Alice Bertaina, MD, PhD

Stanford School of Medicine | Stanford, CA | Section Chief, Stem Cell Transplantation & Regenerative Medicine

Dr. Alice Bertaina completed her MD degree at the University of Pavia in Italy, her fellowship in hematopoietic stem cell transplantation (HSCT) at the Bambino Gesù Children’s Hospital in Rome, and her PhD degree in Immunology and Biotechnology at Tor Vergata University in Rome. Until joining Stanford University in 2017, she was Head of the Stem Cell Transplant Unit in the Department of Hematology and Oncology at the Bambino Gesù Children’s Hospital in Rome (this institution currently has the largest number of children transplanted with hematopoietic progenitors/stem cells in Europe).

Dr. Bertaina is an expert in the field of allogeneic HSCT in pediatric patients affected by hematological malignancies or nonmalignant disorders. In particular, she has pioneered the novel approach of graft manipulation based on the physical elimination of alfa/beta T cells and B cells. Dr. Bertaina has excellent clinical and biological expertise, as demonstrated by her publications in the field of pediatric hematology and oncology. Moreover, she is expert in different aspects of immunological reconstitution of children given an allograft of hematopoietic stem cell, paying particular attention to innate immunity.

Jaap Jan Boelens, MD, PhD

Memorial Sloan Kettering Cancer Center, USA | New York, NY | Chief, Pediatric Transplant and Cellular Therapy Service

Dr. Jaap Jan Boelens is a pediatric hematologic oncologist and Chief of the Pediatric Transplant and Cellular Therapy Service (TCT). He specializes in bone marrow and cord blood transplantation to treat blood diseases in children and young adults. He has particular expertise caring for people with rare diseases, such as lysosomal storage disorders like Hurler's disease and leukodystrophies like metachromatic leukodystrophy, adrenoleukodystrophy, and Krabbe disease. He is also part of a dedicated team of experts working to develop new treatments for young patients. At MSK Kids, his goal is to better control children's disease while improving their well-being, as well as that of their families.

Before coming to Memorial Sloan Kettering in 2018, Dr. Boelens spent a decade developing an umbilical cord blood transplant program in the Netherlands that is now the largest in Europe. Cord blood transplants are a promising treatment for people who don't have a suitable bone marrow match. He is continuing to build on this work by looking for ways to combine transplants with new cellular therapies. His goal is to raise the chance of eliminating disease while having less of an impact on his patients' quality of life.

In his lab research, Dr. Boelens is passionate about finding better ways to help young people with blood disorders lead longer, healthier lives. His team is developing advanced therapies made from cord blood that will target blood disorders at the cellular level. They are also creating mathematical models that can help predict how a young person's immune system will respond to these treatments. He is committed to taking discoveries out of the lab and to the bedside to help his patients.

When Dr. Boelens meets with patients and their families, they are often dealing with a lot of uncertainty. They have received a life-changing diagnosis and face questions about treatment, possible complications, the chance of responding to treatment, and how this experience will affect their family. He guides his patients and their families through these unknowns by giving them the time and space they need to ask questions and process the information they hear. He strives to be completely open and honest so his patients feel heard, respected, and cared for, spending as much time together as needed to make sure this happens.

Bruce Levine, PhD

University of Pennsylvania | Philadelphia, PA | Barbara and Edward Netter Professor in Cancer Gene Therapy

Bruce Levine is a pioneer in the field of cell and gene therapy whose work has helped change how we treat cancer. At the University of Pennsylvania’s Perelman School of Medicine, he serves as the Barbara and Edward Netter Professor in Cancer Gene Therapy and is the Founding Director of the Clinical Cell and Vaccine Production Facility (CVPF).

After earning his B.A. in Biology at Penn and his Ph.D. in Immunology and Infectious Diseases at Johns Hopkins, Dr. Levine went on to lead the cell and gene therapy product development for several “firsts” in human trials, including the first use of lentiviral vectors, the first gene-edited cell infusions, and the first use of engineered T cells to treat cancer. His work contributed directly to the development and approval of Kymriah, the first FDA-approved gene therapy, which reprograms a patient’s own immune cells (CAR T cells) to fight leukemia and lymphoma.

Dr. Levine is co-inventor on more than 30 U.S. patents and has published over 200 scientific articles and book chapters. His innovations have also led to the creation of new biotechnology companies, including Tmunity Therapeutics and Capstan Therapeutics, both spinouts from Penn. He has served as President of the International Society for Cell and Gene Therapy, as a past Board Member of the Alliance for Regenerative Medicine, and currently serves on the Board of the philanthropy Alliance for Cancer Gene Therapy.

Beyond the laboratory, he is committed to training the next generation of scientists, engaging the public in science and medicine, and expanding equitable access to advanced therapies worldwide. He has also written for outlets such as Scientific American and Wired to help explain the promise, and the challenges, of cell and gene therapy to broader audiences. Most recently he has co-written a song about CAR T cells with acclaimed country artist Mags McCarthy, “Ring That Bell”.

Megan Melody, MD, MS

Tampa General Hospital Cancer Institute | Tampa, FL | Malignant Hematologist, Lymphoma and Cellular Therapy

Dr. Megan Melody is a hematologist-oncologist specializing in blood cancers and cellular therapy. She is actively involved in clinical research, focusing on new immunotherapeutic strategies such as cellular therapies and bispecific antibodies for patients with lymphomas. Immunotherapeutics are treatments that use the body’s own immune system to fight disease and bispecific antibodies are designed to bridge two immune cells or target two different cells in the body. Tampa General’s Cellular Therapy program includes advanced treatments such as chimeric antigen receptor (CAR) T-cell therapy, which uses the body’s own immune T cells to identify and kill cancer cells.

