Launch of Jun Takahashi IPS Cell Trial For Parkinson’s Disease

Jun Takahashi, stem cells parkinson's

Jun Takahashi, stem cells parkinson'sA much-anticipated induced pluripotent stem (IPS) cell trial for Parkinson’s Disease reportedly will soon launch led by Professor Jun Takahashi.

The news broke on Yahoo Japan, which included an unusual number of appropriately sober statements regarding the trial, even though it is an exciting trial as well, compared to most media stories on stem cells.

Parkinson’s Disease is a result of a loss of dopaminergic neurons in the brain so it is a particularly attractive target disease for stem cells, which can be readily used to produce dopamine neurons for transplant.

Here are some key details in the article on the Jun Takahashi IPS cell-based trial. It is anticipated that there will be some insurance coverage for this trial for patients. The target patients generally will be those who do not have the most severe disease. This is an interesting choice as it potentially allows for patients earlier in the course of the disease to get the cells, but could have somewhat higher risks. Generally, early phase trials often treat the sickest patients to minimize risks.

Jun Takahashi, stem cells parkinson's
Graphic from Yahoo Japan article

Here’s an example of the more sober aspect of the article, as translated by Chrome so forgive mistakes:

“Side effects must also be carefully judged. The nerve made from iPS cells is transplanted to a place different from the nigra that originally had dopamine nerve. Dopamine may overdone and cause side effects such as involuntary movements. Even though we confirmed the safety by monkey experiments, attention is required.
Even if this treatment method using iPS cells is established, it is not to become “dream medicine” that can return to a completely healthy state. While possessing the possibility of opening a new way of treatment, it is necessary to examine the significance properly together with other treatments.”

Overall, I think there’s real hope for stem cells for Parkinson’s Disease given the work on a number of fronts by a variety of international researchers conducting rigorous research.

10 Comments


  1. I am so glad to see that this trial will go forward. This is exactly the kind of thing we need if we hope to offer desperate people possibilities other than stem cell clinics. As long as trials like this are not happening, delayed and delayed and delayed, canceled, back to the drawing board (looking at you, Astellas), etc etc etc ad nauseum, people will take huge risks with the clinics. And yes, since I seem to need to add this EVERY time, I had an incurable disease several years ago and was just about desperate enough to go to a stem cell clinic. The only available treatment is keeping it in remission. But I have to take it day by day, and I 100% get it when it comes to the desperation that drives people to try something, anything, rather than give up. We’ve got to have at least the realistic hope that approved treatments will exist at some point down the road, or patients will continue making that choice, and it doesn’t make sense to blame them for it.


    • You concisely describe how I feel about this. I understand and agree we don’t want to sabotage progress with unnecessary lawsuits, or take repeated actions that cause the general population to take an opposing stance. The clinics making a profit on disease they have no evidence of having the ability to cure are despicable and must be shut down. Those in people running those clinics should be put on trial and jailed for such actions. There must be laws that reasonably regulate treatments to ensure a certain level of safety.

      The general population really has little idea how far this science has come. Nor do they understand the potential it quite likely will have in the not so far away future. Having said that, I know from personal experience as you do, when faced with the possibility of having a life threatening disease, you will do anything in your power and take any risk to avoid the consequences of that disease.There must be balance between safety and risk. Both are necessary. Lawmakers have to take seriously the fact that people will go so far as break a law if not doing so means their own death. Those lawmakers must remember they serve the public. I don’t believe the majority public would argue against a treatment that might help treat a disease if the person with the disease has no other options left. But notice the qualifier being that the treatment shows at least some promise to help.


