leukemia Archives - Sanford Burnham Prebys

Tag: leukemia

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How community collaboration shapes leukemia research at Sanford Burnham Prebys

AuthorMiles Martin
Date

October 4, 2022

Rena Johnson, Ani Deshpande, PhD, and Todd Johnson holding a large cardboard check for $25,000 from the Luke Tatsu Johnson Foundation

Since 2020, Todd and Rena Johnson, co-founders of the Luke Tatsu Johnson Foundation (LTJF), have helped fund the research of Associate Professor Ani Deshpande, PhD

But it all started with their son Luke. He had a very rare subtype of acute myeloid leukemia, one of the most difficult-to-treat cancers, and, sadly, he passed away from the disease in 2016. This inspired the Johnsons to become involved with fundraising and advocacy for cancer research.

“Our foundation started with a fundraising golf tournament to honor Luke, and that was about taking something so horrific and so horrible and finding a way to turn it into something positive,” says Rena. “If you can take that tragedy and put a positive spin on it, then everything around Luke and his name and his memory becomes positive.”

How “the stars and planets aligned” to bring the Johnsons to the Institute

In a remarkable coincidence, the Johnsons discovered on their first visit to the Institute that Deshpande’s research focuses on AF10 fusion AML, an extremely rare subtype of the disease that accounts for about 5 percent of cases. It’s also the subtype of AML that Luke had.

“It was a goosebumps-raising moment,” says Todd. “Once we visited Ani and saw his lab, we realized there was a lot more in common with our story and his research than we had realized before.”

“The stars and planets aligned and brought us to Ani,” adds Rena. 

Luke Tatsu Johnson

Luke Tatsu Johnson

As well as helping fund Deshpande’s research through LTJF and their partnership with the Rally! Foundation, the Johnsons are also on the Community Advisory Board (CAB) for the Institute’s Cancer Center, which advocates for cancer research by engaging the community. 

“The CAB does such a wonderful job of connecting the community with the scientists, and we’re so excited to be involved in that,” says Todd. “That’s fundamentally what we do as a foundation—we support the folks doing this work so that children and families down the road can have a different outcome from Luke’s.”
 

AML research “needs more support and needs more funding”

The Johnsons’ support helped the AML research team discover a new potential treatment for AML, which is currently in preclinical studies, after which they hope it will advance to clinical trials. The research team maintains that it would have been impossible to secure the NIH grants necessary to do these studies without the jump start given by the LTJF and the Rally! Foundation.

“We couldn’t do what we do without the Johnsons’ support,” says Deshpande. “We are so grateful to have them in our corner, and we’re confident that our work will help improve outcomes for kids like Luke down the line.”

Despite this progress, more research into AML and other leukemias is still needed. Leukemia is the most common cancer in children and teens. About 4,000 children are diagnosed with leukemia each year, and AML accounts for about a third of these cases.
 

Studying AML from all angles

To tackle this pressing problem, the Institute has established an AML disease team composed of researchers across labs and clinician partners. The team’s research falls into several large categories, including studying the genetics of AML, studying how the disease works in animal models and working to develop drugs that can target specific mutations associated with the disease, which are numerous. 

“AML has many different subtypes, so it’s been difficult for researchers to make major advances to treat all cases of AML,” says Deshpande, who co-leads the AML team with Professor Peter D. Adams, PhD “Most patients with AML are given the same treatments that have been used since the ’70s, which is why we want to look at AML from as many angles as possible.”

In addition to being difficult to treat, it is also challenging to get funding for AML research, particularly for the rarer subtypes. This makes the support of foundations such as LTJF even more vital to researchers like Deshpande. 

“This is exactly why AML research needs more support and needs more funding, because this is a much more difficult disease than other forms of leukemia,” says Todd. “Many patients don’t have positive outcomes, and the only way to turn that pendulum is to intensify our efforts and increase the amount of research being done.”

