Month: April 2023

#Take6ForPD: CIRM Board member David Higgins shares why Parkinson’s awareness matters

/ Katie Sharify / 1 Comment

April is Parkinson’s Awareness Month and the California Institute for Regenerative Medicine (CIRM) is joining the campaign to #Take6ForPD—take 6 minutes to raise Parkinson’s awareness and highlight our commitment to funding research for the disease. 

In addition to highlighting our commitment to funding research for Parkinson’s Disease, we’re also giving patients and patient advocates the opportunity to share their stories and experiences.

In this blog, CIRM Board Member David Higgins shares why spreading awareness of Parkinson’s is important to him.

Dr. David R. Higgins grew up in West Virginia with four siblings. He earned his Ph.D. in molecular biology and genetics from the University of Rochester, followed by a postdoctoral fellowship at the National Cancer Institute before moving to San Diego in 1990 to work in biotech.

In December 2011, Higgins was diagnosed with Parkinson’s. Since then, he has become an advocate for people with Parkinson’s and their caregivers. He uses his personal experiences to guide this advocacy work, focused on improving quality of life issues through education, support, training, networking and promoting increased research funding to find a cure.

He currently serves as the Parkinson’s patient advocate member of CIRM’s governing board.

What is your personal connection to Parkinson’s Disease (PD)?

For me, Parkinson’s disease (PD) is a family legacy. My maternal grandmother suffered from PD and participated in some of the earliest Levodopa clinical trials held in the late 1960s.

In early 2014 my mother died with Lewy Body Dementia. My maternal uncle and great uncle also suffered from PD. In December 2011, I was diagnosed with PD completing the Parkinson’s Trifecta: care partner, scientist, and patient.

Additionally, in my biotech career, I worked for a company that is developing a gene therapy product to treat PD. This product currently is in human clinical trials in Europe.

What do you want people to know about PD?

PD is the second most common neurodegenerative disease, behind only Alzheimer’s disease in prevalence and with a rapidly growing incidence. Currently there are about 1 million people in the US diagnosed with PD.

PD is unique in that we know a lot about what the underlaying defect is. And there are likely several factors that influence whether you are going to develop PD. The most common model is a genetic predisposition to PD that exists in some people. If a person with a predisposition to PD is exposed to certain environmental factors, then development of the disease follows.

However, not every person who follows this pattern will develop PD. There is a familial form of PD that accounts for about 10% of the diagnosed cases. These tend to manifest earlier in life — in the patient’s 20s rather than the usual 60s.

There are a number of human genes known to be involved. However, identification of all of the so-called PD genes is not exhaustive. For example, there are about 80+ mutations that have been associated with the development of PD in humans. So clearly the situation is more complex.

Why is it important to you to raise awareness about PD? 

It is critical that the stake holders, PD patients, California citizens, understand the impact that new treatments and even a cure for PD could have:

  • Social — Quality of life, decrease pain and suffering
  • Medical — Decrease huge medical costs to society to support and care for chronic illnesses
  • Economic — Positive impact on the economy by maintaining and increasing productivity of a sector of society that is at its peak productivity
  • Diagnosis — Fast accurate differential diagnoses of PD and related movement disorders such as MSA and SMA.
Higgins on a recent scuba diving trip

Why is it important to continue investing in all PD research and treatments — such as those that improve certain symptoms — and not just research that aims at finding a cure?

The goal in PD research as well as any new therapy is that new and effective treatments are just as important—and likely more within reach—than the elusive Holy Grail cure. Effective treatments that slow progression or work to treat chronic symptoms are incredibly beneficial to patients and the kinds of treatments that patients ask about most.

Why is it important that an agency like CIRM continues to invest in regenerative medicine research and treatment for PD?

Basic research and clinical development both need funding to understand the disease better, as well as to design testing in humans. Developing new therapeutics for PD and the research effort that supports it will require a huge multi-discipline collaborative effort and the financial funding to enable it. CIRM has the resources to put together the collaborative effort needed and is uniquely positioned to build appropriate collaborations needed.