    • It seems some people just can’t help themselves on this blog and take every available opportunity and post by Dr. Knoepfler to verbally stick it to what they refer to as “Stem Cell Clinics”. To me this misnomer of a term seems for the most part brought to you by those who have had absolutely no real patient experience with what I refer to as Regenerative Medicine Clinics. And I do have real world patient experience with the Regenerative Medicine Clinic that I have frequented since about October of 2014. (About 8 times total) Two of the Health Care Providers at my Regenerative Clinic (a PA and an MD) have both told me that stem cells derived from bone marrow and/or adipose tissue comprise only a minority of their procedures which include, among others, the following….. Physical Therapy, Cortisone and Synvisc Injections, Prolotherapy, Platelet Rich Plasma and Ultrasonic Diagnosis. This clinic limits itself to musculo-skeletal conditions only and are in no hurry (and neither am I) to inject my knees with my own adipose or bone marrow derived stem cells. It’s nice to know that I have that option if I so choose.
      That some advances apparently are being made with cells other than just the multi-potent variety….all I can say to that is “Congrats! I wish you well! Keep going and bring it to the clinic!” However, I will not hold my breath for (AT LEAST) another 5-10 years (PROBABLY LONGER) on the mere HOPE of Induced Pluripotency while I’ve already enjoyed success with the regenerative medicine clinic I’ve been a patient of. This particular trial of Dr. Takahashi will probably rely on immunosuppresion drugs according to Jeanne Loring. I have yet to take a single immunosuppresive pharmaceutical during my 8 treatments at the regenerative medicine clinic that I frequent.
      The patients I’ve met at this regenerative clinic do not strike me in any way as “desperate” either. And neither am I. Furthermore, those running the clinic while charging quite a bit more than the mainstream standard of care (which also profits off of disease) don’t belong in jail either, in my opinion. For those who are that frightened of regenerative clinics, I think the best thing to do is not become a patient there. But until you do, your information on anything other than experience seems fairly incomplete to me. I don’t need your protection and I did not ask for it.
      That I opted for the least invasive regenerative procedure first rather than surgery on my arthritic knees in 2014 is, I think, the right option. And, nothing that I’ve read on this blog has ever convinced me that my decision to step out of the mainstream of medicine was the incorrect choice that I exercised back then. To this day I believe that my patient experiences in regenerative medicine supersede an awful lot of what on this blog appears to me as scientific and medical politics; a politics that are driven by a speculation that is in my opinion, more increasingly starting to resemble a mirage.


      • @Douglas,
        Thanks for your comment. One mission of this blog is to be a forum for diverse opinions and discussions that don’t really take place anywhere else. I value the fact that it can be a platform for many patients to discuss their experiences and opinions with each other, with scientists, physicians, funders, etc.
        Paul


  2. This is great news. I’m very pleased that Jun Takahashi is going to start recruiting patients for his clinical trial using iPSC-derived dopamine neurons. He is an excellent, careful scientist, and his trial will be done well.

    Jun is a member of GForce-PD, and we shared our plans for clinical trials with him and two other groups last year in Kyoto.

    While we are also using iPSCs to make dopamine neurons for replacement therapy, there are some differences between the Japanese approach and ours (Summit for Stem Cell) – one difference is while Jun’s study will use iPSCs that are not matched to the patients, and will need to give the patients immunosuppressive drugs, we are using dopamine neurons made from iPSCs from each patient, so that the patients will not need to receive immunosuppression.

    Because of the broader approach we are taking, we’ve focused our efforts on developing robust methods for making reproducible preparations of dopamine neurons from every patient-derived iPSC line, and have designed genomic quality control methods to ensure the safety and efficacy of every transplant. We will be seeking approval from the FDA, whose regulations are not identical to the Japanese regulatory authorities.

    I’m honored to be working in parallel with Jun and with the director of his institute, Shinya Yamanaka.


  3. This is an important milestone in the advancement of Pluripotent derived stemcells – the 4th iPSC trial indication area to-date, 3 of which are appropriately being conducted in Japan, as a prime mover in this technology discipline due to Yamanaka’s groundbreaking work and Japan’s united Industrial$ & Governmental$/Regulatory support for the RegenMed sector.

    The 2nd in the sequence of iPSC trials is in the clinic in Australia & the UK in GvHD (Cynata).

    The order playout goes: EYE (Wet AMD – Japan); IMMUNE (GvHD); BONE (FOP) and now BRAIN (Parkinson’s).

    Congratulations to Jun Takahashi who is the leading scientist on the Pakinson’s trial and best of luck with this important effort to translate his work to the bedside for those in need. Notwithstanding the cautious tone, this is a highly positive event in the field as is indicative of the progress pluripotent science generally has been making in its early clinical translational work.

