A groundbreaking therapy based on transplanting mitochondria into blood stem cells has shown encouraging results in an ongoing, phase 2 trial. If successful, the therapy could not only treat rare, incurable diseases, but also potentially help reverse some of the effects of ageing.

What is mitochondrial dysfunction?

Mitochondria are tiny organs (organelles) found within cells, which work to produce the energy the cell needs to perform the rest of its functions. When they stop functioning correctly, the cells they are in are starved of energy, leading to a wide variety of potential issues depending on the cells affected.[1]

This dysfunction is the hallmark and cause of several diseases and conditions, collectively referred to as mitochondrial disease. These are complex diseases frequently involving multiple organs in the body; although they range in severity, many are ultimately fatal, and there are currently no treatments other than palliative care.[2][3] Many are early onset, developing in babies and young children.[4] This is the case for Pearson syndrome and Kearns-Sayre syndrome, the two conditions on which the therapy is currently being tested.[2][5]

In addition to these, mitochondrial dysfunction has also been linked to several other currently untreatable conditions, including neurodegenerative diseases such as Alzheimer’s[6], Parkinson’s[2] and amyotrophic lateral sclerosis[7], as well as cardiovascular diseases, chronic kidney disease, diabetes and more.[2]

How is this connected to ageing?

As we age, our bodies become less able to function well, and this includes the mitochondria. When they are working correctly, mitochondria produce limited quantities of Reactive Oxygen Species (ROS), which are unstable, highly reactive molecules containing oxygen. ROS are a normal byproduct of cellular metabolism, and play a role in helping cells communicate and remain balanced. Mitochondria that function poorly, however, produce excessive quantities of ROS, which can cause damage to various parts of the cell, including mitochondria. This leads to a vicious cycle where mitochondria functionality continues to worsen and ROS production continues to increase.[8][9][10] There is also evidence that inefficient energy production in mitochondria is what causes the loss of muscle mass and function in the elderly (sarcopenia).[11][12]

This growing body of research, combined with the connection between mitochondrial dysfunction and conditions which occur more frequently in the elderly, such as Alzheimer’s, suggests that treating mitochondrial dysfunction could slow, or even potentially reverse, the ageing process.[2][3][8][9]

How could a mitochondria transplant help?

The therapy, which has been in development for over a decade at Minovia, an Israeli company, hinges on extracting mitochondria from healthy cells from a donor. Scientists then take blood stem cells from the patient and enrich them with the healthy mitochondria. The enriched cells are then infused back into the patient’s bloodstream.[2][3]

The first generation of the therapy used mitochondria from white blood cells. The second generation currently being trialled uses mitochondria collected from the placenta; this is a young, healthy organ, which contains what has been described as ‘super mitochondria’.[3]

On young patients suffering from Pearson syndrome and Kearns-Sayre syndrome, the therapy is safe and has had marked improvements on physical development, energy, and quality of life.[3][5]

The research team believes that the same therapy could also help elderly patients suffering from mitochondrial dysfunction. A clinical trial for myelodysplastic syndrome, a rare type of blood cancer potentially linked to mitochondrial dysfunction, is ongoing.[13] The team is working on the development of biomarkers to test whether older patients are experiencing mitochondrial dysfunction, with an ultimate goal of trialling the therapy on elderly patients beginning in 2026.[3]

The power of the placenta

Although the placenta is frequently discarded as medical waste after birth, it is a valuable source of young, powerful cells. These could be the key to the treatment of diseases for which we currently have no cure. What’s more, they could prove to be invaluable for anti-aging treatments that could enable us to live longer, healthier lives. “We could,” says Dr Natalie Yivgi-Ohana, CEO and cofounder of Minovia, “find it to be the fountain of youth.”[3] To learn more about the placenta, and how you could preserve it for future use rather than discarding it, fill in the form below to request our free guide.

