ICMR to conduct research in the area of 'Therapeutics for inherited rare diseases'

To promote discovery of newer therapeutic agents and approaches in the area of inherited rare diseases, the Indian Council of Medical Research (ICMR) has called for proposals on “Therapeutics for inherited rare diseases” from scientists working in medical and research institutes. Full project submission will end on January 25, 2023 and webinars will be held on December 20, 2022 to discuss queries. Inherited rare diseases are infrequently occurring disorders and have been defined by countries in terms of prevalence in their own population. WHO defines rare diseases as often debilitating lifelong disorders with a prevalence of 1 or less, per 1,000 population.

Only 5% of inherited rare disorders have treatment among which most of the treatments are highly priced and often not available worldwide. Availability and access to medicines are important to reduce morbidity and mortality associated with rare diseases. “There is a need for research and development in intervention, technology transfer and indigenously manufactured therapeutics for rare diseases which could be achieved by collaborative efforts,” according to an ICMR official. The project proposal should be submitted for financial support through online mode only. The researcher should have relevant experience in the field as evident from publications of the past three years.

The proposal must have at least one Co-Principal Investigator (PI) from the host institute. Research Groups working or having expertise in discovery and development of drugs/therapies are encouraged to apply. Collaborations are encouraged. An outstanding project requiring funding more than Rs. 1.5 crore per study, maybe converted to a Task force by ICMR, wherein the proposed Principal Investigator (PI) will be the PI (single centric study) or will be one of the PIs (multicentre study). The project is also aimed at supporting research and development towards developing intervention strategies on the following aspects of inherited rare diseases related to development of therapeutics like drugs repurposing, biologics, genetic error corrective measures, management to improve quality of life of patients with rare diseases such as orthopedic /surgical approaches, innovative management protocols etc.

Following groups which will be considered under inherited rare diseases are storage disorders, small molecule inborn errors of metabolism, primary immunodeficiency disorders (PID), neuromuscular disorders (NMD), haematological disorders (excluding Sickle cell diseases and Thalassemia) and skeletal dysplasia. Approaches may include development of resources such as cellular and animal models, vector for genetic error corrective measures, and mechanistic aspects that are required for drug development as a part of the project. These projects are time-bound with a goal-oriented approach and clearly defined targets, specific time frames.

The proposed research and development should be a potential therapeutic molecule (novel/repurpose drug) or strategies to improve quality of life of patients with rare diseases. Proposals in multidisciplinary collaborative mode will be encouraged. This call excludes proposals focused on descriptive studies, other systems of medicines, rare cancers, Early stage of development i.e., in-silico and in-vitro only, biomarker, registry/patient database. The proposal should cover achievable targets, specific time frames, justifiable budget requirements and engagement with prospective technology transfer partners or technology transfer facilitating bodies/technology business incubators. Investigators should mention if they are likely to have intellectual property from their study.

“Proposal should be focused on outcomes that can be translatable. The host institutions should be well-equipped and have the essential equipment and infrastructure for the proposed development. Only minor equipment may be supported based on the requirements of the proposed objectives. The projects should be time-bound normally for a duration of 2 to 3 years. Evaluation will be based on the significance of the proposal, innovation/novelty, project description, research team’s profile, and infrastructure,” according to ICMR.