Cell Therapy and Regenerative Medicine are widely misunderstood.
“As an industry, we in cell therapy haven’t done a good enough job telling our story,” began Lee Buckler, cell therapy and regenerative medicine consultant. “No wonder there are so many misconceptions about cell therapy!
Lee sat down with #MedDevice to set the record straight in this enlightening conversation.
Joe Hage: @CellTherapy, I’m sure you saw: Shire ($SHPGY) ditches Dermagraft for leg ulcers. For our readers’ benefit, $SHPGY is a UK-based pharma company + bought Advanced BioHealing (ABH) in June, in part for Dermagraft.
Lee Buckler: Well, it’s tough news to hear whenever a pivotal trial fails. Shire’s buying ABH was a big milestone for the industry. I think the headline was misleading. When I first read it, I thought Shire was ditching the product but it’s just the indication for Venous Leg Ulcers (VLU) that got “ditched.”
Dermagraft is a successful product for ABH (now Shire) for diabetic foot ulcers but they were looking to expand into Venous Leg Ulcers which I understand is a bigger indication.
Joe Hage: You consider leg ulcer indications “low-hanging fruit.” Why do you say that?
Lee Buckler: Wounds are a nice indication for regenerative medicine cell therapy for a number of reasons. One is because we can see what’s going on. Endpoints are clear for topical healing. The wound closes, improves (or not) and it’s obvious.
I think another reason is the mechanisms of wound healing are clearer than how to regenerate hearts or pancreatic function, etc.
So this is a setback for Shire and ABH but hopefully not a death knell. By “death knell” I mean not for either company or for the product … but I fear for Shire’s commitment to #regenmed
Oganogenesis is a competitor of ABH that has a regenerative medicine cell therapy in VLU so I’m keen to see how ABH failed. I haven’t seen any rationale yet for the failed Phase III result.
Anyway … ups and downs of therapeutic development especially in an emerging industry where every twitch feels like an earthquake.
Lee Buckler: Misconceptions about #celltherapy is one of my fav topics.
(1) I think when people hear “stem cells” they think embryonic.
Embryonic stem cells are now in clinical trial buy only recently so. Adult stem cells are much more advanced in clinical testing. Adult stem cells are stem cells from a living human. As early as fetal stem cells (controversial) but also include stem cells from cord blood, placenta, amniotic fluid which are more embryonic like than stem cells from an 80 year old man.
So, for example, Celgene has a very strong intellectual-property position in placental-derived “stem cells” and are in clinical testing.
(2) Even within the industry there is a misconception that we haven’t got cell therapy products to market yet.
Joe Hage: What would you consider “the most successful” stem cell therapy on the market today? Using what criteria?
Lee Buckler: Organogenesis Appligraf and Shire’s Dermagraft are so far the products which have been the most successful in terms of revenue.
Genzyme products, Epicel and Carticel have taught us all a lot as early stage cell therapy products but not very profitable.
(3) Cell therapy = stem cells.
True but not exclusively so. There are lots of #celltherapy products which do not involve stem cells -embryonic or adult.
There has yet to be a product approved using stem cells although Osiris‘ Prochymal has come close if you believe MSCs (Mesenchymal stem cells) are “stem cells.”
Dendreon‘s Provenge, for example, is a cell therapy that is based on an immune system cell called a dendritic cell.
(4) Pharma is our enemy and is not willing to do cell therapy.
Very untrue. In fact, it’s harder to find a pharma that’s NOT involved in some way …
Pharma is involved, admittedly in small and early-stage ways appropriate with our maturity. Pfizer, GSK, Roche, Sanofi, Takeda, Shire, etc all involved in cell therapy in some way or another.
Darshan Kulkarni: (A previous #MedDevice guest, see @FDALawyers on Regulatory Crackdowns) Is the lack of blockbuster therapies due to the “personalized nature” of cell therapies? Or are there other reasons?
Lee Buckler: @FDALawyers Good question. I don’t think of #celltherapy as inherently or necessarily “personalized medicine.”
