CIRM Approves Up to $12 Million in Funding for Rare Immune Disorder

The governing board of the California Institute for Regenerative Medicine approved almost $12 million in funding for the development of an experimental therapy for patients with IPEX syndrome, a rare autoimmune condition.

Photo: Maria Millan, president and CEO of CIRM

Children born with IPEX syndrome have abnormalities in the FOXP3 gene, which controls the production of a type of immune cell called a T Regulatory or Treg cell. Without a normal FOXP3 +Treg cells other immune cells attack the body leading to the development of IPEX syndrome, Type 1 diabetes, severe eczema, damage to the small intestines and kidneys and failure to thrive. The syndrome mostly affects boys, is diagnosed in the first year of life and is often fatal.

Current treatments include the use of steroids to suppress the immune system or a bone marrow stem cell transplant, which requires a healthy, closely matched donor to reduce the risk of graft vs host disease where the donated immune cells attack the recipient’s tissues.

The CIRM award will go to Rosa Bacchetta and her team at Stanford University who have developed a therapy using the patient’s own natural CD4 T cells that, in the lab, have been genetically modified to express the FoxP3 gene and converted into Treg cells. Those cells are then re-infused into the patient with a goal of determining if this approach is both safe and beneficial.

Because the cells come from the patients there will be fewer concerns about the need for immunosuppressive treatment to stop the body rejecting the cells. It will also help avoid the problems of finding a healthy donor and graft vs host disease.

Bacchetta received approval from the U.S. Food and Drug Administration to test this approach in a phase 1 clinical trial for patients suffering with IPEX syndrome.

“Children with IPEX syndrome clearly represent a group of patients with an unmet medical need, and this therapy could make a huge difference in their lives,” said Maria Millan, president and CEO of CIRM. “Success of this treatment in this rare disease presents far-reaching potential to develop treatments for a larger number of patients with a broad array of immune disorders resulting from dysfunctional regulatory T cells.”

Besides a strong scientific recommendation for funding the project, the review team said the project commits to the principles of Diversity, Equity and Inclusion by proposing to focus on enrolling people who are low-income, uninsured or members of traditionally overlooked racial and ethnic minority communities.

CIRM was created by the people of California to accelerate stem cell treatments to patients with unmet medical needs and funded with $5.5 billion. With more than 150 active stem cell programs in its portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality.

Author: Rare Daily Staff