Update on SCID patient enrolled in CIRM-funded gene therapy trial

Photo credit: UCSF

Hataalii Tiisyatonii Begay (HT) is paving the road for newborns with SCID. When HT was born in 2018 in a remote part of the Navajo nation, he was quickly diagnosed with a rare and -usually fatal- condition. Today, thanks to a therapy developed at UCSF and funded by CIRM, he’s a healthy four-year-old boy running around in cowboy boots.

The California Institute for Regenerative Medicine (CIRM) has invested $12 million to test this therapy in a clinical trial at UC San Francisco.

The disorder is Artemis-SCID, a form of severe combined immunodeficiency disease. Children born with this condition have no functioning immune system so even a simple infection can prove life-threatening or fatal.

Currently, the only approved treatment for Artemis-SCID is a bone-marrow transplant, but many children are unable to find a healthy matched donor for that procedure. Even when they do find a donor, they often need regular injections of antibodies to boost their immune system.

Dr. Morton Cowen and Dr. Jennifer Puck. Photo credit: UCSF

In this clinical trial, UCSF doctors Morton Cowan and Jennifer Puck are using the patient’s own blood stem cells, taken from their bone marrow. In the lab, the cells are modified to correct the genetic mutation that causes Artemis-SCID and then re-infused back into the patients. The goal is that over the course of several months these cells will create a new blood supply, one that is free of Artemis-SCID, and that will in turn help repair the child’s immune system.

In April 2022, HT finally moved back home to Arizona. Nowadays, HT is off his medication and living the life of a normal and happy young child. On the Arizona ranch, there are horses to pet, cattle and sheep to tend, and streams to cool his hands in.

Watch the video below to find out more about HT’s journey and the team at UCSF behind the pioneering trial.

Video courtesy of UCSF

Funding development of a vaccine for acute myelogenous leukemia (AML)

Dr. Karin Gaensler. Photo credit: Steve Babuljak/UCSF

Adult acute myelogenous leukemia—also known as acute myeloid leukemia (AML)—is a blood cancer in which the bone marrow makes a large number of abnormal blood cells. 

About 20,000 new cases of AML are diagnosed each year in the US with a 5-year survival rate of around 29%. In 2022, there were nearly 12,000 deaths from AML. Many AML patients—a majority of which are over 60 years old—relapse after treatment. Blood stem cell transplant can be curative, but many older patients do not qualify, showing that there is a significant unmet medical need in treating AML. 

That’s why the California Institute for Regenerative Medicine (CIRM) awarded $6,000,000 to Dr. Karin Gaensler at the University of California, San Francisco (UCSF) to support development of a safe and effective vaccine for the blood cancer AML to improve relapse-free survival. 

To develop the cancer vaccine, Dr. Gaensler and her team will engineer the patient’s blood stem cells to maximize stimulation of leukemia-specific killing activity and reintroduce engineered cells back to the patient to target and kill residual leukemia stem cells.  

This approach holds the potential for long-term effectiveness as it targets both AML blasts and leukemic stem cells that are often the source of relapse.  

This award is a continuation of a previous CIRM grant that will support the manufacture of the vaccine and the completion of late-stage testing and preparation needed to apply to the US Food and Drug Administration (FDA) for permission to begin a clinical trial. 

The Most Read Stem Cellar Blog Posts of 2022

This year was a momentous one for the California Institute for Regenerative Medicine (CIRM). You can read some of our achievements in our 2021-2022 annual report.  

As always, we shared our most exciting updates and newsworthy stories—topics ranging from stem cell research to diversity in science—right here on The Stem Cellar. More than 100,000 blog visitors followed along throughout the year!  

In case you missed them, here’s a recap of our most popular blogs of 2022. We look forward to covering even more topics in 2023 and send a sincere thank you to our wonderful Stem Cellar readers for tuning in!   

In Memory of Kevin McCormack 

We cannot close out the year without honoring our dear friend and colleague Kevin McCormack, who passed away suddenly in December. Kevin was CIRM’s Director of Patient Advocacy and loved writing for The Stem Cellar. He did a wonderful job in translating complex science for the general public and was a great mentor to the CIRM team. Many of his closest friends and colleagues wrote memories about him, and we compiled them in this blog post honoring his life and dedication to CIRM and patients everywhere.  

How stem cells helped Veronica fight retinitis pigmentosa and regain her vision 

We shared the story of Veronica McDougall, who thought everyone saw the world the way she did: blurry, slightly out-of-focus and with tunnel vision. When she was 24, she went to see a specialist who told her she had retinitis pigmentosa, a rare degenerative condition that would eventually leave her legally blind. Click through to read about her experience participating in a CIRM-funded clinical trial with a company called jCyte

Smoking marijuana could be bad for your heart, but there is an unusual remedy 

Millions of Americans use marijuana for medical reasons, such as reducing anxiety or helping ease the side effects of cancer therapy. Millions more turn to it for recreational reasons, saying it helps them relax. Now a new study says those who smoke marijuana regularly might be putting themselves at increased risk of heart disease and heart attack. Check out this blog to learn how a team at Stanford Medicine used the iPSC method to create human endothelial cells and, in the lab, found that THC appeared to promote inflammation in the cells. 

