CIRM-Funded Clinical Trials Targeting the Heart, Pancreas, and Kidneys

This blog is part of our Month of CIRM series, which features our Agency’s progress towards achieving our mission to accelerate stem cell treatments to patients with unmet medical needs.

This week, we’re highlighting CIRM-funded clinical trials to address the growing interest in our rapidly expanding clinical portfolio. Today we are featuring trials in our organ systems portfolio, specifically focusing on diseases of the heart/vasculature system, the pancreas and the kidneys.

CIRM has funded a total of nine trials targeting these disease areas, and eight of these trials are currently active. Check out the infographic below for a list of our currently active trials.

For more details about all CIRM-funded clinical trials, visit our clinical trials page and read our clinical trials brochure which provides brief overviews of each trial.

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CIRM Board Appoints Dr. Maria Millan as President and CEO

Dr. Maria Millan, President and CEO of CIRM, at the September Board meeting. (Todd Dubnicoff, CIRM)

Yesterday was a big day for CIRM. Our governing Board convened for its September ICOC meeting and appointed Dr. Maria Millan as our new President and CEO. Dr. Millan has been serving as the Interim President/CEO since July, replacing former President Dr. Randal Mills.

Dr. Millan has been at CIRM since 2012 and was instrumental in the development of CIRM’s infrastructure programs including the Alpha Stem Cell Clinics Network and the agency’s Strategic Plan, a five-year plan that lays out our agency’s goals through 2020. Previously, Dr. Millan was the Vice President of Therapeutics at CIRM, helping the agency fund 23 new clinical trials since the beginning of 2016.

The Board vote to appoint Dr. Millan as President and CEO was unanimous and enthusiastic. Chairman of the Board, Jonathan Thomas, shared the Board’s sentiments when he said,

“Dr. Millan is absolutely the right person for this position. Having seen Dr. Millan as the Interim CEO of CIRM for three months and how she has operated in that position, I am even more enthusiastic than I was before. I am grateful that we have someone of Maria’s caliber to lead our Agency.”

Dr. Millan has pursued a career devoted to helping patients. Before working at CIRM, she was an organ transplant surgeon and researcher and served as an Associate Professor of Surgery and Director of the Pediatric Organ Transplant Program at Stanford University. Dr. Millan was also the Vice President and Chief Medical Officer at StemCells, Inc.

In her permanent role as President, Dr. Millan is determined to keep CIRM on track to achieve the goals outlined in our strategic plan and to achieve its mission to accelerate treatments to patients with unmet needs. She commented in a CIRM press release,

“I joined the CIRM team because I wanted to make a difference in the lives of patients. They are the reason why CIRM exists and why we fund stem cell research. I am humbled and very honored to be CIRM’s President and look forward to further implementing our agency’s Strategic Plan in the coming years.”

The Board also voted to fund two new Alpha Stem Cell Clinics at UC Davis and UC San Francisco and five new clinical trials. Three of the clinical awards went to projects targeting cancer.

The City of Hope received $12.8 million to fund a Phase 1 trial targeting malignant gliomas (an aggressive brain cancer) using CAR-T cell therapy. Forty Seven Inc. received $5 million for a Phase 1b clinical trial treating acute myeloid leukemia. And Nohla Therapeutics received $6.9 million for a Phase 2 trial testing a hematopoietic stem cell and progenitor cell therapy to help patients suffering from neutropenia, a condition that leaves people susceptible to deadly infections, after receiving chemotherapy for acute myeloid leukemia.

The other two trials target diabetes and end stage kidney failure. ViaCyte, Inc. was awarded $20 million to fund a Phase 1/2 clinical trial to test its PEC-Direct islet cell replacement therapy for high-risk type 1 diabetes. Humacyte Inc. received $14.1 million to fund a Phase 3 trial that is comparing the performance of its acellular bioengineered vessel with the current standard of dialysis treatment for kidney disease patients.

The Board also awarded $5.2 million to Stanford Medicine for a late stage preclinical project that will use CRISPR gene editing technology to correct the sickle cell disease mutation in blood-forming stem cells to treat patients with sickle cell disease. This award was particularly well timed as September is Sickle Cell Awareness month.

The Stanford team, led by Dr. Matthew Porteus, hopes to complete the final experiments required for them to file an Investigational New Drug (IND) application with the FDA so they can be approved to start a clinical trial hopefully sometime in 2018. You can read more about Dr. Porteus’ work here and you can read our past blogs featuring Sickle Cell Awareness here and here.

