Stem Cell Clinic

Longeveron Initiates Phase 2b Stem Cell Therapy Trial to Treat Aging Frailty – Markets Insider

By Stem Cell Treatment

MIAMI, Sept. 6, 2017 /PRNewswire/ --Longeveron LLC, a regenerative medicine company developing cellular therapies, announced today that it treated its first patient in the Company's Phase 2b clinical trial evaluating the safety and efficacy of Longeveron human Allogeneic Mesenchymal Stem Cells (LMSCs) in patients with Aging Frailty Syndrome. This trial is being conducted pursuant to an Investigational New Drug Application (IND) in conformance with U.S. Food & Drug Administration (FDA) regulations. Aging Frailty is a common geriatric medical condition that is serious and life-threatening, and for which there are currently no U.S. Food and Drug Administration-approved therapeutics available.

The clinical trial is designed to enroll 120 subjects from approximately 10 medical centers around the U.S. The primary objective of the study is to evaluate the effect that LMSCs have on functional mobility and exercise tolerance in elderly Aging Frailty subjects. Three different LMSC dose groups will be compared to placebo over 12 months in a randomized, double-blinded, parallel arm design.Specifically, the trial will evaluate changes to the following:

"Frailty Syndrome is a very common and difficult situation to manage from a clinician's and caregiver's standpoint," stated Marco Pahor, M.D., Director of the Institute on Aging at the University of Florida. "The goal of intervention is to stop or slow the progression towards dependence and adverse health outcomes common to the syndrome, and to restore the patient to a state of healthy aging and functional independence. Longeveron's regenerative medicine trial is an important step towards the development of an effective therapeutic."

Allogeneic mesenchymal stem cells (MSCs) were previously tested in a Phase I/2 proof-of-concept study conducted by investigators at the University of Miami'sMiller School of Medicine. In that study, MSCs were shown to be safe and well-tolerated in frail, elderly subjects in a Phase 1 open label single ascending dose trial (publication link here) with a similar safety profile observed in the randomized, placebo-controlled Phase 2 study (publication link here) Subjects treated with a dose of 100 million MSCs showed significant improvements in six minute walking distance, and significant decreases in systemic inflammation, both relative to baseline.

"As individuals age, stem cell production and proliferation decreases, systemic inflammation increases, and a person's ability to repair and regenerate worn out or damaged tissue diminishes," remarked Suzanne Liv Page, Longeveron Chief Operating Officer. "In frail individuals this is particularly problematic. Our hypothesis is that exogenously infused allogeneic mesenchymal stem cells that are derived from the bone marrow of a healthy young donor, and culture expanded in our lab, will have potent regenerative and restorative effects."

Participants in this study must be between the ages of 70 and 85, be diagnosed as mildly to moderately frail due primarily to aging, and be able to walk between 200 and 400 meters over six minutes. Detailed information about the trial, subject eligibility and participating centers can be found by clicking here or by visiting the website http://www.clinicaltrials.gov and entering trial ID: NCT03169231.

About LMSCs

LMSCs is an allogeneic product, which means it is produced from stem cells derived from human donor bone marrow, and not from the patient's own stem cells, (referred to as autologous). LMSCs are manufactured at Longeveron's Cell Processing Facility in Miami, Fl. using a proprietary ex vivo culture expansion process.

About Longeveron

Longeveron is a regenerative medicine therapy company founded in 2014. Longeveron's goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life. The company's research focus areas include Alzheimer's disease, Aging Frailty and the Metabolic Syndrome. Longeveron produces LMSCs in its own state-of-the-art cGMP cell processing facility. http://www.longeveron.com

Contact: Suzanne Liv Page rel="nofollow">spage@longeveron.com 305.909.0850

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SOURCE Longeveron LLC

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Longeveron Initiates Phase 2b Stem Cell Therapy Trial to Treat Aging Frailty - Markets Insider

Zika Virus Targets and Kills Brain Cancer Stem Cells – UC San Diego Health

By Stem Cell Treatment

In developing fetuses, infection by the Zika virus can result in devastating neurological damage, most notably microcephaly and other brain malformations. In a new study, published today in The Journal of Experimental Medicine, researchers at the University of California San Diego School of Medicine and Washington University School of Medicine in St. Louis report the virus specifically targets and kills brain cancer stem cells.

The findings suggest the lethal power of the virus notorious for causing infected babies to be born with under-sized, misshapen heads could be directed at malignant cells in adult brains. Doing so might potentially improve survival rates for patients diagnosed with glioblastomas, the most common and aggressive form of brain cancer, with a median survival rate of just over 14 months after diagnosis.

