From bench to bedside: a Q&A with stem cell expert Jan Nolta

At CIRM we are privileged to work with many remarkable people who combine brilliance, compassion and commitment to their search for new therapies to help people in need. One of those who certainly fits that description is UC Davis’ Jan Nolta.

This week the UC Davis Newsroom posted a great interview with Jan. Rather than try and summarize what she says I thought it would be better to let her talk for herself.

Jan Nolta
Jan Nolta

Talking research, unscrupulous clinics, and sustaining the momentum

(SACRAMENTO) —

In 2007, Jan Nolta returned to Northern California from St. Louis to lead what was at the time UC Davis’ brand-new stem cell program. As director of the UC Davis Stem Cell Program and the Institute for Regenerative Cures, she has overseen the opening of the institute, more than $140 million in research grants, and dozens upon dozens of research studies. She recently sat down to answer some questions about regenerative medicine and all the work taking place at UC Davis Health.

Q: Turning stem cells into cures has been your mission and mantra since you founded the program. Can you give us some examples of the most promising research?

I am so excited about our research. We have about 20 different disease-focused teams. That includes physicians, nurses, health care staff, researchers and faculty members, all working to go from the laboratory bench to patient’s bedside with therapies.

Perhaps the most promising and exciting research right now comes from combining blood-forming

stem cells with gene therapy. We’re working in about eight areas right now, and the first cure, something that we definitely can call a stem cell “cure,” is coming from this combined approach.

Soon, doctors will be able to prescribe this type of stem cell therapy. Patients will use their own bone marrow or umbilical cord stem cells. Teams such as ours, working in good manufacturing practice facilities, will make vectors, essentially “biological delivery vehicles,” carrying a good copy of the broken gene. They will be reinserted into a patient’s cells and then infused back into the patient, much like a bone marrow transplant.

“Perhaps the most promising and exciting research right now comes from combining blood-forming stem cells with gene therapy.”

Along with treating the famous bubble baby disease, where I had started my career, this approach looks very promising for sickle cell anemia. We’re hoping to use it to treat several different inherited metabolic diseases. These are conditions characterized by an abnormal build-up of toxic materials in the body’s cells. They interfere with organ and brain function. It’s caused by just a single enzyme. Using the combined stem cell gene therapy, we can effectively put a good copy of the gene for that enzyme back into a patient’s bone marrow stem cells. Then we do a bone marrow transplantation and bring back a person’s normal functioning cells.

The beauty of this therapy is that it can work for the lifetime of a patient. All of the blood cells circulating in a person’s system would be repaired. It’s the number one stem cell cure happening right now. Plus, it’s a therapy that won’t be rejected. These are a patient’s own stem cells. It is just one type of stem cell, and the first that’s being commercialized to change cells throughout the body.

Q: Let’s step back for a moment. In 2004, voters approved Proposition 71. It has funded a majority of the stem cell research here at UC Davis and throughout California. What’s been the impact of that ballot measure and how is it benefiting patients?

We have learned so much about different types of stem cells, and which stem cell will be most appropriate to treat each type of disease. That’s huge. We had to first do that before being able to start actual stem cell therapies. CIRM [California Institute for Regenerative Medicine] has funded Alpha Stem Cell Clinics. We have one of them here at UC Davis and there are only five in the entire state. These are clinics where the patients can go for high-quality clinical stem cell trials approved by the FDA [U.S. Food and Drug Administration]. They don’t need to go to “unapproved clinics” and spend a lot of money. And they actually shouldn’t.

“By the end of this year, we’ll have 50 clinical trials.”

By the end of this year, we’ll have 50 clinical trials [here at UC Davis Health]. There are that many in the works.

Our Alpha Clinic is right next to the hospital. It’s where we’ll be delivering a lot of the immunotherapies, gene therapies and other treatments. In fact, I might even get to personally deliver stem cells to the operating room for a patient. It will be for a clinical trial involving people who have broken their hip. It’s exciting because it feels full circle, from working in the laboratory to bringing stem cells right to the patient’s bedside.

We have ongoing clinical trials for critical limb ischemia, leukemia and, as I mentioned, sickle cell disease. Our disease teams are conducting stem cell clinical trials targeting sarcoma, cellular carcinoma, and treatments for dysphasia [a swallowing disorder], retinopathy [eye condition], Duchenne muscular dystrophy and HIV. It’s all in the works here at UC Davis Health.

There’s also great potential for therapies to help with renal disease and kidney transplants. The latter is really exciting because it’s like a mini bone marrow transplant. A kidney recipient would also get some blood-forming stem cells from the kidney donor so that they can better accept the organ and not reject it. It’s a type of stem cell therapy that could help address the burden of being on a lifelong regime of immunosuppressant drugs after transplantation.

Q: You and your colleagues get calls from family members and patients all the time. They frequently ask about stem cell “miracle” cures. What should people know about unproven treatments and unregulated stem cell clinics?

That’s a great question.The number one rule is that if you’re asked to pay money for a stem cell treatment, don’t do it. It’s a big red flag.

When it comes to advertised therapies: “The number one rule is that if you’re asked to pay money for a stem cell treatment, don’t do it. It’s a big red flag.”

Unfortunately, there are unscrupulous people out there in “unapproved clinics” who prey on desperate people. What they are delivering are probably not even stem cells. They might inject you with your own fat cells, which contain very few stem cells. Or they might use treatments that are not matched to the patient and will be immediately rejected. That’s dangerous. The FDA is shutting these unregulated clinics down one at a time. But it’s like “whack-a-mole”: shut one down and another one pops right up.