To date, CIRM has invested more than $60 million in helping research for Parkinson’s progress from a basic or Discovery level through clinical trials. To learn more about CIRM’s investments in finding treatments for the disease, visit this page on our website.

CIRM commits $60 million for Parkinson’s research, joins #Take6ForPD awareness campaign 

/ Esteban Cortez / 1 Comment

April is Parkinson’s Awareness Month and the California Institute for Regenerative Medicine (CIRM) is joining the campaign to #Take6ForPD—take 6 minutes to raise Parkinson’s awareness and highlight our commitment to funding research for the disease. 

What is Parkinson’s Disease? 

Parkinson’s disease (PD) is a neurodegenerative disease that affects nearly 1 million people in the United States and more than 10 million people around the world. In California, more than 106,000 people live with PD. 

Symptoms of PD include tremors, slow movement, muscle rigidity, balance issues and lack of facial expressions. Parkinson’s disease occurs when the neurons or nerve cells in the portion of the brain that controls movement die off. These neurons send signals by releasing a chemical called dopamine and are referred to as dopaminergic neurons.  

Parkinson’s is the second-most common neurodegenerative disease after Alzheimer’s disease. No cure exists for the disease. 

CIRM’s Commitment to Funding Parkinson’s Research

CIRM remains committed to funding regenerative medicine research—including stem cell and gene therapy research—for PD treatments to improve quality of life for patients.  

To date, CIRM has invested more than $60 million in helping research for PD progress from a basic or Discovery level through clinical trials. 

This year, CIRM awarded $4 million to a late-stage preclinical project by Ryne Bio aiming to improve treatment for idiopathic PD. In the proposed stem cell therapy, investigators at Ryne Bio are aiming to deliver dopamine-producing cells to replace the lost neurons to the brain of PD patients to restore or improve motor function. 

“Receiving a Parkinson’s diagnosis is a difficult and life-changing event,” says Ryne Bio CEO Nick Manusos. “This Parkinson’s Awareness Month, we honor patients and their loved ones by reflecting on the clinical progress made so far, and looking forward to a brighter, healthier future.” 

Other regenerative medicine approaches CIRM has funded to target PD involve understanding the disease and looking for new drugs to treat it.  

CIRM grantees have taken skin cells from people with Parkinson’s disease, reprogrammed them back to an embryonic-like state, turning them into the kind of stem cell that can be transformed into any other cell in the body, then coaxing those cells to become dopaminergic neurons that are lost to the disease.  

Those cells showed signs of the disease in the lab dish and were distinctly different from the same cells created from people without PD.  

Another approach involves using CRISPR gene editing technology to reduce the levels of a toxic protein called alpha synuclein, which builds up in the dopaminergic brain cells affected by PD. 

Overall, CIRM has awarded more than 30 grants aiming to better understand the disease and find regenerative medicine-based treatments.  

“CIRM’s commitment to funding research for Parkinson’s is unwavering,” says CIRM President and CEO Maria Millan. “Our goal is to fund the most promising regenerative medicine research to support finding the best treatments to fight this devastating disease that affects millions.” 

CIRM’s Task Force on Neuroscience & Medicine 

As part of its Strategic Plan, CIRM has also launched a Task Force on Neuroscience and Medicine to set a plan for the $1.5 billion allocated for the support of research and the development of treatments for diseases and conditions of the brain and central nervous system. These include Parkinson’s, Alzheimer’s, stroke, dementia, epilepsy and other diseases and conditions. 

The Task Force will host public meetings to gather community input to identify potentially high-impact opportunities in basic neuroscience, neurodegenerative disease, neuropsychiatric disease, neurodevelopment, and normal brain aging.   

The goal of the task force is to provide final recommendations to CIRM and its governing board within six months of inception. 

To see a listing of upcoming and past Task Force meetings, visit our website. 

Meet CIRM’s growing world class team 

/ Katie Sharify / Leave a comment

The California Institute for Regenerative Medicine (CIRM) continues to build its world class team to deliver the full potential of regenerative medicine to the people of California and around the world. CIRM is rebuilding and expanding its team to meet new challenges and advance the mission of the Agency. 

