Rare disease gets go-ahead to run clinical trial

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A young girl with cystinosis: Photo courtesy CRF

Cystinosis is one of those diseases most people have never heard of and should be very grateful they haven’t. It’s rare – affecting only around 500 children and young adults in the US and just 2,000 people worldwide – but it’s nasty. Up to now the treatments for it have been very limited. But a new clinical trial, just given the go-ahead by the Food and Drug Administration (FDA), could help change that.

Cystinosis usually strikes children before they are two years old and can lead to end stage kidney failure before their tenth birthday. It is caused by a genetic mutation that allows an amino acid, cysteine, to build up in and damage the kidneys, eyes, liver, muscles, pancreas and brain.

There is one approved therapy, cysteamine, but this only delays progression of the disease, has severe side effects and people taking it still require kidney transplants, and develop diabetes, neuromuscular disorders and hypothyroidism.

All those are reasons why, in September 2016, the CIRM Board approved $5.2 million for U.C. San Diego researcher Stephanie Cherqui, Ph.D. and her team to try a different approach. Their goal is to take blood stem cells from people with cystinosis, genetically-modify them to remove the mutation that causes the disease, then return them to the patient. The hope is that the modified blood stem cells will create a new, healthy, blood system free of the disease.

Results from pre-clinical work testing this approach in mice have been so encouraging that the FDA has given the go-ahead for that work to now be tested in people.

In a news release Nancy Stack, the Founder and President of the Cystinosis Research Foundation (CRF), the largest provider of grants for cystinosis research in the world, says this is exciting news for a community that has been waiting for a breakthrough:

“We are thrilled that CRF’s dedication to funding Dr. Cherqui’s work has resulted in FDA approval for the first-ever stem cell and gene therapy treatment for individuals living with cystinosis. This approval from the FDA brings us one step closer to what we believe will be a cure for cystinosis and will be the answer to my daughter Natalie’s wish made fifteen years ago, ‘to have my disease go away forever.’ We are so thankful to our donors and our cystinosis families who had faith and believed this day would come.”

Dr. Cherqui says if this is successful it could help more than just people with cystinosis:

“We were thrilled that the stem cells and gene therapy worked so well to prevent tissue degeneration in the mouse model of cystinosis,. This discovery opened new perspectives in regenerative medicine and in the application to other genetic disorders. Our findings may deliver a completely new paradigm for the treatment of a wide assortment of diseases including kidney and other genetic disorders. If so, CRF, through their years of support will have helped an untold number of patients with untreatable, debilitating diseases.”

Those with questions on the trials can call toll free: 844-317-7836 (STEM) and/or visit www.cystinosisresarch.org

Frustration, failure and finally hope in the search for treatments for spina bifida

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Dr. Diana Farmer and her team at UC. Davis

By any standards Dr. Diana Farmer is a determined woman who doesn’t let setbacks and failure deter her. As a fetal and neonatal surgeon, and the chair of the Department of Surgery at UC Davis Health, Dr. Farmer has spent years trying to develop a cure for spina bifida. She’s getting closer.

Dr. Farmer and her partner in this research, Dr. Aijun Wang, have already shown they can repair the damage spina bifida causes to the spinal cord, in the womb, in sheep and bulldogs. Last year the CIRM Board voted to fund her research to get the data needed to apply to the US Food and Drug Administration for permission to start a clinical trial in people.

That work is so promising that we decided to profile Dr. Farmer in our 2018 Annual Report.

Here’s excerpts from an interview we conducted with her as part of the Annual Report.

I have been working on this since 2008. We have been thinking about how to help kids with spina bifida walk. It’s not fatal disease but it is a miserable disease.

It’s horrible for parents who think they are about to have a healthy child suddenly be faced with a baby who faces a life long struggle with their health, everything from difficulty or inability to walk to bowel and bladder problems and life-threatening infections.

As a fetal surgeon we used to only focus on fatal diseases because otherwise kids would die. But as we made progress in the field, we had the opportunity to help others who didn’t have a fatal condition, in ways we couldn’t have done in the past.

I’ve always been fascinated by the placenta, it has lots of protective properties. So, we asked the question if we were able to sample fetal cells from the placenta, could we augment those cells, and use them to tissue engineer spinal injuries, in the womb, to improve the outcome for kids with spina bifida?

Dr. Aijun Wang and I have been working on this project for the last decade.  Ten years of work has taken us to this point where we are now ready to move this to the next level.

