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Notable Labs: Revolutionizing Pediatric Leukemia Treatment

Syeda Abeer Iqbal • July 29, 2024

Notable Labs, Inc. is a promising therapeutic company that uses its unique Predictive Precision Medicines Platform (PPMP) to find the most effective treatment for cancer on a patient-by-patient basis. Living up to its name, Notable has made significant advances in the treatment of pediatric leukemia by reducing the time and cost required to find the right treatment for the patients and increasing the predictive precision of treatment by 97%, as proven by various validation trials.


Overview of Pediatric Leukemia


Pediatric leukemia is a type of cancer that affects the bone marrow and blood cells in children. It is the most common childhood cancer, with a prevalence of 7.4 per 100,000, accounting for 25.1% of all cancer cases in individuals below the age of 20.


Depending on the types of blood cells that become malignant and how acute (fast-growing) the tumor is, leukemia can take various forms. In pediatrics, however, two forms are more common: Acute Lymphoblastic leukemia (ALL) and Acute Myeloid Leukemia (AML).


ALL is the most common type of pediatric leukemia and typically affects children between ages 2-5. It is caused by abnormal production of lymphocytes (a type of white blood cell). AML affects children from age 2 through the teenage years and is caused by overproduction of red blood cells, platelets, or myeloblasts (another type of white blood cell).


AML has resulted in 20,800 cases and 11,220 deaths in the US this year so far.


As with all cancers, early diagnosis and proper treatment are necessary to save the lives of leukemia patients. Despite its prevalence and high mortality rate, treatment options for patients are often limited or unsuccessful, with the response rate (RR) of available treatments for AML standing at approximately 15%.


Fortunately, Notable Labs, through its groundbreaking research and diagnostic techniques, is set to change the landscape of pediatric leukemia treatment.


Technical Background


Notable Labs aims to revolutionize the diagnosis and treatment of cancer patients by advancing personalized medicine. By combining high-throughput screening and machine learning, they bio-simulate cancer treatment and predict whether a patient will respond to specific treatments. This makes their therapeutic approach data-driven and patient-centric, treating each case as unique and notable.


The world is no stranger to the groundbreaking work and achievements of Notable Labs. They presented their findings related to their predictive medicine platform in oncology treatment, especially Leukemia, at the 2023 Annual Meeting of the American Association for Cancer Research (AACR) held in Orlando, Florida.


"Together with our collaborators at Washington University, today we are reporting on the fourth successful validation study of our Predictive Precision Medicine Platform (PPMP)," said Thomas Bock, M.D., Chief Executive Officer of Notable.

Notable Labs' commitment to precision medicine sets them apart in the field.


Notable's Approach


PPMP is a high-throughput automated platform designed to generate a massive data repository related to leukemia and other cancers. It has analyzed 190 billion lines of data across thousands of patient samples and therapeutic compounds, powering the identification of the most promising therapeutic assets and patient-response predictions with high precision.


By testing a patient's leukemia cells against a panel of FDA-approved drugs or treatments, Notable Labs identifies the most effective treatment options.


The primary goals of PPMP are to identify the most promising therapeutic option and predict patients' responses to available treatments with great accuracy. This not only saves time and therapeutic costs but also spares patients from receiving treatments that will not work for them based on their genetic markers and disease profiles.


This personalized approach ensures that each patient receives tailored therapy, as Notable Labs believes that each patient is notable.


Clinical Outcomes


Notable Labs has conducted several clinical validation studies to assess the accuracy and use of PPMP prediction, especially in hematological malignancies. They have focused on two main drugs:


  1. Volasertib (a potent PLK-1 inhibitor), which has been approved for therapies in several cancers, including acute myeloid leukemia (AML) patients who are ineligible for aggressive front-line treatments and bone marrow transplants.
  2. Fosciclopirox, a patented, parenterally administered prodrug of a commercially available topical antifungal agent, ciclopirox, which is being developed for the treatment of bladder cancer and AML.


