Starting from 2014, I was trying to capture results of clinical studies in cell therapy. Today, Id 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
Exclusion criteria
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
Demographics 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:
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
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. Im open to suggestions and collaboration.
How to cite: Bersenev Alexey. Results of regenerative medicine clinical studies from 2014. CellTrials blog. March 1, 2015. Available: http://celltrials.info/2015/03/01/results-regenmed-studies-2014
Continue reading here:
Cell Trials
- Stem Cells In Human Trials - September 15th, 2011 [September 15th, 2011]
- NZ Stemcell Trials - Part One - September 15th, 2011 [September 15th, 2011]
- CIRM Major Facilities Speed Stem Cell Science and Create Jobs - September 15th, 2011 [September 15th, 2011]
- Geron's Embryonic Stem Cell Clinical Trial for Spinal Cord Injury - September 15th, 2011 [September 15th, 2011]
- Hans Keirstead - Stem Cell Research Human Clinical Trials - September 15th, 2011 [September 15th, 2011]
- Stem-Cell Human Trials - September 15th, 2011 [September 15th, 2011]
- Geron: FDA Clearance for Human Clinical Stem Cell Trials - September 15th, 2011 [September 15th, 2011]
- Eric Legrand Rutgers University shout out, Listen up! - September 20th, 2011 [September 20th, 2011]
- How to Mend a Broken Heart - September 20th, 2011 [September 20th, 2011]
- Human Embryonic Stem Cell History! - September 20th, 2011 [September 20th, 2011]
- Robert Lanza - ACT - Stem cells and organ cloning interview - September 20th, 2011 [September 20th, 2011]
- Toonari Post Health News: Europe's first clinical trial using human embryonic stem cells - September 23rd, 2011 [September 23rd, 2011]
- Diabetes: Spotlight on Stem Cell Research - Ed Baetge - September 23rd, 2011 [September 23rd, 2011]
- Talking Apes being created by Science | Planet of the Apes, here we come! - September 24th, 2011 [September 24th, 2011]
- Health news: Internet use, sleep, human stem cell research - September 24th, 2011 [September 24th, 2011]
- Geron - September 24th, 2011 [September 24th, 2011]
- Batten Disease: Spotlight on Stem Cell Research - A Father's Story - September 24th, 2011 [September 24th, 2011]
- The Frankenstein Syndrome Trailer - September 24th, 2011 [September 24th, 2011]
- Leukemia: Spotlight on Stem Cell Research - Patient Stories - September 24th, 2011 [September 24th, 2011]
- Geron Corp. Initiates First Human Study Of Embryonic Stem-Cell Therapy - September 24th, 2011 [September 24th, 2011]
- Cardiovascular Therapies: Spotlight on Stem Cell Research - Yung-Wei Chi - September 24th, 2011 [September 24th, 2011]
- Stemcells come alive: Fix Mouse with severed spine ABCnews - September 24th, 2011 [September 24th, 2011]
- StemCells Gains 5% on Neural Stem Cell Trial in Spinal Cord Injury - September 24th, 2011 [September 24th, 2011]
- Stem-Cell Research on Animals - September 24th, 2011 [September 24th, 2011]
- First hESC Therapy Clinical Trial - September 24th, 2011 [September 24th, 2011]
- Stem Cell Testing on Human Subjects [Officially] Begins • NBC Nightly News • October 11th, 2010 - September 24th, 2011 [September 24th, 2011]
- Leukemia: Spotlight on Stem Cell Research - Catriona Jamieson - September 24th, 2011 [September 24th, 2011]
- First stem cell trial begins - September 24th, 2011 [September 24th, 2011]
- Parkinson's Disease: Spotlight on Stem Cell Research - Arnold Kriegstein - September 24th, 2011 [September 24th, 2011]
- Becoming a Blood Stem Cell Donor - September 24th, 2011 [September 24th, 2011]
- Stem cells might be able to reverse the effects of a heart attack. - September 24th, 2011 [September 24th, 2011]
- Horse Stem Cell Video.mp4 - September 25th, 2011 [September 25th, 2011]
- Stem cell trials to begin - September 25th, 2011 [September 25th, 2011]
- Lou Gehrig's Disease (ALS): Progress and Promise in Stem Cell Research - September 25th, 2011 [September 25th, 2011]
- Hans Keirstead - September 25th, 2011 [September 25th, 2011]
- Stem Cell Quiz - September 25th, 2011 [September 25th, 2011]
- NZ Stemcell Trials - Part Two - September 25th, 2011 [September 25th, 2011]
- Drug Trials in a Dish - September 25th, 2011 [September 25th, 2011]
- Rise and Walk - September 25th, 2011 [September 25th, 2011]
- Catriona Jamieson: Therapies Based on Cancer Stem Cells - September 27th, 2011 [September 27th, 2011]
