What does the research tell us about Stem Cells treating PSP?

Honestly, it’s still really early days. There hasn’t been loads of research done in this area but we’ve broken down

•  What’s been tested so far

• What researchers found
• What might be coming next

Best Stem Cell Therapy clinics for PSP

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We speak to every clinic directly and review their medical team, the treatments they offer, how their stem cells are sourced and how they follow up with patients after treatment.

We also review licensing documents to confirm they meet the legal and medical standards of the country they operate in. For example, whether cells are processed in a GMP-certified facility or if they provide documentation on stem cell quality.

Clinics must also agree to let us collect and publish independent patient reviews. Both positive and negative.

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Top Stem Cell Therapy Clinics in India for PSP

Neurologist Specialist Autologous Stem Cells Certified Lab

Plexus is a leading center for neurological rehabilitation and stem cell therapy, helping patients with stroke, Parkinson’s, Autism, and more. Their facility is India’s first ISO-certified stem cell research center.

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Current PSP Stem Cell Trials

NEST Study – Neurologic Stem Cell Treatment for PSP (2025)
(You can read more about this study here)

• Participants: Recruiting
• Who’s running this Trial: MD Stem Cells
• Number of Participants: The study aims to enroll approximately 500 participants.
• Phase: Early safety trial (Phase 1)
• Details
  This study is trying something new for people with PSP: using your own stem cells to see if they can help protect the brain.
  
  Doctors take stem cells from your bone marrow (yes, from your own body), and then they give them back to you two ways:
  
  – Through a drip (IV) into your bloodstream
  – Through your nose (intranasal) to try and reach the brain directly
  
  The big hope? These cells might calm down inflammation in the brain and maybe help with symptoms like movement or balance. But right now, they’re just testing if it’s safe and if people show any signs of improvement.
  
  The study spans across several countries including the United States, Austria, and the United Arab Emirates.
• Why It Matters:
   PSP is super hard to treat. There’s no cure right now
  Since the cells come from your own body, there’s a lower chance of rejection
  Delivering cells through the nose is pretty cutting-edge. It could make future brain treatments easier
  

Previous PSP Stem Cell Trials (Since 2020)

IRCCS Casa Sollievo della Sofferenza Pilot Study. Intrathecal MSCs for PSP (2021)

• Completed in Italy: Small early-phase trial
• Who’s running it? Italian research group from IRCCS Casa Sollievo della Sofferenza
• How many people? Only 5 patients with PSP
• Phase: Phase 1 feasibility study

What’s this study about?

• This was an early test to see if it’s safe to inject stem cells directly into the fluid around the brain and spine of people with PSP. The researchers used mesenchymal stromal cells (MSCs) taken from the patients’ own bone marrow. Each person received the treatment twice, six months apart.

• Instead of giving the cells through a vein, doctors injected them straight into the cerebrospinal fluid (via lumbar puncture), hoping the cells could reach affected areas of the brain more directly.

What They Found

• No serious side effects from the procedure
  None of the 5 patients experienced serious adverse events after receiving stem cell injections
  Minor side effects (like temporary headaches or discomfort from the lumbar puncture) were reported but were manageable.
  This supports that intrathecal injection of MSCs appears safe. A big deal since it’s a direct-to-brain delivery method.
•  Some patients showed signs of their condition slowing down
  PSP usually gets worse pretty quickly. People tend to lose balance, movement, and thinking skills over time.
  In this study, doctors tracked each person’s symptoms using a special PSP score sheet.
  After the stem cell treatment, a few of the patients didn’t decline as fast as expected. Their symptoms slowed down a bit. They didn’t get better, but they didn’t get worse as quickly either.
  The researchers didn’t call it a breakthrough, but they were encouraged. It suggested the stem cells might be doing something to slow the disease down, at least for some people.
  
•  Inflammation markers in the brain fluid seemed to improve slightly
  They measured inflammatory markers in the cerebrospinal fluid (like IL-6, IL-8, TNF-alpha).
  After MSC treatment, these markers trended downward, suggesting the stem cells may have had an anti-inflammatory effect on the brain environment.
• However, the small sample size means these findings aren’t statistically powerful. They’re more of a hint than proof.

Why this matters:

This wasn’t a cure or a miracle, but it was a green light for moving forward with larger trials. It showed:

• First real-world trial showing intrathecal MSC delivery is safe in PSP
• Laid the groundwork for larger trials now being planned by the same Italian team
• Early signs of benefit. But we need more patients and better data
  
  If you want to read the full study, you can check it out here.
  

Case Report

Umbilical Cord Blood Stem Cells for PSP (China, 2021)

•  Single-patient case from Hainan Medical University, China
• Who’s running it? Dr. Xiaokun Qi and team at the First Affiliated Hospital of Hainan Medical University
• How many people? Just 1 patient (61-year-old male with advanced PSP)
• Study Type: Peer-reviewed case report (not a formal clinical trial)

What’s this Case study about?

This was a one-off, hospital-led treatment of a patient with severe Progressive Supranuclear Palsy (PSP) using umbilical cord blood stem cells (UCBSCs). The goal wasn’t to cure the disease, but to see if it might help slow things down.

The patient received a total of 6 doses of stem cells:

• 2 given through an IV (into the bloodstream)
  
• 4 injected directly into the spinal fluid (called intrathecal delivery)

Each dose contained 45 million mononuclear cells, totaling 270 million cells over the 2-week treatment period.

What they found:

• Improved muscle rigidity after 3 months
• No serious side effects were reported
• Brain scans looked stable even 2 years later. There was no further shrinkage in key brain areas
• Lived longer than expected (over 8 years after symptoms started, which is above average for someone at his stage)

Why this matters:

• This is one of the few detailed reports showing how umbilical cord stem cells might affect PSP
• The patient had advanced disease, yet his condition stabilized instead of rapidly worsening
• Helped justify launching larger clinical studies in China
• Shows stem cell therapy might be worth exploring for PSP, but much more research is needed

To read more about this case study, the full article is here.

Conclusion

Right now, the body of research on stem cell therapy for PSP is limited but growing. Here’s what we know:

The Good:

• Early-stage studies  including a small Italian Phase 1 trial and a Chinese case report  suggest that stem cell therapy may be safe and could help slow disease progression in some individuals.
  
• These findings have triggered larger studies in both countries, though results haven’t yet been published.
  
• A global, multi-center trial (NEST) is now recruiting PSP patients to study bone marrow stem cell therapy, showing increased international interest.

The Unknowns:

• However, there are still no large-scale, placebo-controlled trials proving that stem cell therapy can reliably improve PSP symptoms or outcomes.
  
• Most stem cell work in neurodegenerative disease still focuses on Alzheimer’s or Parkinson’s, not PSP directly.

In short: the science is early, but it’s advancing. The safety data is encouraging, and multiple trials are underway.

If you’re interested in understanding the actual treatment process, delivery methods, and types of stem cells used in these studies, head over to our page looking at Stem Cell Therapy for PSP.