Stem cell image of the week: Two Experts and an Advocate (Adonica Shaw)

Our stem cell image for the week comes from our “Ask the Stem Cell Team” Facebook live event this week. Moderated by Adonica Shaw Porter of CIRM, the panel discussed the latest in stem cell research and therapies for Sickle Cell Disease. We featured Dr. Mark Walters, a pediatric hematologist/oncologist from Children’s Hospital Oakland Research Institute; Dr. Don Kohn, Professor, Microbiology, Immunology and Molecular Genetics at UCLA; and Adrienne Shapiro, a patient advocate for SCD and the co-founder of the Axis Advocacy SCD patient education and support website.

This event, which has reached more than 8300 people, has also viewed more than 1,800 times on our Facebook page. You can see the full interview here — https://bit.ly/2o4aCAd.

Stem Cell Model Suggests Parkinson’s Drug May Help ALS patients (Kevin McCormack)

A drug that has already been approved to help people with Parkinson’s disease may also be useful in helping people who are battling ALS (also known as Lou Gehrig’s disease).

One of the biggest problems in developing a therapy for ALS is that the disease itself is sporadic, meaning only between 5-10 percent of cases are inherited or familial, so we have little idea what causes the other 90-95 percent of cases. But thanks to some clever scientific sleuth work, featured in an article in ALS News Today, researchers in Japan may have identified a potential new treatment.

Using iPSC techniques they took ordinary cells from 32 patients with the sporadic form of ALS and turned those cells into motor neurons, the kind of nerve cell destroyed by the disease. They then tested around 1,000 different medications to see if any had any impact on these cells. They found that one drug did, Requip (ropinirole).

Requip has already been approved by the US Food and Drug Administration (FDA) for use in treating Parkinson’s disease (PD). The drug helps replace the dopamine in the brain that is attacked by PD.

When tested on the iPSC-generated motor neurons that had ALS, Requip was shown to suppress cell death, abnormal protein aggregation, nerve cell atrophy, and the production of oxygen-related damaging molecules in most of the models.

The findings, which are published in the journal Nature Medicine, suggest Requip might help people with ALS. That would be excellent news as there are only two FDA-approved therapies for the condition and they only work for a small number of patients.

CIRM is funding two clinical trials targeting ALS. One is out of Cedars-Sinai Medical Center and the other with Brainstorm Cell Therapeutics. Brainstorm recently announced that an analysis of the first 31 patients they have treated showed no significant safety concerns, meaning they can go ahead and treat more patients.

San Francisco Chronicle hosts Facebook Live event featuring CIRM President & CEO

For the past two months, San Francisco Chronicle reporter, Erin Allday, has been publishing a four-part, in-depth examination of California’s investment in stem cell research called, “The Miracle Cell”. The first three installments covered the impact on patients, the controversy over unregulated, for-profit stem cell clinics and the research behind high quality clinical trials. The fourth and final part in the series will be published next Thursday and will focus on the politics of stem cell research, specifically the efforts of CIRM to accelerate stem cell treatments to patients with unmet medical needs.

In anticipation of that report, Allday hosted a Facebook Live show that discussed CIRM accomplishments since 2004, when it was created by the voter-approved initative Prop 71. The broadcast featured CIRM President and CEO, Dr. Maria T. Millan, M.D., and UC Davis stem cell researcher Paul Knoepfler, writer of the stem cell blog, “The Niche”.

A video recording of the Facebook Live event is available on the SF Chronicle website.