Cell based therapies continue to emerge as one of the most transformative and impactful areas of healthcare we have ever seen.    

From treating autoimmune diseases to regenerating and healing damaged tissues and organs, the potential of using adult living cells to heal and restore the human body is moving into the stratosphere. 

Many clinical studies have now completed their third and fourth trial phases with significant endpoints in terms of patient outcomes and safety. 

Advancements in terms of reprogramming certain cell types back to an earlier state is also significant and bypasses the ethical stigma that clouded and restricted progress for years.   

In 2025 we are seeing many more of these therapies and procedures move into the primary, secondary and community healthcare sectors.  Here we look at four significant developments. 

In March we reported on groundbreaking stem cell treatment that enabled a paralyzed Japanese patient to stand again.  The patient had been paralyzed from the neck down and after receiving an injection of induced pluripotent stem (iPS) cells to his spinal cord, he was able to regain the ability to stand again without any assistance in just four weeks. 

For this particular study, stem cell scientist, Hideyuki Okano, injected induced pluripotent stem (iPS) cells into the injury site on the spinal cords of four patients. These types of cells are created by taking mature cells, such as skin or blood cells and reprogramming them back to an earlier pluripotent state that allows them to develop into any type of cell in the human body. 

Two million of these iPS cells were injected into four patients who participated in the trials. All individuals were adult males, with two being over the age of 60. They were injected with stem cells within two to four weeks following their injury and were provided with immunosuppressant drugs to prevent their bodies from rejecting the iPS cells.

Those stem cells have the power to transform into any kind of cell the body needs, which has made them incredibly valuable for doctors looking for new ways to treat severe injuries.  Two patients showed no signs of improvement in their condition. Another patient did show improvements and has some arm and leg muscle movement. 

“The patient who received the biggest recovery is now training to walk,” Hideyuki Okano, the stem-cell scientist at Keio University in Tokyo who ran the trial, told Nature. “This is a dramatic recovery.”

This latest achievement offers new hope to those suffering from paralysis. Over 15 million people worldwide are currently living with a spinal cord injury and the majority of these patients are men.

2.     Wider Expansion of Cell-Based Therapies for Chronic Diseases

Cell-based therapies have already shown tremendous promise in terms of treating leukemia, lymphoma, and certain types of blindness.  These therapies are now expanding significantly into the treatment of chronic, non-cancerous diseases, such as heart disease, diabetes, and neuro-degenerative disorders.

A New Approach To Treat Diabetes 

For diabetes, traditional approaches, such as whole pancreas transplantations come with many barriers including the high cost, the scarcity of donor organs and invasive nature of transplant procedures. There is a great need for more scalable and cost-effective solutions.

Diabetes affects 537 million people globally.  While type 1 diabetes (T1D) and type 2 diabetes (T2D) differ in their etiology, they share an important common feature, a marked reduction in the number of functional, insulin-producing pancreatic beta cells.

For over 15 years, researchers at the Icahn School of Medicine at Mount Sinai in New York City have been at the forefront of diabetes research, striving to develop a drug capable of inducing the regeneration of human beta cells. In a significant breakthrough in 2015, they discovered harmine, a small molecule that is capable of stimulating beta cell replication.  These cells are crucial for regulating blood sugar levels. The team’s latest in vitro study involved harmine increasing and converting alpha cells to beta cells. This is a significant breakthrough as alpha cells are abundant in individuals with diabetes.

Esra Karakose, assistant professor in the Department of Medicine, is now forging ahead with studies on the molecular pathways involved for this regeneration process. 

Artificial intelligence (AI) is also playing a major role in advancing these developments. For diabetes management, a new clinical trial at the University of Virginia aims to assess an AI-powered insulin delivery system. This system is called the Bolus Priming System with Reinforcement Learning (BPS_RL). It automates the dosing process based on glucose history and meal detection. This is particularly important for children and young adults. Current insulin delivery systems require user input to adjust insulin dosages, which leads to errors and risks. 

