The Bridges program is one of CIRM’s educational initiatives that gives students the chance to take coursework at California state schools and community colleges while conducting stem cell research at top universities and industry labs. The goal is to train the next generation of stem cell scientists by giving them the skills and experience they need to succeed.

The annual Bridges conference is the highlight of the program and a celebration of the students’ achievements. It gives students a platform to showcase the research they’ve worked on over the past year and to network with fellow students and scientists.

Bridges students also took part in a networking pitch event focused on stem cell research.

CIRM opened the conference with a fast‑paced “Stem Cell Pitch” activity. Students were divided into groups, assigned one of the four questions above, and given ten minutes to introduce themselves, discuss the question, and choose a spokesperson. Despite the tight deadline, each team delivered a polished, thoughtful 30‑second pitch. Their answers were unique and inspiring to both students and scientists in the audience.

Getting to the clinic and into patients

The bulk of the Bridges conference featured student poster presentations and scientific talks by leading academic and industry scientists. The theme of the talks was getting stem cell research into the clinic and into patients with unmet medical needs.

Here are a few highlights and photos from the talks:

On the clinical track for Huntington’s disease

Leslie Thompson, a professor at UC Irvine, discussed her latest Huntington’s disease (HD) research She described it as a “race against time.” HD is a fatal progressive neurodegenerative disorder with no cure. Her lab is moving toward the clinic with human embryonic stem cell‑derived neural stem cells, which they transplant into mouse models of HD. So far, the transplanted neural stem cells have produced encouraging results, including improved behavioral and motor function and reduced buildup of toxic mutant Huntington protein in the mice’s nerve cells.

Leslie noted that because the transplanted stem cells are GMP‑grade her team has a clear path toward testing their disease‑modifying potential in clinical trials. Before reaching that stage, however, they must complete the required regulatory steps with the US Food and Drug Administration and run additional studies to assess safety, determine proper dosage in other mouse models, and evaluate other potential GMP‑grade stem cell lines.

Gene therapy for SCID babies

Morton Cowan, a pediatric immunologist at UC San Francisco, followed Leslie with a talk on his efforts to move gene therapy for SCID—severe combined immunodeficiency—out of the lab and into the clinic. SCID, often called bubble‑baby disease, is caused by the absence of a functioning immune system. Babies with SCID lack normal T and B cell function and typically die from infections or related complications within their first year of life.

Morton explained that the gold‑standard treatment for SCID—hematopoietic, or blood stem cell, transplantation—is only safe and effective when a patient has an HLA‑matched sibling donor. Many patients don’t have this option and face life‑threatening risks from transplant rejection, or graft‑versus‑host disease. To address this, Morton and his team are using gene therapy to correct SCID patients’ blood stem cells and transplant them back so the patients can produce healthy immune cells.

They are now developing a gene therapy for a particularly difficult form of SCID caused by a deficiency in Artemis, a protein essential for immune‑system development and DNA repair. His group is currently conducting the preclinical studies needed to launch a gene therapy clinical trial for children with Artemis‑SCID.

Treating spinal cord injury in the clinic

Casey Case, Senior VP of Research and Nonclinical Development at Asterias Biotherapeutics, gave an update on the CIRM-funded clinical trial for cervical (neck) spinal cord injury (SCI). They are currently testing the safety of transplanting different doses of their oligodendrocyte progenitor cells (AST-OPC1) in a group of SCI patients. The endpoint for this trial is an improvement in movement greater than two motor levels, which would offer a significant improvement in a patient’s ability to do some things on their own and reduce the cost of their healthcare. You can read more about these results and the ongoing study in our recent blogs (here, here).

Opinion: Scientists should be patient advocates

David Higgins gave the most moving speech of the day. He is a Parkinson’s patient and the Patient Advocate on the CIRM board and he spoke about what patient advocates are and how to become one. David explained how, these days, drug development and patient advocacy is more patient oriented and patients are involved at the center of every decision whether it be questions related to how a drug is developed, what side effects should be tolerated, or what risks are worth taking. He also encouraged the Bridges students to become patient advocates and understand what their needs are by asking them.

“As a scientist or clinician, you need to be an ambassador. You have a job of translating science, which is a foreign language to most people, and you can all effectively communicate to a lay audience without being condescending. It’s important to understand what patients’ needs are, and you’ll only know that if you ask them. Patients have amazing insights into what needs to be done to develop new treatments.”

Bridging the gap between research and patients

The Bridges conference is still ongoing with more poster presentations, a career panel, and scientific talks on discovery and translational stem cell research and commercializing stem cell therapies to all patients in need. It truly is a once in a lifetime opportunity for the Bridges students, many of whom are considering careers in science and regenerative medicine and are taking advantage of the opportunity to talk and network with prominent scientists.

If you’re interested in hearing more about the Bridges conference, follow us on twitter (@CIRMnews, @DrKarenRing, #CIRMBridges2016) and on Instagram (@CIRM_Stemcells).