Leukemia Awareness

Genzyme Must Further Study the Drug for Adults

NEW YORK – Genzyme Corp. must collect more data on the leukemia drug Clolar before the Food and Drug Administration will consider expanding the use of the therapy to previously untreated adults with acute myeloid leukemia.

Based on findings from a limited trial, Genzyme sought approval to market the drug to patients with the most common form of blood and bone marrow cancer in adults, the Cambridge, Mass., company said yesterday.

The FDA recommended a randomized, controlled clinical study before it would consider expanding Clolar’s use.

Clolar is approved for children with acute lymphoblastic leukemia (a cancer in which the bone marrow makes too many white blood cells) who have relapsed or are resistant to other treatments.

Genzyme plans to request a meeting with the FDA to discuss which studies will satisfy its requirements to use the drug for untreated adults.

From the Genzyme “About” page:

One of the world’s leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 11,000 employees in locations spanning the globe and 2008 revenues of $4.6 billion. In 2007, Genzyme was chosen to receive the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.

With many established products and services helping patients in nearly 100 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company’s products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune disease, and diagnostic testing. Genzyme’s commitment to innovation continues today with a substantial development program focused on these fields, as well as cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need.

BOSTON (Reuters) – Children can be treated for a common form of childhood leukemia without bombarding the brain with radiation, reducing the risk that they will suffer additional tumours and thinking problems, U.S. researchers said on Wednesday.

They said chemotherapy injected into the blood and the fluid that bathes the brain and spinal cord produced results that were just as good.

“We believe children with ALL (acute lymphoblastic leukemia) do not need to get cranial irradiation preventively, which is different from what some centers recommend,” Mary Relling of St. Jude Children’s Research Hospital in Memphis, who worked on the study published in the New England Journal of Medicine.

Radiation was once a routine therapy for acute lymphoblastic leukemia, the most common form of childhood cancer. It is still given to 20 percent of the 3,400 youngsters in the United States who are diagnosed with ALL each year in the hopes of preventing a relapse.

But the treatment can cause second cancers, stunted growth, hormone imbalances and cognitive deficits.

In the new study, Relling and colleagues found 86 percent of the 498 children given aggressive chemotherapy survived, cancer-free, for five years.

Among 71 patients who normally would have received brain irradiation in the past, the five-year survival rate was 91 percent, much better than a comparison group consisting of children who had previously received the radiation therapy for their ALL. For them, the survival rate was 73 percent.

“These are the best results reported to date,” Relling said.

The amount of chemotherapy was personalized for each child, depending in part on how many leukemia cells were detected after initial treatment.

Relling said that in the 1960s, radiation offered a big advantage in survival at a time when only 20 percent or fewer lived for five years, in part because new tumours would appear in the central nervous system. Using radiation increased the survival rate to 50 percent.

“That was a very dramatic increase in cure rates, so there was a time where almost every patient with childhood leukemia would get irradiation,” she said.

“Then there was a gradual backing away from that,” so only children in the highest-risk group got it, where the risk of recurrence outweighed the risk of serious side effects.

Acute lymphoblastic leukemia is a cancer of the white blood cells, the cells in the body that normally fight infections.