Analysis of published results of CAR-T cell therapy trials

by Alexey Bersenev on September 28, 2017 · 1 comment

in Uncategorized

Recently, Cell Trials Data started to track results of clinical trials from publications. Unlike news, social media, press releases and conference abstracts, published data allow in-depth analysis of trial settings and outcomes. For example, you can learn whether a death case was really cell product related or why dropout rate between enrollment and cell infusion was so big or why some patients were not available for evaluation at certain time points after experimental therapy and so on. Here I want to share analysis of some data, captured in “All Published CAR-T Trial Results” dataset.

Number of publications
All publications were tracked via PubMed database, using multiple combinations of search keywords. Analysis cutoff date: September 1, 2017.
74 publications, covering clinical outcomes of CAR-T cell therapy were identified, including 18 case reports and 56 trials reports. Clinical case reports, describing 1-3 clinical cases, were excluded from analysis. Most of these cases (patients) were included in trial results, published later. Trial publications reported interim or completed results. Some trials had more than one result publications and some publications covered more than one trial. 36 publications reported results of 35 CAR-T cell trials in liquid malignancies (2 trials were not registered in databases) and 20 publications reported results of 18 CAR-T cell trials in solid malignancies (3 trials were not registered).

Surprisingly (to me), 75% of published CAR-T cell trials results were available in open access. Paywalled 25% were mostly recent publications.

Output by country
As you may know, China now has the biggest number of active CAR-T cell trials. So, I was curious to look how China is publishing results of these trials. Based on my analysis, 43 (77%) publications originated from US, 9 (16%) from China and 4 (7%) from other countries (Netherlands, UK, Australia). The output from China is much lower than input because the first trial in China started in 2012 while most publications reported results of earlier trials, started in US before 2012.

Outcome in CD19 liquid malignancies
I adapted the model and graphical representation of published clinical trial results from Jessica Hartmann. Number of responses were summed from all published trials and subdivided for 3 groups: (1) overall response (complete + partial CR+PR), (2) stable disease (SD) and (3) no response/ progression of disease (NR/PD). Only evaluable patients were included. Here is results for CD19+ liquid malignancies:


Total number of CD19 publications 25, total number of evaluable patients: 379 (as of Sep 1, 2017).

Outcome in liquid malignancies – beyond CD19
10 publications reported results of CAR-T cell trials in liquid malignancies with other than CD19 target. Total number of evaluable patients: 80 (click picture to enlarge)

liquid targets

Outcome in solid malignancies
20 publications reported results of CAR-T cell trials in solid malignancies, including 11 targets and 174 evaluable patients (click picture to enlarge):

solid targets

It is important to notice that among 174 patients with solid malignancies, treated with experimental CAR-T cell therapy, only 3 CRs were observed in pediatric GD2- glioblastoma trial (NCT00085930).

All CAR-T cell trials in liquid malignancies met primary and secondary endpoints. In solid malignancies: 12/18 trials met primary and secondary endpoints, 2/18 trials did not meet safety endpoints (1 was terminated earlier due to toxicity NCT01212887), 3/18 trials had mixed results (passed safety, but no evidence for any clinical response), 1/18 – no access to the data.

Importantly, all published trials were in early phases (1 or 1/2), none of them were in late phases (2 or 3).

This is an example of data analysis, which you can utilize in your scientific presentations and market analysis. All CAR cell trials datasets you can get here. With these datasets Cell Trials Data is offering FREE 3-year subscription for semi-annual updates.

{ 1 comment… read it below or add one }

Sojin Jung October 4, 2017 at 12:36 am

Thanks for good information.


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