With the Board’s vote yesterday, CIRM’s clinical trial count rises to 40 funded trials since its inception. 23 of these trials were funded after the launch of our Strategic Plan bringing us close to the half way point of funding 50 new clinical trials by 2020. With more “shots-on-goal” CIRM hopes to increase the chances that one of these trials will lead to an FDA-approved therapy for patients.


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CIRM-funded life-saving stem cell therapy gets nod of approval from FDA

Cured_AR_2016_coverIf you have read our 2016 Annual Report (and if you haven’t you should, it’s brilliant) or just seen the cover you’ll know that it features very prominently a young girl named Evie Padilla Vaccaro.

Evie was born with Severe Combined Immunodeficiency or SCID – also known as “bubble baby disease”; we’ve written about it here. SCID is a rare but deadly immune disorder which leaves children unable to fight off simple infections. Many children with SCID die in the first few years of life.

Fortunately for Evie and her family, Dr. Don Kohn and his team at UCLA, working with a UK-based company called Orchard Therapeutics Ltd., have developed a treatment called OTL-101. This involves taking the patient’s own blood stem cells, genetically modifying them to correct the SCID mutation, and then returning the cells to the patient. Those modified cells create a new blood supply, and repair the child’s immune system.

Evie was treated with OTL-101 when she was a few months old. She is cured. And she isn’t the only one. To date more than 40 children have been treated with this method. All have survived and are doing well.

Orchard Therapeutics

 FDA acknowledgement

Because of that success the US Food and Drug Administration (FDA) has granted OTL-101 Rare Pediatric Disease Designation. This status is given to a treatment that targets a serious or life-threatening disease that affects less than 200,000 people, most of whom are under 18 years of age.

The importance of the Rare Pediatric Disease Designation is that it gives the company certain incentives for the therapy’s development, including priority review by the FDA. That means if it continues to show it is safe and effective it may have a faster route to being made more widely available to children in need.

In a news release Anne Dupraz, PhD, Orchard’s Chief Regulatory Officer, welcomed the decision:

“Together with Orphan Drug and Breakthrough Therapy Designations, this additional designation is another important development step for the OTL-101 clinical program. It reflects the potential of this gene therapy treatment to address the significant unmet medical need of children with ADA-SCID and eligibility for a Pediatric Disease Priority Review voucher at time of approval.”

Creating a trend

This is the second time in less than two weeks that a CIRM-funded therapy has been awarded Rare Pediatric Disease designation. Earlier this month Capricor Therapeutics was given that status for its treatment for Duchenne Muscular Dystrophy.

Two other CIRM-funded clinical trials – Humacyte and jCyte – have been given Regenerative Medicine Advanced Therapy Designation (RMAT) by the FDA. This makes them eligible for earlier and faster interactions with the FDA, and also means they may be able to apply for priority review and faster approval.

All these are encouraging signs for a couple of reasons. It suggests that the therapies are showing real promise in clinical trials. And it shows that the FDA is taking steps to encourage those therapies to advance as quickly – and safely of course – as possible.

Credit where credit is due

In the past we have been actively critical of the FDA’s sluggish pace in moving stem cell therapies out of the lab and into clinical trials where they can be tested in people. So when the FDA does show signs of changing the way it works it’s appropriate that that we are actively supportive.

Getting these designations is, of course, no guarantee the therapies will ultimately prove to be successful. But if they are, creating faster pathways means they can get to patients, the people who really need them, at a much faster pace.

 

 

 

 

 

Humacyte Receives Prestigious Technology Pioneer Award for Kidney Failure Treatment

This month, a CIRM-funded company called Humacyte was named one of the World Economic Forum’s 30 Technology Pioneers for 2017. This prestigious award “recognizes early-stage companies from around the world that are involved in the design, development and deployment of new technologies and innovations, and are poised to have a significant impact on business and society.”

Humacyte is a North Carolina-based company that’s developing a promising human-tissue based treatment for kidney failure. They’ve developed a technology to manufacture a bioengineered human vein that they hope will improve kidney function in patients with end stage kidney disease and patients on hemodialysis. We’ve blogged about their exciting technology previously on the Stem Cellar (here).

The technology is fascinating. The first step involves stimulating human smooth muscle cells from donor tissue to develop into tubular vessels. After the vessels are made, the cells are removed, leaving a 3D extracellular matrix structure composed of molecules secreted by the cells. This decellularized tube-like structure is called a human acellular vessels or HAV.

