Bayer reports positive early data for Parkinson’s cell therapy

[Farooqji]

The therapy, developed by Bayer’s Bluerock Therapeutics subsidiary, appeared safe and well-tolerated in a 12-person clinical trial.

biopharmadive.com/news/baye...

[park_bear]

Good results:

bluerocktx.com/bluerocks-ph...

"the high dose cohort showed an improvement of 2.16 hours in time spent in the “ON” state without troubling dyskinesia compared with baseline after one year. Time spent in the “OFF” state showed a corresponding decrease of 1.91 hours after one year. Participants in the low dose cohort showed an improvement of 0.72 hours in the “ON” state without troubling dyskinesia time compared with baseline and a corresponding decrease of 0.75 hours in “OFF” state time.

In the high dose cohort, a one-year measurement of the effects of bemdaneprocel using MDS-UPDRS Part III measured in the “OFF”-medication state, showed a reduction of 13.0 points compared with baseline. The low dose cohort showed a reduction of 7.6 points."

[curlscurls in reply to park_bear]

What do you think of those results? Is 13 pts a lot on the UPDRS?

[Farooqji in reply to curlscurls]

UPDRS reduction of 3.3 points or more is considered as clinically significant.

[curlscurls in reply to Farooqji]

Thanks. How does it compare to feeling better? I don't have any sense of the scale compared to symptoms.

[Farooqji in reply to curlscurls]

UPDRS score calculator assesses signs and symptoms of Parkinson's disease as part of diagnosis and progression monitoring

The Unified Parkinson's Disease Rating Scale (UPDRS) is a rating tool that includes series of ratings for typical Parkinson’s symptoms which result in the movement hindrances of this disease.

The UPDRS consist of four sections:

Mentation, Behavior And Mood

Activities Of Daily Living (for both “on” and “off”)

Motor Examination

Complications Of Therapy (In the past week)

mdapp.co/unified-parkinson-...

[curlscurls in reply to Farooqji]

Thanks. The link goes into it a little bit. Thanks that's the first I've seen!

I was very surprised when I started searching that the range of results and more specifics of what it means, wasn't available in results that came up.

[curlscurls in reply to curlscurls]

So since it doesn't specify which type of changes, it'd be 13 out of 199, so 6.5 % change in symptoms. Question would be whether those changes continue to increase.

[Farooqji in reply to curlscurls]

Although the progression varies from person to person, the overall score of someone who is 5 ~ 6 years into the disease is around 20 ~ 25. UPDRS scores increased in a linear fashion over 5 years in patients. The reduction of score equal to 13 is quite significant. The improvement (change) in the Bluerock's trial will be clear with the passage of time (whether it further improves or remains constant )

[curlscurls in reply to Farooqji]

That puts it in much better context. And sure 13 out of 20 to 25 is a huge change. It's still be a huge change at a later point too.

Well that's exciting. Though I agree that I'd much rather see how the stem cells from skin cells testing is doing. Taking immuno suppressant drugs is a big deal.

Thanks for explaining!

[Obiora]

Results look extremely promising

[Farooqji in reply to Obiora]

Yes its so. But I am surprised that the Kyoto University trial is still under the carpet which was started in 2018. Japanese technology uses patient's own skin cells therefore chances of rejection by the immune system are very low. Bluerock uses Human Embryonic Stem Cell-Derived Midbrain Dopamine Neuron Cell Therapy (MSK-DA01) which necessitates the use of medicines for suppressing the immune system

[jeffreyn in reply to Farooqji]

"Japanese technology uses patient's own skin cells"

The Kyoto uni trial is using allogenic (donor-derived) iPSCs:

healthunlocked.com/cure-par...

The Aspen trial is using autologous iPSCs:

healthunlocked.com/cure-par...

[Farooqji in reply to jeffreyn]

thanks for clarifying. I'll check again

[MarionP in reply to Farooqji]

This about Bluerock cell selection neccesitating having to use an immune suppressor because of host rejection of cells IN YOUR BRAIN??? Cell rejection in your brain. Not exactly the same as a kidney.

[WinnieThePoo in reply to MarionP]

There are some advantages. Cost and scalability is one. Screening for mutations and abnormalities is another. And building neurons from a different gene pool to the one which went wrong is another. Roger Barker of Cambridge did u very good presentation on the subject.This video was contemporary with my diagnosis. One of the first things my early research turned up. How lucky to be in that group of patients. By 2023 they would be cured. How many have had this surgery?

kpbs.org/news/evening-editi...

"if all goes well during the first trial in 2019..."

Edit: Jeanne Lorings video was from 2018. She is about to graft her first patient. Maybe. It's August 2023

[jeffreyn]

Study Record:

classic.clinicaltrials.gov/...

[Gallowglass]

I’m skeptical. Bayer = bad IMO. Will wait ten years to see the long term effects

[Farooqji in reply to Gallowglass]

We should not speculate anything at this stage. Neither we should attach high hopes nor should we be pessimistic

[LeharLover62 in reply to Gallowglass]

Some of us don’t have 10 years unfortunately.

[Gymsack]

So your saying we should just ignore it ? hahahahahhaha

thanks for the post

[Farooqji in reply to Gymsack]

Good point. Just keep an eye on it

[Farooqji]

A very interesting commentary on this trial :

The small-scale trial, whose main aim was to demonstrate the safety of the approach, was sponsored by BlueRock Therapeutics, a subsidiary of the drug giant Bayer. The replacement neurons were manufactured using powerful stem cells originally sourced from a human embryo created an in vitro fertilization procedure.

These clues that the transplants helped came from brain scans that showed an increase in dopamine cells in the patients’ brains as well as a decrease in “off time,” or the number of hours per day the volunteers felt they were incapacitated by their symptoms.

However, outside experts expressed caution in interpreting the findings, saying they seemed to show inconsistent effects—some of which might be due to the placebo effect, not the treatment.

“It is encouraging that the trial has not led to any safety concerns and that there may be some benefits,” says Roger Barker, who studies Parkinson’s disease at the University of Cambridge. But Barker called the evidence the transplanted cells had survived “a bit disappointing.”

Because researchers can’t see the cells directly once they are in a person’s head, they instead track their presence by giving people a radioactive precursor to dopamine and then watching its uptake in their brains in a PET scanner. To Barker, these results were not so strong and he says it’s “still a bit too early to know” whether the transplanted cells took hold and repaired the patients’ brains.

technologyreview.com/2023/0...

[jeffreyn in reply to Farooqji]

Great article. Thanks.

See also this "backgrounder" from the same source.

After 25 years of hype, embryonic stem cells are still waiting for their moment:

technologyreview.com/2023/0...