Eyeing a future of improved patient outcomes, the Medical College of Wisconsin is working to advance the prevention, diagnosis and treatment options of personalized medicine. Personalized medicine, sometimes called precision medicine, is an emerging practice that uses an individual’s genetic profile to guide decisions about prevention, diagnosis and treatment of diseases such as cancer. MCW
Eyeing a future of improved patient outcomes, the Medical College of Wisconsin is working to advance the prevention, diagnosis and treatment options of personalized medicine.
Personalized medicine, sometimes called precision medicine, is an emerging practice that uses an individual’s genetic profile to guide decisions about prevention, diagnosis and treatment of diseases such as cancer.
MCW is part of the Wisconsin Biohealth Tech Hub’s consortium of 18 member companies or institutions, which include BioForward Wisconsin, GE HealthCare, Exact Sciences, the University of Wisconsin-Madison, Rockwell Automation and more. The tech hub recently received $49 million in federal funding to support six interconnected regional projects aimed at advancing biotechnology in precision and predictive medicine.
[caption id="attachment_605478" align="alignleft" width="300"] Mara Lord[/caption]
In collaboration with biohealth tech hub partners, the MCW aims to advance two forms of personalized medicine: theranostics and gene therapy. Both are emerging methods of treatment that advance personalized medicine, said Mara Lord, chief strategy and growth officer at MCW.
Theranostics combines therapy and diagnostics to treat cancer. It’s a $2.3 billion industry today and is expected to reach $4.3 billion by 2028, Lord said. Gene therapy, which edits an individual’s genes to treat diseases, is a $3.3 billion industry today but will grow to $24.6 billion by 2033, Lord said.
MCW is also working with the biohealth tech hub to advance multi-cancer early detection tests, which are related to personalized medicine as a prevention tool allowing individuals to receive different diagnoses and treatments earlier. In a single blood draw, the tests can detect more than 70 cancers, including pancreatic, prostate, kidney, lung, breast, skin, ovarian and liver cancers, Lord said.
“Personalized medicine is global already,” Lord said. “It has been emerging over the past several years. Now we’re creating more options for prevention, diagnoses and treatment.”
Combining therapy and diagnostics
Radiation oncologist Dr. William Hall is a professor of radiation oncology and surgery and chair of the radiation oncology department at MCW, and the inaugural Bob Uecker Endowed Chair in Pancreatic Cancer Research. He sees theranostics as one of the most promising ways to improve outcomes for patients who have cancer.
But how does theranostics work?
The diagnostics component of theranostics involves diagnosing and staging a patient’s cancer with personalized PET scans. A PET scan looks for cancer cells throughout a person’s body, typically by identifying cells that are using higher amounts of sugar. Theranostics takes that a step further, Hall said. Instead of only looking for cells that use more sugar, theranostics involves looking for particular characteristics and types of protein structures on the outside of the cancer cell that it can attach to and light up.
“It’s a very precise way of diagnosing a patient with cancer, and that’s the diagnostic component,” Hall said. “The reason that’s so powerful is because we want to know exactly where the patient’s cancer cells are, and if we know exactly where the patient’s cancer cells are, then we can therapeutically attack and target those cancer cells using either surgery or radiation or ablation techniques.”
The therapy component comes in once the cancer cells have been located. Imaging technology can be used to determine whether the cancer has spread from the original tumor, and then the cancer cells can be treated with a therapeutic dose of radiation designed to eliminate them. The therapeutic is “tailored” to an individual patient’s cancer, Hall said.
Today, theranostics is typically used to treat patients with prostate cancer and some types of neuroendocrine tumors. Prostate cancer is the third most common cancer in Wisconsin, according to the Wisconsin Cancer Collaborative. It’s also one of the most common cancers among men. From 2017 to 2021, there were 3,304 new cases of prostate cancer reported in Milwaukee County, according to Centers for Disease Control and Prevention data.
In five to ten years, theranostics may be used to treat a variety of other cancers. “The sky’s the limit right now,” Hall said, but some possibilities include primary brain tumors, breast cancer, lung cancer, pancreatic cancer and tumors in the liver.
“The excitement around theranostics really comes from the fact that there are many other types of cancers that will be also treatable with theranostics in the future,” Hall said. “That’s where our team and the group here and many people internationally are getting very excited about this as a whole new category of cancer therapy.”
Driving ‘clinical innovation’
There are currently 53 ongoing theranostics clinical trials across the U.S., and some of them are happening at MCW. So far, MCW has offered 12 theranostics clinical trials to date. More are “coming down the pipeline,” Hall said.
Theranostics clinical trials require tremendous infrastructure to be implemented, beyond the standard pharmaceutical trial, Hall said. Patients who undergo theranostics treatment are temporarily radioactive afterwards, so they require special shielding and precautions to prevent exposing others to radioactive material.
The equipment needed to make the radioactive agents that are used to kill cancer cells is highly expensive. The agents must be made on-site using a cyclotron, a type of particle accelerator, in close proximity to the patient because the radioactive decay has a half-life.