Rafael Aguirre-Sacasa and Koren Temple-Perry join the growing ranks of new team members that CIRM has hired since the passage of Proposition 14 in November 2020. We recently shared an announcement welcoming them to the team here.

Among the new hires is Emily Reyes, who joins CIRM as a project manager in the Medical Affairs and Policy team. She earned her MPH from California State University, Fullerton and her BS degree from Oregon State University. Emily comes to CIRM from UCSF, where she was a clinical research coordinator overseeing several research studies with responsibilities including patient coordination, data collection and manuscript preparation. 

Marivel De La Torre also joins the Medical Affairs and Policy team as a project manager and is responsible for overseeing critical activities as CIRM continues to build and execute the medical affairs strategic plan. Marivel comes to CIRM from Sacramento, where she has over 18 years of State service managing numerous projects within the Department of Transportation, Department of Healthcare Services, Department of Corrections and Rehabilitation, and California Air Resources Board. 

Ben Chau is CIRM’s new Associate Director of IT. He has worked in management for over 13 years and brings a wide range of skills and leadership to this role. Before joining CIRM, he was the IT Director for Asian Health Services where he put forward IT strategic plans to ensure systems were up to date with technology and security compliance.  

James (Jim) Campanelli, PhD joins CIRM as a Senior Science Officer in Therapeutics Development. Prior to CIRM, Jim was employed at Q Therapeutics where he directed research and development efforts for past 17 years.Jim received a PhD in Biological Sciences from Stanford and was an assistant Professor of Biochemistry at University of Illinois at Urbana-Champaign. 

Elizabeth (Liz) Noblin, PhD joins the Review team as a Senior Science Officer. Before joining us at CIRM, Liz was a program and project manager at 23andMe, where she supported both the Research and Therapeutics divisions. Liz received a PhD in Genetics from Yale and completed her postdoc with Dr. Anne Brunet at Stanford.  

Charlie Shaw, PhD is a Senior Science Officer and the newest member of the Business Development & Alliance Management team. Charlie joins CIRM after more than 12 years’ experience in academic technology licensing offices. His most recent role was at the Moffitt Cancer Center where he was the Associate Director of Licensing. Charlie has a PhD in Molecular Genetics and Microbiology from Duke University, post-doctoral training in genetic medicine from University of Chicago, BS in Biology from Davidson College and is currently obtaining his MBA from East Carolina University. 

Scott Tocher, CIRM’s former General Counsel, returned to the agency last November as a retired annuitant to help fill CIRM’s workload gap in the legal division, and rejoined as a permanent employee as Senior Director of Board Governance in February. Scott originally came to CIRM’s legal department in 2005, assisting in the development of CIRM’s policies, programs and regulations through 2019.  

Janie Byrum, PhD, joins the Scientific Programs team as a Science Officer. In her most recent role as an R&D engineer at CZ Biohub, she led and built imaging pipeline projects to interrogate the impacts of viral infection on cell function as part of the Infected Cell and Quantitative Cell Science departments. Janie has a PhD in Biomedical Sciences from the University of New Mexico Health Sciences Center and a post-doctoral training in immunology from Columbia University Irving Medical Center. 

Chan Lek Tan, PhD, a Science Officer in CIRM’s Scientific Programs team, is an experienced and inquisitive scientist with a background in neurodevelopmental and neurodegenerative diseases. In his most recent roles as Principal Scientist at Genentech and BridgeBio, Chan furthered his expertise in pre-IND development of small molecule and RNA-based therapeutics for neuro-based indications. Chan has a PhD in Molecular and Cellular Neuroscience from The Rockefeller University, New York where he studied under Dr. Paul Greengard.  

Gemma Domingo joins CIRM as Sr. Finance Officer.  She attended the University of the East in Manila and earned a Bachelor of Science degree in accounting. In 1990, she immigrated to the U.S. to join her family and joined state service in 1998. Throughout her time with state service, Gemma previously worked at the Department of Parks and Recreation, Department of Child Support Services, and California Office of Emergency Services. 