Institute News

Top Sanford Burnham Prebys research stories of 2021

AuthorSusan Gammon
Date

December 14, 2021

top 10 sign

This year’s most popular research stories include scientific breakthroughs in COVID-19, cancer, schizophrenia and more.

As we bid farewell to 2021, let’s celebrate our most newsworthy research breakthroughs. Despite the continuing challenges brought on by COVID-19, Sanford Burnham Prebys achieved important milestones on the frontiers of biomedical science.

The following 10 research-related stories received top views on Newswise—the press release distribution service for journalists seeking health and science news.

  1. COVID-19: Scientists identify human genes that fight infection
    A research team was able to pinpoint specific human genes that control viral infection. The information sheds new light on factors that lead to severe disease and guides therapeutic options.
  2. Tumor marker may help overcome endocrine treatment-resistant breast cancer
    The study discovered a new approach to select breast cancer patients for HER2 therapy and could help individuals avoid disease relapse or progression of endocrine-sensitive disease.
  3. Scientists identify potential drug candidates for deadly pediatric leukemia
    Two existing drugs—JAK inhibitors and Mepron—show promise for a subtype of acute myeloid leukemia (AML) that is more common in children. The drugs are proven safe in humans, which could accelerate clinical studies.
  4. Leprosy drug holds promise as at-home treatment for COVID-19
    Scientists found that the leprosy drug clofazimine, which is FDA approved and on the World Health Organization’s List of Essential Medicines, exhibits potent antiviral activities against SARS-CoV-2, and could become an important weapon against future pandemics.
  5. Researchers dig deeper into how cells transport their waste for recycling
    Research describing how the “trash bags” in a cell—called autophagosomes—are tagged for recycling opened new paths to understand age-related diseases such as cancer and neurological disorders. 
  6. New drug combination shows promise as powerful treatment for AML
    Researchers identified two drugs that are potent against acute myeloid leukemia (AML) when combined, but only weakly effective when used alone. The study provides a scientific rationale for advancing clinical studies of the drug combination. 
  7. Biomarker could help diagnose schizophrenia at an early age
    A study described how elevated levels of a protein called CRMP2—found in the brain and blood—could become a format for a rapid, minimally invasive blood test to support the diagnosis of schizophrenia.
  8. Scientists identify “immune cop” that detects SARS-CoV-2
    Researchers discovered the sensor in human lungs that detects SARS-CoV-2 and signals that it’s time to mount an antiviral attack. The sensor activates interferon, the body’s own frontline defender against viral invasion.
  9. Study finds promising therapeutic target for colitis
    Scientists identified an enzyme in the gut that triggers an inflammatory cascade leading to colitis. Therapeutically targeting the enzyme may be a viable approach to help the millions of people worldwide affected by the disorder.
  10. Scientists shrink pancreatic tumors by starving their cellular “neighbors”
    For the first time, blocking “cell drinking,” or micropinocytosis in the thick tissue surrounding a pancreatic tumor, was shown to slow tumor growth—providing more evidence that micropinocytosis is an important therapeutic target.
Institute News

Meet cancer researcher Karina Barbosa Guerra

AuthorMonica May
Date

February 3, 2021

Karina Barbosa Guerra in the lab

Barbosa Guerra is working to find better treatments for a deadly leukemia

For Karina Barbosa Guerra, touring a lab and meeting scientists as part of her Girl Guides troop—Mexico’s equivalent of the Girl Scouts—was a life-changing experience. Suddenly, she could see herself as a scientist.

Today, Barbosa Guerra is a graduate student in the Deshpande lab at Sanford Burnham Prebys, where she’s working to find better treatments for a blood cancer called acute myeloid leukemia (AML). We caught up with Barbosa Guerra as she prepares to take the virtual stage at the Diversity and Science Lecture Series at UC San Diego (DASL) to learn more about when she decided she wanted to be a scientist and where she can be found when not in the lab.

