Can genetic data be a magic bullet for drug research and development? –

Drug development has long been a problem for the pharmaceutical industry due to the cost and high failure rate of potential treatments. Ben Hargreaves finds that the vast amount of genetic data available today can help provide a faster and more targeted way to develop new drug candidates.

Recent years have brought a revolution in the way patients receive treatment. Instead of having a therapy that has been tested across a wide range of the population, there are now many therapies on the market that are designed specifically to work for subpopulations within disease areas. This includes treatments, such as immune checkpoint inhibitors, that are extraordinarily effective but only work for certain patients. This form of medical treatment is called personalized therapy, and it is quickly becoming a major focus of the pharmaceutical industry.

The logical extension of this kind of approach is the treatment of individual patients with their individual genetic makeup. Although cases of this type of therapy are rare, it is a growing field. One of the reasons is that researchers now have far more genetic data to work with than was ever possible before. The costs of testing per human genome in 2006 was approximately $14 million, and in less than two decades, an average consumer-purchased genetic test costs $100. With such a drop in costs, it means researchers can test far more often and work with far more genetic data than previously possible. Pharma has used this to begin creating targeted treatments, such as gene therapies, which are able to hone in on gene mutations that cause health problems and eradicate or replace the faulty gene.

Availability of data

It is often repeated, but it is true that the vast amount of data available to all industries has rapidly changed how they operate. The same is becoming true for the healthcare industry, and one of the first major breakthroughs in the field was the 100,000 Genomes Project. The initiative started in 2012 with the aim of collecting 100,000 genetic sequences from participants to then learn more about disease and disease progression. The information gathered from the project still provides insight today, a decade later.

The focus of research projects on the collection of data, especially genetic data, is now commonplace in nationwide health-focused initiatives. As part of the US government’s Cancer Moonshot initiative, a Cancer Moonshot biobank was launched, asking participants to donate biospecimens and associated health information to aid research. A recent British project, known as Our Future Health, is also taking a similar approach, combining genetic information with details about participants’ health and lifestyle, as well as their health records. The difference between these projects and the 100,000 Genomes Project is the scale – the Our Future Health initiative hopes to involve five million adult volunteers.

The future of R&D

The Our Future Health project was funded from various agencies, but the pharmaceutical industry was willing to include £100 million in investment in return for access and insight from the information gathered. The decision makes sense for all parties – the pharmaceutical companies receive analyzes that can help them develop their next medicine, while the researchers behind the project have the funds and additional expertise to pursue their goals. In the case of the Cancer Moonshot Biobank, participants could also receive free genetic testing and help receiving treatments specific to their type of cancer.

Outside of public research, there are vast sources of health data that have gathered it in a completely different way: by appealing to consumers. One of the most prominent is 23andMe, a company that provides users of its service with a genetic test that can provide information about parentage and health.

But despite providing genetic testing services to consumers, 23andMe describes itself as a human genetics and biopharmaceutical company. As such, the company signed a research agreement with GlaxoSmithKline in 2018, which saw the two companies use the genetic data collected by 23andMe to discover new, genetically validated drug targets. In 2020, the two companies advanced their first joint asset, GSK’608, to the clinic as a potential treatment for cancer. In 2022, the partners extended their partnership for another two years, stating that the collaboration had identified over 40 therapeutic programs over four years.

Accelerate development

The ability to use genetic data to accelerate the R&D process is one of the most promising aspects of the growth of data consumption. Adam Auton, VP of human genetics at 23andMe, told pharmaphorum that this approach has so far enabled the company to generate more than 50 early-stage drug development programs. “Our hypothesis, supported by published research, is that drug targets based on human genetics are at least twice as likely to become successful drugs than those that have no underlying human genetic evidence,” he said.

As for how the company works, Auton stated that it currently has more than 13 million genotyped customers, 80% of whom have consented to their health data being used in research. Researchers are able to study the aggregated, de-identified genetics of these individuals. The data can be paired with more than four billion self-reported health data points, enabling researchers to perform analyzes to identify underlying genetic causes of disease. In this way, the company is able to generate drug targets.

In addition to the drug candidate discovered with GSK, 23andMe has also developed its own investigational antibody, 23ME-00610. The potential therapeutic agent has reached the phase 1 stage of development, where it is being tested in patients with advanced solid malignancies. The monoclonal antibody binds to the CD200R1 receptor, preventing the interaction between CD200 and CD200R1. The interaction is thought to play a role in the maintenance of immune tolerance, and CD200 is highly expressed on certain tumors.

Alongside its genetic testing service, 23andMe is also expanding the range of medical information it can provide. The company has received US Food and Drug Administration approval for pharmacogenetic reports to be delivered to users based on their genetics. Certain genetic variants affect the way the body responds to specific medications. Through its Service, 23andMe may notify a consumer whether they have such a genetic variant, in a select number of approved cases, to potentially discuss the information with their healthcare provider.

When asked if this provides a window into the future where individuals take genetic tests to receive relevant health information and then potentially their own personalized treatment, Auton stated that this is still far from reality at the moment. He stated that genetic information may allow for the creation of a proactive health plan for individuals: “with the increased availability of genetic data, it is likely that genetics will increasingly be used in health care decision-making,” he concluded.

Can genetic data be a magic bullet for drug research and development? –

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