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Eculizumab Trial Begins for PNH Patients
November 1, 2004 - Alexion Pharmaceuticals, Inc. has initiated the treatment phase in the pivotal Phase III TRIUMPH trial, to evaluate effectiveness of the investigational drug eculizumab in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). The study is expected to enroll approximately 75 patients in the U.S., Canada, Europe and Australia. The study includes an observation phase for each patient prior to treatment.
A companion safety trial, called SHEPHERD, will be aimed at generating additional safety data with eculizumab in hemolytic PNH patients with a history of transfusion. The study is expected to enroll approximately 75 patients in the U.S., Canada, Europe and Australia. Alexion expects to initiate the SHEPHERD trial in the coming months.
Alexion Reaches Agreement with FDA for Phase III Study of Eculizumab
Tuesday, July 20, 2004 - Alexion Pharmaceuticals, Inc. announced today that it has received written confirmation from the U.S. Food and Drug Administration (FDA) indicating agreement with the two protocols constituting the pivotal Phase III program of its investigational drug eculizumab for the chronic orphan blood disorder Paroxysmal Nocturnal Hemoglobinuria (“PNH”). The Phase III program includes the TRIUMPH pivotal trial together with a companion safety trial called SHEPHERD. The agreement for the Phase III program was reached under the FDA’s Special Protocol Assessment (SPA) process, a procedure by which the FDA provides official evaluation and guidance on proposed protocols for pivotal Phase III clinical trials. It is expected that, if successful, the trials will complete the filing package that will serve as the primary basis of review for the approval of a Biologics License Application for the PNH indication.
Alexion will host a conference call and audio webcast to discuss matters mentioned in this release today, July 20th, at 9:00 a.m., Eastern Time. To participate in this call, dial 913-981-5509, confirmation code 595961, shortly before 9:00 a.m. The audio webcast can be accessed at: www.alexionpharm.com. A replay of the call will be available for a limited period following the call, beginning at 12:00 p.m., Eastern Time. The replay number is 719-457-0820, confirmation code 595961.
Find out more by visiting Alexion web site at www.alexion.com. Also, review recent article posted on BioWorld Online at www.bioworld.com.
Effect of Eculizumab in Patients with PNH
Thursday, February 5, 2004 - Results from a clinical trial study of Eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) were reported in today's issue of the New England Journal of Medicine. Eleven transfusion-dependent patients were enrolled in this 3-month study. All experienced less red blood cell destruction, reducing the frequency of blood transfusions and improving the quality of life in patients. Eculizumab is an investigational drug therapy being developed by Alexion. The drug was granted Orphan Drug Status from the U.S. and European regulatory agencies to treat PNH.
For more information, see Alexion's press release.
Read a summary of the New England Journal of Medicine article on Eculizumab.
Kailee Wells Improves After Bone Marrow Transplant
ALBUQUERQUE, N.M. - February 8, 2005 - Kailee Wells, the 8-year-old Albuquerque girl who went through a bone marrow transplant two weeks ago in a Wisconsin hospital, appears to be improving.
The girl suffers from aplastic anemia, a rare blood disease that has slowly destroyed her health. She had been searching for a suitable donor for more than a year.
Her parents told Action 7 News Monday that Wells' white and red blood cell counts are improving. They also showed pictures of Wells as she prepared for some tests on Saturday.
Previous Stories:
March 9, 2004: Kailee Wells Fights For Life
February 21, 2004: Bone Marrow Drive To Be Held Saturday For 7-Year-Old
November 16, 2003: Family's Long Search For Donor May Be Over
November 1, 2003: Mom Circumnavigates Globe For Daughter
February 25, 2003: Possible Donor is Not a Match for Kailee Wells
Neighbors rally for ailing young woman with aplastic anemia
MARTY MINCHIN
Mar. 06, 2005 - Madison Ridge subdivision in Stallings has kicked into high gear to help a young neighbor who is suddenly facing huge medical bills.s
Amy Harris, 25, was diagnosed with aplastic anemia in mid-February. That means her bone marrow has stopped producing enough red blood cells, and she is undergoing expensive immunosuppressive therapy in Chapel Hill.
