Biotechnology company Talaris Therapeutics, Inc. submitted an Investigational New Drug (IND) application to the FDA for their novel cell therapy treatment FCR001. As per a press release, the FDA approved the application, allowing for an evaluation of the therapy. FCR001 is designed to treat patients with diffuse systemic sclerosis (SSc).
Diffuse Systemic Sclerosis (SSc)
Diffuse systemic sclerosis (SSc) is a form of systemic sclerosis, an autoimmune disorder characterized by organ and skin fibrosis (hardening and scarring). While localized scleroderma only affects some parts of the body, diffuse Ssc can cause:
skin thickening over large areas of the body, usually the fingers, hands, arms, anterior trunk, legs and face. There can be significant associated organ damage, including to the gastrointestinal tract, kidneys, lungs and heart.
Females are 4x more likely to develop SSc than males are. Unfortunately, SSc has a higher mortality rate compared to other forms of scleroderma. Symptoms include:
- Swelling of the hands and fingers
- Open sores on fingers
- Heartburn
- Raynaud’s phenomenon
- Skin fibrosis
- Joint pain
- Gastrointestinal distress
- Shortness of breath
- High blood pressure
- Organ problems
Learn more about SSc here.
IND for FCR001
At its core, an Investigational New Drug application asks for permission to administer a drug to humans. Once approved, researchers can begin clinical trials to test the safety, tolerability, and efficacy of drug therapies. In this case, Talaris Therapeutics will begin Phase 1/2a trials for FCR001 throughout the country.
FCR001 is an allogenic cell therapy, which means it transplants stem cells from another donor to the patient. This is important, as in prior studies on stem cell transplants, patients received their own stem cells. While stem cell transplants did prevent organ damage, patients risked disease recurrence. For FCR001, patients only require one treatment to build immune tolerance. So far, the drug received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation.
Its approval is based on data from a Phase 2 clinical trial in living recipients of kidney transplants. In addition to improving immune function and tolerance, patients were able to stop using anti-rejection medications and immunosuppressants. Although 7 of the 37 patients had kidney failure due to a previous autoimmune disorder, none experienced symptoms or issues associated with that disorder following FCR001.
