Since the inception of 23andMe, our mission has been to help people access, understand and benefit from the human genome. 
<img loading="lazy" decoding="async" src="https://blog-api.23andme.com/wp-content/uploads/2018/07/Anne-headshot_2018-e1532470981667-252x300.jpeg" alt="" class="alignright wp-image-26375" width="280" height="334" srcset="https://blogcms.23andme.com/wp-content/uploads/2018/07/Anne-headshot_2018-e1532470981667-252x300.jpeg 252w, https://blogcms.23andme.com/wp-content/uploads/2018/07/Anne-headshot_2018-e1532470981667-859x1024.jpeg 859w, https://blogcms.23andme.com/wp-content/uploads/2018/07/Anne-headshot_2018-e1532470981667-990x1180.jpeg 990w, https://blogcms.23andme.com/wp-content/uploads/2018/07/Anne-headshot_2018-e1532470981667-768x915.jpeg 768w, https://blogcms.23andme.com/wp-content/uploads/2018/07/Anne-headshot_2018-e1532470981667-739x880.jpeg 739w" sizes="auto, (max-width: 280px) 100vw, 280px" />
We have also built a significant research team that has published over 100 scientific papers. In 2015, we launched 23andMe Therapeutics to develop novel treatments and cures based on <a href="https://www.23andme.com/dna-health-ancestry/">genetic insights</a> from the consented 23andMe community.
<h6>Novel Treatments</h6>
Today, I am thrilled to <a href="https://mediacenter.23andme.com/press-releases/gsk-and-23andme-sign-agreement-to-leverage-genetic-insights-for-the-development-of-novel-medicines/">announce</a> that we are launching a collaboration with GlaxoSmithKline (GSK) to accelerate our ability to make those novel treatments and cures a reality. <code style="display: none;"> </code>
I hear regularly from customers that they want to be part of a solution that is improving health care. We all have some disease or health issue that we care about. 23andMe has created a research platform to enable customers to actively participate in research — to not wait for solutions to appear, but for people to come together and make discoveries happen.  By working with GSK, we believe we will accelerate the development of breakthroughs. Our genetic research — powered by millions of customers who have agreed to contribute — combined with GSK’s expertise in drug discovery and development, gives us the best chance for success.
<h6>Choice</h6>
Our top priority is our customer, and empowering each individual with the options to participate in research. As always, customers choose whether or not to participate in research. Customers can choose to opt-in or opt-out at any time. 
I started 23andMe with the belief that by getting people interested in learning about themselves and participating in research, we would all benefit. Today is an important milestone in how 23andMe will be able to impact the lives of those with disease and how we will be able to improve the future of healthcare.
Finally, I want to recognize GSK for their commitment to empowering consumers to play a role in creating impactful new treatments and helping redefine how we discover novel medicines in the future.
<img loading="lazy" decoding="async" src="https://blog-api.23andme.com/wp-content/uploads/2015/10/sig_small_transparent.jpg" alt="" class="aligncenter wp-image-23161 size-full" width="318" height="133" srcset="https://blogcms.23andme.com/wp-content/uploads/2015/10/sig_small_transparent.jpg 318w, https://blogcms.23andme.com/wp-content/uploads/2015/10/sig_small_transparent-300x125.jpg 300w" sizes="auto, (max-width: 318px) 100vw, 318px" />

23andMe Blog

23andMe Blog Team

A Note On 23andMe’s New Collaboration with GSK

Anne Wojcicki, 23andMe&#8217;s CEO and co-founder, spoke recently with Kai Ryssdal, the host and senior editor at 
<img loading="lazy" decoding="async" class="alignright size-medium wp-image-22084" src="https://blog-api.23andme.com/wp-content/uploads/2015/01/Anne_Color_-Seated-250x300.jpg" alt="Anne_Color_ Seated" width="250" height="300" srcset="https://blogcms.23andme.com/wp-content/uploads/2015/01/Anne_Color_-Seated-250x300.jpg 250w, https://blogcms.23andme.com/wp-content/uploads/2015/01/Anne_Color_-Seated-768x923.jpg 768w, https://blogcms.23andme.com/wp-content/uploads/2015/01/Anne_Color_-Seated-852x1024.jpg 852w, https://blogcms.23andme.com/wp-content/uploads/2015/01/Anne_Color_-Seated-732x880.jpg 732w, https://blogcms.23andme.com/wp-content/uploads/2015/01/Anne_Color_-Seated-982x1180.jpg 982w" sizes="auto, (max-width: 250px) 100vw, 250px" />Marketplace, about the company, genetics, and the future of health care.