It’s amazing to me how long this process takes and that’s why we are so grateful to CIRM because this is a rare disease and finding funding for those is hard. A lot of people are scared about funding fetal surgery and CIRM has been a perfect partner in helping bring this approach, blending stem cell therapy and tissue engineering, together.

If this therapy is successful it will have a huge economic impact on California, and on the rest of the world. Because spina bifida is a lifelong condition involving many operations, many stays in the hospital, in some cases lifelong use of a wheelchair. This has a huge financial burden on the family. And because this doesn’t just affect the child but the whole family, it has a huge psychological burden on families. It affects them in so many ways; parents having to miss work or take time off work to care for their child, other children in the family feeling neglected because their brother or sister needs so much attention.

In the MOMS Trial (a study that looked at prenatal – before birth – and postnatal – after birth – surgery to repair a defect in the spinal cord and showed that prenatal surgery had strong, long-term benefits and some risks) we showed that we could operate on the fetus before birth and help them. The fact that there was any improvement – doubling the number of kids who could walk from 20 to 40% showed this spinal cord injury is not a permanent situation and also showed there was some plasticity in the spinal cord, some potential for improvement. And so, the next question was can we do more. And that’s why we are trying this.

It’s pretty amazing. We are pretty excited.

The thing that makes surgeon-scientists feel so passionate is that we don’t just ask the fundamental questions, we ask questions in order to cure a problem in patients. I grew up in an environment where people were always asking “how can we do it better, how can we improve?”

There were many times of frustration, many times when cell types we explored and worked with didn’t work. But it’s the patients, seeing them, that keeps me motivated to do the science, to keep persevering. That’s the beauty of being a clinician-scientist. We can ask questions in a different way and look at data in a different way because we are driven by patient outcomes. So, whenever we get stuck in the rabbit hole of theoretical problems, we look to the patients for inspiration to keep going.

I am very cognizant of stirring up false hope, knowing that what occurs in animal models doesn’t always translate into humans. But we are optimistic, and I am anxious to get going.

 

By the numbers – a look at how the field of Regenerative Medicine is growing

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ARM State of the Industry briefing

The Golden State Warriors, the current US basketball champions – and your favorite Stem Cell Agency’s neighbors in Oakland – have a slogan, “Strength in Numbers”. That could well apply to the field of Regenerative Medicine because the field is growing in numbers, growing in strength, and growing in influence.

Yesterday, the Alliance for Regenerative Medicine (ARM), the organization that represents the field, held its annual State of the Industry briefing in San Francisco, detailing what happened in 2018. It was pretty impressive.

In fact, just the number of people in the room was impressive. More than 800 RSVP’d for the event, more than for any previous meeting, but even then the room was filled over capacity with many standing around the edges because there were no seats left.

ARM itself is growing, 32 percent last year, and now has more than 300 members. Other impressive numbers include:

  • 906 gene and cell therapy companies worldwide
  • 484 gene and cell therapy companies in the US alone
  • 1,028 clinical trials taking place worldwide
  • 598 of those clinical trials (58 percent of the total) are targeting cancer
  • 59,575 patients are slated to be enrolled in those trials

All those numbers are up dramatically on last year. You can see all the details on the ARM website.

Another sign the industry is growing comes in the amount of money being invested. When people are willing to pony up hard cash you know it’s a sign they believe in you. Last year the field raised $13.8 billion worldwide, that’s up a whopping 73 percent on 2017. That represented a strong year across all fronts from corporate partnerships to Initial Public Offerings (several CIRM-supported companies such as Orchard Therapeutics and Forty Seven Inc. are in that number) and venture capital investments.

Clearly there are still challenges ahead, such as figuring out ways to pay for these therapies when they are approved so that they are available to the people who need them, the patients.

One of the issues that is going to be front and center in 2019 is reimbursement and developing new payment models. But that in itself is a sign of a maturing field. In past years the emphasis was on developing new treatments. Now that those are in the pipeline, we’re working on ways to pay for them.

That’s progress.

The most popular Stem Cellar posts of 2018

The blog

You never know when you write something if people are going to read it. Sometimes you wonder if anyone is going to read it. So, it’s always fun, and educational, to look back at the end of the year and see which pieces got the most eyeballs.

It isn’t always the ones you think will draw the biggest audiences. Sometimes it is diseases that are considered “rare” (those affecting fewer than 200,000 people) that get the most attention.

