Taking even the most promising therapy and moving it out of the lab and into people is an incredibly complex process and usually requires a great team. Now, two great teams have paired up to do just that with a therapy for type 1 diabetes (T1D). ViaCyte and CRISPR Therapeutics have put their heads together and developed an approach that has just been given clearance by Health Canada to start a clinical trial.
Regular readers of this blog know that CIRM has been a big supporter of ViaCyte for many years, investing more than $72 million in nine different awards. They have developed an implantable device containing embryonic stem cells that develop into pancreatic progenitor cells, which are precursors to the islet cells destroyed by T1D. The hope is that when this device is transplanted under a patient’s skin, the progenitor cells will develop into mature insulin-secreting cells that can properly regulate the glucose levels in a patient’s blood.
One of the challenges in earlier testing was developing a cell-based therapy that could evade the immune system, so that people didn’t need to have their immune system suppressed to prevent it attacking and destroying the cells. This particular implantable version sprang out of an early stage award we made to ViaCyte (DISC2-10591). ViaCyte and CRISPR Therapeutics helped with the design of the therapeutic called VCTX210.
In a news release, Michael Yang, the President and CEO of ViaCyte, said getting approval for the trial was a major milestone: “Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies. This approach builds on previous accomplishments by both companies and represents a major step forward for the field as we strive to provide a functional cure for this devastating disease.”
The clinical trial, which will be carried out in Canada, is to test the safety of the therapy, whether it creates any kind of reaction after being implanted in the body, and how well it does in evading the patient’s immune system. In October our podcast – Talking ‘Bout (re)Generation – highlighted work in T1D and included an interview with Dr. Manasi Jaiman, ViaCyte’s Vice President for Clinical Development. Here’s an excerpt from that podcast.
When the COVID-19 pandemic hit and the 2020 election became one of the most contentious in living history it suddenly made trying to get a proposition on the ballot in California a lot harder. That meant the fate of Proposition 14, a ballot initiative refunding CIRM, California’s Stem Cell Agency, was in doubt. And if the agency went down, then a vital source of future funding for scientific research that could change and even save lives would also disappear.
It was a pretty nerve-racking time for all of us involved. We waited day after day after day after day before the election was finally called. Happily, it was in our favor. But only just!
In this podcast we talk to two of the key figures in this saga. Melissa King and Maria Bonneville. Melissa was part of the team that helped secure the votes needed to pass Proposition 14, and Maria helped keep CIRM on track to cope with whatever the outcome of the election was.
As someone with a family history of type 1 diabetes (T1D) I know how devastating the condition can be. I also know how challenging it can be to keep it under control and the consequences of failing to do that. Not maintaining healthy blood sugar levels can have a serious impact on the heart, kidney, eyes, nerves, and blood vessels. It can even be fatal.
Right now, controlling T1D means being careful about what you eat, when you eat and how much you eat. It also means regularly checking your blood throughout the day to see if the glucose level is too high or too low. If it’s too high you need to inject insulin; if it’s too low you need to take a fast-acting carbohydrate such as fruit juice or glucose to try and restore it to a healthy level.
That’s why two new approaches to T1D that CIRM has supported are so exciting. They both use small devices implanted under the skin that contain stem cells. The cells can both monitor blood sugar and, if it’s too high, secrete insulin to bring it down.
We sat down with two key members of the Encellin and ViaCyte teams, Dr. Crystal Nyitray and Dr. Manasi Jaiman, to talk about their research, how it works, and what it could mean for people with T1D. That’s in the latest episode of our podcast ‘Talking ‘Bout (re)Generation’.
Adrienne Shapiro and Marissa Cors are a remarkable pair by any definition. The mother and daughter duo share a common bond, and a common goal. And they are determined not to let anyone stop them achieving that goal.
Marissa was born with sickle cell disease (SCD) a life-threatening genetic condition where normally round, smooth red blood cells are instead shaped like sickles. These sickle cells are brittle and can clog up veins and arteries, blocking blood flow, damaging organs, and increasing the risk of strokes. It’s a condition that affects approximately 100,000 Americans, most of them Black.
Adrienne became a patient advocate, founding Axis Advocacy, after watching Marissa get poor treatment in hospital Emergency Rooms. Marissa often talks about the way she is treated like a drug-seeker simply because she knows what medications she needs to help control excruciating pain on her Sickle Cell Experience Live events on Facebook.
Now the two are determined to ensure that no one else has to endure that kind of treatment. They are both fierce patient advocates, vocal both online and in public. And we recently got a chance to sit down with them for our podcast, Talking ‘Bout (re) Generation. These ladies don’t pull any punches.
Anyone who knows anything about CIRM knows about Bob Klein. He’s the main author and driving force behind both Proposition 71 and Proposition 14, the voter-approved ballot initiatives that first created and then refunded CIRM. It’s safe to say that without Bob there’d be no CIRM.
Recently we had the great good fortune to sit down with Bob to chat about the challenges of getting a proposition on the ballot in a time of pandemic and electoral pandemonium, what he thinks CIRM’s biggest achievements are (so far) and what his future plans are.
According to the Podcasthosting.org website there are some two million podcasts in the world. Make that two million and one. That’s because CIRM is launching its own podcast and doing it with one of the biggest names in biotech.
Our podcast is called – with a nod to The Who – “Talking ’bout (Re)Generation” and the first episode features our President & CEO Dr. Maria Millan interviewing Dr. Derrick Rossi, the co-founder of Moderna. Moderna, as I am sure you know, is the maker of one of the most effective vaccines against COVID.
In the interview Dr. Rossi talks about his early days as a postdoc at Stanford – supported by CIRM – and the career arc that led him to help create the company behind the vaccine, and what his plans are for the future. It’s a fun, chatty, lively interview; one you can listen to in the car, at home or wherever you listen to your podcasts.
We want the podcast to be fun for your ear holes and interesting and engaging for your brain. We’re going to be talking to scientists and researchers, doctors and nurses, patients and patient advocates and anyone else we think has something worth listening to.
We have other episodes planned and will share those with you in the near future. In the meantime, if you have any ideas or individuals you think would make a good subject for a podcast let us know, we are always happy to hear from you.