#StoriesofHope

Stories of Hope: Stroke

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Six months after surviving a stroke, Sonia Olea wanted to die. Her right leg was weak, her right arm useless. She had trouble speaking and even small tasks were challenging. Just making a phone call was virtually impossible. One morning, she woke up with her arm pinned in an awkward, painful position. After finally repositioning it, she wanted to call her fiancé, but knew she couldn’t get the words out. That’s when it hit her.

Sonia has seen first hand how a stroke can rob you of even your most basic abilities.

Sonia has seen first hand how a stroke can rob you of even your most basic abilities.

“I thought, I’m only 32,” says Sonia. “How could this be happening to me?”

Nobody really had an answer. A stroke occurs when a blood clot blocks a vessel in the brain and cuts off blood flow. Brain cells begin to die within minutes when they are deprived of oxygen and nutrients. Stroke rates are on the rise for young adults for a variety of reasons but no one could pinpoint specifically what caused hers.

Slowly, Sonia fought back from her depression and realized she could do this. She would find a way to recover. Just one year later, she got a call from Stanford University; asking if she would be willing to participate in a cutting-edge, stem cell-based clinical trial.

Was she ever. The answer, says Sonia, was a no-brainer.

Rescuing Brain Cells
Led by CIRM grantee Gary Steinberg, M.D., Ph.D., chairman of the Department of Neurosurgery at Stanford School of Medicine, the early phase clinical trial tested the safety of transplanting bone marrow stem cells into the brain. It was a revolutionary approach.

“The old notion was that you couldn’t recover from a stroke after around three months,” says Steinberg. “At that point, the circuits were completely dead—and you couldn’t revive them.”

While this was partially true, it was thought that brain cells, or neurons, just outside the stroke damage might be saved. Steinberg and collaborators at the University of Pittsburgh recognized that stem cells taken from bone marrow wouldn’t transform into functioning neurons. However, the transplanted cells could release molecules that might rescue neurons that were impaired, but not yet dead.

Brain Surgery
Sonia had surgery to transplant bone marrow stem cells into her brain in late May 2013. The improvement was almost instantaneous. “When I woke up, my speech was strong, I could lift up my feet and keep them in the air, I even raised my right hand,” says Sonia. Though the trial was primarily designed to study the stem cell therapy’s safety, researchers were also interested in its effectiveness.

“Sonia was one of our two remarkable patients who got better the day after surgery and continued to improve throughout the year,” says Steinberg. 18 patients in total were treated in that study.

Although Sonia’s treatment results are still very preliminary, they bode well for a separate CIRM-funded stroke research project also led by Steinberg. In this study, cells grown from embryonic stem cells will be turned into early-stage neuron, or brain, cells and then transplanted into the area of stroke damage. The team has found that transplanting these neural cells into mice or rats after a stroke helps the animals regain strength in their limbs. The team is busy working out the best conditions for growing these neural cells in order to take them into clinical trials.

In the meantime, Sonia continues to improve. “My leg is about 95 percent better and my arm is around 60 percent there,” says Sonia. “My speech isn’t perfect, but I can talk and that’s something I never could have done before the surgery.”

The added function has made a huge difference in her quality of life. She can walk, run, drive a car, call a restaurant to make a dinner reservation—simple things she took for granted before having a stroke. But most importantly, she has confidence in the future.

“Everything is good,” says Sonia, “and it’s only going to get better.”

To learn about CIRM-funded stroke research, visit our Stroke Fact Sheet. Read more about Sonia’s Story of Hope on our website.

Stories of Hope: Sickle Cell Disease

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This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

Adrienne Shapiro pledged she would give her daughter Marissa the best possible life she could have—wearing herself out if necessary. Her baby girl had sickle cell disease, an inherited disorder in which the body’s oxygen-carrying red blood cells become crescent shaped, sticky, rigid, and prone to clumping—blocking blood flow. Doctors warned Adrienne that Marissa might not live to see her first birthday. When Marissa achieved that milestone, they moved the grim prognosis back a year, and then another year, and then another.