Melody’s research focuses on lymphoma (primarily Hodgkin’s and large B-cell lymphoma), CAR T-cell therapy and bispecific antibodies. During her fellowship training at Northwestern she was extremely productive with several peer-reviewed publications, abstracts, book chapters and presentations of her research, which is of the highest caliber at regional, national and international platforms, including coveted oral presentations at the 2024 American Society of Hematology meeting and the 2025 Tandem American Society for Transplantation and Cellular Therapy and the Center for International Blood and Bone Marrow Transplant Research meetings.

Melody earned her medical degree in 2018 from the USF Health Morsani College of Medicine, where she developed a deep interest in blood cancers. She completed her residency in internal medicine at the Mayo Clinic in Jacksonville, before finishing her fellowship in hematology and medical oncology at McGaw Medical Center of Northwestern University. While at Northwestern, she gained specialized training in the treatment of complex blood cancers, including leukemia, lymphoma and multiple myeloma, with a particular focus in cellular therapy.

Sarah Nikiforow, MD, PhD

Dana-Farber Cancer Institute | Boston, MA | Technical Director, Immune Effector Cell Program

In 2020, Sarah Nikiforow, MD, PhD, embarked on a journey to standardize safety procedures for commercial chimeric antigen receptor (CAR) T-cell therapies with the goal of improving patient access to these transformative therapies. The work, supported by the American Society for Transplantation and Cellular Therapy, required collaboration across the field, including with makers of the therapies, quality accreditation bodies, medical experts, and the FDA.

Her vision became a reality when the FDA removed product-specific Risk Evaluation and Mitigation Strategy (REMS) requirements for six CAR T-cell therapies and instead deferred to the safety programs already in place at Dana-Farber and most other CAR T-cell centers. A recently approved seventh therapy followed the same approach, requiring no REMS program.

"The change signals that CAR T-cell therapies have matured from novel, unfamiliar medicines to well-understood therapies with familiar safety approaches," says Nikiforow, medical director of Connell and O’Reilly Families Cell Manipulation Core Facility. "These changes do not compromise safety for patients, and they could make receiving CAR T-cell therapy less burdensome for some individuals. They will also enable Dana-Farber and other cancer centers to offer new CAR T-cell therapies much faster once they are approved by the FDA."

The first-approved CAR T-cell therapy included this special FDA REMS safety requirement. The therapy had potentially life-threatening side effects that were unfamiliar to many providers. To mitigate the risks of these side effects, the FDA enacted extra cautionary steps mandating provider training, specific side effect management procedures, patient tracking, reporting, and auditing. In quick succession, five more CAR T-cell therapies were also approved. Each came with its own FDA-mandated REMS, meaning that each approved therapy had a different set of safety procedures.

"At a certain point, the product-specific safety parameters were actually becoming roadblocks to patient access to therapies because there was so much work needed to support each individual product," says Nikiforow. "We saw an opportunity to leverage existing standardized safety approaches, data registries, and accreditation programs."

Nikiforow partnered with Frederick Locke, MD, at Moffitt Cancer Center to bring colleagues together to advocate for application of existing safety standards to the common risks across all approved CAR T-cell therapy products. Through Nikiforow’s leadership, the collaborative working group agreed upon and published recommended management, monitoring, and reporting approaches and resources to ensure safety for these types of shared risks. The remaining list of product-specific safety concerns are managed through safety procedures unique to each product.

As new types of cell therapies enter the clinic, safety programs will be continuously reviewed and updated by experts across the field.

Matthew Porteus, MD, PhD

Stanford University School of Medicine | Stanford, CA | Associate Professor of Pediatrics (Hematology/Oncology & Human Gene Therapy)

Dr. Porteus was raised in California and was a local graduate of Gunn High School before completing A.B. degree in “History and Science” at Harvard University where he graduated Magna Cum Laude and wrote an thesis entitled “Safe or Dangerous Chimeras: The recombinant DNA controversy as a conflict between differing socially constructed interpretations of recombinant DNA technology.” He then returned to the area and completed his combined MD, PhD at Stanford Medical School with his PhD focused on understanding the molecular basis of mammalian forebrain development with his PhD thesis entitled “Isolation and Characterization of TES-1/DLX-2: A Novel Homeobox Gene Expressed During Mammalian Forebrain Development.”

After completion of his dual degree program, he was an intern and resident in Pediatrics at Boston Children’s Hospital and then completed his Pediatric Hematology/Oncology fellowship in the combined Boston Children’s Hospital/Dana Farber Cancer Institute program. For his fellowship and post-doctoral research he worked with Dr. David Baltimore at MIT and CalTech where he began his studies in developing homologous recombination as a strategy to correct disease causing mutations in stem cells as definitive and curative therapy for children with genetic diseases of the blood, particularly sickle cell disease.

Following his training with Dr. Baltimore, he took an independent faculty position at UT Southwestern in the Departments of Pediatrics and Biochemistry before again returning to Stanford in 2010 as an Associate Professor. During this time his work has been the first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients and is considered one of the pioneers and founders of the field of genome editing, a field that now encompasses thousands of labs and several new companies throughout the world.

His research program continues to focus on developing genome editing by homologous recombination as curative therapy for children with genetic diseases but also has interests in the clonal dynamics of heterogeneous populations and the use of genome editing to better understand diseases that affect children including infant leukemias and genetic diseases that affect the muscle. Clinically, Dr. Porteus attends at the Lucille Packard Children’s Hospital where he takes care of pediatric patients undergoing hematopoietic stem cell transplantation.