting for you in the finance committee meeting." "Thank you." "Hello?" "[Dr. Judalon] Augusto, I just got off the phone... to Gus Nikolais, Professor of Neurology... at the Institute of Childhood Diseases here in Washington." "If there is a so-called world expert on the leukodystrophies, well, Nikolais would be it." "Ah, good." "I don't want to raise any false hopes, but he's working on an experimental protocol based on a diet." "Wonderful." "[Panting, Labored Breathing]" "Peanut butter, red meat, cheese..." "Unpeeled fruits, spinach, olive oil." "But these are healthy foods." "But they contain very long-chain saturated fats." "L..." "I still do not understand why they are so harmful to Lorenzo, why they're destructive to his brain." "How can spinach and peanut butter make the difference?" "The body needs these fats in order to create cells." "The excess is burned off;" "that's normally, of course." "In an A.L.D. Boy, these saturates can't be broken down... and they build up in the brain." "Somehow this buildup strips the myelin covering?" "Yes, exactly." "For the moment, we don't know why this is." "But we feel that by withdrawing the saturated fats from the diet, we will prevent them accumulating in the brain." " And if we could?" " There's no way to reverse neurological damage." "All we can hope for... is to slow the cascade of symptoms." "If it's any consolation, you'll be helping us understand the biochemistry of this... heartless disease." "So, can we enroll Lorenzo in our trial?" "I suppose so." "Good." "Then I will pass you over to our dietician... and meanwhile, we should arrange some genetic counseling." "Dr. Nikolais, it's highly unlikely that we will have another child." "No, but your sisters and their daughters may all be carriers." "We need to test them." "Only my family?" "What about his?" "No, no." "A.L.D. Is passed only through the mother." "But I thought you understood all this." "We, uh, we knew it was inborn." "We assumed that meant a combination of our genes." "That's true in other inherited diseases, but A.L.D. Is only carried on the female chromosome." "Excuse me." "Are you saying that Lorenzo got this directly from me?" "Well, in the sense that A.L.D. Is sex-linked, yes." "It goes from mother to son." "Then how did I get it?" "The woman gets it from her mother." "If I inherited the defect, why don't I have the disease?" "No, the woman is only the carrier, nothing else, but with each conception, she has a 50-50 chance of passing on the defect." "And when that happens, it's the cruelest kind of genetic lottery." "Lottery?" "It's a clumsy word." "I use it because it's..." "It's all arbitrary." "No one is to blame." "Um, if Michaela's mother had this, and her mother's mother, why was there no warning?" "I suppose if you went back into the family tree, you'd..." "But remember, a short time ago... this disease didn't even have a name." "Mrs. Odone, you have enough to put up with without..." "You have nothing to blame yourself for." "Thank you." "[Woman Over Phone] I don't think it's a good idea." "So she doesn't want me." "What about mother?" "She's packed and ready." "Don't you get it?" "Michaela doesn't want anybody around." "Well, what about Father Killian?" "Hell no." "She's not even going to mass now." "What is happening there, please?" "Let it rest, okay?" "No.!" "What about Lorenzo's birthday?" "I'll call you." "Bye." "Deirdre." "Deirdre..." "This is wrong." "She needs her family here." "She doesn't want nobody." "She's too angry." "We cannot afford the time to be angry." "Look what she's dealing with." "She does not have to deal with this alone." "She feels incredibly alone and incredibly unlucky, like she's been singled out by God." "Oh, Deirdre!" "Here's Kenise, a carrier with two girls; me, a carrier with no kids;" "and Mariah's got a boy, but she's not a carrier;" "so, poor Michaela gets this laid on her." "This did not happen only to Michaela." "Sure." "You got two kids already..." "[Michaela] Let me make you something, sweetheart." "I'll get you a big quart of fruitjuice." "She waited all this time to have one child." "She's not gonna have another." "[Laughter, Indistinct Chatter]" "[Knock At Door]" "Francesco." "Cristina!" "Ah, buon giorno." "Oh, Francesco." "[Speaking Italian]" "[Michaela, Speaking Italian]" "[Michaela] Everyone, this is Lorenzo's sister and brother... who came all the way from Rome." "[Simultaneous Greetings From Crowd]" "[Laughter]" "[Uproarious Laughter]" "This does not make sense." "Lorenzo's saturated fats have risen." "We eliminate them from his diet, and in his blood they increase." "If these very long-chain saturated fats are destroying his mind, then surely we should take him off the diet." "[Nikolais] Mr. Odone, please be patient." "Your son has been on the diet only six weeks." "Wait until the end of the trial." "What about the other boys?" "What results are you seeing in them?" "As with Lorenzo, it's too early to tell." "We need the study to run for the full six months." "That will tell you what is obvious now, that avoiding apple skins and pizza... has no effect on this brutal disease." "Augusto..." "Please, Doctor, is there anyone... who is chasing a definitive therapy, hmm?" "You judge us too hastily." "The French have tried bone marrow transplants." "In Boston, we're about to try immunosuppression." "Why have we not heard of this before?" "The transplants were disastrous... and the immunosuppression is highly experimental, so the protocol has been limited to six boys, all more profoundly afflicted than your son." "For God's sake, how afflicted does he need to be?" "[Augusto] It's a school, Lorenzo, like any other school." "It's full of doctors?" "Yes, darling, some of the cleverest in America." "What color's that cross?" "Red." "That's right, it's red." "I'm going to speak to your parents, but Leah's gonna stay and take your blood pressure, okay?" "I'll be right back." "[Leah] I'll just put this around your arm." "Look, it's important you both know exactly what's going on." "Cyclophosphamides are no picnic." "This is all-out chemotherapy." "We're gonna be suppressing his immune system." "It's very high-risk." "We're gonna need... to keep him here at least three weeks." "[Leah] That's it." "Lorenzo's still walking, he's still talking, he's still communicating, but that can all change." "Now, knowing all of this, do you still want us to go ahead with it?" "Given the alternatives, yes." "[Lorenzo Breathing Heavily]" "The faculty is very grateful to Lorenzo for doing this." "[Indistinct Chatter]" "[Doctor] This is Lorenzo Odone whom I've spoken about." "Please." "Now, young Mr. Odone, you need to come with me." "You are getting so big." "I'm gonna put you up here so we can all see you, okay?" "Attaboy." "Ten weeks after diagnosis we observe a hemianopia... with transient horizontal nystagmus." "The pupillary light reflexes are still intact, yet there's no optic atrophy." "However, there is evidence of early occipital lobe involvement." "[Slurred Speech]" "Beg your pardon, Lorenzo." "Why... are... aw..." "these... people... here?" "He asks why all these people are here." "Well, they're all doctors and they want to learn... how to help other boys in the future." "[Slurred] Other boys with the boo-boo?" "Yes, darling." "Mm-hmm." "Thank you, Lorenzo." "Now, in the scanning speech, we look for both ends of the pathology:" "Centrally, for the dysphasia;" "and peripherally, for the dysarthria." "Now, Lorenzo, would you walk for me, please?" "That a boy." "That's right." "Hold my hands." "That's right." "Two months ago, there was simply motor lag." "Now, note the characteristic gait." "[Gasping and Straining]" "It's due to hyperreflexia, but exacerbated by encroaching paresis." "[Labored Breathing]" "You're doing very well, Lorenzo." "Keep coming." "Only if you stop talkin' like that." "[Audience Whispering]" "[Man] Hello, Mr. Oh-Don." "Have I got that right?" "[Odone] It's "Odon-nay." Odone." "I'm sorry." "Ellard Muscatine." "I'm with the A.L.D. Foundation." "We heard about your boy, Lorenzo, and we'd like to offer some help." "You, you're a doctor?" "Heck no." "I'm a machinist from Aliquippa, Pa., but I'm an A.L.D. Parent." "This is my second time around, so I know exactly what you're going through." "Uh, my wife Loretta and I, we run the foundation." "We organize counseling, conduct seminars, fund research." "We have 500 member families, and not only American, but from Portugal, Israel," "Japan, Australia." "And we're growing all the time." "[Heavy Breathing]" "And we wanted you and