Human acellular vessel (HAV) from Humacyte.

The HAV is then implanted under a patient’s skin, where it recruits the patient’s own stem cells to migrate into the HAV and develop into vascular smooth muscle cells that line the insides of actual blood vessels. For patients with kidney failure, HAVs provide vascular access for hemodialysis, the process of collecting and filtering a patient’s blood through an artificial kidney and then returning “clean” blood back to the body. It would provide an alternative to the current procedures that insert a plastic tube called a shunt into the patient’s vein. Shunts can cause infection, blood clots, and can also be rejected by a patient’s immune system.

In July of 2016, CIRM awarded Humacyte almost $10 million to launch a Phase 3 trial in California to test their bioengineered blood vessels in patients with kidney failure. Since launching the trial, Humacyte received Regenerative Medicine Advanced Therapy or RMAT designation from the US Food and Drug Administration in March of this year. This designation is a sign that the FDA sees promise in Humacyte’s stem cell-based therapy and “will help facilitate the efficient development and expedited review of the HAV for vascular access to patients in need of life-sustaining hemodialysis.”

Humacyte’s technology has wide-ranging applications beyond treating kidney disease, including peripheral arterial disease, “repairing or replacing damaged arteries, coronary artery bypass surgery, and vascular trauma.” Other key benefits of this technology are that HAVs can be designed on demand and can be stored for later use without fear of a rapidly degrading shelf-life.

In a recent Humacyte news release, Carrie Cox, Chair and CEO of Humacyte, commented on her company’s purpose and vision to help patients.

“Keeping patient care at its core, Humacyte’s scientific discoveries are designed to create ‘off-the-shelf,’ or ready to use, bioengineered blood vessels. Today these conduits are being investigated clinically for patients undergoing kidney dialysis who require vascular access and for patients with peripheral arterial disease. However, this technology may be extended into a range of vascular applications in the future, with the potential for better clinical outcomes and lower healthcare costs. Our vision is to make a meaningful impact in healthcare by advancing innovation in regenerative medicine to produce life-sustaining improvements for patients with vascular disease.”

The potential impact that Humacyte’s technology could have for patients with unmet medical needs was compelling enough to earn the company a coveted spot in the World Economic Forum’s Technology Pioneer community. This recognition will likely foster new partnerships and collaborations to further advance Humacyte’s technology down the clinical pipeline. Fulvia Montresor, Head of Technology Pioneers at the World Economic Forum, concluded in a news release.

“We welcome Humacyte in this group of extraordinary pioneers. We hope that thanks to this selection, the World Economic Forum can facilitate greater collaboration with business leaders, governments, civil society and other relevant individuals to accelerate the development of technological solutions to the world’s greatest challenges.”

According to coverage by North Carolina Biotechnology Center, Humacyte and the other Technology Pioneers will be honored at the “Summer Davos” World Economic Forum Annual Meeting of the New Champions later this month in China. You can learn more about this meeting here.


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Stem Cells Profiles in Courage: Frank’s final gift

frank-st-clair

Not every story has a happy ending. But they do all have something to teach us. In the case of Frank St. Clair the lesson was simple: live life fully and freely, love those around you, and never give up.

We were fortunate enough to get to know Frank as one of the people we profiled in our 2016 Annual Report. Frank was a patient in a clinical trial we are funding to test a new kind of bioengineered vein needed by people undergoing hemodialysis, the most common form of dialysis.

It was an all too brief friendship. Frank passed away on December 17th due to complications from heart disease. But in that time he touched us with his warmth, his kindness, his sense of humor and his generosity. Frank never gave up. He kept fighting to the end. His courage, and compassion for others is a reminder to us that we need to work as hard as we can, to bring treatments to those who need them most.

This is Frank’s story, in his own words:

“I have kidney disease. Had it about four years. When I first started dialysis I had a shunt in my chest.  I had to be careful with the shunt, especially at night, in case I pulled it out. It kept clogging up on me and I’d have to go in and get it reopened and that was a terrible thing.

One time when they were opening up the shunt in my chest I ran into the doctor and I got talking to him. He knew how miserable I was and he asked if I wanted to take part in this clinical trial. I said I did and they arranged for me to get this, the device. I just lucked out and was in the right place at the right time. Best move I ever made. Didn’t know anything about stem cells then, sure didn’t, I just knew I was miserable and if there was any way to make life better I just wanted to do it or try it.

And then I did this and it was like day and night.

