Drug produces remarkable results

Patient's blood counts normal after 2 weeks

By Dawn Schuett

Before the drug Gleevec, a bone marrow transplant was the best chance at a cure for patients suffering from chronic myelogenous leukemia, according to Mayo Clinic pediatric oncologist Dr. Peter Anderson.

Such a transplant requires a donor that's a close genetic match and is followed by high doses of chemotherapy.


"The cure comes at a price," Anderson said.

With Gleevec, patients have very few side effects, according to Anderson. Although a year's supply of Gleevec costs about $20,000 to $30,000, it is much less than a transplant, he said.

The drug was developed by Dr. Brian J. Druker of the Oregon Health Sciences University.

With CML, white blood cells in the bone marrow are overproduced. Druker researched how to block white blood cells from overproducing by using a small molecule known as "signal transduction inhibitor 571."

Novartis is now the pharmaceutical company that makes the drug under the brand name of Gleevec. The generic name of the drug is imatinib.

For Shuo Liu, Gleevec is the reason his leukemia is in remission, said Anderson and Shuo's parents, Xing Zhong and Yanping Zhao.

Shuo's first two blood tests at Mayo Clinic last year revealed that 909 cells out of 1,000 cells were leukemia, Anderson said. Two weeks after Shuo began taking Gleevec, his blood counts were normal.

"We felt comfortable sending him home back to China," Anderson said.


Since the drug is not available in China, Mayo Clinic sent a supply of Gleevec along with the family. Shuo continued to see his doctor in China regularly and each month, Anderson received an e-mail report on Shuo's blood counts.

Another family friend from Waseca took more of the drug to China in January and brought back a blood sample from Shuo for Anderson. The sample showed that only five of 1,000 cells were leukemia. The most recent tests done on this visit show that zero out of 6,000 cells have the leukemia marker.

"This is one of the more gratifying experiences I've had treating leukemia," Anderson said. "It's a remission without even losing your hair and provides hope for future small molecule drugs in different diseases."

Recently, Anderson attended an international conference on cancer in Oslo, Norway, where he talked with officials from Novartis and Pedro Rivarola, chief executive officer of the Max Foundation based in Edmonds, Wash., about helping Shuo.

Novartis and the Max Foundation are partners in an international patient assistance program, which makes Gleevec available to eligible patients in other countries.

Rivarola said the program helps 15,000 new patients a year in countries around the world.

"It is a miracle every single day," said Rivarola, whose own son, Max, died in 1991 from CML, a decade before Gleevec received regulatory approval in the United States.

Before the drug can be shipped to patients' physicians, Rivarola meets with officials in that country to get government approval. He'll go to China next week. Rivarola said he expects Shuo and other patients in China will be able to receive Gleevec through the patient assistance program starting in September.


Shuo and his parents leave Rochester for Beijing on Aug. 5. Anderson said it's uncertain how long Shuo will continue to take Gleevec.

Zhao and Zhong said they're grateful Anderson, their friends in Minnesota and the Max Foundation made it possible for Shuo to get the drug.

"I'm so thankful to the people who helped us to make this treatment available," Zhao said.

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