Not so long ago using the words ‘HIV/AIDS’ and ‘cure’ in the same sentence would have been considered inappropriate, even reckless. Although there were many antiretroviral medications that were effective at helping control the virus, there was nothing that was even remotely close to a cure on the horizon. And those therapies that had been approved were not always readily available, even in the U.S., let alone in the hardest hit countries in Africa.

Today the picture looks quite different. Cure is no longer just a distant dream, it’s a goal. CIRM has two projects that we are funding in clinical trials – one led by Calimmune Inc. and one by City of Hope and Sangamo BioSciences – whose ultimate goal is to replicate the experience of the “Berlin patient” and effectively cure people with the virus.

And we are not alone, the National Institute for Allergy and Infectious Diseases (NIAID) supports a large portfolio of investigator-initiated grants in HIV cure research, including programs to identify where HIV hides, known as the HIV reservoir, and to determine how these hideouts are established and maintained and then to get rid of them.

With this progress in mind we are partnering with the AIDS Project Los Angeles (APLA), Being Alive and the University of Southern California (USC) to host an HIV/AIDS Town Hall event called “Countdown to a Cure: California Leads the Way.”

The goal of the event is to bring together members of the HIV community and leading researchers in the field to talk about the clinical trials that are underway now, and the other approaches that are being tried to cure AIDS.

Speakers at the Town Hall are Dr. Paula Cannon, USC; Dr. David Hardy, Calimmune; Dr. John Zaia, City of Hope; and Dr. Dale Ando, Sangamo BioSciences. The conversation will be moderated by Jeff Sheehy. Jeff is not only the CIRM Board Patient Advocate member for HIV/AIDS, he’s also a long time community activist in the fight against the virus. You can hear Jeff talk about his commitment to the cause in this video.

The event is on Thursday, July 30^th from 5.30p to 8.30p and we’re providing food and refreshments. The location is Fiesta Hall, Plummer Park, 7377 Santa Monica Boulevard, West Hollywood, Los Angeles. And, of course, it’s free.

If you are interested in joining us and being part of the discussion please RSVP to: daniel@beingalivela.org or call 323-874-4322.

We look forward to seeing you there.

Some things never get old. Like watching the sunset over the Grand Canyon. Listening to a baby laugh. Watching the San Francisco Giants win the baseball World Series. Now you can add to that list learning that one of the clinical trials we are funding has just treated their first patient.

The latest to join that growing list is Caladrius Biosciences (previously called NeoStem). We recently awarded them $17.7 million to carry out a Phase 3 metastatic melanoma clinical trial targeting cancer stem cells. These cells are believed to be able to survive chemotherapy and other cancer-targeting treatments, and can cause a relapse by enabling tumors to grow and spread.

Caladrius’ approach is a personalized one. They use the patient’s own tumor cells to create a therapeutic vaccine called (for now at least) CLBS20. It’s designed to engage the patient’s own immune system and destroy the cancer.

This first patient was treated at Thomas Jefferson University Hospital in Philadelphia. Altogether Caladrius hopes to enroll some 250 patients at more than 40 sites worldwide, for the trial. Seven of those sites are here in California; that’s the portion of the project we are funding.

Because this is a randomized, double blind study it’s not known if the patient was treated with CLBS20 or a placebo. But in a news release Dr. David J. Mazzo, CEO of Caladrius Biosciences, says it’s a big first step:

“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 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.”

In the earlier Phase 2 trial, 72 percent of those who got the therapy were still alive after two years, compared to 31 percent of people who got a placebo therapy. There was another bonus for patients; the treatment was well-tolerated with few side effects, the most common being irritation and a reaction at the site of the injection.

There’s a big need for this approach. In 2014 there were approximately 20,000 new cases of metastatic melanoma and nearly 10,000 deaths. It usually causes death within one to two years and only 10 to 15 percent of patients survive five years.

Here’s where to go if you would like more information on the Intus Study or you can also visit the NIH clinical trials site.

Adult nerve stem cells fall in the category of allusive creatures. A few scientists still question their existence and most suggest they exist in small numbers only in one or two locations in the adult brain. In any case, all agree they are not particularly good at the normal function of stem cells—making repairs to their surrounding tissue.

A research team at the University of California, San Francisco, recently published results providing two reasons why adult nerve stem cells are not very robust. First they don’t self-renew—make more copies of themselves—on a regular basis.

While we have many types of nerves in our brains, our adult stem cells seem preprogrammed to form certain ones.

Second, the ones you were born with were preprogrammed before birth to become only a narrow subset of the many nerves we need for a fully functioning brain.