A pioneering couple uproot their lives to help their baby 

This year, we shared some encouraging news about a CIRM-funded stem cell clinical trial for spina bifida at UC Davis Health. Spina bifida is a birth defect that occurs when the spine and spinal cord don’t form properly and can result in life-long walking and mobility problems for the child, even paralysis. This blog told the story of parents Michelle Johnson and Jeff Maginnis, who learned 20 weeks into the pregnancy that the fetus had spina bifida. Read the whole story to learn about their experience and the status of their baby Tobi.  

And that wraps up The Stem Cellar’s top blog posts of 2022! If you’re looking for more ways to get the latest updates from The Stem Cellar and CIRM, follow us on social media on Facebook, Twitter, LinkedIn, and Instagram.

Finding a treatment for Tay-Sachs disease

The California Institute for Regenerative Medicine (CIRM) has awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease.  

Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of certain fats in the brain and nerve cells and causes progressive dysfunction.  

There are several forms of Tay-Sachs disease, including an infant, juvenile, and adult forms. Over a hundred mutations in the disease-causing Hex A gene have been identified that result in enzyme disfunction. There are currently no effective therapies or cures for Tay-Sachs. 

Illustration by Kateryna Kon

The UC Davis team will genetically modify the patient’s own blood stem cells to restore the Hex A enzyme that is missing in the disease.  

The goal is to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.  

“The successful development of this therapy will not only help patients with Tay-Sachs but will demonstrate the use case of this therapeutic approach for other monogenic neurodegenerative diseases,” the UC Davis team said. 

This work is a continuation of a CIRM grant that the team received. 

CIRM funds clinical trial to make cancer therapy safer, less toxic

Blood stem cell transplantation following high dose chemotherapy is standard of care and potentially curative for aggressive forms of lymphoma. However, this treatment regimen is limited by severe toxicity and life-threatening complications due to delayed recovery of the blood system and vascular related damage of multiple organs.

Today the governing Board of the California Institute for Regenerative Medicine (CIRM) funded a Phase 3 clinical trial to support development of a safer, more tolerable alternative.

This brings the number of clinical trials funded by CIRM to 86.

The Board awarded $15,000,000 to Dr. Paul Finnegan and Angiocrine Bioscience to test AB-205, human endothelial cells engineered to express a pro-survival factor.

Prior data suggest that, in the setting of chemotherapy and stem cell transplantation, AB-205 cell therapy can accelerate the recovery of the blood system and protects from toxicity by enhancing the recovery from vascular damage. AB-205 is being studied in a Phase 3 trial in adults with lymphoma undergoing high-dose chemotherapy and autologous blood stem cell transplant.

“If successful, this approach can overcome hurdles to the success of chemotherapy and blood stem cell transplantation for the treatment of advanced blood cancer,” says Dr. Maria T. Millan, President and CEO of CIRM. “This Phase 3 trial is the culmination of preclinical research and the initial clinical trial previously funded by CIRM.”

Lymphoma is the most common blood cancer and one of the most common cancers in the United States, accounting for about 4% of all cancers according to the American Cancer Society and the 6th most commonly diagnosed cancer among men and women in California.  It is estimated that there will be 89,010 new cases of lymphoma and 21,170 lymphoma related deaths in the US in 2022 alone.  In California, it is estimated that there will be over 9,250 new cases of lymphoma with over 2,100 deaths.

“Angiocrine Bioscience is honored to be awarded this grant from CIRM to support our AB-205 Phase 3 trial,” commented Angiocrine CEO Dr. Paul Finnegan. “CIRM has been an instrumental partner in our development of AB-205, a novel therapeutic that acts on the patients’ endogenous stem cell niches. The grant award will considerably aid in our effort to bring forth a solution to the unmet need of transplant-related complications.”

In Memory of Kevin McCormack 

CIRM lost its voice this weekend.   

Sunday evening, our beloved Director of Patient Advocacy and frequent face and voice of the Institute, Kevin McCormack, passed away unexpectedly, leaving an unfillable void in our hearts.  

As our chief chronicler of CIRM milestones and celebrator of patients, Kevin brought his unique brand of grace, style and humor to all he did and to all he touched. Whether in person or in prose, Kevin’s twinkle in his eye and wry sense of humor shone through always, infusing all of our work with enthusiasm as well as the reminder that we’re all in this together. 

Our dear friend embodied the spirit and mission of CIRM, working every day to make patients seen and their stories heard, to give the advocate community a platform and a voice to amplify our shared passion for medical breakthroughs. He helped bring hope for a better future for all. 

Kevin made a career of bringing awareness to the public about medical advancement and patient experiences, serving as KRON-TV’s Health/Medical producer for 18 years. He leveraged that experience as a media relations manager for Kaiser and California Pacific Medical Center, where he honed his skills in medical journalism, helping the public understand how research breakthroughs might be translated to patient impact. 

Our good fortune came in 2012 when Kevin joined us as Senior Director of Public Communications and Patient Advocate Outreach. Earlier this year, Kevin assumed the role of Director of Patient Advocacy, where he remained committed to raising awareness about CIRM’s impact and putting patient voices at the forefront. 

And he did so with indefatigable optimism and cheer, for both patients and his friends at CIRM. 

As you’ll see from memories of Kevin shared below, he was not only extraordinarily talented at his work, he was one of those prized colleagues who also made work a joy. His warmth, sincerity and sense of humor were a tonic during long days and constant deadlines.

Kevin made us feel special, individually and collectively. He made his world, our world, a better place to be. 

Remembering Kevin McCormack

One of my many fond memories of Kevin was the “Elevator Pitch” initiative.  In his kind and effective way, he got “buy in” from scientists that it was important to communicate their work in an understandable and engaging fashion…He turned a “critique” into action and fun!

