Tag Archives: regenerative medicine

First Patient Enrolled in Ocata Therapeutics’ Phase 2 Study for Dry AMD Results from First Cohort Expected in the Second Quarter of 2016

Here’s the press release.
(STARES at today’s news)

*^()&)*(&)*(&)*( I DON’T EVEN.
(RUNS around yelling incoherently, but happily.)

INFINITE YAY.
THIS… this is what we’ve been waiting for. Ocata is officially now going to start Phase 2 of the AMD study.

There’s so much to be said here… it’ll have to get done later on… but for now… HAPPY. Just plain… happy. 🙂

UC-Davis Stem Cell Ethics Conference, Part 4

So here it is, y’all– the fourth installment! This is the LAST “notes from raw slides” section, SO please keep hanging in there… because this is important. Reading these notes may not the most entertaining activity on earth; believe me, I know. (I had to transcribe them from horrible blurry pics!) But there’s a lot to be gleaned here. If you’ve ever wondered how a drug goes from concept to market, this is one you want to read. (Just go down to Slide 9.)

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THE NEXT SPEAKER AT THE UC-DAVIS CONFERENCE…
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The UC-Davis Stem Cell Ethics Conference, The Whole Story: Part Two

At the end of part one, Tim Caulfield, keynote speaker at the conference, was addressing the problem of illegal stem cell clinics worldwide…

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Tim Caulfield looked up. “So what’s the harm?” he asked.

I wasn’t sure if anyone in the audience was supposed to answer the question, but I did not have a good feeling about whatever was coming next.
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The UC-Davis Stem Cell Ethics Conference, Part One

So… I’ve been promising to post material from the Feb 12th conference for a while, and here it FINALLY is! I’ve been moving to a new house… and I didn’t have net access for weeks and weeks… and the dog ate my homework… and, okay; enough already. I think I’ve been trying to fit these notes into literary form, because they will become an important aspect of the book. But it’s more important to get them out there for people to read. So… keeping in mind that these are fairly rough… here’s Part One!

The scene opens on February 11, at Tim Caulfield’s introductory talk…
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The State of Alzheimer’s Research

So I just finished Dancing With a Stranger, by Meryl Comer, and… it’s not exactly the feel-good book of the year. She is a caregiver for BOTH her husband AND her mother, and both have Alzheimer’s. Oh, yeah– and she has both of the genes associated with the disease. Just the fact that some people are able to keep going in the face of unbearable problems is hard to believe.

I’ve been looking into the current state of stem cell based AD trials, and here’s what I’ve found out. As of December 2014, there’s not a lot going on at the clinical level right now. There’s one trial in South Korea using cord blood-derived cells, but even though they completed the preliminary outpoint in 2012, there does not seem to have been any information released on what the outcome was, which isn’t very encouraging to me. Dr. Lanza’s article about preliminary interim results in Ocata’s RPE trials for AMD was released months into the study. So, for it to be fully two years after the completion of Phase I, and still nothing… not that great. Another study in China is now recruiting, but I’m not sure how much better that one looks.

There are a few meds in development that look promising… but the problem with drug trials is that there’s a long history of meds looking great for AD until they’re actually released, and then they turn out to not accomplish much of anything over the long term. I’ve seen a couple of drugs really help people in the early stages, but only for about one year (Aricept and Namenda are good examples.)

The most promising thing I’ve seen, IMHO, is Neuralstem’s animal studies on Alzheimer’s. They released results in October, and the conclusion is that their cells “rescued spatial learning and memory deficits in mice with an animal model of Alzheimer’s disease.” The problem obviously is that we’re only talking about mice at this point, and there is, unfortunately, a history of stem cell treatments looking wonderful in mice and then not translating to humans. Still, if I had more money, I would definitely invest in this company!

So that’s pretty much the AD story for now!