Tell us about the moment you realized you wanted to be a scientist.
According to my mother, I stated that I wanted to become a chemist to develop vaccines when I was ten years old. However, it wasn’t until middle school that I started cultivating my own sense of scientific curiosity. At that time, I was in a Girl Scouts program centered on HIV/AIDS peer education, so I began to read a bit more about viruses. It was incredibly amazing that they could linger undetected in our bodies—and that many questions about their biology remained unanswered. The more I learned, the less I felt I knew, and I wanted to follow that endless string of questions.

What do you study, and what is your greatest hope for your research?
I study a cancer called acute myeloid leukemia—specifically, subtypes that are hard to treat. Certain cancer cells, like stem cells, are pretty resilient and can self-renew. This enables them to resist therapy, so we want to discover better ways to target this particular feature. My research aims to find ways in which we can treat these leukemias based on their stem cell–like capabilities. My hope is that we can ultimately benefit the patients enduring harsh treatments and disease relapse, and along the way, illuminate the fascinating aspects of the biology behind effective treatments.

What do you wish people knew about science?
That it’s a team effort. The current coronavirus pandemic has really shown us that collaboration is at the heart of transformative science. I think that great ideas are best developed through discussion—and the thrill of putting the pieces together is way more enjoyable with company.

How do you think your lab colleagues would describe you?
Maybe as the girl with a bunch of notebooks. I like to make notes of everything. My notebooks are way more reliable than my memory.

What is the best career advice you’ve ever received?
Early in the graduate program, one of my mentors told me, “Be there,” meaning that I had to spend time with my science. If I were to discover something or make a great insight, I had to be there to do it, think it or see it.

What do you wish people knew about Sanford Burnham Prebys?
That this is such a welcoming community. I felt this the very first time I visited the campus, and I feel so at home here as a student. There are plenty of opportunities to engage with others and help each other out. I really enjoy the collaborative spirit of our little community.

Learn more about the Institute’s Graduate School of Biomedical Sciences.

Institute News

A year in review: Our top 10 discoveries of 2019

AuthorMonica May
Date

December 4, 2019

hands holding up Most Popular sign

At Sanford Burnham Prebys, we uncover the origins of disease and launch bold new strategies that lay the foundation for achieving cures. This year our scientists made significant progress—revealing new insights into how we treat some of the deadliest cancers, address neurological disorders such as Parkinson’s and amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) and more.

Read on to learn more about our top 10 discoveries of the year. To receive more frequent updates on our discoveries, subscribe to our monthly newsletter at the bottom of this page.

