Choice of the right target is a key to successful development of CAR cell therapy. Until now the major success of CAR-T cell therapy was related to just one target – CD19. But what about other targets? Is anything else promising in the pipeline? What targets are the most popular for liquid and solid malignancies? How is interest to different CAR cell targets changing over the years? I was trying to answer these questions using All CAR-T Trials database. Analysis cutoff date is October 1, 2017. Here I’m sharing some data.

Total number of targets
I was able to identify 51 targets, mentioned in database listings – 19 targets in liquid malignancies and 32 targets in solid malignancies. Some trials (from China) are describing multiple targets, for example NCT03291444 (CD19, CD20, CD22, CD10, CD33, CD38 CD56, CD117, CD123, CD34) or NCT03267173 (mesothelin, PSCA, CEA, HER2, MUC1, EGFRvIII). Some trials are covering 2 targets as “and/or/bispecific”, for example EUCTR2016-004680-39 (CD19/CD22 bispecific) or NCT03207178 (CD19 and CD20 sequential). Overall, 91% of trials have a single target and 9% of trials have 2 or more targets.

Value of CD19
50% of all registered CAR cell trials are targeting CD19. A small fraction of it is CD19 with other targets (and/or/bispecific):
CD19 share

Targets in liquid malignancies
What is the next most popular target after CD19? Look like CD22, BCMA and CD20 are leading, but bear in mind that most of the trials, involved CD20 and CD22 are done in conjunction with CD19 (and/or/bispecific) – click picture to enlarge:
liquid targets # trials

Targets in solid malignancies
Mesothelin captured the most attention of CAR cell therapy developers in solid malignancies. But it tightly followed by GPC3, GD2, EGFR and CEA – click picture to enlarge:
solid targets # trials

It is important to look at how interest to CAR targets was changing with time. Here is one example of the trend:
CD30 vs BCMA

As you can see from this graph, the interest to CD30 spiked last year, but now is trending down. BCMA took off in 2015 and now is trending up high. The reason – great clinical results.

You can look at any other target and draw the trend line to understand where the field is moving. Get all raw CAR cell trials data and play with it!

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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.


Historic moment for Chinese CAR-T biotech

by Alexey Bersenev on June 6, 2017 · 0 comments

in Uncategorized

The biggest and the most prestigious conference in clinical oncology (ASCO) could be a turning point for Chinese CAR-T biotech. Yesterday, presentation of preliminary data from CAR-T trial in multiple myeloma by China’s company Nanjing Legend Biotech (Legend), was a bomb! Repercussions of this success could be historic for perception of Chinese CAR-T biotech by “Western world”.

First of all, ASCO committee recognized the importance of Legend’s data and accepted their late-breaking abstract. Right after presentation, ASCO issued an immediate press release. A buzz was rapidly escalated by big media outlets (Reuters, NBC, CBS and others) as well as by coverage of specialized biothech/ medical news journalists (see: EP Vantage, Endpoints, FierceBiotech and STAT).

The data are spectacular – 100% response rate with 74% CR at 4 months (n=19). Much anticipated data release from very similar trial, run by US-based bluebird bio, was overshadowed by Chinese Nanjing Legend, presented in the same day.

Why is it a big deal for Chinese CAR-T biotech? Well, I don’t remember so much attention from “western word” to any CAR-T data (and even for any cell therapy data), coming from China, before. China has a lot of CAR-T trials going on and it has published their results (see here, here and here) and presented on prestigious conference (for example, ASH 2016 – see here and here). Despite quite good (comparable to “western world”) results, it had never got covered by western media and biotech analysts. The common perception of data from China among “westerners” was something like “it is unreliable” and “untrusted”. Would Legend’s ASCO success be a game changer for Chinese CAR-T biotech? I hope so. Well, at least now we got to know what city of Nanjing is.


Analysis of published results of clinical trials in regenerative medicine

May 6, 2017

About two years ago I attempted to analyze published results of regenerative medicine clinical studies. It was very hard and time consuming thing to do, so I’ve ditched my efforts. Recently, I was very happy to find a study by Tania Bubela’s group from University of Alberta, published in Stem Cell Reports. This is the […]

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Historical analysis of cell-based therapeutic products marketed and approved worldwide

April 7, 2017

2017 marks 20 years since the first regulatory approval of cell-based therapeutic product on a market. In 1997 Carticel (manufactured by Genzyme Corp.) was approved by FDA for US market. One year before Carticel approval, 2 cell-based products were launched and marketed for the first time in Europe (Italy) – Laserskin and Hyalograft by Fidia […]

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CAR cell therapy trials in China

February 13, 2017

Many of you know that China is very active in CAR cell therapy field. However, it is not easy to track this activity. I made an attempt to analyze CAR cell trials data from China, using All CAR-T Trials dataset from Cell Trials Data. Here are some of my findings. Until 2010, all CAR cell […]

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Number of CAR cell therapy trials worldwide

February 10, 2017

CAR (Chimeric Antigen Receptor)-based cell clinical trials is one of the hottest areas in cell therapy right now. Today, I’d like to share with you a trend for total number of CAR cell trials, registered worldwide. Importantly, I made figures available for free download on FigShare. You can get it in high resolution and use […]

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Cell therapy trials data

January 25, 2017

Dear readers, as you know I have been collecting cell therapy clinical trials data for several years now. I posted some snapshots, based on my own raw data analysis as “annual reports“. However, I have not shared or offered raw data before. I’m happy to tell you that now everyone can get raw data and […]

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Cell Therapy 2016 – Year in Review (part 2)

January 2, 2017

This is the second part of my overview of the most significant events in cell therapy in 2016. CRISPR-modified cells go clinical This year, for the first time, CRISPR gene-edited cells were used in human. Chinese oncologists from Sichuan University used CRISPR editing method to knockout PD-1 gene on autologous T-cells of the patient with […]

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Cell Therapy 2016 – Year in Review (part 1)

December 31, 2016

In the last day of 2016 I’d like to look at back and overview the most interesting events (in my opinion) and trends of the year. I’m going to split this overview for 2 parts without any particular order. Results of pivotal CAR-T cell trials Two major commercial developers of CD19 CAR-T cell therapies – […]

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