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New resources help people take action to prevent inherited diseases

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Vanderbilt University Medical Center offers access to new resources that help relatives connect and join forces to protect their families from inherited diseases. 

VUMC is one of the first cancer centers to partner with the ConnectMyVariant program, which offers resources for families with hereditary cancer risk. The resources make genetic medicine approachable, can help people discover distant relatives they didn’t know before, and can assist families in talking across dinner tables about a potentially sensitive topic. It’s akin to an ancestry game that allows people to amplify the family benefits of cancer prevention, and the person who starts it can be a superhero. 

To launch a connection search, someone with a specific gene variant signs up to meet others who share that variant. When people have the same gene variant, there is more than 90% chance they are related, even if it is several generations back. This begins the process of sharing stories with others who are likely to be relatives, discovering new connections with unknown relatives, and encouraging family members to get tested. The program is designed around the perspective and experience of lay people — not clinicians. It is an innovative approach to encourage genetic testing, and it’s needed, said Tuya Pal, MD, professor of Medicine in the Division of Genetic Medicine and Clinical Pharmacology at VUMC. 

Tuya Pal, MD

“Family communication, which is the basis of what we call cascade testing, is hugely important,” said Pal, who is also Ingram Professor of Cancer Research and an associate director at Vanderbilt-Ingram Cancer Center. “BRCA genes were identified 30 years ago, but we’ve found maybe 20% of adults who are at risk for cancer. There’s a lot of adults walking around with a BRCA mutation. They could get ongoing prevention, but they don’t know it. This is a challenging nut to crack, but it is of huge public health relevance. If we can get to these people, we can save lots of lives.” 

BRCA1 and BRCA2 gene variants, which increase risk for breast, ovarian, pancreatic and prostate cancer, have received the most media attention. However, in recent years, several other gene mutations have been discovered that can also put people at risk for cancer. Pal stressed that people should seek genetic testing from reputable and accredited laboratories, which are different from many direct-to-consumer testing companies that offer testing for “recreational genetics.” While direct-to-consumer labs offer ancestry testing, sometimes they also delve into other genes; however, the quality of the data they provide is not always accurate for disease risk. For this reason, it is important to get tested through providers knowledgeable about genetic testing options, such as the Vanderbilt Hereditary Cancer Clinic. Insurance typically covers the cost for those at high risk, such as those with a personal history of specific cancers (and diagnosed at certain ages), family history of specific cancers, and those who already have a relative found to have a gene mutation. The tests, which can be done with either blood or saliva samples, cost about $250 for self-paying patients.  

Pal likens a gene mutation to a spelling mistake. 

“When we’re talking about an inherited predisposition, you can think of each gene as a big book, and a mutation is like a ‘spelling mistake’ in that book — leading to the gene not working quite right and thereby raising risk of certain cancers,” she said. “If you’ve got the same spelling mistake between two people, they are somehow related. People sometimes have trouble sharing broadly with family members, so this tool can make this experience of family communication and testing more enjoyable. You can connect with people you don’t know that are related from maybe eight generations ago.” 

Brian Shirts, MD, PhD

ConnectMyVariant, which is operated as a nonprofit organization, was developed by Brian Shirts, MD, PhD, associate professor of Pathology, Microbiology and Immunology and of Biomedical Informatics, who was recruited to VUMC in December 2024. He is the service medical director of the Molecular Genetics Laboratories. 

“Scientific modeling has shown that if we could reach out to second and third cousins effectively of people with known variants, we could identify everyone who has a hereditary cancer risk,” said Shirts, who is also President of ConnectMyVariant’s Board of Directors. 

However, he stressed that ConnectMyVariant is not a research program or a direct clinical resource.  

“It’s an educational program,” Shirts said, noting that people should schedule a visit with a genetic counselor affiliated with a medical provider if they discover they have relatives with mutations that put them at increased risk for cancer.  

ConnectMyVariant offers educational resources for adult-onset hereditary diseases for which there are established preventive actions people can take. It is not intended for use by people younger than 18, so it does not offer resources for autosomal recessive pediatric diseases.  

