NOVEL T REGULATORY CELL THERAPY
AUTOIMMUNE DISEASE, TRANSPLANTATION
The ultimate goal of the research in the field of transplantation is to develop immunological tolerance. It would allow for a patient's body to accept a transplanted organ or cells without the need to take anymore toxic anti-rejection medications.
We have spent last several years developing conditions for a novel cell therapy, which may ultimately lead to the immunologic tolerance and replace need for immunosuppression.
T-regulatory cells (Tregs) are known to have immunomodulatory properties and can prevent immunologic rejection as well as control autoimmune disease.
In order to utilize Tregs in the clinical setting, we optimized a Tregs ex vivo expansion protocol and conducted a full-scale clinical grade Treg expansion in our cGMP facility at The University of Chicago.
Our subsequent goal is to conduct a phase 1/2 clinical study testing safety and efficacy of the approach in the context of kidney, liver or islet transplantation.
Independently, the same Tregs can apply in many autoimmune diseases bringing hope for full recovery in patients with new onset of type 1 diabetes, multiple sclerosis, Crohn disease, ulcerative colitis, rheumatoid arthritis and primary sclerosing cholangitis.
This cutting edge complex technology is advanced to the level of clinical application and , developed only in a few centers all over the world besides University of Chicago.
We have also established close collaborations with those centers promoting the progress in the field.
Dr. Witkowski is seeking funding for the first phase 1/2 clinical studies allowing to implement this cutting edge technology.
Cost of a single Treg cell processing is $100,000. So, the cost of the study for 10 patients is $1M.
We have developed a close and fruitful collaboration with Professor Piotr Trzonkowski from the Medical University of Gdansk in Poland (in the first row). He has been a pioneer in the field of Treg clinical therapy. He implemented it first in patients with GVHD and then in children with new onset of Type 1 diabetes mellitus.
Prof. Trzonkowski as well as Dr Natalia Marek-Trzonkowska visited us in 2010. Adam Krzystyniak remained at our GMP facility for over 2 years setting up the Treg processing line.
Since then, Dr. Karolina Golab took over and has been leading the Treg ex-vivo expansion program at our laboratory.
We have optimized Treg cell ex vivo expansion protocols adapted them to the US clinical standards regulated by the FDA with help of the world leaders in Treg therapy: Dr Qizhi Tang and Dr Jeff Bluestone from UCSF.
We have established a structured collaboration and scientific exchange related to Treg
therapy as well pancreatic islet isolation and transplantation with Professor Trzonkowski's
group at the Medical University of Gdansk, Cell T and other universities in Poland in the frame of the Scientific Program StrategMed sponsored by the Government of Poland.
We have also been involved in activity with the A FACTT collaborative group (Action to Focus and Accelerate Cell-based Tolerance-inducing Therapies) supported by COST European Collaboration in Science and Technology.
Dr. Witkowski was invited as a speaker during A FACTT Meeting in 2015 and Dr. Karolina Golab gave a talk in 2016 and 2017.
Recently, A FACTT group published consensus guidelines related to Treg ex-vivo expansion protocol: " Minimum information about T regulatory cells (MITREG): a step towards reproducibility and standardization. Frontiers in Immunology, 2018 in press."