Jupiter Medical Center is using the latest medical breakthrough—gene therapy—to improve the quality of bladder cancer patients’ lives and stop the disease from spreading.
It’s common knowledge that treating cancer is a complex undertaking that often involves intensive regimens, such as chemotherapy, radiation treatment, surgery—or all three. These remedies are often accompanied by physical and emotional side effects that can linger, even when the procedures are successful.
Now gene therapy is offering hope. A team of multidisciplinary specialists at the JMC Anderson Family Cancer Institute is using gene therapy to combat bladder cancer—the seventh most-common male malignancy and tenth worldwide, according to the National Institutes of Health (NIH). This innovative treatment protocol is being led at by urologist Dr. Patrick Tenbrink; Dr. Ryan Devine, a medical oncologist and hematologist; and Dr. Matthew Hartwig, oncology pharmacy director.
“This is a groundbreaking advancement. It’s been a major unmet need within our community,” Hartwig says. “It’s a great therapy that enhances access to advanced care for our patients and improves their quality of life.”
Gene therapy began to emerge in 1928 when British bacteriologist Frederick Griffith determined that bacteria present in a strain of pneumonia could be altered, allowing genes to transfer from one bacterium to another, according to the NIH.
Over the next several decades, scientists and medical professionals doggedly conducted genetic research related to genetics, figuring out how to modify DNA to combat diseases along the way. In 2015, the U.S. Food and Drug Administration (FDA) granted its first approval for gene therapy to treat acute lymphoblastic leukemia for pediatric and young adult patients. Since then, many more have been approved for an array of illnesses, including blood disorders (sickle cell, lymphoma, and hemophilia), skin cancer, muscular dystrophy, and spinal muscular atrophy.
The NIH describes gene therapy as “the introduction of nucleic acid into the hosts cells to achieve a therapeutic effect.” Tumor cells are directly targeted to restore mutated “suppressor functions,” which stop uncontrolled cell division that may promote cancer. The treatment also moderates the immune response which defends against tumors, the NIH says.
At the JMC Anderson Family Cancer Institute, the protocol involves injection of Adstiladrin (the brand name for the generic nadofaragene firadenovec). The only FDA-approved gene therapy delivered directly in the bladder for non–muscle-invasive bladder cancer, it is used solely to treat malignancies on the inner lining of the bladder before they breach muscle.
Determining whether a patient is suitable for treatment begins with diagnosis. A patient may describe certain symptoms experienced during urination—the presence of blood, discomfort, frequency, or urgency—indicating that diagnostic testing is necessary. A urologist then conducts a cystoscopy, during which a video scope is inserted through the urinary tract to look for abnormalities in the bladder.
Anomalies may appear as “carcinoma in situ,” which means a “group of abnormal cells that are found only in the place where they first formed in the body [and did not spread],” according to the National Cancer Institute (NCI). Imaging may also detect a raised, tumor-like mass that has not yet invaded muscle.
If abnormalities are discovered, the patient is assessed for risk factors, including smoking, occupational exposures to chemicals or radiation, and their medication history, says board-certified, fellowship-trained Devine.
When a patient is deemed an appropriate candidate, the treatment designed to prevent the abnormal cells or mass from growing and spreading is set to begin. "When we have superficial bladder cancer, it’s at risk for superficial recurrence and/or can evolve into muscle-invasive bladder cancer,” Devine says. When that happens, "the cancer is at risk for becoming metastatic and spreading [outside] to either lymph nodes, internal organs, or bone."
Last year, more than 83,000 bladder cancer cases were reported in the United States, making up 4.2 percent of all cancer cases, according to the NCI. In 2024, there were about 16,800 deaths from bladder cancer—about 2.8 percent of all cases. The survival rate between 2014 and 2020 was 78.4 percent.
The therapy is administered by injecting the liquid medicine—5 milliliters, or about a teaspoon—into the organ via the urethra. It attaches to the cells and delivers an encoded gene that blocks bladder cancer growth; the patient’s body is rotated to ensure complete coverage of the drug, Devine says. The outpatient procedure lasts about an hour and uses a local anesthetic. The side effects are mild, Devine says, consisting mostly of fevers and chills for a few days.
A gene called interferon alpha-2 “gets taken up only by the cell lining, causing a continuous immune effect to surveil and hopefully prevent new superficial bladder cancer,” Devine says. One treatment of Adstiladrin lasts for three months before a patient is reassessed.
Before Adstiladrin, a medicine called Bacillus Calmette-Guérin (BCG) was used for treatment and applied in the same manner. “BCG is an immune stimulatory, so it basically causes a local reaction in the bladder that helps the immune system to look for and kill off any potential developing or abnormal cancer cells,” Devine says. But the effectiveness of BCG, which is used at Anderson Family Cancer Institute, can be short-lived, with between 30 and 70 percent of patients experiencing a reoccurrence of the superficial cancer, Devine says.
And that’s the challenge when treating the disease. “Bladder cancer has such a high propensity for reoccurrence in the same spot it was before or in spots within the bladder,” says Tenbrink, medical director at the JMC Barb and Joe Charles Center for Urology.
BCG is typically applied once a week for three to six weeks followed by a surveillance cystoscopy. This process is repeated every three to six months for up to three years, Tenbrink says. If conditions persist, the cancerous material is scraped and then the process is repeated. Sometimes, liquid drugs used for chemotherapy are used in tandem with BCG to rinse the inside of the organ to help strengthen the regimen.
“This cancer can often come back, especially if the BCG is stopped or ineffective,” he says. Sometimes patients will discontinue the BCG treatment because of its frequency or strong side effects such as painful urination and other symptoms that prompted medical help in the first place, Tenbrink says.
In the 10 years that gene therapy has expanded and evolved, it’s been considered by the medical community as a highly promising option. And when it is used, caution and vigilance are of great importance. “It’s a new approach to [cancer] treatment and may have risks,” says the NIH.
Development—funding, research, and clinical trials—can take six to 12 years before one product can be submitted for approval by the FDA. On top of that, it’s expensive. The NIH estimates that it costs nearly $2 billion on average to get just one treatment up and running.
The FDA and the NIH are closely watching the gene therapy clinical trials underway in the U.S., ensuring patient safety is a top priority during research to expand gene therapy options for diseases and cancers for which there are few options.
Thus far, three patients who have received the Adstiladrin gene therapy are responding well.
Most insurances, including Medicare, cover the treatment. “It’s an interesting medication. It must be stored in an ultra-low freezer at -80°C,” Hartwig says. “One of the major hurdles to getting the medication and being able to provide it in this community is the supply chain—from ordering it, having it shipped, receiving it, and storing it properly.”
But those concerns are outweighed by the results, and Jupiter Medical Center is poised to deliver and grow the program. Since starting the use of Adstiladrin, the Anderson Family Cancer Institute has introduced other therapies for conditions such as multiple myeloma and lung cancer, Hartwig says.
“Jupiter [Medical Center] is one of the only places locally that is offering Adstiladrin, so it's unique for us to have that opportunity for our patients,” Tenbrink says. “I think it’s going to save a lot of patients from complications, cancer reoccurrences, or the need to progress to large, complex surgeries.”