Top 20 2017 stem cell predictions: score card at 1/2-way point

In December of each year I make a list of stem cell predictions for the coming year, and I made 20 such predictions for 2017 so I’m wondering how I’m doing so far on these now that it’s June. Below is my work-in-progress scorecard for these so far.

  1. Positive news from Asterias on trial for stem cell-based therapy for spinal cord injury. Status: Correct, trial looking encouraging.
  2. Upbeat news from ViaCyte on stem cell-based therapy trial for diabetes. Status: Correct, raising $10M and things looking promising.
  3. More positive news from the old Ocata now under Astellas umbrella on trial use of stem cell-derived RPE for Macular Degeneration. Status: Not Yet.
  4. Good news on the adult stem cell front on trials for one or more major diseases. At least one and probably more positive developments here. Status: Correct, lots of good news. The potential for stem cell generated blood is just one example.
  5. Fake news hits stem cell arena. Stem cell clinics use fake news. For instance, this might be a media mouthpiece for one or more stem cell clinics actively using fake news-like approaches to promote them. Status: Correct, unfortunately.
  6. More clarity on clinics: data. More academic publications on the practices and outcomes of stem cell clinics are published, bringing greater clarity to what is going on with actual data. Status: Not Yet.
  7. More lawsuits against stem cell clinics. There has been a lot of buzz on this behind the scenes already and cases popping up in 2016. This is going to grow in 2017. Status: Not Yet.
  8. Concrete clinic harms. We learn more about additional examples of patient who feel they’ve been harmed by American stem cell clinics including in particular alleged clinic-caused blindness. Status: Not Yet.
  9. Some other federal agency besides the FDA makes news on stem cells. This may not be until 2018, but we’ll see. Status: Not Yet.
  10. At least one FDA guidance is finalized. The FDA finalizes at least one of its four recent stem cell-related guidances, but probably not all four. Status: Not Yet.
  11. More than one warning letter. The FDA issues more than one warning letter to stem cell clinics in this year. Will it still be a drop in the bucket or some kind of decisive action? The FDA may have more difficulty taking action within the Trump context and much will depend on who is the new Commissioner.  Status: None Yet.
  12. Japan IPSC trial starts. Great news as at least one IPSC trial begins in Japan. Maybe two. Status: Correct.
  13. Cures yields regen med IND. The FDA takes at least one accelerated stem cell-related IND action traceable to the Cures Act related to a promising new stem cell/regenerative medicine therapy. Hopefully no direct to consumer businesses try to tap in. Status: Looking correct based on many RMATs granted.
  14. Athersys, Cytori, and Mesoblast have some ups & downs amongst them. Status: Correct.
  15. Prop 71 2.0. CIRM and/or Prop 71 supporters start more openly talking about a new round of CIRM funding. This may include mention of Trump as problematic for the stem cell field and the continuing need for California to take the lead. . Status: Correct. Will they pull the trigger though this year?
  16. Trump somewhat, but not entirely limits ES cell funding. The Trump administration probably does not outright ban federal funding of embryonic stem cell research, but there may be some effort to limit it in some way such as not supporting generation of new lines perhaps à la Bush.  Status: Not yet.
  17. Fetal tissue research restriction effort. The Trump administration and/or the GOP attempt to restrict human fetal tissue research.  Status: Not yet.
  18. CRISPR of human embryos is blocked or limited in some way in the U.S. (e.g. FDA is not permitted to review applications related to this area as was the case with the rider on spending bill for 2016).  Status: Not yet.
  19. Trump creates something like Bush’s President’s Council on Bioethics. It’s packed with conservatives including someone tied to the Witherspoon Institute. Deja vu all over again.  Status: Not yet.
  20. Florida acts on clinics. The state of Florida takes some action on stem cell clinics, which are out of control there. Things are a mess clinic wise here in California too, but I’m not so sure the state will do anything helpful to deal with it.  Status: Not yet.

Overall, I’m doing OK so far, but much is up in the air for the final 6 months of the year.

Upbeat prospects for some California clinical trial efforts from CIRM

Over at the California Stem Cell Report, David Jensen is reporting on some good news from CIRM for California on the stem cell clinical trial front.

