How Sylvester’s Horowitz Initiative Is Advancing Sarcoma Treatment Through Clinical Trials and Precision Medicine
A philanthropic gift fuels research, a first-of-its-kind patient registry and a promising clinical trial as Sylvester Comprehensive Cancer Center accelerates new therapies for rare solitary fibrous tumors.
Joel Horowitz is living with a solitary fibrous tumor (SFT), a rare and complex sarcoma affecting the bone and soft tissue that impacts just one in a million people worldwide. Like many rare diseases, solitary fibrous tumors have historically received limited attention and funding, which has slowed progress toward understanding its cause and developing life-saving treatments. Determined to change that trajectory, Horowitz is driving efforts to accelerate research and bring hope to patients facing this devastating disease.
Three years ago, he made a transformational gift to Sylvester Comprehensive Cancer Center, a part of UHealth — University of Miami Health System, and the region’s only National Cancer Institute-designated cancer center, to establish the Horowitz Solitary Fibrous Tumor Initiative (HSFTI). His vision was, and remains, to position Sylvester as the nation’s leading center for sarcoma and solitary fibrous tumor.
“I’m fortunate that I’ve been successful in business,” said Horowitz, who was an entrepreneur before retiring. “But to me, real success is measured by what you do to help other people. That’s why I made an investment with the Horowitz Solitary Fibrous Tumor Initiative. I’m confident it will make a profound difference. Hopefully, it will save my life and many others.”
Assembling a Sarcoma Dream Team
Sharing this vision is Horowitz’s sarcoma medical oncologist at Sylvester, Gina D’Amato, M.D. ’98, the HSFTI’s lead physician-scientist. Dr. D’Amato, a professor of medical oncology at the Miller School, has assembled what Horowitz refers to as the “Sarcoma Dream Team.” This group of research scientists are advancing tumor profiling, cell line development and therapeutic discovery and accelerating precision medicine for solitary fibrous tumor patients.
The HSFTI research team consists of:
• Jonathan Trent, M.D., Ph.D., director of Sylvester’s sarcoma molecular research program, associate director clinical research and a professor in the Division of Medical Oncology at the Miller School
• David Lombard, M.D., Ph.D., co-leader of the Cancer Epigenetics Program, professor and and vice chair of clinical and translational research for the Miller School’s Department of Pathology and Laboratory Medicine
• Andrew Rosenberg, M.D., a researcher and director of bone and soft tissue pathology at Sylvester, clinical professor in the Division of Pathology and Laboratory Medicine and co-chief of anatomic pathology at the Miller School
• Zhenfeng Duan, M.D., Ph.D., research professor of orthopaedics at the Miller School
Dr. D’Amato says all have made remarkable strides in three short years.
“I explained to Mr. Horowitz when he first approached us and said he wanted to find a cure for solitary fibrous tumors that you need to start by discovering the cause,” said Dr. D’Amato, assistant director of clinical research, medical director of the Advanced Practice Provider Oncology Fellowship Program and medical director of patient education at Sylvester. “We must find out what’s going on molecularly, and then we can find drugs to attack the disease. Thanks to his generosity, we are making measurable progress.”
Decoding Solitary Fibrous Tumors at the Molecular Level
Dr. D’Amato says that the HSFTI is using a three-pronged process to conduct its research. The first step is to try to understand the disease at genetic, molecular and epigenetic levels.
Dr. Rosenberg is studying tissue samples, looking for specific gene mutations and other biomarkers to better understand the disease’s prognosis and natural history. He and his team were able to review more than 100 pathology samples from patients seen at Sylvester over the past 15 years. Their findings were recently presented as a mini-oral abstract presentation at the American Society of Clinical Oncology (ASCO) annual meeting by Keerthena Sureshkumar, a third-year medical student at the Miller School.
Additionally, Dr. Duan has created a cell line from Mr. Horowitz’s tumor. This is significant because it is almost impossible to create a cell line for solitary fibrous tumors. Dr. Lombard is using laboratory-grown cell lines to create organoids, three-dimensional cultures of solitary fibrous tumor cells, which help identify new treatment strategies and drug therapies.
