A biotech boom manifesting real results in rare pediatric diseases

lungs cystic fibrosis

By Bob Coughlin, managing director, life sciences, JLL

For the last two years, the pandemic has resulted in the explosive growth in life sciences. The world watched in awe and anticipation as biotech successfully raced to develop vaccines, therapeutics and diagnostics for Covid-19.

It has been a good moment for an industry that deserves such acute attention, but the growth trajectory of life sciences has been building for years, with benefits extending well beyond the pandemic. 

Today, biotech companies have the opportunity to make rapid advancements in critical and previously under-researched areas like rare pediatric genetic diseases.

Deeply personal

The issue is deeply personal to me. More than 20 years ago, while my wife was pregnant with our third child, we learned through genetic screening that our baby would be born with cystic fibrosis, a fatal and rare genetic disease that affects the lungs and digestive system.

Since then, I have been on my own race to find a therapy and/or cure.

There were not good options for treatment back then. At the time, I served in the Massachusetts House of Representatives, where I quickly learned that funding and good policy would both play a crucial role in addressing rare genetic conditions for babies and children.

In my search for treatments to save my son’s life – alongside my career in the life sciences industry that includes a stint as the CEO of the Massachusetts Biotechnology Council – I have seen first hand how the biotech boom has produced substantial results in pediatric drug development, and it is saving lives.

Research of rare pediatric disorders: a narrow market

The expansion of the biotech industry has created space for the research of rare pediatric disorders that have smaller patient populations compared to more common diseases like diabetes and hypertension. The value that these therapies bring to society is amazing.

In the case of cystic fibrosis, Vertex Pharmaceuticals developed a series of CFTR modulators. One of these drugs, Trikafta, is a breakthrough medication that is transforming patients’ lives, including my son. He took his first dose on November 8, 2019, and he has since grown eight inches and gained 50 pounds. His lung function is back to what it was when he was five years old, and he is now a junior in college. He is thriving, and my family and I are filled with gratitude.

These drugs don’t materialize overnight. It took decades and billions of dollars for Vertex Pharmaceuticals to produce these transformative treatments, but recent growth in the biotech market might help expedite ongoing drug development efforts.

Adopting a collaborative mindset

The pandemic might not deserve total credit for biotech growth, but it is responsible for encouraging collaboration across research platforms. By working together, the scientific community was able to develop three vaccines in 12 months, a dramatically condensed timeline compared to the five or six years vaccine development normally takes. There is no reason why we can’t have that same collaboration for all diseases.

Shared information will also help scientists fail and succeed quicker, and that is critical if we expect the cost of these therapies to decrease. Research and development can account for up to three quarters of healthcare costs, but expediting drug development through shared information will reduce the massive capital investment required to deliver treatments to the market.

It is amazing to see the industry change its attitude. We are creating therapies that change the course of disease, and, in some cases, cure disease, and it is all happening in our lifetime.

New companies have options

Advancing pediatric drug development will not only require collaboration from existing pharmaceutical companies but contributions from budding startups as well. Early-stage biotech companies were responsible for two-thirds of new drug developments over the last decade, and it is important that these companies have adequate lab space to pursue research.

The biotech industry in the U.S. was once isolated to three hubs: Boston, San Francisco and San Diego. Now, emerging life science hubs are popping up around the country, and it is helping to fuel more research. 

There are sick people everywhere; there are great hospitals everywhere; and there are smart people everywhere across the globe. There is no reason why you can’t build your own new cluster, and it is happening. 

For pediatric research, hubs are emerging near best-in-class children’s hospitals and in innovation communities complete with live-work-play amenities, public transit and attainably priced homes to help attract top talent.

With more options, including ready and built-out lab space, research and development companies have leverage to secure a more affordable workspace, which will allow them to put more money under the microscope.

We have the opportunity to do this for every single unmet medical need. When you focus on rare genetic disorders, it unlocks the mysteries of rare diseases and brings hope to families. 

It is truly miraculous.

Explore other topics: cystic fibrosisRare diseaseUSA

Newsletter Signup - Under Article / In Page

"*" indicates required fields

Subscribe to our newsletter to get the latest biotech news!

This field is for validation purposes and should be left unchanged.

Suggested Articles

Show More