Maria T. Millan, M.D., President and CEO, CIRM

Kevin was truly a gentleman: a gentle man with much integrity. He and his voice will be missed.

Jenifer Raub, President, Summit for Stem Cell Foundation, Patient Advocate

His beer of choice at San Diego airport: Stone Arrogant Bastard Ale.

Geoff Lomax, Senior Officer, Therapeutics Development

Kevin took a chance on me when I applied to CIRM in 2015. I was a postdoc with a passion for science communications but without much experience. Kevin saw something in me and I am so grateful that I had the opportunity to work on his communications team and at CIRM for three wonderful years. I learned so much from him and was always inspired by his selflessness and commitment to helping patients and to CIRM’s mission. Our days were filled with laughter and jokes but at the end of the day Kevin was always someone who you could count on to come through. He always went that extra mile whether it was remembering a story you told him years back, developing you a personalized travel itinerary for your honeymoon trip, or reaching out to you during the pandemic just to see how you were doing. I know he has touched so many lives and he will be missed so dearly. But I’m thankful that his legacy lives on through his family, friends and the amazing work he has done in his 10-year career at CIRM. RIP.

Karen Ring, Fmr. CIRM Associate Director Of Communications

Kevin was universally loved for his kindness and wit. Simply put, he is irreplaceable. I will greatly miss him.

Kelly Shepard, Associate Director, Scientific Programs

Kevin’s kindness, witty sense of humor and enthusiasm will be sorely missed at CIRM. Before the pandemic, in the office, I could always count on walking over to Kevin’s space to chat about the latest news, to have a good laugh and to have my day become a thousand times brighter for it.

Externally he was a tireless advocate for CIRM’s mission and within the team he was an equally tireless advocate for the patients and the citizens of California. In many of my professional interactions with him he helped me broaden my perspective and sharpen my messaging to be more universally effective.

Shyam Patel, Senior Director of Business Development and Alliance Management

Kevin always brought levity, compassion, and good cheer to every conversation and interaction we had. His kind jokes and colorful suits will be sorely missed. My whole family is gutted that he is gone, suddenly and entirely unbelievably.

Doug Kearney, Associate Director, Grants Management 

We all knew and loved Kevin’s humor. I especially liked the self-deprecating kind. One evening, in 2013 or so, Kevin and I were scheduled for a science café public lecture on ‘the remarkable powers of stem cells’ in a Fremont public library. For some reason, Shirley was with us. As Kevin was driving us on a dark highway from San Francisco to Fremont that evening, in anticipation of the upcoming event he at some point declared ‘I know how to show my wife a good time’. Very hard to imagine CIRM without Kevin.

Uta Grieshammer, Senior Science Officer, Scientific Programs

Kevin and I used to exchange poems once in a while. Here is one poem from a Palestinian poet Mahmoud Darwish that I handed to him many years ago when we were in King Street office in San Francisco, that he liked:

Remainder of Life  

 If I were told: 
By evening you will die, 
so what will you do until then? 
I would look at my wristwatch, 
I’d drink a glass of juice, 
bite an apple, 
contemplate at length an ant that has found its food, 
then look at my wristwatch. 
There’d be time left to shave my beard 
and dive in a bath, obsess: 
“There must be an adornment for writing, 
so let it be a blue garment.” 
I’d sit until noon alive at my desk 
but wouldn’t see the trace of color in the words, 
white, white, white . . . 
I’d prepare my last lunch, 
pour wine in two glasses: one for me 
and one for the one who will come without appointment, 
then I’d take a nap between two dreams. 
But my snoring would wake me . . . 
so I’d look at my wristwatch: 
and there’d be time left for reading. 
I’d read a chapter in Dante and half of a mu’allaqah 
and see how my life goes from me 
to the others, but I wouldn’t ask who 
would fill what’s missing in it. 
That’s it, then? 
That’s it, that’s it. 
Then what? 
Then I’d comb my hair and throw away the poem . . . 
this poem, in the trash, 
and put on the latest fashion in Italian shirts, 
parade myself in an entourage of Spanish violins, 
and walk to the grave!

Sohel Talib, Director, Therapeutics Development

Kevin was the embodiment of CIRM. He was the heart and soul of our organization and made an impact on all of us. He was compassionate, sincere, funny and a champion for patients and the community and will be sorely missed.

I have been reading old emails to smile as he always had something cheeky to say. This one was to all employees about a blog post last month and I just keep thinking it was an honor to know him and work with him.

‘Where I come from Thanksgiving was called Thursday, so it’s not a tradition I grew up with. But I have to say it’s now my favorite holiday. It’s simple, fun, not packed with the need to exchange gifts or forced jollity, and definitely not fueled by ridiculous amounts of alcohol (you know who you are St. Patrick’s/Cinco de Mayo). And it’s a chance to give thanks for the many blessings we have, among them is you, my colleagues. It’s a pleasure and an honour to work with so many fine, kind, committed individuals. I wish you all the most lovely of Thanksgivings.”

Jennifer Lewis, Senior Director, Grants Management & IT

Usually, when you say, “How are you doing?” to a friend or colleague, the typical response is “I’m good.” But not Kevin. He always replied with “Grand!” or “Fabulous!” It was almost like a ritual or mantra as we greeted each other every morning at the CIRM office. And it sums up what I so appreciated about Kevin: his unbounding optimism, his friendliness, and his ability to find humor even in challenging situations. Kevin, I can’t believe you’re gone and I will miss your sparkling spirit. May your memory be a blessing and an inspiration to all who were lucky enough to know you.

