Late in 2015, one of the pioneering stem cell and regenerative medicine biotechs, Ocata Therapeutics (fka as ACT or Advanced Cell Technology) was acquired by Astellas Pharma. At the time of purchase, Ocata had a number of areas of focus, but was most closely followed for its development of human embryonic stem cell-based retinal pigmented epithelial cells (RPEs) for treatment of multiple kinds of macular degeneration. These products were advancing in promising clinical trials and had demonstrated good safety profiles.
After the purchase of Ocata by Astellas, many in the field wondered what impact this development might have on these clinical trials. Could the additional resources within the Astellas environment give a big boost to the former Ocata work? Some on the other hand worried that within the giant Astellas, these stem cell pipelines might get a bit lost, especially if Ocata leader Dr. Robert Lanza ended up leaving Astellas.
So what’s the scoop on all of this now?
Dr. Lanza, Head of Astellas Global Regenerative Medicine, discusses the future of #regenerativemedicine at the Astellas R&D Meeting. pic.twitter.com/HxRXl0cv0P
— Astellas Pharma US (@AstellasUS) December 8, 2016
The Ocata work is very much alive and well, and Bob Lanza is still at Astellas (see above tweet from December of last year). He is Head of Astellas Global Regenerative Medicine & Chief Scientific Officer, Astellas Institute for Regenerative Medicine. I reached out to him to ask for an update on how things are going with the regenerative medicine efforts at Astellas.
Dr. Lanza was able to share this comment, “ASP7317 for Dry AMD is currently in Phase 2 studies, while the Stargardt study is in Phase 1/2a. It would preliminary to comment on timing for study expansion and next steps.”
I know that’s not a ton of information, but it is a bit encouraging at least. Also from emailing with him, I’m hoping in coming months to have an interview with Dr. Lanza with more detail.
Would be very grateful if you could let me know whether this treatment is also applicable to patients of central retinal artery occlusion. I have suffered this last year, lost partial vision in my right eye. Desperately seeking treatment. Would sincerely appreciate a feedback.
Hemalatha
India.
Well Astellas is continuing to increase the number of participating eye centers for the hrpe trial, they are up to 8 now.
https://clinicaltrials.gov/ct2/show/NCT03178149?term=Astellas+Institute+for+Regenerative+Medicine&draw=1&rank=2
Later
Bill
So Astellas has given a couple of updates on the “Cell Therapy” wing of their companies efforts, Here are the links and one thing I noticed is they seem to have moved to the phase 2 aspect or POC of the new clinical trial for AMD, or at least they are saying this as compared to their update in December where they categorized the trails as 1b and phase 2 and now are showing only phase 2 which is the POC phase of the current trial.
December:
https://sw4503.swcms.net/en/ir-library/ir-meetings/inframe/main/00/teaserItems1/03/linkList/0/link/FINAL_2_181212_RD%20Day_eg.pdf
January:
https://sw4503.swcms.net/en/ir-library/pipeline/inframe/main/01/teaserItems1/0/linkList/0/link/3q2019_pipeline_en.pdf
They are also showing some pretty interesting future moves in the “Cell Therapy” moves they intend, like the IND for Hemangioblast MSC’s Dr Lanza was working on before, in 2020.
Later,
Bill
Thank you SO much for the update, Bill!! 🙂 Some of us are waiting, hoping, praying, and everything else-ing for news on this front. Please keep sharing anything you find out.
I am wondering if there is anyone that can ask Dr. Lanza or someone else like Astellas any follow up questions regarding the ongoing trial? Mr. Paul, Irv, anyone?
The newly started trial has been going since July of 2018 when the first patient was dosed, I would have to guess there has been progress in the patients status or they would not have decided to add to this portfolio and the trial would have been stopped if any sort of complication had occurred.
They have expanded the number of sites recruiting, increased their investment in the cell therapy space, which includes gene therapy with the Quethera aquistion and the expansion of the manufacturing facilities in Japan and the US for cell production presumably for the Universal Cell technology of allogenic cells.
Something must be going pretty good, just my opinion.
Later,
Bill
Here a couple of small articles about Astellas’s continued commitment towards cell therapy and such.
https://www.biopharma-reporter.com/Article/2018/08/21/Astellas-prioritises-ophthalmology-with-gene-therapy-acquisition
https://www.biopharma-reporter.com/Article/2018/11/07/Astellas-invests-256m-in-cell-therapy-manufacturing-buildout
I thought it interesting they are expanding the manufacturing side so dramatically.
Later,
Bill
The Astellas trial is updating it’s locations, including Wills Eye and Jules Stein Eye and a couple of others.
The first patient was dosed July 13th, so I would guess they already have seen some results.
Here is a link to the “Changes” on the Clinicaltrials.gov site:
https://clinicaltrials.gov/ct2/history/NCT03178149?A=1&B=8&C=merged
Later,
Bill
Thanks, Bill. Very interesting. I’m hoping this is an encouraging sign of picked up activity.
