On July 27th, a new medicine candidate for Amyotrophic Lateral Sclerosis (ALS) has become an FDA Orphan Drug Designation. It was developed by Pusan National University (PNU) department of Molecular Biology professor Park Bum-Joon’s research team. Thereupon, the Hyowon Herald looked into details through an interview with Professor Park.

   Q1. Would you please explain about the material that you developed and designated as FDA ODD?

  A1. The material is a treatment material for an illness called Amyotrophic Lateral Sclerosis(ALS). ALS is an illness in which the motor nerve that came out of the spinal nerve is dying selectively. The motor nerve that manages breath dies, and the host eventually dies. After the occurrence of the disease, the living period is usually about three to five years. Generally, there is currently no appropriate treatment. The material that we had designated controls abnormal cohesion of SOD1 protein, assumed as the cause of ALS and has the effect of the remedy. We checked its preservative effect on the motor nerve and the extension of life span. FDA acknowledged these results and designated it as a medicine for ALS henceforth. In particular, our material is a low molecular weight compound, so it can be taken orally when the material’s development is finished. It has the merit that taking this medicine is more straightforward for the patients compared to the medical treatments that are developing now.

   Q2. Did you have any hardships during the research progress?

   A2. I just had to watch patients around me get worse, and it was burdensome internally. However, our team suffered many hardships, rather than me. We progressed well up to the confirmation of the target ALS illness. Nonetheless, the investigation step of the actual compound was challenging and labor-intensive because we could not be sure that we would find suitable candidates for the medicine. Through this magazine, I want to express my appreciation to our laboratory’s graduate students and doctors.

   Q3. As the material you developed became FDA ODD, what do you expect? And please give your im-pressions of it.

   A3. I think we have to shorten the remaining development schedule as soon as possible to shorten the period for applying it to patients. If the person is diagnosed with ALS and is not treated, too many nerve cells will die, so that person can’t live normally. Hence, our research team founded “PRG S&Tech” company to develop rare disease medicine professionally. Our company tries to bring the treatment development process of this material forward to confer a benefit on more patients. Furthermore, I am delighted to benefit from shortening these processes because of  FDA ODD, of course.

   Q4. I heard you also developed a medicine candidate for Pediatric progeria before. What made you do this rare disease part research?

   A4. One of the dreams that I had when I had started my bio-scientific research is doing a job that can save people’s lives, even if it’s only one person. I progressed my early research about anticancer drugs. However, I realized that there are so many people already doing research on it, so I need to do a rare disease field that people think of as minority and uneconomical. Naturally, the rare disease field is not a field for big money because there are few patients. I thought that the patients were excluded from the new medicine development or scientific research because their number is few. I also thought that it is inappropriate that researchers to avoid researching minor fields because they could not earn much money. But my small efforts or fruits can be applied to patients fast and directly, so I am doing this.

   Q5. What are you planning to research from now on? Or do you have any goals to achieve?

   A5. My first goal is to get approval for new drugs for three new medicine candidates that our company is progressing. The rest, I hope for the research success for four or five kinds of rare genetic diseases and developing medicine for them. If I have the chance, developing treatment for more genetic diseases is my goal. Eventually, the final goal is to establish a rare, nonprofit disease research institute. It would let more researchers study rare diseases and making a personalized new medicine development chain that affords new medicines from patients’ demands.

   Q6. Do you have something to say for your junior or future researchers who admire you?

   A6. I usually say “think freely” to researchers. Although great scholars’ or textbooks’ contents are necessary, we cannot think creatively if we are bound by them too much. For others and junior professors, I would like to say, “It would not be easy, however, to do researches for real humans, and not for theoretical researches or thesis.”