California Institute for Regenerative Medicine

At UCLA Health California Center for Rare Diseases, “rare” represents more than 7,000 conditions affecting 3 million Californians. Through multidisciplinary care, genetic diagnosis, registries, clinical trials, and novel therapies, UCLA is advancing rare disease research and treatment in real time.

Recent milestones reflect that momentum, from the launch of the nation’s first Multidisciplinary Bloom Syndrome Clinic to advances in gene therapy for Duchenne muscular dystrophy and other genetic conditions. Among these efforts is promising investigational gene therapy research led by Dr. Donald Kohn, whose work, funded by CIRM, is helping expand what may be possible for families affected by rare diseases.

Stories like these really highlight how important it is to keep investing in research that translates into real treatments, and how working together in teams can help bring new therapies to patients.

Read more: https://bit.ly/4tUh4Z5

UCLA research prioritizes hard-to-treat patients through rare disease innovation At the UCLA California Center for Rare Diseases (CCRD), “rare” is a word that includes over 7,000 conditions and affects 3 million Californians. With hundreds of thousands of patients and families touched by rare diseases nationwide, CCRD, directed by Stanley Nelson, MD, has leveraged UCLA’s l...

How do we get better therapies to patients faster?

On the Sanford Stem Cell Institute's reGEN Podcast, City of Hope's Dr. Leo Wang shares how the CIRM Alpha Clinics Network is helping expand access to cutting-edge therapies across California, not just in major research hubs.

He highlights the network’s collaborative model, its focus on implementation and dissemination, and the importance of building infrastructure to enable more patients to access life-saving treatments closer to home.

Listen to the full episode:

Novel Therapeutics and Discoveries | Leo D. Wang, MD, PhD Our conversation with Leo D. Wang, MD, PhD, includes unique insight...

Like many at CIRM, Maria Gonzalez Bonneville's work is personal.

CIRM's vice chair joined the agency in 2011, not long after her father's Parkinson's diagnosis. The job offered her a chance to combine her deep commitment to public service in California with her desire to support this voter-supported effort to accelerate treatments and cures for conditions like her father's.

Maria's role at CIRM now extends to serving as Chair of CIRM's Accessibility and Affordability Working Group, where she's helping ensure that the potentially life-changing therapies that come from CIRM-supported research and clinical trials are accessible to all Californians who need them.

CIRM supports stem cell and genetic research and clinical trials into treatments for hundreds of different conditions, including Parkinson's disease, which affects about a million people in the United States alone.

Maria's work reflects a broader commitment that her work and CIRM's aren't just about finding breakthroughs but about ensuring that patients can access them.

"It isn't just about funding the science," she says. "It's about making sure it gets to people."

Read Maria's story: https://bit.ly/3QZj052

It is hard to create or identify a life-saving therapy if you are not trained to recognize one. James DeKloe, PhD, a distinguished professor of biotechnology and biomanufacturing at Solano Community College, knows this firsthand, and it is one reason why he spoke so passionately about the importance of the Creating Opportunities through Mentorship and Partnership Across Stem Cell Science (COMPASS) program at Solano in a recent presentation for CIRM’s "Closer to Cures" speaker series.

“I have saved dozens of lives in this year and a half,” said Joel Saenz, a graduate of the COMPASS program at Solano Community College who now works at the CIRM-funded UC Davis Good Manufacturing Practice Facility, which manufactures cell, gene, and drug products, including CAR-T cell therapies for cancer.

“Whether it's someone's mom, dad, brother, sister, whoever it may be, I know that I am able to help with that,” said Joel. “And if it wasn't for CIRM, I can't say that I would have that opportunity and be in the position that I'm in today.”

16 California colleges and universities received COMPASS program grants. This education program pairs undergraduate students with mentors who are experts in stem cell research and related fields to help students gain hands-on experience in the lab.

Students in Solano’s COMPASS program learn about the sciences, work in the lab, and participate in outreach opportunities, including volunteering with local K-12 and adult schools and the NMDP. They also learn about FDA regulations and the business part of the industry.

“Our goal is to make sure that every kid knows that if they want to pursue something more, here is a great opportunity for them to do it, and the lives that you save won't just be the patients that you serve,” said another Solano COMPASS trainee alum, Michael Silva, EdD.

Read the full recap of the presentation on our blog: https://bit.ly/4uivoeW

Hear how California leads the way in scientific research funding for vital stem cell and gene therapies on the Podcast.

This episode features CIRM's President and CEO, Jonathan Thomas (JT). Beyond highlighting California's unique approach to supporting research, JT highlights CIRM's Alpha Clinics Network, which gives the people most in need of these new therapies access to important clinical trials across the state.

Listen to the full podcast episode: https://bit.ly/4u84YfQ

A Conversation with CIRM’s President and CEO | Jonathan Thomas, PhD, JD This episode features our conversation with Jonathan Thomas, PhD, J...

Helping the people of California live healthier lives is a core part of Maria Gonzalez Bonneville’s work as vice-chair of the CIRM’s governing board and as chair of CIRM’s Accessibility and Affordability Working Group (AAWG).

A critical part of her work is crafting and guiding the AAWG's recommendations to CIRM’s governing board to increase the affordability and accessibility of CIRM-funded treatments and clinical trials.

“This is top of mind for us now across the organization,” said Bonneville. Our goal “isn't just to fund the science, it is to fund the science because it needs to get to people.”

Creating a framework to help deliver newly developed stem cell therapies to the people of California is an important part of CIRM’s mission and legacy as a founder in the field.

“CIRM has been an important part of stem cell research for over 20 years,” said Bonneville. “It helped create an ecosystem in California that otherwise would not have existed… California is amazing. We have the economy and the infrastructure to be able to support something like this. So, if it was going to happen anywhere, it was going to happen here."

Read more of Maria's story: https://bit.ly/3QZj052

In 2021, CIRM helped fund a clinical trial that involved an experimental stem cell treatment for a frequently paralyzing form of spina bifida.

This clinical trial, called the CuRe Trial, is co-led by Diana Farmer, MD, the surgeon-in-chief at UC Davis Health Children’s Hospital and the chief of pediatric surgery at Shriners Children's Northern California.

Recently, Farmer provided an update on the progress of CIRM-funded clinical trials for an experimental stem cell therapy to treat severe forms of spina bifida.

Currently, the CuRe trial is enrolling participants in a clinical trial that will follow the progress of infants treated with the stem cell therapy from birth to six-years of age. This study will help the researchers better understand the stem cell therapy’s safety and long-term effects.

“We don't [yet] know that wiggling your toes when you're born means you're going to walk when you're two-and-a-half,” said Farmer. “But we are, incredibly optimistic that we might be able to get all these kids out of a wheelchair. And that would be pretty exciting.”

Read the full story: https://bit.ly/496XIIGhttps://bit.ly/496XIIG