Working in hospice , Part One

So today was the wrap party for NaNo (National Novel Writing Month,) and I read from the stem cell book I worked on all through November. (Yep, it’s the planned book on this site, And the Blind Shall See: The Promise of Stem Cell Cures.) It came from the section on working in hospice with Alzheimer’s patients. People were really touched (and I’m not going to try to claim that it was from the amazing writing quality!) No, it’s because everyone can relate to the desperate need for cures for degenerative diseases. And yet it’s basically a situation where even the most educated people never know almost anything about what’s really going on with truly effective stem cell research (like Ocata’s.) If you’ve ever lived with the situation of caring for a relative who has a degenerative disease like Alzheimer’s, you know how utterly draining it is, how much it takes out of you. I saw it every single day when I worked hospice. And it isn’t just older people, believe me. There was one woman I knew who died of MS when she was only thirty-six years old. I’m going to put up a series of posts on working with patients who had degenerative diseases, and there’s quite a range of those. Alzheimer’s, other types of dementia, MS, COPD, severe arthritis, and on and on and on. These might not be the most cheerful stories in the world– but we’re working on a happy ending through regenerative medicine! 🙂

More thoughts about Japanese stem cell trials right now…

I was working on the section of my book that deals with iPSC stem cell trials in Japan– a little bit about the history, Yamanaka’s discovery of iPSC cells in 2006, and how it all got to where we are now. There’s a lot of material about where we actually are at this point, too. Reading over what’s there so far, it’s kind of hard to avoid the conclusion that I’m sounding pretty negative about the Japanese research. It’s not that I want to take a negative attitude about it– I’d much rather see that research succeed– but they’re moving so fast, and I just can’t get on the rah-rah bandwagon. Here’s a good example of what I mean.

Over the past couple of years, Paul Knoepfler has posted a lot of concerns and critical thoughts about the process of getting iPS cells to clinical trials, and he’s used some pretty strong language. For example, here’s part of what he posted in October 2012:

Are iPS cells being rushed to the clinic or has their time come?
Just the title alone kind of tells us what his thoughts might be, but there’s much more. Here’s a very relevant quote:

The iPS cell field has run fast and furious over the past 6 years reaching a big milestone surprisingly quickly on Monday with Shinya Yamanaka winning the Nobel Prize.

But is the field going too fast?

In August I argued that iPS cells are not quite ready for primetime (i.e. clinical trial studies).

Now in October I mostly feel the same way.

But reportedly, some iPS cell researchers are working to start clinical studies wherein iPS cells would be transplanted into human patients as early as next year in Japan.

This is both exciting, but also potentially very risky if not flirting with disaster.

It would be tragic if the excitement and creativity exploding from iPS cells became diminished in the future by a rush to the clinic that harmed patients.

Pretty strong words. In fact, let’s look back at the August 2012 blog post that Paul referenced:

iPS cells not quite ready for primetime?

I like and support the idea of iPS cell banks (as discussed by Yamanaka). However, I do not believe the field has advanced far to support clinical use of iPS cells in the near future.

I realize that clinical use may nonetheless be on the horizon just a few years out in certain countries such as Japan, but in my opinion iPS cells are “not ready for primetime”, meaning not ready for clinical use. Not yet.

I also realize that some people in the stem cell field are going to be mad at me for saying this, but I believe it is true. People don’t read this blog to get sugar-coated, politically correct statements, right?

Are we ready to start using iPS cells in patients in the near future? Say in just a couple years?

Are they ready for primetime?

I think the answer is clearly “no”.

Guess what? This blog post was written in August 2012; Takahashi and company performed the first human transplant in September 2014. This means that the EXACT situation that Paul warned about, the EXACT one that he said absolutely should not happen, is EXACTLY the one that happened. (!!!) (feel free to add more exclamation points.)

