Autoimmune Sufferers May Benefit from Stem Cell Therapies

7 Min. Read | August 12, 2021

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How Are Stem Cells Being Used to Treat Autoimmune Diseases?

Blood stem cell transplants have become a potentially curative option for some severe forms of scleroderma (systemic sclerosis), multiple sclerosis (MS), and lupus (SLE or systemic lupus erythematosus). Clinical trials are also underway for treatment-resistant or refractory rheumatoid arthritis and juvenile idiopathic arthritis. Recent research suggests Parkinson’s disease also has an early autoimmune component.

We all have learned to love and respect our immune systems during this time of COVID. But if you have one of those autoimmune diseases, it may be more of a love-hate relationship. Those diseases occur when your system—designed to defend against invading pathogens—turns against your own tissues. A hot area of stem cell therapy research today is focused on ways to get an abnormal immune system back on track to defending you, and not attacking you.

How Do Stem Cell Therapies Work?

Stem cell therapies work by resetting the immune system after a conditioning treatment that removes most of the misbehaving immune cells. A patient’s own stem cells, collected and preserved prior to the treatment, are infused after the conditioning.  Long-term follow up of treated patients shows that the effect is not just the upfront suppression of the bad cells, but results from resetting the person’s immune response, resulting in stable remission of their disease.

Autoimmune diseases have become more prevalent over the past 25 years according to a recent study from the National Institutes of Health (NIH). A group at Northwestern University has tested stem cell transplants in 23 different autoimmune diseases.

Is Multiple Sclerosis (MS) a Candidate?

Yes, when it is severe and resistant to existing medical treatment. Blood stem cell transplant (HSCT, Hematopoietic Stem Cell Transplant) is very effective in people with relapsing MS with highly active inflammation. It can also help some people with early progressive MS who are experiencing active inflammation. In one study, nearly 70 percent of people who received HSCT didn’t have a single relapse in five years.

A clinical trial that might open up the option of curative stem cell transplants to more MS patients began at the National Institute of Allergy and Infectious Diseases (NIAID) last year. The trial will test HSCT versus the best available drugs and compare the safety, efficacy and cost-effectiveness over time of the two approaches.

NIAD head, Dr. Anthony Fauci, said at the time (squeezed in between COVID briefings) that HSCT “has the potential to halt the progress of relapsing MS, eliminate the need for a person to take lifelong medication, and allow the body to partially regain function.”

Can Scleroderma/Systemic Sclerosis Benefit from Stem Cells?

Yes. Three high quality clinical trials in the past decade have shown that blood stem cell transplants can be an effective option for some patients with progressive systemic sclerosis, also called scleroderma.  An NIH-funded study released in 2018 showed that transplanting a person’s own blood-forming stem cells was superior to treatment with Immune-suppressive drugs in improving survival and quality of life.

Scleroderma patients have hardening of their skin and internal connective tissues that can be quite debilitating. Again, Dr. Fauci weighed in, this time pre-COVID. “These results add to the growing evidence that stem cell transplants should be considered as a potential treatment option for people with poor-prognosis scleroderma.”

Are Stem Cell Therapies an Option for Treating Systemic Lupus?

Refractory Lupus, the form that is not responsive to available drugs, has been shown to benefit from blood stem cell transplant as early as 2007. The risks associated with the conditioning treatments to remove the bad cells were considered pretty risky in those early days, but those have been dramatically improved bringing down the risk and complexity of the therapy.

In 2018, the Lupus Foundation of America launched a trial using a type of stem cell that might not require as much pre-treatment because the cells directly tame inflammatory cells. That type of cell, mesenchymal stem cells (MSCs), however, have been shown in some studies to be somewhat defective in patients with autoimmune diseases. So, in order to preserve the value of using a patient’s own cells, some researchers are testing the idea of creating new personal MSCs from the type of stem cell known as iPSCs (induced pluripotent stem cells). Those iPSCs can be generated from healthy cells stored by GoodCell and can then make any desired cell type including MSCs.

What’s in the Future for Stem Cells and Autoimmune Disease?

Many teams are working on ways to make cell therapies more broadly available. The company gamidaCell has developed a way to expand the small number of blood stem cells in cord blood to be sufficient to transplant for an adult. They have shown positive results in the early phases of the clinical trials and are now in the final pre-marketing Phase 3. The number of blood stem cells stored by GoodCell are not sufficient for transplant today, but with the rapid advances in research like gamidaCell, that should change in the not-too-distant future.

As mentioned with Lupus, research teams are looking to make various therapeutic cells from iPSC-type stem cells.  Others are looking to employ another type of immune cell, called regulatory T Cells or Tregs. Abata Therapeutics plans a trial using them for MS and Sonoma Bio has a trial planned for rheumatoid arthritis.

Since GoodCell preserves all types of your immune cells, these research paths show the incredible potential that exists in your blood. With your biobanked cells, you will be prepared when research turns cell science into cell medicine.