Scripps Research Institute

Mourning the death of Susan Solomon

/ Kevin McCormack / Leave a comment
Susan L. Solomon, Photo courtesy of NYSCF

We were saddened to learn today of the death of Susan Solomon, the CEO and co-founder of the New York Stem Cell Foundation (NYSCF), a non-profit organization that supports stem cell research around the world. As CEO, Ms. Solomon raised over $400M for stem cell research, helping to catalyze the field and transform the future of medical research.

The foundation announced the news on its website, saying she died after a long battle with ovarian cancer.

CIRM’s Chair Jonathan Thomas said she will be greatly missed. “We were so terribly sorry to hear about Susan’s passing.  She was a titan in our field who did immeasurable good for patients everywhere.  We have so valued our relationship with her and NYSCF through the years.”

Like many patient advocates Ms. Solomon became active when a family member was hit by disease. In her case, it was in 1992 when her ten year old son Ben was diagnosed with type 1 diabetes. A lawyer by training and a longtime business executive she put her skills to work to identify the best way to help her son, and others with type 1 diabetes. In an interview in the Wall Street Journal she says that background really helped: “As a lawyer, you learn how to learn about a new field instantly,” and, she added, “I’m really comfortable asking dumb questions.”

After much research and many conversations with scientists she concluded that stem cells were the most promising way to help patients. In 2005 she co-founded NYSCF.

Dr. Jeanne Loring, the Director of the Center for Regenerative Medicine at the Scripps Research Institute, says Ms. Solomon’s death is a huge blow to the field: “I have worked with NYSCF for the last 5 years, on the project to study neuroinflammation in space using iPSC-derived neurons.   Susan was one in a billion, she threw all of her considerable energy into starting and sustaining the only stand-alone research institute that I know of in the US dedicated to stem cell research.”

Could a common herb help in the fight against COVID-19 and other inflammatory diseases?

/ Esteban Cortez / 1 Comment

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The culinary herb rosemary is commonly used in our kitchens to season an array of dishes, and is also considered a good source for vitamins and minerals.  

Rosemary is also valued for its medicinal properties, and has traditionally been used to help alleviate muscle pain, boost the immune and circulatory system, as well as many other health benefits. 

Now, scientists at Scripps Research have found evidence that a compound contained in rosemary could be a two-pronged weapon against the SARS-CoV-2 coronavirus that causes COVID-19.  

The research was partly funded by the California Institute for Regenerative Medicine (CIRM). 

Published in the journal Antioxidants, the study found that the compound, carnosic acid, can block the interaction between the SARS-CoV-2 outer “spike” protein and the receptor protein, ACE2, which the virus uses to gain entry to cells. 

“We think that carnosic acid, or some optimized derivative, is worth investigating as a potentially cheap, safe, and effective treatment for COVID-19 and some other inflammation-related disorders,” says study senior author Dr. Stuart Lipton of Scripps Research. 

The team also reviewed prior studies and presented new evidence that carnosic acid could inhibit a powerful inflammatory pathway that is active in severe COVID-19, as well as in other diseases including Alzheimer’s.   

They also proposed that this effect could be beneficial in treating the post-COVID syndrome known as “long COVID” whose reported symptoms include cognitive difficulties often described as “brain fog.” 

While the research is preliminary, the researchers propose that carnosic acid has this antiviral effect because it is converted to its active form by the inflammation and oxidation found at sites of infection. In that active form, they suggest, the compound modifies the ACE2 receptor for SARS-CoV-2—making the receptor impregnable to the virus and thereby blocking infection. 

Lipton and his colleagues are now working with Scripps Research chemists to synthesize and test more potent derivatives of carnosic acid with improved drug characteristics for potential use in inflammation-related disorders. 

The full study was co-authored by Takumi Satoh of the Tokyo University of Technology; and by Dorit Trudler, Chang-ki Oh and Stuart Lipton of Scripps Research. Read the source news release here. 

Disclaimer: This research is still in its early phase and there is no suggestion that sprinkling rosemary on everything you eat could help prevent or fight COVID-19. For the latest guidance on COVID-19, see the official CDC website. 

CIRM Board Approves Clinical Trials Targeting COVID-19 and Sickle Cell Disease

/ Kevin McCormack / Leave a comment
Coronavirus particles, illustration.

Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved new clinical trials for COVID-19 and sickle cell disease (SCD) and two earlier stage projects to develop therapies for COVID-19.

Dr. Michael Mathay, of the University of California at San Francisco, was awarded $750,000 for a clinical trial testing the use of Mesenchymal Stromal Cells for respiratory failure from Acute Respiratory Distress Syndrome (ARDS). In ARDS, patients’ lungs fill up with fluid and are unable to supply their body with adequate amounts of oxygen. It is a life-threatening condition and a major cause of acute respiratory failure. This will be a double-blind, randomized, placebo-controlled trial with an emphasis on treating patients from under-served communities.

This award will allow Dr. Matthay to expand his current Phase 2 trial to additional underserved communities through the UC Davis site.