Todd Dubnicoff, Fmr. CIRM Multimedia Editor/Science Writer

Kevin McCormack was a friend, a patient advocate, and a warrior for stem cell research. Every time we needed him he was there, always with the right words, the right tone, and with his heart in the right place.

To know Kevin was to be his friend and love him as much. He always was there for us when we needed him and it was our honor to say we were always there for him because he is a true friend and he will be missed.

Roman Reed, Founder, The Roman Reed Spinal Cord Injury Research Act

To me, Kevin was the face of CIRM. In fact, appropriately, his face is on the first video of CIRM’s YouTube channel. He was full of life, positivity and humor.

When I did my interview for CIRM, Kevin was on the zoom call at the center of the Hollywood squares and every time I looked at him, I felt a sense of comfort. He had a smile on his face the entire time. So when I was asked a tough question, my first reaction was always to look at Kevin’s face just to calm down. He was a joy and I feel fortunate to have had the opportunity to know him.

I’m certainly going to miss receiving those weekly emails that start with: “Hello, lovely people”. Kevin, may you rest in peace.

— Mitra Hooshmand, Senior Science Officer, Special Projects

I had the privilege of working closely with Kevin on the communications team at CIRM. Whether speaking with patient advocates or writing for The Stem Cellar, he always did so with a bright smile and passion for his work. I’ll never forget his impeccable fashion, quick-witted sense of humor, and, above all, his incredible kindness.

Yimy Villa, Fmr. CIRM Marketing Communications Manager

Kevin was simply a good human with a good soul people liked to be around. He was very good at focusing on what needed to get done even when others around him were dabbling in politics. He got things done because he was so good at so many aspects of our work together in communication and outreach. When I Left CIRM, he was what I missed the most.

Don Gibbons, CIRM 2008-2016

I don’t know what to say other than I miss him. I miss his great smile. His daily greetings full of warmth and happiness.  I miss his lovely hats. His beautiful suits. His empathy and understanding. His unflappable demeanor. We’ve all suffered a great loss in our lives and this outpouring of emotion and love is a testament to what an amazing human he was.

Maria Bonneville, Vice President of Public Outreach & Board Governance

Kevin once wrote, “At CIRM we are funding research to understand how to harness the power of stem cells to repair the damage caused by disease, to restore function to a heart damaged by a heart attack, or a brain injured by a stroke.” His passing leaves us heartbroken in a way that cannot be mended by stem cells or any other medicine, but we can keep his legacy alive by carrying forward his commitment to patient advocacy and the promise of regenerative medicine.

He made a difference in his self-described role as “the official translator of the agency” as he turned complex concepts about equally complex science into everyday language that anyone could understand. As a friend, colleague, empath, translator, advocate, intrepid traveler, dog-lover, tie-wearer, slayer-of-deadlines, and humorist, Kevin left an indelible mark on many. His spirit, his words, and his compassion will never be forgotten.

Christina Divigard, Valvespring

I only worked briefly with Kevin this past summer, but looked forward to many future engagements together with the Valvespring team. His wit and his facility with words made everything he wrote a pleasure to read, and also laugh-out-loud funny. He could find the humor in anything, and embraced as kin those who laughed along with him. Even last summer he was looking forward to visiting New York, and I’m so glad he had the chance to visit the Big Apple, and likely a pub or two, once again. Godspeed to Kevin — a memorable soul who shared his gifts with such generous camaraderie and fellowship.

Susan Millerick, Valvespring

Working with Kevin was like putting the exclamation point at the end of a sentence. Much much more than a subject matter expert in regenerative medicine, he infused it with his wisdom, attention to detail and compassion – for those he worked with, and all those he hoped his efforts would help. A true gentleman, and profound loss to us all.

— Bob Demetrius, Valvespring

I remember Kevin being part of my interview panel when I was being interviewed. He instantly gave me a warm and welcoming feeling which made it easier for me to feel at ease. When I finally met him in person, he walked in with such style and confidence. I loved it. He was always willing to lend me a helping hand. You can tell that he truly cares about the impact of the work we support. It showed in the relationships he’s built with our patient advocates. He truly cares for people of this world. I am thankful for the short time I’ve known him since I have only been here for a little over a year. But no matter how short it has been, it has been well worth it and I have been honored to call him a colleague but most importantly a friend. I have so much love and respect for the fantastic Kevin McCormack.

Marianne Dequina-Villablanca, Associate Director of Board Relations

Some of our Favorite Irish Chum’s fabulous vocabulary of expressions honoring him: Chum, Excellent, Lovely people (recycled from Katie Sharify’s), Cheeky, “Barzelona” (to me in passing), Fabulous, Folks want to talk to you ladies!, ‘Be well, my friend’, Cheers.

Rosa Canet-Aviles, Vice President of Scientific Programs 

I remember interviewing Kevin for the job ten years ago and being drawn immediately to his infectious enthusiasm, sunny attitude and sincere interest in CIRM’s mission and programs. Throughout his time with us, he brought all of those qualities and so much more to the office every single day. He was a true professional who unfailingly did an amazing job bringing CIRM to life for the outside world and who was genuinely esteemed by all whom he came in contact with. Kevin was the rare personality that brought a smile to your face on every occasion and encounter. Very few people can do that.