Maybe it’s because those diseases have such a powerful online community which shares news, any news, about their condition of interest with everyone they know. Whatever the reason, we are always delighted to share encouraging news about research we are funding or encouraging research that someone else is funding.

That was certainly the case with the top two stories this year. Both were related to ALS or Lou Gehrig’s disease.  It’s a particularly nasty condition. People diagnosed with ALS have a life expectancy of just 2 to 5 years. So it’s probably not a big surprise that stories suggesting stem cells could expand that life span got a big reception.

Whatever the reason, we’re just happy to share hopeful news with everyone who comes to our blog.

And so, without further ado, here is the list of the most popular Stem Cellar Blog Posts for 2018.

All of us in the Communications team at CIRM consider it an honor and privilege to be able to work here and to meet many of the people behind these stories; the researchers and the patients and patient advocates. They are an extraordinary group of individuals who help remind us why we do this work and why it is important. We love our work and we hope you enjoy it too. We plan to be every bit as active and engaged in 2019.

Stem Cell Agency celebrates 50 clinical trials with patient #1

Yesterday the CIRM Board approved funding for our 50th clinical trial (you can read about that here) It was an historic moment for us and to celebrate we decided to go back in history and hear from the very first person to be treated in a CIRM-funded clinical trial. Rich Lajara was treated in the Geron clinical trial after experiencing a spinal cord injury, thus he became CIRM’s patient #1. It’s a badge he says he is honored to wear. This is the speech Rich made to our Board.

Rich Lajara

Hello and good afternoon everyone. It’s an honor to be here today as the 50th clinical trial has been officially funded by CIRM. It was feels like it was just yesterday that I was enrolled into the first funded clinical trial by CIRM and in turn became California’s’ 1st embryonic stem cell patient.

I became paralyzed from the waist down in September 2011. It was Labor Day and I was at a river with some close friends. There was this natural granite rock slide feature next to a waterfall, it was about 60 feet long; all you had to do was get a bucket of water to get the rocks wet and slide down into a natural pool. I ended up slipping and went down head first backwards but was too far over and I slid off a 15’ ledge where the waterfall was. I was pulled from the water and banged up pretty bad. Someone said “look at that deformity on his back” and tapped my leg and asked if I could feel that. I knew immediately I was paralyzed. I thought this was the end, little did I know this was just the beginning. I call it being in the wrong place at the right time.

So, after a few days in the hospital of course everyone, as well as myself, wanted a cure. We quickly learned one didn’t exist. A close family friend had been making phone calls and was able to connect with the Christopher & Dana Reeve Foundation and learned about a clinical trial with “stem cells”. One of my biggest question was how any people have done this? “Close to none”, I was told.

I was also told I most likely would have no direct benefit as this was a safety trial? So why do it at all? Obviously at that time I was willing to overlook the “most likely” part because I was willing to do anything to try and get my normal life back.

Looking back the big picture was laying the ground work for others like Kris or Jake (two people enrolled in a later version of this trial). At the time I had no clue that what I was doing would be such a big deal. The data collected from me would end up being priceless. It’s stories like Jake’s and Kris’ that make me proud and reinforce my decision to have participated in California’s first stem cell clinical trial funded by prop 71.

Like I said earlier it was just the beginning for me. A couple of years later I became a patient advocate working with Americans for Cures. I have been able to meet many people in the stem cell industry and love to see the glow in their face when they learn I was California’s first embryonic stem cell patient.

I can’t even fathom all the year’s of hard work and countless hours of research that had lead up to my long anticipated surgery, but when I see their glowing smile I know they knew what it took.

I also enjoy sharing my story and bridging the gap between myths and facts about stem cells, or talking to students and helping inspire the next generation that will be in the stem cell industry.  As a matter of fact, I have 13 year old sister, Maddie, dead set on being a neurosurgeon.

Fast forward to today. Life in a wheelchair is not exactly a roll in the park (no pun intended) but I have grown accustomed to the new normal. Aside from some neuropathic pain, life is back on track.

Not once did I feel sorry for myself, I was excited to be alive. Sure I have bad days but don’t we all.

In the last 14 years CIRM has funded 50 human clinical trials, published around 2750 new peer-reviewed scientific discoveries, and they’ve transformed California into the world leader in stem cell research. As I look around the posters on the wall, of the people whose lives have been transformed by the agency, I can’t help but be struck by just how much has been achieved in such a short period of time.