“If it’s given too long after it’s made, it won’t be effective,” Hall said. “For that reason, there’s sort of a challenge there, where these types of therapeutics require new facilities that some centers may not have.”
GE HealthCare provides the isotopes, imaging, informatics and molecular imaging agents to clinicians practicing and working to advance theranostics treatment, said Jay Hill, vice president of advanced technology at GE HealthCare.
The limited access, paired with the significant equipment and infrastructure costs, contribute to the high cost of theranostics treatments. Each treatment can be about $50,000, and patients typically receive three treatments, Lord said.
“We’re going to need to first have some of the institutional infrastructure developed, and really modeled out and enhanced before we can start substantially ramping up our pipeline of these clinical trials,” Hall said. “But it’s definitely something very futuristic looking that we hope to be doing in, say, the next three to five years.”
MCW’s departments of radiation oncology and nuclear medicine are working in close collaboration to improve theranostics treatment. They’ve been working on dosimetry, where they map the radiation dose distribution for patients undergoing radiation therapy, Hall said. This allows for more precise theranostic administration adjusted to a person’s anatomy.
“Bringing that research together with some of the clinical innovation is one of the most powerful ways to bring highly cutting edge, very effective therapies to patients,” Hall said. “By the time a cancer therapy gets to be a standard of care, it has already been iterated on tremendously. There’s a certain sense of, I would say less cutting-edge innovation when something is a standard of care than when something is being looked at in a research capacity. We really pride ourselves in bringing tremendously innovative therapies to patients because of our academic missions and because of our research that we conduct. It enables us to bring these therapies to patients in ways that have not been done before, which we’re very, very excited about.”
Theranostics as another standard care option
Once more theranostics clinical trials have proven effective with positive and improved outcomes, patients may see these theranostics treatments become available as more accessible standard care options, Hall said.
Theranostics is a new category of cancer therapy, so it would not replace chemotherapy, immunotherapy, surgery or external beam radiation. This is powerful because it will give more treatment options to patients in Milwaukee and across the state, Hall said.
“Milwaukee has a lot of patients with cancer, unfortunately, and the number of patients with cancer is significantly increasing as the population ages,” Hall said. “Having access to incredibly cutting-edge therapeutics is critical for the Milwaukee population.”
Having more cancer treatment options “brings us closer to a durable, long-term control” of patients’ cancers, Hall said. It will continue to be challenging to find one agent that cures cancer, so patients need therapies to control their cancers for a long time – enabling them “to live normal lives with normal quality of life,” he said.
“We’re trying to change the mindset of cancer,” Hall said. “We’re trying to make it more like a chronic disease, something that someone has, sort of like someone has diabetes, someone has high blood pressure. Some of them may have high cholesterol. But it’s very effectively treated and controlled.”
Gene therapy and CRISPR
[caption id="attachment_610677" align="alignleft" width="300"] Chun Liu[/caption]
Chun Liu, Ph.D., assistant professor of physiology and medicine at MCW, runs a research lab that utilizes CRISPR, a technology that can be used to precisely modify DNA, to develop new treatments for heart disease. His lab’s mission is to “reprogram cells to repair damaged heart tissue or even prevent cardiovascular diseases before they strike.”
Gene therapy can involve using CRISPR as a tool to edit a patient’s genes as a form of treatment. A person’s genes can be edited to combat diseases in three key ways: replacing a gene with a healthier copy, inactivating a disease-causing gene, or introducing a new gene to help fight a disease.
In 2023, the U.S. Food and Drug Administration approved the first gene therapies to treat sickle cell disease. One of the approved therapies, called Casgevy, utilizes CRISPR. Casgevy, a one-time treatment, also came with a $2.2 million sticker price, according to a Business Insider report.
Some gene therapy treatments cost less than Casgevy, but due to the high cost associated with gene therapy treatments, many insurance companies don’t cover them, Liu said.
MCW’s cancer and blood centers participate in and host gene therapy clinical trials addressing sickle cell disease. MCW has 51 active cellular and gene therapy research studies with human participants. Three of these studies involve CRISPR.
MCW has ongoing clinical trials involving the use of CRISPR in CAR T-cell therapy. In this case, gene editing is used to target and eliminate cancer cells. Liu said that in the next year or two, he anticipates there will be advancements in utilizing CRISPR as a part of these cancer treatments.
“We’re not only doing gene editing on one gene,” Liu said. “New technology can do what we call CRISPR-based screening. You can screen all the genes across the genome, every gene in our genome.”
While the future of gene therapy is bright, there needs to be more clinical trials along with the required large-scale manufacturing capabilities to support the treatments, Liu said.
More clinical trials can help address a concern surrounding CRISPR-based gene therapy that there may be some “off target,” Liu said. This means there’s a chance that when aiming for a specific gene to edit, other genes could be unintentionally edited in the process. Years of continued development can improve this.
If safety concerns are addressed and the cost of treatment decreases, CRISPR screening could be adapted to treat a wider range of diseases, Liu said.
“Then any type of disease can have a quick cure in the near future,” Liu said.
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