Starting from 2014, I was trying to capture results of clinical studies in cell therapy. Today, I’d like to share some results of this attempt. I decided to narrow down my analysis to regenerative medicine, since most of cell-based therapies with published results belong to this category.


Inclusion criteria and definitions

  1. Clinical study defined as any study, which involves administration of alive cells as therapy in human and includes more than 3 subjects. It may include: registered clinical trials and “other human studies”.
  2. Analysis of published clinical trials could be interim (trial is not completed), final (trial is completed) or retrospective (post hoc analysis, long-term outcome).
  3. “Other clinical studies” may include (i) cohort studies, (ii) case series, (iii) clinical cases (3 subjects or more) and (iiii) undefined reports. “Other clinical studies” are not registered in clinical trial databases or could not be ID’d. Reports of compassionate use or hospital exemption could belong to both categories – registered as trial or have no ID.
  4. All published in medical literature reports of studies results, indexed in PubMed database.
  5. Regenerative medicine is defined (for the purpose of this analysis) as administration of alive cells to replaces or regenerates human cells, tissues or organs, to restore or establish normal function.
  6. Results of any clinical study, appeared in PubMed and/or journal web-site in the period between Jan. 1, 2014 to December 31, 2014.
  7. Mesenchymal stromal cells as immunomodulators were included in the analysis, because of multiple mechanisms of action, including tissue repair (example: GVHD)

Exclusion criteria

  1. Conference reports, companies press releases and mass media mentions.
  2. Clinical cases – reports, which describe less than 3 cases.
  3. Studies, which only describe design of the trial, but not results.
  4. Reviews.
  5. Other than “regenerative medicine” studies, which include cellular immunotherapies with immune cells in oncology, hematopoietic cell transplantation in hematology.

Search strategy
I set very loose filter for search of clinical studies results by using PubMed database with query “stem cell”. I receive notifications about new publications from this query via RSS feed. “Stem cell” query captures about 50-100 publications daily, <1% of them are clinical studies. I did random check to assess RSS feed accuracy and confirmed that it captures everything. I find that such filter allows to capture the most (if not all) clinical studies, related to cell-based therapies (by any type of cells) in regenerative medicine.  I also compared “stem cell” filter with “cell therapy” and some others. A stronger filter in PubMed – “stem cell” + “clinical trial” (check box), misses >90% of clinical studies.

Total number of studies and data capture

  • I was able to identify 116 clinical studies, which involve administration of cells as regenerative medicine.
  • Reports of cell-based regenerative medicine clinical studies results appear with a rate of 1 every 3 days.
  • I captured link to original publication, trial ID (if any), indication, country, number of subjects, type of cells, cell manipulations and some other additional information.
  • You can look at sample of raw data here.
  • 59 of 116 studies (~51%) did not have indication to any clinical trial ID.
  • 59 registered clinical trials IDs were identified in 57 published studies (2 studies described 2 registered trials).
  • About 80% of all trials were registered in NCT database (

A great variety of countries published results of clinical studies in 2014. The biggest number of reports came from China. US was the second biggest contributor. I summarized major contributing countries (more than 4 reports) in this figure:


Cell types
As one may predict, Mesenchymal Stromal Cells (MSC) was the major cell type (35%) in cell-based regenerative medicine clinical studies. Mobilized hematopoietic stem/progenitor cells (HPC-A) and bone marrow mononuclear cells (BM MNC) were another popular type of cells for tissue regeneration. Interestingly, 2 reports were about results of studies, involved embryonic stem cell-based (ESC) products. Few studies used 2 different types of cells cell simultaneously or concurrently.


Results interpretation
I loosely judged all results of clinical studies as (1) positive, (2) mixed/ inconclusive and (3) failed.
Positive are the results, reported no safety and feasibility concerns and/or provided at least some evidence of efficacy.
Mixed/ inconclusive results included:

  • end points are defined, but were not analyzed/ described in report (example)
  • efficacy end points were not defined or different end points were reported (example)
  • some end points were met, but some missed (example)
  • only some subsets of patients responded – highly depends on approach to analysis (example)
  • number of patients is too low to conclude efficacy (example)
  • efficacy declined or deterioration observed in different time points (example)

Failed studies were considered, based on termination of studies due to safety issues, lack of feasibility and/ or lack of efficacy. Failed efficacy usually reported by authors as lack of difference between control group (example: placebo) and experimental (“cells”) group. Few trials were not described as failed by authors, but judged as such by me, based on lack of significance between groups and missed end points.

The first figure shows total number of positive, mixed/ inconclusive and failed studies from analysis of all reported studies (116):


Next, I looked at only registered clinical trials and broke it down by phases – 1, 1/2, 2, 2/3 and 3:


Only one trial is failed in phase 1 and almost 90% of them reported as “positive”. The only trial, which failed in phase 1 was designed with efficacy end points. There were no trials, which failed safety. A number of mixed/inconclusive and failed trials were increased from phase 1 to 2/3. Because efficacy of therapy is usually assessed in phase 2 trials, studies labeled as “phase 1/2” are not necessarily included efficacy end points. Despite lack of any controls many studies concluded by authors as “positive” or “promising”. Placebo control was very rare. Most controls include: (i) historic, (ii) baseline and (iii) standard therapy.

