Bridget Hoyt

Emerging treatments like rusfertide may reduce phlebotomies and improve life for patients with polycythemia vera, according to Dr. Aaron Gerds.

New Therapies Advance for Rare Blood Cancer Polycythemia Vera

Dr. Aaron Gerds, assistant professor of medicine at Cleveland Clinic Taussig Cancer Institute, emphasized the growing momentum in treatment development for polycythemia vera, a rare blood cancer. While the disease may not receive widespread attention, he noted that researchers are actively pursuing new therapies, including rusfertide and other agents targeting the hepcidin pathway.

Rusfertide, in particular, may reduce or eliminate the need for phlebotomies, potentially improving patients’ quality of life. Gerds highlighted that multiple new therapies are being studied — such as givinostat in a phase 3 trial — and stressed that even in rare diseases, progress is being made.

What would you like patients to take away from today's conversation?

There are a couple of important things to note. One is that we are developing — and continue to develop — new therapies for polycythemia vera. Even though it’s a rare disease and not often highlighted in the broader pantheon of diseases presented at ASCO, it’s still a very important condition, especially for the people who have it.

There is a group of us who are very invested in this disease and are working hard to develop new therapies for it. Rusfertide isn’t the only one in development — there are also other drugs targeting the hepcidin pathway, as well as agents outside that pathway. For example, givinostat is currently in a randomized phase 3 trial as well as a registration-type study, also seeking approval.

Even though it’s rare, we are working hard to develop better treatment options. That’s the most important takeaway.

Specifically, with rusfertide, it’s exciting because it’s a new therapy that reduces or even eliminates the need for phlebotomies. That can really improve patients’ quality of life, which is important. Having another tool in our arsenal to care for these patients is exciting and can offer a lot to our patients.

Transcript has been edited for clarity and conciseness.

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New clinical trials are advancing treatments for cutaneous T-cell lymphoma, advanced solid tumors, pediatric sarcomas and ALK-positive lung cancer.



Recent progress in cancer research is driving new treatment options for people with cutaneous T-cell lymphoma, advanced solid tumors, pediatric sarcomas and ALK-positive lung cancer. Several clinical trials have reached important milestones, including new site activations, first patient dosing and completion of early-phase studies.

VCU Massey First U.S. Site in Global Phase 2a Trial of PTX-100 for Cutaneous T-Cell Lymphoma

VCU Massey Comprehensive Cancer Center is the first site in the United States to be activated in a global phase 2a trial evaluating PTX-100 (also known as GGTI-2418) for the treatment of cutaneous T-cell lymphoma, a rare and often treatment-resistant form of T-cell lymphoma, according to a news release from the cancer center.

PTX-100 is a first-in-class small molecule that blocks geranylgeranyl transferase-1, an enzyme involved in cancer cell survival.

The global phase 2a trial is enrolling people with relapsed or treatment-resistant cutaneous T-cell lymphoma to further assess the safety and effectiveness of PTX-100. It is taking place at leading academic cancer centers in the United States, Australia and Europe.

For more information about the study or to inquire about eligibility, patients and referring physicians can contact Massey’s Clinical Trials Office or submit a clinical trial request form.

First Patient Dosed in Phase 2 of BIPAVE-001 Trial for Advanced Solid Tumors

The first patient has been dosed in the phase 2 portion of the phase 1/2 BIPAVE-001 trial evaluating AI-081, a potential best-in-class PD-1 and VEGF bispecific antibody.

The BIPAVE-001 phase 1/2 trial is evaluating the safety and effectiveness of AI-081, along with how the body processes the drug, in two parts. Part A is an ongoing first-in-human phase 1 study testing different doses in patients with advanced solid tumors. Part B is the phase 2 portion, focusing on finding the best dose by evaluating AI-081 either alone or combined with standard treatments, depending on cancer type. The study is underway at 11 sites across the United States and has also been approved by China’s regulatory agency.