Allogeneic cell therapy (a cell-based product created from a donor) is not personalized at all. It’s “off-the-shelf.” Even autologous #celltherapy (where it is made from the person’s own material) is not necessarily “personalized medicine.”
Personalized medicine, in my opinion, is about the ability to design a med for patient sub-population based on genetic markers we know.
We have found even autologous cell therapy has no better success rate than drugs often because there are patient sub-populations.
Opexa Therapeutics is an example of a #celltherapy (not stem cell) company doing a personalized medicine approach. Opexa Therapeutics has the ability to create different versions of its therapy based on diagnostic results.
In conclusion, @FDAlawyers, I think the lack of blockbuster to-date is nothing more than our nascent state. We’re very early in this game. If you track our progress against the maturation of antibodies for example, we’re on track….
Lee Buckler: @FDALawyers, I totally agree “blockbuster” is so yesterday. Pharma is onto niche markets now. See Sanofi and Genzyme.
Joe Hage: Offline you told me: “All Stem Cell Tourism (where Americans go abroad, outside the FDA’s reach of approvals in order to get treatments approved elsewhere) is a plague which should be shut down.” Please elaborate.
Lee Buckler: Yes!
(5) “All stem cell tourism is bad” is another great misconception, in my opinion.
@medicalmarcom This issue of stem cell tourism is a big one of contention for me. No doubt there are a lot of charlatans, but to say all stem cell tourism is bad is the same as saying all medical tourism is bad. It doesn’t compute.
I continue to believe there is a legit way to offer patients cell therapies in other countries when the therapies are not yet approved by the FDA.
Take Cytori Therapeutics, for example. CE ‘approved’ for their autologous cell therapy device in Europe. Not approved in USA.
[Note to readers: “Would it be so wrong…?” was “retweeted” a number of times by #MedDevice chat followers.]
Darshan Kulkarni: @celltherapy I agree. Conversely, there may be a reason the FDA hasn’t approved. It’s tough as a pt. to tell the difference. The key is doctor supervision (which is sometimes unfortunately lacking when you go abroad.)
Lee Buckler: @FDALawyers I agree. I come down on the side of informed consent and enabling patients to decide.
Joe Hage: There are opportunities to sell “picks and shovels” to cell therapy companies on their quest for the Holy Grail.
Lee Buckler: I think there a lot of very exciting opps for marketing medical devices in the space … including scaffolds/matrices, bioprocessing, and point-of-care.
Cytori, Tissue Genesis,Thermogenesis are examples of point-of-care devices/systems companies.
There are a plethora of scaffolds, matrices, stents, etc. being explored as in vivo delivery mechanisms. Lots of opportunities, too, in growth media/culture, reagents, antibodies, etc.
Depending how far afield you want to go there are great opportunities for unique storage containers, storage media, etc.
Joe Hage: Do you think #MedDevice / #Cell Therapy synergies are there to the point where you expect to see mergers?
Lee Buckler: So far no medical device M&A in the space that I can think of but they are invested and collaborating. Medtronic comes to mind.
David E. Albert, M.D: (@DrDave01) @MedicalMarcom Not yet as personalized diagnostics and therapeutics are slow to develop.
Lee Buckler: There is a interesting bidding war for KCI right now and I think the whole orthobiologics space is really going to heat up.
I think there a lot of interesting ancillary product and service plays succeeding in the sector…
• Fisher Bioservices (cold chain logistics)…
• Lonza, Progenitor Cell Therapy, and PharmaCells (contract manufacturers) …
• BioLife Solutions, Celsense, Thermo Scientific (reagent companies) ….
I saw a great interview of the CEO of Harvard Apparatus recently. They are betting heavily on regenerative medicine cell therapy.
Joe Hage: @CellTherapy, at the risk of being overly colloquial, this interview was awesomely awesome. Thanks so much for joining us. We’ll have to have you back!
Lee Buckler: @medicalmarcom I really enjoyed chat. I look forward to seeing future chat. Great idea. Great hosting. Great guy. Thanks! I’d love to come back anytime for a #MedDevice chat!