AGAINST THE PLAGUE: The California Stem Cell Program Fights Back

Stem Cell Battles
By Don C. Reed
May 08, 2020

We cannot overestimate the stakes of the battle we are in.

In America alone, approximately 70,000 children, women, and men have died from the COVID-19 virus — and nearly a million and a half are infected.

— https://www.cdc.gov/coronavirus/2019-ncov/cases-updates/cases-in-us.html#anchor_1586782138

At 74, I am a prime target for the coronavirus threat. Although fit and healthy at the moment, I am also reasonably ancient, and had asthma-bronchitis as a child.

So, I stay all day in my lonely house. If I do go out for a morning run, it is in the dark so I won’t meet many people. And of course I wear the mandatory face mask, lest I breathe poisoned air and catch the plague, or give it to somebody else.

The good news? I live in California, which has a terrific stem cell program, the absolute best way to fight this vile disease. Its official name is the California Institute for Regenerative Medicine (CIRM) and it began as a citizen’s initiative, Proposition 71.

Begun in 2004, Prop 71 was designed and led by Bob Klein, patient advocate father of a diabetic son. The voters approved the measure strongly, with 59.1% saying YES! CIRM’s full implementation was delayed by frivolous lawsuits for nearly three years, but at last they were all defeated.

It was worth the wait. Taking on diseases and disabilities long considered incurable, CIRM has saved more than 50 lives. It restored health to children with a previously fatal condition, the “Bubble Baby” disease. It brought back upper body motion to paralyzed people, and returned vision to a blind woman — who had never seen her teenage children.

Importantly, CIRM built a structure, so its successes could be repeated. Not only did it help build twelve new stem cell research institutions, where scientists could work on top-notch equipment — without fear of government harassment — but it also set up educational programs, including free stem cell modules for high schools and colleges, and scholarship programs to help less fortunate students become scientists, working for pay in outstanding colleges.

Reliable funding allowed scientists to work without worry, concentrating on the science instead of the endlessly nagging fear of where the next grant was coming from. CIRM shared what it learned: a state-wide network of knowledge, with results published, verifiable, and open. Cooperation with scientists in different states and nations invigorated the research; California paid our scientists, the other entities paid theirs: more bang for the research buck.

Does it work? More than eighty new therapies or products are in human trials right now.

The bad news? Sixteen years after it began, CIRM’s funding is essentially gone.

What a loss it would be, if the program were to shut down: something so powerful and effective, slipping through our fingers in this hour of greatest need?

But we have not given up. (Important: when I say “we”, I mean California; I have no personal connection with CIRM, except pride.)

In November, if all goes well, California will authorize a $5.5 billion renewal for the stem cell program — and then, it is not hard to predict, there will be an all-out war against COVID-19. (Even now, California scientists are working to see if they already have something applicable; but this is nothing compared to when they start getting major grants.)

With its last dollars, CIRM is fighting to make change happen. In the past few weeks, the leadership of CIRM somehow scraped together a war chest of $5 million to fight COVID-19, the coronavirus. Five million may not be a lot, in research terms, but it will scratch a match to start the fire.

The immediate plan? Small projects, intensely focused in their impact, as a martial artist concentrates power in a strike.

First, a $750,000 grant to Dr. John Zaia of the City of Hope.

John Zaia, City of Hope photo

John Zaia, City of Hope photo

Dr. Zaia is taking plasma from the blood of survivors of COVID-19, those who had the disease but did not die. Since plasma carries antibodies to fight infection, there may be a way to use what Zaia calls “convalescent” plasma to help sick people get well. What makes some people’s immune defenses so strong, while others are weak? Figuring out that answer could help us design new biomedical weapons.

— https://www.cirm.ca.gov/about-cirm/newsroom/press-releases/04242020/cirm-board-funds-its-first-clinical-study-covid-19

Fighting from another angle, UCLA scientists Dr. Gay Crooks and Christopher Seet are taking healthy T-cells (part of the body’s natural immune system) and exposing them to parts of the virus in the lab: to figure out which parts provoke the biggest response.

Gay Crooks, UCLA

Gay Crooks, UCLA

“Our approach will allow us to focus vaccine and therapeutic development on those parts of the virus that induce strong T-cell immunity,” said Dr. Seet.

UCLA Newsroom photo of Dr. Seet and other UCLA scientists

UCLA Newsroom photo of Dr. Seet and other UCLA scientists

As Dr. Crooks put it, “The … cells we are able to make using this process are really good at chopping up the virus…”

— “UCLA scientists receive grants for COVID-19 research from California’s stem cell agency”, Tiare Dunlap, Friday, April 24, 2020

Another approach by a UCLA scientist involves a three-dimensional miniature lung “organoid”. Invented by Dr. Brigitte Gomperts, the stem cell-derived blob of living tissue is microscopic, smaller than the point of a pin, yet the structure closely resembles certain aspects of a human lung. This may be extremely useful in the rapid testing of anti-COVID-19 drugs, including many which are already FDA-approved.

Brigitte Gomperts, UCLA Stem cell expert on organoids

Brigitte Gomperts, UCLA Stem cell expert on organoids

https://stemcell.ucla.edu/research-focus

Passed in 2004, Prop 71 was designed and led by Bob Klein, patient advocate father of a diabetic son. The voters approved the measure strongly, with 59.1% saying YES! CIRM’s full implementation was delayed by frivolous lawsuits for nearly three years, but at last they were all defeated.

It was worth the wait. Taking on diseases and disabilities long considered incurable, CIRM has saved the lives of more than 50  children in just one trial. It restored health to children with a previously fatal condition, the “Bubble Baby” disease. It brought back upper body motion to paralyzed people, and returned a significant measure of vision to a blind woman– who had never seen her teenage children.