Since I’ve done this my life has improved 100%. I can do a lot now that I couldn’t do before. My wife and I are so grateful that we can have this. Now we can go out to dinner and do anything we want. We could go out before but we had to always be careful because of the thing in my chest. But now I don’t even think about it. It’s like getting my life back.

I don’t notice it all. I don’t feel it at all. I hate to say it, but I can’t believe I’m on dialysis. I would like to have a kidney but I’ll be honest with you this is the next best thing.

When I go to the clinic there’s a lot of old people there and I just try to make them laugh, tell them jokes, I just can’t believe how good I feel and I want to make others feel good too.

I take the time to talk to them, and give them gum and that cheers them up. My wife has to keep me supplied with gum.

I’ve been married 45 years. We met in high school chorus. I didn’t care too much about singing but I went to chorus because I wanted to meet girls. That’s where I met Paula. Best move I ever made.

I sure don’t feel old. My wife and I are two people that love each other very dearly, that’s my blessing, with her help I couldn’t get old.

I’m a workaholic but until I got the Humacyte device I couldn’t work. I had to sell my business.

I used to be a private detective. It had its moments. My wife used to get mad because I got up at 2 or 3 in the morning to get someone who was in hiding. I had one guy, he was about 6’ 7”, big guy. I knocked at the door and said the name of the guy I was looking for, and asked if he was there. He asked why, so I told him why I was there and he said “It’s me,” and ran right over me and knocked me on the ground and ran away. But I managed to talk him into coming back.

We served a lot of papers on foreclosures and I hated that, and I would always try and help those people if I could.

One time I ran into an old lady, she was a nice woman, and her husband handled all the bills but he died and they had stock in Bernie Madoff’s company and when he went under it left her broke.  They had $1.7 million in a company that went bankrupt. She lost it all. She didn’t know what to do. When I went to serve her papers she hadn’t eaten in two days,  so I went and bought her and brought some groceries and made sure the electric bill got paid and then called her son and made sure she was taken care of.

My wife said we were going broke helping so many people, but I felt that if you help people it comes back to you and it has.

I volunteer at the VA, help out there when I can. Just trying to give back. Always have. I think if you can help someone you need to do it.

I feel damn lucky, really lucky, more ways than one. You have to understand I have lived 50 years longer than I should have; I could have died in Vietnam, so I would just say do not give up. Don’t give up. My wife wouldn’t let me give up, and things happen. If they are meant to be, of course. Something will happen and I’m telling you. The key is making people around you feel like they want to be around you.”

We are forever grateful to Frank for being willing to be part of a clinical trial that will, hopefully, improve the quality of life for many others. That is his legacy. Our thoughts and wishes go out to his wife Paula

Cured by Stem Cells

cirm-2016-annual-report-web-12

To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual Report for 2016, now online, is our way of checking that we are still on the right path.

I think, without wishing to boast, that it’s safe to say not only are we on target, but we might even be a little bit ahead of schedule.

The Annual Report is chock full of facts and figures but at the heart of it are the stories of the people who are the focus of all that we do, the patients. We profile six patients and one patient advocate, each of whom has an extraordinary story to tell, and each of whom exemplifies the importance of the work we support.

brenden_stories_of_hope

Brenden Whittaker: Cured

Two stand out for one simple reason, they were both cured of life-threatening conditions. Now, cured is not a word we use lightly. The stem cell field has been rife with hyperbole over the years so we are always very cautious in the way we talk about the impact of treatments. But in these two cases there is no need to hold back: Evangelina Padilla Vaccaro and Brenden Whittaker have been cured.

evangelina

Evangelina: Cured

 

In the coming weeks we’ll feature our conversations with all those profiled in the Annual Report, giving you a better idea of the impact the stem cell treatments have had on their lives and the lives of their family. But today we just wanted to give a broad overview of the Annual Report.

The Strategic Plan was very specific in the goals it laid out for us. As an agency we had six big goals, but each Team within the agency, and each individual within those teams had their own goals. They were our own mini-maps if you like, to help us keep track of where we were individually, knowing that every time an individual met a goal they helped the Team get closer to meeting its goals.

As you read through the report you’ll see we did a pretty good job of meeting our targets. In fact, we missed only one and we’re hoping to make up for that early in 2017.

But good as 2016 was, we know that to truly fulfill our mission of accelerating treatments to patients with unmet medical needs we are going to have do equally well, if not even better, in 2017.

That work starts today.

