Nagpur: Incurable Duchene Muscular Dystrophy (DMD), a rare genetic disease, takes a toll on children in India. As there is no cure or treatment available so far in India for DMD, children (boys) suffering from the disease do not live beyond 18-20 years of age due to progressive degeneration and weak limb muscles 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 Calcutta and Bengaluru are nearing completion of the basic work needed to develop 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 might have 8-10 lakh patients of various types of muscular dystrophy.
Professor Surajit Sinha from the Indian Association for the Cultivation of Science (IACS), Dr Apurba Ghosh, from the 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 DMD drugs.
In fact, DART with the support of Professor Sinha has already successfully conducted a single patient trial of an RNA (Antisense Oligonucleotide or AON) molecule with the permission of the Indian Council for Medical Research (ICMR ) from Bengaluru and has already requested clinical trials for the same from 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 the drugs the three scientists are working on with the hope 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, 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 lakh children across India with different forms of muscular dystrophy (DMD being one of them) are living a life of abject desperation and watching the death. Therefore, various groups of parents and individual parents demand a policy on the disease and its inclusion in “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 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 orthoses, 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 medical professionals, patient advocacy groups, pharmaceutical companies and other key stakeholders in addition to raising awareness of medicine and evolving protocols. care with an emphasis on 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 chromosome ” X ”, 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, Calcutta – Professor Surajit Sinha, School of Applied and Interdisciplinary Sciences, developed the basic ingredient, the “Morphilonos” molecules needed to make the drug based on DNA 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 the number US 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 want the government to fund more manufacturing of the drug it developed. He says he did not borrow or copy the 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 medicine will be similar to Exondys-51 but that it is a generic version. He appealed for initial financial support of Rs 5-6 crore required for a 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 ArunShastrya already developed an RNA-based antisense oligonucleotide (AON) about six months ago, which was tested on a KaranveerAnand patient 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 DM, “I didn’t create a new molecule. But I trained in Australia at Murdoch University with the guidance of Steve Wilton, who is a pioneer of the “exon jump” technique. Shastry also admitted that he was working with Professor Sinha in Kolkata for the DNA based mat because it is more efficient.
3.The third research of the Kolkata Institute for Personalized Medicine:
Saubhik Sengupta, director of the PMIS laboratory said that in Kolkata, 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 will be 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 that it will have huge ramifications in the treatment of incurable diseases like girdle muscular dystrophy, spinocerebellar ataxia, huntingtons chorea, 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