Stem cell biotechs Asterias and Capricor have stem cell trials supported by 20+ million in CIRM funding each and have been hitting milestones. These trials are progressing and so far have good safety profiles. Asterias and CIRM have mentioned some possibly encouraging early hints at efficacy as well in its trial, and apparently there are hopeful hints from the Capricor trial too.

See the posts from CIRM here (a weekly summary kind of post that begins discussing Asterias) and here. For background, also see past posts I’ve done on both companies here and here in the archives, and see especially my interview with Asterias leadership from a few months back.

It’s early days for these trials and at these phases they are not really designed to look for efficacy so a conservative approach to discussing such trials is in order given the stage, but at this phase of the game for early clinical trials the news has been all one could hope for so far in both cases.

asterias-cells

Asterias cells

The Asterias and Capricor trials are for spinal cord injury and Duchenne muscular dystrophy, respectively. The latter trial utilizes the Capricor CAP-1002 product, which is a cool allogeneic cardiosphere technology made from donor human heart tissue. A beating cardiosphere from a different source (IPSCs) can be seen in the video above. Asterias’ trial employs their OPC product made from hESCs, which is also inherently allogeneic. The idea of potentially repairing the injured spine via stem cells is intriguing.

I’m hoping in the next month or so to do a broader update on the stem cell and regenerative medicine biotech arena. By way of disclosure, I do not have any financial stake in either company discussed here.

Early, but encouraging data from Asterias on stem cells for spinal cord injury

asteriasAs I posted recently, Asterias Biotherapeutics has had good early safety results so far in its historic stem cell trial for spinal cord injury and now the company presented some early, encouraging hints at efficacy.  However, a caveat here is that this is not an RCT (randomized controlled trial).

Ed Wirth, CMO of Asterias, presented the early data at the 55th Annual Scientific Meeting of the International Spinal Cord Society. You can take a look at the very interesting talk slides here. Asterias is a subsidiary of BioTime.

According to the press release (PR) from the company, while only 4 of 5 patients are 90 days or more after treatment, all patients have shown some improvement in motor function and a subset have a substantial improvement, meeting the target at this point. This trial is funded in part by CIRM so kudos to our state’s stem cell agency on this.

MIT Tech Review is on the story to for more background. I’ve also posted from Dr. Wirth’s PowerPoint the study design image below.asterias-sci-stem-cell-study-design

Bottom line. Overall, I think this development is encouraging. While still early in the clinical trial and drug testing process, the fact that there is a good safety profile and early indications of possible efficacy yield real hope. Still, it is important to be cautious in interpreting early data. We’ll all need to follow this trial and then look for future RCT results to clarify how promising this drug is overall in the long haul.

I’ve posted the key bullet points from the Asterias PR below the fold.

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The backstory on my opinion piece in SF Chronicle critical of CIRM lobbying

Today an opinion piece that I wrote about CIRM was published in the San Francisco Chronicle. The unusual element here is that the article is critical of CIRM. More specifically I raised concerns about a recent political trend at CIRM under its new President Randy Mills lobbying for dramatically weaker stem cell regulatory oversight. The Chronicle piece was first posted online on Thursday on the paper’s website. I encourage you to read it when you get a chance.

CIRM 2.0

For a decade I have been one of the most consistent voices advocating for CIRM, its future, and all the great things that it does so it wasn’t an easy decision to publicly criticize the agency at this point. I’m still a big backer of CIRM, which made this even harder.

For me the tipping point to action was Mills’ recent opinion piece on Fox News with former Republican Senator Bill Frist that I believe was anti-FDA in tone and utilized hype (e.g. words like “miracles”, “gift”, and “beautiful” medicine) to promote a deregulatory agenda. This extreme plan includes conditional approval of unproven stem cell therapies and elimination of the requirement for Phase III clinical trials in certain cases, which would be very risky to patients and the stem cell field.

Mills also recently gave a speech at a conservative group (the self-styled “Bipartisan” Policy Center or BPC at which Frist is a leader) that counterproductively characterized the FDA as the one big problem for the field. Note that BPC also wants to charge patients to be in clinical trials to get experimental therapies. Historically if anything changed hands in such situations it was patients getting something in return for being so brave to be in clinical trials.

