Govt to fund gene therapy research on NCDs, rare diseases – CNBCTV18

The central government has decided to support research on gene therapy in a big way to tackle Indias escalating burden of non-communicable diseases (NCDs) and inherited critical disorders such as spinal muscular atrophy (SMA), thalassemia and haemophilia. Scientific institutions and universities conducting research on genomics and its therapeutic applications will soon get a financial fillip from public coffers, under an initiative facilitated and funded by the Indian Council of Medical Research (ICMR).

Gene therapy is a technique that uses genetic modifications to treat or prevent diseases. The experimental technique allows clinicians to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery or replacing a mutated gene that causes diseases with a healthy copy of it.

According to the proposal note, a copy which CNBCTV18.com has reviewed, the funds will be disbursed for research projects across various therapeutic areas including gene editing, gene transfer techniques and immunotherapy based on genetic modifications. The apex body for biomedical research promotion has already started accepting proposals from scientists and clinicians.

Why gene therapy is crucial

In the current era of genomics and genetic testing, it is evident that a large swath of the Indian population harbours causative mutations leading to various disorders. Conversely, for many inherited and complex disorders, the underlying genetic causes are known.

And most of these illnesses are currently not treatable by molecule drugs or traditional therapies. According to patient advocacy groups, though there are major developments in gene therapy for cancer and other chronic diseases in the developed world, most of them are not accessible or affordable to a majority of the Indian population.

For instance, the US Food and Drug Administration (FDA) recently approved Zolgensma, the first gene therapy to treat children less than two years of age with SMA, a leading genetic cause of infant mortality. Though the drugs clinical trial in older SMA-affected children remains partially halted because of an inflammatory response observed in an animal study, there are very few options left for these patients.

Majority of these rare diseases, including SMA, are progressive and require life-long treatment and continuous support, says Archana Panda, co-founder of Cure SMA India, a non-profit group that works for making SMA treatment accessible to Indians.

In the case of SMA, a disease that leads to loss of motor neurons and early death, Nusinersen is the only drug approved by the US FDA. Developed by US-biotech major Biogen and marketed as Spinraza, the drug is administered directly to the central nervous system intravenously and the therapy could cost around $750,000 in the first year.

But in recent years, significant strides have been made in the field of gene therapy. This has culminated in the recent FDA approvals for Luxturna (gene therapy for retinitis pigmentosa) and Yescarta (CAR-T cell therapy for lymphoma). Many such approaches are currently under investigation or in early clinical trials. Now, the ICMR-backed initiative aims to fill the gaps in research thrust in India by providing emphasis on the focus areas which require more attention to address the needs for the large existing patient base, the note reads.

Hope on the horizon for patients

Apart from multi-factorial diseases such as cancer, diabetes and lung ailments, the research will also focus on treatment for rare diseases such as neuro-muscular diseases (including Duchenne muscular dystrophy, Becker muscular dystrophy, SMA and myopathies), retinal or corneal disorders (including Retinitis Pigmentosa, Stargardt disease and Fuchs dystrophy), inherited heart diseases and blood disorders including Thalassemia, Sickle Cell Disease and haemophilia.

The initiative assumes significance as it is estimated that one in 20 Indians is affected by one of the 7,000 diseases listed by the World Health Organisation as rare diseases. For many of these illnesses, gene therapy is the final hope. About 16 lakh Indians are diagnosed with cancer every year, and 8 lakh people lose their life to the disease.

While the fund support for research which can lead to human trials is only a baby step, experts such as Dr Mamta Muranjan, who heads the Genetic Clinic at KEM Hospital in Mumbai, emphasise the need for such focused long-term policies. Most patients with rare diseases remain undiagnosed for a long time. Even if the diagnosis is on time, the family of the patient is usually unable to afford the treatment cost. Insurance policies generally do not cover these life-long treatment expenses, she says.

Possibility of translation into human trials

According to official sources, the three-year state-funded research projects would be selected on the basis of their potential for development of functional treatment options. The strategies proposed should have the possibility of translation into future human trials.

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Govt to fund gene therapy research on NCDs, rare diseases - CNBCTV18