Cancers that were once almost always fatal for children are now curable because of advancements in cellular therapies that Vanderbilt-Ingram Cancer Center pediatric oncologists and hematologists have introduced to Tennessee.

The advancements have included improvements for HLA (human leukocyte antigen) matching with stem cell donors, better medicines for infection prevention, new therapies for graft-versus-host disease (GVHD) and CAR-T therapies.

“We were one of the first sites in the state to be able to use CAR-T to treat our patients, and that’s really been a big game changer for some of our patients who had relapsed or refractory leukemia that would have otherwise been fatal,” said Carrie Kitko, MD, Ingram Professor of Pediatric Oncology and medical director of the Pediatric Stem Cell Transplantation Program.

The program has established itself as a leading innovator in preventing and treating GVHD, which occurs when donor immune cells begin attacking the stem cell recipient’s healthy tissue. Vanderbilt-Ingram is part of an international consortium called MAGIC that has developed risk-stratified treatments according to both GVHD symptoms at presentation and biomarkers, or certain proteins in their blood, to predict patients who are more or less likely to respond to treatments for GVHD.

“We have had several clinical trials to potentially improve outcomes for patients with GVHD. If you’re a low-risk patient, we’ve been able to offer trials where we avoid steroids, which are normally what we use to treat our GVHD patients, but steroids have lots of side effects that can be quite unpleasant,” Kitko said.

“You can identify those low-risk patients that don’t need steroids, and you can use a less toxic therapy. And then for the high-risk patients, if we know that they’re unlikely to respond to steroids, why wait for them to fail on steroids. We have been able to offer some of these studies to both pediatric as well as adult patients.”

Kitko is the senior author of a study published in 2024 in The New England Journal of Medicine that led to the Food and Drug Administration approving a new drug for recurrent or refractory chronic GVHD. Patients who were given the drug, axatilimab, had an overall response rate of 74%.

“Now, patients have access to more drugs to treat chronic GVHD, which is really a very morbid condition for many of our patients,” she said. “Moderate to severe chronic GVHD really impacts their quality of life and their ability to do normal day-to-day activities.”

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Vanderbilt-Ingram Cancer Center is committed to doing more than treating patients with the latest advancements in cellular therapies. Its physician-scientists are focused on expanding their use for more types of cancer and improving response rates among patients.

Andrew Jallouk, MD, PhD, assistant professor of Medicine, is a translational scientist committed to hastening the progression of laboratory discoveries into better treatments for cancer patients. After finishing an undergraduate degree in biomedical engineering and chemistry from Vanderbilt University, he then obtained a dual MD/PhD degree from Washington University in St. Louis followed by a clinical fellowship at MD Anderson Cancer Center in Houston, where he conducted clinical and laboratory research on the use of engineered cellular therapies for lymphoma. He was recruited to Vanderbilt-Ingram in 2023.

Q: Can you describe the role of a translational scientist in the medical realm?

A: Historically, there have been clinical investigators that run clinical trials, and there have been physician-scientists who work in the lab. That’s been great, but what this field needs are people who can speak both languages and really bring the two together. That’s what we are trying to do.

Q: What’s the next step for the advancement of cellular therapies?

A: With cellular therapies, we take the immune cells out of the patient, reprogram them and then put them back in. What’s really unique about this is the manufacturing step. If the treatment doesn’t work for some patients or is not working as well as we would like, we can figure out what’s going on there and then make tweaks during the manufacturing process to improve the therapies. What fascinates me about this technology is the bench-to-bedside (science lab progression to clinical use) and back to bench perspective. We’re putting these cellular therapies into patients, and we can see what’s happening — what is going well and what is not going well; what are the toxicities and how can we avoid these toxicities. We have a lot of ability to then feed that information back into the lab, optimize our manufacturing, optimize our patient selection and really try to make things better.

Q: What you’ve described is CAR-T therapy or chimeric antigen receptor T cell therapy. Isn’t that primarily for blood cancers?

A: There are no CAR-T cells approved for any solid tumors right now. There are other therapies called TIL therapies or TCR therapies. One of those, TIL, has just been approved for melanoma, and there’s a TCR therapy that’s been approved for synovial sarcoma. They all fall within the realm of immune therapies, where you’re using cells to fight cancer. The issue with that historically has been that solid tumors have a hostile tumor microenvironment, so it’s been hard to develop immunotherapies that overcome that.

