From lab to trials: Delhi University's stride towards a Parkinson's cure

New Delhi, Nov 12, 2023, IANS

Parkinson's disease (PD) is a serious illness, affecting more than 10 million people worldwide. Tremors, excessive shaking of the entire body or limbs, stiffness, slowed walking, and difficulty in movement are its primary manifestations.

The disease also becomes a cause for severe depression among patients. Currently, there is no cure for PD available in the market. 

However, India has now discovered a potential treatment for this incurable disease, and its clinical trials have begun in the US.

Delhi University has identified a specific protein that could lead to the treatment of this disease. 

The clinical trials for the molecule (ATM 399A) developed by Professor Diwan S. Rawat of Delhi University and Professor Kim at the McLean Hospital in the US have commenced.

PD is a neuro-degenerative disorder, with common symptoms such as tremors, stiffness, slow movements, and difficulty in walking, leading to noticeable behavioural issues like depression and anxiety.

The primary cause of this disease is the death of neurons in the substantia nigra, located in the middle part of the brain. This results in a deficiency of dopamine and some proteins are essential for the existence of dopamine neurons.

In the pursuit of finding a cure for this serious ailment, researchers under the leadership of Rawat have initiated clinical trials for a synthesised molecule (ATH-399A). 

The research collaboration involves assistance from McLean Hospital in Boston, the US.

Rawat said: "It has been a long journey. Creating something like this requires a tremendous amount of energy. Sometimes you lose patience because one has to overcome numerous obstacles, from obtaining research funding to filing patents and signing technology transfer agreements. However, we remained focused, and now our molecule has reached a level where we can hope for some benefits to humanity."

In 2021, Delhi University and McLean Hospital reached an agreement, with NurrOn Pharmaceuticals joining as a partner to develop this molecule. 

Later, HanAll BioPharma and Daewoong Pharmaceutical collaborated with NurrOn Pharmaceuticals, initiating the administration of the first dose to a human participant in Phase 1 of clinical trials.

The Phase 1 study involves healthy participants aged 18 to 80, receiving oral doses of ATH-399A to assess safety, tolerability, pharmacokinetics, and food effects.

The study comprises both single ascending dose (SAD) and multiple ascending dose (MAD) groups. 

Initial results for Phase 1 clinical trials of ATH-399A are expected in the second half of 2024. 

The Michael J. Fox Foundation (MJFF) for Parkinson's Research supports Phase 1 clinical trials of ATH-399A.

According to researchers, animal model studies have revealed that this molecule activates the crucial Nur1 enzyme, preventing the death of dopamine neurons and inhibiting the aggregation of synuclein protein. These findings were recently published in Nature Communications.

Rawat said: "Recently, Neurone Pharmaceuticals gave a research grant of Rs 1 crore. However, as I am currently on probation, DU rules do not permit us to enroll PhD students."

He told IANS that the collaborative work with the US began in 2012. 

Kim from McLean Hospital contacted Rawat for potential collaboration on developing a molecule for Parkinson's disease treatment. 

Since then, both teams have worked diligently, and Delhi University researchers have tested over 600 newly-synthesised compounds.

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