By Stephanie Desmon | firstname.lastname@example.org
October 16, 2008
The news, in the first week of August, jolts this loose-knit community of women participating in a trial of an experimental breast cancer vaccine. Just a year ago their friend had triumphantly shared that she was cancer-free, practically shouting it across a crowded waiting room. Now she is the first of their group to die.
Susan Marangi and Anita entered the Johns Hopkins trial together and became fast friends. They had a feeling of invincibility, feeling lucky to be part of a trial they were sure would produce a cure. They chatted about how they would spend their days growing old. Strong and independent, Anita couldn't stand to be around negativity, refusing to listen to anyone who reminded her that remission can be fleeting.
But now, Anita's friends in the trial are forced to confront their own mortality, plunged into grief and filled with panic about what her death means for their own chances of survival.
"If this thing isn't working, tell me so I can go out and find something else," says Marangi. "I won't get angry, but I need to find something else that will."
These eight or nine women aren't just drawn together for support but also by an overwhelming need to know more about the vaccine than they get through official channels. They want to know if it is working, how others are faring. At the very least they need validation that the time, energy and blood they have donated is pushing the scientific process forward.
Many say they don't get enough of that from Dr. Leisha Emens, the Hopkins oncologist who enrolled them in her two small studies. As much as the women admire Emens, the paucity of information that she offers, that she can offer, frustrates them, deepening the tension they sometimes feel between their needs and those of science.
Defining successThough bound together by hope, Emens and the women she has recruited since the study began in 2004 part company in defining success. For Emens, the success of her two trials will be measured by the degree to which the drug produces a measurable immune response in the women, not by the number who stay alive.
Her horizon extends well into the future, encompassing women who don't yet have cancer. Emens is one of the growing number of scientists testing vaccines as a means of training the immune system to attack cancer cells. The pace of scientific discovery is agonizingly slow. Emens thinks she could have a vaccine perfected in eight to 10 years, if all goes well. She is already anticipating the next stage of her research and planning to recruit a new group of women.
But for the women who have already received the vaccine, survival is the goal. Only a miracle will do, though no one has promised one. There's no time for more experiments. As Marangi says, they are the "almost dead." For all the women whose breast cancer is halted by surgery and current therapies, 40,000 die each year because it has spread through their bodies. On average, patients live just one to two years - and not necessarily healthy ones - after they are diagnosed with Stage IV of this disease.
From the time Marangi got her first shots, in December 2006, she wanted to know more about the 18 women who had taken the leap before her. Emens told her very little about them or those who came next. Emens must stay true to her research. Sometimes that is tough, especially when she has grown close to her patients. "I'm actually not allowed to answer," Emens says.
Paucity of informationClinical trials are by their nature secretive. Researchers may require years to track patients and even then may not fully understand how a drug worked. And health privacy laws are so strict Emens can't introduce participants to each other, let alone share information about them. She can't even tell individual patients whether they have had an immune response, afraid they could take the information even she doesn't fully understand yet and use it to make a bad treatment decision.
Marangi was determined to break through the barriers. Every time she went to Hopkins - and there were weeks when she drove the 16-mile round trip from her Parkville home every day - she would look out for others in the study. It might be a woman getting blood drawn at the same time, or a woman speaking to the study's research nurse. Then she'd sidle up and ask, "Are you in Dr. Leisha's study?"
The study participants she met found others. Her list grew to about nine e-mail addresses, nine of the only people on the planet who could understand her situation and give her those hints about how things were going.
The gregarious and straight-shooting Marangi, a former sales and marketing executive, became the unofficial leader and cheerleader of "Leisha's Girls," as one woman dubbed their small group. She organized a dinner last year with a half dozen of the women. She sends out upbeat messages, while others share stories of new research and possibilities for what else might keep them cancer-free, especially if this vaccine turns out not to be the answer.
And it was Marangi who shared the news of Anita's death with the others.
Marangi is one of the fortunate ones. Despite coming up on the 20th anniversary of her original breast cancer diagnosis that led to two mastectomies, despite learning two years ago that the cancer was back and had eaten a hole in her femur, Marangi showed no sign of the disease when she turned 60 in May.
It looks like it's the vaccine that is keeping her healthy, but who is to say? Emens' initial study is not intended to do more than determine whether the vaccine is safe, whether it elicits an immune response and how much chemotherapy should be given before the shots to prepare the immune system to attack cancer. It is not designed to measure whether the vaccine prolongs survival. How long Marangi's reprieve from cancer will last, no one knows. For now, what matters is that she feels good, save for a few aches and pains she gladly blames on aging.
