Lechevallier Charlotte

Stem cells strike again: Promising results from a new Parkinson’s trial just published in the journal Cell. Dopaminergic progenitors are early-stage brain cells that can mature into dopamine producing neurons. These are some but not all of the cells (and circuits) lost in Parkinson’s disease. Jin Woo Chang and colleagues just dropped a new paper on a phase 1/2a clinical trial testing human embryonic stem cell derived dopamine progenitors (A9-DPCs) transplanted into the brains of 12 persons living w/ Parkinson’s disease. Key Points: - Bilateral transplantation of high purity human embryonic stem cell–derived A9 dopamine progenitors into the putamen was safe and well tolerated. - Participants experienced meaningful improvements in off medication motor scores w/ greater benefit in the high-dose group. - Dopamine transporter PET imaging showed increased uptake in the posterior putamen suggesting graft survival and dopamine restoration. My take: These results were presented at the Hawaii MDS International Congress, and I was pleased to see the full paper published this week. Big 3 points: embryonic cells, use of immunosuppression and it was safe. Not enough information yet on how effective and the results were similar to the two other trials recently published in the journal Nature. Here are 5 points that resonated w/ me: 1- Dopamine producing cells can be grown, purified and safely transplanted into the human Parkinson's brain. 2- The Korean research team found that these grafts may begin to restore dopamine signaling and improve motor function. 3- No tumors, no dangerous overgrowth and only minor temporary side effects during the first year. 4- Imaging studies confirmed that transplanted cells took hold and were functionally active in the brain. 5- The results are early and open-label and therefore the question remains whether well designed cell replacement therapies may one day complement other Parkinson's therapies like medication, focused ultrasound, pumps and DBS. Immunosuppression (drugs) were used in this trial. cell.com/cell/fulltext/S0092… #parkinson @CellPressNews @ParkinsonDotOrg @movedisorder @SfNtweets @PdAvengers