Jonathan Thomas, Chairman, Independent Citizens Oversight Committee

As a new CIRM employee, I was very recently reading Kevin’s blog posts and watching his YouTube videos as I prepared for interviews. In this way, Kevin was my first glimpse into the culture of CIRM. His love for science and commitment to patients are embodied across the agency, and Kevin’s work was an important part of my hope to join the CIRM team. I dearly wish that I had more time to know and work with him. Thank you, Kevin, for the impact you’ve made on me and the impacts you will continue to make through the stories and memories that you leave behind.

Elizabeth Noblin, Senior Science Officer, Portfolio Development and Review

Kevin McCormack was a colleague who was one-in-a-million. He was an Irishman with a not-so-thick-anymore accent, a great sense-of-humor, and a dedication to his work. He was always a dapper dresser, complete with a tie, coat and a carefully folded handkerchief poking from his breast pocket – but he was the opposite of a stuffed shirt. He was incredibly down-to-earth, with an optimistic and friendly personality, always willing to help anyone with whatever needed to be done.

Kevin came to CIRM from a career in journalism, not in science or medicine, but he was perfect for his roles in communication and patient advocacy. He quickly learned about the science of stem cells and regenerative medicine, and how new medicines are developed, and then figured out how to translate it all back into language that the general public could understand – and in a most engaging way, e.g. his “Stem Cells In Your Face” postings on YouTube. He was also an incredible resource for connecting CIRM to patient communities, and bringing their voices to our advisory panels, conferences and blogs.

In a job where we get many emails, it was always a pleasure to get one from Kevin – at a minimum, it would be friendly and positive, but usually it was funny as well. He always emailed the CIRM staff when he had posted a new blog – this one from a few weeks ago captures much about him:

“Hello Lovely People,

Where I come from Thanksgiving was called Thursday, so it’s not a tradition I grew up with. But I have to say it’s now my favorite holiday. It’s simple, fun, not packed with the need to exchange gifts or forced jollity, and definitely not fueled by ridiculous amounts of alcohol (you know who you are St. Patrick’s/Cinco de Mayo). And it’s a chance to give thanks for the many blessings we have, among them is you, my colleagues. It’s a pleasure and an honour to work with so many fine, kind, committed individuals. I wish you all the most lovely of Thanksgivings.”

Kevin, the pleasure and honour was most certainly ours. We were lucky to know you, and we will miss you tremendously.

Lisa Kadyk, Associate Director, Therapeutics Development

I had the privilege of crossing paths with Kevin on numerous occasions. He always had a spark about him! I was new to the stem cell world and he had a way of making my butterflies relax. I will always remember his true genuine dedication to the field of stem cells and all the progress that was made because of Kevin. His mark in this world will never be forgotten.

Rich Lajara, ICOC Board member

This feels like a death in the stem cell family. I usually came away from talking to Kevin feeling better than before the conversation. He had a positive, uplifting effect on people. I appreciated his bluntness, which was usually coupled with that unique sense of humor of his.

I’ll miss being able to just talk to him about anything. Maybe get his thoughts on dilemmas in the stem cell field or what the best IPA in Portland might be, which was the last thing that he and I discussed recently. I was at a conference there and he told me to get outside and go have a beer. Cheers to Kevin. Many great memories.

Dr. Paul Knoepfler, Professor, UC Davis

Long before CIRM, Kevin was a valued member of the KRON TV news team in San Francisco. He was always the one who kept us calm in the face of pressing news deadlines. We could count on his consummate professionalism, his sense of humor and that lovely Irish brogue! Working with him at CIRM simply reminded me of what journalism has lost but CIRM had gained.

Ysabel Duron, Board Member, Chair, Communications Subcommittee

Kevin was such a gentleman. He was always very helpful to me, and with a smile on his face.

Dr. Joseph Wu, Director, Stanford Cardiovascular Institute

I feel a sense of loss when I think of Kevin but, immediately after, I know he is and will be present in my memories forever. He was and will always be such a wonderful, kind and sensitive human being. We never met personally -I’ve done many translations for CIRM and sent them by email- but we connected in a profound way because both of us cared very much for the evolution of humanity and the importance of science.

He valued my good intentions and dedication to help CIRM. I always thanked him for that and will always cherish that he knew I resonated with his noble cause.

My deepest condolences to his family and loved ones.

May the spirit of Kevin remain in our hearts and may he inspire us to be more sensitive and kinder towards humanity, and believe -perhaps sometimes like a Quixote- that the impossible quest of a better world… if we dream, may become possible.

And Kevin, may your new journey be full of light and peace.

— Marcela Grad, CIRM Translator

Dear Kevin,

You were a wonderful, kind, warm colleague whom I considered a friend. You always responded quickly and said “yes” with a smile. Your memory will always be for a blessing. When it’s 10am on Thursdays I will think of you.

Jacqueline Hantgan, Senior Advisor, Community Outreach and Engagement

Kevin was someone I could always turn to for guidance, support, and reassurance that things would be okay. I will forever remember him as a friend and mentor. I miss him.

Esteban Cortez, Director of Marketing & Communications

I met Kevin for the first time in late 2012 when I was in a dark phase in my life. I had just become paralyzed from a spinal cord injury a year earlier and was having a very hard time adjusting. Kevin was the one who got me interested in patient advocacy. His enthusiasm, optimism, and passion were so contagious that he always somehow managed to fool me into believing that I too was articulate and smart enough to give public presentations and interviews like he did.

