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.

Jim Allison's groundbreaking work with T cells helped him net the award. Photo courtesy of MD Anderson Cancer Center

Houston scientist wins Nobel Prize for breakthrough cancer treatment

Research Recognition

A University of Texas MD Anderson Cancer Center scientist has been lauded for his cancer research. Jim Allison, Ph.D., was announced as the recipient of the 2018 Nobel Prize in Physiology or Medicine on October 1.

Allison, who is the chair of Immunology and executive director of the Immunotherapy Platform, is the first MD Anderson scientist to receive the world's most coveted award for discoveries in the fields of life sciences and medicine. Allison won for his work in launching an effective new way to attack cancer by treating the immune system rather than the tumor, according to a release.

"I'm honored and humbled to receive this prestigious recognition," Allison says in a statement. "A driving motivation for scientists is simply to push the frontiers of knowledge. I didn't set out to study cancer, but to understand the biology of T cells, these incredible cells to travel our bodies and work to protect us."

Allison shares the award with Tasuku Honjo, M.D., Ph.D., of Kyoto University in Japan. When announcing the honor, the Nobel Assembly of Karolinska Institute in Stockholm noted in a statement that "stimulating the ability of our immune system to attack tumor cells, this year's Nobel Prize laureates have established an entirely new principle for cancer therapy."

The prize recognizes Allison's basic science discoveries on the biology of T cells, the adaptive immune system's soldiers, and his invention of immune checkpoint blockade to treat cancer. According to MD Anderson, Allison's crucial insight was to block a protein on T cells that acts as a brake on their activation, freeing the T cells to attack cancer. He developed an antibody to block the checkpoint protein CTLA-4 and demonstrated the success of the approach in experimental models.

Allison's work led to the development of the first immune checkpoint inhibitor drug which would become the first to extend the survival of patients with late-stage melanoma. Follow-up studies show 20 percent of those treated live for at least three years, with many living for 10 years and beyond, unprecedented results, according to the cancer center.

"Jim Allison's accomplishments on behalf of patients cannot be overstated," says MD Anderson president Peter WT Pisters, M.D., in a statement. "His research has led to life-saving treatments for people who otherwise would have little hope. The significance of immunotherapy as a form of cancer treatment will be felt for generations to come."

"I never dreamed my research would take the direction it has," Allison adds. "It's a great, emotional privilege to meet cancer patients who've been successfully treated with immune checkpoint blockade. They are living proof of the power of basic science, of following our urge to learn and to understand how things work."

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This story originally appeared on CultureMap.

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Houston researchers develop breakthrough device that could bypass spinal injuries

breakthrough research

Scientists at Houston Methodist have announced a significant leap forward for spinal cord injury recovery.

The researchers have developed a device that essentially bypasses spinal injuries, allowing signals from previously “lost” functions to reach the brain, a new study published in Nature Communications shows.

“Most current technologies try to improve whatever function remains after a spinal cord injury,” Dr. Damiano Barone, assistant professor of neurosurgery in the Department of Neurosurgery at Houston Methodist and co-lead on the study, said in a news release. “Our goal is different. Rather than fixing the injury itself, we want to bypass it completely and create an alternative route for signals to travel.”

The study involved a single ultrathin circumferential electrode array made to conform around the spinal cord without penetrating neural tissue, which was implanted into rodent and pig models with spinal injuries. The electrode array was able to interpret motor, sensory and autonomic signals around the injury. Think of it as a set of detours that restore road access to isolated towns after a disaster destroys the highway instead of just rebuilding the highway.

Over the course of three days, the arrays detected signals of intended movement from low-frequency spinal oscillations with more than 94 percent accuracy. This worked across species and was replicated in feasibility studies on human cadavers.

This research could serve as a new foundation for neuroprosthetic implants that could restore connectivity to the 2.5 million people worldwide suffering from spinal injuries that result in loss of ability. Future development could result in everything from restored organ function to mobility, according to Houston Methodist.

George Malliaras, the Prince Professor of Technology in the Department of Engineering at the University of Cambridge, who co-led the study, sees it as a fundamental restructuring of the science of spinal trauma.

“This could represent a paradigm change in how we think about spinal cord injuries,” Malliaras said. “Instead of starting from the idea that what is lost is gone forever, this approach asks whether we can restore function by carrying the signal around the injury.”

Further work involving laboratory models will need to be completed before launching human trials.

Grants from the National Institutes of Health, Houston Methodist Katz Investigator Award, Helaers Research Award and the Engineering and Physical Sciences Research Council helped support the study. Other collaborators on the study include Salim Hadwe, Ruben Serrano, George Psaltakis, Margaux Forner, Chaeyeon Lee, Sydney Swedick, Moleca Ghnnam, Tawfique Hasan and Alejandro Carnicer-Lombarte from the University of Cambridge; and Anton Banta and Xueer Zhang from Houston Methodist.

Abbott assembles expert team to help lure U.S. Space Academy to Texas

space race

State Rep. Greg Bonnen of Friendswood has been tapped to lead a new team that will promote Texas as the future home of the U.S. Space Academy.

Bonnen, a neurosurgeon, chairs Houston Physicians’ Hospital and the powerful Texas House Appropriations Committee. His House district is close to NASA’s Johnson Space Center.

Gov. Greg Abbott appointed the seven-member team. Last month, President Trump signed an executive order establishing the Presidential Commission on the U.S. Space Academy. Commission members, who held their first meeting this month, will recommend a permanent location for the academy.

Texas officials are pushing a site near Johnson Space Center to host the academy. Alabama, Colorado and Florida are among Texas’ competitors.

In a joint statement, U.S. Sen. Ted Cruz and U.S. Rep. Brian Babin, both of Texas, issued a statement backing the state’s bid for the academy. Cruz lives in Houston. Babbin lives in Woodville, about 55 miles south of Beaumont.

“America’s space program is built across the country, but Texas is where the pieces come together,” the lawmakers said. “We are ready to lead the next generation of space pioneers and look forward to showing why Texas is the right home for the U.S. Space Academy.”

The academy’s curriculum will include technical education, leadership development and public service components. Graduates will be set up for careers in the U.S. military, civil service, and aerospace sectors.

The Abbott-appointed team will work with the Texas Space Commission to prepare the state’s proposal for the academy.

In addition to Bonnen, team members with ties to Houston include:

  • Robert Ambrose, who grew up in Houston. He worked at Johnson Space Center before becoming associate director of the Texas A&M Space Institute.
  • Former NASA astronaut Nancy Curry-Gregg, director of the Texas A&M Space Institute. She earned a doctoral degree from the University of Houston and previously worked at Johnson Space Center.
  • Former NASA astronaut Jack Fischer, senior vice president of Houston-based Intuitive Machines. The company builds spacecraft, delivers payloads to the moon and launches satellites.

“Texas is the home of America’s human spaceflight program,” Abbott said in a release. “No state can match what Texas brings to this mission.”

“NASA’s Johnson Space Center, world-class universities, a premier commercial space industry, major military installations, and an unmatched aerospace workforce give Texas every asset the United States Space Academy requires,” the governor added.