Sometimes it feels like finding a cure is the easy part; getting it past all the hurdles it must overcome to reach patients is just as big a challenge. Fortunately, many rather brilliant minds are hard at work finding the most effective ways to do just that.

Last week, at the grandly titled Second Annual Symposium of the CIRM Alpha Stem Cell Clinics Network, some of those minds gathered to talk about the issues around bringing stem cell therapies to the people who need them, the patients.

Alpha Clinics

The Alpha Clinics Network aims to speed the development and delivery of stem cell therapies to patients. One major issue is cost: how much should a life‑changing, even life‑saving, treatment cost? For example, medications that cure Hepatitis C cost more than $80,000. So what would a cure for Severe Combined Immunodeficiency cost? CIRM-funded researchers have developed a cure for SCID, but only 40–100 newborns each year have the disease, meaning the high development cost falls on very few patients.

The same approach used to cure SCID could also cure sickle cell disease, which affects about 100,000 people in the U.S., most of them African American. As more people become eligible for treatment, should the cost drop, or will it stay the same to increase profits?

Jennifer Malin of UnitedHealthcare explained the questions involved in pricing a therapy. She also addressed the key issue of who should pay: patients, insurers, or the state? As she noted, a cure is useless if it’s priced so high that no one can access it.

Joseph Alvarnas, Director of Value‑based Analytics at City of Hope, said decisions about stem cell therapies must remain “mindful of economic reality and inequality” to ensure access for everyone, not just the wealthy.

“Remember, the decisions we make now will influence not just the lives of those with us today but also the lives of all those to come.”

Clinical Trials

The regulatory process for cell therapies is slow and complex, even in the best circumstances. Many researchers have little experience with it, which helps explain why cell therapies take more than eight years, on average, to reach a clinical trial. Traditional medications reach trials in about 3.2 years.

Sunil Kadam of QuintilesIMS outlined the skills needed to navigate this pathway. QuintilesIMS partners with CIRM to run the Stem Cell Center, which helps researchers apply for and conduct clinical trials and provides guidance to keep promising work on track.

At the heart of the conference were patients and advocates. They offered inspiration and a clear reminder of why this work matters: finding treatments and cures for people in need.

The Alpha Clinic Network is only a few years old but already runs 35 clinical trials involving hundreds of patients. The conference focused on lessons learned and best practices so the number of trials and patients can continue to grow.

CIRM Board

The CIRM Board is also helping speed progress by approving funding for up to two additional Alpha Clinic sites. The deadline to apply is May 15, and details are available on our funding page.

Since joining CIRM, I have attended many conferences, but this one was the best. It brought together experts from across the field and offered a clear view of where we stand and where we’re headed. The talks were engaging and inspiring.

Kristin Macdonald was left legally blind by retinitis pigmentosa, a rare disease that destroys vision. A few years ago, she became the first person treated with a CIRM‑funded therapy aimed at restoring sight. She says it helps. For years she lived in darkness, and while she still cannot see clearly, she can now see light. She says stepping out of darkness has changed her world.

In future years, the Alpha Clinics Network hopes to bring similar hope—and more—to many people in need.

In the years to come, the Alpha Clinics Network hopes to be able to do the same, and much more, for many more people in need.

To read more about the Alpha Clinics Meeting, check out our Twitter Moments.