While my journey might not yet be over, Evie and 40 other children like her, (children born with SCID) will never remember what it was like to live with the horrible condition they were born with because they have been cured thanks to CIRM. There are hundreds of others whose lives have been transformed because of work the agency has funded.

CIRM has proven how much can be achieved if we invest in cutting-edge medical research.

As most of you here probably know CIRM’s funding from Proposition 71 is about to run out. If I had just one message I wanted people to leave with today it would be this. Everyone in this room knows how much CIRM has done in a little over a decade and how many lives have been changed because of its existence. We have the responsibility to make sure this work continues. We have a responsibility to make sure that the stories we’ve heard today are just the beginning.

I will do everything I can to make sure the agency gets refunded and I hope that all of you will join me in that fight. I’m excited for the world of stem cells, particularly in California, and can’t wait to see what’s on the horizon.

 

Stem Cell Agency Board Approves 50th Clinical Trial

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Rich Lajara, the first patient treated in a CIRM-funded clinical trial

May 4th, 2011 marked a landmark moment for the California Institute for Regenerative Medicine (CIRM). On that day the Stem Cell Agency’s Board voted to invest in its first ever clinical trial, which was also the first clinical trial to use cells derived from embryonic stem cells. Today the Stem Cell Agency reached another landmark, with the Board voting to approve its 50th clinical trial.

“We have come a long way in the past seven and a half years, helping advance the field from its early days to a much more mature space today, one capable of producing new treatments and even cures,” says Jonathan Thomas, JD, PhD, Chair of the CIRM Board. “But we feel that in many ways we are just getting started, and we intend funding as many additional clinical trials as we can for as long as we can.”

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The project approved today awards almost $6.2 million to Angiocrine Bioscience Inc. to see if genetically engineered cells, derived from cord blood, can help alleviate or accelerate recovery from the toxic side effects of chemotherapy for people undergoing treatment for lymphoma and other aggressive cancers of the blood or lymph system.

“This is a project that CIRM has supported from an earlier stage of research, highlighting our commitment to moving the most promising research out of the lab and into people,” says Maria T. Millan, MD, President & CEO of CIRM. “Lymphoma is the most common blood cancer and the 6th most commonly diagnosed cancer in California. Despite advances in therapy many patients still suffer severe complications from the chemotherapy, so any treatment that can reduce those complications can not only improve quality of life but also, we hope, improve long term health outcomes for patients.”

The first clinical trial CIRM funded was with Geron, targeting spinal cord injury. While Geron halted the trial for business reasons (and returned the money, with interest) the mantle was later picked up by Asterias Biotherapeutics, which has now treated 25 patients with no serious side effects and some encouraging results.

Rich Lajara was part of the Geron trial, the first patient ever treated in a CIRM-funded clinical trial. He came to the CIRM Board meeting to tell his story saying when he was injured “I knew immediately I was paralyzed. I thought this was the end, little did I know this was just the beginning. I call it being in the wrong place at the right time.”

When he learned about the Geron clinical trial he asked how many people had been treated with stem cells. “Close to none” he was told. Nonetheless he went ahead with it. He says he has never regretted that decision, knowing it helped inform the research that has since helped others.

Since that first trial the Stem Cell Agency has funded a wide range of projects targeting heart disease and stroke, cancer, diabetes, HIV/AIDS and several rare diseases. You can see the full list on the Clinical Trials Dashboard page on our website.

Rich ended by saying: “CIRM has proven how much can be achieved if we invest in cutting-edge medical research. As most of you here probably know, CIRM’s funding from Proposition 71 is about to run out. If I had just one message I wanted people to leave with today it would be this, I will do everything I can to make sure the agency gets refunded and I hope that all of you will join me in that fight. I’m excited for the world of stem cells, particularly in California and can’t wait to see what’s on the horizon.”

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The CIRM Board also took time today to honor Dr. Bert Lubin, who is stepping down after serving almost eight years on the Board.

When he joined the Board in February, 2011 Dr. Lubin said: “I hope to use my position on this committee to advocate for stem cell research that translates into benefits for children and adults, not only in California but throughout the world.”

Over the years he certainly lived up to that goal. As a CIRM Board member he has supported research for a broad range of unmet medical needs, and specifically for curative treatments for children born with a rare life-threatening conditions such as Sickle Cell Disease and Severe Combined Immunodeficiency (SCID) as well as  treatments to help people battling vision destroying diseases.

As the President & CEO of Children’s Hospital Oakland (now UCSF Benioff Children’s Hospital Oakland) Dr. Lubin was a leader in helping advance research into new treatments for sickle cell disease and addressing health disparities in diseases such as asthma, diabetes and obesity.

