Ordinarily, we end each week at the Stem Cellar with a few stem cell stories that caught our eye. But, for the past couple of weeks we’ve been busy churning out stories related to our Month of CIRM blog series, which we hope you’ve found enlightening. To round out the series, we present this “caught our eye” blog of CIRM-specific stories from the last half of October.

Stopping neurodegenerative disorder with blood stem cells.

CIRM-funded scientists at the UC San Diego School of Medicine may have found a way to treat a progressive neuromuscular disorder called Fredreich’s ataxia (FA). Their research was published last week in the journal Science Translational Medicine.

FA is a genetic disease that attacks the nervous tissue in the spinal cord, leading to the loss of sensory nerve cells that control muscle movement. Early on, patients with FA experience muscle weakness and loss of coordination. As the disease progresses, FA can cause scoliosis (curved spine), heart disease, and diabetes. 1 in 50,000 Americans are afflicted with FA, and there is currently no effective treatment or cure for this disease.

Cherqui’s approach

UCSD scientists, led by CIRM grantee Stephanie Cherqui, previously showed that transplanting blood stem and progenitor cells prevented cystinosis in mice. CIRM is currently funding her late‑stage preclinical cystinosis research.

In this new study, the team tested whether the same approach could treat FA. They used a transgenic mouse model carrying two human FXN mutations. FXN produces frataxin, a mitochondrial protein. Mutations reduce frataxin and cause FA symptoms.

The researchers transplanted healthy blood stem and progenitor cells into FA mice. These cells became microglia and macrophages. Microglia appeared in the brain and spinal cord; macrophages appeared in the spine, heart, and muscle. The healthy immune cells produced frataxin and delivered it to affected nerve and muscle cells.

Cherqui said the transplant rescued FA‑affected cells. Frataxin levels returned, mitochondrial function normalized in the brain and heart, and skeletal muscle atrophy decreased.

Her team noted that this mouse model does not fully mirror human disease. They plan to test their method in other models next.

Brainstorm’s CIRM-funded clinical trial for ALS enrolls its first patients

“We have been conducting ALS clinical trials for more than two decades at California Pacific Medical Center (CPMC), and this is, by far, the most exciting trial in which we have been involved to date.”

Dr. Robert Miller said the ALS trial is the most exciting effort his team has joined in two decades at California Pacific Medical Center. Brainstorm Cell Therapeutics announced on October 16 that it had enrolled the first patients in its CIRM‑funded stem cell trial for ALS.

ALS, or Lou Gehrig’s disease, is a devastating disorder that destroys motor neurons. As neurons die, muscles weaken and eventually become paralyzed. Most patients die within three to five years, and no effective therapy exists.

Brainstorm’s therapy, NurOwn®, uses the patient’s own bone marrow mesenchymal stem cells. Researchers modify these cells to release factors that protect motor neurons. Because the cells come from the patient, no immunosuppressive drugs are needed.

The Phase 3 trial aims to enroll 200 patients and builds on a promising Phase 2 study. CIRM’s $16 million grant supports trial sites across California. Abla Creasey noted that Brainstorm will run the study at CIRM Alpha Clinics and manufacture its product using CIRM‑funded facilities.

Initial results from the Phase 3 trial are expected in 2019.

CIRM President Maria Millan reflects on her career, CIRM’s successes, and the outlook for stem cell biology 

RegMedNet published an interview with Maria Millan, MD, CIRM’s new President and CEO. The piece highlights her major accomplishments before joining CIRM, including details even some staff may not have known.

Dr. Millan also describes CIRM’s achievements during her time as Vice President of Therapeutics. She shares her outlook on the agency’s future and the direction of stem cell biology over the next five years and beyond.

It’s a must‑read article.