Patient Advocacy

CIRM approves $2.5 million to establish a Patient Support Program 

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment
Hataalii Tiisyatonii Begay (HT) participated in a CIRM-funded clinical trial at UCSF for Artemis-SCID, a condition that can be life-threatening or fatal. Photo courtesy Barbara Ries / UCSF. 

The California Institute for Regenerative Medicine (CIRM) funds innovative cell and gene therapy research and educational programs to advance regenerative medicine in the Golden State. Just as important is CIRM’s commitment to ensuring that Californians from diverse backgrounds have access to groundbreaking clinical trials for a variety of diseases and condition resulting from the research.  

That’s why CIRM has approved awarding $2.5 million to EVERSANA—a leading provider of global commercial services to the life sciences industry—to establish a Patient Support Program (PSP) to assist patients enrolled in CIRM-funded clinical trials. 

Addressing Barriers to Clinical Trials 

For many patients battling diseases and chronic health conditions, getting access to a clinical trial can be lifesaving, but it can also be very challenging. Clinical trial patients often face financial challenges, long-distance travels, and require family commitments that can make it difficult to maintain participation.  

Through this award, CIRM and EVERSANA will address informational, financial and logistical bottlenecks experienced by clinical trial patients and their family members. The Patient Support Program will be particularly important for providing equal access to California clinical trial participants.  

Evie Junior (left) participated in a CIRM-funded clinical trial at UCLA for sickle cell disease. Photo courtesy UCLA Broad Stem Cell Research Center.  

Evidence shows that support programs positively impact clinical trial accrual rates, patient diversity, trial adherence, humanistic outcomes, reduced healthcare utilization costs, and quality of life. 

Encouraging Trial Participation in Underserved Communities 

Services offered by the PSP will include maintaining a Patient Support Center to refer patients to clinical trials, verifying participation and financial support eligibility, as well as administering Patient Assistance Fund (PAF) reimbursements to cover travel expenses, meals, accommodations, childcare, and other out of pocket expenses. 

Funds for the PSP are set aside under Proposition 14, the voter-approved initiative that renewed CIRM’s funding in 2020.  

Under Prop 14, royalty revenues that CIRM grantees earn from licensing, inventions or technologies are to be spent “offsetting the costs of providing treatments and cures arising from institute-funded research to California patients who have insufficient means to purchase such treatment or cure, including the reimbursement of patient-qualified costs for research participants.” 

Currently, CIRM has been appropriated $15.6 million from the Patient Assistance Fund to support patients. 

“CIRM is committed to supporting patients through the clinical trial process to continue advancing transformative regenerative medicine therapies to the benefit of all Californians,” said Jonathan Thomas, PhD, JD, CIRM’s Interim President and CEO. “The Patient Support Program is just one initiative designed to get us a step closer to that vision.” 

EVERSANA is expected to initiate work on the approved project plan within 120 days of the final contract. 

Reasons to be thankful this Thanksgiving: creative nerds

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We at the California Institute for Regenerative Medicine have a lot to be thankful for this Thanksgiving. We get to work with some extraordinary colleagues, we get to know some remarkable patient advocates who are pioneers in volunteering for stem cell and gene therapies, and we have a front row seat in a movement that is changing the face of medicine.

We also get to work with some brilliant scientists and help support their research. As if we needed any reminders of how important that funding is, we thought we would share this video with you. It’s from the talented post docs and researchers at the University of California San Diego. It’s a delightful parody of the Cyndi Lauper classic “Girls Just Wanna Have Fun”. Only in this case it’s “Nerds Just Wanna Have Funds.”

Enjoy, and Happy Thanksgiving.

A timeless message about stem cells

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Dr. Daniel Kota

The world of stem cell research is advancing rapidly, with new findings and discoveries seemingly every week. And yet some things that we knew years ago are still every bit as relevant today as they were then.

Take for example a TEDx talk by Dr. Daniel Kota, a stem cell researcher and the Director, Cellular Therapy – Research and Development at Houston Methodist.

Dr. Kota’s talk is entitled: “Promises and Dangers of Stem Cell Therapies”. In it he talks about the tremendous potential of stem cells to reverse the course of disease and help people battle previously untreatable conditions.

But he also warns about the gap between what the science can do, and what people believe it can do. He says too many people have unrealistic expectations of what is available right now, fueled by many unscrupulous snake oil salesmen who open clinics and offer “treatments” that are both unproven and unapproved by the Food and Drug Administration.

He says we need to “bridge the gap between stem cell science and society” so that people have a more realistic appreciation of what stem cells can do.

Sadly, as the number of clinics peddling these unproven therapies grows in the US, Dr. Kota’s message remains all too timely.

Patient Advocacy is its own reward

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It’s always nice to be told you are doing a good job. It’s even nicer when it’s unexpected. That’s certainly the case when we, the Communications Team at the California Institute for Regenerative Medicine, found out we’d been named as a finalist for the Patient Advocacy Award (non-profit category) as part of the Phacilitate Advanced Therapies Awards.