Leyla Najmi, a seasoned Senior Human Resources (HR) leader and business partner, joins CIRM as the Director of HR. Leyla has used her strong HR acumen and a blend of experience in various positions within private and public sectors to affect positive change in the organizations where she has worked.  Effectively balancing big picture thinking and strategic planning with hands-on execution has allowed Leyla to combine decisiveness, strong business acumen and formal education to achieve objectives both professionally and personally. 

“We are delighted to have these talented individuals join the CIRM team,” says CIRM President and CEO Dr. Maria Millan. “They all bring unique qualifications and critical skills to support our strategic plan and advancement of CIRM’s mission to accelerate world class science for California and the world. We look forward to working with all of them.” 

CIRM welcomes two additions to its leadership team

/ Katie Sharify / 1 Comment
Left to right: Rafael Aguirre-Sacasa, General Counsel and Koren Temple-Perry, Senior Director of Marketing Communications

The California Institute for Regenerative Medicine (CIRM) continues to build its world class team to deliver the full potential of regenerative medicine to the people of California and around the world. 

CIRM is pleased to welcome two new members to its leadership team, including Rafael Aguirre-Sacasa as General Counsel and Koren Temple-Perry as Senior Director of Marketing & Communications. 

Rafael has over 25 years of experience in corporate law and joins CIRM after seven years at Standard BioTools (formerly Fluidigm Corp). There, he provided worldwide commercial, strategic and transactional legal support for all functions of the NASDAQ-listed life science company.  
 
Prior to this role, he held a variety of management positions and provided legal support for a wide range of commercial, intellectual property and corporate matters at Teradici Corporation, PMC-Sierra, Inc., Autodesk, Hyperion solutions, Grupo Financiero and Xilinx Inc. 
 
Rafael received his bachelor’s degree in history and government from Dartmouth College and law degree from the University of California-Hastings. As CIRM’s new General Counsel, Rafael will support the President, Board (ICOC), management and working groups on all legal matters affecting the agency. 
 
“Joining the Institute as General Counsel is not just a job to me,” says Raphael. “It’s an opportunity to be part of an organization that is changing lives. I am enthusiastic about helping to implement CIRM’s strategic plan and contributing to their mission of accelerating stem cell treatments to patients in need, especially those in underserved and underrepresented communities.” 
 
The agency also welcomes Koren Temple-Perry as Senior Director of Marketing Communications.  
 
Koren joins CIRM after spending the last 15 years translating complex scientific information into compelling content for diverse audiences.  
 
As a former journalist, she has worked extensively in the areas of patient and community health education, health advocacy, and scientific communications for hospitals, research institutes, and healthcare organizations.  
 
Prior to joining CIRM, she founded Temple Communications LLC, which specializes in scientific copywriting and marketing communications for public health organizations.  
 
She served as consulting communications director for March for Science, where she led the media and communications campaign for the global marches. She spent many years as a communications writer and digital marketing manager at academic medical centers throughout New York.  
 
Koren earned her master’s degree in journalism from the S.I. Newhouse School of Public Communications at Syracuse University and a bachelor’s degree in communications from Santa Clara University. Her passion for improving health equity led to her appointment as District 5 Commissioner to the Alameda County Public Health Commission and to the Board of Directors for Jamal’s Helping Hands, a non-profit dedicated to helping families of color navigate rare diseases.  
 
“I am truly honored to lead CIRM’s marketing communications team as the Agency aims to bring promising stem cell and gene therapy science to diverse communities,” says Koren. “I see incredible potential in building meaningful partnerships and elevating our outreach strategies to amplify cutting-edge treatments so that all Californians have equitable access to them. I am eager to make a positive impact in my new role and contribute to CIRM’s overall success.”   
 
“We are delighted to have these talented individuals join the CIRM team,” says CIRM President and CEO Dr. Maria Millan. “They all bring unique qualifications and critical skills to support our strategic plan and advancement of CIRM’s mission to accelerate world class science for California and the world. We look forward to working with all of them.”