Limitations of analysis

  1. The data are not validated by independent researcher.
  2. Definitions, inclusion and exclusion criteria, data interpretation were set by me and could be subjective.
  3. Thorough analysis was limited by the lack of access to some publications (I’d estimate ~10%).
  4. Only PubMed database was used for data capture.
  5. Results of many studies were very hard to interpret, because end points were not defined or/and were not reported, absence of control groups, low number of subjects. Mixed/ inconclusive results category is especially difficult to analyze. It could be interpreted differently by different people.

It was a snapshot of some data that I was able to capture in 2014. Please feel free to give me feedback and discuss these data in comments. I’m open to suggestions and collaboration.

How to cite:
Bersenev Alexey. Results of regenerative medicine clinical studies from 2014. CellTrials blog. March 1, 2015. Available:


Post updated on Feb. 17, 2015

Today I’m sharing some of my data for the last 4 years. This is a snapshot of trends in cell therapy trials from 2011 to 2014. This year, I’m planning to make few posts on cell therapy trends. I’d like to analyze some trends in mesenchymal stromal cells, adipose tissue-derived cells, industry versus academia and, finally, get to results of the trials. Some of these posts we will find on StemCellAssays.


Total number of trials
Total number of cell therapy trials, registered in international databases, continue to grow from year to year. From 2011 to 2014 a total number of trials increase more than 2 times. In 2014, every day cell therapy trial got registered in database. Please note that “registered” is not necessarily “new”.


About 75% of all databases cell therapy listings posted on US-based NCT registry ( The contribution of NCT database remains constant over the years, with range of 71-79% from all cell therapy trials, registered worldwide.

Similarly to the previous 2 years, US and China dominated all other countries in number of cell therapy trials in 2013. The US trend is keep going up, but China’s trend is getting flattened. Japan, South Korea and UK went down in 2014.

This graph shows a value of the most representative countries as % of total trials number.


There is no big difference from year to year in contribution by regions of listed cell therapy trials. However, the value of North America (read USA) steady increased from 28% to 36%.


Cell types
The positive trend for 2 “major advancers” – mesenchymal stromal cells (MSC) and T-cells is continued. T-cell curve looks especially impressive. MSC trials continue to surpass all other cell types in 2014. Another steady growing cell type is adipose-derived cells – very good positive trend. It seem like field lost interest to bone marrow mononuclear cells (BM MNC) and dendritic cells (DC) in 2014. Yet another decliner is NK cells. Embryonic stem cell trials are coming back! Three ESC trials were registered in 2014.


Malignancies is the most frequent indication for cell therapy trials. It skyrocketed in the last 3 years (increase about twice every year) with some decline in 2014. Of course, it coupled with interest to cellular immunotherapy and strictly correlates with use of immune cells (T-cells, DC, NK cells…). Interest to cell therapy in cardiovascular medicine remains pretty much flat. Muskoloskeletal diseases have a prominent positive trend. Yet another “advancer” of 2014 is neurology. Interest to cell therapy for liver diseases seem to be decreasing.


It was a snapshot of dataset analysis for the last 4 years. I’ve picked only few trends to demonstrate that the data could be “sliced and diced” in many different ways. Stay tuned for some other interesting results and trends!

How to cite:
Bersenev Alexey. Trends in cell therapy clinical trials 2011 – 2014. CellTrials blog. February 14, 2015. Available:


Cell therapy clinical trials – 2014 Report

by Alexey Bersenev on January 22, 2015 · 6 comments

in annual reports

This is 2014 report of registered cell therapy clinical trials. Every year I give a snapshot of some tracked data, captured from international clinical trials databases. You can see previous annual reports here.


Definitions and criteria
I tracked clinical trials which fall in definition of cell therapy: administration of living cells in human with therapeutic purpose. Besides “traditional cellular products”, I also included tissue engineered constructs with cells, cellular gene therapy and use of cells as a vehicles for therapeutic agent delivery. I tracked all clinical trials which were registered from Jan.1, 2014 to Dec. 31, 2014 in international registries.

The following categories were excluded from analysis:
1. Cells for homologous use:

  • hematopoietic cells for recovery of blood formation in hematological malignancies or for recovery of hematopoiesis after chemotherapy for treatment of solid tumors;
  • gene-modified hematopoietic cells for correction of metabolism errors and inherited immune diseases (example: SCID);
  • ex vivo expanded hematopoietic cells for enhancement of engraftment in hematological malignancies;

2. Platelet rich plasma trials
3. Extracorporeal devices with cells (no administration of cell inside of body).

Data mining strategy
“Hand coding” included:

  • using multiple key words and phrases
  • exclusion of overlaps between search results
  • exclusion of duplications between databases
  • reading trial description and “coding” the following categories: trial ID, country, phase, status, indication, cell type, donor type, type of sponsorship, name of company-sponsor, study acronym, number of patients in enrollment.