Importantly, CIRM built a structure, so its successes could be repeated. Not only did it help build 17 new stem cell research institutions, where scientists could work on top-notch equipment— without fear of government harassment– but it also set up educational programs, including free stem cell modules for high schools and colleges, and scholarship programs to help less fortunate students become scientists, working for pay in outstanding colleges.

Reliable funding allowed scientists to work without worry, concentrating on the science instead of the endlessly nagging fear of where the next grant was coming from. CIRM shared what it learned:  a state-wide network of knowledge, with results published, verifiable, and open. Cooperation with scientists in different states and nations invigorated the research; California paid our scientists, the other entities paid theirs: more bang for the research buck.

Does it work? More than eighty new therapies or products are in human trials right now.

The bad news? Sixteen years after Prop 71 passed, CIRM’s funding is essentially gone.

What a loss it would be, if the program were to shut down: something so powerful and effective, slipping through our fingers in this hour of greatest need?

But we have not given up. (Important: when I say “we”, I mean California; I have no personal connection with CIRM, except pride.)

In November, if all goes well, California will authorize a $5.5 billion renewal for the stem cell program—and then, it is not hard to predict, there will be an all-out war against COVID-19. Even now, California scientists are working to see if they already have something applicable; but this is nothing compared to when they start getting major grants.

FLASH!!!

Below is what I consider amazing news. You can read the full press release at:

https://tinyurl.com/ybz7x9sn

“A Covid-19 Success, 300 Percent Stock Price Increase and the California Stem Cell Agency

“A company that the California stem cell agency has backed with nearly $18 million scored big this morning on news that one of its products had generated a 100 percent survival rate with a small group of (6) critically ill Covid-19 patients.

“The company is Capricor, Inc., a publicly traded company based in Beverly Hills, Ca.

“The company said in a news release that it had generated “100 percent survival in critical Covid-19 patients who were treated with Capricor’s lead asset, off-the-shelf …cardiac cell therapy CAP-1002, at Cedars-Sinai Medical Center as part of six compassionate care cases.”

“The company said the federal government has approved 20 additional patients for treatment. Capricor also said it is developing ‘a randomized, placebo-controlled trial (for) patients with moderate and severe disease…”

“Capricor and its underlying research have long been (funded by) the state stem cell agency … the California Institute for Regenerative Medicine (CIRM). In addition to $17.8 million given directly to the company, another $7 million went to Cedars-Sinai for much of the early development work that led to the creation of Capricor.

“Speaking to the agency’s governing board in 2016 concerning CAP-1002, CIRM director Jeff Sheehy, chair of its Science Committee, said:

“This is pretty much a pure CIRM product. They came into our first disease team to develop the product. We’ve supported two of the three clinical trials. So if this turns out to be a major success, it will be a real feather (in the cap) for CIRM. We’ve been with them all the way…”

“Linda Marban, CEO of Capricor, said:

Linda Marban, Capricor CEO

Linda Marban, Capricor CEO

“As the global medical community continues to come together in its battle against COVID-19, the results of our initial compassionate care cases are extremely promising and what we had anticipated. We look forward to continuing to treat additional patients under our recently approved expanded access program Investigational New Drug application.

“CAP-1002 is an easy-to-deliver intravenous therapy that has been administered successfully to over 150 patients to date. Given its novel mechanism of action, it could be a potential game-changer in helping countless Covid-19 patients.”

“The Capricor product initially targeted Duchenne muscular dystrophy (DMD), “a muscle wasting condition that steadily destroys the muscles in the arms and legs, heart and respiratory system” of boys and “never lets up,” CIRM says.

“The CAP-1002 treatment has demonstrated immune-system modulating properties that come into play with Covid-19, Capricor said.

“… current understanding of Covid-19’s later stages are thought to be due to overstimulation of the immune system, which triggers a cytokine storm in which the body is overwhelmed with pro-inflammatory molecules. This immune response may become excessive…, inducing pneumonia, organ failure and death. Therefore, it can be the body’s overreaction to Covid-19, rather than the virus itself, that delivers the fatal blow.”

The Marban/Capricor therapy must of course go through more rigorous testing and validation, and there is no guarantee of success.

I am neither scientist nor doctor. My opinions should be taken with a pinch of salt, or maybe a handful.

But my gut instinct on the Marban/Capricor trial? It is important. It deserves investigation, and that means funding: yet another reason to support the renewal of funding for the California program.

So, where do you stand on stem cell/gene research? Do you want regenerative medicine therapies to move forward in a responsible and verifiable basis? If so, there is something you can do:

If you are a voting-age person and you live in California, on November 3rd, vote YES! on the bill called: “THE CALIFORNIA STEM CELLS FOR RESEARCH, TREATMENTS AND CURES INITIATIVE OF 2020”.

— https://tinyurl.com/y9hsccf3

Be sure you are registered to vote! Then, when you are actually in the voting booth (or filling out the form to vote by mail) hunt for that initiative on the ballot — find it — and vote for it.

In the meantime, talk it up. Tell your friends. Make sure everyone in your circle of friends knows about this incredible opportunity; the $5.5 billion renewal of this superbly effective program.

Want more info? Go to the Californians for Cures website: https://caforcures.com/

Have you given your endorsement yet, as an individual, or a member of a group? If not, click on this easy ONE STEP ENDORSEMENT LINK:

https://caforcures.com/become-a-part-of-this-initiative-sign-an-endorsement-form/

It will cost you nothing but a moment of your time.

We need everybody’s help — in this war against the plague.