Cellenkos Therapeutics has completed promising Phase 1b testing of its Treg cell therapy, CK0804, in the fight against myelofibrosis. Photo via Getty Images

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.

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Axiom Space-tested cancer drug advances to clinical trials

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A cancer-fighting drug tested aboard several Axiom Space missions is moving forward to clinical trials.

Rebecsinib, which targets a cancer cloning and immune evasion gene, ADAR1, has received FDA approval to enter clinical trials under active Investigational New Drug (IND) status, according to a news release. The drug was tested aboard Axiom Mission 2 (Ax-2) and Axiom Mission 3 (Ax-3). It was developed by Aspera Biomedicine, led by Dr. Catriona Jamieson, director of the UC San Diego Sanford Stem Cell Institute (SSCI).

The San Diego-based Aspera team and Houston-based Axiom partnered to allow Rebecsinib to be tested in microgravity. Tumors have been shown to grow more rapidly in microgravity and even mimic how aggressive cancers can develop in patients.

“In terms of tumor growth, we see a doubling in growth of these little mini-tumors in just 10 days,” Jamieson explained in the release.

Rebecsinib took part in the patient-derived tumor organoid testing aboard the International Space Station. Similar testing is planned to continue on Axiom Station, the company's commercial space station that's currently under development.

Additionally, the drug will be tested aboard Ax-4 under its active IND status, which was targeted to launch June 25.

“We anticipate that this monumental mission will inform the expanded development of the first ADAR1 inhibitory cancer stem cell targeting drug for a broad array of cancers," Jamieson added.

According to Axiom, the milestone represents the potential for commercial space collaborations.

“We’re proud to work with Aspera Biomedicines and the UC San Diego Sanford Stem Cell Institute, as together we have achieved a historic milestone, and we’re even more excited for what’s to come,” Tejpaul Bhatia, the new CEO of Axiom Space, said in the release. “This is how we crack the code of the space economy – uniting public and private partners to turn microgravity into a launchpad for breakthroughs.”