On the other hand, the Alpha Clinic is part of our mission is to help the public get to the right therapy, treatment or clinical trial. The big difference between those who make patients pay huge sums of money for unregulated and unproven treatments and UC Davis is that we’re actually using stem cells. We produce them in rigorously regulated cleanroom facilities. They are certified to contain at least 99% stem cells.

Patients and family members can always call us here. We can refer them to a genuine and approved clinical trial. If you don’t get stem cells at the beginning [of the clinical trial] because you’re part of the placebo group, you can get them later. So it’s not risky. The placebo is just saline. I know people are very, very desperate. But there are no miracle cures…yet. Clinical trials, approved by the FDA, are the only way we’re going to develop effective treatments and cures.

Q: Scientific breakthroughs take a lot of patience and time. How do you and your colleagues measure progress and stay motivated?   

Motivation?  “It’s all for the patients.”

It’s all for the patients. There are not good therapies yet for many disorders. But we’re developing them. Every day brings a triumph. Measuring progress means treating a patient in a clinical trial, or developing something in the laboratory, or getting FDA approval. The big one will be getting biological license approval from the FDA, which means a doctor can prescribe a stem cell or gene therapy treatment. Then it can be covered by a patient’s health insurance.

I’m a cancer survivor myself, and I’m also a heart patient. Our amazing team here at UC Davis has kept me alive and in great health. So I understand it from both sides. I understand the desperation of “Where do I go?” and “What do I do right now?” questions. I also understand the science side of things. Progress can feel very, very slow. But everything we do here at the Institute for Regenerative Cures is done with patients in mind, and safety.

We know that each day is so important when you’re watching a loved one suffer. We attend patient events and are part of things like Facebook groups, where people really pour their hearts out. We say to ourselves, “Okay, we must work harder and faster.” That’s our motivation: It’s all the patients and families that we’re going to help who keep us working hard.

Regulated, reputable, and reliable – distinguishing legitimate clinical trials from predatory clinics

Here at CIRM, we get calls every day from patients asking us if there are any trials or therapies available to treat their illness or an illness affecting a loved one. Unfortunately, there are some predatory clinics that try to take advantage of this desperation by advertising unproven and unregulated treatments for a wide range of diseases such as Diabetes, Alzheimer’s, Parkinson’s, Amyotrophic Lateral Sclerosis (ALS), and Multiple Sclerosis (MS).

A recent article in the Los Angeles Times describes how one of these predatory stem cell clinics is in a class action lawsuit related to false advertising of 100% patient satisfaction. Patients were led to believe that this percentage was related to the effectiveness of the treatment, when in fact it had to do with satisfaction related to hospitality, hotel stay, and customer service. These kinds of deceptive tactics are commonplace for sham clinics and are used to convince people to pay tens of thousands of dollars for sham treatments.

But how can a patient or loved one distinguish a legitimate clinical trial or treatment from those being offered by predatory clinics? We have established the “fundamental three R’s” to help in making this distinction.

REGULATED

The United States Food and Drug Administration (FDA) has a regulated process that it uses in evaluating potential treatments from researchers seeking approval to test these in a clinical trial setting.  This includes extensive reviews by scientific peers in the community that are well informed on specific disease areas. Those that adhere to these regulations get an FDA seal of approval and are subject to extensive oversight to protect patients participating in this trial. Additionally, these regulations ensure that the potential treatments are properly evaluated for effectiveness. The 55 clinical trials that we have currently funded as well as the clinical trials being conducted in our Alpha Stem Cell Clinic Network all have this FDA seal of approval. In contrast to this, the treatments offered at predatory clinics have not gone through the rigorous standards necessary to obtain FDA approval.

REPUTABLE

We have partnered with reputable institutions to carry out the clinical trials we have funded and establish our Alpha Stem Cell Clinic Network. These are institutions that adhere to the highest scientific standards necessary to effectively evaluate potential treatments and communicate these results with extreme accuracy. These institutions have expert scientists, doctors, and nurses in the field and adhere to rigorous standards that have earned these institutions a positive reputation for carrying out their work.  The sites for the Alpha Stem Cell Clinic Network include City of Hope, UCSF, UC San Diego, UCLA, UC Davis, and UC Irvine.  In regards to the clinical trials we have directly funded, we have collaborated with other prestigious institutions such as Stanford and USC.  All these institutions have a reputation for being respected by established societies and other professionals in the field. The reputation that predatory clinics have garnered from patients, scientists, and established doctors has been a negative one. An article published in The New York Times has described the tactics used by these predatory clinics as unethical and their therapies have often been shown to be ineffective.

RELIABLE

The clinical trials we fund and those offered at our Alpha Stem Cell Clinic Network are reliable because they are trusted by patients, patient advocacy groups, and other experts in the field of regenerative medicine. A part of being reliable involves having extensive expertise and training to properly evaluate and administer treatments in a clinical trial setting. The doctors, nurses, and other experts involved in clinical trials given the go-ahead by the FDA have extensive training to carry out these trials.  These credentialed specialists are able to administer high quality clinical care to patients.  In a sharp contrast to this, an article published in Reuters showed that predatory clinics not only administer unapproved stem cell treatments to patients, but they use doctors that have not received training related to the services they provide.

Whenever you are looking at a potential clinical trial or treatment for yourself or a loved one, just remember the 3 R’s we have laid out in this blog.

Regulated, reputable, and reliable.