Adrienne has seen first hand how difficult it is to live with this blood disease.

Adrienne has lived through several generations of the inherited blood disease.

Adrienne worked tirelessly to help Marissa. “I was constantly asking questions,” Shapiro says. And for a long time, it worked.

However, things began to unravel for Marissa as she reached adulthood. A standard treatment for sickle cell disease—and the excruciating pain caused by blocked blood vessels—is regular blood transfusions. A transfusion floods the body with healthy, round red blood cells, lowering the proportion of the deformed, ‘sickle-shaped’ cells. But when she was 20, a poorly matched blood transfusion triggered a cascade of immune problems. Later, surgery to remove her gall bladder set off a string of complications and her kidneys shut down temporarily. After that, her immune system couldn’t take any more insults. Now, at age 36, she’s hypersensitive.

“She can’t be transfused. She can’t even have tape next to her skin without her body reacting,” Adrienne said.

Pain control is the newest and continuing nightmare. Adrienne tells harrowing stories of long waits in hospital emergency rooms while her daughter suffers, followed by maddening arguments with staff reluctant to provide enough drugs to control the intense pain when her daughter is finally admitted.

“When she was a kid, everyone wanted to make her feel good,” Adrienne says. “But when we moved from the pediatric side to the adult side, they treated her as a drug seeker and me as an enabler. It’s such a slap in the face.”

For Adrienne, the story is all too familiar. She is the third generation in her family with a sickle cell child. Another daughter, Casey Gibson, does not have the disease but carries the sickle cell mutation, meaning she could pass it to a child if the father also has the trait. One in 500 African Americans has sickle cell disease, as do 1 in 36,000 Hispanic people.

There is only one sure way to stop this story from repeating for generations to come, Adrienne says, and that’s research. She believes stem cell science will be the answer.

“I’ve been waiting for this science to get to the point where it had a bona fide cure, something that worked. Now we’re actually nearing clinical trials. It’s so close.”

In fact a CIRM-funded project led by Don Kohn, M.D. at UCLA aims to start trials in 2014. Kohn and his team intend to remove bone marrow from the patient and fix the genetic defect in the blood-forming stem cells. Then those cells can be reintroduced into the patient to create a new, healthy blood system.

“Stem cells are our only hope,” Adrienne continues, “It’s my true belief that I’m going to be the last woman in my family to have a child with sickle cell disease. Marissa’s going to be the last child to suffer, and Casey is going to be the last one to fear. Stem cells are going to fix this for us and many other families.”

For more information about CIRM-funded sickle cell disease research, visit our Sickle Cell Disease Fact Sheet. You can read more about Adrienne’s Story of Hope on our website.

Stories of Hope: Spinal Cord Injury

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This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

Katie Sharify had six days to decide: would she let her broken body become experimental territory for a revolutionary new approach—even if it was unlikely to do her any good? The question was barely fathomable. She had only just regained consciousness. A week earlier, she had been in a car crash that damaged her spine, leaving her with no sensation from the chest down. In the confusion and emotion of those first few days, the family thought that the treatment would fix Katie’s mangled spinal cord. But that was never the goal. The objective, in fact, was simply to test the safety of the treatment. The misunderstanding – a cure, and then no cure — plunged the 23-year-old from hope to despair. And yet she couldn’t let the idea of this experimental approach go.

Katie never gave up hope that stem cell-based therapies could help her or others like her living with spinal cord injury.

Katie never gave up hope that stem cell-based therapies could help her or others like her living with spinal cord injury.

Just days after learning that she would never walk again, that she would never know when her bladder was full, that she would not feel it if she broke her ankle, she was thinking about the next girl who might lie in this bed with a spinal injury. If Katie walked away from this experimental approach—what would happen to others that came after her?