Mrs. Odone..." "[Gasping For Air] To know that you're not alone." "Hi." "Ellard Muscatine." "Welcome to the conference." "Augusto Odone and..." "Oh!" "Odone, oh!" "Michaela." "Very nice to meet you." "Please come in." "This is my wife, Loretta." "These are the Odones." "Great." "I'm so happy you were able to come." "[Ellard] A lot of it is denial." "We don't want to say there's anything going on, that we're strong and we can handle it." "But this is the place and this is the time... to let it all out, let the barriers come down, and to share your feelings and get it off your chest." "It's hard to believe that things can get worse, but they will." "One thing nobody wants to talk about... is what happens or doesn't happen between the two of you in the bedroom." "One whole year we couldn't touch one another." "[Woman] My husband stood by me all the way, but does it test the men." "The weak ones up and go." "Even the strong ones leave." "My guy hung in there when our first boy took sick, then we had Jake tested." "Arlen wanted a bunch of boys, a whole ball team." "He's gonna have 'em too, with some other gal." "[Loretta] We'd like to thank the parents... who gave generously to the fund... for the immunosuppression trial." "The doctors have told us that this experiment... contributed greatly to the understanding of A.L.D." "Betty and Tom Knolls, $250 in memory of Corey;" "Dr. And Mrs. Liebowitz, $500 in memory ofJoel;" "Mr. And Mrs. Higgins, $1,000 in memory of Adrian and Julian." "26 people have sent in recipes for the newsletter." "We still need ideas for our snack foods and breakfast treats." "Remember, all recipes should be low-fat... and use only the foods allowed on the institute diet." "[Michaela] Excuse me." "Yes, ma'am." "Our son's complied with this diet... and yet his levels of saturated C-24 and C-26... have risen for two months." "That's exactly what's happened toJaybird." "Ah, well, perhaps, before we talk about publishing cookbooks, we should ask ourselves... if the diet is working at all," "shouldn't we?" "Damn right we should." "This is not the way we do things." "There are two families here with this paradox." "Maybe there are more." "Shouldn't we open it for discussion?" "My boy's levels are rising." "Now there are three." "So, uh, maybe we should..." "Could we have a show of hands..." "Yes." "And see how many families there are?" "This is a formal pilot study." "It has to run the full six months." "We don't take it on ourselves to interpret experiments." "That's the responsibility of the doctors." "Come on!" "Let's at least have a show of hands!" "That would be misleading." "The medical folks call this evidence anecdotal." " What's wrong with a show of hands?" " It wouldn't have any meaning." "Excuse me." "May I say something?" "Sure." "The only way the doctors can get useful results... is with a strict protocol... and statistical samples in a control group." "And a proper time frame." "Doctors have to be very careful." "The clinical trials must withstand a tremendous amount of scrutiny." "This is the way that medical science works." "That's the only way scientists can get the information they need." "So what you're saying... is that our children are in the service of medical science." "How foolish of me." "I assumed medical science... was in the service of the sufferers." "Yes, uh, thank you." "Now I think it's time we get back to our agenda." "Why, so we can discuss... further salvaging our marriages and managing our grief?" "What about the children?" "No one here is discussing the children.!" "Thank you." "If Mary has nothing further to add," "I would like to introduce Dr. Chapel... who will talk about the nasogastric tube and suction machine." "Would you join me in giving him a warm welcome?" "[Applause]" "Thank you, Madame President." "Thank you, Ellie." "[Lorenzo] Now hold my hand." "Yes." "Now the story." "What story is that, sweetheart?" ""La Notte de San Lorenzo."" "San Lorenzo who is..." "My patron saint... and saint of my father's village." "That's right." "And what happened to him?" "He was in Rome..." "Mm-hmm?" "And I forgot." "Mm-hmm." "Well, many years ago, the bad guy... says to him:" ""Bring us the riches of your church."" "And Lorenzo, he brings them the beggars... and the sick people, and he says:" ""These are our riches."" "Yes." "Bravo, Lorenzino." "Oh, that's wonderful, sweetheart." "Bravo." "So clever." "And tonight, August 10th is..." "La Notte de S-San Lorenzo." "[Michaela] The night of the shooting stars." "[Augusto] When anything can happen." "[Italian Opera]" "Shh-shh-shh!" "When we first went to the Comoros, what did we do?" "We got to know the country, right?" "Yes." "We studied, we got to know the language, resources, its laws." "We studied, right?" "We should treat Lorenzo's illness like another country." "I don't quite see the analogy." "All right, A.L.D. Has many dimensions, right?" "Yes." "So in order to understand it, we need a command of, uh, genetics, biochemistry, microbiology, neurology, - ology, -ology, -ology." "We don't have time to go to medical school." "The doctors are in the dark." "They're groping in the dark." "They've got Lorenzo on a turvy-topsy diet." "And that bloody immunosuppression, it's brutal, useless." "Michaela, we should not... have consigned him blindly into their hands." "He should not suffer by our ignorance." "We take responsibility, so we read a little... and we go out and inform ourselves, hmm?" "But... to miss time with him... while he can still speak to us..." "Oh, yes, I know, I know." "But he expects it of us." "I want to take Lorenzo off the diet." "He's been eating cardboard for 19 weeks." "What's it getting us?" "And feed him what?" "Normal foods, things a child likes." "Full of the long-chain fats that we know destroy his myelin?" "Look, on the diet, his blood fats are still rising." "Yes, I know." "It doesn't make any sense." "But we don't know enough." "We don't know enough." "We don't know it clearly enough." "Here, Michaela, let me show you something." "All right." "Un piccolo acquaio." "A kitchen sink?" "Yes, yes, a little kitchen sink." "All right." "Now here is the tap... and here's the sink basin and the plug hole, right?" "Very long-chain saturated fats... are introduced by what we eat, all right?" "Yes." "Also produced by the body, or manufactured by the body, these very same fats." "Biosynthesis." "Biosynthesis, right." "All right." "Now normally they are not harmful because... this enzyme eliminate the excess, right?" "But in Lorenzo, this enzyme is defective from genetics." "His plug hole is blocked, right?" "Hmm?" "Yes." "Now, we know that... very long-chain saturated fats... are made up of carbon atoms:" "C-2, -4, -6, -8, all the way up the chain, all right?" "In Lorenzo, his saturated C-24... and C-26... rise to four times the normal level." "[Sighs] Okay, now, here's where we have a little paradox, our mystery, okay?" "Why, when we reduce the dietary intake of saturated fats, does the level in our sink, which we would expect to go down, go up, hmm?" "So, logically, we should take him off the diet." "Well, Michaela, just as logically, we should not." "Let's not do what the doctors did." "Let's not act without knowing why." "Why, when we turn off this tap, does the level in our sink rise, hmm?" "I married a plumber." "You married a man... with a simple mind, who asks simple questions." "Here, Michaela, brain food." "Come on." "Oh, thank you." "Polish rats!" "I think I have an explanation for the paradox." "Lorenzo, kiss your clever mother." "Polish rats with a fatty acid storage disease." "Here, let me use your kitchen sink." "Here's our tap for what we eat." "Right." "All right." "And here's our tap... for biosynthesis." "I see, biosynthesis, mm-hmm." "When the rats were deprived... of a specific fat in their diet, their body cells compensated by overproducing it." "If this holds true for humans..." "Then the diet is useless... unless we can stop it from overproducing, slow down biosynthesis, see?" "Yes, yes, and there's more." "For this, I deserve two kisses, my darling." "Ah!" "In this summary, it talks about the concept... of fatty acid manipulation." "[Deirdre] What's that?" "They stop the rats producing one kind of fat... by loading their diet with another kind." "Maybe we could stop Lorenzo from producing saturated C-24 and C-26... by loading his diet with another kind of fat, you know, one that's less harmful." "We should have this translated as soon as possible." "I have some news t-t-too." "It's a letter from Omouri." "He wouldn't open it