Integrated personalized cell technologies for improving health outcomes in children and adults

CLINICAL TRIALS

MSC in Patients With Xerostomia Post XRT in Head and Neck Cancer

NCT04489732

Current Status: Enrolling Patients

Pilot Study of autologous MSCs for Radiation-Induced Xerostomia (Dry Mouth) in Head and Neck Cancer Patients Completed at University of Wisconsin-Madison.

The UW Program for Advanced Cell Therapy has completed enrollment for the pilot MARSH (Marrow-derived Autologous Stromal cells for the Restauration of Salivary Hypofunction) clinical trial.

Six patients with radiation-induced Xerostomia (Dry Mouth) were treated in the pilot study led by Randall Kimple. The therapy delivers a single dose of GMP-manufactured, autologous Interferon gamma-stimulated bone marrow-derived mesenchymal stromal cells (MSCs) delivered “fresh” into one submandibular salivary gland.

Results from the study will be used to inform the safety and tolerability for feasibility of a UW Program for Advanced Cell Therapy-sponsored Phase 1, dose escalation clinical trial that includes 24 to 30 additional patients. Salivary gland function will be used to evaluate efficacy.

Full specifications of this trial are listed on Clinicaltrials.gov

Additional details for this trail can be found in this Press Release.

BK With VST for Kidney Transplant Patients

NCT05042076

Current Status: Enrolling Patients

Kidney transplant patients face an age-old problem to protect their new kidney and bodies after transplant surgery: how to prevent infections while also safeguarding their new kidney from damage or rejection.

The BK virus causes symptoms similar to a common cold, and most people are infected with this virus at a young age. The human immune system typically fights it off, but the virus continues to linger quietly in the body. The immune system usually keeps it in check, but during transplantation, medications are used to suppress the activity of the immune system to keep the body from rejecting the transplanted kidney.

While immunosuppression is necessary to protect the new organ, it comes with a tradeoff: dormant viruses like BK can start to proliferate, resulting in a balance between preventing infection and causing damage to the kidney or outright rejection. But there just haven’t been many good options for treating BK reinfection.

Traditionally, no antiviral drugs have been effective for fighting BK reinfection, and some are even harmful to new kidneys, without much benefit, so PACT looked to provide a better option.

Early in 2021 PACT launched a first-of-its-kind phase 1 trial using T-cells – a type of immune system cell – donated from a close relative of Adolph to treat the infection. T-cells used for this procedure are collected from donor blood, and those that target BK specifically are purified for transfusion at PACT’s manufacturing laboratory at University Hospital. It takes six hours to prepare the T-cells. Once they are ready, they are infused into the patient to treat the infection.

Like other PACT cell therapies, a significant advantage of these approaches is a very low risk of side effects other than possible injection site irritation because the treatments use the body’s own immune system to fight off the virus.

The goal of a phase 1 trial is to determine what dose is safe for patients to take. Establishing how well a drug or device works comes in later trial phases. PACT hopes to enroll 20 patients. Given the hundreds of kidney transplant patients UW Health cares for in a year, there will be no short supply of potential participants.

Full specifications of this trial are listed on Clinicaltrials.gov

Additional details for this trail can be found in this Press Release.

Treatment of Cytomegalovirus (CMV) Infections With Viral-Specific T Cells

NCT03798301

Current Status: Enrolling Patients

PACT’s first clinical trial, a study to examine cell therapy to treat CMV infection in BMT recipients, will deploy virus-specific white blood cells to treat the potentially lethal reactivation that can occur after a bone-marrow transplant.

Cytomegalovirus (CMV) can affect one in three children by age 5 and can cause fever sore throat, fatigue and swollen glands, but for healthy people with stable immune systems the virus is usually kept in check and doesn’t cause any symptoms. It’s only when a patient’s immune system is suppressed that there is nothing to stop the virus from spreading in the body.

This type of treatment is currently the standard of care in some European countries, but is still considered experimental and is being attempted only at a few elite academic medical centers around the United States.

Full specifications of this trial are listed on Clinicaltrials.gov

Additional details for this trail can be found in this Press Release.

 

A Dose-Escalation Study Evaluating Safety and Tolerability of Viral-Specific T Cells Against CMV in Adult Kidney Transplant Recipients

NCT03950414

Current Status: Enrolling Patients

For the first time in the United States, PACT’s research team will test a personalized cell therapy to treat a common and serious complication facing kidney transplant patients. PACT will study a cutting-edge therapy to treat a viral infection faced by around 30 to 40 percent of kidney and/or pancreas transplant recipients.

The study will deploy virus-specific white blood cells to treat severe cytomegalovirus (CMV) infection after kidney transplantation. UW Health is the largest kidney and pancreas transplant program in the state, performing a majority of Wisconsin’s kidney and kidney-pancreas combined transplants with 315 in 2018.

In such patients, the infection can be fatal, and some anti-viral treatments have serious side effects, such as reducing blood counts and kidney function. Additionally, viruses can develop resistance to antiviral drugs. These factors make PACT’s new approach a potentially safe, effective treatment to stop this virus.

Additional details for this trail can be found in this Press Release.

Full specifications of this trials are listed on Clinicaltrials.gov

 

TCRa/b and CD19 Depleted HSCT for Non-Malignant Hematologic Diseases

Current Status: Not Yet Active

This clinical trial will evaluate hematopoietic stem cell transplant to treat children and young adults with various blood disorders for which transplant is currently the only possibility of cure. These disorders include inherited bone marrow failure syndromes, Myelodysplastic Syndrome, Sickle Cell Disease, Thalassemia, and Paroxysmal Nocturnal Hemoglobinuria with bone marrow failure.

Donor cells come from an HLA half-matched relative, or a closely-matched non-relative.  Some donor cells can be problematic if transplanted, such as alpha/beta T cells that can cause graft-versus-host disease, or B cells that can cause post-transplant lymphoproliferative disease.  In this study using a special technology, the unwanted cells are removed while the cells that have therapeutic benefit, such as 1) stem cells, 2) infection-fighting natural killer and gamma/delta T cells, and 3) cells that facilitate engraftment, are preserved in the transplanted “graft”.

Hematopoietic stem cell transplant, using this special technology, is anticipated to result in faster engraftment and fewer infections.