FDA Approves New Approach to Stem Cell Transplants

Roxanne Laveau gets regular blood panel tests every 10 years because there is a history of lupus in her family. In June 2023, she had her blood drawn before a cruise vacation. But when she got the results upon her return, it was nothing like her previous decade of results.

“My doctor revealed that my blood results were critical and that I needed to head to the ER immediately for two blood transfusions,” Laveau said.

After the transfusions and two nights in the hospital, Laveau was discharged and told to follow up with physicians at Moffitt Cancer Center.

“I still don’t think it had registered that cancer could be a diagnosis. Sure, I had been feeling a little tired and run down. The low blood counts would explain that, but I still wasn’t sure what was happening,” Laveau said. 

She was journeying into the unknown. An aggressive diagnosis, followed by a chance to join a clinical trial evaluating a new kind of stem cell transplant called Orca-T. A trial that would lead to the U.S. Food and Drug Administration approval of the treatment.

An Unexpected Diagnosis

Laveau was diagnosed with acute myeloid leukemia, a cancer that starts in the bone marrow, where blood is made. In acute myeloid leukemia, the body produces large numbers of abnormal white blood cells that don’t function properly and crowd out healthy cells. As a result, patients can develop dangerously low levels of red blood cells, platelets and healthy white blood cells, leading to fatigue, infections or bleeding.

Acute myeloid leukemia can appear suddenly and progress quickly, especially in older adults. While it is considered an aggressive cancer, it is also treatable. Care often includes chemotherapy, targeted therapies and, for some patients, a stem cell transplant to replace diseased bone marrow with healthy donor cells.

Doctors suggested Laveau start treatment immediately, which consisted of chemotherapy to eradicate the leukemia, increase her blood counts and get her healthy enough for a stem cell transplant. In July 2023, she was admitted to Moffitt for 30 days of therapy, but because her case was so severe, she ended up spending three months in the hospital receiving treatment. Halfway through, planning began for her transplant. The best treatment option was to have an allogeneic transplant, which required identifying a compatible donor.

Laveau did get a short break to go home over Thanksgiving to prepare for her transplant. During that time, doctors called to go over the transplant and provide another option: a clinical trial evaluating a new kind of treatment for stem cell transplant called Orca-T.

The Next Generation of Stem Cell Transplant Care

Orca‑T is a precision-engineered cell therapy that offers a new approach to stem cell transplant designed to reduce some of the risks associated with a traditional donor transplant. In a typical allogeneic transplant, donated stem cells rebuild a patient’s blood and immune system. However, the donor’s immune cells can sometimes recognize the patient’s body as foreign and attack healthy tissues, leading to a potentially serious complication known as graft‑versus‑host disease.

“Our goal with Orca‑T is to be more intentional with the donor cells by using novel precision engineering methods designed to lower the risks of serious side effects of transplant,” said Rawan Faramand, MD, a medical oncologist in the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt and one of the investigators of the clinical trial. “By carefully engineering the graft ahead of time, we aim to reduce harmful immune reactions while still allowing the donor cells to do what they’re meant to do: help eliminate cancer.”

By carefully engineering the graft ahead of time, we aim to reduce harmful immune reactions while still allowing the donor cells to do what they’re meant to do: help eliminate cancer.

Rawan Faramand, MD

To accomplish this, Orca‑T uses a precise combination of donor cells selected before the transplant. In addition to blood‑forming stem cells and conventional T cells, the transplant includes regulatory T cells, which help keep immune responses in check. This balance is designed to maintain the transplant’s cancer‑fighting effect while limiting damage to healthy tissues.

Another key difference is how Orca‑T is prepared and delivered. Rather than infusing donor cells and then relying mainly on medications to control immune complications, Orca‑T is manufactured in advance with specific proportions of cell types and administered alongside standard immune‑suppressing drugs such as tacrolimus. This strategy is intended to reduce the risk of graft‑versus‑host disease and may lessen the need for long‑term immune suppression, although some risk remains.

Taking A Chance on a New Therapy

After carefully weighing her options, Laveau decided to enroll in the trial, only to learn a few weeks later that it was full. So, her care team prepared for a traditional allogeneic transplant, finding her a 24-year-old female donor who was a 100% match. But as fate would have it, a trial spot opened up a short time before her transplant, and she was eligible to participate.

Laveau checked into Moffitt on Dec. 1, 2023, for her transplant. It started with chemotherapy to deplete her immune system and make room for the new cells.

“I had a few side effects from all the chemo. My mouth was dry. I spiked a fever. But I met each one head on and even worked the entire time I was in the hospital, setting up a makeshift office in my room. Working provided me a sense of normal,” Laveau said. “And my nurses and care team were at my side, cheering me on the entire journey. They explained every step of what to expect and offered advice, such as switching out my toothbrush once a week to lower the risk of infection.”

She drew strength from others who had faced cancer before her. “My inspiration and encouragement came from hearing Farrah Fawcett’s story. I have a bracelet from her foundation that I wear every day.”

Eight days after checking in for her transplant, it was time for Laveau to receive her donor cells. Orca-T is administered in three parts over the course of two to three days. One part contains blood-forming stem cells that help rebuild the patient’s blood and immune systems, and the second part contains regulatory T cells that help prevent donor cells from attacking the patient’s body. The third part, administered two to three days later, contains conventional T cells to accelerate immune reconstitution and help fight the disease. Putting those pieces together in a controlled way is designed to make the transplant safer and lower the risk of graft-versus-host disease.

Ready for the Next Adventure

Now, two and a half years later, Laveau is ready for her next adventure. She says she has lived a bit in a bubble for the last couple of years, but for good reason.

“I have been given the ultimate gift. Nothing is more precious than the gift of life, and I want to protect and respect it,” she said. “Cancer picked me. I feel it’s my purpose to do something good with it. I want my story to help other patients.”

She has had minor bouts with graft-versus-host disease, such as skin rashes and she still prefers to mask up when she heads out and about, but Laveau has gotten back to some of the things she enjoyed before her diagnosis. She went to the NHL Winter Classic at Raymond James Stadium in February. She also plans to visit family in Ohio. Coincidentally, her donor lives about 40 minutes outside her hometown. While they have emailed briefly, Laveau would like to visit her, too, if possible.

“I am here because of someone else’s unselfish gesture. I want to thank her in person,” Laveau said.

New Hope for More Blood Cancer Patients

The U.S. Food and Drug Administration approved Orca‑T on June 30 based on results from the Precision‑T clinical trial in which Laveau participated. The study enrolled 187 adults with acute leukemia or myelodysplastic syndrome and found that patients who received Orca‑T experienced significantly fewer serious cases of chronic graft‑versus‑host disease than those who underwent a standard donor transplant.

With FDA approval now in place, Orca‑T will be more widely available to patients with hematological malignancies including acute myeloid leukemia, acute lymphoblastic leukemia and myelodysplastic syndromes, expanding access to a treatment that helped change the course of Laveau’s own cancer journey.