In 1996, Kathy Giusti, then a 37-year-old pharmaceutical sales exec with a new baby, was told she had a blood cancer called multiple myeloma and three years to live. She refused to act like her life was over. “I decided I was going to live like I was going to live, not like I was going to die," she says.
It's an attitude that has continued until this day. She and that baby (Nicole, now 23) recently went skydiving in South Africa, and Giusti frequently visits her daughter in Uganda, where Nicole works at a nonprofit focused on maternal and newborn health. “It’s going to be an incredible trip, and none of it is going to revolve around the fact that she has cancer,” Nicole says, anticipating the next one.
Giusti's survival has a lot to do with luck. Cancer is unpredictable. But it's also in part due to the philanthropic organization she founded in 1998, the Multiple Myeloma Research Foundation. The MMRF has helped accelerate ten myeloma drugs to market, from Takeda's Velcade to Johnson & Johnson's Darzalex, by speeding up clinical trials. It has pioneered investing in companies, not just giving grants, in order to get biotechs to focus on myeloma, which is diagnosed 30,000 times a year in the U.S. and kills 12,000 Americans annually. It also sponsors cutting-edge research, and pushes researchers to share data they would otherwise keep to themselves.
“It’s fair to say that patients today are living three to four times longer and many have a chronic illness with a cure on the horizon,” says Ken Anderson, director of the Lebow Institute for Myeloma Therapeutics at the Dana-Farber Cancer Institute, a past president of the American Society of Hematology and Giusti’s doctor. "Kathy and the MMRF have played a central role in spearheading this progress."
Giusti has inspired other patients to follow her. “She has truly moved mountains in the multiple myeloma field and set an example of what’s possible for the role patients can play,” says Josh Sommer, 30, the executive director and cofounder of the Chordoma Foundation and a Forbes 30 Under 30 alumnus. He says Giusti's story helped him to take on chordoma, a rare bone cancer, when he was diagnosed with it at age 18.
“We don’t have to look like a traditional nonprofit. We can take on a more aggressive role and bring stakeholders to the table to develop drugs faster, efficiently, more cost-effectively than anybody else can do," says Julie Fleshman, who became the president and CEO of the Pancreatic Cancer Action Network after her dad died from the disease. "We’ve certainly looked to Kathy and the MMRF in being the leader in doing that.”
And drug companies? They adore her. Tony Coles, the former chief executive of cancer-focused Onyx Pharmaceuticals, says he misses her energy now that his new company, Yumanity, focuses on neurodegenerative disease. "I wish I had five Kathys who were as worried about Alzheimer’s and Parkinson’s,” says Coles. “If we had five Kathys we would have found a cure for Alzheimer’s by now. I’ll even take one Kathy.”
In December 1995, Giusti wanted a referral to a fertility specialist. She felt run down, but attributed it to work stress. But her doctor spotted an abnormal blood test and put it together with her fatigue. He suspected myeloma. A bone marrow biopsy confirmed it. The doctor held her hands and told her the bad news. Well, what does it mean, she asked. How long would she live? He told her three years.
She checked with several oncologists about her options – and then decided to wait on treatment in order to have a child. “People thought I was crazy,” Giusti says, but she went through IVF anyway. “I'm not a religious person at all, but I do remember saying to myself, ‘If you make this happen for me, please God, I promise I will do whatever I can.’” In 1997, her son David was born, and the Giustis moved from Illinois to Connecticut to be closer to family.
She and her twin sister Karen founded the MMRF in 1998 because of her kids, she says. “I wanted to live long enough that they'd remember me.” They raised roughly $450,000 at their first fundraiser. Kathy started having meetings to get industry scientists, academic researchers and clinicians working together.
In October 2000, the power of just having those meetings became apparent. Julian Adams, a curly haired chemist at Millennium Pharmaceuticals in Cambridge, Massachusetts, visited an MMRF meeting in Boston to present data from a single patient who had taken the company's drug – and whose cancer had become undetectable. One physician told him he hadn't seen data like that in 25 years. Meeting Giusti for the first time, Adams asked if he could take over the meeting to plan a clinical trial. All the right doctors were there. She said yes, and he says it saved him nine months. “I was eternally indebted to her,” Adams says. “We just saved so much time.” The drug, Velcade, was approved in 2003 and became a billion-plus seller for Millennium, which was later bought by Takeda.
Another drug was showing promise around the same time: thalidomide, infamous for causing birth defects when it was used to treat nausea in pregnant women. Summit, New Jersey-based Celgene was selling it as a leprosy drug, but an oncologist named Bart Barlogie had tried it in myeloma patients with fantastic results. Keith Stewart, director of Mayo Clinic's Center for Individualized Medicine, remembers an MMRF meeting right after 9/11—the doctors traveled on empty planes—in which the group hashed out how a new research consortium could share tissue samples, produce genetic data and create a network to run clinical trials better. It helped speed studies of thalidomide and of Celgene's follow-up drug, Revlimid. The success of those drugs is one reason Celgene now has a $65 billion market capitalization. The MMRF's research consortium has grown from 4 cancer centers to 25.
Onyx Pharmaceuticals bought the drug that would become Kyprolis in 2009 for $276 million. The MMRF helped with the clinical trials. It was approved in 2012, and Onyx sold to Amgen for $10.4 billion the next year. “With hindsight, we were also kind of kicking ourselves. These drugs were doing incredibly well, and we had not done anything in terms of getting any kind of [financial] return,” Giusti says. So the MMRF started investing in companies, among them: Constellation, Epizyme, Karyopharm, Astex, and Intellikine. The investments got the companies interested in myeloma, but also offered the MMRF the potential of a financial return.
By 2003 the foundation became so successful that Kathy and her husband feared it would have a side effect: that they could no longer keep her cancer secret from her kids. They sat them down and gave a rehearsed speech. “It went way over our heads,” Nicole remembers. “I remember going up and hugging my mom and saying, ‘Is my mom going to die?’” David had a different question. Sensing an opportunity, he looked at his mom and asked, “Can I have some ice cream?”
Giusti took Velcade and Revlimid, the very drugs she'd helped speed development on. But her cancer did progress. In 2005 she received a stem-cell transplant from her identical twin sister Karen. Leaving her kids for three-week stretches was the worst part. And then she returned home hairless, so frail that she looked like she might break at her son's sporting events. "When I walked into that first basketball game, nobody knew who I was," she says. David shaved her head. The kids encouraged her to wear scarves that brought out her eyes because her wig itched. But it worked: The transplant put her in remission.
Giusti thinks she did so well in part because of a genetic mutation her cancer had. At first she was devastated to hear she had it. Generally speaking, it meant she would die sooner. But it also seemed to indicate she was more likely to benefit from Velcade. On to the MMRF's next quest: funding DNA sequencing to understand myeloma. In 2011 an MMRF-funded study of DNA sequences from 38 patients was published in Nature. The result showed that some patients with myeloma might respond to the drugs Zelboraf and Tafinlar. Early clinical trial results are promising. A much bigger DNA-sequencing study of myeloma patients launched in 2011.
Robert Kraft, the billionaire owner of the New England Patriots, gave the Harvard Business School a $20 million grant focused on genetic medicine in 2016. Giusti is helping to run it. One goal: combine the efforts of patient groups like PanCAN and the MMRF. “We're appalled at how much we've all spent doing the exact same thing," she says.
Every two months Giusti goes to Dana-Farber for her blood tests with her husband, Paul. “You would think we would get used to it, but we don't,” she says. As she drives back to Connecticut, she thinks about what she'll do if the cancer comes back. And how to make the odds better.
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