References

[1] Newman, T. (2018). Mitochondria: Form, function, and disease. MedicalNewsToday. https://www.medicalnewstoday.com/articles/320875

[2] Minovia. (2025). Mitochondrial Augmentation Technology (MAT). https://minoviatx.com/therapy/

[3] Knapton, S. (2025). The pioneering therapy that could roll back ageing. The Telegraph. https://www.telegraph.co.uk/news/2025/08/16/the-pioneering-therapy-that-could-roll-back-rigours-ageing/

[4] Rahman, S. (2020). Mitochondrial disease in children. Journal of Internal Medicine, 287(6). doi:https://doi.org/10.1111/joim.13054

[5] GlobeNewswire. (2025). Minovia Therapeutics Announces Interim Data from Phase 2 Trial in Pearson Syndrome Demonstrating No Treatment-Related Serious Adverse Events and Preliminary Signal for Efficacy Measured by Growth.

[6] Morteza Abyadeh, et al. (2021). Mitochondrial dysfunction in Alzheimer’s disease – a proteomics perspective. Expert Review of Proteomics, 18(4), pp.295–304. doi:https://doi.org/10.1080/14789450.2021.1918550

[7] Muyderman, H. and Chen, T. (2014). Mitochondrial dysfunction in amyotrophic lateral sclerosis – a valid pharmacological target? British Journal of Pharmacology, 171(8), pp.2191–2205. doi:https://doi.org/10.1111/bph.12476

[8] López-Otín, et al. (2013). The Hallmarks of Aging. Cell, 153(6), pp.1194–1217. doi:https://doi.org/10.1016/j.cell.2013.05.039

[9] Wei, P., et al. (2025). Mitochondrial dysfunction and aging: multidimensional mechanisms and therapeutic strategies. Biogerontology, 26(4). doi:https://doi.org/10.1007/s10522-025-10273-4

[10] Somasundaram, I., et al. (2024). Mitochondrial dysfunction and its association with age-related disorders. Frontiers in Physiology, 15. doi:https://doi.org/10.3389/fphys.2024.1384966

[11] Marzetti, E., et al. (2013). Mitochondrial dysfunction and sarcopenia of aging: From signaling pathways to clinical trials. The International Journal of Biochemistry & Cell Biology, 45(10), pp.2288–2301. doi:https://doi.org/10.1016/j.biocel.2013.06.024

[12] Marzetti, E. and Leeuwenburgh, C. (2006). Skeletal muscle apoptosis, sarcopenia and frailty at old age. Experimental Gerontology, 41(12), pp.1234–1238. doi:https://doi.org/10.1016/j.exger.2006.08.011

[13] Clinicaltrials.gov. (2025). A Study to Evaluate the MNV-201 in Patients with Low Risk MDS. https://clinicaltrials.gov/study/NCT06465160

A team of scientists at UCLA, United States, has shown it’s possible to reprogram haematopoietic stem cells to generate a regular supply of cancer-killing T cells. This novel approach, tested in a first-of-its-kind clinical trial the results of which were published in Nature Communications, could potentially offer longer-lasting protection from cancer.

T-cell cancer therapies and their challenges

T cells are a type of lymphocytes, white blood cells which are part of the immune system and move around the body finding and destroying abnormal cells to fight infection and disease. However, sometimes it can be difficult for T cells to tell the difference between cancerous cells and normal cells. This enables the cancerous cells to avoid destruction.[1]

T-cell cancer therapies collect T cells from the patient’s blood through a process called apheresis. Following this, the T cells are modified in the laboratory to make them better able to find and destroy cancerous cells. Then, the patient undergoes conditioning chemotherapy to weaken the immune system, in order to ensure the modified T cells have little competition from other T cells and can therefore be as effective as possible. Lastly, the modified T-cells are infused back into the patient.[1]

One type of T-cell therapy is CAR T-cell therapy. This therapy modifies T cells by adding a protein, called a Chimeric Antigen Receptor (CAR), to their surface, enabling them to recognise a specific protein on the surface of cancer cells. CAR T-cell therapies are approved in the UK for children and adults with B cell acute lymphoblastic leukaemia, as well as adults with some types of lymphoma.[1][2]

These therapies have been successful in treating blood cancers where other types of treatment have failed. However, solid tumours, such as melanoma or breast cancer, are more difficult to target with cell therapies. This is due to the cancerous cells being more challenging to target without harming other tissues, as well as the need for cell therapies to get through body tissue to get to the tumour. Some cell therapies have recently been approved for use in clinical care in the United States, with more  currently in development,[3] including the novel approach developed by the research team at UCLA.