AI-081 is a bispecific antibody that targets PD-1 and VEGF for advanced solid tumors. It combines two high-affinity antibodies in a proven design to boost immune response. Preclinical studies showed AI-081 binds both targets strongly and works better than similar drugs by enhancing PD-1 blockade while blocking VEGF. This dual action may improve treatment effectiveness.

To learn more about the trial, use the clinical trial ID NCT06635785 at clinicaltrials.gov, or visit www.oncoc4.com.

Actuate Completes Phase 1 Trial of Elraglusib Combos for Pediatric Cancer

Actuate Therapeutics announced the completion of the phase 1 portion of its clinical study evaluating elraglusib monotherapy or in combination with irinotecan, irinotecan plus temozolomide, or cyclophosphamide plus topotecan in pediatric patients with refractory malignancies.

Elraglusib inhibits glycogen synthase kinase-3 beta, a target involved in cancer progression. Early signs of activity, especially in treatment-resistant Ewing sarcoma, support advancing the program to a phase 2 study in children, adolescents and adults with relapsed or refractory disease.

The open-label Phase 1/2 trial enrolled 10 patients with relapsed or refractory Ewing sarcoma treated with elraglusib plus topotecan and cyclophosphamide. The study is ongoing at multiple centers.

For more information about the trial or to inquire about eligibility, use the clinical trial ID NCT04239092 at clinicaltrials.gov or contact Actuate Therapeutics.

ALKAZAR Phase 3 Trial Evaluating Neladalkib for Lung Cancer Subset

The first patient has been dosed in the phase 3 trial ALKAZAR, evaluating neladalkib for tyrosine kinase inhibitor (TKI)-naive patients with advanced anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer, versus Alecensa (alectinib), a front-line standard of care, according to a news release from Nuvalent.

Neladalkib is a brain-penetrant ALK inhibitor designed to work in patients whose lung cancer has stopped responding to earlier ALK-targeted treatments, as per the release. It may help control tumors with tough-to-treat mutations and brain metastases while avoiding certain side effects linked to other drugs.

The ALKAZAR trial is a global phase 3 randomized, controlled study. Around 450 patients who have not previously received a TKI will be enrolled and randomly assigned to receive either neladalkib or Alecensa. The primary endpoint is progression-free survival. Secondary endpoints include overall survival, progression-free survival by investigator assessment, time to intracranial response and intracranial objective response rate (assessed by blinded independent central review), intracranial duration of response, objective response rate, duration of response, time to intracranial progression and safety.

The trial is recruiting in St. Louis, Missouri and New Hyde Park, New York.

For more information about the trial or to inquire about eligibility, use the clinical trial ID NCT06765109 at clinicaltrials.gov.

“Massey becomes first U.S. site to enroll patients in global phase 2a trial of first-in-class therapy for T-cell lymphoma.” VCU Massey Comprehensive Cancer Center, July 18, 2025.

“OncoC4 doses first patient with potential best-in-class PD-1 VEGF bispecific antibody AI-081 in phase 2 portion of BIPAVE-001 trial for advanced solid tumors in US.” OncoC4, July 22, 2025.

“Actuate Therapeutics advances clinical program in Ewing sarcoma after positive phase 1 trial demonstrates complete and partial responses in difficult-to-treat pediatric sarcomas.” Actuate Therapeutics, July 17, 2025.

“Nuvalent announces initiation of ALKAZAR phase 3 randomized controlled trial evaluating neladalkib for patients with TKI-naive ALK-positive NSCLC.” Nuvalent, July 21, 2025.

Articles

Recent advancements in cancer research are expanding treatment options for cutaneous T-cell lymphoma, advanced solid tumors, pediatric sarcomas, and ALK-positive lung cancer. Key clinical trials have achieved significant milestones, such as VCU Massey being the first U.S. site for a global phase 2a trial of PTX-100 for cutaneous T-cell lymphoma. The BIPAVE-001 trial has dosed its first patient with AI-081 for advanced solid tumors. Actuate completed a phase 1 trial of elraglusib for pediatric cancer, and the ALKAZAR phase 3 trial is evaluating neladalkib for ALK-positive lung cancer.

