Stem Cell Research Parkinson’s

What They’re Testing

• UX-DA001: a personalized stem cell therapy for idiopathic Parkinson’s disease.
• Goal: evaluate whether a one time treatment can safely improve motor symptoms (tremor, rigidity, bradykinesia).

What Makes This Treatment Different?

• The Cells Come from the Patient: A small blood sample is taken and reprogrammed into induced pluripotent stem cells (iPSCs).
• Programming the Cells: iPSCs are guided to become midbrain dopaminergic progenitor cells (dopamine-producing neurons).
• Why That Matters: Using autologous cells avoids immunosuppressive drugs, reducing risks like infections, tumors, or organ damage.

How It’s Delivered

• Each patient receives a single dose of UX-DA001.
• Cells are implanted into both sides of the putamen (movement-control region).
• Delivery via stereotactic neurosurger(precise, minimally invasive) under general anesthesia.

How It’s Delivered (Recap)

• Single-dose UX-DA001 implanted bilaterally in the putamen.
• Stereotactic neurosurgery under general anesthesia.

Primary Measures (Safety)

• Adverse events tracked within the first 4 weeks and throughout 2 years.
• Monitoring for serious complications related to surgery or the implanted cells.

Secondary Measures (Efficacy)

• PET/MRI to assess cell survival, appropriate growth and absence of tumor formation.
• Changes in UPDRS Part II & III (motor function).
• Changes in daily levodopa requirements.
• Non-motor symptoms (e.g.,sleep, mood).
• Overall disease stage (Hoehn–Yahr scale).

What They Found (Early Case)

• First patient showed improvements in sleep quality and motor function within 1 month post surgery.
• Patient described gradual, day-by-day symptom improvement.
• Trial is ongoing with up to 2 years of follow-up for long term safety and effectiveness.

What Makes This Trial Important?

• China’s first registration directed clinical trial for personalized iPSC-derived Parkinson’s therapy.
• Among the few globally that: uses autologous cells, avoids immunosuppressants & has approvals from both NMPA and the U.S. FDA
• If successful, could shift care from daily drug management to long-lasting, personalized cell therapy that aims to restore dopamine function.

What They’re Testing

• Whether new dopamine-producing brain cells made from the patient’s own skin or blood can be safely implanted to improve movement and reduce medication needs.
• These cells are created using iPSCs: adult skin or blood cells are reprogrammed back to a stem cell state.
• The iPSCs are guided to become dopamine progenitor cells — early-stage brain cells that can mature into dopamine-producing neurons.
• The cells are injected into the putamen, a movement-control region particularly affected in Parkinson’s disease.

Cell Count

• Each participant receives 4 million cells per side of the brain (total 8 million dopamine-producing cells).

How the Stem Cells Were Made

• Begins with a small sample of either skin or blood from the patient.
• Cells are reprogrammed into iPSCs using iCamuno’s method (iCam-iPSC), designed to yield stable, high-quality stem cells.
• These cells behave like embryonic stem cells but are patient-derived, lowering ethical concerns and the risk of immune rejection.
• They are then directed in the lab to become dopamine-producing neurons.
• AI and advanced testing are used to ensure cell health, consistency, and readiness for clinical use.

Tumor Testing & Safety Checks

• Screening for harmful mutations or genetic abnormalities.
• Toxicity testing to confirm the cells don’t cause damage.
• PET and MRI scans to evaluate the brain before and after treatment.
• Post-surgery monitoring for a full year for side effects or cell-related issues.

Primary Outcome

• Safety: tracking any serious side effects or complications from the treatment.

Secondary Outcomes

• Changes in motor symptoms (UPDRS-III).
• Changes in Parkinson’s stage (Hoehn and Yahr scale).
• Quality of life (PDQ-39 questionnaire).
• Post-treatment Parkinson’s medication requirements.
• Brain dopamine activity via PET (18F-DAT).