To be honest, we didn’t even know we’d been nominated. But who cares. We are now in the final. And we are in good company. Our friends at Americans for Cures, were also nominated. They are advocates for stem cell research in California and were hugely instrumental in getting Proposition 14 passed in 2020, that’s the voter initiative that refunded CIRM with $5.5 billion.

The other finalists are the Alliance for Cancer Gene Therapy and the Rare Advocacy Movement.

While we may focus on different areas we all share a common goal, a desire to ensure that the voice of the patient is front and center in all that we do. At CIRM we have patient advocates on our Board and on the panel of experts who review applications for our funding. We have patient advocates helping guide the clinical trials we fund. And now, as we expand our efforts to reach out in every community in California, we have patients and patient advocates guiding that work as well.

We do this work because it’s important and because, without the support of the patient advocacy community, we wouldn’t be here.

It’s an old cliché that when you are in this position you say, “it’s an honor just to be nominated.” But in this case, it’s true.

Strength forged from adversity

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Regina Karchner – Photo courtesy Nancy Ramos Photography

Our 2021-22 Annual Report is now online. It’s filled with information about the work we have done over the last year (we are on a fiscal calendar year from July 1 – June 30), the people who have helped us do that work, and some of the people who have benefited from that work. One of those is Regina Karchner. 

Regina Karchner says she feels as if she’s been a patient advocate for people with brain cancer almost from birth. When she was three, her father died of a brain tumor. When she was 16 Regina was diagnosed with brain cancer. While she was in the hospital she heard about the Children’s Brain Tumor Foundation (CBTF) and as soon as she was able she became a volunteer with the organization. Today she is a social work regional coordinator at CBTF.  

She says that as an advocate she feels she has a responsibility to help families deal with devastating news, to talk about death, and how to cope with the emotional trauma of it. She also advocates on behalf of survivors, like herself.  

“I am just such an advocate for the need for long term programming for brain cancer survivors, because it’s so different from other cancers. The emotional, cognitive and physical impacts of brain tumors are dramatic, that’s even if the individuals survive.  

“We are working with people in their 40’s who were the first group of childhood survivors and there’s nowhere to go that matches their needs, they can’t function enough to live independently and work full time. It’s a big problem in the medical world and even in schools, they don’t understand brain tumors, they don’t see it as a traumatic brain injury which it is and even the most well-intended schools don’t really know what to do or handle the patients.” 

“We found that survivors with better social skills have a better quality of life, so we are now trying to focus on kids in elementary school, giving them the social skills they need to survive and that are hard to catch up on later in life. They can get math or history or other subjects anytime, but the social skills are essential” 

Regina also serves on a CIRM Clinical Advisory Panel or CAP for a clinical trial for children with brain cancer. She says having the patient advocate at the table is vital to the success of the trial. “I help the researchers understand the needs of the patient, even understand why families don’t enroll in trials. 80% of families who have kids with brain tumors are on Medicaid so it’s a select group of people who can afford to be in these trials. Letting the researchers know that and coming up with ways to help them is so important.” 

She says it’s challenging work, but also very rewarding. “It feels wonderful to help families in a time of need. I feel I grow as a person and as a parent, I have learnt so much that helps me in my personal life and being grateful for having a healthy family and being a healthy survivor myself.”  

Fighting for his life and the lives of other stroke survivors

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Sean Entin, stroke survivor and founder of Stroke Hacker

The word “miraculous” gets tossed around a lot in the world of medicine, mostly by people who have made an unexpected recovery from a deadly or life-threatening condition. In Sean Entin’s case calling his recovery from an almost-fatal stroke could be called miraculous, but I think you would also have to say it’s due to hard work, determination, and an attitude that never even considered giving up.

Sean had a stroke in 2011. Doctors didn’t think he’d survive. He was put into a coma and underwent surgery to create an opening in his skull to give his brain time and space to heal. When he woke he couldn’t walk or talk, couldn’t count. Doctors told him he would never walk again.

They didn’t know Sean. Fast forward to today. Sean is active, has completed two 5k races – that’s two more than me – and has created Stroke Hacker, a program designed to help others going through what he did.

Sean is a remarkable man, which is why I sat down to chat with him for the latest episode of the California Institutes for Regenerative Medicine’s podcast, ‘Talking ‘Bout (re)Generation’.

He is a fascinating man, and he makes for fascinating company. Enjoy the podcast.

The California Institute for Regenerative Medicine (CIRM) has invested more than $80 million in stroke research, including one clinical trial currently underway.

Taking to the streets with Pride

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Yesterday the CIRM team were honored to be part of the San Francisco Pride Parade. To walk along the route with colleagues and friends, surrounded by hundreds of thousands of cheering people was such a fun way to spend the day, and gave us a chance to introduce ourselves to many people who may not have known who we were (although I did get several people saying “I voted for you” and “Go Regenerative Medicine”). To be able to share in the joy that people clearly felt at having the parade back again after a Covid-inflicted absence was just a delight. Here’s some images of the day.

People were creative in finding the best spot to view the parade
Treecy brought Uma Thurman (no, that is her name) along for the march
There were lots of great signs, some of which we can even show on a public website!
Even the dogs were stylish

Can regenerative medicine turn back the clock on aging?