I admired Kevin so much. I wanted to emulate him as much as I could. When Kevin complimented my writing, it felt like I had just won an Oscar. I still had so many questions to ask him. I still had so much to learn from him. Our time was cut too short.

I feel like I’ve experienced so many major life moments with Kevin over the last 10 years. Kevin was the first one to volunteer to help me when I was applying to transfer from USC to Stanford. After getting rejected, he wrote me a lengthy email reassuring me that everything happens for a reason. I had to go back and re-read it. He was right. Not getting into Stanford that year was the best thing that happened to me.

At every milestone, Kevin was there cheering me on. Graduating from university, moving to Paris and then London. Getting married. House hunting. Everything I did, Kevin was a part of it. He heard about it first and he was always so ecstatic for me. For years I’d tell him, “Kevin one day I’m moving back to the Bay Area so I can work for you.” I wouldn’t be at CIRM if it wasn’t for Kevin. So many things in my life have happened because of the direction and guidance he gave me early on in my injury.

Kevin, you were a legend. An extraordinary writer, human being, and friend. I will miss your sense of humor. I will miss your kind words. Thank you for believing in me. Thank you for giving me purpose. I’m so lucky to have met you in this lifetime and to have called you, my friend.

Katie Sharify, Communications Team Coordinator

Kevin was a man of great wit and humor and I greatly appreciated his contributions to CIRM. He was brilliant at anticipating what information was insightful to share and I valued his efforts to keep the website current with great information. I also greatly appreciated his ability to distill complicated science down to understandable and fun whiteboard presentations. I learned so much from him and will miss him tremendously. I am sorry to hear about his passing.

James Stewart, Multiple Sclerosis Patient Advocate

I am devastated by the news about Kevin. He was a consummate professional, committed to telling the stem cell stories, with just the right blend of seriousness and humor. His passing is a big loss for biomedical research, the patient communities for which he was an excellent advocate, and humanity. He was a mensch and I will miss him.

Steven Peckman, Deputy Director, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research

Kevin’s energy, enthusiasm, humor and personal touch lifted CIRM and all of us working towards CIRM’s mission. I am already missing you Kevin.

Andrew McMahon, Director, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research

What sad news, and what a significant loss. I remember Kevin as incredibly dedicated, endlessly resourceful, upbeat, humorous and kind spirited. He left CIRM and the world a better place.

Michael Yaffe, Fmr. CIRM Associate Director of Research Activities

I met Kevin at a meeting shortly after the creation of CIRM. He had organized a presentation for patient advocates at the Broad Center in downtown Los Angeles. He seemed like a good guy, but you have to understand what it means to be a patient advocate for a chronic disease like Sickle Cell. At that time, around 2008, nothing much had been happening in terms of research. Sickle cell is at the nexus of poverty, racism, and a poorly funded healthcare system, no matter how nice a person might seem, we advocates are very cautious about who we trust, we have been disappointed too many times.

Kevin McCormack earned our trust many times over. He reached out for our input and listened to our concerns. He helped us become patient advocates for promising research studies. Through his influence I was able to speak on a panel at the ISSCR conference, held last June in San Francisco. Researchers at the highest levels heard my presentation about the gaps between findings in the lab and treatment at the bedside. Our panel discussion from the conference, under the leadership of Dr. Jeremy Sugarman, will be published in 2023.

Kevin knew I was a retired educator so our talks and email exchanges often focused on education. These discussions led to me making a presentation at the Bridges Conference in San Diego. It was very rewarding to see the hundreds of bright young people looking forward to a career in scientific research. I have a feeling that Kevin was a large part of this effort.

Kevin’s leadership style should serve as a model for CIRM: he listened, he showed appreciation, he followed through. He ended every email with his signature response, “cheers”. Kevin’s influence will be missed by sickle cell patients and their advocates in the community.

Nancy Rene, Educator/Advocate Sickle Cell Disease and Autism

The Irish have a saying that only the good die young. In the case of Kevin, this is particularly true. So it is only appropriate that we send him off with a bit of poetry, a glass to his lips and a scholarly reference.
“The Parting Glass” is a Scottish/Irish traditional poem, often sung at the end of a gathering of friends. It is only fitting that we send Kevin off with the “Parting Glass”. Historically, this was the final hospitality offered to a departing guest today or yesteryear. The “Parting Glass” can be sung and done so with flourish and ornamentation, becoming a fluttering kind of melody. Like Kevin himself. The flourish should remind us all of what a profoundly inspirational enrichment Kevin had on all of our lives. He also would also have wanted an appropriate reference.

Robert Burns (1786). Poems, chiefly in the Scottish dialect. Kilmarnock: J. Wilson. p. 228. hdl:2027/uc1.31175019497166.

The Parting Glass

Of all the money that e’er I had
I spent it in good company
And all the harm I’ve ever done
Alas it was to none but me
And all I’ve done for want of wit
To mem’ry now I can’t recall
So fill to me the parting glass
Good night and joy be to you all

So fill to me the parting glass
And drink a health whate’er befall,
And gently rise and softly call
Good night and joy be to you all

Of all the comrades that e’er I had
They’re sorry for my going away
And all the sweethearts that e’er I had
They’d wish me one more day to stay
But since it falls unto my lot
That I should rise and you should not
I gently rise and softly call
Good night and joy be to you all

If I had money enough to spend
And leisure time to sit awhile
There is a fair maid in this town
That sorely has my heart beguiled.
Her rosy cheeks and ruby lips
I own she has my heart in thrall
Then fill to me the parting glass
Good night and joy be with you all.