As I pointed out in my opinion piece in the Chronicle, the stem cell status quo is not getting the job done, but this is only in part due to the FDA and the changes that BPC and CIRM are advocating for are too radical. These proposed changes also fit with the REGROW Act, a bill that would codify many of them. REGROW would force the FDA to conditionally approve stem cell therapies that frankly are just not ready for primetime, putting patients at great risk. Note that the big changes being lobbied for in FDA regulations would only apply to adult stem cells. I favor more careful changes at the FDA such as using Breakthrough designation for stem cells, something the FDA has not yet done. They should.

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FDA meeting on stem cell regs: mostly who’s who of anti-regulation forces

FDAThe FDA in the past year or so has issued draft guidances on regulation of stem cells that would, if finalized, make it crystal clear that what many American stem cell clinics are selling are unapproved drugs.

That would be a good thing for patients who are often being put at risk by many of the clinics. This is the reality no matter what some of the patient advocates who promote the clinics might say. Such finalized FDA regs that unambiguously state that, for example, fat stem cells are drugs, would be bad news for the clinics that are after patients’ money. Update: It is important to note that not all patients or patient advocacy groups are pro-clinic and anti-FDA. Every patient has a different story and I’ve met many that know the clinics aren’t some kind of Robin Hoods of stem cells.

It is perhaps not surprising then that at the planned upcoming public FDA meeting on stem cell regs in April, there’s going to be a crowd of anti-FDA, anti-regulation forces converging to speak. The agenda of this meeting, with a few notable exceptions, reads like a who’s who of the for-profit stem cell clinic world. I’ve pasted the agenda at the bottom of this post.

An interesting additional factor going on here is that there are some highly respected organizations also coming to the meeting that, reading the tea leaves, are also almost certainly going to advocate that the FDA be less comprehensive in its regulation of stem cells to accelerate translation. For instance, CIRM recently has made some statements about the FDA needing to speed up and maybe not be as strict.

CIRM probably realizes that there will be many folks at this FDA meeting who have no particular interest in participating in the standard FDA approval process at all. These stem cell clinic folks may cheer on statements from CIRM, if such are made, that come off as wanting a weaker FDA regulatory sphere for stem cells. I cannot imagine that CIRM would want that and I know that CIRM does not support these clinics.

As a result, there’s a vital need for CIRM to choose its approach to this meeting very carefully and cautiously. It may be a fine tightrope to walk. Accelerating evidence-based treatments based on stem cells is one thing, but weakening much-needed regulatory oversight that leads in the near future to clinics taking more money from more patients that they put at risk is quite another.

In short, CIRM’s agenda is very different than that of the clinics. Yet the stem cell clinic crowd could even be hoping to use CIRM’s words at this meeting for their own future publicity. They might even try to get their picture taken with CIRM staff to use on their websites. Historically the stem cell clinics have been very anti-CIRM, but that doesn’t mean that they won’t pounce on a PR opportunity if it arises.

The Alliance for Regenerative Medicine (ARM), which has done and continues to do amazing things, is also likely to push for a relatively more permissive stem cell regulatory construct at this meeting too. I hope that they are cautious not to align themselves with the dubious clinics. The membership of ARM consists of good citizens of the stem cell and regenerative medicine biotech community who work with the FDA and follow regs. This is a night and day difference from the stem cell clinics.

Regardless of how this meeting plays out, it will be fascinating to see and hear the views at this unprecedented gathering.

The agenda as of Feb. 10.

Draft Guidances Relating to the Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products Part 15 Public Hearing Draft Agenda April 13, 2016 FDA White Oak Campus 10903 New Hampshire Ave, Building 31, Room 1503, Great Room Silver Spring, Maryland 20903