Senator Art Torres said he has known Dr. Lubin since the 1970’s and in all that time has been impressed by his devotion to patients, and his humility, and that all Californians should be grateful to him for his service, and his leadership.

Dr. Lubin said he was “Really grateful to be on the Board and I consider it an honor to be part of a group that benefits patients.”

He said he may be stepping down from the CIRM Board but that was all: “I am going to retire the word retirement. I think it’s a mistake to stop doing work that you find stimulating. I’m going to repurpose the rest of my life, and work to make sure the treatments we’ve helped develop are available to everyone. I am so proud to be part of this. I am stepping down, but I am devoted to doing all I can to ensure that you get the resources you need to sustain this work for the future.”

Peddling hope for thousands of dollars – a TV expose on one clinic offering unproven stem cell therapies

David Goldstein

You may have seen an ad in your local paper, promoting a seminar on the “wonders” of stem cell therapies. They are becoming increasingly common all around the US.

The ads talk about the ability of stem cells to heal everything from arthritis to autism. But what they don’t talk about is that they are not approved by the FDA for use in patients, and that they are not proven to do anything except remove large amounts of money from your wallet.

One TV reporter decided to see exactly what was on offer at these clinics. So CBSLA Investigative Reporter David Goldstein went to a free stem cell seminar in the City of Orange, put on by the Stem Cell Institute of Orange County, and found that there was a huge gap between what was being promised and what was being delivered.

You can watch that TV report here.

 

Promising Approach to Curing Spina Bifida Gets $5.6 Million from Stem Cell Agency

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Every day in the U.S. four children are born with spina bifida. It is the most common cause of lifelong paralysis and also frequently leads to other serious health problems affecting the bowel and bladder. The impact on families is enormous. A new approach to repairing the defect that causes spina bifida was today awarded $5.66 million by the Board of the California Institute for Regenerative Medicine (CIRM).

In spina bifida the spinal cord doesn’t form properly, in many cases leaving a section of it open, exposing tissues and nerves. The current standard of care is surgery, but even this leaves almost 60% of children unable to walk independently. Diana Farmer MD, and Aijun Wang PhD at U.C. Davis will use mesenchymal stem cells, taken from a donor placenta, and place them on a form of synthetic scaffold over the injury site in the womb. Tests in animals show this approach was able to repair the defect and prevent paralysis.

“Spina bifida is a devastating condition for babies born with this disorder and the families who care for them,” says Maria T. Millan, MD, President & CEO of CIRM. “CIRM has funded this important work from its earliest stages and we are committed to working with Dr. Farmer’s team to moving this work to the stage where it can be tested in patients.”

The CLIN1 award will provide funding to enable the UC Davis team to do the final testing and preparations needed to apply to the FDA for permission to start a clinical trial.

Dr. Farmer says she and Dr. Wang, have been working on this approach for more than ten years and are excited about being able to take the next step.

“There were many times of frustration, many times when cell types we explored and worked with didn’t work,” says Dr. Farmer. “But it’s the patients, seeing them, talking to them and working with them, that keeps me motivated to do the science, to keep persevering.”

If this therapy is successful it will have a huge economic impact on California, and on the rest of the world. Because spina bifida is a lifelong condition involving many operations, many stays in the hospital and, in some cases, lifelong use of a wheelchair this has a huge financial, and psychological, burden on the family.

“It affects them in so many ways; parents having to miss work or take time off work to care for their child, other children in the family feeling neglected because their brother or sister needs so much attention,” says Dr. Farmer. “That’s why we are so grateful to CIRM. Because this is a rare disease and finding funding for those is hard. CIRM has been a perfect partner in helping bring this approach, blending stem cell therapy and tissue engineering, together to help these families.”

This video shows English bulldogs treated with this approach who are now able to walk:

Stories that caught our eye: Is a Texas law opening up access to stem cell treatments working? Another CIRM-funded company gets good news from the FDA.

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Texas Capitol. (Shutterstock)

In 2017 Texas passed a sweeping new law, HB 810, which allowed medical clinics to provide “investigational stem cell treatments to patients with certain severe chronic diseases or terminal illnesses.” Those in favor of the law argued that patients battling life-threatening or life-changing diseases should have the right to try stem cell therapies that were involved in a clinical trial.