“The way I like to think about what we offer is ‘If I talk to my relatives, are they going to be able to do something?’ If the answer is yes, and they can get genetic testing and do something that will improve their health, then that’s included,” Shirts said. 

The ConnectMyVariant program also offers participants access to resources for ancestry research, such as professional assistance from the Center for Family History and Genealogy at Brigham Young University.  

For individuals with inherited cancer predisposition, Vanderbilt-Ingram has the Inherited Cancer Registry (ICARE), which Pal founded in 2010. It has grown into one of the largest registries for inherited cancers, with almost 8,000 participants enrolled to date. Through the registry, the latest care and research updates are shared with participants by newsletter, email and links to presentations, and it is a way for people to be part of the larger research mission. 

Pal serves as vice chair of the National Comprehensive Cancer Network Genetics/Familial Guidelines Committee for Breast, Ovarian and Pancreatic Cancer. She leads a team of certified genetic counselors at the Vanderbilt Hereditary Cancer Clinic.

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New NCI-funded project targets polyp recurrence to prevent colon cancer

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A multidisciplinary team led by investigators at Vanderbilt University Medical Center has received a $4.2 million grant from the National Cancer Institute (NCI) to probe the genetics of colorectal adenomas — polyps that can develop into colon cancer — and to identify drug candidates that could reduce adenoma recurrence. 

Xingyi Guo, PhD

Colorectal cancer is the second most common cause of cancer-related death in the United States, according to the NCI, part of the National Institutes of Health. Removing precancerous polyps during colonoscopy procedures significantly reduces the burden of colorectal cancer, but about 30% of patients who have a colorectal adenoma removed will develop recurrent adenomas. 

“Patients who have recurrent adenomas are at higher risk for developing cancer,” said Xingyi Guo, PhD, associate professor of Medicine in the Division of Epidemiology and lead principal investigator for the project. “We will integrate cutting-edge genomic research with real-world patient data from electronic health records, with the goal of translating genetic discoveries into actionable strategies to prevent colorectal cancer.” 

Zhijun Yin, PhD

Zhijun Yin, PhD, MS, associate professor of Biomedical Informatics, is co-principal investigator for the four-year project. 

The team previously conducted genome-wide association studies (GWAS) of about 8,000 colorectal adenoma cases from European American and African American participants included in BioVU, VUMC’s de-identified DNA biobank and linked electronic health records. Using a large-scale analysis of electronic health records and pathology reports, the investigators also established the Vanderbilt Colonoscopy Cohort of colorectal adenoma cases after polyp removal, which includes 76,664 cases. 

With the new funding support, the team will extend its efforts to establish the largest-ever genetic study of colorectal adenoma, drawing on BioVU, the Mass General Brigham Biobank, and the NIH All of Us Research Program to include over 25,000 cases in European Americans and 6,500 cases in African Americans, with thousands of recurrences. African Americans are about 20% more likely to have colorectal cancer and about 40% more likely to die from it compared to other racial groups, according to the American Cancer Society. 

“Our approach will allow us to examine racial differences in adenoma recurrence and colorectal cancer risk,” Guo said. 

In addition to GWAS, the team will conduct transcriptome-wide, methylome-wide, and proteome-wide association studies to identify genes and proteins associated with colorectal adenomas and their recurrence. The investigators will integrate findings from these “omics” studies with electronic health record data from the Vanderbilt Colonoscopy Cohort and the Mass General Brigham Colonoscopy Cohort and use machine learning frameworks to identify candidate drugs that could prevent colorectal adenoma recurrence. They will test the most promising drug candidates in colorectal adenoma and cancer cells, patient-derived organoids, and animal models. 

“This project is an innovative integration of multiomics analyses with electronic health record-based real-world clinical evidence,” Yin said. “We anticipate that our findings will inform personalized colorectal polyp surveillance, guide therapeutic prevention strategies, and ultimately reduce the burden of colorectal cancer nationwide.” 

Guo holds a secondary appointment in the Department of Biomedical Informatics at VUMC, and Yin holds secondary appointments in the Department of Computer Science and the Department of Electrical and Computer Engineering at Vanderbilt University. Guo and Yin have both received NCI R37 MERIT Awards, which provide long-term grant support to outstanding investigators. 