What They’re Testing

• Whether lab-grown brain cells can improve Parkinson’s symptoms such as tremor, stiffness, and slow movement.
• The cells are dopaminergic progenitor cells — the type that make dopamine, which is reduced in Parkinson’s.
• Cells are derived from induced pluripotent stem cells (iPSCs) made by reprogramming blood cells from a healthy donor.
• Designed as an “off-the-shelf” therapy that doesn’t need to be custom-made for each patient.
• Cells are implanted into the putamen on both sides of the brain using a precise surgical method.

Cell Count

• Two dosing groups in the study:
• Low Dose Group: exact dose not disclosed
• High Dose Group: exact dose not disclosed

How the Stem Cells Were Made

• iPSCs created from blood cells donated by a healthy adult.
• Episomal reprogramming used to generate iPSCs (non-viral, does not permanently alter DNA).
• Cells expanded and guided over several months to become dopaminergic progenitor cells.
• Final cells are described as transgene-free, with batch consistency suitable for treatment.

Primary Outcome

• Safety: any adverse events within 24 weeks of transplantation.

Secondary Outcomes (24 months)

• Changes in movement symptoms (e.g., shaking, balance, mobility).
• Overall quality of life and daily activity impact.
• Levels of depression and anxiety.
• Disease progression over time.
• Brain scans to assess survival of transplanted cells.
• Changes in Parkinson’s medication needs after treatment.

Results So Far

• The trial is not yet recruiting; no results have been posted.

What They’re Testing

• Evaluating whether programmed human embryonic stem cells can safely improve Parkinson’s symptoms.
• Parkinson’s is caused by the loss of dopamine-producing brain cells.
• TED-A9 therapy replaces these cells using lab-grown ones that develop into dopamine-producing neurons after transplantation.
• The cells were surgically placed into the putamen — a brain region controlling movement.

Cell Count

• Participants divided into two groups:
• Low Dose Group: 3.15 million cells total.
• High Dose Group: 6.30 million cells total.

How the Stem Cells Were Made

• TED-A9 cells originated from human embryonic stem cells.
• Cells were guided with specific chemicals to become dopamine-producing progenitor cells.
• This process avoids genetic modification and ensures cell safety, consistency, and effectiveness.

Cell Quality Checks

• Researchers verified that TED-A9 cells could produce dopamine under different lab conditions.
• All tests confirmed active dopamine production.

Tumor Testing & Safety Checks

• Continuous MRI and PET monitoring to detect tumor formation or abnormal growth.
• Regular medical check-ups and lab tests for adverse reactions.
• No tumors or serious health issues have been linked to the treatment so far.

What They’re Measuring

• Main Goal: safety — monitoring side effects like tumor growth, infection, or bleeding.

Other Goals

• Improvement in movement symptoms measured by Parkinson’s scales.
• Cognitive improvements.
• Better overall quality of life.
• Brain imaging to confirm transplanted cell survival and function.

Results So Far (12 months)

• High-dose group showed 44.4% improvement in movement and daily function.
• Low-dose group showed 19.4% improvement.
• Brain scans confirmed transplanted cells were active and producing dopamine.
• Significant gains in movement, reduced medication wearing-off, and better daily living.
• Notable improvements in non-movement symptoms as well.

Why It Matters

• TED-A9 therapy could replace lost dopamine cells, improving quality of life for Parkinson’s patients.
• Provides a more consistent and reliable option compared to fetal tissue therapies.
• Early results suggest a potential breakthrough for Parkinson’s treatment.

What’s Next

• Ongoing monitoring to confirm long-term safety and effectiveness.
• Larger clinical trials planned to validate these early promising results.

What Are They Testing?

• Skin sample taken from behind the ear or arm
• Skin cells reprogrammed into induced pluripotent stem cells (iPSCs)
• iPSCs converted into early-stage dopamine-producing brain cells
• Cells surgically implanted into the putamen on both sides of the brain

Do We Know How They Make the Cells?