It&#8217;s a good overview of some of what we&#8217;ve been doing lately here at 23andMe.
Anne takes time to explain some of 23andMe&#8217;s core mission about helping people access and benefit from their genetic information, as well as explain how this information will become increasingly important in the future.


Genetic information is already changing health care, but it is also changing drug development and discovery. 23andMe recently announced the creation of its new therapeautics group, lead by renowned scientist Richard Scheller, Ph.D. Anne talked a little bit about this effort and how it aligns with 23andMe&#8217;s overall mission.


It&#8217;s worth taking a few minutes to listen to the piece.

The Future of Health Care

Before a medicine reaches pharmacy shelves, it goes through extensive and rigorous evaluation to prove it is safe and <img loading="lazy" decoding="async" class="alignright size-full wp-image-21214" src="https://blog-api.23andme.com/wp-content/uploads/2014/07/IBD-IMage-.jpg" alt="IBD IMage" width="240" height="160" />effective.
We want to shed some light on a few of the steps involved in drug development and highlight how the work being done by 23andMe with the <a href="https://www.23andme.com/ibd/" target="_blank" rel="noopener noreferrer">Inflammatory Bowel Disease Study</a> can help inform this process in the future. 

<h6>
The basics 
</h6>

On average, it takes somewhere between 11 and 14 years for a drug to go through development. This extensive process can cost up to $5 billion. Of all the medicines that start the clinical development process, less than 10 percent make it through all the stages of clinical trials and through Food and Drug Administration (FDA) approval. The majority of the drug
<div id="attachment_22135" style="width: 310px" class="wp-caption alignright"><img loading="lazy" decoding="async" aria-describedby="caption-attachment-22135" class="size-medium wp-image-22135" src="https://blog-api.23andme.com/wp-content/uploads/2015/01/Global-Drug-Dev-Spending-300x223.jpg" alt="Image: The Center for Information and Study on Clinical Research Participation " width="300" height="223" srcset="https://blogcms.23andme.com/wp-content/uploads/2015/01/Global-Drug-Dev-Spending-300x223.jpg 300w, https://blogcms.23andme.com/wp-content/uploads/2015/01/Global-Drug-Dev-Spending.jpg 548w" sizes="auto, (max-width: 300px) 100vw, 300px" />Image: The Center for Information and Study on Clinical Research Participation</div>
development timeline is taken up by conducting the clinical trials (6-11 years), and one of the most time-intensive parts of running clinical trials is finding study volunteers. Only 7 percent of Americans say their doctor has ever suggested they consider participating in a clinical research study.

<h6>
What happens before clinical trials? 
</h6>

Before a medicine enters clinical development, it goes through years of testing in the laboratory, using cell culture and animals to better understand how the drug works and to ensure it’s safe.