until you got home." "Omouri." "Yes." "Michaela, by tomorrow morning, your Polish rats... will be speaking to us in English, hmm?" "I've been thinking." "I found that article by accident." "I just as easily could have missed it." "And it was written by a Polish biochemist, who, I'm sure, knew nothing about A.L.D." "Well, Michaela, still it give us a new line of inquiry." "Yes, of course, but it could take months, and there will be other leads and that will take more time, and it's time we don't have." "All these experts, working in isolation, each one on his own piece of the jigsaw." "[Indistinct Conversation]" "Yes, a symposium is the best way to accelerate the exchange of information." "In that sense, it's a very good idea, Mr. Odone." "Only, uh, may I say something?" "Have you any idea how many children in the United States... die every year from choking on French fries?" "Many more than from adrenoleukodystrophy." "Ours is what is known as an orphan disease." "It's too small to be noticed, too small to be funded, especially with the iron hand of Reaganomics." "So, while, as I say, this remains a very good idea," "I'm afraid it's a luxury we just can't afford." "We appreciate that, so we have, uh, prepared a budget... based on 40 participants for two days." "We think it can be done for $35,000." "My wife and I will raise the money." "We can't ask you to bear the cost." "No, no," "That's out..." "no, no, no." "That's the least of our concerns." "And, of course, your institute will not be at risk, because the Odones will underwrite any shortfall." "Hmm?" "And, uh, we took the liberty of drafting an invitation list." "Forgive me, Dr. Nikolais." "We wish to make two conditions." "We would like the symposium to emphasize therapy, and Michaela and myself would like to participate in the discussions." "Martha, I'm afraid we'll have to add to your work load." "But it's in a very good cause." "We've decided to convene the first symposium on A.L.D." "We should notify the Muscatines." "Of course." "Well, the foundation could help with the arrangements." "They could even, perhaps... find some funds." "Now, how can we make this work?" "How about we hook it to the back of the next foundation family conference?" "[Michaela] When would that be?" "Next summer, fourth ofJuly weekend." "That's nine months away." "It'll take every bit of that time to invite those busy doctors and raise money." "I don't think so." "What's your time frame, Mrs. Odone?" "Five weeks from now, November 10th and 11 th." "[Labored Breathing]" "That's not possible." "I think it is." "Pardon me, but what is the point of rushing it?" "As A.L.D. Parents, we both know the answer to that inane question." "Look, if you want to make the symposium happen, you're going about it the wrong way." "Mrs. Muscatine, at least we are going about it." "[Lorenzo Mumbling]" "I'm sorry, sweetheart." "I don't understand." "He wants his diaper changed." "[Dial Tone] Mrs. Muscatine, look..." "Hello?" "Hello." "[Clicking Receiver]" "They should be hung by their eyelashes." "They're not gonna help?" "Doesn't look that way." "I'll take him." "No, no, no, I got it." "What are you gonna do, mortgage the house?" "Not till I have to." "Michaela.!" "I've spent the last couple of months pulling on the heart strings of Washington... to raise money for multiple sclerosis, sudden infant death syndrome... and cystic fibrosis." "Then why exclude adrenoleukodystrophy?" "Because, you see, I also cover politics, the arts, women's issues and stuff, and my readership actually likes to have a little balance of what I report." "I cannot do another medical story right now." "Maybe in another month." "Joyce, I think you should meet my son." "My friends, this is so generous." "You know, Augusto, our wives are eager to form a committee... to perhaps help with hospitality." "Oh, wonderful, wonderful." "[Excited Chatter] So much money." "Not only will we be saving money, but they'll be extremely well fed." "We have for breakfast French pastry, correct?" "Lunch, U.N. Finger food." "The British will do a ripping afternoon tea, [Knock At Door]" "And for dinner, I know a marvelous Italian cook." "[Laughter]" "Are you needin' another pair of hands?" "I'll lick envelopes, serve coffee, whatever." "Oh, this is for your boy." "I grow the best organic produce in Virginia." "Thank you." "You don't remember me." "Wendy Gimble." "Foundation family conference." "I yelled louder than you." "Yes, of course." "You have two boys." "Well, we lostJaybird, butJake here is fine." "We think what you're doing is fantastic." "[Deirdre] It's okay." "Hello, Jake." "Come on in." "Thank you." "It's all right." "I read in the newspaper about what you were doing." "It was about time somebody did something about this." "Come meet the others." " Is that Lorenzo?" " You'll make it." "Yes, Lorenzo, this is Jake." "He's a young man of your age." "Look, Lorenzo, a great big pumpkin." "Let's go down and feel how big it is." "[Indistinct]" "[Lorenzo Mumbling] What, sweetheart?" "[Garbled] I want to play.!" "I'm sorry, Lorenzo." "Let's, let's try that again." "[Indistinct Shouting]" "[Shouting Continues]" "This morning I could still understand him." "If we could clone the errant gene, we'd be able to identify the deficient enzyme, mass-produce the gene, implant it into A.L.D. Sufferers... and normalize their metabolism." "How long would that take?" "Qu'est-ce que vous pensez, professor?" "Si tout va bien, une dizaine d'annes." "[Man] Oui." "Seven to ten years." "You see, science has its own time, and very often, any new advancement will be a matter of serendipity." "[Professor Nikolais] For the last six months, all our A.L.D. Boys have been on a regimen... which excludes saturated C-24 and 26." "But despite this restriction in the diet, these long-chain fatty acids have remained the same, in some cases, even increasing." "Now this compensating increase..." "I put down to biosynthesis." "But, uh, Gus, if you could maintain the diet and somehow inhibit biosynthesis, you'd have a therapy." "[Nikolais] No, no, we wouldn't... quite have a therapy." "You'd have a way of lowering the fatty acids to normal." "[Speaking Japanese]" "If you wish to inhibit biosynthesis, have you considered fatty acid manipulation?" "[Speaking Japanese]" "There is some literature." "A rat study, Russian or Polish." "[Michaela] Polish." "Straszak." "I have copies right here." ""Polish Journal of Biological Sciences," 1979, Volume II." "They loaded the diet with one fat... and decreased biosynthesis of another." "Yes, I remember this article." "And also in Canada, they have seen the same thing in miniature swine." "As a matter of fact, I've seen it in human cells, in human A.L.D. Cells." "From patients with A.L.D., I took fibroblasts, skin cells, okay?" "Each one carries the same genetic defect." "I incubated them with oleic acid and reduced the levels... of saturated C-24 and C-26 by more than 50%." "Excuse me, Doctor, you said, uh, oleic acid?" "Yes." "Monounsaturated C-18." "It's the principle component of olive oil." "Surely this is the basis for a therapy, then." "Slow down." "These are preliminary studies in cultured cells." " We don't know it will work in a human being." " Well, take the olive oil... and try it in a patient and see." "Yes." "Olive oil is forbidden in the diet." "C-24, C-26." "Yes, but use whatever Dr. Rizzo... fed his fibroblast." "Exactly." "Pure oleic acid would be very toxic to an intact organism, an animal or a human." "It would need to be the triglyceride form, which is edible, but that's not available." "Then can't you take ordinary olive oil... and extract the C-24 and C-26?" "You could, but it's a very complicated procedure, and it would be very, very expensive, especially to produce the quantities needed for a clinical trial." " Have you tried contacting chemical companies?" " No one will tool up for that, not for an experiment, not for something with no market." "Then you haven't tried." "No." "[General Conversation]" "[Augusto] If we were able to give him triglyceride, then..." "[Indistinct] Yes, it has to be triglyceride to be edible." "[Michaela] Edible form." "Triglyceride, yeah." "[Man On Phone] No one in this department can help you." "Do you know anyone who does?" "I'll send you back to the switchboard." "Ask for RD." "[Operator] Which extension, please?" "Research and development." "Hello?" "[Woman] RD." "Yes, I'm seeking a triglyceride form of oleic acid." "Your name, Doctor?" "No, I'm not a doctor." "Are you with a company?" "No." "I'm the mother of a sick child." "A