• Aspen reprograms skin cells into iPSCs and differentiates them into dopamine-producing brain cells
• The exact proprietary methods have not been disclosed

Tumor Risk and Safety Checks

• Genetic and quality-control testing performed at each stage with AI-based analysis
• Screening likely includes checks for mutations and developmental abnormalities

What They’re Measuring

• Main goal: safety and identification of any treatment-related problems
• Reduction in OFF time (when medications stop working)
• Increase in ON time (when symptoms are controlled)
• Daily movement, mood, and quality of life

What Have They Seen So Far?

• First 3 patients at 6-month follow-up: no serious side effects
• Minor issues reported: incision site pain and tongue swelling
• Average 45% improvement in motor symptoms during OFF periods
• 71% improvement in daily living tasks
• About 2 fewer hours of OFF time per day
• About 1.5 more hours of ON time per day
• Early results are encouraging

Why It Matters

• First multi-site U.S. trial using a person’s own reprogrammed cells to restore dopamine neurons
• Potential to move beyond symptom management toward replacing lost brain cells

What They’re Testing

• Doctors are trying to see if they can safely transplant lab-grown dopamine-making brain cells into the putamen and help improve Parkinson’s symptoms.
• These new cells were designed to replace the ones lost because of Parkinson’s, and possibly help the brain function more normally again.

Cell Dose (How Many Were Given)

• Each patient had surgery to place the cells into both sides of their brain. There were two different dose groups:
• Low Dose: 0.9 million cells per side (1.8 million total)
• High Dose: 2.7 million cells per side (5.4 million total)

How the Cells Were Made

• The cells were created in a lab from human embryonic stem cells using a carefully controlled process to turn them into dopamine-producing cells, the type lost in Parkinson’s. These are NOT IPSC cells.
• They were frozen and stored, so they could be used anytime. Like a ready-made medicine.
• Before being used in people, the cells were tested for safety, checked to make sure they wouldn’t form tumors, and screened to remove any unwanted types of cells that might cause side effects.

Tumor Testing & Safety Checks

• To make sure the cells were safe:
• They were tested in animals to rule out tumor growth
• They were carefully screened to ensure they wouldn’t become any other type of cell
• After surgery, all patients were closely monitored with brain scans and checkups

What They Found

• No tumors
• No abnormal tissue growth
• No dangerous side effects caused by the cells

Main Goal

• Safety: Were there any serious side effects from the surgery, the cells, or the immune-suppressing drugs?
• → No major issues were found.

Other Goals

• Did the transplanted cells survive and work?
• → Yes — brain scans showed increased dopamine activity where the cells were placed.
• Did the patients’ movement problems get better?
• → Yes — especially in the high-dose group.
• Did it reduce the amount of time they had symptoms each day?
• → Yes — by about 2.7 hours per day in the high-dose group.
• Did they need more medication?
• → No — medication use stayed about the same or decreased slightly.
• Were there any involuntary movements (called dyskinesias) caused by the graft?
• → No and that’s a big deal. Previous stem cell trials had problems with this.

Results So Far

• Everyone completed at least 18 months of follow-up
• People in the high-dose group improved the most, with a 23-point improvement on a standard Parkinson’s motor scale (that’s considered a major improvement)
• Brain scans showed the new cells survived and were making dopamine
• No one developed graft-related movement problems (a major issue in older types of cell therapy)

Why It Matters

• This is the first time a ready-to-use, lab-made dopamine cell product from embryonic stem cells was safely used in humans.
• It avoided the ethical and technical problems of using fetal brain tissue
• It showed early signs that the cells can survive, function, and improve movement
• It could become a scalable therapy for Parkinson’s — something that can be made in large batches and used when needed

What’s Next

• Based on the positive results, the same therapy (bemdaneprocel) is now moving into a larger Phase III clinical trial called exPDite-2.
• Led by BlueRock Therapeutics (a Bayer subsidiary), this will be the first-ever registrational Phase III trial of an off-the-shelf, embryonic stem cell–derived therapy for Parkinson’s.
• The study will enroll around 100 participants, include a placebo control group, and measure motor function, quality of life, and safety over 78 weeks.