Other collaborators for the new NCI grant (R01CA297582) include VUMC Department of Medicine investigators Wei Zheng, MD, PhD, MPH, Qiuyin Cai, MD, PhD, and Wanqing Wen, MD, MPH, Division of Epidemiology; Bhuminder Singh, PhD, Division of Gastroenterology, Hepatology and Nutrition; and Kristen Ciombor, MD, MSCI, Division of Hematology and Oncology; and Harvard T.H. Chan School of Public Health investigator Mingyang Song, ScD, Departments of Epidemiology and Nutrition.

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Mark Kelley retires, having ‘profoundly shaped surgical oncology’ at VUMC

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Mark Kelley, MD, MMHC
Mark Kelley, MD, MMHC

Mark Kelley, MD, MMHC, medical director of the Williamson County General Surgery Division in the Department of Surgery at Vanderbilt University Medical Center, has retired, effective Oct. 1, after 28 years of exceptional service and leadership.

“Dr. Kelley transformed the Division of Surgical Oncology and Endocrine Surgery into one of the nation’s largest and most productive academic surgical oncology programs,” said Carmen Solórzano, MD, John L. Sawyers Chair in Surgical Sciences and chair of the Department of Surgery. “His remarkable contributions span clinical excellence, innovative research and impactful education.”

Kelley, an associate professor of Surgery in the Division of Surgical Oncology and Endocrine Surgery, is lauded by his colleagues and surgical leaders for his contributions both at VUMC and nationally.

“Dr. Kelley’s career reflects an unwavering commitment to excellence in clinical care, research, education and leadership,” said Seth Karp, MD, H. William Scott Jr. Chair in Surgery and chair of the Section of Surgical Sciences. “His legacy has profoundly shaped surgical oncology at Vanderbilt and beyond. We are grateful for his dedication and leadership.”

In 1997, Kelley joined the VUMC faculty as an assistant professor in the newly established Division of Surgical Oncology and Endocrine Surgery and served as clinical director of the Vanderbilt Breast Center until 2005. His leadership skills led to his appointment as chief of the Division of Surgical Oncology and Endocrine Surgery in 2002, a role he held until 2015.

“As clinical director of the Vanderbilt Breast Center, he led its development and transition from a small practice in the Village at Vanderbilt to a comprehensive breast center at One Hundred Oaks,” said Solórzano. “In 2005, he passed the clinical directorship to Dr. Ingrid Meszoely, whom he recruited back to Vanderbilt after her surgical oncology fellowship. Today, the Vanderbilt Breast Center is one of the largest and most comprehensive programs in the United States.”

Recognizing the importance of focused training in breast surgical oncology, Kelley developed the framework for a breast surgical oncology fellowship. Expansion of the curriculum under the leadership of Mary Hooks, MD, MBA, and Ingrid Meszoely, MD, led to accreditation of the program by the Society of Surgical Oncology in 2016.

Under Kelley’s leadership, the Division of Surgical Oncology and Endocrine Surgery expanded from four surgeons to a multidisciplinary team of more than 20 surgeons, advanced practice providers (APPs) and research scientists. He also played a pivotal role in recruiting and mentoring key faculty who now serve as VUMC surgical and Vanderbilt-Ingram Cancer Center leaders, including Rondi Kauffmann, MD, MPH; Christina Bailey, MD, MSCI; Kamran Idrees, MD, MSCI, MMHC; Meszoely, Solórzano and others.

Kelley was a sought-after mentor throughout his career, and assisted numerous medical students, surgical trainees and junior faculty as they participated in projects and developed their own research. Under his leadership, the Division of Surgical Oncology and Endocrine Surgery was highly ranked, year after year, as a favorite learning environment for general surgery trainees. More than 30 residents completed surgical oncology fellowships during Kelley’s tenure, and many are leaders in the field today, including five current VUMC faculty members.

He was an early advocate for the integration of APPs into clinical roles. In 1999, he established training and mentorship programs for APPs specializing in breast health and surgical oncology, and these programs served as models for integrating APPs into surgical practices throughout VUMC. Today, there are 10 APPs practicing in inpatient and outpatient roles in the Division of Surgical Oncology and Endocrine Surgery.