“We’ve screened a fairly small drug library so far, consisting of a few hundred compounds, as a proof-of-concept,” said Dr. Lombard. “We’re now gearing up to screen much larger collections of medicines, and also to use cutting-edge approaches to identify genes that are required for SFT survival and growth.”
He added, “This kind of discovery science paves the way for new therapies. I think that SFT patients can be optimistic that there will be new therapeutic options available in the next few years.”
From Lab Discovery to Clinical Trials
That confidence comes in knowing that Sylvester is participating in a phase one clinical trial of a fibroblast activation protein (FAP) inhibitor. It’s an image-guided alpha-particle therapy study of the drug PSV359, which uses radioactive particles to target FAP and has shown promising preliminary results in Germany. Sylvester will be the only cancer center in the southeast United States to participate in the trial for patients with solid tumors.
Dr. D’Amato says Sylvester scientists are working to develop other technologies to target FAP, such as an antibody drug conjugate, and hopes to bring that to a clinical trial. She notes they continually pivot and produce innovative ideas based on what they discover in the lab, noting their ultimate goal is gene therapy.
A First-of-Its-Kind Patient Registry Expands Global Data
Of course, none of this is possible without data. That is why Dr. D’Amato believes the HSFTI’s greatest accomplishment so far is the creation of the first comprehensive patient registry for solitary fibrous tumors.
“Because SFT is such a rare cancer, we need to be as inclusive as possible to best understand this disease,” said Dr. D’Amato. “The more patients we enroll, the more powerful the data will be as we uncover the causes, biology and natural history of this rare disease.”
Help advance research on rare sarcomas
Patients diagnosed with solitary fibrous tumor are encouraged to join Sylvester’s patient registry to support discovery and accelerate new treatments.
Join the Patient RegistryThe HSFTI is collaborating with sarcoma experts worldwide to enroll patients, compiling data from medical records and through questionnaires about environmental and genetic factors to understand treatment responses and recurrence patterns. The registry will also collect blood samples and tumor specimens for analysis, with researchers looking for potential biomarkers.
Horowitz is channeling his efforts into promoting the patient registry program because of the critical data it provides.
“The more patients we enroll, the more scientists can learn, and that will bring them closer to finding cures,” said Horowitz, who volunteers through Sylvester’s Sarcoma Peer Mentorship Program. “I’m so grateful to Dr. D’Amato and the Sylvester Dream Team for sharing my passion and providing greater hope for patients like me.”
Accelerating Hope for Patients with Rare Cancers
Like Mr. Horowitz, Dr. D’Amato sees a promising future.
“Every question we answer leads to 10 more, but each one brings us closer to treatment breakthroughs,” said Dr. D’Amato. “The pace of discovery has dramatically increased thanks to support from donors like Mr. Horowitz, who understand that rare doesn’t mean hopeless.”
Learn more about enrolling in the patient registry and making a donation to support the Horowitz Solitary Fibrous Tumor Initiative.
More from Sylvester Comprehensive Cancer Center
A Sylvester study identifies genetic markers that may predict aggressive solitary fibrous tumors, encouraging personalized cancer treatment.
Sylvester researchers are studying lifestyle, exercise and wearable devices for pediatric and young adult sarcoma patients.
Sylvester Comprehensive Cancer Center is one of 20 institutions contributing to GENIE, a registry of data from thousands of cancer patients.
Sarcoma expert Dr. Emanuela Palmerini is uniting experts to bring more precise and less toxic treatments to young patients with rare cancers.
Tags: cancer research, Department of Pathology and Laboratory Medicine, Dr. Andrew Ronsenberg, Dr. Andrew Rosenberg, Dr. David Lombard, Dr. Gina D'Amato, Dr. Jonathan Trent, Dr. Zhenfeng Duan, Horowitz Solitary Fibrous Tumor Initiative, sarcoma, solitary fibrous tumor, Sylvester Comprehensive Cancer Center