mother." "That's right." "And you want?" "Oleic acid." "Lines are busy." "I'll wait." "Papa makes you spaghetti al pomodoro, seasoned with basilico, the royal herb for our prince, huh?" "[Affectionate Chatter]" "There's really no remedy." "It's a very rare disease." "Take a little." "That's a boy." "The most positive lead came from the symposium, and that was the oleic acid in triglyceride form." "There you go." "Right now it's our only hope." "[Whispering]" "[Woman] Don't fret, Mrs. Odone." "You will hear from him within the hour." "You be sure of it." "Thank you." "That's 301..." "Don't worry." "I have the number." "Thank you very much." "You're welcome." "Bye-bye." "Bye." "So as the swallowing reflexes weaken, he's less and less able to handle his own saliva." "We'll all have to be pretty vigilant." "I mean, we really have to stay on top of this." "If we allow any saliva to get down into his respiratory tract, we've compromised his airway." "[Phone Ringing]" "We don't want to do that." "Okay, put your thumb over the valve." "When you no longer want to suction, just release your thumb." "Hi, it's Protochem again." "I'll connect you to our Mr. Pellerman." "Yes, I believe you're looking for some oleic acid?" "[Michaela] Yes, in triglyceride form." "You're a very lucky lady." "We've been testing a glycerol trioleate here... as an industrial lubricant." "Is it fit for human consumption?" "I don't see why not." "It's a purified olive oil." "Without saturates?" "Pure, monounsaturated C-18 in triglyceride form?" "Yes, ma'am." "You're sure?" "Happens we have one bottle here, one liter sitting idle on the shelf." "Marvelous." "How can we get our hands on it?" "I'm sorry." "Go ahead?" "How can we get our hands on it?" "This is..." "it's beautiful." "And, of course, it's entirely harmless." "Gus, you're talking to an Italian." "It's only olive oil." "Yes, it's harmless." "It's like, uh, sugar." "And yet to a diabetic, sugar can be lethal." "The trouble is we don't know to what extent..." "Lorenzo can metabolize fats." "Well, Gus, it's been seven months... since Lorenzo was diagnosed." "Now he's silent." "He's immobile." "Augusto, I am a scientist, and I'm of absolutely no use to you whatever... unless I can maintain my objectivity." "And I am not a scientist." "I am a father." "And nobody can tell me what dressing I put on my kid's salad." "Okay?" "Augusto." "This science of medicine, you know, it's not like physics." "There's no mathematical certainty." "And because we deal with human beings who suffer, it can appear heartless." "I know." "You realize, of course, that any collaboration of mine would have to be unofficial." "Si, si, of course." "Now, the dose." "We think, uh, 40 grams a day." "No, that's too much." "Too much?" "Say 30." "Thirty." "Yes, any more than that might damage your son's liver." "Si, si." "Excuse me." "Thank you." "Darling, they're about to start." "[Opera]" "We're going to send this blood away to establish a baseline, my brave boy, because today, we all start the fight against the boo-boo." "There." "All done." "Lorenzino, this year we celebrate Thanksgiving a little early, with a fine barracuda like we used to eat in the Comoros." "And Papa has cooked it with aromatic dill, huh?" "And now, we add this special olive oil, which Mama found, hmm?" "All right." "Here we go, hmm?" "Deirdre." "All right, Lorenzo, here we go." "[Children Chattering]" "There we go." "You did good, buddy boy." "You did real good." "Your auntie means "well," darling." "In this house, we cherish our adverbs, and I expect you to do the same when you get your voice back." "[Lorenzo Wheezing]" "[Phone Ringing]" "Hello?" "Mrs. Odone?" "Yes." "This is Dr. Nikolais'office." "I have Lorenzo's results, and there's been a small drop in saturated C-24 and 26." "About 15%." "15%?" "Dr. Nikolais said not to get carried away." "It could be a spontaneous variation." "Am I allowed a tiny squeak of joy?" "We'll know more next month." "Thank you." "[Coughing, Choking]" "[Indistinct Shouting]" "[Nurse] Don't move him.!" "Get something to hold down his tongue.!" "[Lorenzo Screaming] [Michaela] Oh, my baby.!" "[Panting]" "So, this is a kind of seizure like epilepsy." "No, not a seizure." "It's a paroxysm." "It's triggered by saliva in the windpipe." "But we suction him." "Regularly." "Yes." "Even so, some saliva gets caught in the trachea." "What, in any other child, would just be a cough to get rid of it, in Lorenzo becomes this scrambled chain of reflexes... that amplifies into what you have just seen." "If this happens again, what can we do about it?" "Just help him try to ride it out." "[Indistinct]" "We're all flying' blind here, folks." "The best I can offer is sedation." "But if it's not epilepsy, then he's aware of what's happening while it's going on." "Well, it's possible." "And if he has another episode, we could talk him through it." "It's possible, Michaela." "It's possible." "[Lorenzo's Labored Breathing Continues]" "Si, si, a 50% drop, right?" "[Woman] That's correct." "Thank you very much." "Thank you." "Fantastico.!" "Fantastico.!" "I very well understand that we are not scientists, but we have observed this in..." "[Nikolais] Let us do it our way, Augusto." "All right." "Yes." "Mm-hmm." "I've got to go." "Okay." "Thank you." "Good night." "Okay, bye." "Nikolais says it is very interesting." "Interesting?" "A 50% drop?" "But he said it's too early to draw a conclusion." "Rizzo might consider a trial, and he asked us... not to tell the other parents." "What?" "That's immoral." "Yes, it is." "Here's something that can finally..." "But the parents have to hear!" "Yes, they do." "We'll tell them." "They can judge for themselves." "But the foundation has the mailing list." "We'll call the Muscatines." "You're kidding." "No, I can eat crow in the name of a good cause." "Michaela, now go easy." "[Ringing]" "Mmm." "Delicious." "That was worth driving 300 miles for." "Now, my friends, dessert." "[Muscatine] Here, let me move this." "And, Loretta, a little science." "Thank you." "There you are." "Loretta." ""Dear A.L.D. Parents."" " You want us to send this out." " Yes, we'd be happy to pay for copying and postage." " But you're advocating a therapy here." " Oh, no." "We're merely reporting a positive advancement in the diet." "It would be wrong for us to keep this information from the other parents." "Mrs. Odone, we have an advisory board of eminent doctors." "We parents take our guidance from them." "After all, they are the ones with medical degrees." "Yes, my dear, but you see, Nikolais already knows about this," " and the wretched man does absolutely nothing." " Because Dr. Nikolais..." " is a responsible..." " No, because Dr. Nikolais has another agenda." "[Speaking Italian]" "You know, Loretta, sometimes... the interest of the scientists... is not the same as the interest of the parents." "Scientists are human, and sometimes they can be wrong." "As president of the foundation, you represent us, the families, see." "And you serve the families best by informing them." "And all we ask... is that you please send this letter to them, that's all." "Our parents suffer enough... without being made the victims of false hopes." "But..." "No, we can't give credence to every jerk that comes along..." " with a pocketful of apricot..." " This is not Laetrile." "We're talking about an extract of olive oil, Olive oil." "An idea put forth by the scientists... at the symposium." "And it works." "It works in Lorenzo." "It works in an A.L.D. Boy to a degree that is significant." "Very significant." "For God's sake, don't you think... that other parents have a right to know this?" "I mean, we know this is a deadly disease, so they have a right to choice, and if they feel as we do, they can put pressure on the doctors, because as parents, we should challenge these guys." "We push them." "Unless somebody..." "someone is willing... to question, to provoke, how would there be progress?" " We feel there is progress." " Loretta, we know that, but all we say is start a dialectic with the doctors." " Are you gonna tell us how to run the foundation?" " We're merely asking you... to disseminate information." "You're going to teach the doctors?" "We call that arrogance." "Arrogance?" "Yes, uh, it is..." "arrogance." "It comes from the Latin word..." ""arrogare."" "You know the root, what it means?" "It means to claim for oneself." "That's the root." "It means to claim for oneself." "And I claim the right to fight for my kid's life, and no doctor, no researcher, no bloody foundation... has the