Her medical team provided a crash course in stem cell therapy to help Katie think things through. In this case the team had taken stem cells obtained from a five-day old embryo and converted them into cells that support communication between the brain and body. Those cells would be transplanted into the injured spines. Earlier experiments in animal models suggested that, once in place, these cells might help regenerate a patient’s own nerve tissue. But before scientists could do the experiment, they needed to make sure the technique they were using was safe by using a small number of cells, too few to likely have any benefit. And that’s why they wanted Katie’s help in this CIRM-funded trial. They explained the risks. They explained that she was unlikely to derive any benefit. They explained that she was just a step along the way. Even so, Katie agreed. She became the fifth patient in what’s called a Phase I trial: part of the long, arduous process required to bring new therapies to patients. Shortly after she was treated the trial stopped enrolling patients for financial reasons.

That was nearly three years ago. Since then, she has been through an intensive physical therapy program to increase her strength. She went back to college. She tried skiing and surfing. She learned how to make life work in this new body. But as she rebuilt her life she wondered if taking part in the clinical trial had truly made a difference.

“I was frustrated at first. I felt hopeless. Why did I even do this? Why did I even bother?” But soon she began to see how small advances were moving the science forward. She learned the steep challenges that await new therapies. Then this year, she discovered that the research she participated in was deemed to be safe and is about to enter its next phase, thanks to a $14.3 million grant from CIRM to Asterias Biotherapeutics. “This has been my wish from day one,” Katie says.

“It gives me so much hope to know there is an organization that cares and wants to push these therapies forward, that wants to find a cure or a treatment,” she says. “I don’t know what I would do if I thought nobody cared, nobody wanted to take any risks, nobody wanted to put any funding into spinal cord injuries.

“I really have to have some ray of hope to hold onto, and for me, CIRM is that ray of hope.”

For more information about CIRM-funded spinal cord injury research, visit our Spinal Cord Injury Fact Sheet. You can read more about Katie’s Story of Hope on our website.

Stories of Hope: Leukemia

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This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

Stem cells create life. But if things go wrong, they can also threaten it. Theresa Blanda found that out the hard way. Fortunately for her, CIRM-funded research helped her fight this threat, and get her life back.

Theresa's battle with leukemia took a happier turn after entering into a stem cell-based clinical trial.

Theresa’s battle with leukemia took a happier turn after entering into a stem cell-based clinical trial.

In the first few days of human development embryonic stem cells are a blank slate; they don’t yet have a special, defined role, but have potential. The potential to turn into the cells that make up our kidneys, heart, brain, every other organ and every tissue in our body. Because of this flexibility, stem cells have shown great promise as a way to regenerate dead, diseased or injured tissue to treat many life-threatening or chronic conditions.

But some studies have suggested a secret, darker side to stem cells—so-called cancer stem cells. Like their embryonic cousins, these cells have the ability to both self-renew— to divide and make more copies of themselves – and specialize into other cell types. Many researchers believe they can serve as a reservoir for cancer, constantly reinvigorating tumors, helping them spread throughout the body. To complicate matters, these slow-growing cells are often impervious to cancer therapies, enabling them to survive chemotherapy.

For Theresa Blanda, cancer stem cells were dragging her down a slippery slope towards disease and possibly death. In 2003, she was diagnosed with polycythemia vera (CV), which causes the body to produce too many red blood cells. As sometimes happens with CV patients, her body began producing too many white blood cells as well. Eventually, she developed an even more serious condition, myelofibrosis, a form of bone marrow scarring that results in an enlarged spleen, bone pain, knee swelling and other debilitating symptoms.

“You couldn’t even breathe my way or I’d bruise,” says Theresa. “I didn’t think I was going to make it.”

Her doctors wanted to do a bone marrow transplant, but were having difficulty finding the right donor. “Finally, I just asked if there was some kind of clinical trial that could help me,” says Theresa.

Fortunately, there was.

The Root Cause
At UC San Diego’s Moores Cancer Center, Catriona Jamieson, M.D., Ph.D., had made a discovery that would have a big impact on Theresa’s health. In research funded in part by CIRM, Jamieson found a key mutation in blood-forming stem cells. Specifically, a mutation in a gene called JAK2 was being passed on to Theresa’s entire blood system, causing CV and myelofibrosis. Without effective treatment, her condition could have progressed into acute myeloid leukemia, a blood cancer with a very poor survival rate.

“These malignant stem cells create an inhospitable environment for regular stem cells, suppressing normal blood formation,” says Jamieson. “We needed to get rid of these mutated stem cells so the normal ones could breathe a sigh of relief.”

The answer was a JAK2 inhibitor being developed by San Diego-based TargeGen. Though the trial had already started, they made room for Theresa and the results were amazing. Within weeks, her discomfort had faded, her spleen had returned to normal and she was back at work.

“In a month or two I was feeling pretty good,” says Theresa. “I could climb stairs and the swelling in my knee had gone down.”

She continued on the drug for five years but safety issues forced the trial to be suspended. But the work continues. With continued support from CIRM, Jamieson and others are investigating new JAK2 inhibitors, and other alternatives, to help myelofibrosis patients.

“Because of CIRM funding, we’ve managed to develop a number of agents that have gone into clinical trials,” says Jamieson. “That means patients have lived to hold their grandchildren, attend their mom’s hundredth birthday party and live fruitful lives.”

For more information about CIRM-funded leukemia research, visit our Leukemia Fact Sheet. You can read more about Theresa’s Story of Hope on our website.

Stories of Hope: Diabetes

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This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

The last thing Maria Torres expected was to be diagnosed with type 2 diabetes. She exercised, ate well and kept her weight under control. There had to be some mistake. Maria asked her doctor to repeat the tests, but the results were the same. At 43, for reasons no one could fully explain, she had diabetes, and her life was going to change dramatically.

Maria Torres' diabetes diagnoses was frightening—but she is hopeful that stem cell therapies could one day change how doctors treat this devastating condition.

Maria Torres’ diabetes diagnoses was frightening—but she is hopeful that stem cell therapies could one day change how doctors treat this devastating condition.

“It really scared me,” says Maria. “I thought I was going to die soon.”

That Maria doubted her diagnosis is no surprise. Type 2 diabetes is often associated with obesity, and she didn’t fit the profile. Most likely, some undiscovered genetic component had made her susceptible to the disease.

Regardless, she now had to rework her life to manage the diabetes. Her cells had developed a condition called insulin resistance. Though her pancreas was producing insulin, which tells cells to take in blood sugar, the cells were not cooperating. As a result, glucose was accumulating in her blood, putting her at risk for heart disease, nerve damage, eye issues and a host of other problems.

To help her cells absorb glucose, she needs regular insulin injections. Maria injects the hormone five times a day and must often measure her blood sugar levels even more frequently.

Faithfully following this regimen has kept her alive for 20 years, but insulin is not a cure. Even with the regular injections, she faces dramatic mood swings and more serious complications as glucose levels rise and fall.

Working for a Cure
One of the most promising strategies to cure diabetes is to transplant beta cells, which sense blood sugar levels and produce insulin to reduce them. Patients with type 1 diabetes would benefit because new beta cells would replace the ones they’d lost to disease. Type 2 patients, like Maria, could increase their body’s ability to produce insulin, lowering blood sugar levels and alleviating the need for injections.

With almost $40 million in funding from CIRM, a San Diego-based company named ViaCyte is working on this solution. They have spent years developing new methods to turn human embryonic stem cells into insulin-producing beta cells. It hasn’t been easy. Stem cells are promising because they can form any tissue. However, to make a specific type of cell, researchers must replicate the exact signals that transform a stem cell into a beta cell, rather than a neuron or muscle cell.

In 2008, the company succeeded, but with a clever twist. They created progenitor cells, one step shy of mature beta cells, and allowed them to finish developing in the body. In animal studies, the hardier progenitor cells survived the transplant process and, once mature, began producing insulin. The project has another innovation up its sleeve: these progenitor cells are first placed in a porous capsule, about the size of a credit card, before transplantation under the skin. This device allows transfer of blood sugar, insulin, oxygen, and other molecules but keeps cells out, thus avoiding the possible attack and rejection by the patient’s own immune system.

