As They Lay Dying

Breast cancer activists rage at Genentech for withholding an experimental drug

Marti Nelson, 40, died first.
A physician herself, Nelson found the lump in her breast in 1987 when she was 33 and had just joined the medical staff at Kaiser Permanente in Fairfield. On her first wedding anniversary, she had a mastectomy. Two years later she discovered a lump in her remaining breast. By the summer of 1994, Nelson's cancer had metastasized to her bones, liver, and lungs. Nothing worked: not surgery, chemotherapy, or radiation — not any of the “slash, poison, and burn approaches,” as her husband, Bob Erwin, puts it.

But there was one drug, Erwin and Nelson believed, that might help. Known as HER-2/neu monoclonal antibody, a genetically engineered substance that works with the body's own immune system to control certain tumors, the drug is manufactured by Genentech, the South San Francisco biotechnology company, but is years away from Food and Drug Administration (FDA) approval.

Through her doctor, Nelson asked Genentech officials to let her have “compassionate use” of the drug on an experimental basis. She had heard of the small, but promising, studies showing how injections of HER-2/neu monoclonal antibody — referred to simply as HER-2/neu — prolonged the lives of some cancer patients without the debilitating effects of chemotherapy.

She also understood the risks. A dedicated activist, Nelson was a co-founder of the Solano Breast Cancer Support Group and a member of the Breast Cancer Task Force at Kaiser. All she wanted, Nelson said, was for people with breast cancer and other grave illnesses to have access to medicines, approved or unapproved, that might help them. All she wanted, she said, was for drug companies to do for everyone what they are now, after years of AIDS activism, beginning to do for AIDS sufferers: offer at least some experimental drugs to people with nowhere to turn.

Genentech refused to give Nelson HER-2/neu. She died on Nov. 9, 1994.
Evelyn Moulton, 51, a teacher and Ph.D. psychologist — her professional name was Ricki Dienst — also tried to convince Genentech to give her HER-2/neu. Moulton, brilliant and creative, devoted herself to expanding and shaping the Cancer Support Community, which offers support groups for those suffering from the disease. In between three courses of chemotherapy and a bone marrow transplant, she also joined Breast Cancer Action (BCA), a 5-year-old group of breast cancer survivors and activists who are taking cues from longtime health activists at ACT UP/Golden Gate.

The groups' goal, according to BCA Vice President Nancy Evans, is to bring about the prevention and cure of the disease — the nation's second most prevalent malignancy, after lung cancer. Breast cancer strikes one out of eight U.S. women. In the Bay Area, the region with the world's highest rate of breast cancer among white women — for reasons still unknown — the disease causes three deaths each day, according to the National Cancer Institute.

“We're trying to educate people and change the system so that women can make good decisions about their health care, so that people have access to the most promising treatments,” Evans says.

“Please Urge Genentech Inc. to do the Right Thing For Women With Breast Cancer,” read the ACT UP/Golden Gate press release on the Moulton case. “Evelyn Moulton must get this drug immediately,” it declared.

Genentech wouldn't give Moulton its drug. She died Aug. 1 of this year.
“We would like to know how many women have to die on Genentech's doorstep before they do even one compassionate use for HER-2/neu,” said Katie Krauss, a member of the breast cancer committee of ACT UP/Golden Gate, upon hearing the news of Moulton's death. Moulton, she noted, was too ill to qualify for Genentech's clinical trials for HER-2/neu, so — like many others — she could only get the drug if Genentech agreed to provide it, and the FDA (which it almost always does in such cases) gave its blessing.

“We are not going to let this rest,” Krauss said. “What Genentech is doing is really ugly. And believe me, this train is barreling forward and I think Ricki's death is really going to make people start working on this issue: men, women, gay, straight, good health or not.”

AIDS activists, notes Marilyn McGregor, ACT UP/Golden Gate's point person on the Genentech issue, have successfully challenged the FDA and drug companies to allow the gravely ill at least some access to AIDS drugs on a compassionate use basis (where drugs are provided to one person) or an expanded access basis (in which the company allots the drug to many people).

