Developing Next Gen Multiple Sclerosis Therapies: Part 1

The following is part of an MStranslate exclusive series which highlights the struggles and obstacles in developing the next generation of therapies and drugs for human disease. Dr. Travis Stiles is a neuroscientist who has worked to develop regenerative therapies capable of reversing neuronal damage caused by disease and trauma, such as multiple sclerosis and spinal cord injury.

You can help support his efforts by clicking here. Every contribution helps move this work closer to these revolutionary discoveries becoming therapeutic realities.

PART 1

For most people, medicine can be another language. It can be intimidating, even scary. However, modern medicine can also be miraculous and a tremendous source of hope. If you’re reading this, you’re very likely coming to this article from the experience of MS. You may be a patient, or you may be a loved one or a caregiver, but either way you have been affected by the realities of human disease. As such, you have seen both the blessings and pain that hope can bring. So, what we are going to do here is arm that hope with knowledge, and hopefully direction. This series won’t be about MS specifically. What we are going to discuss goes beyond any 1 disease. Let me be clear, while I truly hope readers find this compelling and informative, there will be no cheerleading and inspirational thoughts. Instead, I’m going to do my very best to empower readers with an understanding of the modern complexity of medical advancement.

I hope this series reinforces the fact that modern medicine is a marvel, one that can bring all new cures and treatments to improve the human condition and advance the way we approach disease. What I also hope to convey to you is that the old way of doing things is outdated, and the changes are coming rapidly and deservedly. From these discussions I hope that I can offer a renewed optimism for the future and an informed outlet for the passion and motivation that I see in patients and advocates. At the end of the day passionate advocacy is the most powerful tool any of us have to promote change, and my sincere desire is to arm you with the perspective, knowledge, and reinvigorated hope to push for the types of actions that will most efficiently lead to significantly improved patient outcomes. If I accomplish that, we all win. So lets get to it….

Preface

The bottom line is that we just aren’t curing diseases or making revolutionary drugs the way we used to.  This reality has led to a lot of negative perceptions of the pharma and biotech industries that have begun to sway public opinion against these institutions. While there has been decreased productivity in terms of cures, that is not to downplay the advancements that we make. Regardless, the simple reality is that the pace of such innovations has slowed. So how do we address this? How do we adapt the system to address the slowing of medical innovation? The good news is, those changes are happening, but by understanding the process of medical innovation and how the system is evolving will help all of us advocate for the activities that will expedite the next generation of medical treatments and cures. But to have this conversation, we need a foundational understanding of what it takes to make those things happen.

Let’s start with a truth we all understand; if you want to cure a disease, you need cash…a LOT of cash. As a result, if you aren’t working for a wealthy research institution, the federal government, or a large pharmaceutical company (commonly referred to as large or “big” pharma), you are starting off at a gross disadvantage. While such large operations are incredibly valuable, they are also notoriously limited by bureaucracy, making innovation difficult. Innovation requires outside-the-box thinking, flexibility, adaptability, and depth of understanding. Oh yeah….and did I mention $$$CASH$$$!!!???

….what do I know? Who is this guy?

Before I go any further I think it may be worthwhile to explain to you why I know anything about this and where I am coming from. I began my career as a plain old scientist. I chose a career path where my job is to cure disease. Specifically, my life’s work is dedicated to repairing nerve damage. Currently, I am focused on reversing damage caused by multiple sclerosis and spinal cord injury by promoting the regeneration of damaged nerves. Such discoveries have many potential additional applications, but we are starting with these diseases and hope to expand to others as we progress. As you are likely very aware, the consequences of these diseases are largely irreversible, and tackling such problems is no small task. Unfortunately, I am not a large pharmaceutical company, government researcher, or backed by a large academic institution, meaning I lack the vast resources of such entities. As such, I am not a representative of such groups, so when I speak of such entities know that I am not trying to sell you on the merits. Now, I am biased towards groups like mine; small start-ups doing the high-risk, high-reward work of translating basic science into future treatments and cures.

As you can probably imagine, it is no small feat to tackle problems such as these that have defied medical experts for basically ever. What I do is hard, but what makes me an entirely unique entity relative to drug developers of the past is that I chose the freedom of life outside of the bureaucracy of large institutions so I could focus on one thing only: making the best treatments possible. This unique perspective will frame a lot of my discussion, and we will get MUCH more in depth on these issues, but for now let me clarify that as much as my smaller and more agile operation enables innovation, it lacks that almighty dollar so crucial to my goals. This is a calculated and necessary compromise between the vast resources and capabilities of large pharma/academia and the agility and focus of being a small company. Small companies like mine are often spinouts of pharma or academia and are often referred to as biotech “start-ups”. The distinction between such start-ups and the entities they both derive from, and bridge the gap between, is an important and little understood aspect of modern drug development, and I will elaborate more throughout this series.

But before we can break down the nuances of drug development and talk about the ways in which we can do it all better, we must first understand the process overall. So how exactly does a drug come to the market?

Progress to Part 2 of Dr Stiles’ series by clicking here.