A man may drink and not be drunk
A man may fight and not be slain
A man may court a pretty girl
And perhaps be welcomed back again
But since it has so ought to be
By a time to rise and a time to fall
Come fill to me the parting glass
Good night and joy be with you all
Good night and joy be with you all

John Cashman, President, Human BioMolecular Research Institute (and a fellow Irishman)

There are few critical pillars in Ronav’s life and Kevin was one of them as he first introduced CIRM to us when Ronav was fighting SCID at UCSF. We immediately came close to each other and met several times after Ronav’s treatment, sometimes as patient advocates, sometimes advising congress for funding at Sacramento Capitol but most importantly we recently met in July at our home for lunch. We agreed to meet soon at Biryani party. He was thrilled to meet all of us. He loved Ronav. We always admired his generosity. Everting he met us with same kind of love and warmth. This is very sad.

This a photo from his recent visit in July at our home in Sacramento. This is so painful to write. May his soul rest in peace. Our thoughts and prayers for his family.

— Pawash Priyank and Upasana Thakur

We will miss Kevin forever. His enthusiasm for medical journalism was contagious.

Several years ago, I asked him if he could talk to our local high school journalism class; as expected, he did a fabulous job. His professional talent was matched by unwavering kindness. He always went beyond the call of duty to meet people where they were in their life, help them and make them feel better.

My sincere condolences to his immediate family and the CIRM team, his second family. Since his abrupt and tragic departure from our world, the days are not as bright as they seem when looking outside in sunny California.

— Anne-Marie Duliege, ICOC Board Member

If you would like to share any words, memories or photos to pass on to Kevin’s family, please send them to Katie Sharify at ksharify@cirm.ca.gov.

Three women with ties to CIRM featured in 2022 Women in Biopharma list 

Endpoints News released its 2022 Women in Biopharma list, which recognizes 20 of the top women leading biopharma research and development (R&D).  

This year, the publication received more than 500 entries and selected 20 women “who have blazed trails and are still promising to reshape biopharma R&D for years to come.” 

There are many amazing finalists featured this year, but three in particular stood out for their ties to the California Institute for Regenerative Medicine (CIRM). Those three are:  

Jennifer Gordon, Ph.D. — Senior Vice President of Research and Development at Excision BioTherapeutics 

Dr. Jennifer Gordon and the team at Excision Bio Therapeutics have developed a therapeutic candidate called EBT-101. CIRM is funding a clinical trial to test EBT-101 in patients with HIV. 

This is the first clinical study using the CRISPR-based platform for genome editing and excision of the latent form of HIV-1, the most common form of the virus that causes AIDS in the US and Europe.  

The goal of the treatment is to eliminate or sufficiently reduce the hidden reservoirs of virus in the body to the point where the individual is effectively cured. 

Barbara Wirostko — Co-founder and CMO at Qlaris Bio 

Barbara Wirostko is the co-founder and CMO at Qlaris Bio, a clinical stage biotech company committed to developing therapies for patients suffering from serious and debilitating ophthalmic diseases.  

In addition to her work there, Barbara is a member of CIRM’s Grants Working Group (GWG), which is responsible for evaluating the scientific merit of all applications submitted to CIRM and provides funding recommendations to the CIRM board.  

In the Endpoints News profile, Wirostko shares that she was inspired by her father, also an ophthalmologist, and his desire to help people. 

“I think that was the other thing that really drew me to ophthalmology — is that you were able to work with patients, make a difference in people’s lives, also have a surgical as well as a medical aspect, practicing medicine, and then also have a family,” she said. 

Dr. Lili Yang – UCLA associate professor, Co-founder of Appia Bio and Immune Design 

We’ve written about Dr. Lili Yang’s work on the Stem Cellar blog.  

Dr. Yang at UCLA was recently awarded $1.4 million by CIRM to develop an off-the-shelf cell therapy for ovarian cancer, which causes more deaths than any other cancer of the female reproductive system. 

With support from several CIRM grants, Dr. Yang has developed a platform that can use healthy donor blood stem cells to produce clinical scalable “off-the-shelf” iNKT cells. That has led to the creation of start-up company Appia Bio, and talks with the FDA about testing a series of iNKT cell products in clinical trials. 

“I have this dream that cell therapy can become off-the-shelf, and how this would really help all cancer patients in need. The current cancer cell therapy requires treating patients one-by-one, resulting in a steep price that is hard to afford,” Dr. Yang says.  

“Not everyone lives near a hospital capable of handling such a personalized therapy or can afford such a steep price. If we can make this therapy with centralized manufacturing, pre-quality controlled and ready for wide use then we don’t need to worry about the gender or age or location of the patient.” 

CIRM congratulates all the extraordinary women featured in the Endpoints News 2022 Women in Biopharma list. To see all the finalists, read the official announcement here or visit the Endpoints News website.  

Update on spinal cord injury patient enrolled in CIRM-funded stem cell clinical trial

Jake Javier and his parents at Duke University

A spinal cord injury (SCI) is devastating, changing a person’s life in an instant. Every year, around the world, between 250,000 and 500,000 people suffer a spinal cord injury. Most of these are caused by trauma to the spinal column, thereby affecting the spinal cord’s ability to send and receive messages from the brain to the body’s systems that control sensory, motor and autonomic function below the level of injury.