Women with breast cancer, on the other hand, “haven't had the constituency that AIDS activists have built,” McGregor says. Gender handicaps haven't helped, either. “Ricki told me once, and it really struck home: 'When men fight for drugs, they're called heroic, but when women fight for drugs, they're called greedy,' ” McGregor says. “Women have been too polite, too isolated, too quiet.”

Last winter, a confluence of activism helped turn up the volume. In a protest organized by ACT UP/Golden Gate — and dedicated to Nelson — a contingent of breast cancer activists and gay and lesbian groups descended on Genentech, honked horns, blasted sirens, and lay in the street to demand that the company provide HER-2/neu. That day and several times in the months following, Genentech officials met with activists, both local and nationwide, to discuss their demands. McGregor submitted specific suggestions for releasing the drug; she traveled to a Genentech meeting in Virginia to push her points home.

But Genentech was not yet prepared to offer HER-2/neu. The company had only a limited supply, officials said; in addition, it was not yet proven whether the shots were safe and effective.

“We believe we've done the responsible thing,” explains Laura Leber, a Genentech spokesperson. “This is an extremely difficult decision to make because there are so many issues.” If the company gives the drug experimentally, Leber says, it must track how the patient is doing, a responsibility that can be time-consuming and expensive. The company also has to put in place a system — typically a lottery — for deciding who and how many will get the drug (which would not generate any profit since it's not yet on the market).

“And we're also talking about a drug made through biotechnology, genetic engineering, which is difficult to make and expensive,” Leber says.

How expensive?
“I can't say. But it's very, very expensive,” she says.
But the cost of not getting it, at least in certain cases, is much higher, says McGregor.

In one study, when the HER-2/neu antibody was given to patients, 11 percent — five out of 44 patients — showed a positive response. In another study, the antibody was used in conjunction with chemotherapy, and nine out of 36 people were helped, Genentech reports.

The drug is only useful in women, typically young women, who have a very aggressive form of breast cancer in which the surfaces of certain cells overproduce the HER-2/neu protein, sparking rapid tumor growth. The HER-2/neu antibody therapy, unlike traditional chemotherapy — which kills healthy cells and cancer cells indiscriminately — stops or retards only the secretion of the offending protein, and doesn't cause hair loss, vomiting, and other toxic effects. About 20 to 30 percent of women have the type of breast cancer that might respond to the drug.

Marti Nelson suspected she was one of them. When she first inquired about HER-2/neu, Genentech told her she had to take a company laboratory test to establish if she had the right sort of cancer, says her husband, Bob Erwin. Three months went by before the company called to say, yes, she did. But it was too late, says Erwin. Nelson died the next day.

“If only they said from the start, 'There's no way you're going to get this drug — forget it,' ” Erwin says. “We could have saved three months and gone to another lab for results and tried to get the drug somewhere else.

“I think any company that experiments on human beings has the responsibility to at least provide some drugs to people who have no other hope,” says Erwin, who along with his wife, before her death, founded the Marti Nelson Cancer Research Foundation out of their Vacaville home, an organization aimed at funding support groups and providing grants to innovative cancer researchers.

“To say, 'We're just going to let you die until we can market this drug and make our profits' — that's just morally wrong,” Erwin says.

Breast cancer activist Gracia Buffleben, meanwhile, says the new struggle is to convince the FDA to require expanded access or compassionate use programs in all drug companies. “We have to stop fighting this one company at a time,” says Buffleben, an ACT UP/Golden Gate attendee since 1991. Her own breast cancer is not the type that can be helped by HER-2/neu, but she continues to fight for access for all those who might need it.

And last Monday, two working days after Moulton's death, news came of what appeared to be at least a partial victory. Genentech spokesperson Leber announced that the company had made a decision: “We've decided to go forward with an expanded access program,” Leber said.

“I'll believe it when I see the drug go into women's bodies,” says ACT UP's Krauss. “It's one thing to say something, and another thing to do it.

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