“Dr. Matthay indicated in his public comments that 12 patients with COVID-related ARDS have already been enrolled in San Francisco and this funding will allow him to enroll more patients suffering from COVID- associated severe lung injury,” says Dr. Maria T. Millan, CIRM’s President & CEO. “CIRM, in addition to the NIH and the Department of Defense, has supported Dr. Matthay’s work in ARDS and this additional funding will allow him to enroll more COVID-19 patients into this Phase 2 blinded randomized controlled trial and expand the trial to 120 patients.”

The Board also approved two early stage research projects targeting COVID-19.

  • Dr. Stuart Lipton at Scripps Research Institute was awarded $150,000 to develop a drug that is both anti-viral and protects the brain against coronavirus-related damage.
  • Justin Ichida at the University of Southern California was also awarded $150,00 to determine if a drug called a kinase inhibitor can protect stem cells in the lungs, which are selectively infected and killed by the novel coronavirus.

“COVID-19 attacks so many parts of the body, including the lungs and the brain, that it is important for us to develop approaches that help protect and repair these vital organs,” says Dr. Millan. “These teams are extremely experienced and highly renowned, and we are hopeful the work they do will provide answers that will help patients battling the virus.”

The Board also awarded Dr. Pierre Caudrelier from ExcellThera $2 million to conduct a clinical trial to treat sickle cell disease patients

SCD is an inherited blood disorder caused by a single gene mutation that results in the production of “sickle” shaped red blood cells. It affects an estimated 100,000 people, mostly African American, in the US and can lead to multiple organ damage as well as reduced quality of life and life expectancy.  Although blood stem cell transplantation can cure SCD fewer than 20% of patients have access to this option due to issues with donor matching and availability.

Dr. Caudrelier is using umbilical cord stem cells from healthy donors, which could help solve the issue of matching and availability. In order to generate enough blood stem cells for transplantation, Dr. Caudrelier will be using a small molecule to expand these blood stem cells. These cells would then be transplanted into twelve children and young adults with SCD and the treatment would be monitored for safety and to see if it is helping the patients.

“CIRM is committed to finding a cure for sickle cell disease, the most common inherited blood disorder in the U.S. that results in unpredictable pain crisis, end organ damage, shortened life expectancy and financial hardship for our often-underserved black community” says Dr. Millan. “That’s why we have committed tens of millions of dollars to fund scientifically sound, innovative approaches to treat sickle cell disease. We are pleased to be able to support this cell therapy program in addition to the gene therapy approaches we are supporting in partnership with the National Heart, Lung and Blood Institute of the NIH.”

Researcher claims to have made first gene-edited baby. But did it really happen?

/ Kevin McCormack / Leave a comment

Raelians

Claude Vorilhorn, founder of Raelism; Photo: courtesy thoughtco.com

Remember the Raelians? Probably not. But way back in 2002 the group, some described them as a cult, claimed it had created the world’s first cloned baby. The news made headlines all around the world raising fears we were stepping into uncharted scientific territory. Several weeks later the scientist brought in by the Raelians to verify their claims called it an “elaborate hoax.”

hejiankui

He Jiankui: Photo courtesy MIT Technology Review

Fast forward 16 years and a Chinese scientist named He Jiankui of Shenzhen claims he has created the first genetically modified humans. Again, it is generating headlines around the world and alarming people. In an interview with CNBC, Hank Greely, a bioethicist at Stanford, said if it happened it was “criminally reckless and I unequivocally condemn the experiment.”

The question now is did it happen, or is this just another “elaborate hoax”?

The concerns about this story are real. The scientist claims he used CRISPR to modify embryos during fertility treatments, resulting in the birth of twin girls.

CRISPR has been making headlines all of its own in the last few years as a fast, cheap and efficient way of editing genes. CIRM supports research using CRISPR for problems such as sickle cell disease. The difference being that our research works with adults so any changes in their genes are just for them. Those changes are not passed on to future generations.

The work making headlines around the world used CRISPR on embryos, meaning a child born from one of those embryos would pass those changes on to future generations. In effect, creating a new kind of human being.

This approach raises all sorts of serious issues – scientific, ethical and moral – not the least of which is that the technique could create unknown mutations down the road that would be passed on to future generations.  That’s why in the US the editing of embryos for pregnancy is banned.

But almost as soon as the news was announced there were questions raised about it. The research was not published anywhere. A hospital that the researchers named in their ethical approval documents is denying any involvement.

If it did happen, it could open a new, and potentially frightening chapter in science. In an interview on CNN, Julian Savulescu, director of the Oxford Uehiro Centre for Practical Ethics at the University of Oxford, called the use of CRISPR in this manner as “genetic Russian Roulette.”

“If true, this experiment is monstrous. Gene editing itself is experimental and is still associated with off-target mutations, capable of causing genetic problems early and later in life, including the development of cancer.”

And in an interview on the BBC, Prof Robert Winston, Professor of Science and Society at Imperial College London, said: “If this is a false report, it is scientific misconduct and deeply irresponsible. If true, it is still scientific misconduct.”

Our best hope right now is that this is just a repeat of the Raelians. Our worst fear, is that it’s not.