By Rajaneesh K.Gopinath, Ph.D.
Ruchi Jhonsa, Ph.D.
TAIPEI, TAIWAN – Day 1 of BIO Asia-Taiwan 2020 saw remarkable talks from eminent scientists who shared their cutting edge research and biotech professionals who discussed the impact of the COVID-19 on global biotech trends. In part 2, we put together selected takeaways from the day. Link to Part 1
Milasen and Prospects for Hyper-Personalized Medicine
Nearly 30 million patients are suffering from rare diseases in the United States without any therapeutic options. Despite the progress achieved in genetic testing technology, such diseases prove to be a significant challenge for drug developers.
Professor Timothy Yu, Principal Investigator and Attending Physician at Boston Children’s Hospital shared a remarkable case. Milasen, an antisense oligonucleotide (ASO)-based drug, was manufactured, and FDA approved rapidly to save a single patient suffering from a rare, lysosomal storage disorder. Dr. Yu and colleagues designed an oligonucleotide to treat a fatal neurodegenerative condition called Batten disease in a 6-year-old patient, Mila Makovec.
Milasen performed well in clinical evaluations by reducing epileptic events and did not exhibit any significant adverse reactions. In the beginning, the patient had 15 to 30 seizures a day, each lasting 1 to 2 minutes. Milasen reduced the frequency of seizures to 0-20 times a day and decreased the duration of each seizure to less than a minute. Following successful trials, several stakeholders, including drug manufacturers and the FDA raced to deliver the drug for Mila, setting a record in the timeline of drug development. By virtue of the positive trial data, the drug received FDA approval within just three months. Milasen is a pioneering example of personalized medicine and a model for other drug developers to emulate and seek approval for rare disease drugs.
Organoids to Model Human Disease
Prof. Hans Clevers, Principal Investigator at the Hubrecht Institute, enlightened the audience by mentioning that the concept of personalized medicine is not new. He proceeded to explain that the 60-year-old practice of performing an antibiotic resistance screen to administer the right antibiotic to a pneumonia patient is essentially “personalized medicine”. He then segued into the concept of organoids, the three-dimensional structures derived from stem cells, and their promise in advancing personalized medicine.
Professor Clevers pointed out that “the gut harbors the champion of all stem cells” since intestinal stem cells (ISCs) plays a crucial role in renewing cell populations. The harsh environment of the gut damages the intestinal epithelial lining, and therefore every 4-6 days, ISCs differentiate to help renew the population. Stem cell-derived mini-gut organoids grown in a dish could be successfully transplanted to cure patients suffering from inflammatory bowel disease (IBD).
Today, many organs can grow into organoids, and they can be derived from both healthy and disease tissues. This opens up multiple avenues for studying diseases and facilitates drug development. For instance, researchers could grow organoids from both cancer and healthy cells and perform various analyses, including sequencing, to identify cancer-causing mutations. Besides, cancer organoids serve as a wonderful platform for testing drug combinations, and a cancer organoid biobank can be established to achieve personalized medicine. Extensive research in this area has culminated in the launch of Hubrecht Organoid Technology (HUB), “a paradigm-shifting platform for drug discovery and development, companion diagnostics, and (pre)clinical patient stratification.”
In summary, Prof. Clevers described organoids as “patients in the lab” and reemphasized the merits of the organoid technology platform and the immense potential it holds in advancing different aspects of drug discovery.
Emerging from the Pandemic: A Global Perspective
Dr. Graham Lewis, Vice President, Global Pharma Strategy, IQVIA, presented a perspective on changes that will happen to the pharmaceutical industry and health care sector post-COVID.
During the pandemic, there is a growing demand for pharmaceutical companies to support health care systems, increasing net sales. However, Dr.Lewis reckons the overall sales would be threatened in the future due to an increase in rebates and discounts, price raises and market hurdles, and delays in revenue streams and clinical development. He forecasts a lower growth over the next few years, and net prices will transfer to virtually zero levels.
Companies will need to develop vaccines at the same time, ensuring that chronic conditions are managed effectively. This demands new ways of working. “There’s going to be a great need for more flexibility and efficiency in research and development. These methods, of course, have been available now for a number of years. But the industry has been slow to take them up. But because communication is moving pressingly into a digital or virtual framework, then the acceleration of skill sets and scale is going to be essential,” said Dr. Lewis. Post the pandemic, pharma companies will have to re-assess missions and their overall strategy to support health systems, accelerate digital transformation, build or strengthen capabilities to enable novel patient support programs, and re-evaluate everything from launching, resourcing models, deployment development, portfolio priorities, and supply chain.
Dr. Lewis opined that one interesting dimension of the changes that we are seeing is the gradual growing importance of BRICS nations, Brazil, Russia, India, and China. “China was already the second-largest pharmaceutical market in 2019. And we expected it to retain that position in 2022 and indeed beyond that as well. We do see Brazil, though, becoming more important. And our current estimates suggest that Brazil will be the fifth-largest pharmaceutical market by 2022. India will enter the top 10 for the first time, in our view, and Russia will be just below in 12th position.” he concluded.
©www.geneonline.news. All rights reserved. Collaborate with us: firstname.lastname@example.org