Crusader Against Cancer

Oct 14, 2014, 9:00 AM

For the 25th anniversary of the Robert Wood Johnson Foundation’s Summer Medical and Dental Education Program (SMDEP), the Human Capital Blog is publishing scholar profiles, some reprinted from the program’s website. SMDEP is a six-week academic enrichment program that has created a pathway for more than 22,000 participants, opening the doors to life-changing opportunities. Following is a profile of Jacqueline Barrientos, MD, a member of the 1994 class.

As far back as Ancient Egypt, cancer has frustrated medical practice. Papyri written around 1600 BC describe various cases, with one concluding that “there is no treatment.”

But there’s hope for patients diagnosed with chronic lymphocytic leukemia (CLL)—a cancer that attacks the blood and bone marrow—thanks to Jacqueline Barrientos, MD, who isn’t intimidated by the history surrounding the disease.

She’s busy helping to rewrite it.

Barrientos is part of a team researching new CLL therapies at the North Shore-LIJ Cancer Institute’s CLL Research and Treatment Center on Long Island. In clinical trials, the pioneering drug treatments produced unprecedented results—considerably better than those achieved with chemotherapy, and minus the brutal side effects.

“We’ve never seen response rates like this before,” says Barrientos. “It’s astonishing.” When the FDA approved the use of the new treatments earlier this year, she and her team were elated. “We’re giving life to patients who once had no hope of surviving because the cancer was so aggressive.”

To those patients, Barrientos and her colleagues are saviors. “You have given us hope where there wasn’t any,” wrote one. “I thank you, my children thank you, and my eight grandchildren thank you.”

Journey of a Thousand Miles

For Barrientos, the newly approved CLL therapy is the zenith of a long journey. “I always wanted to treat cancer,” she says.

Her passion for the work is personal: when she was 6 years old, her mother was diagnosed with refractory lymphoma, a form of cancer that is resistant to treatment.

“We didn’t have good cancer doctors in Peru,” explains Barrientos, who was born in that country’s Chincha Province. Her father, a general practitioner and surgeon, took her mother to Houston’s MD Anderson Cancer Center seeking more advanced therapies, which also were unsuccessful. “She didn’t respond, ever. She suffered a lot.”

Although she couldn’t know it at the time, her mother’s illness set Barrientos on the path she travels today. “I felt we needed to find better ways to treat the disease. We treat infections with antibiotics...why couldn’t we do the same with cancer?”

As an undergraduate in Puerto Rico, where Barrientos moved with half of her family in the wake of Peru’s political turmoil in the 1980s, she joined a pre-med club and began to explore the possibilities. That’s where she saw an ad for the University of Virginia (UVA) Medical Academic Advancement Program (MAAP), which later became the Robert Wood Johnson Foundation’s Minority Medical Education Program (MMEP, now SMDEP).

“When I was accepted to MAAP, I was jumping for joy!” she exclaims. Getting there was logistically taxing—she had to take a plane from Puerto Rico to New York City and then a Greyhound bus to UVA’s campus in Charlottesville, Va.—but once she arrived, she was introduced to like-minded people, including doctors who had once participated in the program.

“They talked about their experiences and how they made it, and I thought, ‘Oh, my God, I hope that will be me one day,'” she says. “And about 10 years ago, I was invited to go back and speak to the new students.”

Entering the Promised Land

Although she was determined to become a doctor, Barrientos felt an equally strong pull toward research. Back home, there was a line of demarcation between the two disciplines: “In Puerto Rico, most MDs are clinicians only, while the people who want to do research are PhDs.” But at MAAP, Barrientos partnered with a black physician who was a researcher as well. “I was surprised to learn I could do both.”

It’s hard when you know that there is a drug that could save someone’s life, but you’re unable to offer it because it hasn’t been approved yet.

She also found a mentor who, two decades on, continues to influence her: Moses Woode, PhD, who led MAAP and then MMEP at UVA for 25 years.

“Jacquie is an intelligent, highly motivated individual with great determination and perseverance,” says Woode. “She will continue to make outstanding contributions to health care and is a wonderful role model.”

Barrientos says Woode told the students, “Medicine is the Promised Land. You have to study a lot. You cannot slack off. You have to make an effort.”

She adds, laughing, “I just realized: his name is Moses, and he told us about getting to the Promised Land. It took me 20 years to put that together!”

Over the years, there have been many other mentors who have helped Barrientos pursue her goals. One of them, Kanti R. Rai, MD, invited her to interview four years ago for her current position at the CLL Research and Treatment Center. But Rai is no run-of-the-mill researcher; when it comes to CLL, he’s a veritable rock star. The main system used to classify stages of the disease is named for him, and he has received numerous awards in recognition of his groundbreaking clinical research and contributions to oncology.

Breakthrough Treatment

With Rai’s support, Barrientos opened up clinical trials of two new drugs, ibrutinib and idelalisib, that she believed had the potential to change the lives of their CLL patients.

“The moment that I opened up those clinical trials, we couldn’t stop people from banging on our doors to get into the trials,” she says. The drugs worked immediately, without many of the side effects of chemotherapy.

“These are targeted drugs that essentially put you into remission without the toxicity that you usually think of with chemotherapy—none of the usual vomiting or tiredness,” she explains, adding that even patients who hadn’t responded to other therapies went into remission.

When the program began to grow, Rai made sure that Barrientos had the patients and staffing she needed to continue her research. “I don’t think I could be here without his support,” she says. “I’ve been very blessed.”

The success of the trials were accompanied by disappointments. “In one of the studies, the FDA allowed crossover to the new drug only after some time, even if the patients had progressed,” says Barrientos. “People died waiting for the miracle drug.”

We’re giving life to patients who once had no hope of surviving.

“I do understand the need for clinical trials and comparisons to ensure patient safety,” she adds. “It’s just hard when you know that there is a drug that could save someone’s life, but you’re unable to offer it because it hasn’t been approved yet.”

The results of the trials were published in a series of articles in the prestigious New England Journal of Medicine, and the FDA eventually approved both drugs to treat patients with CLL.

Making Dreams a Reality

One of those patients, Ronald E. Tolkin, was diagnosed in March 2007. Although chemotherapy had lowered his white blood cell (WBC) count from a high of 264,000—the normal range is between 4,500 and 10,000—he suffered serious side effects. Then, in late 2012, his WBC count began to rise again.

“The night before I was scheduled to go into the hospital for more chemotherapy, Dr. Barrientos called me to find out if I wanted to participate in a new study,” says Tolkin. Instead of chemotherapy, he would take three pills a day of ibrutinib—a welcome change in light of his previous experience with chemo.

Tolkin began taking ibrutinib in January 2013. Within months, his WBC count started to fall, and CAT scans showed improvement in his spleen and lymph glands. “At my last visit my white blood count was 9,000, which is within the normal range,” he says. “My CAT scan shows that there is no sign of CLL.”

Ever the scientist, Barrientos is careful to note that the treatment’s longest follow-up so far is three years. “But to me, it’s very encouraging,” she says. “The hope is that we can make this cancer a chronic, treatable illness, like HIV.”

Ronald Tolkin is less reserved about her accomplishments.

“This was a life-altering event for me and my wife,” he says. “We can now plan for our future. We have eight beautiful grandchildren, ages 4 to 12. It’s our hope and desire to be able to dance at all of their weddings.

“Dr. Barrientos has made that dream a reality for us.”

This commentary originally appeared on the RWJF Human Capital Blog. The views and opinions expressed here are those of the authors.