Clinical trial for type 1 diabetes reaches milestone

SIOUX FALLS, S.D. –- Dr. Kurt Griffin knows the toll that type 1 diabetes can take on a child.

He sees it every day in his Sanford Health clinic, where newly diagnosed children and their parents try to understand the autoimmune disease and come to the realization that they will have to spend every day for the rest of their lives managing it.

“New ways of monitoring blood sugar and dosing insulin are coming,” said Griffin, M.D., Ph.D. “But until we figure out a way to keep the body from attacking itself, we are limited to replacing insulin the body can no longer make.  This is lifesaving, but it requires constant vigilance, and there is no way to take a break from therapy. That can be very frustrating.”

A desire to get to the root of the disease helps drive Griffin’s research, including the ongoing T-rex clinical trial.

This Phase 2 clinical trial is conducted collaboratively by Sanford Health and Caladrius Biosciences Inc., (Caladrius)(Nasdaq: CLBS).

The study opened in March 2016, and the first enrollee recently received his final evaluation as part of the study.

“This is a significant milestone, but it’s only the beginning of getting these children through the trial successfully,” said Griffin, lead investigator for the trial.

The trial has completed enrollment of 110 children with type 1 diabetes. The study started with two sites at Sanford Health in Sioux Falls, South Dakota, and Fargo, North Dakota, and expanded to 13 additional sites across the United States.  Griffin and Fargo, North Dakota-based pediatric endocrinologist Luis Casas, M.D., are the study’s principal investigators at Sanford Health.

Experts from Sanford Health recently talked about how public/private partnerships can accelerate research and discoveries at the “Unite to Cure: “The Fourth International Vatican Conference – How Science, Technology and 21st Century Medicine Will Impact Culture and Society,” in Rome.

The T-rex trial is testing the potential of CLBS03, Caladrius’ cell therapy consisting of each patient’s own regulatory T cells, or Tregs, to help the body fight type 1 diabetes. After a single dose of expanded autologous Treg cells or placebo, subjects are followed for two years, with the primary endpoint of persistence of insulin production at one year after treatment.

People with type 1 diabetes experience a loss of insulin-producing beta cells as their immune system targets these cells inappropriately. Treg cells usually keep the immune system under control, but they are lacking in number and activity in people with type 1 diabetes.

The Sanford Project: T-Rex Study is exploring whether expanding the body’s supply of Treg cells can rebalance the immune system, stop destruction of beta cells and preserve insulin production. Participants were randomized to either of two doses in the treatment groups or to placebo. For those in the treatment groups, the participant’s own Treg cells were extracted from the body, purified, expanded in culture, and then returned to blood circulation. The cell identification and expansion process is patented technology licensed by Caladrius, a cell-therapy development company.

The therapy being used in this trial, CLBS03, has received fast-track designation from the U.S. Food and Drug Administration (FDA), a first for any type 1 diabetes intervention. That designation is reserved for drugs or biologics that address a serious health condition, like type 1 diabetes, where there is an unmet medical need.

This status allows for more frequent communication with the FDA and faster feedback about the therapy during the approval process. It also allows researchers to submit data and reports on a rolling basis.  CLBS03 also has been granted European Medicines Agency’s Advanced Therapeutic Medicinal Product classification and FDA Orphan Drug designation as a potential new treatment for recent-onset T1D.

The Sanford Project is a cornerstone initiative at Sanford Health focusing on finding a cure for type 1 diabetes. The initiative was launched as part of a $400 million gift from philanthropist Denny Sanford in 2007.

About Caladrius Biosciences
Caladrius Biosciences, Inc. is a clinical stage biopharmaceutical company with multiple technology platforms targeting autoimmune and select cardiology indications. The Company is investigating its lead product candidate, CLBS03, an ex vivo expanded polyclonal T regulatory cell therapy for the treatment of recent-onset type 1 diabetes, in an ongoing Phase 2 trial.  CLBS14, CD34+ cell therapy intended as a treatment for coronary microvascular dysfunction, is Caladrius’ proprietary and patent protected formulation of CD34 cells designed specifically to enhance the potency of the CD34 cells for repair and regeneration of cardiovascular tissue. Its companion product, CLBS12, is specifically formulated for intramuscular administration for the treatment of lower extremity ischemia.  A phase 2 study of CLBS12 as a treatment for critical limb ischemia has initiated in Japan, a successful outcome of which will qualify the program for consideration of early conditional approval based on discussions with the Japanese regulatory authorities as provided for under Japan’s progressive regenerative medicine regulations.   For more information about Caladrius please visit www.caladrius.com.

About Sanford Health
Sanford Health is one of the largest health care systems in the nation, with 44 hospitals and nearly 300 clinics in nine states and nine countries. Headquartered in Sioux Falls, South Dakota, and serving the Upper Midwest, with nearly 1,400 physicians, Sanford Health is dedicated to several initiatives, including global clinics, genomic medicine and specialized centers researching cures for type 1 diabetes, breast cancer and other diseases. Sanford Health has 28,000 employees, making it the largest employer in the Dakotas. Nearly $1 billion in gifts from philanthropist Denny Sanford over the past decade have transformed how Sanford Health can improve the human condition. For information, visit sanfordhealth.org.

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