Currently, there is nothing that completely reverses SCI damage and most treatment is aimed at rehabilitation and empowering patients to lead as normal a life as possible under the circumstances. Improved treatment options are necessary both to improve patients’ overall quality of life, and to reduce associated healthcare costs.

In 2010, the Geron trial became not only the first clinical trial to be funded by the California Institute for Regenerative Medicine (CIRM), but the first clinical trial in the world using embryonic stem cells.

By 2014, Asterias Biotherapeutics (now Lineage Cell Therapeutics Inc.), acquired the cell therapy assets of Geron and launched its Phase 1/2a clinical trial with the goal of determining the safety of the therapy and the optimal dose of cells to transplant into patients.

In 2016, Jake Javier became the fifth patient to participate in the revived Asterias trial. Regular readers of our blog will remember that Jake is the young man who broke his neck the day before he graduated high school, leaving him paralyzed from the upper chest down.

After enrolling in the CIRM-funded Asterias clinical trial, and receiving a transplant of ten million stem cells, Jake regained enough use of his arms and hands to be able to go to Cal Poly and start his life over.

This video highlights the struggles and challenges he faced in his first year, and his extraordinary spirit in overcoming them.

Video courtesy of Matt Yoon and his team at Cal Poly

Today, Jake is set to graduate from Duke University with his master’s degree in Biomedical Engineering, with plans to help those impacted by neurological injuries or disease.

Watch the video below to learn more about Jake’s personal perspective on his clinical trial participation, the OPC1 clinical study, his future plans and his message to the SCI community.

Video courtesy of Lineage Cell Therapeutics Inc.

Lab-made retinas offer a new approach to battling vision loss

Age-related macular degeneration (AMD) is the leading cause of vision loss in the elderly. Now, new research using 3D organoid models of the eye has uncovered clues as to what happens in AMD, and how to stop it. 

In AMD, a person loses their central vision because the light sensitive cells in the macula, a part of the retina, are damaged or destroyed. This impacts a person’s ability to see fine details, recognize faces or read small print, and means they can no longer drive. 

AMD causes blurry and distorted vision 

No one is quite sure what causes AMD, but in a study in the journal Nature Communications, German researchers used miniature human retina organoids to get some clues.  

Building a better model for research

Organoids are 3D models made from human cells that are grown in the lab. Because they have some of the characteristics of a human organ—in this case the retina—they help researchers better understand what is happening in the AMD-affected eye. 

In this study they found that photoreceptors, the light sensitive cells at the back of the retina, were missing but there was no sign of dead cells in the organoid. This led them to suspect that something called cell extrusion was at play.  

Cell extrusion is where a cell exports or sends large particles outside the cell. In this case it appeared that something was causing these photoreceptors to be extruded, leading to the impaired visual ability.  

In a news release Mark Karl, one of the authors of the study, said, “This was the starting point for our research project: we observed that photoreceptors are lost, but we could not detect any cell death in the retina. Half of all photoreceptors disappeared from the retinal organoid within ten days, but obviously they did not die in the retina. That made us curious.” 

Using snakes to fight AMD 

Further research identified two proteins that appeared to play a key role in the process, triggering the degeneration of the retinal organoid. They also tested a potential therapy to see if they could stop the process and save the photoreceptors. The therapy they tried, a snake venom, not only stopped the photoreceptors from being ejected, but it also prevented further damage to the retinal cells. 

Karl says this is the starting point for the next step in the research. “This gives hope for the development of future preventive and therapeutic treatments for complex neurodegenerative diseases such as AMD.” 

CIRM’s fight against blindness 

The California Institute for Regenerative Medicine (CIRM) has funded six clinical trials targeting vision loss, including one for AMD. We recently interviewed Dr. Dennis Clegg, one of the team trying to develop a treatment for AMD and he talked about the encouraging results they have seen so far. You can hear that interview on our podcast “Talking ‘Bout (re)Generation.” 

Bubble baby treatment cleared to restart clinical trial

Evie Vaccaro: Photo courtesy Nancy Ramos

Three families battling a life-threatening immune disorder got some great news last week. A clinical trial that could save the life of their child has once again been given the go-ahead by the US Food and Drug Administration (FDA).

The clinical trial is the work of UCLA’s Dr. Don Kohn, and was strongly supported by CIRM. It is targeting ADA-SCID, a condition where the child is born without a functioning immune system so even a simple infection could prove fatal. In the past they were called “bubble babies” because some had been placed inside sterile plastic bubbles to protect them from germs.

Dr. Kohn’s approach – using the patient’s own blood stem cells, modified in the lab to correct the genetic mutation that causes the problem – had shown itself to be amazingly effective.  In a study in the prestigious New England Journal of Medicine, the researchers showed that of 50 patients treated all had done well and 97 percent were considered cured.

UCLA licensed the therapy to Orchard Therapeutics, who planned to complete the testing needed to apply for permission to make it more widely available. But Orchard ran into problems and shelved the therapy.

After lengthy negotiations Orchard returned the therapy to UCLA last year and now the FDA has given clearance for UCLA to resume treating patients. That is expected to start early next year using CIRM funds left over when Orchard halted its work.

One of the people who played a big role in helping persuade Orchard to return the therapy to UCLA is Alysia Vaccaro. She is the mother of Evie, a child born with ADA-SCID who was cured by Dr. Kohn and his team and is now a thriving 9 year old.

You can watch an interview we did with Alysia about the impact this research has had on her family, and how important it is for other families with ADA-SCID kids.