  1. One-two punch drug combination offers hope for pancreatic cancer therapy. Ze’ev Ronai, PhD, identified a combination of two anti-cancer compounds that shrank pancreatic tumors in mice—supporting the immediate evaluation of the drugs in a clinical trial. The study was published in Nature Cell Biology.
  2. Targeted treatment shrinks deadly pediatric brain tumors. Robert Wechsler-Reya, PhD, reported that a targeted therapy that blocks a protein called LSD1 shrank tumors in mice with a form of pediatric brain cancer known as medulloblastoma. LSD1 inhibitors are currently under evaluation in clinical trials for other cancers, which could speed their potential path to children. The study was published in Nature Communications.
  3. Epigenetic change causes fruit fly babies to inherit diet-induced heart disease. Rolf Bodmer, PhD, showed that reversing an epigenetic modification or over-expressing two genes protected fruit fly children and grandchildren from the negative heart effects of their parents’ fatty diet. These findings help explain how obesity-related heart failure is inherited and uncover potential targets for treatment. The study was published in Nature Communications.
  4. Amyotrophic lateral sclerosis (ALS) research reveals new treatment approach. Huaxi Xu, PhD, extended the survival of mice with ALS-like symptoms by elevating levels of a protein called membralin using a gene therapy approach. The study was published in the Journal of Clinical Investigations.
  5. How prostate cancer becomes treatment resistant. Jorge Moscat, PhD, and Maria Diaz-Meco, PhD, identified how prostate cancer transforms into an aggressive, treatment-resistant subtype called neuroendocrine prostate cancer (NEPC) following treatment with anti-androgen therapy. Their findings uncover new therapeutic avenues that could prevent this transformation from occurring and reveal that an FDA-approved drug holds promise as an NEPC treatment. The study was published in Cancer Cell.
  6. Boosting muscle stem cells to treat muscular dystrophy and aging muscles. Alessandra Sacco, PhD, uncovered a molecular signaling pathway that regulates how muscle stem cells decide whether to self-renew or differentiate—an insight that could lead to muscle-boosting therapeutics for muscular dystrophies or age-related muscle decline. The study was published in Nature Communications.
  7. Functional hair follicles grown from stem cells. Alexey Terskikh, PhD, created natural-looking hair that grows through the skin using human induced pluripotent stem cells (iPSCs), a major scientific achievement that could revolutionize the hair growth industry. Stemson Therapeutics has licensed the technology.
  8. Potential targeted treatment for acute myeloid leukemia identified. Ani Deshpande, PhD, showed that a protein called BMI1 is a promising drug target for an AML subtype in which two normally separate genes fuse together. The findings, published in Experimental Hematology, provide a rationale for evaluating a BMl1-inhibiting drug that is currently in clinical development as a potential treatment for this subtype.
  9. Antimicrobial protein implicated in Parkinson’s disease. An immune system protein that usually protects the body from pathogens is abnormally produced in the brain during Parkinson’s disease, Wanda Reynolds, PhD, reported in Free Radical Biology & Medicine. The discovery indicates that developing a drug that blocks this protein, called myeloperoxidase (MPO), may help people with Parkinson’s disease.
  10. Digestion-aiding herbs alter gut microbiome. Scott Peterson, PhD, found that four herbs—turmeric, ginger, long pepper and black pepper—promoted strong shifts in the gut bacteria that are known to regulate metabolism, providing insights that could help us protect our health. The study was published in Evidence-Based Complementary and Alternative Medicine.
Institute News

5 things to know about acute myeloid leukemia (AML)

AuthorMonica May
Date

September 26, 2018

Ani Deshpande, PhD

It’s no surprise that our blood is important. The cargo it transports—nutrients, infection-fighting cells, clotting factors, waste and more—keeps our body healthy and running smoothly. So when blood cells don’t form properly, serious cancers can occur. 

Scientists divide blood cancers into three broad categories—leukemia, lymphoma and myeloma—based on the cell type affected. Leukemias disrupt white blood cell production; lymphomas affect the lymphatic system, which removes extra fluid from the body; and myelomas affect plasma cells, which produce intruder-fighting antibodies. There are many subsets within each category.

In honor of Blood Cancer Awareness Month, we spoke with Sanford Burnham Prebys Medical Discovery Institute scientist Ani Deshpande, PhD, to learn more about the blood cancer he studies: acute myeloid leukemia (AML). Of the 60,000 American children and adults diagnosed with leukemias each year, nearly 30 percent will have AML. 

  • Most patients receive the same treatment used nearly five decades ago. Drug developers have created medicines for AML patients who have certain changes in their DNA, called mutations. But the majority of AML patients receive the treatments used in the ’70s: chemotherapy, radiation and possibly a bone marrow transplant. This isn’t last-decade science; it’s last-century science.
  • It’s deadly. The five-year survival rate for adults with AML—the number of people who are alive five years after diagnosis—is only 24 percent, according to the American Cancer Society. New medicines and treatment approaches are urgently needed. 
  • Sequencing is making strides. Now, scientists can sequence patients’ genomes to learn the underlying mutation driving their cancer. This technology has advanced our understanding to the point that about 60 to 70 percent of the time, their doctor knows the mutation involved. Our new problem is that we don’t have effective medicines that target most of these mutations. 
  • Speaking of sequencing. Because of DNA sequencing, we also know that a large fraction of the mutations in AML are epigenetic changes—alterations that affect which genes turn on but don’t change the DNA itself.

To better understand how epigenetic changes work, imagine a cookbook. If recipes are DNA,               then epigenetic changes are bookmarks. These bookmarks signal whether the recipe should be made or not, without altering the underlying text of the recipe.

