Incurable DMD disease takes a toll on children in India

Nagpur: The incurable Duchene muscular dystrophy (DMD), a rare genetic disease, takes a heavy toll on children in India. As there is as yet no cure or treatment available in India for DMD, children (boys) with the disease do not live beyond the age of 18 to 20 due to progressive degeneration and severe disease. weakness of the muscles of the limbs ultimately affecting the heart and lungs, resulting in death. But now there is a silver lining to fighting the disease. Scientists from three different institutes in Kolkata and Bengaluru are about to complete the basic work required for the development of a drug against the disease.

In India, 54 babies with DMD are born every day. One in 3,500 people is affected by DMD according to the WHO report. India may have 8-10 lakh patients with various types of muscular dystrophy.

Professor Surajit Sinha from the Indian Association for the Cultivation of Science (IACS), Dr Apurba Ghosh, Institute of Child Health (ICH) in Kolkata and Arun Shastry from the Dystrophy Annihilation Research Trust (DART) in Bengaluru are at different stages of the development process of three different types of drugs for DMD.

In fact, DART, with the support of Professor Sinha, has already successfully conducted a single patient trial of an RNA (Antisense Oligonucleotide AON) molecule with permission from the Indian Council for Medical Research (ICMR ) in Bengaluru and has already requested clinical trials for the same to the Drug Controller General of India (DCGI). But it will take another 10 to 12 years before these drugs are available on the market. Yet the parents of almost all children want their children to participate in future clinical trials in India of drugs the three scientists are working on in the hopes that the drugs could help treat the disease for at least those who are small like. well those who might be born with this disease in the future

Although in the United States a drug – ‘Exondy51’ – was released in September 2016, but it is well beyond the reach of Indian patients as the drug costs Rs 2-3 crore per year with one injection per week for 52 weeks.

With no cure or treatment, parents of over 10 lakhs of children in India suffering from different forms of muscular dystrophy (DMD being one of them) live a life of abject despair and watch death. . This is why various groups of parents and individual parents are calling for a policy on the disease and its inclusion in the “Ayushman Bharat”.

Parents across the country want the government to prepare a patient directory to realize the high incidence of the disease as 20% of the total muscular dystrophy patients are in India. Another request is to include rare diseases like this in insurance plans. Parents also want physiotherapy and other necessary supports in the form of free medicinal supplements for patients whose parents cannot afford the expensive steroids that are currently used for disease management, in addition to reducing blood pressure. cost of assistive devices such as wheelchairs, transfer boards, hoists, stirrups, AFO, leg / knee braces, etc.

Parents expect the government to work on effective treatment for all forms of DM including BMD, DMD, FSHD, all types of LGMD, etc. They require DMD care centers with health and healthcare professionals, patient advocacy groups, pharmaceutical companies and other key stakeholders in addition to raising awareness about DM and evolving care protocols. emphasizing the availability of relevant literature and videos in local languages.

Since the disease is genetic and is due to different types of effects such as mutations and deletions on any exon from 1 to 79 on the gene forming dystrophin, the largest gene in the human body, on the “X” chromosome, one drug cannot be used to treat all defects. Medicines have to be tailor-made for each type of muscular dystrophy and hence the huge cost involved in drug development.

The three searches –

1. Indian Association for the Cultivation of Science (IACS), a reputable university, Kolkata – Prof. Surajit Sinha, School of Applied and Interdisciplinary Sciences developed the basic ingredient, the “Morphilonos” molecules necessary for the manufacture of DNA-based medicine for the treatment of muscular dystrophy. DNA drug is better and more effective than RNA drug.

Sinha already has a US patent (Morphilino antisense agents) with US number 9,914,745B2. He also worked with Arun Shastry, DART scientific lead for the RNA drug already tried on a patient. Almost 90-95% of parents of MD children are aware of his work and want their children to participate in clinical trials for his drug. They and want the government to finance more the manufacture of the drug developed by him. He says he hasn’t borrowed or copied Morphilonos’ manufacturing technology but is the result of 13 years of research. “At the start, my target was not DMD. I was working on drug development for genetic diseases in general, including cancer, ”he said.

Professor Sinha has written to Prime Minister Narendra Modi to inform him that his drug will be similar to Exondys-51 but is a generic version. He appealed for an initial financial support of Rs 5-6 crore required for the preclinical study with a hospital and a pharmaceutical company with good laboratory practices. This will be followed by large scale trials and treatments. The government asked him to submit his proposal.

2. Second research by DART – Dystrophy Annihilation Research Trust Bengaluru
DART chief scientist ArunShastry already developed an antisense RNA-based oligonucleotide (AON) about six months ago that was tried on a KaranveerAn patient and with positive results in the past six months. Shastry said this research was funded by Ravdeep Singh Anand, the patient’s father as well as 20 other families of patients with some form of MD, “I didn’t create a new molecule. But I trained in Australia at Murdoch University with the guidance of Steve Wilton, who is the pioneer of the “exon jump” technique. Shastry also admitted that he was working with Professor Sinha in Kolkata for the DNA mat as it is more efficient.

3.The third Personalized Medicine Institute Kolkata research:

Saubhik Sengupta, director of the PMIS laboratory said that in Calcutta, his laboratory was starting the first clinical trial for DMD in India. Antisense oligonucleotides (AON) will be designed after careful analysis of each individual DMD gene and designing unique AONs for each child. The study aims to reduce the cost to around 4 lakhs per year.

Dr Apurbo Ghosh (Principal Investigator) of the project will lead the trial at the Institute of Child Health, Kolkata with Dr Manjari Mitra. DrSEngupta will be the co-principal investigator and is responsible for the genetic analysis and design of AON for each individual.

The study is funded by Muscular Dystrophy Group, an NGO made up of parents whose children have DMD.

Sengupta claimed that AON therapy is being tried in India for the first time and will have huge ramifications in treating incurable diseases like girdle muscular dystrophy, spinous muscular atrophy, spinocerebellar ataxia, chorea. huntingtons, several cancers, etc.

Types of DMD

There are a total of 9 types of muscular dystrophy

1 Duchenne muscular dystrophy

2. Becker’s muscular dystrophy

3. Myotonic muscular dystrophy

4. Girdle muscular dystrophy

5. Facioscapulohumeral muscular dystrophy

6. Congenital muscular dystrophy

7. Oculopharyngeal muscular dystrophy

8. Distal muscular dystrophy.

9. Spinal muscular dystrophy

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