How MSK Donors Drive Early Research to Help People with Cancer

Miguel Perales and his patient Jay

Jay O'Brien (right) came to MSK to be treated by Miguel Perales for leukemia.

Creating a new drug or testing an existing drug for a new purpose takes time. It first requires an idea and years of laboratory tests. After that, many more years of clinical trials follow to make sure the treatment is safe and effective for people. Only then will the US Food and Drug Administration approve a drug so it is available to the people who need it.

Once studies begin to show promising results, the National Institutes of Health (NIH) and drug companies may invest in further research. But those results build on the early phases, including investigations and experiments in the lab. Without the initial work, drug discovery would cease. Drug companies and the NIH do not usually fund this time-consuming and important exploratory work. Yet these big risks can yield the biggest rewards in cancer medicine.

Philanthropic donations big and small are the main source of support for early-stage research. Memorial Sloan Kettering’s doctors and scientists rely on thousands of philanthropic partners. These generous donors live all around the world, giving what they can to support curiosity-driven explorations and, ultimately, improve patient care.

How Donations Drive One Scientist’s Research — and Why He Runs to Raise Funds

Physician-scientist Marcel van den Brink is a world leader in bone marrow transplants (BMTs). Like all scientists at MSK, he relies on various funding sources to conduct his most creative, forward-thinking work. Recently, he and medical oncologist Jonathan Peled found that having a healthy balance of microorganisms in the body before a BMT is associated with higher survival rates after the procedure. This discovery highlights for people undergoing BMTs the importance of achieving this balance through diet and other means.

Previously, Dr. van den Brink’s lab conducted a clinical trial in which patients received a weekly injection of recombinant interleukin-7 (IL-7) after a bone marrow transplant. This protein plays a role in T cell development and survival. The goal was to improve the recovery of their immune systems. The study proved successful, and this treatment is in further development at MSK.

Dr. van den Brink appreciates the impact that philanthropy makes on research and has personally raised money by running marathons with Fred’s Team since 2005. Fred’s Team is a community of runners committed to fundraising in support of cancer research at MSK. Dr. van den Brink knows firsthand the importance of this support.

“Because NIH funding continues to be low, we are more and more dependent on private donations to keep our research going,” he says. “Philanthropy is especially important in investigator-initiated trials, which come about because of an exciting idea. Donations allow us to explore these innovative ideas and make a real impact on our ability to advance cancer research.”

One Patient’s Story of Overcoming Leukemia

Jay O’Brien is one of the many people who have directly benefited from Dr. van den Brink’s research. A former college athlete and coach who has always maintained a healthy, active lifestyle, Jay is an executive in the athletic footwear business. He and his wife enjoy taking long walks together. In November 2008, during one of these walks, he became uncharacteristically tired and breathless. He was unable to make it up a hill he had climbed many times.

“My heart was pounding, but I wasn’t sweating,” he says. “From playing soccer and basketball, I knew that when your heart rate goes up, you usually perspire. But in this case, my heart rate went up like crazy, and I wasn’t sweating. I knew something was wrong.”

Jay’s primary care doctor in New Jersey took one look at him and ordered a blood test. On the drive home from the doctor’s office, he got a phone call telling him to go to the local hospital immediately. But no one told him why.

After a long wait in the emergency room, someone finally told him: “You have leukemia.”

At the advice of his colleagues, Jay transferred to MSK the next morning. He was admitted on November 15 and immediately began chemotherapy infusions.

As Christmas approached, Jay was told he would likely still be in the hospital over the holidays. He remembers saying to himself, “That’s OK. As long as I continue to get better, that’s the only thing that matters to me. If I have to be here in the hospital, I have to be here.”

And then, just days before Christmas, he and his family received some happy news: He was well enough to go home. “It was a really strange feeling, to all of a sudden be out after being in the hospital for so long. I had to take a great deal of caution, and I couldn’t be around anyone except my wife and children.”

Philanthropic donations are the main source of support for early-stage research at MSK.

Although Jay was doing well, his doctors told him that a BMT would greatly reduce his risk of the cancer coming back in the future, from 50% to closer to 5%.

Jay is one of six siblings, and one of his brothers was a perfect bone marrow match. After considering the risks of this complex procedure, Jay and his wife decided to proceed with the transplant under the care of Miguel-Angel Perales.

After a BMT, the risk of infection is high. Dr. Perales told Jay about the clinical trial of IL-7 that he and Dr. van den Brink were running to boost infection-fighting T cells and strengthen recovering immune cells. IL-7 was initially developed to strengthen the immune systems of people with AIDS, and Drs. van den Brink and Perales and their collaborators were testing how well it worked after bone marrow transplants.

Jay decided to enroll in the trial, and his immune system recovered. He has now been in remission for nine years. “I felt pretty special to be part of this trial,” he says.

Jay is grateful to so many people for helping him get well — his wife, children, and brother, as well as the doctors and nurses at MSK and all the donors who support blood cancer research. “Their gifts allow doctors to save lives, including mine,” he says. “They give MSK researchers the ability to keep trying to find cures for this crazy thing called cancer.”