right to stop me from asking questions... which might help me save him!" "And you, you have no right to stop the sharing of information.!" " So you think about it..." " Our job is to be there for the parents, to comfort them, not to get them all stirred up." "If the doctors think you are onto something, they will let us know in their own good time." "Their own good time is not our time." "Oh, that is..." "What, they are so powerful, they are so powerful... that you would be silent?" "What..." "they are not gods." "You know, this, this acquiescence is so disgusting!" "You think you know so much." "Let me tell you something!" "When Michael got sick, we thrashed about for anything that might help him." "You know the best thing that happened?" "He was taken quickly." "Now Tommy, he has lasted three years." "For two of'em, he's been without his sight, his mind, everything that makes him a human being." "He's a vegetable." "Oh, boy." "You know, if you would stop all this denial, you wouldn't do a thing to prolong your boy's suffering and indignity one minute longer." "Has it occurred to you that maybe he doesn't want to be around anymore?" "[Augusto] Si, si, thank you." "It has to be a mistake." "No." "No, Nikolais says not." "Augusto." "Oh, I'm sorry." "Should we risk raising the dose?" "No, he's too fragile." "Any more might harm his liver." "[Bell Ringing]" "[Children Shouting and Cheering]" "Lorenzo, my darling, listen to Mama." "[Gasping]" "You're going to have to be brave as only you know how, sweetheart." "We're going to count together, all right?" "Listen to Mommy's voice, and the boo-boo will let go of you." "One, two, three, four, count to five..." "[Lorenzo Screaming]" "Michaela?" "You know, I've been a nurse for 18 years, eight of them spent in I.C.U. Pediatrics, and I know that you can't always play by the rules, but this boy shouldn't be at home." "He should be placed in a properly equipped hospital." "Why don't you say what you mean?" "I'm not comfortable with this situation." "Well, Lorenzo and I aren't comfortable with your lack of comfort." "I, I can't continue to be a party to this." "I gotta give you my notice." "I'll stay until you..." "No, I think you should finish tonight." "Michaela, come on." "Your agency will receive a check in the morning." "Look, Ruth, we're all a little strung out here." "[Michaela] She's talking about placement, Deirdre." "It's a euphemism for hospice." "Lorenzo has had quite enough to endure." "We are not going to expose ourself to doubt and despair." "She's exhausted." "I mean, that's all it is." "Look, I don't know." "Somebody had to say somethin', you know?" "Good luck, hon." "Buona sera, Odone." "Look what I found." "Look at this." "Would you look at this?" "Let me show you." "Here, where are we?" "Right here." "That was June of'83, just 21 months ago." "You know, it's time you guys take it easy on yourselves." "[Sigh] Deirdre." "If we knew why it only work halfway, then we might know what to do next." "Augusto, how many mornings have I found you asleep... under a pile of research papers with the light still on?" "And then you wake up and drag yourself into the office." "How long can you keep this up?" "If you get sick, and you will, what are you gonna do for money?" "What, what are you telling me?" "For God's sake, he's my kid." "Yeah, so are Cristina and Francesco, and it's been a year since you've seen them." "[Lighter Clicking] Listen to me." "Okay?" "There has to be a life beyond Lorenzo." "What are you telling me?" "That I should give up on this now?" "Oh, man, we both know that even if you get his levels down to normal, his brain can't fix itself." "I mean, how much myelin does he have left?" "You ever ask yourself that question?" "Deirdre, now that's enough." "No." "Don't bullshit me." "That is enough." "I know you do, but she doesn't." "Not ever." "You have to help me with her." "I mean, she doesn't eat;" "she doesn't sleep." "I'm afraid for her." "I think she's losing it." "You've seen how she is with him." "She just can't let him go." "Poor Deirdre." "Spoken as only a woman who's never had a child could speak." "Michaela, Deirdre was speaking out of love." "I am only telling you what I see." "For both your sakes, there has to be... life beyond Lorenzo." "Do you think because he has no voice... that he has no will to live?" "He asserts it through us!" "If you can't understand that, there's no place for you in this house." "Michaela, for God's sake.!" "She is your sister." "I think you're losing it, Michaela." "Well, then I think you should go." "Augusto, help her pack her bags." "I'm going back to our son." "[Speaking Italian]" "You think I'm crazy because I speak for our son?" "[Speaking Italian]" "I am so sorry." "I never wanted this to happen." "How can l..." "How can I enjoy anything when he enjoys nothing?" "How can you talk to me about loving him when you trivialize him... into some kind of biochemical crossword puzzle?" "Don't!" "No!" "[Lorenzo Gasping] Mrs. Odone.!" "Get a tube." "Have you got the tube?" "Don't nod." "Say yes." "My attention's on Lorenzo." "I can't see you." "Darling, look at me." "Open your eyes." "Listen to Mommy's voice." "We're going to count." "One, two, [Screaming]" "Three, four, five, six." "[Screaming Continues]" "[Crying]" "I'm sedating him as much as I dare." "I don't know what else to do." "[Lorenzo Crying]" "How can he endure this for so many hours?" "Augusto," "I don't think he'll have to endure it much longer." "[Lorenzo Screaming]" "[Whimpering]" "Michaela, let me take him." "You need rest." "I'm fine." "I could use a cup of coffee, though." "Okay." "[Lorenzo Sobbing]" "Lorenzo." "Lorenzo, listen to Mama." "Can you hear me, my darling?" "If this is too much for you, my sweetheart, well, then, you fly." "You fly as fast as you can to BabyJesus." "It's okay." "Mama and Papa will be okay." "[Crying]" "[Lorenzo Sobs]" "I have rarely seen anything like this boy's tenacity." "They have a bed available upstairs." "No, no, no." "In a hospice, no." "It'd be easier on everybody." "But it would not do honor to Lorenzo." "Michaela." "Yes?" "Come." "What?" "Come, come." "Nurse." "Yes." "Now, today we eat, moglie mia." "Every last mouthful." "I'm not hungry." "No, no, no." "You eat." "Sit." "Michaela, you sit." "Sit." "From tomorrow morning, the Odones need all their energies." "Now, listen, we set out to normalize Lorenzo's blood, right?" "And with the oleic oil, we got it half right." "But that was luck." "Oh, it was rather more than luck." "No, no, no, it was luck." "It was merely observation." "Rizzo observed something in a test tube, right?" "We tried it on Lorenzo." "Rizzo observed a 50% drop in fibroblasts." "We observed a 50% drop in Lorenzo." "Now, Michaela, that is luck." "That is observation." "That is all it is." "It is not understanding." "And until we understand why this work only halfway, how can we expect to succeed all the way?" "Right?" "Mmm." "I need to understand, Michaela." "You need to understand." "So, tomorrow we go back to the library." "No, no, no." "We review all the literature, everything on fatty acid metabolism, every word published in the last decade." "So eat." "You eat." "Whatever energy, whatever time I have left, I want to spend with Lorenzo." "You will not have to leave his bedside." "I will go to the library, make copies." "I bring it home for you." "Augusto, we are on our second mortgage." "You can't afford not to go to work." "Michaela, I will work." "I fit it in somehow." "Michaela, this is important." "I need you." "I need you, Michaela." "So eat." "Bravo." "Bravissima." "Bravissima." "[Groans]" "[Muttering]" "Betty!" "Betty!" "Betty, all these fatty acids studies, they all concentrate on the middle of the chain... because medium-chain saturates are implicated in cholesterol!" "Shh!" "So what do these researchers do?" "They think, "Cholesterol, cholesterol."" "C-12, C-14, C-bloody-16!" "Augusto, what is it that you're looking for?" "I am looking..." "I am looking for the long end of fatty acid chains!" "Write it down for me." "These researchers should be doing something about it!" "Write it down." "Long-chain fatty acids." "C-18 through C-26... saturated, monounsaturated, okay?" "Okay, now I want you to go off to lunch." "[Muttering]" "I want you to have yourself a good lunch, and I'm going to see what I can do." "This is the best I could do on a moment's notice." "It's one article from a veterinary science journal about pigs." "But at least it's on your long-chain fats." "Oh, well, thank you, Betty." "Listen, I'm, I'm very