CIRM invests $2.7 million in research to develop stem cell-based tendon tissue for shoulder injuries

/ California Institute for Regenerative Medicine (CIRM) / 1 Comment

The California Institute for Regenerative Medicine (CIRM) has awarded a $2.7 million research grant to Scripps Health to investigate the use of stem cells to engineer rotator cuff tendons in the lab that can then be used to repair common shoulder injuries.  

The rotator cuff is a group of four muscles and their attached tendons, which hold the upper arm in place in the shoulder socket. The rotator cuff attaches the humerus to the shoulder blade and helps to lift and rotate the arm.  

Most rotator cuff tears result from natural degeneration of the tendon over time, frequently causing pain and possibly limited use of the arm. As the condition progresses and worsens, the shoulder can become weaker, more painful, and less useful. 

Image credit: Cedars-Sinai

The use of pluripotent stem cells to develop lab-grown tendon tissue to repair rotator cuffs could benefit older patients with significant tears due to age-related degeneration.  

“For this group of patients, the failure rate for conventional repair surgery is about 40%,” said Dr. Darryl D’Lima, director of orthopedic research for the Shiley Center for Orthopaedic Research and Education (SCORE) at Scripps Clinic and the initiative’s lead investigator. “This is a common injury, and as our population ages, there’s a pressing need to find new solutions.” 

Annually, over 100,000 Californians sustain tendon injuries, the majority of which require surgical repair. Failure rates for shoulder rotator cuff tendon repairs vary between 20% and 90%. Failed rotator cuff tendons lead to early development of osteoarthritis, for which the only effective treatment is total joint replacement. Preventing disability offers significant socioeconomic benefits and reductions in healthcare costs also are likely to be significant. 

Image credit: Scripps

The funding from CIRM will support the initiative’s three-year discovery phase at Shiley Center for Orthopaedic Research and Education (SCORE) on Torrey Pines Mesa. Researchers plan to develop a series of lab tests called assays to assess and measure the makeup of the tendon cells and develop the tendon material in the lab.

SCORE researchers intend to explore the possibility of transforming pluripotent stem cells into tendon-like cells, which would be embedded into a scaffold structure of “electrospun” fibers and grown into tendon tissue using bioreactors.  

The lab-engineered tendon tissue also would be stretched to help stimulate the cells to produce more tissue, and to help align the electrospun fibers so they mimic the intricate alignment of fibers in native tissue. Lining up the tendon fibers in the same direction that stretching occurs is needed to resist tensile force, or the stretching force experienced during motion. 

To learn more about the California Institute for Regenerative Medicine, visit our website.

Apply Now! Discovery Stage Funding + Webinar on April 7

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The California Institute for Regenerative Medicine (CIRM) is pleased to announce the opening of our next round of Quest Awards (DISC2) for discovery stage research.

The purpose of the Quest Awards is to promote discovery of promising new stem cell-based and/or gene therapy technologies that could be translated to enable broad use and ultimately, improve patient care. The expected outcome, at the end of the award, is a candidate therapeutic or technology that can immediately progress to translational stage activities.

Applications are due May 2nd, 2023 at 2:00 PM PDT. Please visit this page on CIRM’s website for application instructions.

CIRM encourages the submission of proposals to advance the treatment and/or understanding of central nervous system (CNS) disorders.  

Important Update: Please note that the DISC2 Program Announcement (PA) has been updated since the last round of applications. In addition, CIRM has implemented new guidelines and requirements for data sharing in our Discovery programs.

Please read the new PA and review the Information for Applicants page on our website before submitting your application. Interested applicants may contact discovery@cirm.ca.gov with any additional questions.

Sign Up for an Informational Webinar

Have questions about this funding opportunity? Join the CIRM Scientific Programs team for an informational webinar.

This webinar provides an opportunity for prospective applicants to learn more about CIRM’s DISC2 Quest Awards and how to assemble a competitive application. Click here to register for the webinar.

The CIRM team will elaborate on DISC2 eligibility requirements, the application and review process, and how this program fits into CIRM’s R&D Pillar initiatives as well as our Strategic Plan and Mission.

The webinar includes a half-hour presentation by CIRM staff followed by a half-hour for Q&A.

We look forward to your applications!