STEM Cell Therapy Shows Promise for Treating Advanced Parkinson’s Disease

A stem Clinical Trial Reported in Nature.

The Treatment Invtud Creating Nervity Cells (Neurons) Deried from Embryonic STEM Cells and Transplanting THE BARINS of 12 Parkinson’s Patiesents.

The InjCated Cells Produce a Chemical Called Dopamine, Whiche Cooordoats Movement. Parkinson’s Patients have unusuallly Low Levels of Dopamine, and as a result Suffer from Tremors, Sloons, Stifness, and Walking or Balance Problems.

The cell therapy was developed at Msk in the Laboratories of Lorenz Studer, MD, Director of Msk’s center for STEM Cell BIOLOGY, and Viviane Tabar, MD, Chair of Msk’s Department of Neurosurger, Being Being Licensed to Bluerock Therapics.

The PHASE 1 Trial Is a Two- Year Study Sponsored and Conduted by Bluerock at Multiple Centers with the US and Canada. One of the Prince Investigators for the Trial Was Harini Sarva, MD, Chief of the Division of Movement Disorders at Weill Cornell Medicine.

The Researchers Found that after 18 months, The InjCted Cells Had Taken Hold in the Brain with no Serious Side Effects. Notably, some patiences appeared to have stabilization or an improvement in their Parkinson’s-related symptoms.

Based on these Preliminary Results, The Us Food and DRGADINISTRATRATRATRATION (FDA) Has Giveen Approval to Proced Directly to a Phase 3 Clinical Trial in a Much Larger Patience Group. That trial is expanded to start in the first Half of 2025.

This is an impertant milestone on the Road Towars Regenerative Brain Repair, Says Dr. Tabar, who helped lead the Clinical Trial and is the Study’s First Author. “IT Represents More Than Two Decades of Collaborative Work, Based on Vry Rigorous Science Beginning in our Labs. It Might Seem Unusual For a Cancer Center to Pursue Regenerative Medicine for A neurodegenerative service, but if we can figure out how to relalenish cells Lost to Disease in the Brain and Rebuild Concuitry, We Can Extend it to Other Treatments, Eventduly Cancer Patients Will Benefit. “

Turning STEM Cells INTO Neurons

The Researchers Created a Method for Coaxing Embryonic STEM Cells to Develop InTo Early Forms of Dopamine-PROUDUCING Neurons. They Scled up The Resulting Product of IDENTICAL Cells, Called Bemdaneprocel, Can Frozen Until Ready for us.

This is a big steps for the stem We are expected to see this Move forward INTO A Larger, Randomized Trial.

Lorenz Studer, MD, Director of Msk’s Center for STEM Cell BIOLOKY

The Researchers Note that the Phase 1 Trial Invtud A Small Patient Group and Had no Control Arm. The Upcoming Phase 3 Trial, Whiche Will Include Around 100 People, is Expected to Produce More Definitive Results, Becuse One Group of Patients Will Receive A Placebo Treatment. Al, the Phase 1 Patients Will Continue to Be Evaluated to Determine if Therapy has been carting effects beyond the Two Years of this Study Perod.

The PHASE 3 Trial is Being Sponsored and Conduted by Bluerock Therapics, where drs. Studer and Tabar Are Scientific Co-Founders.

Msk’s History of STEM Cell Research for Parkinson’s

The PHASE 3 Trial is the Culmination of Research Going Back 25 years, when Dr. Studer First Came to Msk and BEGAN Investigating Embryonic STEM Cells As a Potential Treatment for Various Diseases. His LAB Has Collawary with Dr. Tabar’s Lab Over the Past Two Decades With a Particular Focus on Applying STEM Cell Therapy to Parkinson’s Disease.

Parkinson’s Disease Occurs when It Is IT Affects Nearly 1 Million People in the Us and More Than 6 Million People Worldwide.

