Conventional medical care generalizes treatment to all patients with a particular disease. But since a disease is as individual as the person who has it, casting a wide therapeutic net has its limitations. For one, patients with a certain genetic makeup might not respond to a particular drug as well as patients with different genetics, or they might experience different side effects. As personalized medicine becomes a reality, it could rectify these less-than-ideal situations.
From the diagnostic point-of-view, personalized medicine is a shift from reactive to proactive. Based on a person’s health, genetic, and environmental profiles, doctors practicing personalized medicine could assess a patient’s risk for acquiring a genetic disease before any symptoms develop. This might allow them to target the specific genes that account for illness (the BRCA1/BRCA2 genes that predispose a woman to breast cancer, for example), incorporate a prevention strategy, and monitor those genes over time. When it comes to treatment, personalized drugs could be prescribed based on an individual’s molecular “build” and targeting treatment where it will do the most good and the least harm.
While a few tests are now available that can help predict how an individual is likely to respond to certain medications, for the most part personalized medicine is currently making the most headway at the research stage. For example, researchers are now using a technique, referred to as disease in a dish, to re-create a person’s individual disease in a laboratory dish to better study the underlying causes and screen potential drugs tailored to that patient. For instance, researchers can take skin cells from a patient with amyotrophic lateral sclerosis (ALS), and introduce a few genes that turn them into a type of stem cells called induced pluripotent stem (iPS) cells. Like their embryonic cousins, iPS cells can proliferate, generating many more cells, and differentiate, making any type of mature cell. In this scenario, researchers can then direct iPS cells to differentiate back to brain cells with the same molecular and genetic defects that gave rise to ALS in the first place. Disease in a dish gives scientists a limitless number of cells to study, manipulate, and use to perform personalized, miniaturized clinical drug trials in a dish. Whether or not iPS cells can be re-introduced to a patient for therapeutic purposes is still being debated.
Here are a few ways Sanford-Burnham researchers are advancing personalized medicine and disease in a dish:
- Dr. Daniel Kelly’s group is evaluating several genetic markers in diabetic and non-diabetic people who have experienced heart attacks. Heart patients are traditionally treated with a type of drug called a beta-blocker. However, initial results reveal that diabetic patients with different variations in a particular gene respond differently to beta-blockers. For some people, beta blockers are extra beneficial, while those with a different genetic makeup are more likely to experience harmful side effects from the drug. Read more >>
- In cancer research, personalized medicine describes the notion that every tumor is different. If doctors could simply profile every person’s tumor on a genomic, proteomic and metabolomic level, they’d be better able to develop more appropriate and less toxic therapies. Dr. Robert Wechsler-Reya and his team are developing personalized mouse models for a childhood brain tumor called medulloblastoma. Personalized mouse models allow the tumor cells to maintain their biochemical properties, allowing researchers to study the role stem cells play in brain development and tumor formation in a real-life context. Read more >>
- Dr. Jeff Price and Dr. Mark Mercola’s laboratories are using disease in a dish to screen drugs for cardiotoxicity – a side effect that harms the heart (a common reason for pulling pain and weight loss medications off the shelf). This technique allows them to scale up their efforts, using many different combinations of cells and drugs. They can actually see heart cells beating in a dish and, using advanced video technology, they can record and measure contractions before and after drug treatment. This information could eventually be used to more quickly inform the selection and development of safer drugs, ultimately saving many lives. Read more >>
- Reprogramming differentiated adult cells into iPS cells to recreate a disease in a dish requires so many steps and so much time that the efficiency rate is very low – you might end up with only a few iPS cells, even if you started with a million skin cells. Dr. Tariq Rana and his team are improving the process by exploiting microRNAs (miRNAs) during the reprogramming stage as a skin cell makes the transition to an iPS cell. Their findings demonstrate that miRNAs are great enhancers for iPS cell generation. Read more >>