Cell Therapies

Thanks to recent advances, people with type 1 diabetes (T1D) could be freed from their blood-glucose monitors and insulin injections for years, or even decades. The JDRF Beta Cell Therapies Program invests in research and clinical trials to develop and deliver life-changing therapies that place healthy, insulin-producing beta cells back into the bodies of people with T1D.

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Our Approach

In people with T1D, the autoimmune system destroys the pancreas’s beta cells, which produce insulin. One known way to cure T1D is to replace destroyed beta cells with external cells that make insulin and protect them so that they can function for a very long time. Islet transplantation using donor islets from cadavers has provided evidence for a long-term cure through insulin independence and other benefits. Currently, only a small portion of people living with T1D can get this treatment because of the challenges of producing beta cells, and because a beta cell recipient must also take medications to protect the implanted beta cells for the rest of their life.

Program Goals

Develop a renewable beta cell source so that every person with T1D can get this treatment.

Find ways to maintain the beta cells so they stay functional in the body for years—or even decades.

Shield the beta cells from immune attack to prevent the need for anti-rejection drugs or the recurrence of T1D.

Progress

2023
March 9

Vertex Announces Clinical Trial Clearance from FDA

Vertex announced clinical trial clearances from both the FDA and Health Canada for VX-264, their investigational encapsulated cell therapy for the potential treatment of type 1 diabetes. This treatment would not require immunosuppressant drugs.
2022
July 11

Vertex Acquires ViaCyte

Vertex Pharmaceuticals announces that they have acquired ViaCyte to help speed up the development of VX-880, Vertex’s stem cell-derived therapy for people with T1D. This merger brings together two of the biggest companies pursuing cell replacement therapy, and will allow them to combine their resources, technologies, intellectual property, and more.

May 3

Vertex Provides an Update on the VX-880 Clinical Trial

The latest update from Vertex is the biggest yet: Patient #1 is 100% insulin independent 270 days after receiving the therapy with an HbA1c of 5.2%. He is on immunosuppressive therapy, but no longer needs to administer insulin.

February 2

ViaCyte and CRISPR Therapeutics Launch Clinical Trial Testing Gene-Edited Cells

ViaCyte and CRISPR Therapeutics announce the start of a clinical trial of VCTX210, a gene-edited stem cell replacement therapy for T1D. This immune-evasive therapy is designed to enable patients to produce their own insulin.

2021
May 27

JDRF Launches Centers of Excellence to Find Cures

JDRF announces the launch of Centers of Excellence to solve the riddle of the body rejecting stem cell-derived beta cells through immune modulation and gene editing.

May 1

Vertex Launches Clinical Trial

Vertex began a phase I/II clinical trial of VX-880, which uses stem cell-derived beta cells to try to restore the body’s ability to produce insulin, in combination with immunosuppressive therapy to protect the cells from rejection.

2019
May 27

Sernova Demonstrates Insulin Production

Sernova, a clinical-stage regenerative medicine company, showed in its JDRF-funded clinical trial that its cell replacement therapy, the Cell Pouch System™, can produce insulin in people with T1D.

May 1

ViaCyte Shows Insulin Production in People with T1D

ViaCyte, a beta cell replacement company long supported by JDRF, showed—for the first time ever—that its PEC-Directtherapy helps people with T1D produce insulin again.

Looking Ahead

In the past decade, JDRF has invested more than $140 million in cell therapies. Future research will continue to investigate how to keep the beta cells alive and healthy using gene editing, encapsulation, and immune modulation.

Want to learn more? View JDRF scientists Jaime Giraldo, Ph.D., and Esther Latres, Ph.D., below for a presentation on the cell therapies portfolio. You can also view the extended strategy here.