ICMR releases rules to pave way for novel gene therapy

Nine months after the national drug regulator CDSCO recognised “gene therapy” as a new drug, Indian Council of Medical Research has come out with the first guideline for the industry and research establishment on how to make such products on the basis of sound ethical, scientific and regulatory norms.

The guidelines not only outlines the ethical and scientific requirements for gene therapy trials but also asks developers to keep the patient’s safety, efficacy and clinical rigour in mind while developing such therapies.

Two other types of therapies involve knocking out a bad gene that triggers disease and introducing a new gene to fight disease. The first successful gene therapy clinical trial was conducted in 1989 in the USA, for severe combined immunodeficiency.

“In India, there is no gene therapy trials yet. Along with guideline, the ICMR has also sought proposals from researchers for funding opportunities for gene therapy projects targeting human disease,” Geeta Jotwani, programme coordinator for gene therapy and gene editing at the ICMR told DH.

The apex medical research council looks at gene therapy as a treatment option not only for inherited diseases and rare diseases but also for multi-factorial ailments like cancer, diabetes, lung diseases and neurological diseases. Several Indian pharmaceutical companies have shown interest in a particular gene-therapy for cancer known as Car-T cell therapy, in which one particular immune cell of a patient (T-cell) is tweaked using gene therapy in such a manner so that they attack and kill cancer cells.

“While gene therapy is used to look for treatments of rare diseases, the clinical trial procedure for such therapies should be different. The US Food and Drug Administration has a fast track process for such therapies. They are unconventional products for which the protocol should be slightly different,” commented Sridhar Sivasubbu, a scientist at the Institute of Genomics and Integrative Biology, Delhi, who works on rare diseases but wasn’t involved in the making of the guideline.

While products for common diseases like cancer and diabetes may go through the standard three-phase clinical trial, the CDSCO may opt for a different protocol for rare diseases. In the notification, the regulator has defined a rare disease drug as “a medicine intended to treat a condition which affects not more than five lakh persons in India”.

In the past three years, several gene therapy products received approval for patient use from US and EU regulators, paving the way for the development of therapies for previously untreatable disorders. Worldwide, the market for such treatments is predicted to increase at a compound annual growth rate of 11.3% from 2018 to 2024.