July 25, 2019
In our July campaign #RareDiseases, we focus on the Dystrophy
Annihilation Research Trust (DART), that has been working to find a cure for
Duchenne Muscular Dystrophy, a rare genetic disease that affects over five
lakh children in the country. Read the inspiring story of a father behind
India’s first research lab working to annihilate the disease and change the
lives of countless kids.
What do you do when your only child, age seven, is diagnosed with a rare
degenerative disease like Duchenne Muscular Dystrophy (DMD)
that has no cure?
It lead Ravdeep Singh Anand started a research lab,
India’s first, to study DMD in the hopes of finding a cure.
Anand, who has no medical background, is the
Founder-President of Dystrophy Annihilation Research
Trust in Bengaluru, which was in the news recently for coming up
with a genetic intervention procedure for people with DMD.
That procedure is set for clinical trials, a moment that the Anands’ and
countless other families battling this rare condition have been waiting for.
A neurodegenerative disease, DMD occurs only in boys. It is a genetic
condition where the protein dystrophin, needed for muscles to
function properly is present at much lower levels than what is needed. As the
child grows older, the muscles continue to degenerate. This means the child
will have to use a wheelchair for a large part, before being bedridden. There
is no cure for DMD, it can only be managed with steroids and physiotherapy.
Movin and Ravdeep Anand were fortunate as their son
Karan was diagnosed fairly early, but as Ravdeep points out,
the battle had just started.
Diagnosis was one thing. We got him diagnosed pretty early, when he was
two-and-a-half. From there on, once you know what the problem is, then
what? The ‘then what’ question had to answered. How much ever we were
looking around in India and abroad we were not finding a solution or a
path. And as the years went by, that was becoming quite frustrating and as
Karan was growing older, his problems were increasing. We were getting
quite desperate to get a handle on things. – Ravdeep Singh Anand,
Founder-President ,Dystrophy Annihilation Research Trust
India has over five lakh children with DMD, among the highest
numbers in the world but there’s no treatment available at an affordable
cost. Nor was there any research taking place in the country. All the
research was happening in the West, a status quo Ravdeep refused to accept.
“I started figuring out that we have one of the highest numbers of
children, but no one was looking at India. There were so many trials going on
in the United States and Europe but not here. I don’t have a science
background, but I said let me use my experience as a project manager to get
the ABCD together, all the factors together”, he recalls.
His search led him, among others, to Dr Arun Shastry,
Chief Scientific Officer at the DART Lab.
“What we are trying to do is a kind of genetic intervention for DMD”,
says Dr Arun, who has been a part of DART since it was founded in 2012.
“The challenge is that, being genetic, no two children are the same. The
treatment for every child is unique and needs personalised medicine. What we
have done in DART is create new molecules that will help the children
affected”.
Key to DART’s success has been the collaboration with experts from
countries like Australia and Netherlands as well in India, mainly Kolkata.
“When we look at the cost factor, the procedure we have developed is
available in other countries is ₹2 crore a year. Ours will cost a fraction
of that”, says Dr Arun. The procedure will stabilise the condition of the
child by stopping the progression of the disease. “This means that is the
child with DMD is walking, the treatment we have developed will ensure he
will not have to use a wheelchair”.
The aim is to arrest the degeneration, says Ravdeep. “Once we bring a
status quo to the boy, we can focus on regeneration and repair”. He is
confident that with the new procedure developed, the end point is close at
hand. “We are much closer to the final destination. The final chapter is
yet to be written and the good thing is that the whole world is working on
it”.
It is this never say die spirit and optimism that has kept the Anands’
going through some hard times. “There was a time when I needed ₹3 crore
to import a vital piece of equipment and had no idea how I would manage”,
recalls Ravdeep. Many affected families he reached out to help were
surprisingly wary, which was disheartening. But Ravdeep was relentless in his
mission to live up to the A in DART and annihilate muscular dystrophy, and
find the light ahead for his son and other children.
Apart from the R&D lab, DART supports affected children and their
families in many ways by providing information, awareness campaigns,
counselling and rehabilitation support.
To Know More about DART, visit their website.
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