 

Bioengineered veins give hope to kidney disease patients on dialysis

As blood travels around your body, it helps your body get around. Blood is essential for delivering oxygen and nutrients to all the cells in your body and for removing waste products made by these cells. Your body contains approximately 1.5 gallons of blood, which translates to around 7% of your body weight. In order for all this blood to do its job, it needs to be constantly cleaned of waste and extra fluids.

Your kidneys are your blood’s best friend. They act as natural filters that remove those cellular waste products and extra fluid from the blood and pass them off to the bladder, where they are disposed of through urine. Kidneys have the important job of maintaining the proper balance of fluids, electrolytes and chemicals in the blood. They are also involved in other essential biological processes such as regulating blood pressure, making new blood cells, and maintaining healthy bones. It’s a big problem when your kidneys stop working. Without this built-in filtration system, toxic byproducts build up in your blood and cause a multitude of not fun symptoms.

Hemodialysis acts as an artificial kidney to filter the blood of kidney disease patients. (wikipedia)

Hemodialysis acts as an artificial kidney to filter the blood of kidney disease patients. (wikipedia)

More than half a million Americans suffering from kidney dysfunction or failure are being treated by hemodialysis. This process involves connecting a patient to a machine that acts as an artificial kidney. “Old blood” is pumped into the machine from a plastic tube, also known as a shunt, that’s inserted into the patient’s vein. The blood is then passed through a dialyzer which filters out the waste products and extra fluid and allows clean blood to pass through and be put back into the patient (see image).

While hemodialysis is successful at extending the lifespan of kidney disease patients, serious complications can arise from this treatment including uncontrolled changes in blood pressure, bone disease, and anemia. Another common problem occurs with the shunt that’s inserted into a patient’s vein. Shunts can cause infection, blood clots, and can also be rejected by a patient’s immune system. As a result, patients have to get new shunts implanted every year. This is not always feasible for older patients whose veins cannot hold up to this invasive procedure.

A tubular alternative for better hemodialysis

A North Carolina company called Humacyte is trying to improve current hemodialysis technology by engineering human acellular vessels (HAVs) (meaning that the vessels don’t have any cells) that can be transplanted into patients and develop into a human version of a shunt. Sounds complicated, but it’s not really!

First, scientists take muscle cells from human organ donors and coax these cells to grow into tube-like structures. During this process, the cells secrete a compound called cellulose – a component of the extracellular matrix – which forms a biological scaffold that maintains the structure of the cells.

Next, the scientists chemically wash away the muscle cells, leaving an intact scaffold with a hole the diameter of your pinky finger. These scaffolds are then placed under the skin of patients on dialysis. Once transplanted, a patient’s own stem cells migrate to the empty scaffold, set up shop and create a new vein with a wide enough hole that can be used for hemodialysis.

Humacyte’s Chief Medical Officer, Jeff Lawson, explained it an interview with KQED Science:

Jeff Lawson, Humacyte

Jeff Lawson, Humacyte

“This scaffold, once implanted, uniquely becomes repopulated with their own stem cells. That then turns back into something that looks like a vascular cell. And it now transitions over the period of a few months into something that’s indistinguishable from your own tissue. One of the holy grails in vascular surgery is to come up with a prosthetic artificial graft that has the same properties as the patient’s own blood vessels.”

The great news about this promising technology is that Humacyte is testing it in a Phase III clinical trial – the final stage before a drug or treatment is approved by the US Food and Drug Administration (FDA). In a Phase III trial, the treatment has already proven to be safe and shown some effectiveness (in a Phase II trial) and is now being tested in a larger group of patients to hopefully confirm these findings.

In July, CIRM invested $10 million in Humacyte’s Phase III trial in hopes that this technology will improve the lives and health of dialysis patients. Randy Mills, the President and CEO of CIRM, views kidney failure as an unmet medical need that could benefit from a stem cell related treatment:

“This approach has the potential to significantly improve our ability to care for people with kidney disease. Being able to reduce infections and clotting problems, and increase the consistency of care hemodialysis patients get, would meaningfully impact the quality of their lives.”

A patient’s story and CIRM’s efforts to fund clinical trials

Raymund Ramirez

Raymond Ramirez (KQED Science)

Yesterday, David Gorn from KQED Science published a nice piece about Humacyte’s stem cell derived technology and featured the story of a kidney failure patient, Raymond Ramirez. Raymond’s story is very emotional. He is a Vietnam war veteran that has experienced a gauntlet of maladies including bladder cancer and blindness in his right eye. On top of that, his kidneys aren’t functioning well and he is unable to continue his dialysis treatments because his veins aren’t holding up.

