European Stars And Stripes (Newspaper) - February 27, 1988, Darmstadt, Hesse Neer at age 7 while others notice no symptoms Unhul late in to. Ollen though. They become severely anaemic. Their blood does t clot properly so they bleed at the or Galesi injury. Their Livora and spleens Bio enlarged. Over Lime their Bones grow Brit la and break easily. They May suffer deep Bono pain. Removing the spleen can help temporarily. But this May also increase susceptibility to infections and some think it speeds Bone damage. Otherwise Little can be done to control the disease. During Hal tall there was an Aura c expo Lalion in Brady s lab As researchers worked on the enzyme mrs. Berman remembers. But meanwhile Brian s condition quickly worsened. To started to get very sick she said. He Wen Down Hill so that to could t walk up stairs he looked like a cabbage with legs. He had a huge belly and wasting extremities he was extremely anaemic his heart raced at 130 Beals a Minulph. Trying 1o pump enough oxygen poor Wood through his body. He also bled easily. His parents could not discipline him because even a mild spanking would bring spots of blood to a skin when doctors Al the Institute offered to give Brian Ihler incest version of a replacement enzyme the Choice for the bormans was easy. The first week his blood count went up a title bit says mrs. Berman. Everyone was encouraged. The second week it Wen up again. We were walking around on a Cloud. We cancelled his surgery we clearly Icett we were witnessing a alter seven weeks of Progress however the lab ran into problems making the enzyme for seven More weeks he got no Shols. He Well right Back to ground Zero she said. We were to hulled and Yel beside ourselves. It was an unbelievable time. Then to got it again and his blood count won Back up " this happened twice More and each time his haemoglobin Levels Ell after dramatic climbs now his haemoglobin is Normal. Who Laboratory less showed improvement the benefits of the treatment were Clear to anyone who saw Brian. Gradually he regained the Energy of a Normal Little boy running and jumping with his friends. Now to is 44 inches tall and his Waist is 22 inches while still Small Tor his age his growth has begun to Speed up what Brian seems to remember Best about his sickness is the taunts of other children. They used to Call a Falso he says. For Brady Brian s Rompc if ement is one Happy step in a Long often slow struggle against gaucher s disease. When a began working on the disease no one knew what caused it. In 1959, he Pul Forth the theory that victims lacked an enzyme and to finally proved this in 1964. Next he and his co workers developed ways to diagnose tha disease identify carriers who might pass it on and Check fetuses for the disease before birth. When these became available we began to think about what we could do for these he says. Do they respond to exogenous outside enzymes a had no reason to suspect that they would or would by 137. He had purified a Little of Tho enzyme. Although there were signs that it would do some goal it was simply too hard to obtain to be prot Cal. He and his. Colleagues spent to More years developing another purification procedure. This Lima they got sufficient quantities of tha enzyme by separating in lion placentas. But instead of going to the cells that were tilled Wilh the enzyme was gobbled up by other body tissues. After live years of testing it was Clear that tha purified enzyme would not work. So to scientists began tinkering again. This Lime they stripped Oft some of the sugar that makes up the enzyme rebuilt this Way the enzyme seems to Zero in on the swollen cells. Ii was Given initially to seven Pat ions but Only Brian responded. Brady believes in ailed in the others because they did not gel big enough doses. To Lohere however this suggests Hal the enzyme is not the answer to gaucher s disease. Brian s improvement is encouraging says or. Edward i. Ginns. A gaucher s researcher at the National Institute of menial health. Bui his trend men can t be generalized to others. We Don t completely understand what is going on. There Are patients who have a beneficial rec lion to something but it does t help anyone Etc. I think that was an isolated currently the researchers Are in the midst of a so called phase two trial with the modified enzyme. Doctors have taken Sver biopsies before and alter single injections in eight people. The researchers will perform this Experiment on of least 18 people before moving on to the final stage of study. In Hal Case they will give repealed doses to about a dozen people. The goal is objective proof that the enzyme changes the course of gaucher s disease. Barringer who Lei Bethesda last year in a Biller dispute with Broidy says the treatment is being hyped by its supporters. I spent 10 yours trying to make this approach a therapy for these patients but from the paint of View of a truly reasonable rational dependable and reproduce Iva he said up to a year ago. Hal was t done. Ii wan l Clear Hal his worked does in work the answer is yes responds Brady and he promises to publish the Prool. Among those closely watching this work is or f. Scott furbish of enzyme a Boston biotechnology firm Hal makes the enzyme in very Small qua lilies solely Lor Iho Federal studies. He