What They’re Testing

• The study is testing whether a patient’s own blood cells can be used to create stem cell-derived dopamine neurons that replace the ones lost in Parkinson’s disease.
• Blood cells are collected from each patient and converted into induced pluripotent stem cells. These are then turned into midbrain dopaminergic neurons in the lab.
• The final product is implanted into the brain’s putamen region, where dopamine neurons normally reside. The goal is to test whether these new neurons can survive, integrate, and restore dopamine function.

Cell Count

• Not disclosed.

How the Stem Cells Were Made

• The stem cells come from the patient’s own blood.
• These blood cells are reprogrammed into iPSCs and then differentiated into dopaminergic neurons using a protocol developed by the NRI at McLean Hospital.
• The technique avoids donor cells and immunosuppressants. No genetic engineering details were shared.

Tumor Testing & Safety Checks

• No human safety data has been published yet.
• The NRI previously tested this approach in non-human primates and found long-term survival of the cells without tumor formation.
• The Phase 1 trial is designed to follow each participant for at least 12 months to monitor safety and any signs of overgrowth.

Primary Outcome

• Safety and feasibility of implanting autologous iPSC-derived dopamine neurons

Secondary Outcomes

• Signs of motor improvement in Parkinson’s symptoms
• Signs of cell survival and integration using imaging

Results So Far

• No clinical results have been published.
• As of early 2025, three patients have been treated and are being monitored.

What They’re Testing

• This study is testing whether dopamine progenitor cells (immature cells that can grow into functional dopamine neurons) can be safely transplanted into the brain and potentially improve Parkinson’s symptoms.
• The cells were transplanted into the putamen, a key region affected in Parkinson’s that controls movement.

Cell Count

• Participants were split into two dose groups:
• Dose 1: 3.54 million cells per side (7.08 million total)
• Dose 2: Double that dose, 7.08 million per side (14.16 million total)
• Each dose is calculated to target a specific number of surviving dopamine neurons: 100,000 neurons per putamen for Dose 1, and 200,000 for Dose 2.

How the Stem Cells Were Made

• The cells come from human embryonic Stem Cells that were originally created in a lab using leftover embryos from IVF.
• Scientists then spent 16 days carefully turning these stem cells into the kind of brain cells that make dopamine. The same type that are lost in Parkinson’s disease.
• These dopamine making cells, called STEM-PD, are frozen and tested thoroughly to make sure they’re safe before being used in people.
• Once transplanted into the brain, they gradually grow into working dopamine cells over several months and start doing the job of the lost ones.

Tumor Testing & Safety Checks

• Before trying these cells in people, scientists tested the exact same batch in animals. In those tests, the cells:
• Survived long-term and connected properly with the brain
• Helped improve movement symptoms caused by Parkinson’s
• Did not cause tumors
• Stayed in place and didn’t spread to other parts of the body
• Showed no signs of toxicity or harmful side effects
• Now that the trial is in humans, safety is being closely monitored for three full years after the transplant. This includes regular check-ups, brain scans and blood tests to check for any immune reactions.

Primary Outcomes

• Safety: Number and nature of serious adverse events (12 months and again from 12–36 months)
• No signs of abnormal growths on brain scans (MRI)

Secondary Outcomes

• Motor function (UPDRS, peg tests, sit-stand-walk tests)
• Cognitive testing (MoCA, HVLT-R, Stroop, Boston Naming, etc.)
• Quality of life scales (PDQ-39, EQ-5D-5L)
• Medication dose changes

Exploratory Outcomes

• PET brain scans at 6, 12, 24, and 36 months to check if the new dopamine cells are surviving and working properly
• Wearable sensors on the wrist to track movement problems like stiffness, shaking, and symptom ups and downs throughout the day
• Tests on spinal fluid and blood to check for inflammation or immune system reactions
• Response to Parkinson’s medication (L-dopa) measured over time to see if effects improve or change after the transplant

What They’ve Found So Far

• Dopamine Cells Survived: PET brain scans taken 6 to 12 months after surgery show that the transplanted cells are alive and surviving in the brain. A key sign the therapy is working as intended.
• Integration in the Brain: The dopamine progenitor cells appear to have settled into the putamen, which is responsible for controlling movement.
• On Track for Dose Escalation: The first four participants received the lower dose and tolerated it well. Based on these early safety and imaging results, the trial has moved ahead with the higher dose group.
• No Major Safety Issues Reported: So far, there have been no reports of serious complications related to the transplant. Safety monitoring is ongoing and includes brain scans, blood tests, and regular check-ins over 36 months.