Hope for a booster shotSome of the women have fixated on something Emens once mentioned as a future goal: a booster shot, to help sustain the body's new cancer-fighting power. They e-mail one another about it. They talk about raising money for its development. "What if this really does work and you really do need a booster and they don't figure that out until we die?" Marangi wonders.
But Emens hasn't written a grant for money to develop one. It's too early to even know what scientific questions she'd ask. She is exasperated by their obsession with it, catching herself as she starts to lash out at their persistence. She still doesn't fully understand whether what she has already given them is working.
Emens has collected some data. She shared a few tidbits at a meeting of cancer doctors in May: Of the first 27 women in the study, 15 were healthy enough to receive all four rounds of vaccine and nine others were able to receive three. Those who couldn't finish them all either had cancer that was progressing or some other medical problem that kept her from completing the treatment. The blood of nine to 14 of the women revealed some level of immune response.
Marangi pushes Emens for more. When she gets answers, they are vague and unsatisfying. "We need to know it's not a complete failure," Marangi says.
Emens understands. She just can't help. Research science doesn't allow for jumping to conclusions. Emens still needs to write a final paper on the study - and have it reviewed by peers - before anything she has learned can be legitimized. That's a year or two away. The women believe Emens wants to reveal what she knows, but as she told Marangi, "my boss would have my head" if she did.
Doctor as 'rock star'The relationship between doctor and patient, particularly in a trial like this, is complicated. For many, Emens embodies their last hope of survival. Many put her on a pedestal. One patient calls her a "rock star," for her place among a relatively small number of researchers trying to develop vaccines to fight cancer.
Her demeanor isn't warm and bubbly, they say. But if she seems detached to some of them, they're happy that she's working so hard to save lives.
"She's very efficient. We are going to discuss the important facts. We're not going to do the small talk," says Patty Fitzgerald, 44, a former research nurse at Hopkins who joined the trial in April 2007 and showed no signs of cancer in August. "I feel safe in that. I feel I can trust her."
For Emens, the women allow her a chance to put medical theory into practice. They aren't waiting for someone else to take on the risks of being first. They jump in with their eyes open. "Remarkable," she calls them. "There's something about people who are willing to do this."
As cancers go, Marangi figures she has had a pretty good run. She went 11 years cancer-free. Many doctors call that cured. So when the cancer came back two years ago, she was floored. She quickly started to feel as if some doctors give up on women once they are diagnosed as Stage IV.
Marangi's oncologist suggested Emens' trial. Marangi had done this medical pioneer thing once before, participating in a clinical trial for the cancer drug tamoxifen more than a decade earlier. She had just seen a dear friend die as she endured chemotherapy, so Marangi didn't want to head down that road. But neither was she going to sit around and wait to die. She was sold on the trial, despite a lifelong fear of needle sticks, of which there would be too many to count.
Besides, she says, her late father instilled in her a desire to leave things better than she found them.
Marangi was encouraged to learn from a friend at Hopkins about a similar vaccine being developed for pancreatic cancer, a cancer that means quick death for most who get it. The first 14 patients in that Hopkins study got their shots a decade ago. Three are still alive. Marangi says she will be one of the survivors in this trial.
As August rolls around, she still feels pretty good. She does laps in her pool, plays with her three dogs and entertains her 9-year-old grandson, the son of her only child, in town from West Virginia. Sometimes she jokes that her home life is boring. She is pretty happy that it is. She doesn't have a "bucket list"- no cruise to take, no city she must see. She feels as if she has always lived a full and fulfilling life.
But Marangi is nothing if not realistic. She knows breast cancer will kill her, probably not this month or this year but some day. She is getting her affairs in order. She has planned every detail of her funeral - down to burning the CD of disco music to be played. She has picked the spot off Cape Cod where she wants her ashes scattered after her friends toast her at a favorite bar. Her only regret is she will miss the bash.
It is in her will that Marangi inscribes her faith in Emens. Much of her money will be left to the doctor's breast cancer vaccine research.
more on the series Coming tomorrow: Dr. Leisha Emens must move on with her breast cancer research even as the women in her trials face uncertain futures.
Online at baltimoresun.com/vaccine: Watch a video of Susan Marangi talking about the vaccine and its relationship to her quality of life, and find more photos, videos and previous installments of the series.
Copyright © 2014, The Baltimore Sun