Yes I also hope for a good push now.
I expect you know this, but the patient enrollment in the trial is 150 I believe, so I would guess they will have to pick up the pace a bit or enlist a lot of sites.
Later,
Bill
I just saw this, and I’ll read it all!! THANK YOU Bill… you’re a great human being. 🙂
Astellas has finally started recruiting for its clinical trial, time will tell if the new cell line is comparable.
Later,
Bill
@Bill, Do you have a link regarding this new recruitment?
Here is the link to the trial below;
https://clinicaltrials.gov/ct2/show/NCT03178149?term=Astellas+Institute+for+Regenerative+Medicine&rank=2
Of course there are numerous follow up trials also continuing for the previous trials
Later,
Bill
Thanks, Bill.
Nice greetings from Runcaly to all …
Here is the Clinicaltrials.gov link to the new trial being set up.
https://clinicaltrials.gov/ct2/show/NCT03178149?term=Astellas+Institute+for+Regenerative+Medicine&rank=2
Bill
Paul
Irv is correct as i understand Lanza’s progress or lack of it To bad for the Regen fiel
Andy– just in case you signed up to get notifications only on replies to your comment, take a look below!!
Irv’s correct. The MA09 hESC cell line used by Ocata/ACTC’s RPE program was a hot topic during the iCell forum days and many threads were peppered with discussion on the topic. It’s a 2005 Blastomere derived cell line but the embryo was discarded rather than frozen, hence not considered a true non-embryo-destruction (NED) line, which were also created by Ocata/ACTC and submitted to the NIH registry for approval (NED1/2/3/4). They remain pending due to the 2010 proposed NIH guideline redefintion to include the Blastomere technology amended wording (which incidentially still hasn’t been adopted and may never due to the concerns surrounding the remaining embryo post Blastomere cell extraction even though the existing guidelines encompass the standard destructive hESC method from the blastocyst stage ICM).
https://grants.nih.gov/stem_cells/registry/pending.htm
The culture method used to create the cell line was also needed to be updated to adhere to newer/better formulation techniques and perhaps acceptable EPO patent IP method steps in-process in Europe (should Ocata/ACTC wish to rely on that patent estate to protect it as a future marketed product). Also the MA09 cell line wasn’t created in a xeno-free manner – this aspect may or may not have changed also in the new line (as I understand it isn’t mandated by the regulators to be so).
However, all told the evolving complexities of the FDA licensure process seems to have been the fundamental reason for the need to switch the cell line – MA09 evidentially didn’t have all the paperwork needed to pass the strict regulatory guidelines for ultimate market approval.
In Astellas recent presentation they mention the cell line change on Pg 41 > https://www.astellas.com/en/ir/library/pdf/RD_2_en_2016.pdf
A bit of speculation here – Ocata/ACTC may have been sold before this cell line change delay became an issue, as that would have killed the stock and perhaps the company along with it. Hard decision but perhaps necessary considering.
From what I understand the MA09 was the best performing cell line back when hence its use I believe (data on the line’s genetic profile I don’t recall seeing).
Good point on the mutations in hESC lines, and for that matter in iPSC lines also. I guess we can only live and learn that all things biological (whether “natural” or handled) aren’t really going to be perfect or exactly equal due to a host of issues and it’s best to be pragmatic and strive for multiple data points to drive potency testing and filters et al to then select the best of the bunch to work with, accepting certain % variations and passable inheritent mutations.
As a final comment I would say its absolutely necessary to ensure sufficient data across the same methodology from all available cell sources and their derived downstream clincial products in order to produce robust debate on any scientific conclusions imo – statistically relevent peer reviewed patient results being the litmus.
Cheers
THANK YOU for posting something about this, Paul! 🙂 This means more than I could ever say.
Now, as for the reformulating question… I have my own theories about why that would be happening and why there might have been an issue with the original source, and I don’t know if it would even be a good idea to share them… let’s just say that I think it was originally about religion and politics more than science. But I’m so glad to see this. A Japanese company, not being crippled by the insane nonsense garbage baggage that embryonic cell research carries in America, will get it done. There is hope! 🙂
If I read this correctly, the problem isn’t with the cell or how it’s performing or anything like that. The problem is with where the cells origination?
As of now, TX & FL are perhaps the best places to be for those not preferring med.tourism. And ESCs are old hat. Cord blood is the way to go.
See https://www.youtube.com/watch?v=NE_gR_TzIJo
Paul, I believe the program has been delayed so that they can reformulate their stem cell line from another embryonic source – there may have been some “problem” with their original source.
Irv
Thanks, Irv. A number of human ES cell lines including H9s have recently been found to have quite a number of mutations. I don’t recall what ES cells they were using at Ocata/ACT. I’d be interested to learn more.
Paul
MA-09, from what I recall.