Month: June 2024

Supporting a CAR-T cell trial for kidney cancer

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment
Photo Credit: Allogene Therapeutics. Image of the ALLO-316 allogeneic CAR-T cell therapy.

The California Institute for Regenerative Medicine (CIRM) awarded $15 million to Dr. John Le Gall and his team at Allogene Therapeutics. This funding will support the development of ALLO-316, an innovative anti-CD70 CAR-T cell product designed to treat metastatic clear cell renal cell carcinoma (ccRCC), the most common type of kidney cancer worldwide. 

Metastatic renal cell carcinoma (RCC) presents significant challenges with limited treatment options, especially for patients who have not responded to checkpoint blockers and targeted therapies. The five-year survival rate for advanced kidney cancer is less than 17%, highlighting the urgent need for new therapeutic approaches. 

ALLO-316 is an allogeneic CAR-T cell therapy that targets CD70, a protein highly expressed on ccRCC tumors. By focusing on CD70-positive cancer cells, this therapy offers a promising new approach to treating kidney cancer. Initial data from the Phase 1 TRAVERSE trial, presented at the American Association for Cancer Research (AACR) 2023 Annual Meeting, showed encouraging response rates and early anti-tumor activity, suggesting deepening responses over time. 

Dr. Zachary Roberts, Executive Vice President of Research & Development and Chief Medical Officer of Allogene, emphasized the transformative potential of CAR-T therapy for solid tumors.  

“CAR-T has revolutionized the treatment of hematologic malignancies, and we believe this CIRM award validates the remarkable progress we have made in our TRAVERSE trial,” said Dr. Roberts. “ALLO-316 has shown significant promise for patients with advanced RCC who have exhausted standard therapies. We are grateful for the support from CIRM and look forward to advancing this trial.” 

The Phase 1 TRAVERSE trial aims to assess the safety, tolerability, and preliminary efficacy of ALLO-316 in patients with advanced RCC. The grant from CIRM will facilitate the completion of this phase, including expanding clinical sites to enhance patient access and conducting translational and clinical analyses to inform the Phase 2 regimen. 

Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM, highlighted the potential impact of this research.  

“This clinical study has the potential to demonstrate the value of Chimeric Antigen Receptor (CAR) T cell therapy in solid cancers such as kidney cancer, which has a high unmet medical need,” said Dr. Creasey. 

A cornerstone discovery during the initial phase of the TRAVERSE trial is the Dagger® technology, which selectively eliminates CD70-positive, alloreactive host immune cells. This breakthrough helps delay or prevent the patient’s immune system’s premature rejection of AlloCAR T cells, enhancing the therapy’s effectiveness. 

The development of ALLO-316 represents a significant step forward in the fight against metastatic kidney cancer, offering hope to patients and their families. 

CIRM’s $31 million boost for cancer and eye disease research

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment

The California Institute for Regenerative Medicine (CIRM) recently approved nearly $31 million in funding, further cementing our commitment to advancing the field of regenerative medicine. This substantial investment aims to drive clinical research targeting various cancers and eye diseases. 

CIRM’s dedication to fostering innovative treatments is demonstrated through its clinical program, which funds eligible stem cell and gene therapy-based projects at all stages of clinical trial development. With these new awards, CIRM’s clinical portfolio now includes 106 clinical trials. 

During CIRM’s April Application Review Subcommittee (ARS) meeting, the following vital projects were approved for funding: 

Application    Program Title    Principal Investigator/Institution  Amount    
CLIN2-15282  RPESC-RPE Therapy for dry Age-related Macular Degeneration    Stern, Jeffrey H – Luxa Biotechnology  $4,009,675  
CLIN2-15311  A Phase I/IIa Study to Evaluate the Efficacy of a Gene Therapy with Standard of Care Therapy in Newly Diagnosed High Grade Glioma    Kasahara, Noriyuki – UCSF  $11,807,220  
CLIN2-15343  A Phase 1B Study Evaluating the Safety and Efficacy of an Allogenic Cell Therapy in Subjects with Clear Cell Renal Cell Carcinoma (ccRCC)  Le Gall, John – Allogene Therapeutics  $15,000,000  

 
These projects are poised to make significant strides in developing new therapies that could revolutionize the treatment landscape for cancer and eye diseases. By supporting these cutting-edge research initiatives, CIRM continues to pave the way for breakthroughs in regenerative medicine. 

Stay tuned for updates on the progress of these exciting projects and learn how they are making a difference in healthcare by signing up for our monthly newsletter. 

Tackling sickle cell disease from within

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Photo Credit: Jaquell Chandler

Evie Junior was born with sickle cell disease, a life-threatening condition that affects around 100,000 Americans, most of them Black and Latino. People with sickle cell disease have blood cells that are shaped like a hook (or sickle) rather than smooth and round, which can create clogs, causing intense pain, organ damage, and stroke.  

By age 18, Evie experienced severe pain crises once or twice a month. In his mid-20s, he took part in a CIRM-funded clinical trial at UCLA, where researchers collected his blood-forming stem cells and, in the lab, inserted a gene that prevented the sickling of the cells. The modified cells were returned to his body to create a new blood supply.  

Since the treatment, Evie hasn’t had any pain crises requiring medications or trips to the emergency room. For World Sickle Cell Day (June 19), we asked Evie Junior why it’s important to be a champion for SCD.

