StemCell Therapy

Cells Weekly is a digest of the most interesting news and events in stem cell research, cell therapy and regenerative medicine. Cells Weekly is posted every Sunday night!

This week was a week of ISSCR 2015, so read all news from the conference here.

1. The price of gene therapy trial failure A year ago, Celladon became the first gene therapy company, which received designation by US FDA Breakthrough Therapy for their MYDICAR platform in heart failure. It means very good data from Phase 1 trial and impressive efficacy in early Phase 2. Despite the recognition and all excitement, a year later, MYDICAR failed to deliver efficacy as result of Phase 2. The fate of the company was unclear. This week, the company warned their investor about potential termination suspension of MYDICAR and other pre-clinical programs, sale/ merger of the company or liquidation:

We are aggressively pursuing that course, said Paul Cleveland, president and chief executive officer of Celladon. If we are unable to identify a merger or sale that provides superior value to our shareholders, we will move forward with a liquidation and distribution of net cash to shareholders. The Company also announced a second reduction in its workforce, with approximately half of the employees not previously notified of termination of employment being expected to depart in the third quarter.

Very bad news for the field! Many lessons to learn

2. Heart peacemaker activity turned on by light The most interesting study from this week came from 2 Israeli scientists. They were able to modulate cardiac pacing in vivo, using optogenetic approach. AVV vector with light-sensitive protein transgene was injected directly into myocardium:

This allowed optogenetic pacing of the hearts at different beating frequencies with blue-light illumination both in vivo and in isolated perfused hearts. Optical mapping confirmed that the source of the new pacemaker activity was the site of ChR2 transgene delivery. Notably, diffuse illumination of hearts where the ChR2 transgene was delivered to several ventricular sites resulted in electrical synchronization

This is fantastic! The author Lior Gepstein says:

Our work is the first to suggest a non-electrical approach to cardiac resynchronization therapy, Gepstein said. Before this, there have been a number of elegant gene therapy and cell therapy approaches for generating biological pacemakers that can pace the heart from a single spot. However it was impossible to use such approaches to activate the heart simultaneously from a number of sites for resynchronization therapy.

Read more here:
Stem Cell Assays Reproducible Research on Stem Cells

This entry was posted on July 1, 2015 at 3:45 am and is filed under Stem Cell Negative. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |