Precision Medicine in Childhood Leukemia

Discover how HealthTech innovations are transforming chronic disease management. This blog highlights the latest technologies like remote monitoring, AI, and mobile apps, which improve patient care, outcomes, and reduce costs. Explore real-world examples and see how personalized and preventive approaches enhance quality of life for millions.

Notable Labs is a clinical-stage precision oncology company developing new cancer therapies identified by its Predictive Medicine Platform (PMP), today announced progress for the Phase 2 volasertib program following receipt of “Clearance to Proceed” from the FDA and agreement on the dosing plan for its Phase 2 clinical trial. With trial start-up activities launched in the first quarter of this year, Notable intends to initiate enrollment in the Phase 2 study for PMP-enabled development of volasertib for relapsed refractory (R/R) acute myeloid leukemia (AML) in the coming months.

Notable Labs is transforming pediatric leukemia treatment with its Predictive Precision Medicines Platform (PPMP), which boasts a 97% predictive precision. This platform rapidly identifies the most effective treatments for individual patients, significantly reducing the time and cost of cancer therapy. Notable Labs focuses on personalized medicine, combining high-throughput screening and machine learning to predict patient responses and tailor treatments accordingly. Their innovative approach has shown promising results in clinical validation studies, offering new hope for improving outcomes in pediatric leukemia.

Notable Labs is revolutionizing cancer treatment with its innovative approach to combination therapy, which targets multiple cancer pathways simultaneously. Their Predictive Precision Medicine Platform (PPMP) leverages advanced data analysis and machine learning to identify the most effective drug combinations for individual patients. By using high-throughput screening and bio-simulation, Notable Labs accelerates drug development, reduces costs, and improves clinical trial success rates. This approach promises significant improvements in patient outcomes, particularly for pediatric leukemia, by providing personalized, effective treatments and minimizing side effects.

Notable Labs' cutting-edge diagnostic technology, boasting a 95% prediction accuracy per a Stanford study, is set to revolutionize pediatric leukemia treatment by identifying the most effective therapies. Their mission centers on advancing precision diagnostics to enhance therapy efficacy for young patients. Utilizing high-throughput screening and machine learning, Notable's platform offers tailored treatment plans, significantly improving outcomes for rare and aggressive forms of leukemia. This approach holds promise for personalized medicine, ensuring each child receives optimal, life-saving care.

Notable Labs is transforming cancer treatment with its innovative combination therapy approach. By leveraging its Predictive Precision Medicine Platform (PPMP), Notable identifies optimal drug combinations tailored to individual patients. This methodology aims to reduce the development of resistant cancer cells and allows each drug to be used at its best dosage without intolerable side effects. The platform has shown impressive predictive accuracy in clinical trials, particularly for pediatric leukemia. Notable's commitment to precision medicine offers new hope for patients with high unmet medical needs, driving better treatment outcomes and enhancing quality of life.

Notable Labs leverages predictive precision medicine to revolutionize cancer treatment, particularly pediatric leukemia. Their Predictive Medicine Platform (PMP) uses advanced technologies like flow cytometry and machine learning to analyze patient-specific cancer cell responses, offering highly accurate predictions for effective treatments. Clinical validation studies have shown a 97% precision in predicting responders. Collaborations with renowned institutions like Texas Children’s Hospital and UCSF further highlight Notable's commitment to improving pediatric oncology outcomes, making personalized, rapid, and effective cancer treatments a reality.

Precision medicine is revolutionizing pediatric leukemia treatment by offering tailored therapies based on individual genetic profiles. Notable Labs’ predictive precision medicine platform, driven by machine learning, is at the forefront of this transformation. With a 95% accuracy rate, this technology identifies the most effective treatments for rare and aggressive leukemia subtypes, such as pAML and JMML, ensuring better outcomes. This blog explores the advanced technological foundations, life-saving impact, and promising future of Notable Labs' approach in personalized cancer care for children.

