Sylvester Provides 100 Patients Potentially Life-Saving Treatments in its Phase 1 Trial Program

Dr. Jaime Merchan is thrilled that Sylvester Comprehensive Cancer Center has reached the milestone number of 100 patients enrolled in a single year in its Phase 1 clinical trials program. The program, which tests new drugs and other novel treatment strategies for advanced solid tumors and hematological malignances, offers these patients something they couldn’t find elsewhere: hope.

Team members of the Sylvester Phase 1 clinical trials program.

“Phase 1 clinical trials offer promise for patients who have failed prior therapies and have no other options,” said Merchan, a hematologist/oncologist at Sylvester, associate professor of medicine at the Miller School of Medicine, and director of the Phase 1 program. “By growing our program to 100 accruals in a year, we firmly establish Sylvester’s program in the upper echelon of academic Phase 1 programs in the country.

“This is a reflection of our productivity and our growth as a clinical program in experimental therapeutics, with a main goal to offer patients hope.”

Ana Deiter Cruz’s doctors had done all they could for her leukemia, and were out of options – but fortunately they had heard about the innovative research at Sylvester. Deiter Cruz traveled from Kissimmee to see Sylvester researcher Justin M. Watts, M.D., assistant professor of medicine and an active member of the Phase 1 program, who is developing novel therapies for acute myeloid leukemia.

“He said, ‘You are a perfect candidate,’ and the rest is history,” Deiter Cruz said.

“As he told me about all the treatments and the risks, I said, ‘I’ll tell you one thing – I don’t have anything to lose. Even if it doesn’t work for me, I want to be part of it because at least I can be of help to other people.’”

The treatment – an IDH1 inhibitor targeted to a mutation she has – did work for her. “It’s really cutting edge scientifically and the drug’s been working very well for some patients,” Watts said. For Deiter Cruz, it put her bone marrow into remission and served as a bridge to a stem cell transplant, which is her next step. Without the Phase 1 trial, “she never would have been able to get the life-saving transplant,” Watts said.

“It’s been an awesome winter,” Deiter Cruz said. “Not only did they help me, they saved my life.”

The new therapeutic agents – several of which were discovered or developed by UM researchers – include the targeted drugs that are directed against specific mutations within the tumor, novel immunotherapy agents, and new biological agents. In several of the studies, Sylvester is the first center in the world to test the treatment.

“We are developing many areas of strength. One area of our research is the evaluation of viruses that target cancer, called oncolytic viruses,” Merchan said. “We have several of those trials for different types of tumors.”

Therapies that have shown efficacy are moved to the next level of clinical development. This includes enfortumab vedotin for patients with urothelial cancer. “We were part of a multi-center Phase 1 study of this experimental drug that showed promise in patients with urinary tract cancers,” Merchan said. “It’s moving into Phase 2 and 3 studies based on the Phase 1 results.”

“Reaching the 100-patient landmark has been possible thanks to the dedicated Phase 1 investigators and staff of Sylvester who are fully committed to helping patients and advancing science,” said Sylvester Director Stephen D. Nimer, M.D. “We are making many new treatments available to cancer patients around the world as a result of this incredible work.”

Craig Lockhart, M.D., M.H.S., professor of medicine and Chief of the Division of Medical Oncology, said, “Doing Phase 1 studies requires a huge amount of teamwork between the patients and their families, the doctors, the nurses and the research staff. Dr. Merchan is to be commended for assembling such a strong team so that patients in a difficult life situation are reassured by the ability of the group to provide great care.”

In a Phase 1 program, “patients with advanced cancers have access to the newest and most novel drugs, and the highest chance of getting a drug that is going to work,” Watts said. “It has to be at a specialized place – the studies are difficult to do, and patients must have access to a large team of providers and coordinators.”

Many patients in the Sylvester trials are South Floridians, but several have come from other states and other countries. “Being in South Florida makes us a hub to offer new therapies for patients in Latin America,” Merchan said.

Ana Deiter Cruz couldn’t be happier about the progress she has made and the commitment of everyone at Sylvester. “I was very sick and they were so kind, they gave me so much hope,” she said. “Dr. Watts and his staff were always right there when I needed them. I knew even though we are far apart, they were only one phone call away.

“I have met so many people who like to help other people. You can tell it’s not only their job, it’s a passion they have for what they’re doing.”

The future has never looked brighter for patients battling cancer.