Our laboratory is studying the epigenetic changes that drive AML. Our hope is that once we identify these changes, we can create drugs that restore the epigenome to its normal state. 

  • There is hope. After nearly 50 years of little progress, four new drugs have been approved for AML over the last 18 months. And there are currently more than 330 clinical trials enrolling patients in the U.S., so more treatments may soon follow. 

Resources:

Interested in keeping up with Sanford Burnham Prebys’ latest discoveries, upcoming events and more? Subscribe to our monthly newsletter, Discoveries.
 

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V Foundation grant to Ani Deshpande, PhD, supports pioneering research toward better leukemia treatments

AuthorJessica Moore
Date

December 2, 2016

ani deshpanda, ph.d.

Patients with a rare type of leukemia called acute promyelocytic leukemia (APL) have better outcomes than most leukemias because they can be treated with a very effective drug that converts their cancer cells back to normal. This success has convinced many cancer researchers that there’s a way to do the same for other leukemias. And with his recently awarded funding from the V Foundation, Ani Deshpande, PhD, assistant professor at Sanford Burnham Prebys Medical Discovery Institute, can now find targets for future drugs to do just that.

“We’re aiming to rehabilitate the cancer cells, in a sense, instead of destroying them,” said Deshpande. “The advantage to this approach is that, unlike conventional chemotherapy, it doesn’t harm normal cells, so it should have far fewer toxic side effects.”

Deshpande aims to make a big impact with this work—he’s first focusing on a group of acute myeloid leukemia (AML) with very poor survival outcomes. Worse, these leukemias, characterized by fusions of chromosome 11 with another partner chromosome, are especially common among children and infants.

This subgroup of AML is trickier than APL, where the product of the gene created by the chromosomal rearrangement directly blocks the cancer cells from becoming their normal type. In contrast, in the leukemias that Deshpande’s lab studies, the change in the cells’ programming is more complex. The mutation they carry alters the regulation of other genes, but which of these prevent AML cells from becoming normal blood-forming cells is largely unknown.

Fortunately, Deshpande is an expert in studying leukemic gene regulation. His lab specializes in epigenetics—analyzing the chemical tags on genes that influence their activity. The V Foundation funds will allow Deshpande’s team to apply an advanced sequencing-based approach to identify and validate potential targets for drugs that restore cancer cells’ epigenome to normal.

“This grant not only lets me expand my lab by hiring a new postdoc, but it also means I can take risks that wouldn’t be possible if I were proposing research to the NIH,” commented Deshpande. “I’m confident that we’ll get exciting results. The tools we’re using have gotten exponentially better over the last few decades, so we’re poised for a breakthrough.”

About the V Foundation

The V Foundation for Cancer Research was founded by ESPN and legendary basketball coach Jim Valvano with one goal in mind: to achieve victory over cancer. Since its start in 1993, the V Foundation has awarded over $170 million in cancer research grants nationwide.

Watch Dr. Deshpande talk about why foundation funding is important:

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Leukemia research breakthrough: a new way to trigger cancer cell suicide

AuthorJessica Moore
Date

May 18, 2016

Header image

Better therapies for acute myeloid leukemia (AML), a fast-growing cancer of the bone marrow, are urgently needed. Nearly 15,000 people in the United States are diagnosed with AML each year, and it’s the most common acute leukemia in adults. The cause of the disease is unknown, and it is usually fatal within the first five years. Continue reading “Leukemia research breakthrough: a new way to trigger cancer cell suicide”

Institute News

How arsenic cures leukemia

Authorsgammon
Date

June 18, 2015

Header image

For the first time, Sanford-Burnham researchers have shown how the reversible interactions of the small protein SUMO work to facilitate treatment of acute promyelocytic leukemia (APL). The study, published recently in the journal Science Signaling, explains how the on-off associations of SUMO are required to destroy the APL causing oncoprotein and pave the way for an arsenic-based cure. Continue reading “How arsenic cures leukemia”