Kelley was a surgical innovator and the first surgeon in Tennessee to perform sentinel lymph node biopsy for breast cancer and melanoma in 1997. This procedure has transformed the care of these cancers. Kelley developed an Institutional Review Board (IRB)-approved protocol to train surgeons on this technique, leading to the rapid and safe application of the new surgical procedure at VUMC and in the community.

Kelly was also integrally involved in the development of multidisciplinary clinical and research programs at Vanderbilt-Ingram Cancer Center. From 2000-2012, he served as chair of the VUMC Cancer Committee. This group monitors and reports cancer volumes and outcomes, guides quality improvement, and ensures compliance with national cancer treatment standards. During his tenure as chair, VICC was continuously accredited by the American College of Surgeons Commission on Cancer. The program was also routinely recognized as one of the top National Cancer Institute-accredited comprehensive cancer centers nationwide during that time.

Early in his career, Kelley had an independent laboratory that focused on translational research in melanoma tumor biology and contributed to the early development of immunotherapy for melanoma. He established the melanoma and cutaneous malignancy tissue repository in 2003. This IRB-approved research repository has collected tumor tissue samples from patients undergoing surgical resection or biopsy with paired clinical data from more than two decades. This invaluable resource has supported high impact basic and translational studies that have led to novel combinations of immunotherapy and targeted therapy being investigated in clinical trials today.

“Dr. Kelley is highly committed to the advancement of cancer care through research, and we are grateful that he will remain active in clinical and translational research with VUMC and VICC as professor of Surgery, retired, to continue to improve care for patients with cancer,” said Karp.

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Nurse’s wind phone project provides solace from grief 

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Allison Young is easing the pain for people grieving the lost connections when a loved one dies by providing a place for words left unspoken and milestones that can no longer be shared. 

It’s a small phone booth without utility wires or cellular connections nestled along her driveway in East Nashville. The messages are carried by love vibes. Since Young erected the booth two years ago, it has become a sanctuary of solace and garnered attention from local and international media. 

She learned about a “wind phone” while she was working on the thesis for her master’s degree, researching alternative grieving methods, and delving into the continuing bonds theory — a concept that it’s okay for people in mourning to maintain connections to loved ones who have died instead of just having to let them go. 

The first wind phone was erected in 2010 in northern Japan by Itaru Sasaki, who wanted to talk with his cousin who had died from gastric cancer.  

“Since he couldn’t talk to him in person on a regular phone line, he wanted his words to be carried on the wind,” said Young, MS, BSN, RN, a research nurse specialist for clinical trials at Vanderbilt-Ingram Cancer Center. “He lived at the top of a hill right next to the ocean, so there was lots of wind. It was a perfect concept for him. Then unfortunately, four or five months later was when the tsunami hit Japan, the one that killed tens of thousands of people along the northern Japanese coast.” 

The tsunami devastated Ōtsuchi, the town where Sasaki lived, with about 10% of its population of 16,000 perishing. Sasaki’s wind phone became the place where grieving survivors went to connect with lost loved ones. 

 “Now, thousands of people from around the world have made the trek to northern Japan to see the original wind phone,” Young said. “The idea has spread across the entire world. There are wind phones in other places in Asia, in Australia, all over North America, and a ton in Europe as well.” 

The “wind phone” allows people a place for words left unspoken and milestones that can no longer be shared. (photo by Donn Jones)
The “wind phone” allows people a place for words left unspoken and milestones that can no longer be shared. (photo by Donn Jones)

Young put up her wind phone booth in September 2022, then unveiled it to the wider community in February 2023. 

“It is dedicated to my paternal grandparents,” she said. “We opened it on the 24th anniversary of my grandfather’s death, and it is dedicated to my grandma who died in 2016. She is the only person I have ever called on it, and I called her the day I defended my thesis.”  

Young has received notes from more than 200 people who have visited it. The structure is a circa 1940s actual phone booth. Her father, who is a contractor, helped her set it up by putting a slanted roof over its flat top and weatherproofing it with exterior paint. The booth is soundproof with a privacy curtain. 

“There’s a light so that people can use it overnight as well,” she said. “Since I am a nurse, I know what health care hours are like, so I made it available 24 hours a day.” 