Working in mice, the team led by Arturo Alvarez-Buylla found several types of stem cells on the walls of cavities in the brain and each was pre-programmed to be “progenitors” for a specific subset of nerves. Like progenitors appeared to be lumped together by location and the team also tracked the time during embryo development when these destiny designations are made.

These results could make folks reconsider how they might use adult nerve stem cells for therapy. Alvarez-Buylla explained in a UCSF press release picked up by ScienceNewsline:

“It may be unwelcome news for those who thought of adult neural stem cells as having a wide potential for neural repair. Instead, it looks as if that potential is narrowed down very early during embryonic development. It’s almost as if the embryo is planning for the future.”

He went on to argue that the study points out the critical importance of understanding how stem cells develop and change in the embryo because that knowledge will guide how we use the various stem cells in therapy.

CIRM did not fund this study, but we do fund work in the Alvarez-Buylla lab that seeks to create nerve cells that can be implanted into people with diseases like epilepsy that result from an imbalance between different types of nerves.

It’s hard to get where you want to go without a map. Even if you have a pretty good idea of where you are heading it’s all too easy to get sidetracked or take a wrong turn. Having a good map helps you stay on course.

Charting a course for success

That’s why we are creating our own map, to help us reach our goal, of accelerating stem cell therapies to patients with unmet medical needs.

We’re putting together a new Strategic Plan, something that will help shape our future as we head into our second decade. The idea is simple, how can we best use the money we have left (almost one billion dollars) and all our other resources.

To do that we’re asking the usual suspects for their thoughts and ideas, but we’re also asking some unusual suspects, in fact, we’re asking anyone who is interested to help us develop the plan.

As our President and CEO, Dr. C. Randal Mills, said in a news release LINK:

“No one has a monopoly on good ideas, that’s why we want to hear from a diverse group of people, scientists and non-scientists alike, to learn what they think about how we should best use our money, resources, and expertise to reach our goal. This new Strategic Plan will help create a clear vision for how we move forward, one that sets priorities and an actionable approach to accomplish our mission.”

Anyone wishing to add their voice to those helping us develop the plan can take the online anonymous survey. The deadline is the end of the day Friday, June 26^th.

YOU CAN FIND THE SURVEY HERE

As the Chair of our governing Board, Jonathan Thomas, Ph.D., J.D., says:

“We are a state agency. We were created by the people of California and we answer to the people of California. It makes sense that for something this important, a Strategic Plan that will help shape our future for years to come, that we ask the people of California for their thoughts and suggestions.”

Science is filled with uncertainty. Even the most promising therapeutic approach can take a wrong turn. Having a clear road map, a well thought out Strategic Plan, is no guarantee of success, but it certainly means we’ll have a much better idea of how to get where we want to go.

One of the fascinating things about stem cell research is how quickly the field is evolving. It seems like every other day a new study is published that highlights a new discovery that makes us stop and think how this new knowledge affects our understanding of stem cells and the diseases we are trying to treat.

The latest example came this week with research from Canada identifying a new kind of stem cell population found in the colon that can lead to cancer growth.

The study, published in the journal Cell Stem Cell,  is important because colon cancer is the third most commonly diagnosed cancer and the second leading cause of cancer death in both men and women, claiming around 50,000 lives in the U.S. alone every year.

Stem cells are essential for helping replace the lining of our colon and intestine every three or four days. In the intestine there are two kinds of stem cells, a rapidly recycling one called Lgr5+ and a slower one. However, scientists had only been able to identify Lgr5+ stem cells in the colon. Because this stem cell type is sensitive to radiation physicians believed that radiation therapy would be effective against colon cancer.

Now, researchers at Lawson Health Research Institute in Ontario, Canada, have identified another stem cell in the colon, one that is both long-lived and radiation resistant.

They also found that this new stem cell population can not only give rise to tumors in the colon, it can also help sustain and support the growth of the cancer.

In a news release Dr. Samuel Asfaha, a clinician-scientist at Lawson and the lead author of the study, says this new piece of information gives them vital new information in fighting the cancer:

 “The identification of more than one stem cell pool in the colon has proven challenging. These findings are exciting, as we have identified an important new target for cancer therapy. It is also proof that more than one stem cell can give rise to and sustain tumors, telling us that our cancer therapy needs to target more than one stem cell pool.”

Asfaha says knowing that there is a pool of stem cells that don’t respond to radiation means researchers must now look for new, more effective ways of tackling them, so we are better able to help patients with colon cancer.

CIRM is funding a number of therapies that target solid tumor cancers, the kind that includes colon cancer. One, run by Dr. Dennis Slamon of University College, Los Angeles, is now in clinical trials. You can read about that work here.