New clinical trials are advancing treatments for cutaneous T-cell lymphoma, advanced solid tumors, pediatric sarcomas and ALK-positive lung cancer.

New Trials Advance Treatments for Lymphoma, Lung Cancer and Sarcomas

SH-110, a liquid cancer treatment for glioma, earned orphan drug status from the FDA, helping patients who struggle to swallow pills access therapy.

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to SH-110, a liquid medication designed to treat glioma, a rare form of brain cancer, in patients who have trouble swallowing pills.

“For too long, many patients with glioma and their pharmacists and caregivers had no other choice but to break open capsules and expose themselves to unnecessary hazards,” Sharon Cunningham, CEO of Shorla, said in the news release. “SH-110 is a proprietary product that offers them a safer and more convenient formulation to treat this rare brain cancer.”

SH-110 is expected to improve treatment flexibility and access for patients with glioma, which affects about 13,000 adults and 2,000 children in the U.S. annually, according to the National Brain Tumor Society.

While SH-110 offers broader dosing and administration options to support compliance and coverage, its most significant benefit may be for patients who have difficulty swallowing, known as dysphagia, as per the release.

These individuals often depend on specialized compounding pharmacies or must modify their own capsules to take their medication. SH-110 will be part of an expanding catalog of patient-friendly cancer therapies developed by Shorla and recently approved by regulators, according to Shorla.

“This is a difference-maker in the lives of those who suffer from glioma,” Orlaith Ryan, chief technical officer and co-founder of Shorla, said in the news release. “SH-110 complements our other Shorla treatments that are designed to be easier for patients to use and for caregivers and providers to administer.”

SH-110 marks the third oral liquid in Shorla’s portfolio of accessible cancer therapies and reflects the company’s continued focus on developing treatments that improve both safety and usability.

Orphan drug designation is status granted by the FDA to drugs intended to treat rare diseases or conditions that affect fewer than 200,000 people in the United States, according to the FDA. It provides incentives such as tax credits, user fee waivers, and seven years of market exclusivity upon approval.

Liquid Suspension Treatment FDA Approvals

Alongside SH-110, Shorla offers two other liquid suspension treatments: Jylamvo, an oral methotrexate for acute lymphoblastic leukemia (ALL) and other cancers, and Imkeldi, a liquid version of imatinib used in ALL, chronic myeloid leukemia, gastrointestinal stromal tumors and additional indications.

Jylamvo was initially approved by the FDA in November 2022 for select uses in adults with ALL, cutaneous T-cell lymphoma, and relapsed or refractory non-Hodgkin lymphoma. 

The approval was expanded in October 2024

 to support treatment of children with ALL as well.

Jylamvo is an orange-flavored liquid form of methotrexate designed to remove the need for pill splitting, crushing, or compounding into a liquid. According to the National Cancer Institute, methotrexate may work by blocking cells from using folic acid to make DNA, which can help destroy cancer cells and reduce immune activity.

Jylamvo also comes in a formulation that remains stable at room temperature for 90 days, so it does not require cold chain storage before dispensing.

“This approval follows Jylamvo’s successful use in adult patients and represents a critical step forward in addressing the unmet need of pediatric care in oncology and autoimmune diseases,” Cunningham said in a previous news release from Shorla Oncology. “We are pleased to offer a convenient, patient-friendly alternative for both adult and pediatric patients in the U.S., as we continue to develop innovative solutions for those with limited treatment options.”

Imkeldi gained FDA approval in November 2024.

“Shorla Oncology announces FDA orphan drug designation for SH-110 to treat rare brain cancer using oral liquid,” Shorla Oncology. Accessed July 16, 2025.

