"We are here for children"

Aditya Kulkarni and Arun Papaiah make medicines for the rare diseases that hit children the most.

Every parent wants to make his child proud, and Aditya Kulkarni and Arun B Papaiah are no different. Only that their daughters – Kulkarni is two and a half years old and Papaiah's daughters are five and one and a half years old – can be very proud.

In fact, girls are a constant source of inspiration for innovation as the two men work at the two-story Aten Porus Lifesciences (APL) office in Bangalore, and at its US subsidiary – Oraxion Therapeutics . Srinivasan Namala, the CEO of Porus Labs, based in Hyderabad, is the third co-founder, who injected $ 1 million and gave them a good start.

They develop drugs for rare diseases or "orphan diseases" – 350 million of the world's population suffers, of which up to 70% are newborns for adolescents, but less than 5% of these diseases have suffered from diseases proven treatments. Seven thousand diseases are known, and most are fatal.

After nearly three years, a thousand failed experiments, and $ 3 million in R & D, good news arrived in January and messages of congratulations from biologists like Kiran Mazumdar Shaw. APL has licensed its first drug from an undisclosed biopharmaceutical company in the United States for additional studies and clinical trials for a whopping $ 125 million. Called ORX-301, the drug is intended for the treatment of Niemann-Pick type C disorder (NPC) and focal segmental glomerulosclerosis (FSGS). NPC is a neurodegenerative disorder caused by the abnormal accumulation of cholesterol in organs such as the brain, liver and spleen and 3000 patients worldwide, while FSGS affects the kidneys and 50 000 cases in the USA.

India does not have an NPC database but according to population and incidence rates, Kulkarni places the numbers between 10,000 and 20,000. "Regular doctors in India have not even heard of NPC.As a result, if a parent comes to them complaining that their child keeps on falling, they consider it a learning disability, "he adds. Mazumdar Shaw is currently working with the Karnataka government to formulate a research policy on rare diseases in India, says Kulkarnia.

Medicines for rare diseases are just that – rare – because the patient population is small and manufacturers and investors do not see the value of spending millions for something that will not pay them back . So why did both take the risk?

All this goes back to a promise that Kulkarni (chief scientific director) and Papaiah (CEO), 33, had made studying science at the University of Christ in Bengaluru in 2003. " We decided that when the time is right, we will start something that will make the difference, "Papaiah recalls.The time has come 10 years later, after Kulkarni and Papaiah have completed graduate studies in bio-organic chemistry from the States. USA, and MBA from Australia, respectively. "Since I was exposed to the foundation of a patient at Purdue University I knew the disease and the scope of this domain. So why not? "Kulkarni thought, whose parents are scientists." Papaiah jumped away from his family's real estate and construction business.

India has about 70 million patients with rare genetic disorders, so that in these figures they found an unmet medical need. "There is an urgent need to innovate and reduce the costs of treatment because nothing in the US, the treatment of most of these diseases costs $ 200,000 a year. While most families can still afford it, this is not the case in India, "says Papaiah.

It does not matter that they were "freshers, who had just finished their higher education". They then hired 12 new millers to run their chemistry lab. It worked. And as a "family", they beat the odds together.

Their most difficult moment came when a colleague died suddenly. Papaiah says, "When we were in the United States to sign this agreement, one of our project leaders died in a bus accident. She had been working on this medicine since the first day. It was his baby as much as ours, and we were unable to tell him this news. This case is now a tribute to her. "

When the first version of the drug was bombed in 2014, Kulkarni was devastated, but Namala's words – "I've invested in you two, not the drug, if it failed, it's okay." Kulkarni admits that he is anxious, so when he is moody, he confides in Papaya, and who does Papaya go to? " I'm going out for Biriyani. We end up talking about molcules and mice, but it's better, kulkarni.

In drug discovery, a test that works today may not work tomorrow. Or it may not work on humans. For these reasons, Kulkarni detached himself from the result. But Papaya can not stop dreaming. "I see the medicine in the hands of clinicians, it has been given to patients and they are improving," he says enthusiastically.

This dream makes them move forward, especially when others put their hopes there. "Prasanna Kumar Shirol runs the Organization for Rare Diseases in India.His daughter has a rare metabolic disorder called Pump." Probably, a cure for Pump is on their list so you have to put pressure on these guys, "he said to his daughter introducing us," said Kulkarni.

These words strike hard. "It breaks our heart to see our children catch cold or fever, and here are kids fighting for life," says Papaiah. "We are fathers, we feel a lot more."