Q: How much does your training as a biomedical engineer play into what you do?

A: I think it helps in terms of speaking both languages. I have a lot of great scientific training and give a lot of credit to my mentors, both in undergraduate and graduate school and throughout my training. The engineering background has really made it easy to pick up the concept of cellular engineering and the technologies that are associated with that. The PhD work that I did gave me a lot of experience and insight into various laboratory techniques, so it’s made it easier to keep doing what I’m doing on the laboratory end even when I have responsibilities on the clinical end as well.

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Cancers that were once almost always fatal for children are now curable because of advancements in cellular therapies that Vanderbilt-Ingram Cancer Center pediatric oncologists and hematologists have introduced to Tennessee.

The advancements have included improvements for HLA (human leukocyte antigen) matching with stem cell donors, better medicines for infection prevention, new therapies for graft-versus-host disease (GVHD) and CAR-T therapies.

“We were one of the first sites in the state to be able to use CAR-T to treat our patients, and that’s really been a big game changer for some of our patients who had relapsed or refractory leukemia that would have otherwise been fatal,” said Carrie Kitko, MD, Ingram Professor of Pediatric Oncology and medical director of the Pediatric Stem Cell Transplantation Program.

The program has established itself as a leading innovator in preventing and treating GVHD, which occurs when donor immune cells begin attacking the stem cell recipient’s healthy tissue. Vanderbilt-Ingram is part of an international consortium called MAGIC that has developed risk-stratified treatments according to both GVHD symptoms at presentation and biomarkers, or certain proteins in their blood, to predict patients who are more or less likely to respond to treatments for GVHD.

“We have had several clinical trials to potentially improve outcomes for patients with GVHD. If you’re a low-risk patient, we’ve been able to offer trials where we avoid steroids, which are normally what we use to treat our GVHD patients, but steroids have lots of side effects that can be quite unpleasant,” Kitko said.

“You can identify those low-risk patients that don’t need steroids, and you can use a less toxic therapy. And then for the high-risk patients, if we know that they’re unlikely to respond to steroids, why wait for them to fail on steroids. We have been able to offer some of these studies to both pediatric as well as adult patients.”

Kitko is the senior author of a study published in 2024 in The New England Journal of Medicine that led to the Food and Drug Administration approving a new drug for recurrent or refractory chronic GVHD. Patients who were given the drug, axatilimab, had an overall response rate of 74%.

“Now, patients have access to more drugs to treat chronic GVHD, which is really a very morbid condition for many of our patients,” she said. “Moderate to severe chronic GVHD really impacts their quality of life and their ability to do normal day-to-day activities.”

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The cellular therapy programs at Vanderbilt-Ingram Cancer Center, Monroe Carell Jr. Children’s Hospital at Vanderbilt, and the VA Tennessee Valley Healthcare System have received reaccreditation from the Foundation for the Accreditation of Cellular Therapy (FACT).

Founded in 1995, FACT establishes standards for high-quality medical and laboratory practice in cellular therapies. FACT is a nonprofit corporation co-founded by the International Society for Cell and Gene Therapy and the American Society for Transplantation and Cellular Therapy for the purposes of voluntary inspection and accreditation in the field of cellular therapy.

“FACT is an internationally recognized accrediting body for hospitals that offer stem cell transplant and cellular therapy, and recognition by FACT indicates that the accredited institution has met the most rigorous standard in every aspect of cellular therapy,” said Adetola Kassim, MBBS, MS, professor of Medicine and clinical director of the Adult Stem Cell Transplant Program. “This covers the entire spectrum of stem cell therapy from clinical care to donor management, cell collection, processing, storage, transplant, administration and cell release.”

The Vanderbilt and VA Tennessee Valley Healthcare System programs received accreditation notification April 14 after on-site inspections in October 2024. The accreditation is effective for three years.

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The Vanderbilt-Ingram Cancer Center 26th Annual Scientific Symposium, held April 22, focused on the theme “Artificial Intelligence in Cancer Research and Clinical Care.” The event also highlighted the achievements of trainees with a research poster competition and the announcements of graduate and undergraduate students of the year.

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