The door has a sensor to help her keep records of visitors, but there are no cameras or privacy infringements. Visitors are encouraged to park in her driveway because Rosebank is a two-lane road with a narrow shoulder. 

The wind phone has been featured in news stories and documentaries, including coverage by NHK, Japan’s public media organization, and by “This American Life,” a public radio program in the United States. 

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Clip In 4 the Cure 2025

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This year’s Clip In 4 the Cure drew hundreds of participants to Geodis Park for a relay-style team cycling event featuring top Nashville spin instructors and live DJs.

The high-energy fundraiser began in 2022 to raise money for cancer initiatives at Vanderbilt-Ingram Cancer Center and Monroe Carell Jr. Children’s Hospital at Vanderbilt.

Clinicians and patient ambassadors highlighted the impact that the fundraising efforts have on cancer care and research. 

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Types of Cellular Therapies

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There are many types of cellular therapy. Vanderbilt-Ingram has one of the nation’s best stem cell transplant centers based upon procedure volume and patient outcomes. The center was the first in Tennessee to offer CAR-T (chimeric antigen receptor T cell) therapy, which reengineers a patient’s immune cells to recognize and attack cancer. Another cellular therapy Vanderbilt-Ingram recently added is TIL (tumor-infiltrating lymphocyte) therapy for advanced melanoma. TIL became the first cellular therapy approved by the Food and Drug Administrations for solid tumors in February 2024.

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A Second Opinion

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In the summer of 2021, I was diagnosed with Stage 4 renal cell carcinoma. Needless to say, my world was turned upside down. Cancer is such a devastating disease, and no one wants to be a part of that club. No preplanning for this one. Yet, there I was, handed this diagnosis with no plan in hand.

 “What’s next?” I thought. While the radiologist was giving me some direction on my recent discovery, I totally passed out! “This couldn’t be happening, could it? Look at me, I’m a healthy, physically fit, soon to be retired happy-go-lucky male.” Ironically, in all of this, I just received my very first Social Security deposit earlier that morning. Good luck seeing my next one! 

Two weeks later, I was undergoing a serious operation to remove my right kidney and 11 centimeter tumor when the unimaginable happened. Parts of the tumor broke apart during the resection. A loose thrombus from the large tumor traveled up my inferior vena cava and into my heart, which stopped my blood flow for up to 10 minutes. That generally means instant death! Obviously, I didn’t “expire,” as I am here writing this dialogue for you. However, there are many excruciating details of how I ultimately survived that surgery and lived to talk about it. That particular incident and my cancer journey that followed are quite a story. In fact, I authored a bestselling book describing that operation and what really happened in those 10 “lifeless” minutes.

The book recounts that major event along with the six months that followed. It was quite a ride enduring the cancer emotions for both me and my wife along with the continual cancer treatments. My therapy following the operation involved a regimen of pembrolizumab infusions, which is an immunotherapy, along with a daily intake of Inlyta pills, a targeted therapy that inhibits the growth of cancer cells.

After following this schedule for the two years that was prescribed, it was decision time. I had great success with the mix of these two drugs in fighting my cancer, although I encountered many side effects along the way. I was determined to stay the course no matter what the consequences were. Now at this crossroad, I had many options to consider. Keep the medication intake the way it was? Reduce the number of medications? Or a combination of many other variables? After giving this much thought, I asked my oncologist at the Medical University of South Carolina, Dr. Theodore Gourdin, about getting another opinion. Dr. Gourdin was gracious regarding my question and suggested two other oncologists who specialized in kidney cancers. One of the doctors he mentioned was Dr. Brian Rini at Vanderbilt-Ingram Cancer Center in Nashville. I researched Dr. Rini online and felt like he was my guy. After all, he had authored the treatment study that I’d been on for the past two years. 

Hollings Cancer Center at the Medical University of South Carolina contacted Vanderbilt-Ingram and introduced me to them in order to get the ball rolling. I followed their introduction with a call to set up an appointment. I was impressed from the moment the receptionist answered the phone. She was very welcoming, and after some discussion she switched me over to Dr. Rini’s navigator who was already familiar with my case. She scheduled an appointment for a Thursday at 10 a.m. That was only 48 hours from the time I was speaking to her. 

