Research from Germany, using brain organoids and mice, could bring forward new drug therapies for a fatal brain disease.
Amyotrophic lateral sclerosis (ALS), the most common form of motor neuron disease, causes weakness and wasting in the limbs, with most patients not surviving longer than three years.
Researchers at Heidelberg University developed a new drug named FP802, which they found could prevent the connection between healthy neurons and a harmful protein, thereby avoiding ALS symptoms.
Testing FP802 in both genetically altered mice that mimic ALS, and organoids developed from ALS patients’ stem cells, the drug was able to protect nerve cells in both and improve movement in mice.
Hilmar Bading, lead investigator at Heidelberg, said: "The discovery of this new pharmacological class of drugs opens up a promising path for fighting ALS.”
The full study is published in Cell Reports Medicine and FP802 will soon be tested in human clinical trials, in collaboration with FundaMental Pharma, also based in Heidelberg.