Key words:

    • “cell therapy”
    • “stem cell”
    • “cord blood”
    • “umbilical cord”
    • “bone marrow”
    • “cancer vaccine”
    • “tissue engineering”
    • “cellular”
    • “mesenchymal”
    • “adipose”

by name of company

Total number of trials tracked: 372
Number of duplications between databases: 8

The following databases were scanned:
European EUCTR (EudraCT)
Japanese UMIN, JMA CCT
Indian CTRI
Chinese ChiTCR
Iranian IRCT
Australian/NZ ANZCTR
Dutch NTR
South Korean CRIS

All international databases, except NCT, were scanned via WHO Search Portal (ICTRP). Each database was checked separately to capture everything, missed by ICTRP.

Databases representation:





All trials were divided on 2 categories – “academic” or “industry”. The term “academic” combined any monetary support (governments, funds, charities…) other than company-sponsored. Term “industry” also includes (1) companies – collaborators, when sponsorship is not clear from trial description and when company manufactured/ provided cellular material, (2) commercial for-profit clinics with unclear regulatory authorization.


Cell types






Abbreviations: MSC – mesenchymal stromal cells, HSPC – hematopoietic stem/ progenitor cells, TIL – tumor-infiltrating lymphocytes, DC – dendritic cells, BM – bone marrow, MNC – mononuclear cells, NK – natural killer cells, CIK – cytokine-induced killers, SVF – stromal vascular fraction; MB – mobilized blood; T-regs – regulatory T-cells; ESC – embryonic stem cells; CAR – chimeric antigen receptor; CB – cord blood; DLI – donor lymphocyte infusion.



This is a first snapshot from 2014 report. I’m planning to post trends analysis, trials results analysis and some other data. Stay tuned!

How to cite:
Bersenev Alexey. Cell therapy clinical trials – 2014 report. CellTrials blog. January 22, 2015. Available:

You’re free to share these data with appropriate credit under CC BY-ND 3.0 license.


Embryonic stem cells in cerebral palsy – results of clinical study from India

January 11, 2015

One clinical study, which was released 2 weeks before Christmas holidays captured my attention (but not attention of mass media). One of the most scandalous Indian “stem cell tourism” clinic Nutech Mediworld published(!) results of the study, which evaluates embryonic stem cell transplantation in children with cerebral palsy. Yes, you’re reading it correctly – embryonic […]

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Cell therapy clinical trials failures in 2014

January 4, 2015

Today, I’d like to highlight the most interesting, in my opinion, clinical trials failures, reported in 2014. As field is moving to efficacy (Phase 2) trials, we are starting to see more failures. In this overview, I’m going to focus on efficacy results. I hope we can learn a lot from these failures and avoid […]

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Top 10 cell therapy clinical studies in 2014

January 2, 2015

At the end of the year I analyze results of clinical studies in cell therapy. Today, I’d like to highlight 10 most significant, in my opinion, clinical studies with published results. Taking in account excellent safety profile of most cellular therapies, I was trying to focus on efficacy and long-term outcomes. Do cells really work? […]

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Cell therapy highlights from #ASH14

December 8, 2014

Annual 56th meeting of American Society for Hematology (ASH) is about to finish in San Francisco. I was following conference via twitter and I was amazed by stream of tweets – very good tweets, high value tweets, unpublished data tweets, 1 tweet per second! There was no “scientific tweets”, but almost all were “clinical”. Here, […]

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Some thoughts on results of NeoStem cardiac cell therapy trial

November 19, 2014

In the last two days I was involved in lively twitter discussions about the results of Phase 2 PreSERVE-AMI clinical trial, sponsored by NeoStem. Unfortunately, interpretation of results divided professionals for two camps – (1) trial is failed and (2) trial is successful. Why did it happen? Maybe the truth somewhere in the middle? I’d […]

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Stem Cell Autopsy: Survival and fate of fetal neural stem cells in NeuralStem ALS trial

November 16, 2014

I’ve written two years ago about autopsy findings from ALS patients, treated by neural stem cell product candidate, commercialized by NeuralStem. Recently, company published an update on pathology of spinal cords and donor cell fate after transplantation. The article is freely available online. The authors analyze 6 postmortem cases of transplantation investigational product NSI-566 into […]

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Tracking results of clinical studies in cell therapy

May 11, 2014

It has been awhile since I called to collaborate and proposed to track results of clinical trials and studies. Unfortunately, nobody expressed an interest. So, starting from this year, I’ve attempted to do it myself. Today, I’m sharing results of the first 3 months of this experiment. Methodology: I track results of: published clinical trials; […]

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