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One of my favorite phrases is “standing room only”. I got a chance to use it last week when we held a panel discussion on whether regenerative medicine could turn back the clock on aging. The event was at the annual conference of the International Society for Stem Cell Research (ISSCR) and more than 150 people packed into a conference room to hear the debate (so far more than 800 also watched a live stream of the event.)

It’s not surprising the place was jammed. The speakers included:

  • Dr. Deepak Srivastava, the President of the Gladstone Institutes, an expert on heart disease and the former President of ISSCR.
  • Dr. Stanley “Tom” Carmichael, Chair of the Department of Neurology at UCLA and an expert on strokes and other forms of brain injury.
  • Adrienne Shapiro, the mother of a daughter with sickle cell disease, a tireless patient advocate and supporter of regenerative medicine research, and the co-founder of Axis Advocacy, a family support organization for people with sickle cell.
  • Jonathan Tomas, PhD, JD, the Chair of the CIRM Board.

And the topic is a timely one. It is estimated that as many as 90 percent of the people who die every day, die from diseases of aging such as heart disease, stroke, and cancer. So, what can be done to change that, to not just slow down or stop these diseases, but to turn back the clock, to repair the damage already done and replace cells and tissues already destroyed.

The conversation was enlightening, hopeful and encouraging, but also cautionary.

You can watch the whole event on our Youtube channel.

I think you are going to enjoy it.

Two reasons to remember June 19th

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Today marks two significant events for the Black community. June 19th is celebrated as Juneteenth, the day when federal troops arrived in Galveston, Texas to ensure that the enslaved people there were free. That moment came two and a half years after President Abraham Lincoln signed the Emancipation Proclamation into law.

June 19th is also marked as World Sickle Cell Awareness Day. It’s an opportunity to raise awareness about a disease that affects around 100,000 Americans, most of them Black, and the impact it has on the whole family and entire communities.

Sickle cell disease (SCD) is an inherited blood disorder that is caused by a genetic mutation. Instead of red blood cells being smooth and round and flowing easily through arteries and veins, the cells are sickle shaped and brittle. They can clog up arteries and veins, cutting off blood to vital organs, causing intense pain, organ damage and leading to premature death.

SCD can be cured with a bone marrow transplant, but that’s a risky procedure and most people with SCD don’t have a good match. Medications can help keep it under control but cannot cure it. People with SCD live, on average, 30 years less than a healthy adult.

CIRM has invested almost $60 million in 13 different projects, including five clinical trials, to try and develop a cure for SCD. There are encouraging signs of progress. For example, in July of 2020, Evie Junior took part in a CIRM-funded clinical trial where his own blood stem cells were removed then, in the laboratory, were genetically modified to repair the genetic mutation that causes the disease. Those cells were returned to him, and the hope is they’ll create a sickle cell-free blood supply. Evie hasn’t had any crippling bouts of pain or had to go to the hospital since his treatment.

Evie Junior: Photo by Jaquell Chandler

CIRM has also entered into a unique partnership with the National Heart, Lung and Blood Institute (NHLBI) to co-fund cell and gene therapy programs under the NIH “Cure Sickle Cell” initiative.  The goal is to markedly accelerate the development of cell and gene therapies for SCD.

“There is a real need for a new approach to treating SCD and making life easier for people with SCD and their families,” says Adrienne Shapiro, the mother of a daughter with SCD and the co-founder of Axis Advocacy, a sickle cell advocacy and education organization. “Finding a cure for Sickle Cell would mean that people like my daughter would no longer have to live their life in short spurts, constantly having their hopes and dreams derailed by ER visits and hospital stays.  It would mean they get a chance to live a long life, a healthy life, a normal life.”

We will all keep working together to advance this research and develop a cure. Until then Juneteenth will be a reminder of the work that still lies ahead.

Join us to hear how stem cell and gene therapy are taking on diseases of aging

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It is estimated that as many as 90 percent of people in industrialized countries who die every day, die from diseases of aging such as heart disease, stroke, and cancer. Of those still alive the numbers aren’t much more reassuring. More than 80 percent of people over the age of 65 have a chronic medical condition, while 68 percent have two or more.

Current medications can help keep some of those conditions, such as high blood pressure, under control but regenerative medicine wants to do a lot more than that. We want to turn back the clock and restore function to damaged organs and tissues and limbs. That research is already underway and we are inviting you to a public event to hear all about that work and the promise it holds.

On June 16th from 3p – 4.30p PST we are holding a panel discussion exploring the impact of regenerative medicine on aging. We’ll hear from experts on heart disease and stroke; we will look at other ground breaking research into aging; and we’ll discuss the vital role patients and patient advocates play in helping advance this work.

The discussion is taking place in San Francisco at the annual conference of the International Society for Stem Cell Research. But you can watch it from the comfort of your own home. That’s because we are going to live stream the event.

Here’s where you can see the livestream: https://www.youtube.com/watch?v=CaUgsc5alDI

And if you have any questions you would like the panel to answer feel free to send them to us at info@cirm.ca.gov