sorry about..." "Oh, well, you're Italian." "[Lorenzo Crying] [Michaela] 72, 73, 74, and 75," "76, 77, 78, 79, That's nice going, Mrs. Odone." "80." "That's it, my love." "You know only the strongest, bravest boy, only a very special person like you is chosen to fight the boo-boo." "I'll give him a little water, okay?" "20 cc's?" "Yes." "And, Nancy, why don't you tell Lorenzo what an excellent job he did." "[Nancy] What an excellentjob you did, Lorenzo." "Sometimes I wonder, my love, if people realize how incredible you truly are." "Oh, thank you." "Thank you very much." "Is this a new hobby or a nervous tic?" "No, this is a simple mind at work is all." "See, each paper clip, Deirdre," " is two carbon atoms, right?" " Oh, God." "No, no, no, really." "It's two carbon atoms, all right?" "So, we have C-2, C-4, C-6, you know?" "Now here's what I try to do." "I try to make a chain, a very long chain, of monounsaturated fats." " This is the good guys, right?" " Okay, what are these, the bad guys?" "Yes." "These, these here is the bad guys." "All right?" "Now, Deirdre, what I'm trying to do," "I'm trying to understand the relationship... between the good guys and the bad guys." "So, okay, I am the enzyme, right?" "And I reach out and I grab a carbon atom... and I put it on my chain, right?" "Here's my chain, right?" "Now, here, you be the bad guy enzyme." "No, no, no." "Do this for me." "You be bad guy enzyme, all right?" "Take these, all right?" "Now you reach out and you grab carbon atom... and you put it on your bad guy chain, okay?" "Oh, Augusto, I had enough trouble with the kitchen sink." "No, no, you just do this." "Do this and then I explain, okay?" "All right, all right." "So let's each start now... making our chains, okay?" "All right, putting that on." "That's good, Deirdre." "Good." " We put another one on..." " Here's your stuff on enzyme complexes." "Ah, thank you." "Mille grazie." "Prego." "Prego.!" "Very good." "Now, did you make the extra copies?" "Oh, you bet." "Oh, thank you very much." "For Michaela?" "How is she?" "She's fine, she's fine." "So, here we are." "We're making our chains more or less the same rate, right?" "Mm-hmm." "That is what we'd expect, right?" "But now, Deirdre, if we were enzymes... in the bodies of little rats and little pigs and Lorenzo, then, the faster I go, the more you slow down." "Now why would that be?" "Why would the faster I go, why would you slow down... if we're both separate enzymes?" "It means that there has to be some kind of relationship going on." "We affect each other some way." "There's some kind of interplay." "But what is it?" "What is it?" "One, two, three, four, five," "[Soft Murmur] Six, seven candles." "And Mommy will blow them out for you." "Make a wish." "[Child's Voice Murmuring in Italian]" "[Child's Voice Counting "C"Fats in Italian]" "[Counting Continues and Grows Louder]" "C- venti-quattro, c- venti-sei, c..." "Michaela!" "Michaela!" "They are the same enzyme!" "There is one enzyme for both chains!" "It's the same bloody enzyme!" "So, if we keep the enzyme busy making monounsaturates..." "It distracts it from elongating the saturates." "Right!" "So we have a way to trick nature." "Yes, it's a principle called competitive inhibition." "Right." "So, Gus, Gus, you think it's a reasonable hypothesis?" "Oh, I think it's more." "What you've done is to clarify the biochemical pathway." "Augusto, I really want to congratulate you." "This, this would explain why oleic acid is only partly successful." "Yes, it's C-18, you see." "It's too low down in the chain." "Right, right." "So, we add a second barrier." "We block the bad guys further up the chain, the saturates, further up the chain between C-22 and C-24." "Then, we stop the bloody things completely." " And how do you do that?" " By adding monounsaturated C-22." " But that's erucic acid." " Yes, exactly." "How does he get it?" "Um..." "Martha?" "[Intercom] Yes, Doctor." "Among our animal studies, you'll find a, uh, a file on erucic acid, with an "E."" "R-U-C-l-C." "[Martha] Okay." "Thank you." "You, you don't believe it will work?" "Well, I think what you people have done is to postulate a theory... which could be of enormous benefit." "But as for erucic acid, it creates cardiac problems in rats." "And once it's not safe for rats," " I don't know what justification there can be..." " Here we are." "For using it... thank you..." "as a therapy on humans." "But, Gus, Gus.!" "Erucic acid is the chief component in rapeseed oil, right?" " Yes." " Rapeseed oil is a common food in China and India.!" "Yes, and their rate of heart disease... if far lower than in this country." "Look at these documents." ""Cardiac lipoidosis, myocardial lesions," ""cholesterol deposits in the adrenal gland... and damage to the reproductive system. "" "I'm afraid the weight of evidence is much too great, and no human studies review committee... will ever do anything but discard erucic acid out of hand." "But don't you see that the human studies have been accomplished by history?" "They have been eating the stuff in these countries for thousands of years." "You can't expect me to start a protocol..." " based on that kind of..." " Perhaps you should examine your protocol... when children are dying, and find a way to research..." "Michaela, Michaela." "These isolated..." "Michaela!" "It's common sense." "Michaela, we simply have a contradiction, that's all." "We seek more information." "We do not dismiss erucic acid out ofhand." "[Nikolais] Michaela, Michaela, if I ignore all this evidence, and we embark on a therapy based on erucic acid, huh?" "Something goes wrong." "What then?" "Then I suppose the risk/reward ratio is too unattractive for you." "I beg your pardon?" "The life of one boy is not enough reward for you... to risk the reputation of the institution... and the esteem of your peers." "That was uncalled for." "Your responsibility is merely towards your own child." "My responsibility is towards all the boys that suffer... from this disease, now and in the future." "Of course I anguish for the suffering of your boy." "And of course I applaud you for the efforts you make on his behalf." "But I will have nothing to do with this oil." "We are not asking, doctor, for your anguish or your applause." "We are asking merely for a little courage." "Okay." "All right." "[Augusto] Whatever the doctors say, reason tells us, this is a risk worth taking." "But, my friend, we need a sympathetic collaborator." "If it was in my hands, I would do anything to help." "I really would." "I have a boy of my own." "But I don't like our chances." "They said the same thing about the oleic acid... and here it was, sitting on the shelf." "This is another ball game." "Extracting saturated C-24 and 26 from rapeseed oil, that's very tricky." "It would take our best chemists at least a year!" "Then you would never get it past the F.D.A." "Erucic acid for human consumption?" "I'm sorry, but..." "no one in the country's gonna touch it." "What about outside this country?" "Well, I wouldn't know where to send you." "Oh, Mr. Pellerman, we're agents for over a dozen foreign companies." "There must be one that could help." " We need to look at the list." " You and your wife have enough to do." "I'm sure Mr. Pellerman would be happy to make the calls for you." "We'd have to be covered legally." "If anything goes wrong, this company is not going to be held liable." "Oh, we wouldn't hold you liable." "No." "No, no." "And it would be greatly appreciated... if we could circulate this." ""The Interplay of Monounsaturated and Saturated Fatty Acids:" "Therapeutic Application in A.L.D." Wow." "You wrote this?" "Yes." "We felt that if we were asking for help, our rationale needs to be very clear." "[Man] We've read your paper." "And I must say... it's a beautiful piece ofbiochemistry." "[Augusto] We approached over 100 companies." "They all say, "too difficult."" "My first impression was that it's bloody impossible." "But I've been doing difficult fractionations for 40 years." "I'd like to have a stab at it." "How long you think, Mr. Suddaby?" "I've only six months before I retire, so we'll see." "I let you get on with it, but call me if you have any questions." "Righto, bye then." "[Knocking]" "[Michaela] Who could this be?" "Ah!" "It's your good friend Wendy... with all sorts of lovely vegetables and fruits." "Oh!" "Michaela." "Look what you brought us!" "Hi, Lorenzo." "Jake's not in school today?" "He's not feeling so good." "I'm going for a walk with Mrs. Gimble." "We shall be back shortly." "I've given him... his oxcarbamazepine and 50 mils of water." "Would you like me to do anything else?" "He would love a story." "Do you have a problem with this?" "Fine." "He's had a couple of tantrums." "Oh, I'm sorry." "That's okay." "Dr. Rizzo's got him in the oleic acid trial." "That's good." "Hi, Jake." "Hi, sweetie." "Thank you for my potatoes." "Say hello, son." "Hi..." "Mrs..." "O-O-O-Odone." "He saw..." "He saw the way Jaybird went." "He knows what's in store for him." "[Crying]" "[NancyJo] "They forgot their sadness and hunger." ""They followed the bird." "They came to an opening in the forest." ""Before them was a little cottage gleaming in the rays of the setting sun." ""'Look, Gretel,' exclaimed Hansel. 