CIRM: Why is raising awareness of sickle cell disease important to you?  
 
EJ: It’s important to inform doctors and medical professionals about sickle cell disease because it’s an invisible disease. There’s a lot of stigma and a wide variety of symptoms for sickle cell, which can be easy for medical professionals to disregard. It’s also important for people not in the medical field and family members of sickle cell patients because advocacy is important because sickle cell patients are at their worst and can sometimes not convey everything they’re going through. 
 
CIRM: What do you want people to know about sickle cell disease?  
 
EJ: Lifestyle choices, including diet, sleep, and stress levels, can cause a sickle cell crisis.  
 
CIRM: Why is it important to continue to invest in finding treatments for sickle cell disease?  
 
EJ: Having sickle cell disease can really tank your quality of life. It can force you to fall short of your goals and isolate you from your loved ones due to your family and friends not understanding what you’re going through.  

I don’t think anyone wants this for anyone, but kids shouldn’t have to put their future on hold or their goals. We want kids to shoot for the stars and never stop pursuing their goals. Many times, sickle cell got in the way of me pursuing my dreams. I truly don’t wish this on anyone. Sickle cell research is important because everyone deserves a chance.  
 
CIRM: CIRM funds many projects seeking to better understand sickle cell disease and to translate those discoveries into stem cell- and gene-based therapies. Why is it important that an agency like CIRM continues to invest in regenerative medicine research and treatment for sickle cell disease?  
 
EJ: Again, I think all agencies should invest in sickle cell research; the more, the better. The same with all diseases that affect people, everyone deserves a chance at providing themselves with a good quality of life. 

Video Credit: UCLA Broad Stem Cell Research Center

Learn more on how CIRM is championing change for sickle cell in California and how you can help spread SCD awareness at our website, cirm.ca.gov/sickle-cell.

How CIRM’s high school internship program inspired this scientist’s career path 

/ Esteban Cortez / Leave a comment
Photos courtesy of Rashi Ranjan

Each year, interns and trainees from the California Institute for Regenerative Medicine (CIRM) education and training programs celebrate achievements, whether that’s making new discoveries in their cell and gene therapy research or advancing to the next phase of their professional journey.  

This summer, Rashi Ranjan—a former SPARK high school intern—is celebrating her graduation from University of Pittsburgh, where she worked diligently to complete a Bachelor of Science in Computational Biology and a Bachelor of Arts in Gender, Sexuality, and Women’s Studies.

A Unique High School Internship Experience

Rashi participated in the CIRM-funded SPARK internship at City of Hope in the summer of 2019, which she says was a major influence on the trajectory of her studies.  

SPARK, also known as the Summer Program to Accelerate Regenerative Medicine Knowledge, offers California high school students an invaluable opportunity to gain hands-on training in stem cell research at some of the leading research institutes in California. Rashi—a student at Leland High School in San Jose during her internship—is one of hundreds of students across California to participate since the program’s launch in 2012. 

During her summer internship, Rashi spent her time in the City of Hope lab analyzing magnetic resonance imaging (MRI) sequences to find the efficacy of CAR-T cell therapy treated glioblastoma, the most common primary brain tumor in adults. At the end of the program, she presented her research project at the SPARK annual conference and on social media.

Rashi (left) evaluating the T2 sequence of the MRI at City of Hope in 2019

“Learning to perform image segmentation was no easy feat,” Rashi wrote in a 2019 blog about her internship experience. “Even after two months of studying a brain and identifying the different components, I can revisit a study I finished working on three hours ago and find something I missed. Even though I’m not a radiologist, I’ve learned the power of immunotherapy in an era of precision imaging.”

Rashi presented her research project at the SPARK annual conference in 2019

Her CIRM and City of Hope internship experience inspired a new passion alongside research: storytelling in science.

During college, Rashi worked for her school’s newspaper and Massive Science, a science storytelling publication that aims to make research articles easier to understand to the public. At Massive Science, she wrote articles that made complex science more accessible to readers.

Throughout her college years, she continued to conduct research in labs, including immunology and cell and gene therapy, sharing updates on her social media account along the way. 

Now, she is preparing to work full-time as she decides on her next education journey. 

“CIRM’s SPARK internship and City of Hope gave me the confidence to reach out to professors about topics I didn’t know about and get involved in research, and I attribute those early experiences to my wanting to study and work towards a PhD in the future,” says Rashi.

Rashi graduated from the University of Pittsburgh in 2024

About CIRM’s Education & Training Programs 

CIRM’s education and training programs support the next generation of regenerative medicine scientists, ranging from high school and undergrad students to doctoral candidates and clinical fellows.   
 
CIRM funds four unique internship programs, each with its own set of requirements: SPARK (high school), Bridges (Undergraduate and Masters), COMPASS (Undergraduate), and Scholars (Pre-Doctoral, Post-Doctoral, and Clinical Fellows).  

Each program offers hands-on experience to foster a workforce adept in stem cell science and the technical skills that are crucial for present and future scientific and medical advancements.


Partnering with community colleges and universities, research institutions, and biotechnology companies, each program aims to attract diverse talent to regenerative medicine by offering varied career paths and accessibility across a broader educational spectrum.   
 
To date, CIRM’s educational and training programs have supported 3,800 interns and fellows, the majority of whom have achieved employment or chose to pursue further education in regenerative medicine or related fields.  Learn more about CIRM’s programs on our website.