<h6>
Clinical Trials: Phase I to Phase III 
</h6>

The very first study of a new medicine in people is called a “phase I” study. This type of study is very small – just a handful of volunteers are given the drug, starting at very low amounts. The dose is increased incrementally until they find a dose that is safe and also high enough to be considered active.
<div id="attachment_22136" style="width: 310px" class="wp-caption alignright"><img loading="lazy" decoding="async" aria-describedby="caption-attachment-22136" class="size-medium wp-image-22136" src="https://blog-api.23andme.com/wp-content/uploads/2015/01/Time-in-years-to-FDA-approval-300x211.jpg" alt="Image: The Center for Information and Study on Clinical Research Participation " width="300" height="211" srcset="https://blogcms.23andme.com/wp-content/uploads/2015/01/Time-in-years-to-FDA-approval-300x211.jpg 300w, https://blogcms.23andme.com/wp-content/uploads/2015/01/Time-in-years-to-FDA-approval.jpg 566w" sizes="auto, (max-width: 300px) 100vw, 300px" />Image: The Center for Information and Study on Clinical Research Participation</div>
The next study, a “phase II” trial, is larger and looks for safety as well as efficacy. Essentially this phase looks at whether the drug does what it is meant to do. Does it improve the symptoms or alleviate the disease?
A “phase III” study is the largest still and often includes a “control” group – volunteers who are given the standard-of-care medications (not the experimental drug) for comparison.  The FDA then reviews the data collected from these trials. The FDA can then request additional studies, or it can “clear” the drug for use (meaning the manufacturer can go ahead and sell the drug.)
<h6>
How can genetics impact the drug development process?</h6>
Newly approved medicines provide benefits to many people. They alleviate symptoms, or sometimes cure a disease. However, they don’t work well for everyone. Many people take medicines which don’t help their condition, or sometimes lead to unacceptable side effects. There is no way to know which medicines will work for which patients. The method of finding the right treatment for any patient is still very much a “trial and error” process – the doctor keeps prescribing different medicines until he or she finds one that works.


This is where genetics may play a role in improving the process. We know that genetics can influence how people respond to drugs. By studying the genetics underlying diseases, such as Inflammatory Bowel Disease (IBD), we hope to provide more insights into how IBD differs between people. It is possible that there are certain genetic variants which can predict what type of drug would be most effective, or cause the least side effects.
A better understanding of the genetics of IBD can change how clinical trials are done, which can in turn bring medicines to the market faster and provide guidance to physicians about which patients will most benefit from which drugs.
<div id="attachment_22134" style="width: 310px" class="wp-caption alignright"><img loading="lazy" decoding="async" aria-describedby="caption-attachment-22134" class="size-medium wp-image-22134" src="https://blog-api.23andme.com/wp-content/uploads/2015/01/Examples-of-Drugs-Approved-300x241.jpg" alt="Image: The Center for Information and Study on Clinical Research Participation " width="300" height="241" srcset="https://blogcms.23andme.com/wp-content/uploads/2015/01/Examples-of-Drugs-Approved-300x241.jpg 300w, https://blogcms.23andme.com/wp-content/uploads/2015/01/Examples-of-Drugs-Approved.jpg 528w" sizes="auto, (max-width: 300px) 100vw, 300px" />Image: The Center for Information and Study on Clinical Research Participation</div>
Patients who enroll in clinical trials of new medicines must meet several specific eligibility criteria. For IBD, right now those criteria may involve a list of medications the person must have taken (and failed) and/or a specific range of acceptable lab results.
In the future, the eligibility criteria for an IBD trial may include lists of specific genetic variants that participants need to have in order to enroll in the study along with other precise measures of disease such as the composition of their microbiome (the types of bacteria in their gut). In this way, only those patients who would most benefit from the experimental medicine would be enrolled, sparing patients with no hope for efficacy the potential side effects of the drug and allowing them to find other clinical trials better suited for their IBD.


Precision medicine (prescribing the right medication for the right patient at the right time) is already a reality in the treatment of many cancers. Examples of such drugs are: trastuzumab (Herceptinâ“‡) for the treatment of HER2 positive breast cancer, cetuximab (Erbituxâ“‡) for the treatment of colorectal cancer which carries a mutation in the EGFR gene but not the KRAS gene, or crizotinib (Xalkoriâ“‡) for the treatment of lung cancers with an ALK gene fusion.
The reality of genetically targeted medicines is still not yet realized for IBD, but we are helping lay the groundwork through our research efforts and collaboration with Pfizer, a manufacturer of new treatments for IBD.
Observational studies (where no drugs or treatments are involved) such as our IBD study play an important role in providing a basic understanding of the disease. We hope the results of this study will directly inform Pfizer’s drug development efforts and help us move just a step closer to the future of precision medicine approaches in IBD.