What’s Next

• The higher-dose group will now move forward with surgery.
• Participants will continue to be followed for 36 months, with additional imaging and clinical assessments along the way.

The review also pointed out some big challenges:

• 1. Cell Prep Was All Over the Place
  Some studies grew their cells in ultra-clean labs (GMP compliant), while others didn’t say how their cells were made.
  Some tested cell quality; others didn’t.
  Many didn’t explain how they confirmed the cells could help with Parkinson’s.
• 2. Cell Types and Doses Were Inconsistent
  Some used 1 million cells, others used 10 million.
  Some gave one treatment; others gave multiple.
  Not all cells were designed to become brain cells which might explain why some treatments didn’t work.
• 3. Short Follow-Ups
  Most patients were only followed for 3–24 months.
  We still don’t know how long the benefits last. Or if long-term problems might appear later.
• 4. Small Study Sizes
  Most trials had fewer than 30 patients.
  We need bigger studies to confirm results and learn who benefits most.

Was It Safe?

• Yes the treatments were generally safe:
• No tumors or major immune rejection.
• Side effects were rare and mild, like fever or vein inflammation.
• A few patients had involuntary movements after treatment (called GID), but this was very uncommon.

• Cell therapy for Parkinson’s looks promising but it’s not a cure yet.
• It can improve symptoms, especially motor skills, and appears to be safe in the short term.
• Donor-derived MSCs and neural cells may help in different ways. One by healing signals, the other by cell replacement.
• But the field needs more consistency, better-quality trials, and longer follow-up to know how to use it best.

Average Success Rate

• The average improvement across all 9 studies was: WMD = −14.86 (95% CI: −16.62 to −13.10), which is considered a clinically significant reduction in Parkinson’s symptoms.

In simple terms, on average, people who received stem cell therapy improved noticeably compared to those who didn’t. Their symptoms became milder, and their overall condition got better over a 12-month period.

All studies showed some improvement:

• Every trial included in the review showed that patients got better after stem cell treatment, even if they used different types of cells or delivery methods.

The results were very consistent:

• The outcomes across all studies were surprisingly similar, which is a good sign that the treatment really works — even without guiding the cells to the exact spot.

Delivery methods and which was most effective

• Intravascular injection (through a vein)
• Intrathecal injection (into the spine)
• Intraventricular injection (into the brain)
• Intravascular was the most effective, showing the largest and most consistent improvement in symptoms.
• Injecting cells through the bloodstream may help them circulate more widely and survive better than direct brain injections.

How they discussed MSCs helping in Parkinson’s

• MSCs likely don’t work by turning into new brain cells.
• Instead, they seem to release healing signals that reduce inflammation, calm the immune system, and protect existing neurons.
• This supports the idea that MSCs act like “cellular support crews,” improving the brain environment rather than replacing lost dopamine cells directly.

Limitations they acknowledged

• Most studies were small and not randomized, which can introduce bias.
• Follow-up times were usually short. Only one study looked beyond 12 months, so we don’t know if benefits last long-term.
• Differences in how cells were prepared and given (like dose or quality) weren’t analyzed.
• No sham surgery controls were used, so the placebo effect can’t be ruled out.
• Not enough data to compare the different types of MSCs in a head-to-head way.

Their overall conclusion:

• “Data from controlled trials suggest that stem cell transplantation as a therapy for Parkinson’s disease can be effective for at least 12 months. The factors that may influence its curative effect are time after transplantation and stem cell types.”

In Lehman’s terms:

• Stem cell therapy does help people with Parkinson’s, at least in the first year after treatment.
• The results depend on what kind of stem cells are used and how long it’s been since the transplant.
• More long-term studies are needed, but early results are promising.