“Shorla Oncology announces US Food and Drug Administration expanded approval of JYLAMVO (methotrexate), an oncology and autoimmune drug for pediatric indications,” by Shorla Oncology. Accessed July 16, 2025.

“Shorla Oncology announces FDA approval of Imkeldi (imatinib) oral solution, an oral liquid for treatment of certain leukemias and other cancers,” by Shorla Oncology. Accessed July 16, 2025.

The FDA has granted orphan drug designation to SH-110, a liquid medication for glioma patients who struggle with swallowing pills. This formulation aims to enhance treatment flexibility and accessibility, particularly benefiting those with dysphagia. SH-110 is part of Shorla's expanding portfolio of patient-friendly cancer therapies. The orphan drug status provides incentives like tax credits and market exclusivity. Shorla also offers Jylamvo and Imkeldi, liquid treatments for various cancers, enhancing patient compliance and convenience by eliminating the need for pill modification or specialized compounding.

FDA Grants Orphan Status to Liquid Glioma Treatment SH-110

New clinical trials are advancing in hard-to-treat cancers, offering targeted and less toxic options for patients with limited treatment paths.

New clinical trials are opening or progressing in several hard-to-treat cancers, including pediatric brain tumors, colorectal cancer, ovarian cancer, bladder cancer and leukemia.

These studies are testing treatments designed to be more targeted, less toxic or better tailored to individual tumors. In children and young adults with brain cancer, a new therapy will deliver radiation directly into the tumor, while other trials explore new drug combinations, cell therapies, and tumor-matching technologies.

Although still in early phases, each trial represents a step toward expanding options and improving care for patients who often face limited paths forward with standard treatments.

FDA Clears Reyobiq Trial for Pediatric Brain Cancer

The U.S. Food and Drug Administration (FDA) has cleared the investigational new drug application for Reyobiq (rhenium Re186 obisbemeda) for the treatment of pediatric patients with supratentorial recurrent, refractory, or progressive high-grade glioma (HGG) and ependymoma, according to a news release from Plus Therapeutics.

Reyobiq delivers high-dose radiation directly into the tumor using convection-enhanced delivery (CED), which helps protect healthy brain tissue by bypassing the blood-brain barrier.

The trial, called ReSPECT-PBC, will enroll patients ages 6 to 21 (and some up to 25) with recurrent or progressive HGG or ependymoma. The trial will evaluate safety and dosing in phase 1 and effectiveness in phase 2.

Visit respect-trials.com to stay updated on enrollment.

UroGen Completes Enrollment in Phase 3 UGN-103 Bladder Cancer Trial

UroGen Pharma has completed patient enrollment in its phase 3 UTOPIA clinical trial of UGN-103 (mitomycin) for intravesical solution, a next-generation formulation in development for the treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

The study includes 99 patients receiving weekly bladder instillations of UGN-103 for six weeks. The main goal is to measure complete response at three months, with follow-up for up to a year.

UGN-103 uses RTGel®, a hydrogel-based delivery system designed to keep medication in the bladder longer. It is intended to offer similar benefits to Zusduri (another UroGen mitomycin therapy) but with faster preparation and easier use.

For more information on the UTOPIA study, visit clinicaltrials.gov using the trial ID: NCT06331299.

First Patient Dosed in Phase 2 Trial in Colorectal Cancer

The first patient has been dosed in the phase 2 clinical trial evaluating the efficacy of leronlimab in patients with relapsed or refractory microsatellite stable colorectal cancer, according to a news release from CytoDyn.

The trial is enrolling patients across multiple sites with support from Syneos Health. Dr. Ben Weinberg of Georgetown University is serving as the lead investigator.

Leronlimab is an experimental antibody designed to block CCR5, a protein that may play a role in cancer spread and immune system behavior.