Drug Development’s Long And Winding Road

<img loading="lazy" decoding="async" class="alignright size-full wp-image-4747" title="800px-HCV_prevalence_1999" src="https://blog-api.23andme.com/wp-content/uploads/2009/09/800px-HCV_prevalence_1999.png" alt="800px-HCV_prevalence_1999" width="433" height="189" srcset="https://blogcms.23andme.com/wp-content/uploads/2009/09/800px-HCV_prevalence_1999.png 800w, https://blogcms.23andme.com/wp-content/uploads/2009/09/800px-HCV_prevalence_1999-300x132.png 300w, https://blogcms.23andme.com/wp-content/uploads/2009/09/800px-HCV_prevalence_1999-768x337.png 768w" sizes="auto, (max-width: 433px) 100vw, 433px" />
Unlike the flu virus, which the body is generally able to fight off completely, infection with hepatitis C is often chronic.   That means for most of the three to four million people worldwide who are newly infected each year the virus will persist in the body, where it greatly increases risk for chronic liver diseases including cirrhosis and cancer. The only treatment currently available – an almost year long course of drugs – often causes severe side-effects. Worse yet, it fails in about half of all patients.
Last month, a <a href="https://dx.doi.org/10.1038/nature08309" target="_blank" rel="noopener noreferrer">study</a> published online in the journal Nature showed that DNA variations in and around the IL28B gene influence whether a person chronically infected with the hepatitis C virus will fully respond to the standard treatment regimen. Now three new reports further support the importance of IL28B variations in the body&#8217;s response to hepatitis C, a finding that has implications for the future of hepatitis treatment and drug development.
An Australian group headed up by <a href="https://dx.doi.org/10.1038/ng.447" target="_blank" rel="noopener noreferrer">Vijayaprakash Suppiah</a> analyzed the DNA of more than 800 people with European ancestry from Australia, the UK, Germany and Italy infected with hepatitis C.   They found that having one or two copies of the less-common G version of , a SNP near the IL28B gene, doubled the odds that a person would fail to have a sustained response to treatment.
A group headed up by <a href="https://dx.doi.org/10.1038/ng.449" target="_blank" rel="noopener noreferrer">Yasuhito Tanaka</a> studied 326 Japanese hepatitis C patients and also found that predicts whether or not a person will respond to treatment.   In this population, however, the effect was much greater.   Carrying a G increased the odds of treatment failure by at least twelve times.
&#8220;This SNP has such a strong association with response that, in combination with other parameters, genotyping it and other genetic variants should be a useful part of   clinical management,&#8221; write Suppiah et al., who along with Tanaka et al. published their results online this week in the journal Nature Genetics.
Another study, this one published online in <a href="https://dx.doi.org/10.1038/nature08463" target="_blank" rel="noopener noreferrer">Nature</a>, found that variation near IL28B might be part of what allows a minority of people infected with hepatitis C to clear the virus naturally, without any treatment.
David Thomas and colleagues found that individuals with European or African ancestry were about three times more likely to spontaneously clear a hepatitis C infection if they carried two copies of the C version of than if they had CT or TT at this SNP.
Twenty-eight percent of the people with the CT or TT genotypes were able to fight off their hepatitis C infection without treatment, a number very close to the population average.   But 53% of those with the CC genotype were able to become naturally virus-free.
In a <a href="https://dx.doi.org/10.1038/461357a" target="_blank" rel="noopener noreferrer">commentary published in Nature</a>, Shawn Iadonato and Michael Katze express doubts about the importance of these new discoveries.
&#8220;Although these findings raise the tantalizing prospect of a more personalized approach to treating [hepatitis C] by tailoring treatment to patients who are most likely to benefit, the reality is more sobering,&#8221; they write.
Iadonato and Katze worry that testing for IL28B variations would not provide doctors with a straightforward yes-or-no answer to the question of whether a patient will respond to hepatitis C treatment.   This is because not all carriers of the advantageous versions of the variants clear the virus, nor do all patients lacking them fail to benefit from treatment.   Furthermore, they point out, there is currently no alternative hepatitis C treatment available.
What they do not consider, however, is how this new research may help change that. In a separate <a href="https://dx.doi.org/10.1038/ng.453" target="_blank" rel="noopener noreferrer">commentary in Nature Genetics</a>, Thomas O&#8217;Brien suggests that the current findings could increase the interest in developing hepatitis therapies based on the protein encoded by IL28B, interferon-Î»3.   He notes that an early clinical trial testing the effects of a related protein, interferon-Î»1, has already had some success.   And certain properties of interferon-Î»s may cause them to have fewer side effects than current treatments.
O&#8217;Brien is still cautious about the findings linking IL28B variations to hepatitis C treatment response.   He says that more research in this area is needed in diverse populations and in people infected with different forms of hepatitis C.   He also stresses that researchers need to consider how these genetic findings fit in with other factors that affect treatment response, including age and gender.
Overall, however, his view is optimistic.   He concludes: &#8220;With these caveats, predictive models of HCV treatment response hold the potential to inform treatment decisions for millions of patients who are infected with HCV.&#8221;
Map: <a href="https://en.wikipedia.org/wiki/File:HCV_prevalence_1999.png" target="_blank" rel="noopener noreferrer">PhilippN</a>