How is the new therapy different?

One of the problems with T cell therapy for solid tumours is that while the therapy works at first, resulting in shrinking of the tumour in a large proportion of patients, this result is not sustainable, with patients often relapsing within 6-12 months of the treatment. This is because the number of modified T cells in the body decreases over time. What’s more, the ones that remain also become less effective. Thus, the need for a self-renewing source of these cells beyond the original infusion at point of treatment.[4]

Indeed, the therapy developed by the research team at UCLA features a novel, two-pronged approach. First, patients underwent a process called mobilisation, which involves using medication to stimulate the production of peripheral blood stem cells (PBSCs) and then collecting them through apheresis. The stem cells were then modified in the lab to enable them to give rise to T cells able to target a specific tumour marker (NY-ESO-1). Once the modified stem cells were ready and had been tested, T cells were collected from patients through a second apheresis and modified to be able to target the same NY-ESO-1 tumour marker. Following conditioning chemotherapy, both the modified stem cells and the modified T cells were reinfused into the patients.

What were the trial results?

Five patients with relapsed or treatment-resistant metastatic sarcoma, a type of solid tumour, were recruited into the trial; two had to drop out of the study, and three ultimately received the treatment. Of the three, two had a positive response to the treatment, with a reduction in tumour size; the third showed no response, something which could possibly be attributed to the conditioning chemotherapy not being as effective as it could have been.

In one of the patients who had a positive response, the modified stem cells failed to engraft and produce any new T cells. Researchers attributed this to the fact that the process of modifying the stem cells, although successful, produced results that were at the lowest acceptable level for use in the clinical trial.

The last patient had successful engraftment of the stem cells, which began to produce new T cells. Unfortunately, she passed away during the study from a respiratory infection which arose as a complication of the conditioning chemotherapy.

The road towards a treatment for cancer

This was a small, preliminary trial, limited to a handful of patients with advanced cancer. The results demonstrated that the two-pronged therapy approach is feasible and can work. However, the treatment needs further development and more extensive testing before it can be deemed ready for clinical use. What’s more, it may not be suitable for all patients, given the dangers inherent to conditioning chemotherapy for those who are already very weak.

Still, there is no doubt that stem cells could hold the key to more effective treatments than those we already have, as well as cures for conditions and diseases currently considered incurable. Indeed, the researchers posit that variations on the approach tested in this trial might be used to target a wide variety of other diseases, such as HIV.

There are many possible sources of stem cells, such as from cord blood, fat, bone marrow, or peripheral blood, the latter of which was used in this particular trial. Some of these sources are always available throughout your lifetime, although they may be easier or harder to obtain stem cells from at the point of treatment. On the other hand, others, such as cord blood and other perinatal (birth-related) sources of stem cells, can only be obtained at specific times.

It is impossible to know, right now, which sources of stem cells could be most effective for therapies which are still in the early stages of development today. Your baby’s future health could depend on having access to as wide a variety of stem cell sources as possible, to enable the widest range of treatment options. To learn more about how you could preserve a rich source of stem cells for your baby right when they are born, fill in the form below to receive a free guide to stem cell banking.