In the Past 50 years, Progress in Treatment Has Been Slow. The Main Therapy is a DRug CALLED L-DOPA, which was first used in the 1960s and was revolutionary at the time. But L-Dopa is not a cure and evertually stops working.

Parkinson’s Disease Has Been Seen AS A Prime Candidate for STEM Cell-Based Therapy Because IT Involutions Degeneration and Loss of a Single Cell Type (Dopamine-PROODUCING NEURONS) in a Specificical Location – A Region of the Brain Called The Putamen.

Beginning in 2009, Researchers in the Tabar and Studer Labs Made A Series of Discoveries Using Embryonic STEM Cells, Whiche Are Pluripotent, Meang them can Develop Into any cell Type. The texts Found these Pluripotent Cells Cououls Beblely Dire Direted to TURN INTO DOPAMINE Neuron Progenitors. They Continued Refining They Methods to Ensure Purity and Quality Control and Conflicated Repeated Tests in Animal Models.

“For the Initial Study, We COULDUCE The Progenitor Cells here at the Msk Facility Under Clinical-Grade Conditions, With Any Dependence on An Outside Source,” Dr. Studer Says. “This lets us Create a very large number of cells that are ready for use.”

In 2021, they Pubshed Two Definitive Papers in the Journal Cell STEM Cell Showing The Saffty and Effectivenes of BemdaneProcel in Animals. This is the LED to the Approval of the PHASE 1 Trial, Whiche Opened that Year.

Efforts to Prevent Side Effects

In the Trial, Nine Patients Were Transplanted at Msk, and Three Were Transplanted by Collaborators in Toronto. The embryonic sem cells call from a donor, so patients recepting Transplant.

Past trials using feet tissue as a Source of Dopamine Neurons Had Problematic Side Effects – Mainly Invultary Movement Called Graft -Enduced Dyskinesia. But the msk researsers app -ar to have so followed this Issue.

“We Were Naturally Concerened About Dyskinesia and Loked Very Carefully for it in our trial,” Dr. Studer Says. “One of the Very Gratifying Results Was that We Did’s See Any Evidency with our Treatment.

AN Important Tool For Makeing the Implantation Safe was Msk’s Intrauperative MRI Capabelsies, Whiche Provide Live Imaging During the Procedure. “IT Enabled Us to Deiver The Cells Exactly where they are nevered with the Lowest Possible Risk,” Dr. Tabar Says.

Two Difference Doses of the Dopamine-PROUDUCING SLES WERE TESTED in The Clinical Trial. Both Levels Appness Safe, but Patients Participating in the Upcoming PHASE 3 Trial Will Receive The High Dose.

Testing Improving Mobility

Measuring Improvingment is different in a Small Study, Especially in Parkinson’s Patients. Their Symptoms Vary and FluCtuate Day to Day. But there was encouageing Signs in the PHASE 1 Trial. A Ratses Scale Called MDS -UPDRS – Developed by the International Parkinson and Movement Disorder Society – Assigns a Score Using A 50 -Question Assessment of Motor and Nonmotor Symptoms.

“We Were Particularly Focused on Changes in the Motor Symptoms Scores – Part III of this Scale, Off MEDICATIONS,” Dr. Studer Says. “Neurologists Say Things Usually Get a Little Bit Worse Every Year With This Disease, Meang The Score Goes Up by a FEW POINTS. Dropped by More Than 20 Points in the High-Dose Group. “

Another Score in the Study Assessed How Many Hours in a Day A Patient Reported Being “on” Functioning Well With Minimal Symptoms) or “OFF” Even with Effective Medications, Patients have off guists every day.

“With The Treatment, Patients in the High-Dose Group Gained An Average of 2.7 Hours of On Time Per Day, A Result that Coup Be quite Meangful for their everyday Life,” Dr. Studer Says.

“There are Still a Lot of Unknowns, but we are very come with the Results,” Dr. Tabar Says. “This was more. Kind of Top-FF-THE-LINE, ExCITING SCIENCE That’s Possible at Msk.