Harvard Stem Cell Institute

Progress Report: December 2018
Breakthrough of the Year
Science , one of the leading scientific journals, has named research by HSCI scientists as the 2018 Breakthrough of the Year.
  • What they did: HSCI scientists Alexander Schier and Allon Klein analyzed the genetics of individual cells in zebrafish embryos during the earliest stages of development.
  • Why it matters: The technology to track cells in such detail can be used to study what goes wrong in human cells during the progression to cancer, diabetes, and other diseases.
A team led by HSCI scientist Brian Wainger has successfully completed a clinical trial in patients with ALS.
  • What they did: In order to model ALS in a lab dish, researchers used patient stem cells to make motor neurons. The researchers discovered that the ALS motor neurons were hyperexcitable (or overactive), and that the drug retigabine lowered excitability and improved survival.
  • Why it matters: Retigabine has a potentially positive effect in patients with ALS. Additionally, researchers will use patient cells collected in the trial to further test candidate therapies.
Lung and liver cancers: new findings
lung and liver cancers
Left to right: Carla Kim, Samuel Rowbotham, Wei-Chien Yuan, Fernando Camargo.
HSCI scientists have published two new studies that give important insights into lung and liver cancers, and have potential implications for cancer treatment.
  • Carla Kim has identified a caveat for a type of lung cancer therapy: while the therapy fights against general tumor cells, it also boosts cancer stem cells that lead to tumor development. The study identifies an alternate therapeutic approach that takes cancer stem cells into account.
  • Fernando Camargo has identified a protein involved in liver cancer that could be targeted by a therapeutic drug. The protein is the first 'druggable' target identified in the well-studied Hippo biological pathway.
HSCI Seed Grant leads to new class of therapeutics
mRNA therapeutics
Biotech company Moderna Therapeutics, co-founded by HSCI scientist Derrick Rossi, is set to bring a new class of treatments to patients.
  • Moderna's recent public offering is the largest ever for a venture-capital-backed biotech company, reflecting the potential of its new technology: mRNA therapeutics.