References

[1] Cancer Research UK (2025). CAR T-cell therapy. https://www.cancerresearchuk.org/about-cancer/treatment/targeted-cancer-drugs-immunotherapy/car-t-cell-therapy

[2] Anthony Nolan. (2024). What is CAR T-cell therapy? https://www.anthonynolan.org/patients-and-families/what-car-t-cell-therapy

[3] University College London Hospitals NHS Foundation Trust. (2021). UCLH to drive forward research in cell therapies for solid tumours. https://www.uclh.nhs.uk/news/uclh-drive-forward-research-cell-therapies-solid-tumours

[4] Nowicki, T.S., et al. (2025). Human cancer-targeted immunity via transgenic hematopoietic stem cell progeny. Nature Communications, 16(1). doi:https://doi.org/10.1038/s41467-025-60816-z

The liver is a remarkably resilient organ – the only organ in the body capable of regenerating itself when damaged.[1] Damage to the liver does, however, build up over time, eventually leading first to chronic (long-term) liver disease and then, inevitably, to liver failure.[2] If liver failure occurs suddenly and also involves the failure of other organs, it is called acute-on-chronic liver failure (ACLF).[3]

A lack of treatment options

The odds of ACLF occurring increase as a patient’s chronic liver disease worsens, ranging from around 7%[4] in the early stages of cirrhosis (which itself is the final stage of chronic liver disease) to about 35%[5] in the end stage. It can, however, even happen in very early chronic liver disease, although this is quite rare.[6]

ACLF is generally triggered by something negative happening to the liver, such as drinking alcohol, bacterial infections, sepsis, or a hepatitis flare[7][8]. In up to 40-50% of cases, though, the trigger is never conclusively identified.[8] Whatever the cause, the result is an uncontrolled, intense systemic inflammation, which then leads to organ damage and failure. This, in turn, worsens the inflammation, leading to a vicious cycle.[3][9] Depending on the severity of the organ failure, 28-day mortality for ACLF can vary from around 18% to as high as nearly 89%.[7]

Treatment options for ACLF are limited to identifying and treating the triggering event, as well as supporting failing organs and managing complications while helping the body to recover.[7][10] A liver transplant is the only effective treatment, with survival rates for patients with severe ACLF who receive a transplant reaching nearly 80%.[7] However, not every patient may be eligible for a transplant[10] or, for that matter, find a match in time, given how quickly ACLF progresses[3][11]. There is therefore an urgent need for a treatment for ACLF that does not rely on a liver transplant.

Addressing the root cause

Because systemic inflammation is a key component of ACLF, an effective treatment must target this as well as the underlying liver damage. This is not always possible with currently available treatment options. Treating the trigger for the inflammation can make a difference,[11] but this cannot even always be conclusively identified. Moreover, the only available method to treat liver damage once it has reached the chronic stage is a transplant, which, as previously mentioned, is not always an option.

Mesenchymal stem cells (MSCs) are powerful, tissue-forming stem cells, which can help in wound healing and tissue regeneration. MSCs also have a strong potential for regulating the body’s immune response and reducing inflammation. It is these properties that have drawn researchers’ attention to their potential uses in the field of regenerative medicine,[6][12] and that could make a difference in the treatment of ACLF by helping to regenerate the liver as well as reducing systemic inflammation.[3][6]

MSC therapy for ACLF is in the early stages of development; recently, a systematic review and meta-analysis of six clinical trials and one retrospective study was conducted, in order to better assess its safety and effectiveness.[3]

What were the results of the analysis?

Across the board, MSCs proved to be a safe treatment for ACLF, with no adverse events or serious side effects being recorded in any of the individual studies. What’s more, study results indicate the treatment could indeed prove to be effective.

Firstly, the review authors pooled data from a total of 363 patients to analyse scores on the Model for End-Stage Liver Disease (MELD) scale. This scale is used to assess the severity of chronic liver disease and determine the urgency of a liver transplant.[13] MELD scores were found to have significantly decreased in patients who underwent the treatment, indicating an improvement in liver function and a better chance of survival.

Next, the authors analysed the reported data on levels of albumin, a key protein made by the liver. Across the studies which reported them, albumin levels were shown to have increased, confirming the boost to liver function. Other markers of liver function also improved, although these were not found to have been statistically significant.

