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2014

Dr. Jana Portnow, City of Hope

City of Hope

Brain Cancer

Grant Amount: $66,000

“My first trial patient – Jenny, from Seattle, had glioblastoma. She didn’t think the treatment could help her, but she wanted to give hope to other patients who could be helped. Jenny died two months after treatment, but her involvement kick-started the study. News coverage of her situation drew participant interest from all over.” Dr. Jana Portnow

Treating brain tumors with chemotherapy presents special challenges because the blood-brain barrier restricts which chemicals can reach the vast majority of our brain cells. The barrier consists of a network of junctions between our capillaries and the brain cells that they supply with blood. These junctions possess an electrical resistance that permits water, certain gases, lipids, glucose and some amino acids to pass through, but other substances are blocked – creating a significant hurdle to delivering medication.

Phase ONE Foundation immediately saw the potential benefits of Dr. Jana Portnow’s proposal to use neural stem cells to circumvent the blood-brain barrier and deliver targeted chemotherapy to tumor sites.

This novel treatment involves surgically infusing specially designed stem cells into the brain, and then waiting one week for them to migrate to the tumor sites – to which they are naturally drawn. The patient is then given an anti-fungal compound that is known to be able to pass through the blood-brain barrier. Once it reaches the tumor, the stem cells convert the compound into a chemotherapy drug that acts against the tumor.

Stem cell therapies have typically been used to replace or regenerate damaged tissue; in this case, they are creating a gateway to cellular repair that is unique to the treatment needs of the brain.

The trial began in 2010 with the first-in-human study and concluded in January of 2013 with 15 enrolled patients. In addition to the groundbreaking work being conducted within the brain, Dr. Portnow’s team also tested whether they could use a microcatheter to provide a continuous supply of stem cells to the brain in order to eliminate the need for a surgical infusion each time. Indeed, their work with the microcatheters was able to document proof-of-concept.

“I got involved in research because I can’t imagine how any oncologist cannot be involved in it, given the difficulties you witness during treatment, said Dr. Portnow. “I intended to be an oncologist but, after working with so many patients, I realized that I had to try to do more to help them.”

Work continues in this promising field, with the intent that another trial group will soon be able to undergo treatment with microcatheter delivery of stem cells.

PHASE ONE’s support for Dr. Portnow’s stem cell therapy is dramatically improving our arsenal of responses to this form of cancer with a therapy that seems to have no significant side effects. This treatment can work alongside traditional therapies as well, and provide an additional layer of defense.

In 2014, brain cancer was the focus of PHASE ONE’s award-winning video – EveryONE Can Make a Difference. After its premiere, guests at the Gala were able to donate directly to Dr. Portnow’s work using their smartphones — an addition $67,000.

Funding is limited for this area of study, so Phase ONE Foundation’s support has been pivotal in advancing its progress in recent years. Without these resources – and the additional funds that have been leveraged with them, stem cell treatment of brain tumors might not have emerged as the promising therapy that it is.