Policies and guidelines related to healthcare

With an aim to ensure better treatment options for ‘rare diseases’ or inherited genetic diseases the Indian Council of Medical Research (ICMR) has come up with the national guidelines for gene therapy product development and clinical trials. The idea is to promote gene therapies in India and ensure clinical trials for gene therapies can be performed in an ethical, scientific, and safe manner.

What is the Importance of these guidelines?

  • These guidelines will provide the general principles for developing gene therapy products for any human ailment along with the framework for human clinical trials.
  • The guidelines will also serve as an important resource and roadmap for those in the field trying to develop gene and cell therapies.
  • Because of the huge burden of genetic diseases in India there was a need to accelerate the development of advanced therapeutic options.
  • Though the affected population is small, such diseases are often life-threatening, many of whom are in the pediatric age group.
  • For a variety of debilitating diseases caused by genetic mutations, gene therapy offers a treatment modality that cannot be provided by conventional therapy including small molecule drugs or alternative medicines.

Overview of the Guideline

  • The guide includes scientific and ethical considerations that are allowed in gene therapy. It explains the responsibilities of investigators, sponsors, institutions.
  • It also lists considerations like quality assurance, manufacturing, and control, etc. The guidelines give a clear overview of the rules to be followed during clinical trials.
  • The guide explains the principles to hold while signing international collaboration and procurement of Genetic Therapeutic Products. The Genetic Therapeutic Products are entities that deliver nucleic acid by various means for therapeutic benefit to patients.

What are rare diseases?

A rare disease also referred to as an orphan disease, is any disease that affects a small percentage of the population.

Most rare diseases are genetic, and are present throughout a person’s entire life, even if symptoms do not immediately appear. Rare diseases are characterized by a wide diversity of symptoms and signs that vary not only from disease to disease but also from patient to patient suffering from the same disease.

The most common rare diseases in India are Haemophilia, Thalassemia, sickle-cell anemia and primary immunodeficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.

What is Gene therapy?

Gene therapy is a technique which is used in the replacement of defective genes with healthy ones in order to treat genetic disorders. It is an artificial method that introduces DNA into the cells of the human body.

Types of Gene Therapy

There are two types of gene therapy-

Somatic Gene Therapy

This type usually occurs in the somatic cells of the human body. In this method, therapeutic genes are transferred into the somatic cells or the stem cells of the human body. This technique is considered as the best and safest method of gene therapy.

Germline Gene Therapy

It occurs in the germline cells of the human body. Generally, this method is adopted to treat the genetic, disease causing-variations of genes that are transferred from the parents to their children. The process involves injecting a healthy DNA into the cells responsible for producing reproductive cells, eggs, or sperms.