'That cottage is made of gingerbread.'" ""'It really is,' replied Gretel, tasting a bit she had pulled from the wall." ""'The roof looks as though it were shingled with butterscotch.'" ""'The windowsils are made of chocolate,' declared Hansel." "'And the windowpanes are made of clear candy..."'" "NancyJo, do you think you could muster just a tad more enthusiasm?" "Lorenzo loves the sound of language!" "I'm paid to nurse!" "Not read stories." "And you've decided that his mind doesn't need as much nurturing as his body?" "What mind?" "I beg your pardon!" "Nothing." "Get out." "Look, Mrs. Odone..." "Just get out." "I've read up on this disease." "I know what it's done to his brain." "Lower your voice." "Lorenzo doesn't need to hear this from a subliterate!" "Face it, lady." "The lights are out and there's nobody home!" "Just get out!" "Go!" "Well, merry Christmas." "Get out!" "Happy holidays!" ""Near a great forest lived a woodcutter with his children, Hansel and Gretel." ""Times were very hard and a day came..." ""when there was no food except stale bread." "But Hansel..." was like you, my darling." ""He was a clever lad..." ""who knew how to solve difficult problems." "The woodcutter was very sad."" "[Augusto] Are you going to keep doing this?" "She brought it on herself." "She consigned herself to the outer darkness." "Michaela, NancyJo, she was a good nurse." "You trained her well." "But I want someone who can give more than medication." "Someone who'll minister to his mind and soul." "I think I found him." "What?" "I wrote to Omouri." "I asked him to come stay with us." "Omouri?" "Lorenzo is surrounded by women." "I don't want our son to grow up hag-ridden." "You cannot take a boy out of this fishing village... and bring him here to nurse Lorenzo." "Not to nurse him." "To be his friend, to bring the Comoros into this room." "We cannot uproot this young man... from his family, his way of life." "Where will he live?" "Here, in Lorenzo's room." "But..." "he has no English." "He is a Muslim." "An African." "We cannot bring an African to this racist country... to be a second-class citizen." "This is Washington, D.C. He'll be part of this family." "I'll teach him English." "There's so much we can do for him here." "Oh, Michaela." "Here, sit down." "Sit down." "Now, the last time Omouri saw Lorenzo... he was climbing trees." "He was a wild and vibrant boy, full of life." "In the letter that you wrote to Omouri, did you tell him what he will see when he walks into this room?" "I told him he would see a young man... who was very, very ill and had need of a friend." "That is all?" "How would you have me describe him?" "As a biochemical conundrum?" "No, no!" "You know he is not that for me." "Augusto, when you look at him, what do you see?" "Tell me the truth!" "How do I tell the man to give up?" "His health won't take it." "The hours he's working are daft." "Our expectations were unrealistic." "Look, Don's a man who's been working all his life in face creams and cosmetics." "With this oil, he's having the time of his life!" "You tell him, 'cause I won't." "All right." "For his own sake, I'm gonna have to." "Then you can call Mrs. Odone and tell her that you've pulled the plug." "Best leave him be." "I think you're right." "[Airplane Engines]" "Omouri." "[Labored Breathing]" "[Labored Breathing]" "Lorenzo," "Omouri is here." "[Singing An African Song]" "Morning!" "Morning." "Good morning, Mr. Suddaby." "There's a bottle of pure erucic acid triglyceride on my bench." "Will you see that it gets to young Mr. Odone as soon as possible, please?" "I'm off home now." "Bye-bye." "Good-bye, Mr. Suddaby." "Well, we'd better get on with it." "[Telephone Dialing]" "Four parts of our old friend oleic." "And, uh... one part erucic." "And the journey begins, like all good journeys, with... food." "Oh, where's the wretched nurse?" "I'm gonna take my baseline myself if she doesn't get here soon." "I think a month is much too long." "We take my blood levels every week and I have a cardiogram every few days." "We should know enough to start Lorenzo even sooner, don't you think?" "[Knocking] Oh, at last." "Hi!" "How's the mother tiger?" "Augusto didn't tell you." "I'm here to be Lorenzo's rat." "That's a very kind offer, but I can't let you." "I'm a carrier just the same as you." "My blood levels are just as high as yours." "I've had my baseline taken." "And I'm starving!" "Oh, I have been wanting to meet Lorenzo's main man." "[Omouri] I am content to greet Aunt Dierdre." "[Dierdre] It's good to meet you too." "[Augusto] Now, Michaela, I want no argument." "Michaela, you cannot test it." "Lorenzo needs you fit and well." "You would put Dierdre at risk without discussing it?" "Hey, hey." "Dierdre." "I backpacked all through India." "I must've eaten rapeseed oil up the wazoo." "Ah, I've been fasting all day." "All right." "Try it." "Okay." "Here goes." "[Augusto] So, does it taste awful?" "No?" "Good." "It's good." "Good." "Eat." "Eat." "[Suddaby] Your sister had no side effects?" "None." "If we can normalize the levels of a carrier so quickly, who knows?" "We're going to start Lorenzo immediately." "Marvelous." "We're terribly grateful to you." "Oh, no, no." "Not to mention Deedee, the family rat." "[Suddaby Chuckling]" "What dosage will you give the lad?" "We don't have time to be conservative, so we've decided on eight grams." "Okay." "[Whimpering] [Sucking Sound]" "Goin' to Montana to throw the hoolihan [Phone Ringing]" "Gonna feed them..." "Hello." "[Man] Mr. "Odon"?" "Odone." "Yes." "Look, we have a problem with this blood sample." "You have a problem." "What is the problem?" "Could there have been a mistake in the labeling?" "No!" "No mistake." "Why?" "We ran the assay twice... and the levels of C-24 and C-26 read normal." "[Chuckling]" "They read normal?" "Well, ah... ah..." "I, I, I..." "Do you have the sample in front of you?" "Yes." "What is the name on the sample?" "Lorenzo Michael Murphy "Odon." That's Odone." "No, there's no mistake." "But thank you very much." "Okay, bye." "Bye." "Where the fiery are raring to go" "Whoopie-ti-yi-yo get along little dogies" "You know Wyomin' will be your new home [Whispering]" "What's going on?" "Lorenzo's levels are normal." "Play, play!" "Wonderful!" "[Wendy] Drink it up, son, every last drop." "Mama says this is the only bottle in the world." "[Chuckling] Now that we have destroyed Lorenzo's blood fats," "I'm sure that Mr. Suddaby will make us plenty more." "Mama says this is bootleg." "Bootleg." "Look, I know this is only a fraction of what it cost you..." "No, no, no!" "Wendy, no, no." "You gotta take it, please?" "We work on the barter system." "Our oil." "Your produce." "There aren't that many potatoes in the world." "Okay." "Here you go." "Okay." "When are you gonna tell Nikolais?" "We told him and the other doctors and we sent all the figures." "Have you heard back?" "They deliberate." "They want more study." "Wendy, that's enough." "The other kids have to wait?" "Oh, great." "We know what to do about that, don't we?" "The doctors had a hard time swallowing the first oil." "They want to see how it goes with Lorenzo on the second." "But we don't have time, do we?" "No." "So we leave science to its own concerns, huh?" "Grazie." "Good-bye, Jake." "Merry Christmas." "What happened to the suction machine?" "There's no need, Michaela, not for four hours." "I turn the machine off." "All last night he made to swallow for himself." "Augusto?" "Augusto?" "We hope to make an announcement quite soon... about a new development in our diet therapy." "It's going to be quite expensive." "With the help of the government and this wonderful foundation, we are going to start a trial before the end of the year." "I can hear from that that you wish us godspeed." "[Woman] Excuse me, Doctor, are you referring to the oil... the Odones have invented