HSCI researchers use patient-derived motor neurons to screen for clinically relevant targets

HSCI scientists identify gut-brain connection in ALS

Discovery opens doors to improving diagnostics and developing new therapy for majority of ALS patients

Summary

• HSCI-funded research using human pluripotent stem cell models and tissue from ALS patients found that the protein TDP-43 regulates a gene called Stathmin2 (STMN2).
   
...

Clinical trial success based on stem cell models in the lab

SMN protein (red) is necessary for the survival of spinal cord neurons (motor neurons) responsible for breathing and all movement. Harvard researchers have found a compound that stabilized this protein in mouse and human motor neurons. This may lead to the development of new...

President Drew Faust accepts the ALS Ice Bucket Challenge and is doused by Harvard’s ALS research team, led by Harvard Stem Cell Institute Principal Faculty member Kevin Eggan, PhD. She nominates MIT president Rafael Reif, UMass Boston Chancellor Keith Motley, and The Harvard Dorm Crew.

...

Similar results in experimental animals and human cells in dish

A series of studies begun by Harvard Stem Cell Institute (HSCI) scientists eight years ago has led to a report, published in ...

Discovery will lead directly to clinical trials

Harvard stem cell scientists have discovered that a recently approved medication for epilepsy may possibly be a meaningful treatment for amyotrophic lateral sclerosis (ALS)—Lou Gehrig’s disease, a uniformly fatal neurodegenerative disorder. The researchers are now collaborating with Massachusetts General Hospital to design an initial clinical trial testing the safety of the treatment in ALS patients.... Read more about Patient stem cells help identify common problem in ALS

A team of Harvard stem cell researchers has succeeded in reprogramming adult mouse skin cells directly into the type of motor neurons damaged in amyotrophic lateral sclerosis (ALS), best known as Lou Gehrig’s disease, and spinal muscular atrophy (SMA). These new cells, which researchers are calling induced motor neurons (iMNs), can be used to study the development of the paralyzing diseases and to develop treatments for them.... Read more about From skin cells to motor neurons: researchers find success with direct cellular reprogramming

A new finding by Harvard stem cell biologists turns one of the basics of neurobiology on its head by demonstrating that it is possible to turn one type of already differentiated neuron into another within the brain.... Read more about HSCI researchers turn one form of neuron into another in the brain

Using a new, stem cell-based, drug-screening technology that could reinvent and greatly reduce the cost of developing pharmaceuticals, researchers at the Harvard Stem Cell Institute (HSCI) have found a compound that is more effective in protecting the neurons killed in amyotrophic lateral sclerosis (ALS) than are two drugs that failed in human clinical trials after large sums were invested in them.... Read more about New use for stem cells identifies a promising way to target ALS

The Harvard Stem Cell Institute (HSCI) has entered into a strategic partnership with Evotec AG to identify compounds that prevent or slow down the loss of motor neurons, which is characteristic of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.... Read more about Harvard Stem Cell Institute and Evotec collaborate to advance ALS research