More Evidence That Genetic Variations are Important for Hepatitis C Infection and Treatment

<img loading="lazy" decoding="async" class="alignright size-full wp-image-4053" title="Biology experiment on well plate" src="https://blog-api.23andme.com/wp-content/uploads/2009/07/istock_000004124815xsmall.jpg" alt="Biology experiment on well plate" width="283" height="424" srcset="https://blogcms.23andme.com/wp-content/uploads/2009/07/istock_000004124815xsmall.jpg 283w, https://blogcms.23andme.com/wp-content/uploads/2009/07/istock_000004124815xsmall-200x300.jpg 200w" sizes="auto, (max-width: 283px) 100vw, 283px" />
The exact causes of Crohn&#8217;s disease remain a mystery, but scientists do know that genetic factors play an important part. More than <a href="https://blog.23andme.com/articles/snpwatch-number-of-snps-associated-with-crohn%E2%80%99s-disease-triples/" target="_blank" rel="noopener">30 variations</a> have been associated with increased risk for the disease, but changes in one gene, NOD2, have been found to be especially critical. &nbsp; Three different variants in this gene have been associated with significantly increased risk for developing the disease. &nbsp; It&#8217;s estimated that 10-15% of people with Crohn&#8217;s disease have two copies of one of these variants.
Because changes in NOD2 are associated with so many cases of Crohn&#8217;s disease, medications made to interact with the protein the gene encodes would seem to be obvious candidates for drug development. &nbsp; But the NOD2 protein doesn&#8217;t have the type of function that can be modified by a drug.
A new avenue of drug research may have been opened, however, thanks to a study from Derek Abbott and colleagues at Case Western Reserve University. &nbsp; In a paper appearing in the August issue of <a href="https://dx.doi.org/10.1016/j.cub.2009.06.038" target="_blank" rel="noopener">Current Biology</a>, they show for the first time that a protein called ITCH can influence NOD2 biological pathways. &nbsp; This is important because, in the parlance of pharmaceutical development, ITCH is a &nbsp; &#8220;druggable&#8221; target.
NOD2 binds to bacteria that make their way into cells, setting off signals that activate the immune system. Research has shown that the Crohn&#8217;s disease-associated NOD2 variants decrease the signals that go through a particular protein called NFkB. &nbsp; Abbott&#8217;s lab found that ITCH also decreases NOD2-dependent NFkB signals. &nbsp; This is significant because studies have shown that increased NFkB signaling can help slow down the progression of Crohn&#8217;s disease.
&#8220;The thought is that if you could identify patients with NOD2 polymorphisms who also displayed early gastrointestinal trouble, you could block ITCH to increase NFkB signaling and not let the Crohn&#8217;s disease get to the acute stage,&#8221; Abbott said in an email.
Abbott and his fellow researchers are in the very early stages of looking for drugs that could be used to inhibit ITCH and possibly be useful in treating Crohn&#8217;s disease. &nbsp; But the road to drug discovery is a long one. &nbsp; ITCH interacts with many other proteins in cells and it&#8217;s not yet clear if blocking it would have unwanted effects on other important biological pathways. However, in the future this research could lead to a valuable drug against Crohn&#8217;s disease – at least for the 10 to 15% of patients who have variants that cause decreased NOD2 signaling.