The authors also performed a time analysis, hoping to determine the best time to perform the treatment. They found that liver function improved at 4 and 24 weeks post-therapy in the treatment group when compared to the control group. However, there were no statistically significant differences between the two groups either earlier (2-week mark) or longer term. Thus, the authors suggest that the best result may be achieved by repeating the treatment once efficacy begins to wane.

Furthermore, the authors note that more research is needed to identify the best treatment route, as there currently isn’t enough data to conclusively determine this. Scientists are also still investigating the best source of MSCs for treatment; of the many possible sources, most of the studies included in the review used MSCs from the umbilical cord.

The promise of stem cells

Larger, more wide-ranging trials are needed to determine whether MSC treatment for ACLF is truly effective. Still, this research, along with ongoing studies into the use of stem cells for the treatment of other diseases and conditions, highlights the potential of regenerative medicine. Banking your baby’s umbilical cord stem cells at birth could offer a significant advantage, providing a readily available, personal source of cells for future treatments without having to rely on donated cords or other sources. To learn more about harnessing this potential for your baby’s future health, fill in the form below to request your free guide to stem cell banking.

References

[1] British Liver Trust. (2025). About your liver. https://britishlivertrust.org.uk/information-and-support/liver-health-2/abouttheliver/#repair

[2] John Hopkins Medicine (2019). Chronic Liver Disease/Cirrhosis. https://www.hopkinsmedicine.org/health/conditions-and-diseases/chronic-liver-disease-cirrhosis

[3] Lu, W., et al. (2025). Efficacy and safety of mesenchymal stem cell therapy in acute on chronic liver failure: a systematic review and meta-analysis of randomized controlled clinical trials. Stem Cell Research & Therapy, 16(1). doi:https://doi.org/10.1186/s13287-025-04303-8

[4] Mahmud, N., et al. (2019). Incidence and Mortality of Acute‐on‐Chronic Liver Failure Using Two Definitions in Patients with Compensated Cirrhosis. Hepatology, 69(5), pp.2150–2163. doi:https://doi.org/10.1002/hep.30494

[5] Mezzano, G., et al. (2022). Global burden of disease: acute-on-chronic liver failure, a systematic review and meta-analysis. Gut, [online] 71(1), pp.148–155. doi:https://doi.org/10.1136/gutjnl-2020-322161

[6] Khanam, A. and Kottilil, S. (2021). Acute-on-Chronic Liver Failure: Pathophysiological Mechanisms and Management. Frontiers in Medicine, 8. doi:https://doi.org/10.3389/fmed.2021.752875

[7] Kumar, R., Mehta, G. and Jalan, R. (2020). Acute-on-chronic liver failure. Clinical Medicine, 20(5), pp.501–504. doi:https://doi.org/10.7861/clinmed.2020-0631

[8] Shah, N.J., Mousa, O.Y., Syed, K. and John, S. (2021). Acute On Chronic Liver Failure. PubMed. https://www.ncbi.nlm.nih.gov/books/NBK499902/

[9] Zaccherini, G., Weiss, E. and Moreau, R. (2021). Acute-on-chronic liver failure: Definitions, pathophysiology and principles of treatment. JHEP Reports, 3(1), p.100176. doi:https://doi.org/10.1016/j.jhepr.2020.100176

[10] AASLD. (2025). Management of Acute on Chronic Liver Failure in the Hospitalized Patient. https://www.aasld.org/liver-fellow-network/core-series/clinical-pearls/management-acute-chronic-liver-failure

[11] British Liver Trust. (2025). What is cirrhosis? https://britishlivertrust.org.uk/information-and-support/liver-conditions/cirrhosis/#aclf

[12] Margiana, R., et al. (2022). Clinical application of mesenchymal stem cell in regenerative medicine: a narrative review. Stem Cell Research & Therapy, [online] 13(1). doi:https://doi.org/10.1186/s13287-022-03054-0

[13] Cleveland Clinic. (2025). MELD Score: How It’s Calculated & Interpreting Results. https://my.clevelandclinic.org/health/diagnostics/meld-score