JCC-Cellular Therapeutics-Brochure

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CENTER FOR CELLULAR THERAPEUTICS

Transforming Medicine at Houston Methodist

A YEAR OF IMPACT

The year 2024 and early 2025 has been a defining period for the Johnson Center, marked by exceptional growth and achievement.

Our physician-scientists are now working on...

28

50%

cell therapy research initiatives increase from 2023

Their research targets...

17

medical conditions, collectively affecting millions of people around the world.

7

current Johnson Center projects are building collaborations with external partners including The Texas Heart Institute (THI), The University of Texas Health Science Center (UTHealth) and the University of Southern California (USC).

These efforts are remarkably addressing critical unmet health care needs across multiple medical fields. Some exciting accomplishments this past year include:

• securing first Food and Drug Administration (FDA) approval for the use of adult stem cells to treat anterior cruciate ligament (ACL) injury or tear.

• manufacturing first in-house chimeric antigen receptor (CAR) T-Cells to treat gastric cancer.

• validating a first in-human Good Manufacturing Processes (cGMP) facility exosome product to treat amyotrophic lateral sclerosis (ALS).

• playing a critical role in a first-in-human liver therapy with LyGenesis, Inc., a cell therapy company transforming patient lymph nodes into bioreactors for growing functioning ectopic organs.

Additionally, we initiated in-house therapeutic stem cell studies for orthopedic rotator cuff injury and prostate cancer that are expected to begin in 2025. This year, the Johnson Center will start the processing of hematopoietic (blood forming) stem cells and support standard of care bone marrow transplants for Houston Methodist. Cellular therapy collaborations targeting autoimmune diseases with industry partners such as Novartis are also a focus.

SIGNIFICANT FIRST ACCREDITATION

All this is possible because the Johnson Center began the year 2024 with significant first accreditation from both the College of American Pathologists (CAP) and the Foundation for the Accreditation of Cellular Therapy (FACT). These accreditations are allowing the Johnson Center to be one of the only cell therapy facilities in the Texas Medical Center to perform quality tests for itself and others.

NOVEL, DUAL-TARGETED CAR T-CELLS FOR BREAST AND GASTRIC CANCERS

Despite the success of CAR T-cell therapy as a novel cancer treatment against hematological cancers, its effectiveness against the solid tumors typically present in breast, prostate and gastric cancers is more challenged. In pursuit of a CAR T-cell therapy for these cancers, Dr. Jin has developed a novel dual-targeted CAR. To test this novel therapy, Dr. Jin has proposed a dose-escalation, first-phase trial with breast and gastric cancer patients utilizing CAR T-cells manufactured at the Johnson Center. This project is now in the final stages of FDA Investigational New Drug (IND) submission, and the clinical trial is expected to open soon.

ANN KIMBALL AND JOHN W. JOHNSON CENTER

ADVANCING REGULATORY T LYMPHOCYTE (TREG)-DERIVED EXTRACELLULAR VESICLES (EV S ) FOR AMYOTROPHIC LATERAL SCLEROSIS (ALS) THERAPY

Stanley H. Appel, MD

Peggy and Gary Edwards Distinguished Chair in ALS Research Director, Ann Kimball and John W. Johnson Center for Cellular Therapeutics

Over the past year, Dr. Appel and his Houston Methodist team have made significant strides in developing regulatory T lymphocyte (Treg)-derived extracellular vesicles (EVs) as a potential therapeutic for amyotrophic lateral sclerosis (ALS). Building on their prior success with autologous expanded Treg cell therapy in Phase 1 and 2A clinical trials, they are now optimizing and validating manufacturing processes, refining characterization and leveraging Treg-derived exosomes. This is in preparation for first-in-human clinical applications planned to follow in 2026-2027. The advantages of EV-based therapies over Treg cell therapies are compelling and include EVs not being susceptible to microenvironmental changes.

ENHANCING KIDNEY TRANSPLANTATION PREPARATION THROUGH MRNA-ENGINEERED CAR T-CELL THERAPY

Collaborators include John P. Cooke, MD, PhD; Yunki Im, PhD; Bin He, PhD; Jimmy D. Gollihar, PhD; Siddhartha Ganguly, MD; Paul Schroder, MD, PhD; and A. Osama Gaber, MD, along with his medical team.

The success of CAR T-cell therapy in cancers and autoimmune diseases has prompted its application to transplant desensitization. To prepare patients for kidney transplantation, the Johnson Center has chosen mRNA-engineered CAR T-cell therapy to target B cells and long-lived plasma cells and to eliminate anti-HLA antibodies. Studies show that combining CD 19-targeted and BCMA-targeted CAR T-cell therapies — rather than using either therapy alone — can effectively eliminate anti-HLA antibodies in highly sensitized patients and mitigate transplant rejection. Houston Methodist is deeply grateful for the platform of the Johnson Center that is bringing together all our needed expertise.

PROJECT PIPELINE

Our exciting work is only beginning. The Johnson Center Steering Committee plans to continue releasing Requests for Applications (RFA) to identify promising cell therapy research projects throughout the Houston Methodist system. Identified investigators will be funded through The Johnson Center for Cellular Therapeutics Challenge Initiative Fund at least through the proof-of-concept stage, with the goal of generating sufficient data to secure other sources of funding. Thanks to generous benevolence, support is also available through the Ann Kimball and John W. Johnson Center Endowment for Cellular Therapeutics.

Since opening in 2022, the Johnson Center has become an invaluable resource for scientists worldwide. The second annual Johnson Center Symposium on April 26, 2024, included Malcolm K. Brenner, MD, PhD, the Fayez S. Sarofim Chair and Distinguished Service Professor at Baylor College of Medicine, as the keynote speaker. Dr. Brenner is renowned in the field of stem cell transplantation, particularly in treating cancers.

The third annual Johnson Center Symposium held on April 25, 2025, featured Camillo Ricordi, MD, a Distinguished Professor of Medicine and Chief of the Division of Cellular Transplantation at the University of Miami's Miller School of Medicine. Dr. Ricordi is a leading scientist in diabetes cure research, cell transplantation and regenerative medicine.

STEERING COMMITTEE

Stanley H. Appel, MD

Director, Johnson Center for Cellular Therapeutics

Nestor F. Esnaola, MD

Interim Director, Dr. Mary and Ron Neal Cancer Center

John P. Cooke, MD, PhD

Director, Houston Methodist Center for Cardiovascular Regeneration

A. Osama Gaber, MD

Chair, Houston Methodist Department of Surgery

Patrick McCulloch, MD

John S. Dunn Chair in Orthopedic Surgery

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JCC-Cellular Therapeutics-Brochure by Houston Methodist Professional Publications - Issuu