Diagnosed with cancer, a pharmaceutical executive became a patient advocate and changed drug development. It took decades

Kathy Giusti was diagnosed with multiple myeloma in 1996, when she was a 37-year-old executive at the drug company G.D. Searle. She was told she had three years to live.

Twenty-eight years later, Giusti, 65, is thriving. One reason is the patient advocacy group she founded in 1998: the Multiple Myeloma Research Foundation. The group has raised more than $600 million for research, launched nearly 100 clinical trials, and helped bring more than 15 new drugs to market. That includes Revlimid and Velcade, bedrock treatments that Giusti herself took. Between 1998 and 2024, the five-year survival rate for the disease has increased from 33% to 61%, according to the MMRF.

That’s striking progress for a relatively rare cancer that is diagnosed about 35,000 times a year in the U.S., meaning drug companies were initially less interested in developing therapies. Thanks in part to better treatments that help patients stay alive, the number of people in the U.S. who live with myeloma has increased 80% to 180,000.

Yet when Giusti sat down to write a book about her experience and to give advice to other cancer patients, drawing on nearly 30 of her own personal journals, her first instinct was not to feel a sense of triumph — it was to offer apologies to her husband, her daughter, and her son for the toll her work and her illness had taken on their lives.

“I’ve had every resource you can imagine,” Giusti told STAT. “I have a great family. I have great friends. I had good insurance. I had everything I needed to do it right. And even I messed up. A lot.”

Others might disagree. “We would not be where we are today in the treatment of multiple myeloma without Kathy’s leadership,” said Richard Pazdur, the Food and Drug Administration’s top oncology regulator. Her book, “Fatal to Fearless,” was blurbed by everyone from newsman Tom Brokaw to research scientist Eric Topol.

“That she took everything she knew about the pharmaceutical industry, having been part of it, and has really built a powerful advocacy for multiple myeloma patients, is remarkable,” said Tony Coles, who was CEO of Onyx Pharmaceuticals, a myeloma-focused biotech, from 2008 to 2014.

What experts say about Giusti’s impact is this: She changed the way medicines for multiple myeloma are developed by marshaling better science and ensuring that researchers, companies, and regulators actually talked to one another. But what also emerges from Giusti’s story is the strain of being an exceptional patient in a broken health care system — a message she relearned all over again when she developed early breast cancer in 2022 and opted for a double mastectomy, a medically controversial choice.

Even as technology has improved and new medicines have reached the market, the toughest lesson of Giusti’s story relates to the degree to which patients and their families need to take control of their own care.

“In an ideal world, it would happen seamlessly,” Giusti said. “Doctors would be able to see each other’s records and they could say, ‘Oh, OK, I see who takes care of her on the myeloma side….’ It’s not going to happen. Not because they’re bad people. They just don’t have time. They’re running from one surgery to the next surgery, and they’re doing the best they can.”

So the weight falls on patients and their families.

“I know it sounds unfair. And I know people are going to say to me, well, this is crazy. Like, why should we have to do everything?” Giusti said. “And my answer is you can keep asking yourselves why you have to do it. I would ask yourself how are you going to get it done? Because you don’t have a choice.”

That difficulty is apparent from the very first lines of Giusti’s book: “I knew the science and the industry, but I didn’t know how challenging it would be to work with the healthcare system until I found myself in it as a patient,” she wrote.

“In truth, I was scared shitless.”

What was unusual was Giusti’s reaction to that fear: She acted. Faced with a death sentence, she went to a Borders bookstore. And she bought the journals.

“I was just like, I’m going to live three years and I need my daughter to remember me.” She sat on the floor with a copy of “Harrison’s Principles of Internal Medicine” while her husband, Paul, held Nicole, then a toddler.

Giusti went on long-term disability and she, Paul, and Nicole moved from Chicago, where Searle was based, to Connecticut, where her mother and sister lived.

She was, from the start, willing to ignore medical advice. One of her first big decisions was to delay her cancer treatment, because it could have affected her fertility at a time when she wanted to pursue IVF. She wanted Nicole to have a sibling. Her son, David, was born in 1997.

But then she brought an executive’s energy to her own care. She quizzed doctors, changing from one specialist to another, and in time connecting with Kenneth Anderson at the Dana-Farber Cancer Institute, one of the top researchers in myeloma. He helped guide her care along with a team of local physicians who met her stringent desire for communication.

With her twin sister Karen, Giusti started a community fund for myeloma research. It began with donations from friends and family, but within a year it was the nonprofit now known as the MMRF.

“I wanted nothing to do with this,” Giusti said. “I was dying. I was like, I don’t want to start another company.

“But I ended up doing it.”

The personal costs of doing so were high. In 1999, she decided to attend a meeting at the International Myeloma Foundation in Stockholm on the same day that Nicole started kindergarten. Instead, her mother took Nicole so the young girl would feel loved that day.

For her part, Giusti had a rapid impact professionally. The combination of her industry experience and a patient’s desperation catalyzed drug research in surprising ways. One of the foundation’s first big victories involved a drug called Velcade.