ViaCyte’s goal is to start clinical trials for type 1 diabetes by the end of 2014. But the company eventually hopes to also help those with type 2. Maria Torres is eager for them to succeed, both for herself and her family.

“I have three kids, and I know they could have the same thing I have,” says Maria. “If they find a cure, for me, that’s peace of mind.”

For more information about CIRM-funded diabetes research, visit our Diabetes Fact Sheet. You can read more about Maria’s Story of Hope on our website.

Stories of Hope: Alzheimer’s Disease

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This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

Adele Miller knew what came next. She had lived it twice already: her father’s unraveling, due to Alzheimer’s disease, and, a few years later, her mother’s journey through the same erasure of mind and memory. So when doctors told her, at age 55, that she, too, had the disease, she remembered her parents’ difficult last years.

Lauren Miller has seen first hand how Alzheimer's can erase a lifetime's worth of memories.

Lauren Miller has seen first hand how Alzheimer’s can erase a lifetime’s worth of memories.

‘Tell no one,’ she told her family. Keep it secret.

“She was ashamed,” her daughter, actress and writer Lauren Miller, recalls. “She was so embarrassed because there’s such a stigma.” And she worried about her family. How would they handle all this? “I asked her once if she was scared,” Lauren says. “She said she wasn’t afraid for herself. But she was afraid for me, and my dad, and my brother. She knew what she’d gone through with her parents.”

Alzheimer’s disease has been a constant in the actress’s family. Perhaps that made her more attuned to the subtle changes that can herald the onset of the disease. At Lauren’s college graduation, she saw the first clues that something was amiss with her mother. She was repeating herself. Not just, “Oh, have I told you this before?” This was different. A few years later, as she and her mother prepared for a party, Lauren was stunned by the changes in her mother’s behavior. Her mother’s memory no longer seemed to function. She kept forgetting that the taco salad was vegetarian. She kept asking over and over where to throw the garbage. Lauren knew that’s not like her mother, a teacher for 35 years. So she sat down with her brother Dan and their dad. It was time to do something for Mom.

“It’s not that my dad wasn’t noticing things. But I don’t think he wanted to admit there was a problem. And he was simply too close to it,” Lauren says.

It took less than five years for Alzheimer’s disease to rob Adele Miller of nearly everything. Before she turned 60, she couldn’t write. She couldn’t speak. She didn’t even recognize her family.

The loss, the sadness, and the anger that Alzheimer’s families feel is compounded by a sense of utter helplessness against a disease that yields to no drug. But Lauren decided she would not be helpless, and in 2011, she and her husband, actor Seth Rogen, launched Hilarity for Charity, which aims to raise Alzheimer’s awareness in young people while also raising funds for the Alzheimer’s Association. This year Hilarity for Charity sponsored its first college fundraisers. It also hosts support groups for under-40 caregivers.

“Seth has the ability to reach an audience that may not know much about Alzheimer’s. His fans are 16 year old boys who aren’t generally the target for Alzheimer’s awareness,” Lauren said. “But he was able to strike a cord with a lot of these young people. We get emails from people who are 16. ‘Thank you for doing this. I felt alone. Now there’s a voice.’ This is considered an old person’s disease, but it’s really not. It affects everyone.”

In December 2013, Lauren, co-writer, producer and star of For a Good Time, Call, joined the CIRM governing Board, the Independent Citizens Oversight Committee, as a patient advocate for Alzheimer’s disease.

“Alzheimer’s research is woefully underfunded by the government, so it’s important to have bold, innovative approaches like CIRM’s to take research to the next level,” Lauren said. “Stem cell research is at the cusp of something life changing. To me, it’s one of the things closest to making a step toward treatment. I jumped at the opportunity to be part of it.”

For information about CIRM-funded Alzheimer’s disease research, visit our Alzheimer’s Fact Sheet. You can read more about Lauren’s Story of Hope on our website.