8:00-8:15 AM: Presiding Officer Opening Remarks and Introduction of Panel Members 8:15-9:46: Session 1 • 8:15-8:18: Allosource • 8:19-8:22: Atlanta Medical Center • 8:23-8:26: AZ Pain Centers • 8:27-8:30: Birth Tissue Recovery, LLC • 8:31-8:34: California Stem Cell Treatment Center and Cell Surgical Network • 8:35-8:38: CAREStream America • 8:39-8:42: Celebration Stem Cell Center • 8:43-8:46: Centeno-Schultz Clinic • 8:47-8:50: Harvard Apparatus Regenerative Technology • 8:51-8:54: INCELL Corporation LLC • 8:55-8:58: Intellicell BioSciences • 8:59-9:02: Johnson & Johnson • 9:03-9:06: LifeLink Tissue Bank • 9:07-9:10: LifeNet Health • 9:11-9:14: Long Island Plastic Surgical Group • 9:15-9:18: MedCentris • 9:19-9:22: Info Health Global • 9:23-9:26: Millennium Medical Technologies • 9:27-9:30: MiMedx Group, Inc. • 9:31-9:34: Minnesota Medical & Rehabilitative Services • 9:35-9:38: Musculoskeletal Transplant Foundation • 9:39-9:42: National Spine & Pain Centers • 9:43-9:46: Oregon Regenerative Medicine 9:47-9:55: Questions from panel 9:55-10:25 Break 10:25-11:56: Session 2 • 10:25-10:28: Organogenesis Inc. • 10:29-10:32: RTI Surgical • 10:33-10:36: StemGenex, Inc. • 10:37-10:40: U.S. Stem Cell Inc. (FKA BIOHEART Inc.) • 10:41-10:44: Robert W. Alexander, M.D. • 10:45-10:48: Scott R. Graham, M.D • 10:49-10:52: Shay Bess, M.D. • 10:53-10:56: Julie Cerrone • 10:57-11:00: Ann A. Connolly • 11:01-11:04: Shawn Connolly • 11:05-11:08: Elio Coradin • 11:09-11:12: Terri Coutee • 11:13-11:16: Charles S. Cox, Jr., M.D. • 11:17-11:20: Pamela Fricke • 11:21-11:24: Brian Gates • 11:25-11:28: Jarvis P. Green • 11:29-11:32: Candace Hart • 11:33-11:36: David Jacobs, M.D. • 11:37-11:40: Carolyn M. Salafia, M.D. • 11:41-11:44: George U. Sauter • 11:45-11:48: John Klimkiewicz, M.D. • 11:49-11:52: Samantha Wilkinson • 11:53-11:56: Rebecca Baergen, M.D. 11:56-12:00 Questions from panel 12:00-1:00: Lunch: Ala Carte items will be available for purchase on site 1:00-1:35: Session 3 • 1:00-1:03: MaryAnn Chirba, J.D. • 1:04-1:07: Case Western Reserve University • 1:08-1:11: Indiana University School of Medicine • 1:12-1:15: Wake Forest University School of Medicine • 1:16-1:19: Navigant Consulting • 1:20-1:23: OrthoKinetic Technologies, LLC • 1:24-1:27: Parenteau BioConsultants • 1:28-1:31: Alston & Bird LLP • 1:32-1:35: Topher Stephenson, M.D. 1:36-2:52: Session 4 • 1:36-1:42: Alliance for Regenerative Medicine • 1:43-1:49: Alliance of Wound Care Stakeholders • 1:50-1:56: American Association of Tissue Banks • 1:57-2:03: Bipartisan Policy Center • 2:04-2:10: California Institute for Regenerative Medicine • 2:11-2:17: Coalition of Wound Care Manufacturers • 2:18-2:24: Curemonos • 2:25-2:31: Foundation for the Accreditation of Cellular Therapy • 2:32-2:38: International Society for Cellular Therapy • 2:39-2:45: National Center for Health Research • 2:46-2:52: Patients for Stem Cells 2:52-3:00: Questions from panel 3:00-3:30: Break 3:30-4:53: Session 5 • 3:30-3:36: The Amputee Coalition • 3:37-3:43: The Cord Blood Association • 3:44-3:50: The Cure Alliance • 3:51-3:57: Academy of Regenerative Practices • 3:58-4:04: Alliance for the Advancement of Cellular Therapies • 4:05-4:11: American Association of Neurological Surgeons and Congress of Neurological Surgeons • 4:12-4:18: American College of Surgeons • 4:19-4:25: American Society of Plastic Surgeons • 4:26-4:32: Biologic Orthopedic Society • 4:33-4:39: International Cellular Medicine Society • 4:40-4:46: International Federation for Adipose Therapeutics and Science • 4:47-4:53: The Plastic Surgery Foundation 4:53-5:00: Questions from panel 5:00-Close: Closing statement from Presiding Officer