    This area represents the Hope for affordable off-the-shelf patient solutions using “high” science, as Masayo Takahashi likes to call it…

    Let’s trust additional US, Canadian and mainland European players/VCs see the paradigm shifting potential of standardized iPSC derived Allo cell lines and the IP in therapeutically designed programs to add additional momentum behind sector building Phase I translational efforts, of which there are a number already announced around new and existing ventures and also in the wings at institutions being prepped in CARDIO, BLOOD, BRAIN, IMMUNE and CANCER.

    In order for a technology footing in the West to take hold there needs to be the respective regulatory body encouragement of submissions, easing pre-clinical burden and approval of well researched clinical Phase ready iPSC trials (especially when they’re based on pre-existing Phase 1 clinical data). Where are the iPSC program INDs in the US, Canada and mainland Europe??

    What’s holding up the clinical translation in the West? Is there an IP licensure issue curtailing adoption? Is it really Sector Centric, Industry Building, Collaborative and Community with Affordable Off-the-Shelf Patient/Payer Focused at the Core without Seeding through Therapeutic Platform Access?

    Jeanne I know it’s possible to clinically trial without IP (legally) and to push the issue off until commercial launch later but in many cases the approval screening bar has been way too high to-date and the cost implications of the existing clinical license of non-program specific reprogramming tech is an inhibitor to growth and success imo.

    Cheers


  4. Hi Michael:
    Thanks for tracking all of this progress. In response to your comment to me, we’re not worried about IP. Japan is not trying to have a monopoly on iPSCs – they want the technology to be widely used and licensed non-exclusively. They’ll do better financially by licensing rather than blocking (recall PCR…). We have our own IP – methods as well as PluriTest and its spinoffs.
    Jeanne


  5. Hi Jeanne

    Yes. Your Know How, Methods, Tools and Compositions are the valuable aspects of your Product Innovation process and I’m aware from our discussions that you’re well positioned and will tie up any reprogramming layer needed as and when.

    My question/comment was more to your point that Japan’s interest is in circumventing any anti-commons issues and proactively developing the iPSC tool-set authorizations and freedom to operate as a sector strategy for broad market adoption, not just for Research (which is great to see well established now) but equally and more publicly for Therapeutic Product Development.

    Favorable commercial program seeding terms would help stimulate that process, of course. As would perhaps a published criterion for venture technology use?

    Not sure of the non-exclusive therapeutic product dev. license stats as a % of the overall versus research only licenses granted but there were very few commercial licenses issued (single digits) from iPSC Academia Japan, Inc. last I knew. Hopefully that has changed recently, with more programs in the West being authorized (current overall licensing picture from Japan: http://ips-cell.net/e/license/licensees.html). Excellent to see Platelet BioGenesis were recently awarded a license.

    As we know the world of science technology is constantly evolving. Your experience with WARF speaks to the need for product innovation led platform usage on as wide an adoption level as possible – such as what’s happening with gene-editing. Leap-frogging is great for technology momentum, but we’ve seen it leave productization efforts behind in its wake as the ongoing cycles stimulate further research & discovery, which by the nature of the effort uncover additional questions and issues.

    Is iPSC Science ready for more broad-based prime-time warm ups? Is it prudent now to take further clinical steps forward Internationally, in the US, Canada and mainland Europe? Does the safety data warrant it? Should more therapeutic grade iPSC cell lines be made available & more commercial licenses be granted? Questions of this nature come to mind…

    Progress made in early stage trials seem to have paved the way for such a push forward on a number of fronts. It would be a huge positive for stakeholders to see the expansion of clinical program use and more varied pluripotent derived targets announced (incl. more using gene editing).

    A good indication of the state of play in the area would be when international clinical trial INDs are submitted, considering iPSCs by design took the leadership mantle some years ago and investigation using cell lines had to switch.

    Let’s hope the sector affirming patient centric news flow continues.

    Congrats btw with your spin-out! Great development.

    Cheers


  6. It’s a great progress. Really thanks for all the researchers and scientists devoted themselves to parkinson. I wish more and more young people can join in this research which need more scientific reexperiments. If someone is in financial jam, I heard there is a scholarship program provided by a company. Wish it can help you.


  7. Sincerely hope the neurons derived from iPS cells functionally integrate with the recipient cells. This is most crucial since in the heart the iPS derived cardiac cells do not integrate with the recipient cardiac tissue. Hope we have enough data to show this. Secondly do we transplant mature neurons or neuronal progenitors?

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