Cedars-Sinai receives California Institute grant to develop new technique for specific diseases

A team of scientists from the Cedars-Sinai Regenerative Medicine Institute has been awarded a $1.9 million grant from the California Institute for Regenerative Medicine to fund development of a new technique to aid pharmaceutical discoveries for specific diseases.

Additionally, another team from the Cedars-Sinai Department of Surgery will share a $1.5 million grant with a medical technology company aiming to develop a new imaging system.

The Tools and Technology grants from the California stem cell agency support the early-stage development and evaluation of innovative tools and technologies to overcome current roadblocks in translational stem cell research.

"Scientists have to first invent the tools so that regenerative medicine can bring patients and their families hope, relief, and perhaps one day, cures," said Clive Svendsen, Ph.D., director of the Cedars-Sinai Regenerative Medicine Institute. "These grants allow us to continue advancing stem cell science toward the goal of a new era in treatment, especially for diseases that currently have no cure or standard treatment."

Svendsen's team, led by Dhruv Sareen, Ph.D., was granted $1.9 million to invent a new pharmaceutical discovery process that employs pluripotent stem cells in an effort to find a pharmaceutical treatment for Spinal Muscular Atrophy, a neuromuscular condition that is one of the most common lethal genetic diseases in children.

Children born with Spinal Muscular Atrophy harbor a genetic mutation in the SMN1 gene, which doesn't allow their motor neurons to manufacture critical protein (SMN) needed for survival. As motor neurons die, patients become increasingly paralyzed. Another gene, called SMN2, manufactures the necessary protein, but doesn't make enough to compensate the gene deficit and stop the disease progression.

Svendsen's team aims to discover a pharmaceutical drug candidate compound that will stimulate SMN protein development in motor neurons. To that end, they will convert patient skin cells to powerful induced pluripotent stem cells and then motor neurons with SMN1 mutation, but still containing the intact SMN2 gene.

Then, the team will begin testing compounds to see if any help increase motor neurons' production of the SMN protein through the SMN2 gene.

"Pharmaceutical and biotech companies have comprehensive libraries of chemically diverse compounds, some of which potentially could be developed into drug treatments to help SMA patients," Sareen said. "We will be able to test these drug libraries relatively quickly using our high-content screening approach. We hope to identify novel compounds that target the SMN2 gene specifically in patient motor neurons. Any drug lead "hits" we get can be quickly repurposed and pursued, bringing us so much closer to finding a treatment for this debilitating childhood disease."

Another team led by Dan Gazit, Ph.D., DMD and Zulma Gazit, Ph.D. is working with Gamma Medica-Ideas, Inc., which was granted $1.5 million to develop a new imaging technology that for the first time would enable doctors to monitor stem cell treatments in patients in real time.

Their project is to combine magnetic resonance imaging (MRI), which uses magnetic field and radio waves to create images of organs and tissues, with single-photon emission computerized tomography (SPECT), which uses a radioactive substance and a special camera to produce three-dimensional images. The scientists believe the new imaging system will be most applicable at first for muscle, tendon, ligament, cartilage and intervertebral disc regeneration. In the future, however, the new imaging system is likely to be developed to monitor stem cell treatments for other tissues, particularly in the heart and brain.

"A common challenge facing the use of stem cells to regenerate damaged tissue is that we have no immediate way of knowing whether the cells are, indeed, building new tissue," said Zulma Gazit. "By combining these technologies into a new, non-invasive hybrid system, we will be in a position to follow healing."