Breaking down barriers to advance stem cell therapies – the view from the Vatican conference

Perry and the Pope

Pope Francis meets Katy Perry at the Unite to Cure conference at the Vatican

All hands were on deck at the “Unite to Cure” conference, organized by the Cura Foundation and the Vatican Pontifical Council,  and held at the Vatican on April 26-28. Religious leaders, scientists, physicians, philanthropists, industry leaders, government, academic leaders and members of the entertainment industry gathered to discuss how to improve human health and to increase access to relief of suffering for the under-served around the world.

Pope Francis spoke of “the great strides made by scientific research in discovering and making available new cures” but stressed that science also needs to have “an increased awareness of our ethical responsibility towards humanity and the environment in which we live.”

He talked of the importance of addressing the needs of children and young people, of helping the marginalized and those with rare, autoimmune and neurodegenerative diseases. He said:

“The problem of human suffering challenges us to create new means of interaction between individuals and institutions, breaking down barriers and working together to enhance patient care.”

So, it was appropriate that breaking down barriers and improving collaboration was the theme of a panel discussion featuring CIRM’s President and CEO, Maria Millan. She had been invited to attend the conference and participate on a panel focusing on “Public Private Partnerships to Accelerate Discoveries”.

As Dr. Millan put it, “Collaboration, communication, and alignment” is the winning formula for public/private partnerships.

She highlighted how CIRM exemplifies this new approach, how everything we do is focused on accelerating the field and that means partnering with the National Institutes of Health and the Food and Drug Administration to create new regulatory models. It also means working with scientists every step of the way; helping them prepare the best possible application for CIRM funding and, if they are approved, giving them the support they need to help them succeed.

It was a wide ranging, thoughtful, engaging conversation with David J. Mazzo, PhD, President & CEO of Caladrius Biosciences and David  Pearce, PhD, Executive VP for Research at Sanford Health. You can watch the discussion here.

People may find it surprising that government agencies, academic researchers and private companies can all collaborate effectively.  It is absolutely critical to do so in order to rapidly and safely advance transformative stem cell, gene and regenerative medicine to patients with unmet medical needs.  Pope Francis and the Pontifical Council at the Vatican certainly believe that collaboration is essential and the “Unite to Cure” Conference was a powerful demonstration of how important it is to work together for the future of humanity.

Helping patient’s fight back against deadliest form of skin cancer

Caladrius Biosciences has been funded by CIRM to conduct a Phase 3 clinical trial to treat the most severe form of skin cancer: metastatic melanoma. Metastatic melanoma is a disease with no effective treatment, only around 15 percent of people with it survive five years, and every year it claims an estimated 10,000 lives in the U.S.

The CIRM/Caladrius Clinical Advisory Panel meets to chart future of clinical trial

The CIRM/Caladrius Clinical Advisory Panel meets to chart future of clinical trial

The Caladrius team has developed an innovative cancer treatment that is designed to target the cells responsible for tumor growth and spread. These are called cancer stem cells or tumor-initiating cells. Cancer stem cells can spread in the body because they have the ability to evade the body’s immune defense and survive standard anti-cancer treatments such as chemotherapy. The aim of the Caladrius treatment is to train the body’s immune system to recognize the cancer stem cells and attack them.

Attacking the cancer

The treatment process involves taking a sample of a patient’s own tumor and, in a laboratory, isolating specific cells responsible for tumor growth . Cells from the patient’s blood, called “peripheral blood monocytes,” are also collected. The mononucleocytes are responsible for helping the body’s immune system fight disease. The tumor and blood cells (after maturation into dendritic cells) are then combined and incubated so that the patient’s immune cells become trained to recognize the cancer cells.

After the incubation period, the patient’s immune cells are injected back into their body where they generate an immune response to the cancer cells. The treatment is like a vaccine because it trains the body’s immune system to recognize and rapidly attack the source of disease.

Recruiting the patients

Caladrius has already dosed the first patient in the trial (which is double blinded so no one knows if the patient got the therapy or a placebo) and hopes to recruit 250 patients altogether.

This is the first Phase 3 trial that CIRM has funded so we’re obviously excited about its potential to help people battling this deadly disease.  In a recent news release David J. Mazzo, the CEO of Caladrius echoed this excitement, with a sense of cautious optimism:

“The dosing of the first patient in this Phase 3 trial is an important milestone for our Company and the timing underscores our focus on this program and our commitment to impeccable trial execution. We are delighted by the enthusiasm and productivity of the team at Jefferson University (where the patient was dosed) and other trial sites around the country and look forward to translating that into optimized patient enrollment and a rapid completion of the Phase 3 trial.”

And that’s the key now. They have the science. They have the funding. Now they need the patients. That’s why we are all working together to help Caladrius recruit patients as quickly as possible. Because their work perfectly reflects our mission of accelerating the development of stem cell therapies for patients with unmet medical needs.

You can learn more about what the study involves and who is eligible by clicking here.