• The cells were transplanted to both sides of the brain into the putamen using a computer-guided system for precise brain surgery.
• The cells were prepared fresh and injected on the day of surgery.
• Patients also received an immunosuppressive drug starting before surgery, which was gradually reduced and discontinued at 15 months after transplantation.

Cell Type & Source

• Dopaminergic progenitor cells, made from iPS cells donated by someone else (not the patient).
• Dopaminergic progenitor cells are immature brain cells that can grow into neurons which make dopamine, a chemical that helps control movement.
• The iPSCs came from skin cells donated by a healthy adult with a common immune type, making them more likely to be accepted by different patients.
• The donor’s skin cells were reprogrammed using a non-viral method (episomal plasmids), a safer process that doesn’t alter DNA permanently.
• Key genes like OCT4, SOX2, KLF4, L-MYC, and LIN28 were used to reverse the cells back into a stem-like state.
• Cells were tested for mutations & tumors.

Dosage

• Three patients got a low dose of about 2–2.6 million cells on each side of the brain.
• Four patients got a high dose of about 5.3–5.5 million cells per side.

Motor Function (Primary Efficacy Endpoint)

• The Movement Disorder Society Unified Parkinson’s Disease Rating Scale part III OFF score is a clinician-rated scale for motor symptoms when a patient is off medication.
• These improved by an average of 9.5 points (20.4%) at 24 months in all patients.
• The “ON” score, which is when they are on medication, improved by an average of 4.3 points (35.7%) at 24 months. 5 out of the 6 patients showed improvement.
• Hoehn–Yahr stages (a clinical scale for Parkinson’s disease progression) improved in four patients during off-time, with one patient improving by 2 stages and three others by 1 stage.

Dopamine Production & Safety (Secondary Endpoints)

• Brain scans showed dopamine production in the putamen rose by an average of 44.7% after 24 months, with bigger gains in the high-dose group (63.5%) than the low-dose group (7%).
• MRI scans showed the transplanted cells slowly grew in size over time, but there were no signs of tumors or unusual cell overgrowth.
• No patients showed signs of brain inflammation in the putamen or nearby areas on PET scans.

Safety

• No serious adverse events were reported in any patient.
• Dyskinesia scores rose after 24 months, but the increase matched usual medication side effects, not the severe movement problems seen with older cell therapies.
• Dyskinesia is involuntary, uncontrolled movements, such as twisting, jerking, or writhing, that can happen as a side effect of Parkinson’s medication.

How the Cells Worked

• The researchers believe the stem cells helped by surviving in the brain, producing dopamine, and integrating into the existing neural circuits without forming tumors. They propose:
• The transplanted iPS-cell-derived dopaminergic progenitors matured and functioned as dopamine neurons, effectively replacing some of the lost dopamine cells in the brains of patients with Parkinson’s disease.
• By removing cells that make serotonin, the researchers may have avoided the severe movement problems seen in earlier fetal tissue transplants.

What we don’t know

• To fully confirm the transplanted cells survive long-term, cause no inflammation and never form tumors, researchers will need longer follow-up.

Conclusion

• This study shows that allogeneic transplantation of iPS-cell-derived dopaminergic progenitors appears to be a safe and potentially effective regenerative therapy for patients with Parkinson’s disease.
• The trial demonstrated a favorable safety profile.
• In August, Sumitomo applied for marketing approval in Japan for this Stem Cell treatment.
• They’re currently running trials in the US too as well.

What They’re Testing

• This study tested whether autologous mesenchymal stem cells (HB-adMSCs), derived from each participant’s own fat tissue, could be used to reduce inflammation and improve clinical outcomes in Parkinson’s disease when administered through a series of IV infusions.
• The idea is that these cells may help regulate immune responses, reduce neuroinflammation, and potentially protect brain cells involved in movement.