Raymond was the first patient to be treated in Humacyte’s Phase III trial. You can read more about his story here.

Gorn also highlighted CIRM’s recent efforts to fund promising stem cell projects that are further along in development and ready for clinical trials in patients. He ended with a quote from UC San Diego’s director of stem cell research, Larry Goldstein, on how important it is for our agency to continue funding stem cell clinical trials.

Larry Goldstein

Larry Goldstein

“Ten years ago I don’t think there were that many [stem cell] projects that were really ready for clinical trials. The field itself has developed projects that are at clinical stage. If the agency [CIRM] keeps pumping out these types of clinical results, California voters may soon see another ballot measure to keep it going.”

CIRM Board targets diabetes and kidney disease with big stem cell research awards

diabetes2

A recent study  estimated there may be more than 500 million people worldwide who have diabetes. That’s an astounding figure and makes diabetes one of the largest chronic disease epidemics in human history.

One of the most serious consequences of untreated or uncontrolled diabetes is kidney damage. That can lead to fatigue, weakness, confusion, kidney failure and even death. So two decisions taken by the CIRM Board today were good news for anyone already suffering from either diabetes or kidney disease. Or both.

The Board awarded almost $10 million to Humacyte to run a Phase 3 clinical trial of an artificial vein needed by people undergoing hemodialysis – that’s the most common form of dialysis for people with kidney damage. Hemodialysis helps clean out impurities and toxins from the blood. Without it waste will build up in the kidneys with devastating consequences.

The artificial vein is a kind of bioengineered blood vessel. It is implanted in the individual’s arm and, during dialysis, is connected to a machine to move the blood out of the body, through a filter, and then back into the body. The current synthetic version of the vein is effective but is prone to clotting and infections, and has to be removed regularly. All this puts the patient at risk.

Humacyte’s version – called a human acellular vessel or HAV – uses human cells from donated aortas that are then seeded onto a biodegradable scaffold and grown in the lab to form the artificial vein. When fully developed the structure is then “washed” to remove all the cellular tissue, leaving just a collagen tube. That is then implanted in the patient, and their own stem cells grow onto it, essentially turning it into their own tissue.

In earlier studies Humacyte’s HAV was shown to be safer and last longer than current versions. As our President and CEO, Randy Mills, said in a news release, that’s clearly good news for patients:

“This approach has the potential to dramatically improve our ability to care for people with kidney disease. Being able to reduce infections and clotting, and increase the quality of care the hemodialysis patients get could have a significant impact on not just the quality of their life but also the length of it.”

There are currently almost half a million Americans with kidney disease who are on dialysis. Having something that makes life easier, and hopefully safer, for them is a big plus.

The Humacyte trial is looking to enroll around 350 patients at three sites in California; Sacramento, Long Beach and Irvine.

While not all people with diabetes are on dialysis, they all need help maintaining healthy blood sugar levels, particularly people with type 1 diabetes. That’s where the $3.9 million awarded to ViaCyte comes in.

We’re already funding a clinical trial with ViaCyte  using an implantable delivery system containing stem cell-derived cells that is designed to measure blood flow, detect when blood sugar is low, then secrete insulin to restore it to a healthy level.

This new program uses a similar device, called a PEC-Direct. Unlike the current clinical trial version, the PEC-Direct allows the patient’s blood vessels to directly connect, or vasularize, with the cells inside it. ViaCyte believes this will allow for a more robust engraftment of the stem cell-derived cells inside it and that those cells will be better able to produce the insulin the body needs.

Because it allows direct vascularization it means that people who get the delivery system  will also need to get chronic immune suppression to stop their body’s immune system attacking it. For that reason it will be used to treat patients with type 1 diabetes that are at high risk for acute complications such as severe hypoglycemic (low blood sugar) events associated with hypoglycemia unawareness syndrome.

In a news release Paul Laikind, Ph.D., President and CEO of ViaCyte, said this approach could help patients most at risk.

“This high-risk patient population is the same population that would be eligible for cadaver islet transplants, a procedure that can be highly effective but suffers from a severe lack of donor material. We believe PEC-Direct could overcome the limitations of islet transplant by providing an unlimited supply of cells, manufactured under cGMP conditions, and a safer, more optimal route of administration.”

The Board also approved more than $13.6 million in awards under our Discovery program. You can see the winners here.