CTSI Pilot Awardee Taps the Power of Vitamin C to Treat Blood Cancers

Published: 06-03-2021

Vitamin C, an essential dietary nutrient for humans, has been touted for its immune boosting properties for more than half a century. More recently, research has revealed its greater potential as a treatment for blood cancers.

In her ongoing research, Miami Clinical and Translational Science Institute (CTSI) Pilot Award recipient and Principal Investigator Luisa Cimmino, Ph.D., explores the potential of Vitamin C to combat blood cancers such as leukemia.

Dr. Cimmino, assistant professor at the University of Miami Miller School of Medicine, found that Vitamin C restores the activity of TET2 proteins, enzymes that play an important role in regulating the production of blood cells. Dr. Cimmino says, “TET2 enzymes lose their activity in leukemia patients, which is shown to drive disease progression. You can get a poor prognosis if you have a mutation in this particular gene.”

According to Dr. Cimmino, TET2 depends on Vitamin C for its optimal activity. Vitamin C is a “direct activator” of the proteins that help fight cancers including leukemia, she says.

While there are limits to the amount of Vitamin C that can be absorbed by the body when ingested orally, high doses of the vitamin delivered intravenously may have the therapeutic effect needed to slow cancer progression. As her research evolves, Dr. Cimmino hopes to pinpoint the exact dosage of Vitamin C required to improve cancer outcomes, and whether the vitamin is more powerful alone or in combination with other therapeutics.

“The motivation in my research is to try to find safe and effective novel epigenetic therapies to treat blood cancers and blood disorders,” she says.

Dr. Cimmino joined University of Miami in 2018 after being recruited by her current mentor, Maria Figueroa, M.D., associate professor and co-leader of the Cancer Epigenetics Research Program. “I knew her work first,” says Dr. Figueroa. “Then I met her, and I knew she had everything it takes to be successful in this field.”

In 2019, a CTSI pilot grant provided the seed money that allowed Dr. Cimmino to move her research forward. She used the grant to build her team, purchase materials, and collect the preliminary data required for subsequent grant applications.

Grants like those provided by CTSI help young investigators establish credibility and build a data set that often leads to larger funding opportunities in the future. “It’s this type of funding that allows talented investigators like Dr. Cimmino to get their research programs off the ground and to generate
data that then become the foundation for federal funding or state funding applications that are the cornerstone of our research programs,” says Dr. Figueroa.

Established researchers who want to explore a new direction or garner credibility in a new area can also benefit from these seed grants, says Dr. Figueroa. “These are incredibly valuable funding opportunities for the field in general.”

Dr. Cimmino says the support from CTSI was critical in setting the foundation for deeper exploration of the potential of Vitamin C to fight blood cancers. “Our initial goal was to determine whether we can restore activity of the TET2 enzyme as a therapy” she says. “The question now is, how do we maximize the ability of Vitamin C to do the job of restoring the enzyme and slowing the leukemia?”

This is the “epitome of what we want in cancer research,” says Dr. Figueroa. “She has identified a low cost, easy to access, easy to administer vitamin, and has found a way to introduce it into our established protocols.”

The impact of Vitamin C as a therapy for blood cancers might be most immediately felt by elderly patients, who tend to be less tolerant of harsher treatments like chemotherapy. Similarly, high doses of Vitamin C or novel combined therapies with Vitamin C as a component may be preferable options for patients with comorbidities like heart disease and digestive issues, says Dr. Figueroa.

“The important factor is that many patients can’t actually tolerate aggressive chemotherapy, so our goal is to model novel therapies that include vitamin C so that they will be less toxic and more tolerable than chemotherapy. Overall, we’re trying to improve the quality of life of these patients and also help cure the disease,” says Dr. Cimmino.

The research Dr. Cimmino conducted under the CTSI grant has led to further opportunities, including a more recent grant award from the Dresner Foundation. She hopes further research will reveal insights on the preventative power of Vitamin C, and perhaps even the discovery of a dose that can be given orally to prevent the development of blood cancers.

Dr. Cimmino’s research has also shown that patients with blood cancers are more likely to be deficient in Vitamin C, and she would like to find out more about the reasons and potential solutions for this discrepancy.

Even patients who are adverse to interventions would be open to treatment with Vitamin C, says Dr. Figueroa. “If you tell someone I’m going to give you Vitamin C and it’s going to prevent cancer, I can’t imagine anyone not taking it.”