By: Ken Alltucker
Joel Spatt thought his skin-cancer scare was over after a doctor removed a small growth on his cheek eight years ago.
But in 2012, the Cave Creek man began to feel pain through his arm from a disk injury in his neck. He returned to a doctor and underwent tests and scans of his back.
Then he heard news he did not expect. An MRI showed that melanoma had spread through his body. The grim prognosis: He had six months or less to live.
Radiation and chemotherapy failed to keep his cancer at bay. He had signs of cancer in his brain and throughout his body. He lost weight and his muscles withered. He had blood clots in his legs.
“I didn’t know how long I had to live,” said Spatt, 64, a former surgeon who relocated from a Chicago suburb to Cave Creek three years ago. “I had to get things ready for my wife and daughter.”
His doctor suggested one more option: enrolling in a medical study of a then-experimental drug called nivolumab.
The drug is designed to unlock the body’s immune system to attack the cancer. It’s part of a new class of immunotherapy drugs about which cancer researchers are cautiously optimistic for treatment of advanced melanoma.
Every other week, Spatt would go to Mayo Clinic in Phoenix, one of the study sites, to get a dose of the drug.
Today, doctors tell Spatt he has no active signs of cancer. He has a personal trainer and began working out at a local gym in sweat-filled sessions on the elliptical machine, along with curls and bench presses to regain his upper-body strength. He resumed playing golf — his passion — while gradually increasing the number of holes and building his endurance.
Melanoma is the deadliest form of skin cancer. Its rates have doubled over the past 30 years. While most cases are treatable when caught early, the advanced form of melanoma is deadly. The American Cancer Society expects nearly 10,000 U.S. residents to die of this form of skin cancer this year. The Centers for Disease Control and Prevention recommends people take steps to minimize sun exposure to reduce skin damage from exposure to ultraviolet light.
While the number of melanoma cases has increased dramatically, doctors who treat the deadliest form of the disease say more treatment options have emerged in recent years.
“The revolution in melanoma (treatment) over the past five years has been dramatic,” said Dr. Alan Bryce, a Mayo Clinic oncologist who has treated Spatt. “There have been more advances in melanoma than any other cancer.”
More than a half-dozen commonly used immunotherapy drugs have been approved by the Food and Drug Administration or are being tested in current medical studies, according to the Melanoma Research Foundation. Doctors also are testing whether combinations of these drugs can extend lives, though trials can sometimes have substantial side effects for patients.
One example: A study of 945 patients at 137 sites around the globe tested whether the combination of two drugs, ipilimumab and nivolumab, could extend lives when used together, rather than alone. The drug tandem did extend lives, but the study also found that the drugs when used together had more side effects than either one when used alone.
The drugs are also expensive. Nivolumab, first approved by Japan in 2014, carried a price tag there of $143,000 for each year of treatment. When the two drugs are combined, one researcher estimated at the American Society of Clinical Oncology meeting this month that the total cost could reach nearly $300,000. Such costs could strain private health insurers, employers who pay for health insurance and government programs like Medicaid, Medicare and the Department of Veterans Affairs.
Dr. Lee Cranmer, a University of Arizona oncologist, said a big challenge in the coming years will be to decide how to correctly use these drugs in a way that limits side effects and tumor resistance.
“In melanoma, we went for decades where nothing really worked,” Cranmer said. “Immunotherapy has the capacity to generate long-term control of cancer.”
Cranmer emphasized that there are examples of patients who have been treated with the latest form of melanoma drugs and have kept the cancer in check for years.
He added that new treatment options are emerging. One example: A recent study showed that a version of the herpes simplex virus more effectively shrank tumor sizes compared with a control group.
Another local melanoma study from the Phoenix-based Translational Genomics Research Institute, or TGen, and Yale University will seek to tailor treatments to an individual’s DNA.
Armed with $10 million from the celebrity-infused Stand Up to Cancer, the Melanoma Research Alliance, the pharmaceutical industry and other funding sources, the TGen-Yale study aims to recruit about 100 people with melanoma that has spread to other parts of the body and resisted conventional-drug treatment. There will be a half-dozen locations across the nation that will recruit patients for this study, including Mayo Clinic in Arizona.
Organizers of the study are recruiting people whose melanoma does not have a common mutation to a gene called BRAF. Because so many existing drug treatments target this mutation, which is responsible for nearly half of advanced melanoma cases, the local research team wanted to study treatment options for others.
“It’s really providing an opportunity to these patients that they otherwise would not have had,” said Jeffrey Trent, president and research director of TGen, who will help lead the trial.
Using a technology known as sequencing, scientists plan to compare DNA and RNA from each patient’s normal and tumor cells to find the wayward gene or group of genes that may be responsible for the cancerous growth. From there, doctors can choose among more than two dozen drugs — either FDA-approved or experimental — that may be used as treatment options.
Dr. Aleksander Sekulic, a Mayo Clinic dermatologist who will be part of the trial, said one important part of the study is testing the idea that doctors can determine the best treatment for an individual based on the individual’s unique genetic makeup.
The TGen-led team initially announced the Stand Up to Cancer and Melanoma Research Alliance grant in 2011 and hoped to start the trial more quickly. But the FDA asked researchers to complete a five-patient pilot trial first to show that the concept could work.
“Nobody has really taken the time and effort to really study this approach carefully,” Sekulic said.
There will be two groups in the study: A targeted-therapy group will get a drug based on the patients’ unique genetic makeup, and a control group will get standard chemotherapy treatment. The study is designed to allow those who are assigned to the chemotherapy group to join the targeted-therapy group after a period of time.
The study will evaluate whether the targeted-therapy group gets a better response by reducing tumor size and extending lives compared with the control group.
Bryce said one of the drawbacks of a smaller study is that it is difficult to draw strong conclusions about how effective a particular targeted therapy may be. But the trial results might compel researchers to conduct a larger follow-up study if a drug signals that it may be effective against melanoma that is characterized by a unique genetic signature.
Barbara Jacoby is an award winning blogger that has contributed her writings to multiple online publications that have touched readers worldwide.