3X recent stem cell & regenerative medicine good news

stem cells for spina bifida Arthur the dog
A picture of Arthur, a bulldog with spontaneous spina bifida who received a stem cell infusion, visiting the Knoepfler lab

In the past month or two there has been a steady stream of good news in the stem cell and regenerative medicine world. Here are three examples.

stem cell map ARM
Screenshot of ARM map of global stem cell & regenerative medicine related firms

Fate Therapeutics announced the first US IND for an induced pluripotent stem cell (IPSC)-related product. Its cleared product, FT500, is an off-the-shelf natural killer-based cancer immunotherapy.

I expect more to come in the next couple years so good news now and likely more to come. IPSC-based trials are picking up in Japan too with trials related to vision loss and Parkinson’s Disease led by stem cell power couple Masayo Takahashi and Jun Takahashi, as well as a new heart study. Moving forward in the next 5-10 years how much will be allogeneic versus autologous? It’s interesting to follow this given how ~10 years when IPS cells were first reported the emphasis was entirely autologous.

The Alliance for Regenerative medicine (ARM) gave a very up-beat state of the field recently. You can go through the presentation here. The “state of the field briefing” mentions 32% annual ARM growth over recent years and 906 regenerative medicine companies globally (see map from ARM).

stem cells for spina bifida Arthur the dog
A picture of Arthur, a bulldog with spontaneous spina bifida who received a stem cell infusion, visiting the Knoepfler lab.

Other highlights include a number of recent product approvals in different countries: Spark Therapeutics’ LUXTURNA, Avita’s RECELL, Gilead/Kite Pharma’s Yescarta, MiMedx‘s Amniofix & EpiFix, Novartis’ Kymriah, and TiGenix (Takeda)’s Alofisel. The ARM

update also mentions that there are more than 1,000 clinical trials in our area including 92 phase III trials, which is great.

Some cool, promising work right here at UC Davis on a novel stem cell approach for spina bifida got a boost with a big award of CIRM funding. I’ve seen my UCD Health System colleagues Diana Farmer and Aijun Wang present data on this early effort and even met one of the dogs who spontaneously have spina bifida, Arthur, who got a stem cell infusion.

The idea behind the investigational stem cell therapy for spina bifida is that the stem cells will grow to protect the spinal cord from injury. The intervention requires an in utero surgical step. It’s early days on this work, but it looks promising.

6 Comments


  1. Paul – seems some are not aware of the reported clinical safety & early efficacy results of the conducted trials to-date using differentiated cell therapy products derived from iPSC reprogramming technology.

    Eye – http://www.cdb.riken.jp/en/news/2017/researches/0404_10328.html
    GvHD – https://ash.confex.com/ash/2018/webprogram/Paper110432.html

    One study was in Japan using a personalized patient specific “Autologous” approach and the other was conducted in the UK using an off-the-shelf universal “Allogeneic” product.

    See also Jeanne Loring’s recent Guest Post on Auto & Allo approaches: https://ipscell.com/2019/01/autologous-or-allogeneic-a-stem-cell-question-guest-post-from-jeanne-loring/, in additon to a number of other threads here at The Niche on Pluripotent therapies in development.

    To my knowledge the other iPSC clinical applications underway have yet to report.

    There are a handful of iPSC based translational efforts in total to-date and another handful or two nearing the clinic in various prep stages.

    Cheers

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