Now a new study, published in the journal Stem Cells and Development, looks at the impact of the law. The report says that despite some recent amendments t there are still some concerns about the law including:

  • It allows treatment only if the patient has a “severe, chronic” illness but doesn’t define what that means
  • It doesn’t have clearly defined procedures on tracking and reporting procedures so it’s hard to know how many patients might be treated and what the outcomes are
  • There is no Food and Drug Administration (FDA) oversight of the patients being treated
  • Because the treatments are unproven there are fears this will “open up the state to unsavory and predatory practices by individuals preying on vulnerable patients”

The researchers conclude:

“While HB 810 opens up access to patients, it also increases significant risks for their safety and financial cost for something that might have no positive impact on their disease. Truly understanding the impact of stem cell based interventions (SCBI) requires scientific rigor, and accurate outcome data reporting must be pursued to ensure the safety and efficacy behind such procedures. This information must be readily available so that patients can make informed decisions before electing to pursue such treatments. The creation of the SCBI registry could allow for some level of scientific rigor, provide a centralized data source, and offer the potential for better informed patient choices, and might be the best option for the state to help protect patients.”

Another CIRM-funded company gets RMAT designation

Poseida

When Congress approved the 21st Century Cures Act a few years ago one of the new programs it created was the Regenerative Medicine Advanced Therapy (RMAT) designation. This was given to therapies that are designed to treat a serious or life-threatening condition, where early clinical stage trials show the approach is safe and appears to be effective.

Getting an RMAT designation is a big deal. It means the company or researchers are able to apply for an expedited review by the FDA and could get approval for wider use.

This week Poseida Therapeutics was granted RMAT designation by the Food and drug Administration (FDA) for P-BCMA-101, its CAR-T therapy for relapsed/refractory multiple myeloma. This is currently in a Phase 1 clinical trial that CIRM is funding

In this trial Poseida’s technology takes an immunotherapy approach that uses the patient’s own engineered immune system T cells to seek and destroy cancerous myeloma cells.

In a news release Eric Ostertag, Poseida’s CEO, welcomed the news:

“Initial Phase 1 data presented at the CAR-TCR Summit earlier this year included encouraging response rates and safety data, including meaningful responses in a heavily pretreated population. We expect to have an additional data update by the end of the year and look forward to working closely with the FDA to expedite development of P-BCMA-101.”

This means that five CIRM-funded companies have now been granted RMAT designations:

New partnership to make CIRM supported treatment for type 1 diabetes even better

 

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ViaCyte’s PEC-Direct device. Image courtesy of ViaCyte

ViaCyte, a regenerative medicine company long backed by CIRM, announced a partnership with CRISPR Therapeutics to increase the number of people with Type 1 Diabetes (T1D) who could benefit from their PEC-Direct therapeutic implant.

Last year, CIRM granted ViaCyte $20 million to facilitate development of PEC-Direct, a device that both transplants pancreatic progenitor stem cells (the immature version of  islet cells, the insulin-producing cells that are destroyed in TID), and allows those cells to connect to the patient’s bloodstream to help them function more like normal islet cells. This treatment, currently in clinical trials, was initially targeted towards high risk patients because of the need to treat them with immunosuppressive therapy, to ensure that the patient’s immune system does not attack the implanted cells.

ViaCyte’s partnership with CRISPR Therapeutics aims to eliminate the need for immunosuppressive therapy by engineering the transplanted stem cells to evade the immune system prior to implanting in the patient. CRISPR Therapeutics is already using this gene editing approach in CAR-T based cancer therapies and has developed an important knowledge base in “immune-evasive gene editing.” Paul Laikind Ph.D., CEO and President of ViaCyte explains the importance of this partnership in a news release:

“Creating an immune-evasive gene-edited version of our technology would enable us to address a larger patient population than we could with a product requiring immunosuppression. CRISPR Therapeutics is the ideal partner for this program given their leading gene editing technology and expertise and focus on immune-evasive editing.”

Samarth Kulkarni, Ph.D., and CEO of CRISPR Therapeutics adds:

“We believe the combination of regenerative medicine and gene editing has the potential to offer durable, curative therapies to patients in many different diseases, including common chronic disorders like insulin-requiring diabetes.”

The hope is that this new approach could make this treatment available to everyone with T1D. The benefits of such a treatment option would be considerable as TID affects around 1.25 million Americans, and can lead to severe health complications such as kidney damage and heart disease. The initial goals of this collaboration are to develop a stem cell line that successfully evades the immune system, followed by developing a product that can be used in patients.