With progressive advances in technology and medicine, the promise of personalized medicine has loomed large for years. The use of Big Data, CRISPR gene editing techniques, and other innovations have spurned visions of individualized care. Yet, while the progress seems slower than expected, there is increasing evidence that these trends are coming. One such example is the use of customized genetic drugs for a variety of rare conditions. In fact, there are several organizations and companies doing their best to make these available on a broader scale. In the process, millions could benefit from access to this type of care.
Unfortunately, there a few obstacles in the way when it comes to widespread use of customized genetic drugs. In addition to cost issues, data sharing and potential side effects of some drugs pose common problems. At the same time, current models to secure these personalized genetic therapies may not be sustainable long-term. If this industry is to evolve and expand, then new approaches will be required. It will also need major investments that support its growth and expansion. These are the struggles that businesses in the industry face today in making true personalized medicine a reality.
“Ultimately, before this explodes into treating dozens of families per year, they’re going to have to show that [customized genetic drug treatment] works.” – David Corey, Biochemist at the University of Texas Southwestern Medical Center at Dallas
Current Customized Genetic Drugs
Previous reports have described various gene-editing techniques like CRISPR and RLR. However, some of the latest customized genetic drugs are being produced through different strategies. Several institutions and companies are exploring anti-sense drugs, which can target discrete genetic flaws and conditions. In essence, anti-sense DNA drugs provide a template by which messenger RNA can produce specific proteins. And if a particular genetic flaw is resulting in faulty protein production, anti-sense therapies may provide a solution. By bypassing the dysfunctional DNA strand, anti-sense drugs can redirect protein production normally.
The reason these are called anti-sense drugs is because DNA segments are composed of two strands. One DNA strand, called the sense strand, is capable of being read and making “sense.” The other strand, however, only serves as a template for producing messenger RNA “sense” strands. Within a cell, when DNA strands separate, this is the function of the anti-sense portion. But when the naturally occurring anti-sense strand has a genetic defect, the subsequent RNA is similarly flawed. This results in errors in protein production that can have profound effects. In fact, many rare genetic diseases are caused by such defects and flaws. The hope of personalized medicine is therefore that these precise defects can be erased. And anti-sense drugs may be one strategy to achieve this.
“Developing, manufacturing and then giving it away for life for free is an amazing concept, for the most desperate, most underserved patient population we know of.” – Dr. Stanley Crooke, Founder of Ionis Pharmaceuticals and Co-founder of n-Lorem
The Customized Genetic Drugs Industry
At the current time, the overall landscape of companies making anti-sense drugs are rather small. They represent a segment of the entire personalized medicine sector. One such company called Ionis Pharmaceuticals is dedicated to making these medicines for rare genetic disorders. Its founder, Dr. Stanley Crooke, also founded n-Lorem, which is a non-profit with similar intentions. The non-profit partnered with Ionis as well as Biogen to reduce the substantial costs associated with customized genetic drugs. The agreement is that Ionis and Biogen discount or donate equipment, services, and testing. At the same time, n-Lorem provides infusions of the drugs for free for life. In total, this arrangement reduces costs by about 40% on average.
These are of course not the only market players in the anti-sense drug sector. A parent of a child with Batten’s Disease, a rare genetic condition, founded a for-profit company, EveryONE. This company is exploring how to make these customized genetic drugs sustainably. She also partnered with Dr. Timothy Yu, a neurologist and genetic researcher at Boston Children’s Hospital. They formed N=1 Collaborative, designed to create greater accessibility to personalized medicine care to individuals. To date, N=1 Collaborative has over 300 members consisting of scientists, investors, parents and companies. These enterprises alone and cumulatively have raised millions of dollars for research and funding. But the yield thus far has been quite limited, involving only a handful of drugs for a handful of genetic conditions.
“We’re talking about dying children. Companies, academic institutions and foundations should all have a mandate to share data so we can learn what works, because it is unethical not to.” – Julia Vitarello, Founder of EveryONE Medicine and Cofounder of N=1 Collaborative
The Challenges Ahead for Personalized Medicine
When it comes to customized genetic drugs, major hurdles notably exist. The most obvious one involves costs, as these therapies are certainly not cheap. Personalized medicine treatments involving anti-sense drugs can cost as much as $2 million per patient per treatment. This is the main reason only a few dozen individuals with rare genetic diseases have received interventions. The current approach involving donation collection and collaborative discounts has helped a handful of patients. But this algorithm is hardly sustainable long-term. Instead, new models that attract major investors are needed. But in order to attain this, proof of efficacy and utility is needed. And pricing must come down through increased competition and a larger scale of service.
Unfortunately, cost is not the only barrier to date. Two other notable challenges exist when it comes to customized genetic drugs. One involves safety issues as a few patients have developed a neurologic condition called hydrocephalus after receiving anti-sense drugs. In fact, this form of personalized medicine resulted in the death of at least one patient. Secondly, the industry is handcuffed by a lack of data sharing among companies in the sector. Due to philosophical and perhaps proprietary reasons, personalized medicine organizations aren’t pooling their findings that might lead to new findings. This will continue to slow progress in the field and likely deter investors as well. In any case, customized genetic drugs reflect the incredible advances made through science and technology over the past decade.
Personalized medicine could indeed be right around the corner. But it will require innovative ideas and strategies by businesses to make this a practical future.