This study builds on earlier research suggesting potential benefit in patients with relapsed colorectal cancer, as well as prior studies in metastatic triple-negative breast cancer.

CytoDyn hopes this study will help determine how broadly leronlimab’s mechanism of action may apply to other solid tumors.

CERo Reports No DLTs in First CER-1236 AML Patient

CERo Therapeutics has completed the initial safety evaluation of the first patient in its phase 1 trial of CER-1236, a first-in-class engineered T-cell therapy for acute myeloid leukemia (AML), according to a news release from the company.

The patient experienced no dose-limiting toxicities during the 28-day observation period. A second patient with AML has consented to enroll and is expected to be dosed soon.

CER-1236 targets TIM4L, a previously unexplored pathway, and works through a phagocytic mechanism. The treatment is manufactured from a patient’s own cells.

The trial, called CertainT-1, is evaluating safety and early signs of efficacy in patients with relapsed/refractory AML, measurable residual disease, or TP53-mutated AML or myelodysplastic syndromes.

Outcomes being tracked include side effects, response rates and measurable residual disease. CERo also plans to begin a second trial of CER-1236 in solid tumors later this year.

For more information on the study, please visit clinicaltrials.gov using the trial ID: NCT06834282.

Allarity Doses Second Patient in Ovarian Cancer Trial of Stenoparib

Allarity Therapeutics has dosed the second patient in its phase 2 trial evaluating stenoparib for advanced, recurrent ovarian cancer that is resistant to or ineligible for platinum-based chemotherapy, according to a news release from the company.

This trial targets patients with few treatment options, aiming to assess stenoparib’s safety and potential benefit. Earlier data showed the drug was well tolerated and provided durable responses with twice-daily dosing.

Stenoparib is an oral therapy that blocks both PARP and WNT signaling pathways — key drivers in many cancers. Its dual mechanism may offer a more targeted and less toxic alternative to chemotherapy.

The trial also supports further development of Allarity’s Drug Response Predictor, a tool designed to identify patients most likely to respond based on gene expression in their tumors.

Rapid early enrollment signals strong interest from investigators and reflects ongoing need for better therapies in this difficult-to-treat cancer population.

“Plus Therapeutics Announces FDA Clearance of its Investigational New Drug Application using REYOBIQTM for the Treatment of Childhood Brain Cancer” by Plus Therapeutics

“UroGen Announces Completion of Enrollment in the Phase 3 UTOPIA Clinical Trial of UGN-103 for the Treatment of Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer” by UroGen Pharma

“CytoDyn Announces First Patient Dosed in Phase II Oncology Trial Evaluating Leronlimab in Metastatic Colorectal Cancer” by CytoDyn Inc.

“CERo Therapeutics Completes Initial Safety Evaluation in First Patient Treated with CER-1236 in Phase 1 Trial for AML” by CERo Therapeutics

“Allarity Therapeutics Announces Dosing of Second Patient in New Phase 2 Trial of Stenoparib in Advanced Ovarian Cancer” by Allarity Therapeutics

For more news on cancer updates, research and education, don’t forget to 

Recent clinical trials are advancing in challenging cancer types, including pediatric brain tumors, colorectal, ovarian, bladder cancers, and leukemia. These trials focus on targeted, less toxic, and personalized treatments. Notable developments include FDA clearance for Reyobiq in pediatric brain cancer, UroGen's phase 3 bladder cancer trial completion, and initial dosing in colorectal cancer and ovarian cancer trials. CERo's T-cell therapy for AML shows no dose-limiting toxicities. These trials aim to expand treatment options and improve outcomes for patients with limited standard treatment paths.

Bridget Hoyt

New Therapies Advance for Rare Blood Cancer Polycythemia Vera

New Trials Advance Treatments for Lymphoma, Lung Cancer and Sarcomas

FDA Grants Orphan Status to Liquid Glioma Treatment SH-110

New Trials Target Brain, Bladder, Ovarian and Other Cancers

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