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Wantagh Man Trying to Persuade Pharmaceutical Companies to Give Him Access to an Experimental Drug: Cancer Patient in a Bind

Posted on: Friday, 2 June 2006, 15:00 CDT

By Kathleen Kerr, Newsday, Melville, N.Y.

Jun. 2--A Wantagh man battling cancerous tumors in his organs and bones hopes a translucent blob of a sea creature can help extend his life.

But so far, Robert Kudlack, 25, has had no luck accessing the secrets of the sea squirt - a floppy, tube-shaped Caribbean organism that clings to the underwater roots of tropical mangrove trees.

Kudlack, on leave from his job as a supervisor for the U.S. Department of Agriculture, has Ewing's sarcoma, a rare cancer. He has undergone traditional chemotherapy, radiation and spinal surgery, but they have not defeated the tumors that have spread from his spine to his lungs, kidneys, shoulder blades and thigh bones.

"It's an uncommon cancer for someone my age," Kudlack said.

Ewing's sarcoma usually develops during puberty at a time of rapid bone growth but is sometimes seen in adults. Now Kudlack is hoping the pharmaceutical maker Johnson & Johnson can help him.

The New Brunswick, N.J., company and PharmaMar, a Spanish firm, have developed a synthetic version of ET743, a compound found in the sea squirt. The companies are testing an experimental drug, Yondelis, which contains the compound.

Yondelis, delivered to patients as an infusion, binds to a tumor's DNA and interferes with cell repair machinery, slowing a tumor's growth. Johnson & Johnson is testing the effect of Yondelis on soft-tissue sarcomas.

Kudlack does not have a soft-tissue sarcoma. Still, his oncologist, Dr. Joe Phillips of Winthrop University Hospital in Mineola, thinks Yondelis could help him. Yondelis is not available by prescription because it is not yet approved by the Food and Drug Administration.

Phillips has asked Johnson & Johnson to let Kudlack use Yondelis under a special access program. He acknowledges Yondelis is not a cure but argues it might buy Kudlack some time.

So far, Johnson & Johnson has said no.

Kudlack's case highlights the roadblocks desperate patients can encounter when trying to persuade pharmaceutical companies to provide access to experimental drugs. While companies have programs that allow some access to experimental drugs, they also have strict guidelines for such use.

Johnson & Johnson's position is that Ewing's patients are not eligible for Yondelis trials, which include a Phase 2 study for soft-tissue sarcoma and a Phase 3 study for ovarian cancer. Phase 1 trials concentrate on drug safety. Phase 2 and 3 trials concentrate on a drug's effectiveness.

"It's really important we maintain strict medical and ethical standards," said Doug Arbesfeld, a Johnson & Johnson spokesman. "You still have to have reasonable belief that the drug is going to work for a very sick person."

Arbesfeld explained that if Kudlack were allowed to enter the soft-tissue sarcoma trial, it would skew the results.

But what about letting Kudlack use Yondelis outside the trials, as his doctor has requested?

Pharmaceutical companies do sometimes permit experimental use of drugs outside of trials. But Arbesfeld said there's little evidence Yondelis can fight Ewing's. And Arbesfeld pointed to concerns about possible unknown side effects.

But Phillips says some Ewing's patients who were included in past Yondelis trials were helped by the drug. "Each patient is different," Phillips said. "Some of the patients who have been treated with it have tolerated it very well."

However, Dr. Denise Williams, an oncologist and Johnson & Johnson's medical leader for Yondelis, said while there may be a "suggestion of benefit, we really don't have enough data."

A new trial planned by a group of hospitals and oncology centers will include Ewing's patients, Williams said, but it hasn't been finalized yet. And the age range to be studied would be younger than Kudlack, although he could apply for a waiver.

Recently, an Arlington, Va., group called the Abigail Alliance for Better Access to Developmental Drugs, which had sued the FDA, won a legal victory for patients seeking access to experimental drugs.

The Washington, D.C., Circuit Court of Appeals ruled 2-1 May 2 that terminally ill patients have a right to access experimental drugs that might save their lives once they have passed Phase 1 safety trials.

The appeals court overturned a lower court ruling and returned the case to a federal district court for a hearing. The FDA opposes the Abigail Alliance, saying eased access to experimental drugs might put patients in danger.

But the appeals court ruling has no effect on Kudlack. He still wants to try Yondelis, and time is short.

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Copyright (c) 2006, Newsday, Melville, N.Y.

Distributed by Knight Ridder/Tribune Business News.

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Source: Newsday, Melville, N.Y.

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