Are Californians getting their moneys worth for the $3 billion they invested in stem cell science in 2004? Is there cause for optimism that major breakthrough discoveries are about to happen? What is holding back stem cell treatments from reaching patients?

These are some of the issues to be addressed Thursday in San Diego at a special stem cell meeting thats free and open to the public.

The session is sponsored by Californias stem cell agency and UC San Diego, a major hub of stem cell research and experimental treatment.

The event is the first in a statewide outreach tour by the California Institute for Regenerative Medicine, or CIRM.

The agency is projected to run out of money in 2020 unless more money is raised from public or private sources, and the series of forums is partly meant as a way to persuade voters to further support the institute with more funding.

The free event Stem Cell Therapies and You is slated for noon to 1:00 p.m. at the Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive, across from the Salk Institute in La Jolla.

Four speakers at Thursdays event are to discuss the state of stem cell research:

-- Catriona Jamieson, director of the UC San Diego Alpha Stem Cell Clinic and an expert on blood cancers

-- Jennifer Briggs Braswell, executive director of the Sanford Stem Cell Clinical Center, another stem cell clinic at UCSD

-- David Higgins, a patient advocate for Parkinsons on the CIRM board, and a San Diegan

-- Jonathan Thomas, chairman of CIRMs governing board

Boosted with the $3 billion in bond money raised through Proposition 71 (not including the additional $3 billion in interest that taxpayers are also repaying), California has become an international leader in stem cell exploration.

The money has helped attract top-notch scientists from across the country to work in this state, and it has underpinned much of the training for new researchers in this field.

While encouraging reports of individual patients being cured with experimental stem cell therapies have emerged in recent years, no stem cell-based treatment developed in this state has been approved for commercial use.

This lack of therapies on the market has resulted in some criticism that stewards of Californias groundbreaking effort have spent lavishly on researchers and the infrastructure that supports them instead of focusing on how to more quickly turn lab discoveries into usable products and technologies for the public.

In January, the biomedical news site Stat published a lengthy and critical analysis of CIRMs record in clinical trials, quoting critics who said Prop. 71s supporters shamelessly oversold the initiative as providing quick cures.

The airwaves were swamped with guys in white coats who were identified with their academic affiliation even though they were principals of private companies (some of which later got CIRM grants), and basically saying, Were going to have cures by Christmas. Marcy Darnovsky, who directs the Berkeley-based Center for Genetics and Society, was quoted as saying in the Stat article.

Providing answers

Supporters of CIRM and the programs it has backed financially said it can take many years to effectively translate research into treatments, especially when ensuring safety is paramount. The agency is supporting about 30 clinical trials, including some at its own alpha stem cell clinics, combining treatment with research support.

Jonathan Thomas, CIRMs chairman, said the San Diego event and others like it in other parts of the state are meant to update patients and all Californians about how their money has been spent, and to hear from the public. While San Diego will be in the spotlight at this meeting, work throughout CIRM will be discussed.

San Diego has received a lot of money from CIRM, including about $60 million that has gone to ViaCyte, developer of a stem cell-based implant that could produce a functional cure for Type 1 diabetes.

Many San Diego County stem cell researchers have received grants for various projects. These include David Schubert of the Salk Institute for Biological Studies, for stem cell-based development of an Alzheimers drug; Robert Wechsler-Reya of Sanford Burnham Prebys Medical Discovery Institute, to determine the role of neural stem cells in growth, regeneration and cancer; and Bianca Moth of Cal State San Marcos, to train students for a career in stem cell research.

The Sanford Consortium for Regenerative Medicine building, where the Thursday meeting will be held, was constructed with $43 million from CIRM toward its total price tag of $127 million.

Four clinical trials are taking place at UC San Diegos alpha stem cell clinic, said Larry Goldstein, director of the universitys stem cell program.

These are the diabetes treatment being developed with ViaCyte; a treatment for spinal cord injury derived from human fetal cells; a chronic heart failure therapy using mesenchymal stem cells; and a drug called cirmtuzumab that targets cancer stem cells for chronic lymphocytic leukemia. (Yes, the drug was named after CIRM, which supported its research and development.)

Other stem cell treatments are taking place at UC San Diego outside the alpha clinic, Goldstein said. They include one from Kite Pharma of Santa Monica, using genetically modified immune cells called CAR T cells. The trial is being handled through the universitys bone marrow transplant program at Moores Cancer Center because CAR T cell therapy amounts to a bone marrow transplant.

Safety requires time

All these trials need time because patient safety is being evaluated, Goldstein said. That process can consume years.

So far, they all look safe, which is terrific news, Goldstein said.

Other stem cell trials at the alpha clinic are incipient, he said, including for osteoarthritis using mesenchymal and stromal cells, taken from bone marrow and fat tissue. Numerous stem cell clinics offer treatment with these cells, including some operating in a legal gray zone, outside the clinical trial system.

Goldstein said UCSD plans to better study these poorly defined cells, and what they can do, before beginning treatment. Part of that includes building a genetic profile of these cells, using a method called single cell RNA seq.

Once weve got a better handle on what those cells look like, wed like to put them into clinical trials, he said.

Its especially important that we get a handle on patient-to-patient variability, Goldstein said. We expect there will be variability. Most things in humans are. But to my knowledge, the clinics that are using this methodology dont have a logical and rigorous ability to take advantage of that variability to treat human patients.

bradley.fikes@sduniontribune.com

(619) 293-1020

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