Researchers Take A Step Toward Developing New Drugs For Crohn’s Disease

<img loading="lazy" decoding="async" class="alignright size-full wp-image-3892" title="istock_000003316166xsmall" src="https://blog-api.23andme.com/wp-content/uploads/2009/06/istock_000003316166xsmall.jpg" alt="istock_000003316166xsmall" width="332" height="220" srcset="https://blogcms.23andme.com/wp-content/uploads/2009/06/istock_000003316166xsmall.jpg 425w, https://blogcms.23andme.com/wp-content/uploads/2009/06/istock_000003316166xsmall-300x199.jpg 300w" sizes="auto, (max-width: 332px) 100vw, 332px" />
Not surprisingly, there has been intense interest in the genetics of obesity in recent years.   Obesity is a major health problem, resulting in tens of thousands of premature deaths and billions of dollars in healthcare costs each year in the United States, and it is known from twin and family studies that weight is a highly heritable trait.
The genetic mutations and variations identified so far explain only a small percentage of the variability in body mass seen in the population.   Some exciting clues have been found, however, as to why some people are more prone to obesity than others and how this might be counteracted.   But new research, published online this week in the <a href="http://www.cell.com/AJHG/abstract/S0002-9297(09)00238-9" target="_blank" rel="noopener">American Journal of Human Genetics</a>, shows that caution must be taken in moving from genetic discoveries to drug development.
Variations in the FTO gene have consistently been associated with obesity. On average, people who carry two copies of the &#8220;risky&#8221; version of this gene weigh six to eight pounds more than those who carry none. Mice completely lacking the FTO gene were shown to be leaner than their normal littermates, despite being less active.   These results have led some to suggest that inhibiting the FTO protein with a drug could be a good for obesity treatment or prevention.
(23andMe customers can see their FTO data in the Obesity Research Report.)
But an international team of researchers led by Sarah Boissel of the UniversitÃ© Paris Descartes in France has found that a mutation that interferes with the function of the FTO protein is associated with a lethal genetic condition.   Members of a large, inbred Palestinian family born with two copies of this mutation had multiple developmental abnormalities and died before the age of three.   Based on their results, Boissel and her colleagues warn that any research exploring the use of FTO inhibitors as obesity treatments must include a &#8220;careful assessment&#8221; of the potential to cause birth defects and other toxic side effects.
More about FTO:
<a href="https://blog.23andme.com/2008/12/11/snpwatch-gene-variant-linked-to-obesity-affects-food-choices-in-children/" target="_blank" rel="noopener">SNPwatch: Gene Variant Linked to Obesity Affects Food Choices in Children</a>
<a href="https://blog.23andme.com/articles/it%E2%80%99s-not-genes-or-environment-it%E2%80%99s-genes-and-environment" target="_blank" rel="noopener">It’s Not Genes or Environment, It’s Genes AND Environment</a>

drug development — Page 2

A Note On 23andMe’s New Collaboration with GSK

The Future of Health Care

Drug Development’s Long And Winding Road

More Evidence That Genetic Variations are Important for Hepatitis C Infection and Treatment

Researchers Take A Step Toward Developing New Drugs For Crohn’s Disease

Researchers Warn That Drug Development Based On Obesity Genetics Could Deliver More Than Is Bargained For

Stay in the know.