for Lorenzo?" "We'll take questions later." "It's all right, Loretta." "It's a good question." "I should've mentioned it perhaps, but..." "The Odones have been extremely active and we are in their debt." "It's because of them that we are now preparing a protocol for review... by the human studies committee." "If then we can persuade the government to fund us, then we shall know whether this oil... is of more than temporary therapeutic value." "But it works." "The Odones have got Lorenzo's blood levels down to normal." "[Nikolais] It is for that reason we are conducting this trial." "You don't really expect me to endorse a therapy just... on the basis of one hopeful observation." "There's two!" "My boy's been taking the oil for six months." "His blood levels are normal and he's doing just fine." "You are playing with people's hopes." "Where did these Odones get off, thinking they know more than the doctors?" "Where did you get this oil?" "From the Odones." "They bring it from England." "They don't have approval from the F.D.A. It is bootleg." "Give the doctors..." "Come on!" "It's a mixture of two cooking oils." "What's the dosage please?" "Can you tell me?" "What are the side effects?" "How would we pay for it?" "It must be a formally approved protocol before we can raise the funds." "As I understand it, it will cost over $ 1,000 a liter." "Let me say you're not going to get any insurance company, any government to support you unless you have the approval." "The only way to get the approval is through thorough testing." "That's what they told the Al DS people about A.Z.T. They fought and got it." "They were dying and didn't have time to wait!" "We padded our walls yesterday." "I want that oil!" "[Arguing, Shouting]" "There, my darling." "That's a sweet boy." "What a big, strong boy." "Yes, my darling." "Yes." "You see, Michaela, it is as though he is trapped down in some dark cave." "Because he's lost so much myelin, he can't find his way out." "We know his simple functions are intact." "He breaths." "He swallows." "His heart beats." "From the outside world, he receives sensation." "A touch." "Pain." "Cold." "And here are the primal feelings:" "Fear." "Hunger." "Desire." "Up here in the neocortex, the higher brain, locked here... are his reasoning, imagination and memory, the things that make us each ourselves." "So, how does he reach us... when a simple swallow is such a huge task?" "To find... to find the way, the path from his mind... to the outside world... is like willing himself through a brick wall." "So he waits." "He waits." "Michaela," "do you ever think... that... maybe all this struggle," "it may have been for somebody else's kid?" "[Crying] Yes." "[Sighs]" "But I promised, I promised him... his world would never be silent." "He would never be alone." "Yes." "[Michaela] "One morning a little rabbit sat on a bank." ""He pricked his ears and listened to the trit-trot of a pony." ""A gig was coming along the road." ""It was driven by Mr. McGregor and beside him sat Mrs. McGregor." ""As soon as they had passed, Benjamin Bunny..." ""slid into the road and set off..." ""with a hop, skip and a jump to call upon his relations..." ""who lived in the woods at the back of Mr. McGregor's garden." ""That wood was full of rabbit holes." ""In the neatest hole of all, lived Benjamin's aunt and his cousins. ;" ""Flopsy, Mopsy, Cottontail and Peter." ""Old Mrs. Rabbit was a widow." "She..." ""earned her living by knitting rabbit wool mittens." "I once bought a pair..."" "What's the matter, my darling, you don't like this story?" "Lorenzo?" "You don't like the story?" "He does that a lot lately." "When?" "Mainly at story time." "Sweetheart, listen carefully to Mama." "Is your middle name Patrick?" "No." "Is it Peter?" "No, Mommy." "Is it Michael?" "Michael Murphy?" "Yes!" "Only you don't know how to say that, do you?" "Will you teach Mommy how to say no?" "When you close your eyes, does that mean no?" "What an idiot!" "That was a yes question." "You don't want to hear any more Benjamin Bunny, do you?" "That's an emphatic reply." "Very well, young man, no more Benjamin Bunny." "I don't know how Mommy could've been so thoughtless." "We're going to take all these baby books right to the children's hospital." "It's time for the big stuff." "Something heroic." "There you go." "You gotta tell us what you want." "The Once and Future King by T.H. White." ""The boy slept well in the woodland nest where he had laid himself down." ""At first he only dipped below the surface of sleep," ""and skimmed along like a salmon in shallow water;" ""so close to the surface..." ""that he fancied himself in air." ""He thought himself awake when he was already asleep." ""He saw the stars above his face whirling on their silent and sleepless axes," ""and the leaves of the tree rustling against them." ""He heard small changes in the grass." ""Distilled noises of footsteps and soft, fringed wing beats," ""and stealthy bellies drawn over grass blades..." ""or rattling against the bracken at first frightened him... then interested him. "" "[Imagining. ;" "Loud Screeching]" "Okay." "Is it your thumb?" "Is it your index finger?" "Your middle finger?" "Your little finger?" "It's your little finger." "Think you can move your little finger?" "Okay." "Tell your brain to tell your arm to tell your hand... to move your little finger." "Come on, sweetheart, tell your brain... to tell your arm to tell your hand... to move your little finger." "Come on, my friend, what a wonderful thing." "A "yes."" "Then there'll be a "could be" and maybe even a "shut up, Mommy."" "Right now we just have to get to "yes."" "So tell your brain to tell your arm... to tell your hand to move your little finger." "Come on, my love, tell your brain... to tell your hand to move your little finger." " Come on, Lorenzo." " You can do it, can't you?" "You can do it.!" "[Omouri] Come on, Lorenzo." "[Excited Gasping, Laughing]" "[Augusto] They are born without myelin, right?" "[Man] The only naturally dismyelinated higher animals in existence." "And this is passed from mother to son." "Yes." "The mother is from Edinburgh." "That's Jasmine." "She doesn't shake, but her male pups do." "You think you can stop them from shaking?" "We hope so." "By implanting nerve cells, we expect to see the growth of new myelin." "We've been partially remyelinating rats and mice for ten years now... and it's repeatable." "If we could fund this work, it could be the first in higher animals." "Hmm." "And then in human beings, huh?" "Well, in time." "Yes." "Dr. Duncan, if I help you raise these funds, and if we can get the researchers together to collaborate," "I think we can achieve very much in a short time." "Augusto, we scientists are a very competitive lot." "Such a collaboration's a lovely idea, but sadly that is not how science works." "That's not necessarily so." "Because, remember, the Manhattan Project?" "Twenty-eight months, it took them twenty-eight months." "Now, if scientists can come together to build the atomic bomb, surely they come together to remyelinate some puppy dogs." "[Yipping]" "[Michaela] Papa called last night from Italy, my darling." "He's missing his Lorenzaccio very, very much." "But he wants me to tell you he's making a big dinner tonight." "A banquet ofbrain food for the cleverest doctors in the world... who've gathered together to put the myelin back in the shaking puppy dogs." "And if they can achieve that... someday, there might be a way to help all the people who've lost their myelin." "Not only the boys with A.L.D., but the people with multiple sclerosis and many other diseases, Lorenzo." "Then think, my brave boy, if they can ever give you back your myelin, you'll be able to tell your brain... to tell your toes, your fingers, your anything, to do what you want them to do." "[Michaela And Lorenzo's Voice] And then, one day," "[Lorenzo's Voice] I'll hear my voice... and all these words I'm thinking... will get outside my head." "[Laughing]" "Oh-ho!" "Now it broke!" "This is Charles and this is Harry." "They've both been on Lorenzo's Oil for two years." "My name is Michael Benton and I'm 12 and a half years old." "I've been taking Lorenzo's Oil for four and a half years." "I was diagnosed with adrenoleulsodytrophy when I was 13 years old." "I've been talking Lorenzo's Oil for three years." "Some of the activities I'm involved in are baseball, basketball..." "I've been taking Lorenzo's Oil for 16 months." "[Indistinct]" "I've been on Lorenzo's Oil for four years." "I've been taking Lorenzo's Oil for one year."