Southern Research cancer drug approved

Friday, December 31, 2004

DAVE PARKS

News staff writer

The Food and Drug Administration has approved a pediatric leukemia drug developed by Southern Research Institute of Birmingham. It is the first new treatment in a decade for children who have been stricken with the life-threatening disease.

The drug clofarabine, now known commercially as Clolar, was approved Tuesday by the FDA after being sped through the regulatory process. Marketed by Genzyme Corp., the drug will be used for children with acute lymphocytic leukemia, ALL, a cancer of the blood and bone marrow.

It is the sixth anti-cancer drug invented by Southern Research, which holds the patent on clofarabine and will draw royalties from sales. The drug's journey from the lab bench to the bedside of patients has taken more than a decade.

"It's been a long road from where we started to where we are now," said Jack Secrist, vice president of drug discovery at Southern Research.

Clofarabine was initially discovered in the 1980s at Southern Research by Secrist and another well-known researcher, John Montgomery, who died in May. It was first synthesized in a Southern Research lab by Anita Fowler, a chemist.

Southern Research sold development and commercialization rights for clofarabine to Bioenvision Inc., a drug company in Europe. Bioenvision took on a North American development partner, ILEX Oncology Inc., a subsidiary of Genzyme.

Designed for children:

The drug's approval was based upon results from a U.S. trial involving 59 children who had been treated unsuccessfully with other drugs for ALL. A third of the children responded to the treatment with clofarabine, with 20 percent achieving complete remission.

The first pediatric patient was treated with the drug in August 2000. The FDA put the drug on the fast-track for approval a year ago because early trials were promising. Secrist said the FDA sped up the process because doctors needed new options for treating pediatric leukemia.

Genzyme said in a prepared release that it expects to quickly make the drug available, possibly in January.

Dan Sims of Vestavia Hills - whose 5-year-old daughter, Janie, died from ALL two years ago - said approval of the new drug is a major advancement.

"I wish it would have been available two years ago," he said. The drug could have helped his daughter, said Sims, who now raises money to fight childhood cancer through the Janie Sims Children's Foundation.

Sims said ALL is a rapidly progressing disease. When diagnosed, doctors hit it fast and hard, hoping to knock it into remission. Existing treatments are often successful, but sometimes the cancer comes back.

Now doctors have a new weapon, Sims said. "That's very significant."

ALL is the most common form of pediatric leukemia. About 3,800 cases are diagnosed annually in the United States, mostly in children, according to the American Cancer Society.

Secrist said the drug also may be effective against other, more common cancers, as indicated in animal studies. The drug works by attacking DNA in cancer cells and is being tested in other cancer trials.

"The drug is showing very nice effects in certain adult leukemia as well," Secrist said. "I think this will gradually be found to help more and more folks."

E-mail: dparks@bhamnews.com




Southern Research cancer drug approved Friday, December 31, 2004 DAVE PARKS News staff writer The Food and Drug Administration has approved a pediatric leukemia drug developed by Southern Research Institute of Birmingham. It is the first new treatment in a decade for children who have been stricken with the life-threatening disease. The drug clofarabine, now known commercially as Clolar, was approved Tuesday by the FDA after being sped through the regulatory process. Marketed by Genzyme Corp., the drug will be used for children with acute lymphocytic leukemia, ALL, a cancer of the blood and bone marrow. It is the sixth anti-cancer drug invented by Southern Research, which holds the patent on clofarabine and will draw royalties from sales. The drug's journey from the lab bench to the bedside of patients has taken more than a decade. "It's been a long road from where we started to where we are now," said Jack Secrist, vice president of drug discovery at Southern Research. Clofarabine was initially discovered in the 1980s at Southern Research by Secrist and another well-known researcher, John Montgomery, who died in May. It was first synthesized in a Southern Research lab by Anita Fowler, a chemist. Southern Research sold development and commercialization rights for clofarabine to Bioenvision Inc., a drug company in Europe. Bioenvision took on a North American development partner, ILEX Oncology Inc., a subsidiary of Genzyme. Designed for children: The drug's approval was based upon results from a U.S. trial involving 59 children who had been treated unsuccessfully with other drugs for ALL. A third of the children responded to the treatment with clofarabine, with 20 percent achieving complete remission. The first pediatric patient was treated with the drug in August 2000. The FDA put the drug on the fast-track for approval a year ago because early trials were promising. Secrist said the FDA sped up the process because doctors needed new options for treating pediatric leukemia. Genzyme said in a prepared release that it expects to quickly make the drug available, possibly in January. Dan Sims of Vestavia Hills - whose 5-year-old daughter, Janie, died from ALL two years ago - said approval of the new drug is a major advancement. "I wish it would have been available two years ago," he said. The drug could have helped his daughter, said Sims, who now raises money to fight childhood cancer through the Janie Sims Children's Foundation. Sims said ALL is a rapidly progressing disease. When diagnosed, doctors hit it fast and hard, hoping to knock it into remission. Existing treatments are often successful, but sometimes the cancer comes back. Now doctors have a new weapon, Sims said. "That's very significant." ALL is the most common form of pediatric leukemia. About 3,800 cases are diagnosed annually in the United States, mostly in children, according to the American Cancer Society. Secrist said the drug also may be effective against other, more common cancers, as indicated in animal studies. The drug works by attacking DNA in cancer cells and is being tested in other cancer trials. "The drug is showing very nice effects in certain adult leukemia as well," Secrist said. "I think this will gradually be found to help more and more folks." E-mail: dparks@bhamnews.com Copyright 2005 al.com. All Rights Reserved.