Given the complexity of the biological mechanisms that underlie cancer, it is prudent to identify and target more than one Achilles’ heel to attack this multifactorial disease. This can be achieved by employing combination therapy, an approach to cancer treatment that combines multiple therapeutic agents to improve patient outcomes. Notable Labs is at the forefront of revolutionary advancements in combination therapy, one noteworthy example being its long-standing collaboration with Syros Pharmaceuticals. For Syros, Notable developed a highly efficient predictive biological test to screen patients for the clinical trial of a combination therapy against non-acute promyelocytic leukemia (APL) acute myeloid leukemia (AML). Notable Labs’ innovative approach to combination therapy holds immense promise for significantly improving cancer treatment outcomes despite current limitations in commercial pursuit. This blog dives into the science behind the promise of combination therapy, how Notable is contributing toward the evolution of this technology, and why the company’s Board of Directors is excellently positioned to advance this field in the future. Notable Labs' investigation into combination therapy against cancer is backed by decades of scientific research. Combination therapy holds immense promise in the fight against cancer because of its ability to additively or synergistically target two or more cancer pathways. Due to its multi-target approach, combination therapy is also an effective strategy to overcome anti-cancer drug resistance. By collaborating with Syros in 2017, Notable Labs contributed to the phase II clinical trial of a therapy combining the drugs tamibarotene and azacitidine against non-APL AML. For this multi-arm monotherapy and combination therapy trial, Notable developed a blood-based biomarker test to help predict patient response to treatment. Specifically, Notable was involved in conducting ex vivo experiments on peripheral blood mononuclear cells (PBMCs) isolated from the blood samples of non-APL AML patients. Harnessing the power of high-throughput flow cytometry, these experiments revealed that a 72-hour ex vivo exposure of PBMCs to tamibarotene upregulates various markers of myeloid cell differentiation, including the RARα pathway target CD38. Importantly, treatments that can promote myeloid cell differentiation are known to be effective against some subtypes of AML. Therefore, by testing a patient’s blood sample using Notable’s ex vivo approach, it is possible to predict whether the patient is likely to respond well to tamibarotene in monotherapy and combination therapy. Indeed, Notable’s ex vivo assay became one of the strategies Syros adopted when screening patients for enrolment in the phase II clinical trial of tamibarotene (alone as well as in combination with azacitidine). Market Challenges Currently, cancer treatment – and thus, the oncology market as a whole – is faced with a three-pronged challenge. There are millions of non-responders who are not benefiting from chemotherapeutic drugs, thousands of compelling drugs that need to be shelved because of perceived non-response, and hundreds of promising candidate drugs that are struggling to navigate the drug development pipeline. Notable Labs is committed to resolving these challenges through its successfully tested predictive precision medicine pipeline, a significant application of which was demonstrated during the Syros clinical trial.  Notable Labs’ commitment is also one of the reasons why it has decided not to commercialize the combination therapy technology immediately. Notable wants to focus on refining the technology further, expanding its applications, and ensuring that it integrates seamlessly into existing healthcare systems. The company aims to develop more sophisticated prediction models and address regulatory and logistical challenges. This approach will help make Notable's technology accessible to a broader patient population, maximizing the life-saving potential of predictive diagnostics as well as combination therapy. Broader Applications The high-throughput nature of Notable’s ex vivo flow-cytometry-based predictive precision medicine pipeline has helped identify many powerful drug combinations to treat other cancers such as juvenile myelomonocytic leukemia. This pipeline also has great potential for identifying effective drug combinations and guiding patient enrolment in clinical trials of combination therapies for other cancers, including solid tumors. In the case of solid tumors, the Notable pipeline’s blood samples for ex vivo testing could be replaced by biopsy samples, circulating tumor cells, pleural effusion, or ascites, among other sampling options. Founding Story Notable’s CEO, Dr. Thomas Bock (MD, MBA), has ensured that the company is well-positioned to transform combination therapy for cancer. For decades, our CEO has worked on multiple dimensions of oncology, including precision medicine, prevention of inherited cancers, and cancer vaccines. At Notable, Dr. Bock combines his medical and business training to accomplish the company’s mission of dramatically improving patient outcomes and the success, speed, and cost of developing new medicines. Strength of the Board of Directors Notable’s esteemed Board of Directors boasts of more than 150 years of combined professional experience in premier life science companies. Our board members excel in medicine (Dr. Bock), pharmacology (Mr. Tuomo Pätsi), business (Dr. Bock), finance (Mr. Michael Rice, Mr. Peter Feinberg, Mr. Thomas Graney), media communications (Ms. Michele Galen), and law (Mr. Thomas Dubin), fostering a vibrant and eclectic environment for Notable to thrive. This diverse team of bona fide giants from the pharmaceutical and biotechnology sectors is set to efficiently guide Notable Labs on its path to success in combination therapy and beyond. Conclusion Combination therapy for cancer, aided by predictive methods, could significantly improve patient outcomes while also broadening the population of patients who can benefit from a given drug combination. Leading the charge in this direction, Notable Labs’ predictive precision medicine pipeline boasts of massive potential to not only identify effective drug combinations for combination therapy, but also guide patient enrolment in clinical trials through predictive screening. Moving ahead, Notable’s technology is set to transform the treatment of multiple types of cancer by ensuring positive patient outcomes of personalized combination therapies in shorter times and at lower costs. Additional Data Points AML statistics (American Cancer Society): estimated 20,800 new diagnoses and 11, 220 deaths in the US in 2024. Success stories and collaborations in the field of combination therapy: Notable’s collaboration with Syros to develop a predictive test for patient response to monotherapy and combination therapy. Potential impact on cancer treatment outcomes: Combination therapy, aided by predictive methods, has the potential to enhance patient outcome while also broadening the population of patients who can benefit from a given combination of drugs. Future directions for research and development: AML treatment strategies must pivot towards personalized/precision medicine because of overwhelming heterogeneity of the disease. References Combination therapy in combating cancer . (2017). Oncotarget Notable’s collaboration with Syros . (2017). Notable Labs + Syros Differentiation therapy for myeloid malignancies: beyond cytotoxicity . (2021). Blood Cancer Journal New strategies to treat AML: novel insights into AML survival pathways and combination therapies . (2021). Leukemia Precision oncology using ex vivo technology: a step towards individualised cancer care? . (2022). Expert Reviews in Molecular Medicine Targeting RARA overexpression with tamibarotene, a potent and selective RARα agonist, is a novel approach in AML . (2023). Blood Advances Notable Labs’ JMML data . (2023). Notable Labs Key statistics for acute myeloid leukemia (AML) . (2024). American Cancer Society About the Author Dr. Anjaney Kothari is a scientific writer and researcher with a decade of experience in biomedical research. He completed his Ph.D. in Biomedical Engineering from Virginia Tech (USA) in 2019, developing and characterizing holistic in vitro and ex vivo models of hepatic and gastrointestinal toxicity. He has since been working as a freelance writer and researcher for companies operating in diverse niches, including biotechnology and biopharmaceuticals. Disclaimer The content of these blogs reflects the research and opinions of the individual authors and does not necessarily represent the views or positions of Notable Labs or its affiliates. The information provided is for educational and informational purposes only and should not be construed as medical, legal, or financial advice. Notable Labs makes no representations as to the accuracy, completeness, or validity of any information in these blogs and will not be liable for any errors, omissions, or any losses, injuries, or damages arising from their use. These blogs may reference third-party research, studies, or resources. Notable Labs does not endorse or assume responsibility for the content or practices of these third parties. Any reliance on the information provided is at the reader's own risk. 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