Notable Labs aims to predict the responsiveness of these drugs using their PPMP prior to actual treatment in leukemia patients.

In one study, PPMP achieved 100% accuracy in predicting clinical responders to a combination of Venetoclax plus Decitabine (VenDec) for acute myeloid leukemia (AML).


Successful clinical data from PPMP from a Phase 2 Fosciclopirox study have also been reported. With 100% accurate prediction of Fosciclopirox’s clinical trial outcome using their proprietary platform, PPMP, Notable Labs was able to streamline the volasertib program by eliminating an entire patient cohort.


"This further validated our PPMP-guided development strategy to target responding patients, improve patient outcomes, and reduce the risk, time, and cost of drug development," said Thomas Bock, M.D., Chief Executive Officer of Notable Labs.


Notable Labs has made significant progress in the Volasertib Phase 2 program. They have planned to carry out a Phase 2 trial with volasertib in combination with decitabine for patients with relapsed/refractory (R/R) AML. By selecting volasertib-responsive patients prior to treatment, they aim to fast-track the development and commercialization of the drug as a potential therapy for leukemia.


Also, due to its robust clinical outcome in Boehringer Ingelheim's (BI) expansive Phases 1-3 and PPMP data that distinguishes predicted volasertib-responders from predicted non-responders based on samples from AML patients, Notable has in-licensed volasertib.

Based on these advancements, the Phase 2 trial for relapsed/refractory AML is expected to initiate in Q2 2024, and the first complete data is anticipated in Q4 2024, before enrolling PPMP-selected patients.


Drug Development


Notable Labs' fight against cancer does not stop at diagnostics; they are robustly involved in the development of novel and more effective drugs. By identifying potential drug combinations and repurposing existing medications, such as Volasertib and Fosciclopirox, they have accelerated the advancement of effective treatments for leukemia.


To further support drug development, efforts are being made to license undervalued advanced-stage drugs that have been shelved for various reasons. Their model propels advanced-stage drugs over finish lines, bypassing years of risk and millions of dollars in investments.

These efforts demonstrate Notable Labs' commitment to revolutionizing pediatric leukemia treatment by identifying personalized and effective therapies.


Future Potential


The future looks promising for Notable Labs. As they expand their research portfolio, they aim to address other cancers and rare diseases. Their commitment to advancing precision medicine and improving patient outcomes inspires hope for a brighter future.

They are committed to optimizing dosing strategies to make them as effective for patients as possible. Notable Labs aims to de-risk precision medicines by developing them selectively in patients predicted to respond.


In conclusion, Notable Labs’ dedication to pediatric leukemia research and treatment is commendable. By combining technical expertise, clinical insights, and a patient-centered approach, they are saving lives and transforming the field of oncology.


Citations


  1. What we do (notablelabs.com)
  2. https://www.lls.org/facts-and-statistics/childhood-and-adolescent-blood-cancer-facts-and-statistics
  3. Childhood Leukemia | MedlinePlus
  4. Notable Advances Volasertib Phase 2 Program Utilizing Proprietary Predictive Precision Medicine Platform-Guided Enrollment (yahoo.com)
  5. Notable Labs Presents Clinical Platform Validation Data at the 2023 American Association for Cancer Research Annual Meeting (yahoo.com)
  6. VOLASERTIB & FOSCICLOPIROX (notablelabs.com)


About the Author


Syeda Abeer Iqbal holds a Master's degree in Microbiology and Molecular Genetics and has extensive experience in scientific writing and research. Passionate about writing, she has contributed to various scientific and medical blogs over the past 10 years, completing numerous writing and research tasks. Syeda has an international publication of her MS work on identifying and characterizing a specific gene in bacteria. Read the publication here.


Based in Virginia, USA, Syeda worked as a research consultant for GATC Health from 2021 to 2024. She is currently seeking similar opportunities and is dedicated to giving her 100% to any related task.


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.


For biotechnology and pharmaceutical content, note that ongoing research and clinical trials may change the context and results discussed. Always refer to the latest research and guidelines from reputable sources.


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