I would need to step it up as the cancer center was over 500 miles away involving an eight-hour-plus drive. We would have to leave early Wednesday morning to arrive in Nashville at a reasonable hour. Considering that MUSC and VUMC used the same software company, (Epic Systems Corp.), I was able to merge the two systems so Dr. Rini would have immediate access to all my medical records. It was amazing how all of this came together in short order. 

William and Linda Douglas take a stroll on the beach with their standard poodle, Gianni.

My wife, Linda, and I, along with our standard poodle, Gianni, loaded up our SUV Wednesday morning and headed toward Nashville. Ingram Content Group, the company that would print my book on cancer is located in La Vergne, Tennessee, southeast of Nashville. Considering it was along the way, I thought it would be beneficial to stop and see the facility that would ultimately publish my book when it came out. This trip was kind of full circle for me, being that the cancer center that I was heading to was named in honor of the family who made a large contribution to Vanderbilt University Medical Center for the cancer center. This was all very surreal to me in a good way. 

We arrived in Nashville on Wednesday evening and checked into a hotel near the Vanderbilt campus. After unpacking, we drove around the college campus and noted the beauty of it all. We found the cancer center and parked our car in the convenient parking garage adjacent to the large glass building. I had Linda take my picture next to the Vanderbilt-Ingram Cancer Center sign located in front. We noticed that they had complimentary valet parking. We thought that was a nice offering, but we would forgo that service the next morning. After finding a good barbecue restaurant that evening, we went to bed with great anticipation for my appointment the next morning. 

We woke up early and took Gianni for a nice stroll in the downtown area. Nashville is a very friendly town that displays country music everywhere you look. The downtown atmosphere was relaxing and a joy to walk through. After our short dog walk, we got Gianni situated in our motel room and headed to our appointment with Dr. Rini. 

When we walked into the lobby of the cancer center, I immediately noticed the piano situated against the paneled wall of the room. It was a shiny black piano that had a calming appearance to it. It all made sense being we were in Nashville. 

The flooring was carpeted, with comfortable seating scattered throughout the spacious room. I took note of the coffeepot on the self-service bar that I thought was a nice touch. It was obvious that Vanderbilt went to great lengths to make its cancer patients feel comfortable while visiting their oncologists. We checked in with the nice lady at the front desk, who entered my information into their system, then we took a seat in the lobby. It was approximately 10 minutes to 10 a.m. when Dr. Rini’s nurse called my name. “That didn’t take long,” I thought. We followed her to the exam room, where she asked a few questions and said that Dr. Rini would be in shortly. 

At precisely 10 a.m., Dr. Rini entered the room. He didn’t have a medical chart, laptop computer or anything such as this with him. He was sharply dressed; however, he wasn’t wearing the usual white coat that most doctors wear. This created a much more relaxing atmosphere. We shook hands; he introduced himself; and we all took a seat.

He looked me in the eye and said, “I have studied your case and know it very well. Your situation is very unique, and I’m glad you’re doing so well after a rocky start to your journey.” We jumped into some particulars regarding my nebulous cancer outlook. I was very impressed that he didn’t have to refer to any notes on the topics we discussed. After a 30-minute conversation, we jointly came to a satisfactory conclusion as to how I needed to proceed going forward. As we exited the exam room, Dr. Rini said he would keep up with my progress and to never hesitate to reach out to him if I felt the need to do so. I felt comfort in knowing that. 

As I exited the Frances Williams Preston Building, I turned around, took a good look and thought, I know I came to the right place, at the right time, and saw the right doctor. This is probably the most satisfied feeling I’ve had since my cancer diagnosis. To have the validation from this wonderful cancer facility along with the work that my magnificent cancer center in Charleston has already accomplished, I couldn’t be in a better place. I’m very fortunate to have been treated by two outstanding National Cancer Institute-designated Cancer Centers, and my success in fighting this terrible disease will be a reflection of that.  

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Vanderbilt-affiliated cellular therapy programs reaccredited

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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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Children benefit in multiple ways from cellular therapy advancements

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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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Translational scientists aim to improve cellular therapies

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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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