Stem Cell Transplant Developments For Blood Cancers Set To Change 40 Years Of Standard Practice

This week, Australian researchers demonstrated that the use of a new, less toxic drug combination after stem cell transplants for leukemia significantly improves patient outcomes post-transplant, reducing the risk of the life-threatening complication of Graft Versus Host Disease (GVHD).

The trial, led by Monash University and the Australasian Leukemia & Lymphoma Group (ALLG) will transform global blood stem cell transplant outcomes for people with high-risk blood cancers.

“This new treatment triples the chances of a patient being alive, healthy and free of GVHD three years after stem cell transplant,” said lead researcher, Professor David Curtis, Director of Malignant Haematology Research at the Australian Centre for Blood Diseases, Monash University, and Clinical Haematologist and senior bone marrow transplant physician at The Alfred.

Blood stem cell transplants are often lifesaving for leukemia patients, but they come with a high risk of life-threatening complications, especially in the first 100 days after transplant.  Common side effects include infections, organ damage, and the often-debilitating Graft-versus-host disease (GVHD), an irreversible, lifelong complication. 

“The trial showed the new treatment combination is simple, safe and more effective than current methods in preventing GVHD, which contributes to death or life-long illness in 20% of patients undergoing a blood stem cell transplant,” Professor Curtis said.

“The trial has established a new benchmark for treatment in matched related donor transplantation.”

Results were published in the New England Journal of Medicine and presented at the European Hematology Association Meeting in Milan, Italy.

These results are game-changing for stem cell transplant patients, with cyclosporin and cyclophosphamide offering a new standard of care for prevention of GVHD for patients with aggressive blood cancers undergoing transplant from a match-related blood stem cell donor.

The Medical Research Future Fund (MRFF) trial was a five-year, phase 3 randomized controlled trial that was conducted across eight hospital sites in Australia and New Zealand. It tested a new drug combination in 134 patients aged 18–70, most of whom were living with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL).

Researchers compared the standard drug combination used for the last 40 years with a new combination of cyclophosphamide and cyclosporin. The new drug combination tripled the number of patients that were alive, cured of blood cancer and not suffering from GVHD three years after transplant (49.1% vs 14.2% for the standard drug combination).  Additionally, the risk of serious side effects was reduced to 19.7% from 32.4%.

Due to the success of the trial, the integration of this new treatment procedure is to be integrated immediately as the new standard of care in matched sibling transplants. 

3.     Cell Therapies Are Setting The Standard For Personalized Healthcare 

One of the most exciting developments in cell therapy is the move towards customized and personalized healthcare. 

Gene Editing and CRISPR

Gene editing tools (such as CRISPR-Cas9) are playing a major role in revolutionizing cell therapy. By editing the genes of a patient’s own cells, scientists can correct genetic mutations that cause diseases. In the next few years, we can expect to see the first widespread use of gene-edited cells for the treatment of many inherited diseases.

Expansion of Immunotherapies

Immunotherapy is one of the most promising fields in modern medicine, and in the coming years, cell-based immunotherapy will play a major role in the treatment of cancer and autoimmune diseases.

CAR-T Cell Therapy

Chimeric Antigen Receptor T-cell (CAR-T) therapy, which involves modifying a patient’s own T-cells to target cancer cells, has already shown remarkable success in certain cancers, such as B-cell lymphoma.  In 2025 we are seeing CAR-T therapies expand beyond blood cancers to solid tumors. Advancements in genetic engineering and delivery methods are improving efficacy and safety of these treatments.

TCR-T Cells and NK Cells
 

Another type of cell therapy that is gaining prominence is T-cell receptor-engineered (TCR-T) therapies, which involve engineering T-cells to target specific tumor antigens. Natural Killer (NK) cell therapies, which harness the power of the immune system’s innate defense mechanisms, will also become more widely adopted as off-the-shelf treatments, providing faster and more affordable options for patients in need of immune-based therapies.

4.      Market Expansion and Insurance