Showing 3 ideas for tag "hemophilia"

Goal 3: Advance Translational Research

Genome Editing and Gene Therapy

There is a critical need for the establishment of strategies that will determine the efficacy, safety, and toxicity of genome editing techniques specifically in hematologic diseases.

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

Inherited monogenic hematologic diseases such as hemophilia, beta-thalassemia and sickle cell disease are prime targets for future application of genome editing technology. However, studies are still needed to advance our understanding of the biology of genome editing as well as determine which other disorders are amenable to genome editing correction. Emphasis on preclinical research that focuses on determining the accuracy, safety and efficiency of this technology in order to help minimize off-target mutations and reduce toxicity, is essential for effective translation of this technology into the clinic. Once preclinical efficacy is established, support will be needed for clinical vector production, toxicity testing of the vectors/reagents used, and the performance of clinical trials. The gene correction strategies developed for inherited disorders will also be attractive for other hematologic diseases, and autoimmune disorders like lupus, rheumatoid arthritis, and type I diabetes). There is also a critical need for supporting preclinical validation studies, scale-up and GMP cell manufacturing, all of which could be shared infrastructures across multiple diseases in the NHLBI portfolio.

Name of idea submitter and other team members who worked on this idea Alice Kuaban on behalf of the American Society of Hematology (ASH)

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Goal 3: Advance Translational Research

Novel Mechanism for Clinical Trials of New Pro-Hemostatic Agents in Hemophilia

There are new exciting novel pro-hemostatic therapeutics in early phase clinical trials for hemophilia and hemophilia inhibitor patients. Yet, it is difficult to design randomized trials to compare these agents, or compare them with standard treatment, given the small sample size and competing studies for such patients. It is critical to develop novel approaches to compare new agents in rare populations. For example,... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

Should rare disease-specific strategies for clinical trials be developed, it would revolutionize the approach to study of not only rare disease populations, but all disease groups, no matter their size. If validated, rare disease-specific clinical trial strategies, would potentially reduce the cost, time, patient burden, and research effort to conduct clinical trials. If validated, consideration could be given to drug licensure earlier in the trial process, with a requirement for all such trials to initiate and continue ongoing data collection post-licensure for safety and efficacy.

Feasibility and challenges of addressing this CQ or CC

Novel statistical methodologies are greatly needed to help with rare disease research. NHLBI might consider a grant mechanism RFA to encourage development of novel clinical trial strategies utilizing smaller sample size. The proof would be to develop the methodology as part of a feasibility study, and then, if feasible, adapt the novel approach to development and conduct of a clinical trial in a rare disease clinical tria,l to test the concept.

Name of idea submitter and other team members who worked on this idea Margaret V. Ragni, MD, MPH (aspects discussed with Don Brambilla PhD.

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9 down votes
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Goal 3: Advance Translational Research

Immune-Mediated hematologic disorders

What is the optimal approach to prevent and treat immune mediated hematologic disorders (autoimmune hemolytic anemia, immune thrombocytopenic purpura, etc) and complications of hematologic disease (inhibitors in hemophilia, transfusion-related alloimmunization, etc)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

Therapies for these disorders are suboptimal and current treatments are associated with significant side effects. Transfusion is limited by development of alloantibodies..

Feasibility and challenges of addressing this CQ or CC

NHLBI should support clinical trials in this area. Improved understanding of the biology and biomarkers predictive of disease development would aid in defining therapeutic approaches and trials.

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18 down votes
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