How the Stem Cells Were Made

• Each participant underwent a small fat biopsy, from which adipose-derived mesenchymal stem cells (adMSCs) were isolated and expanded in a lab.
• These HB-adMSCs were:
• Autologous (from the same patient)

Cell Count & Dosing

• Each participant in the treatment group received:
• 6 intravenous (IV) infusions
• One infusion every 4 weeks over a 32-week period
• The placebo group received 6 matched saline infusions on the same schedule.

Throughout the study and 52-week follow-up:

• Patients were closely monitored for tumor formation, cell overgrowth, or unusual responses (Ongoing safety surveillance to catch delayed adverse effects).
• No cases of tumor growth, cancer, or abnormal cell proliferation were reported. (Supports a strong safety profile over the treatment duration).
• No serious adverse events were linked to the stem cell infusions. (Indicates the treatment was well tolerated in all participants).

What They’re Measuring

• Primary Outcome: Safety and tolerability: frequency and type of adverse events
• Secondary Outcomes:
• Changes in motor symptoms using the Unified Parkinson’s Disease Rating Scale (UPDRS)
• Neuroinflammatory markers in blood (e.g., monocyte subtypes)
• Changes in neurodegeneration biomarkers (e.g., Neurofilament light chain, NfL)
• General clinical progression

• No treatment-related serious adverse events occurred.
• One participant had a transient seizure (not related to treatment).
• Treated patients showed improvement in UPDRS motor scores, while placebo patients worsened.
• Blood analysis showed a reduction in pro-inflammatory monocytes in treated patients.
• NfL levels trended lower in the treatment group (a positive neuroprotective signal, though not statistically significant).

Timeline:

• Start Date: 2021
• Treatment Period: 32 weeks
• Follow-up Completed: 52 weeks

What They’re Testing

• The study tested whether mesenchymal stem cells (MSCs) from donor bone marrow could improve motor symptoms when given in repeat infusions.
• Three groups were compared:
• Placebo group (no real stem cells)
• 2-dose group (2 real infusions, 1 placebo)
• 3-dose group (3 real infusions)
• Each infusion was spaced 4 months apart, and each dose was based on body weight: 10 million cells per kilogram.
• That’s one of the highest doses ever tested for Parkinson’s.

How the Cells Were Made

• The study did not specify how the MSCs were sourced or processed.
• We know only that they came from donor bone marrow and were allogeneic (not from the patient themselves).
• Details on how the cells were expanded, tested, or purified were not included in the public record.

What They’re Measuring

• Primary Outcome: Motor function, using the MDS-UPDRS Part III scale (a gold standard tool in PD research). It’s basically researchers measuring how well patients could move using a trusted Parkinson’s rating scale. The lower the score, the better the motor function.

Secondary Outcomes:

• Safety and side effects
• Immune response to the donor cells
• Mobility (Timed-Up-and-Go test)
• Daily function and quality of life (PDQ-39, EQ-5D, Schwab & England scales)
• Cognition, mood, anxiety, and smell
• Biomarkers in blood and spinal fluid, including:
• NfL (a marker of nerve damage)
• Cytokines, chemokines, growth factors, and neurotransmitters
• Alpha-synuclein levels

Motor Symptoms (MDS-UPDRS-III scores):

• Placebo Group: Improved by 3 points
• 2 MSC Infusions: Improved by 11 points
• 3 MSC Infusions: Improved by 16 points
• The more MSC infusions, the better the improvement (and the differences were statistically significant – p < 0.001).

NfL Biomarker:

• All groups showed increases in serum NfL, including placebo. Serum NfL is a blood marker that shows how much stress or damage your nerves are under.
• But in the 3-dose group, NfL levels were positively correlated with motor improvement (R² = 0.42, p = 0.006).
• This suggests NfL could be a useful biomarker for tracking recovery.

Why It Matters:

• This trial shows that high-dose, repeated stem cell infusions may have a real impact on slowing or even reversing motor decline in Parkinson’s.
• And importantly, the patients who got all 3 MSC infusions did the best.
• It also hints that NfL, a blood-based biomarker, might help doctors track who’s responding to treatment in the future.