But that’s far from all he had to say. Now, let’s fast forward to April 2013, only a year and a half ago:

As iPS cell studies in humans approach, accessible relevant pre-clinical data remains minimal

This dealt with Japanese scientist Takahashi and her attempts to get the first-ever human trials with iPSC’s off the ground (again, this actually did happen in September 2014.) Here’s what he had to say then, only a little over a year before that happened:

In her ISSCR seminar given 10 months ago, Takahashi presented some safety data from mice on the RPEs, but not from larger animals such as monkeys. To be clear, larger animal studies are not also not required, but this is an important distinction since larger animals are sometimes better models for humans and also because there were some anecdotal reports that said she had in fact presented larger animal pre-clinical safety data at the ISSCR meeting.

My understanding from Geron’s and ACT’s experience at the FDA here in the US is that the short-term nature of this iPS cell safety data along with very low animal numbers and lack of a clinically-relevant transplantation paradigm would be far from satisfying regulators here in the US that human studies should begin.

Unless there are a lot more, longer-term studies (e.g. 1 year or even longer) done on many more animals (e.g. 100s) yielding equally encouraging safety results specifically on transplants in the retina (not just sub-Q teratoma assays), I am deeply concerned as to whether the field is really ready to make the jump to transplanting iPS cell-based therapies into people any time soon.

Those long-term studies simply did not happen. There was never another large animal used in a study besides that one monkey (I have a copy of the study; I might post it later.) No additional long-term studies, certainly no hundreds of animals. I’m not sure about the transplant safety results, but I don’t really see how those could have been done when the long-term, additional studies themselves weren’t done. One thing we do know is that preclinical research wasn’t published until after this article (I want to say July 2013, but I would have to look this up.) Dr. Takahashi was given the go-ahead to start recruiting for the clinical trials in August 2013.

So what happened to the concern? Well, we don’t really know. It hasn’t been brought up in relation to the incredible rush to the clinic that the Takahashi study has turned out to be.

Here’s what I posted on Paul Knoepfler’s blog on November 18:

Paul, do you really, SERIOUSLY feel that only a year and a half later, anyone is really ready to transplant iPS cells into human beings? Isn’t a year and a half later “anytime soon?” Between your articles in 2012/2013 and today, we saw the STAP disaster take place at Riken, the SAME place that is responsible for the Takahashi trials. Some of the people involved are the same (such as Wakayama) Do you feel comfortable with the kind of self-policing that Riken has done since then? Do you think that all of the questions and concerns you had only a year and a half ago have been well addressed? Don’t you think there’s at least a real chance that these scientists are rushing terrifyingly and dangerously fast into human trials?

I just feel like the problem with the situation surrounding these human trials in Japan isn’t exactly rocket science. When pared down to its essentials, it’s pretty simple.

Ultimately, it comes down to this:
1.) Paul K. criticized the imminent rush to the clinic of iPSC’s in 2012 and 2013 in no uncertain terms
2.) He specifically said exactly two years ago that he didn’t think these cells would be ready for that IN TWO YEARS, which would be, well, today
3.) He listed some steps that he thought needed to be taken in order to be even a bit more confident that iPSC’s were ready for clinical human trials, and those steps weren’t taken,
4.) He brought up a ton of serious concerns, and now… can he SERIOUSLY be jumping on the rah-rah train? I don’t know if it’s exactly that bad, but he suddenly seems to have dropped all genuine criticism.

I really, really love Paul K’s blog, I think he’s an amazing scientist, he’s done so much good work, and I have so much respect for him… but when it comes to this issue, I think that he should return to his earlier and much more critical stance. Stem cell research is literally a matter of life and death. It’s worth being critical about.

Stem Cells: The Most Important Medical Issue of Our Time.

Stem cells. The hope, the hype; the successes, the failures; the frauds, the triumphs, and the miracles in the making. It’s all going to be here! Oh, okay; ALL of the information in the world about stem cells is probably NOT going to be found here. 😉 But if I can find it out… will be. There will be much more content here, including the story of just why and how I  became so passionate about the entire issue of stem cells and regenerative medicine.  I can honestly say that some of what you’ll read here is unique and cannot be found anywhere else the world. So just keep watching this space– some incredible things are coming up!