said it takes about a Ion of placentas obtained trom hospitals to extract enough enzyme for one injection. To predicts thai if the treatment is approved by the . Food and drug administration ii will Cost patients $30,000 annually. That will drop to $12.000 after a enzyme Scales up ils product lion. What we am proposing or a Cost is very very reasonable compared to what hospitalization a oils Lor gaucher s patients he said. Insurance companies would be delighted to have something Hal works this meanwhile doctors at the Federal research Institute believe they have Lound an answer to gaucher disease " for the first Lime in this kind of disease there is a says or. Norman Barton clinical director of the program. In Brian there is objective evidence Hal it works unan Gry at last after being Down and out by Christopher Sullivan associated Pressw Hen Dennis Payne was 8, his parents told him to Wail with his sister White Thoy went out. I m 36, be 37 in March he says quietly and i m still wailing or them to get Back from the grocery his tortuous Story starts there. With stops at an orphanage a divorce court alcohol rehab Lillion centers and dark streets hurried away irom a homeless shelter it takes turn today to a hushed Plush Ollice in new York s Rockefeller Center. Ii he s in a hurry now. His Boss less him Call a a mousing. Is he confident Are things of now to answers quickly nodding Bui he holds up his Palm like a cop stopping Trakic and pauses i Don t know what s going to happen tomorrow. I go to a a. Meetings i Don t go every night t Don t have to. Nobody s looking Over my when he applied Lor the Job delivering and picking up documents for a corporate Law firm he was living in a shelter. I told them i was homeless and you Don t really gel Over either condition he says. Having lived by soup Kitchen schedules he still plans for Only one meal a Day his Ssler. With whose family a s living until he yets his own place encourages him to Tat regularly the two have stayed close since the Day they were packed Oft to an orphanage on Slaten Island a Terry ride across new York Harbor from lower Manhattan. Eva Ella never got in trouble. Payne says but he did. Over and Over. He once fought with his sister and her husband after seeking Refuge Wilh them something he d done Only because his drinking and belligerence and beneath those his anger Cost him a Job or a Home yet again. Ii was years before he became unan Gry As he puts it. Somehow the bitter lifetime in Between does t show on Payne s open lace or in his smiling eyes. In school he was a Good student who did other kids Homework for a Dollar Here and there. "1 thought i could buy me and my Sisler s Way out of the he Couin a of course but he managed to buy liquor. From the age of 13. He says he drank in High school in Vietnam afterwards while earning a degree in education. He Laughl. He married and had a family. He bought a House. I drank and drugged All through Hal period Payne says. Except when he got violent he Gol by. Eventually though his Wile moved Back to Indiana and they divorced. In i960 came my first detox he blacked out and found himself in a Hospital shaking. Ii was the beginning of years of shuttling from address to address some institutional Wilh nights Between on the streets. By then there were other Bitle Nesses to add to the deprivations o his childhood and he says. My drinking was my a had this attitude recalled he Rev. Jesse threats a Counselor at the Staten Island shelter called project hospitality where Payne turned up one Alvernoon carrying a knife " Hal everybody was out to get him and he had to get them the shelter turned him away and Payne Calls the Days that followed haunting the streets and Tho lorry terminal a turning Point. A restless Man. He described the wasted Days there was nothing to do. Bum Money and alter dark he sought Refuge in out of Are View ferries and he registers the horror still there s rals on those boats at at project hospitality the executive director is a divinity graduate in Denim overalls her name is Terry Trola. During an interview at the sheiler continuing As she checked the lorry terminal for newcomers needing help Sho dispatched stall questions and gently lectured a would be guest thai he d have to make a decision about his drinking and Tho pills he s supposed to Lake. Soothing or sworn she is the Calm Eye in the storm of dislocated humanity that is project Hosp Flamily. Payne had literally begged his Way into the shelter and. He said. Terry would Lake hours talking to me he can laugh now. I Call her Iho Mother Teresa of the homeless. She Hales working with her and Wilh alcoholics Anonymous he pulled himself together enough to gel another Job running a warehouse in new Jersey he was living independently. But again his drinking forestalled tha Happy ending and this time when he Lound himself Back on the Street he confront cd a corrosive fear. A Friend was Slabbed he said and i saw him bleeding out the it was time past time to sit Down and sum up. I was t getting any younger. And i started losing Taille in myself and that s something t never did. I was in Jersey in another shelter. I d had a Good Jeb Tor a year thai i d lost because of today things seem to be taking a turn Tor the better in his life again the stars and stripes pbqb15
