A Houston-based, female-founded biotech company has developed a treatment that could prove to be an effective therapy for a rare blood cancer.

Cellenkos Therapeutics has completed promising Phase 1b testing of its Treg cell therapy, CK0804, in the fight against myelofibrosis. According to a news release from the Cellenkos team, the use of its cord-blood-derived therapeutics could signal a paradigm shift for the treatment of this hard-to-fight cancer.

Cellenkos was founded by MD Anderson Cancer Center physician and professor Simrit Parmar. Her research at the hospital displayed the ability of a unique subset of T cells’ capability to home in on a patient’s bone marrow, restoring immune balance, and potentially halting disease progression.

Myelofibrosis has long been treated primarily with JAK (Janus Kinase) inhibitors, medications that help to block inflammatory enzymes. They work by suppressing the immune response to the blood cancer, but don’t slow the progression of the malady. And they’re not effective for every patient.

“There is a significant need for new therapeutic options for patients living with myelofibrosis who have suboptimal responses to approved JAK inhibitors,” Parmar says. “We are greatly encouraged by the safety profile and early signs of efficacy observed in this patient cohort and look forward to continuing our evaluation of the clinical potential of CK0804 in our planned expansion cohort.”

The expansion cohort is currently enrolling patients with myelofibrosis. What exactly are sufferers dealing with? Myelofibrosis is a chronic disease that causes bone marrow to form scar tissue. This makes it difficult for the body to produce normal blood cells, leaving patients with fatigue, spleen enlargement and night sweats.

Myelofibrosis is rare, with just 16,000 to 18,500 people affected in the United States. But for patients who don’t respond well to JAKs, the prognosis could mean a shorter span than the six-year median survival rate outlined for the disease by Cleveland Clinic.

Helping myelofibrosis patients to thrive isn’t the only goal for Cellenkos right now.

The company seeks to aid people with rare conditions, particularly inflammatory and autoimmune disorders, with the use of CK0804, but also other candidates including one known as CK0801. The latter drug has shown promising efficacy in aplastic anemia, including transfusion independence in treated patients.

The company closed its $15 million series A round led by BVCF Management, based in Shanghai, in 2021. Read more here.