Her neighbors mobilized immediately when learning that Amy's medical bills could be as much as $750,000, said Margie Huntley, who also lives in Madison Ridge.
"She is such a sweet person," Huntley said. "This has just gripped Madison Ridge, and people have just come together trying to help."
One neighbor sold candy bars door-to-door. Huntley raised more than $500 with a letter she sent to her family and friends about Amy. Another neighbor donated $1,000. Lynn Wren is organizing a golf tournament fund-raiser in May.
Amy and her husband Shawn, 30, have a 6-year-old daughter and a 4-year-old son. Huntley said that Shawn works from home, and the couple have medical insurance for their children but not for themselves.
On March 26, the Harrises' friends are hosting a community-wide spaghetti dinner to raise more money for Amy's medical bills.
The dinner will be from 4 p.m. to 7 p.m. at Mt. Harmony Baptist Church, and tickets cost $7. The organizers also are looking for volunteers to help serve and clean up the dinner and to donate desserts, rolls, pasta and spaghetti sauce.
If you are interested in helping with or attending the spaghetti dinner or with other fund-raisers for Amy, contact Jennifer Smith at (704) 882-3531.
Bone marrow donor drive scheduled for San Luis firefighter diagnosed with blood disease
BY PAIGE LAUREN DEINER, STAFF WRITER
Isidro Lopez stands surrounded by pictures of his eight children. Lopez, who suffers from aplastic anemia, is in need of a bone marrow transplant.
March 1, 2005 - Isidro Lopez, who has aplastic anemia, says he's not afraid of death. He says he knows God is on his side, even if the odds of survival are against him. What scares him is leaving his eight children without a father, should he die.
If the 34-year-old San Luis firefighter and Emergency Medical Technician doesn't receive a bone marrow transplant soon, he will die.
"I have faith in God," he said. "I have never been scared. I know it's going to work."
Finding a bone marrow match is like finding a needle in a haystack.Lynn Hooker, emergency medical service coordinator for Yuma County, said there is a 1 in 3,000 chance that a match will be found among Hispanics and a 1 in 1 million chance of finding a match in other races. A matching donor would enable doctors to stop the aplastic anemia, a disease that attacks the body's red and white blood cells.
Lopez and the San Luis and Yuma fire departments are trying to increase the odds of finding a match by holding a bone marrow donor drive on Thursday from 9 a.m. to 7 p.m. at the Fernando Padilla Community Center, 800 E. Juan Sanchez Blvd. in San Luis and the city of Yuma Fire Station 2, 3284 S. Ave. A.
Hooker said that at the drive potential donors will have one vial, or about two teaspoons, of blood drawn. The blood will be sent to a lab in Phoenix where it will be analyzed for potential matches. Lopez said if a match is found the donor will undergo a series of tests to ensure they are healthy enough to give marrow. Then the marrow will be extracted from the donor's hip. He said there are no side effects for the donor.
Sitting in an armchair Monday, in the children's playroom in his home — where Lopez said he spends most of his time — he talked about his children.
"My life is work and kids," he said, pointing to a picture of one of his daughters. He explained how he dropped some of his children off at day care and others at school this morning. Smiling, he added that his oldest, 11, is in the gifted program and is beginning to take college classes.
Lopez said he has told his children that he is sick, but that they can't seem to grasp that he could die. He said they ask about the large bruises that covers his arms and legs,but "they don't understand the whole meaning."
Saddened by his children's tears and worries, Lopez said he usually wears long-sleeved shirts so the evidence of the disease is hidden.
For him, the most difficult part of having aplastic anemia is the effect its having on his family. He said "the hardest part is seeing my wife suffer. She worries too much."
If a bone marrow match is found these worries will disappear. Lopez is optimistic. "One year from now I will be raising my kids and everything will be back to normal," he said.
Paige Lauren Deiner can be reached at pdeiner@yumasun.com or 539-6872.
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