Velcade was invented by a scientist, Julian Adams, who worked at a company that had been purchased by Millennium Pharmaceuticals, one of the biggest biotech startups at the time. It was, initially, a project executives had little interest in.

But Adams managed to get his bosses interested — and Velcade secured one of the fastest FDA approvals ever. A key moment came when, while at an MMRF meeting at a Boston Hilton, he presented data for a single patient whose disease had disappeared due to treatment with the experimental drug. Researchers at the meeting dropped what they were doing and started to plan Velcade clinical trials. That one evening trimmed nine months off of the drug’s development, Adams would later estimate. Velcade became Millennium’s main product, and was one of the medicines Giusti took.

Giusti “facilitated the getting together of investigators, facilitated the advocacy for accelerated approval, and I think that did help,” said Vincent Rajkumar, a professor at the Mayo Clinic who was in the room that night. Subsequently, he said, the MMRF became a key group for organizing clinical trials, with ideas that were discussed at its meetings also spilling out into studies being funded by other parties.

“She wanted cures,” said Rajkumar. “She had her own life at stake. She went for it and she brought people together, no question about it.”

Amid what had become a full-time job, she underwent her own cancer treatments. In 2006 she had a stem cell transplant using Karen’s bone marrow. It was the hardest moment of her treatment.

“I remember driving home with Paul and I felt so small in the world,” she said. “I just didn’t know how I was ever going to function again.” After Paul went back to work, and the kids went back to school, she sat in their house and simply felt sad. Her treatment had even made most food taste bad.

She called Karen, and insisted on going to the grocery store — something she was not supposed to do because her still-weakened immune system left her at risk of getting an infection. “You have to go with your instincts to say, this may not be what people are recommending. But for right now, emotionally, this is what I need and I have to do it,” she said.

Her 80-hour workweeks at the MMRF also came at a cost. “It was working! I was saving my life. But I wasn’t necessarily living it,” Giusti recalled. Too often, she feels, she wasn’t there. The stem cell transplant came as Nicole was in middle school, and Giusti said Nicole felt the need to be a perfect, straight-A student in order to not cause her mother additional stress.

While working on the book, Giusti apologized to Nicole for the moments she had missed. “You expected way too much,” her daughter, now 29, told her. “You had that vision of what you thought my life should be and you just had that short window to get it done.”

But Nicole also told her that she wasn’t sure things could have been different. In the book, Giusti recounts how her daughter told her that if a person has the gift to change the world for thousands of others, even if there are sacrifices, they should do it.

Things were different with David. By the time he was a teenager, the MMRF was well-established, and Giusti took time off to be around when he was in high school, managing his calendar.

As with Nicole and Paul, Giusti apologized to David for the ways in which her war on myeloma had affected the family. “Why would I need an apology?” he replied. She was sick, he said, and while she may not have always understood him, he always felt loved. “You did listen,” he told her. “I always knew how much you cared.”

Giusti’s decisions about breast cancer treatment also went against medical orthodoxy. Her cancer, diagnosed when she was 63, was early stage, and could easily have been treated with more limited surgery and drugs called aromatase inhibitors, which reduce the odds that tumors will return. But Giusti had watched Karen suffer from the side effects of breast cancer treatment. She decided on a double mastectomy.

“There’s no right treatment, only the right treatment for you,” her doctor told her. Still, she said, the surgery was tough. She ended up with a surgical infection — and she has wondered if she could have prevented it if she had communicated with her doctors better about the risks that came with her myeloma treatment.

Patient advocates, Giusti said, often make the mistake of showing up at meetings with the FDA intent only on pushing regulators to move faster instead of also providing them with information about research.

“When a patient advocacy group reaches out to the FDA, they have to see you,” she said. “So if you’re going to take that opportunity, don’t go alone.” Bring your best scientists, bring representatives from biotechnology companies.

“If you can, keep them updated on what’s going on in your field, as opposed to being the advocacy group that shows up to yell at them,” Giusti said. “That doesn’t get you anywhere.”

That attitude is perhaps why she is held in such high regard by drug company executives, regulators, and disease advocacy organizations alike.

“Working alongside Kathy is inspiring,” said Clifford Hudis, the CEO of the American Society for Clinical Oncology, which has a partnership with the MMRF. He called her “a true champion for patients” and said her work “delivered dramatically better outcomes for people affected by multiple myeloma.”

But Giusti’s approach also contrasts sharply with that taken by many other patient advocacy groups, which can view their main role in drug development as working to lower regulatory standards so that medicines can be more quickly approved — even if, as has recently been the case in amyotrophic lateral sclerosis and Alzheimer’s disease, those treatments are rapidly withdrawn.

Giusti’s efforts have resulted in new drugs, a changed landscape for multiple myeloma drug development, and data repositories of genetic information and tissues. But building that took three decades. It exacted a personal cost. And it meant years of fundraising and focus.

Cancer has been built into the biology of living things at least since the time of the dinosaurs. Fighting back doesn’t come cheap.

Correction: An earlier version of this story misstated the period during which MMRF says the five-year survival rate for multiple myeloma increased from 33% to 61%.