- Vatican lending hand in adult stem cell research - September 28th, 2011 [September 28th, 2011]
- Why STEM-Enhance? - September 28th, 2011 [September 28th, 2011]
- Genetic Underpinnings of Alopecia Areata - September 29th, 2011 [September 29th, 2011]
- Batten Disease: Spotlight on Stem Cell Research - Ann Tsukamoto - September 29th, 2011 [September 29th, 2011]
- stem cell trial for leading causes of blindness - October 9th, 2011 [October 9th, 2011]
- Forbes Discusses Stem Cell Research - Video - October 13th, 2011 [October 13th, 2011]
- Bruce Conklin: Drug screening with stem cells - Video - October 13th, 2011 [October 13th, 2011]
- Human Trials of Embryonic Stem Cell Treatment Beginning - Video - October 13th, 2011 [October 13th, 2011]
- Rise and Walk - Video - October 13th, 2011 [October 13th, 2011]
- Mesenchymal Stem Cell Trials - Video - October 14th, 2011 [October 14th, 2011]
- Spinal Cord Injury: Progress and Promise in Stem Cell Research - Video - October 14th, 2011 [October 14th, 2011]
- Better Drugs Through Stem Cells - Video - October 16th, 2011 [October 16th, 2011]
- Adult Stem Cells Used To Rebuild Heart Tissue / Video - Video - October 16th, 2011 [October 16th, 2011]
- GARY RABIN- ADVANCED CELL TECHNOLOGIES 2011 - Video - October 21st, 2011 [October 21st, 2011]
- Stem cell treatment on horse a success, vet says - Video - October 21st, 2011 [October 21st, 2011]
- MAJOR EMBRYONIC STEM CELL BREAKTHROUGHS - Video - October 22nd, 2011 [October 22nd, 2011]
- Batten Disease: Spotlight on Stem Cell Research - Stephen Huhn - Video - November 13th, 2011 [November 13th, 2011]
- Human Cloning has Begun | Playing God? - Video - November 15th, 2011 [November 15th, 2011]
- Diabetes: Advancing Stem Cell Therapies - 2011 CIRM Grantee Meeting - Video - November 15th, 2011 [November 15th, 2011]
- Brain Tumors: Advancing Stem Cell Therapies - 2011 CIRM Grantee Meeting - Video - November 15th, 2011 [November 15th, 2011]
- Double Blind Trial of Stem Cells for Heart Failure - Video - December 4th, 2011 [December 4th, 2011]
- Cardiac Stem Cell Therapy Clinical Trial - Video - December 4th, 2011 [December 4th, 2011]
- Glasgow Hospital Conducts First Stem Cell Trials on Stroke Patients - Video - December 4th, 2011 [December 4th, 2011]
- Dr Dietrich discusses the Geron stem cell trial to treat acute spinal cord injury - Video - December 4th, 2011 [December 4th, 2011]
- Stem Cell Research Today: Larry Goldstein - CIRM Science Writer's Seminar - Video - December 7th, 2011 [December 7th, 2011]
- Stem Cell Clinical Trial for Heart Failure: Eduardo Marban - CIRM Spotlight on Disease - Video - December 22nd, 2011 [December 22nd, 2011]
- Clinical trials and stem cells: what patients should consider - Video - January 2nd, 2012 [January 2nd, 2012]
- Spare Parts for Humans: Tissue Engineers Aim for Lab-Grown Limbs, Lungs and More - Video - January 4th, 2012 [January 4th, 2012]
- CIRM Grant Writing: RFA's - Where does your Project Fit? - Video - January 5th, 2012 [January 5th, 2012]
- Prometheus and I: building new body parts from stem cells (15 Nov 2011) - Video - January 5th, 2012 [January 5th, 2012]
- News Bulletin 18 November 2011 -- The Christian Institute - Video - January 5th, 2012 [January 5th, 2012]
- The Brain on Trial: How Neuroscience Challenges the Law as We Know It - Video - January 5th, 2012 [January 5th, 2012]
- The Human Genome and Individualized Medicine - David Valle, MD - Video - January 5th, 2012 [January 5th, 2012]
- 2010 Professional of Distinction - Discovery Awards - Video - January 23rd, 2012 [January 23rd, 2012]
- Research at The Nebraska Medical Center Biologics Production Facility - Video - January 24th, 2012 [January 24th, 2012]
- ACT Announces Aberdeen Royal Infirmary in Scotland as Additional Site for Phase 1/2 Clinical Trial Using hESC-Derived ... - January 30th, 2012 [January 30th, 2012]
- StemCells, Inc. Announces Publication of Preclinical Data Demonstrating Its Human Neural Stem Cells Preserve Vision - January 31st, 2012 [January 31st, 2012]
- $30 million donation from Boris family will help McMaster turn stem cell research into therapy - February 6th, 2012 [February 6th, 2012]
- Stem Cell Treatment For Blindness Shows Promise In Trials - February 7th, 2012 [February 7th, 2012]
- ACT Announces Third Patient with Stargardt’s Disease Treated in U.S. Clinical Trial with RPE Cells Derived from ... - February 13th, 2012 [February 13th, 2012]