The Zika virus specifically targets neuroprogenitor cells in fetal and adult brains. Our research shows it also selectively targets and kills cancer stem cells, which tend to be resistant to standard treatments and a big reason why glioblastomas recur after surgery and result in shorter patient survival rates, said Jeremy Rich, MD, professor of medicine at UC San Diego School of Medicine. Rich is co-senior author of the study with Michael S. Diamond, MD, PhD, professor, and Milan G. Chheda, MD, assistant professor, both at Washington University School of Medicine in St. Louis.

Transmission electron microscope image of negative-stained, Fortaleza-strain Zika virus (red), isolated from a microcephaly case in Brazil. Image courtesy of NIAID.

This year, more than 12,000 Americans will be diagnosed with glioblastomas, according to the American Brain Tumor Association. Among them: U.S. Senator John McCain, who announced his diagnosis in July. They are highly malignant. The two-year survival rate is 30 percent.

Standard treatment is aggressive: surgery, followed by chemotherapy and radiation. Yet most tumors recur within six months, fueled by a small population of glioblastoma stem cells that resist and survive treatment, continuing to divide and produce new tumor cells to replace those killed by cancer drugs.

For Zhe Zhu, MD, PhD, a postdoctoral scholar in Richs lab and first author of the study, the hyper-reproductive capabilities of glioblastoma stem cells reminded him of neuroprogenitor cells, which fuel the explosive growth of developing brains. Zika virus specifically targets and kills neuroprogenitor cells.

So Zhu, with Rich, Diamond, Chheda and other collaborators, investigated whether the Zika virus might also target and kill cultured glioblastoma stem cells derived from patients being treated for the disease. They infected cultured tumors with one of two strains of the virus. Both strains spread through the tumors, infecting and killing stem cells while largely avoiding other tumor cells.

The findings, the authors said, suggest that chemotherapy-radiation treatment and a Zika infection appear to produce complementary results. Standard treatment kills most tumor cells but typically leaves stem cells intact. The Zika virus attacks stem cells but bypasses ordinary tumor cells.

We see Zika one day being used in combination with current therapies to eradicate the whole tumor, said Chheda, an assistant professor of medicine and of neurology at Washington University School of Medicine.

To find out whether the virus could boost treatment efficacy in a live animal, researchers injected either the Zika virus or a saltwater placebo directly into glioblastoma tumors in 18 and 15 mice, respectively. Two weeks after injection, tumors were significantly smaller in the Zika-treated mice, who survived significantly longer than those given the placebo.

The scientists note that the idea of injecting a virus notorious for causing brain damage into patients brains seems alarming, but they say Zika may prove a safe therapy with further testing because its primary target neuroprogenitor cells are rare in adult brains. The opposite is true of fetal brains, which is part of the reason why a Zika infection before birth produces widespread and severe brain damage while a normal Zika infection in adults typically causes mild symptoms or none at all.

The researchers also conducted studies of the virus using brain tissue from epilepsy patients that showed the virus does not infect non-cancerous brain cells.

As an additional safety feature, the research team introduced two mutations that weakened the viruss ability to combat natural cellular defenses against infection, reasoning that while the mutated virus would still be able to grow in tumor cells, which have a poor anti-viral defense system, it would be quickly eliminated in healthy cells with a robust anti-viral response.

When they tested the mutated viral strain and the original parental strain in glioblastoma stem cells, they found that the original strain was more potent, but that the mutant strain also succeeded in killing the cancerous cells.

Were going to introduce additional mutations to sensitize the virus even more to the innate immune response and prevent the infection from spreading, said Diamond, a professor of molecular microbiology, pathology and immunology. Once we add a couple more, I think its going to be impossible for the virus to overcome them and cause disease.

Co-authors of the study include: Matthew Gorman, Estefania Fernandez, Lisa McKenzie, Jiani Chai, Justin M. Richner, and Rong Zhang, Washington University, St. Louis; Christopher Hubert, and Briana Prager, Cleveland Clinic; Chao Shan, and Pei-Yong Shi, University of Texas Medical Branch; and Xiuxing Wang, UC San Diego.

Funding for this research came, in part, from the National Institutes of Health (R01 AI073755, R01 AI104972, CA197718, CA154130, CA169117, CA171652, NS087913, NS089272), the Pardee Foundation, the Concern Foundation, the Cancer Research Foundation and the McDonnell Center for Cellular and Molecular Neurobiology of Washington University.

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Zika Virus Targets and Kills Brain Cancer Stem Cells - UC San Diego Health

Cellectis shares slump as death puts cell therapy tests on hold – Reuters

By Uncategorized

LONDON (Reuters) - French cell therapy specialist Cellectis, which is developing a gene-modified cancer treatment similar to Novartiss recently approved Kymriah, has been forced to suspend testing following a patient death.

Cellectis said on Tuesday it was working closely with the U.S. Food and Drug Administration in order to resume trials with a lower dose of the medicine UCART123.

Shares in the company fell 26 percent in morning trade following the U.S. regulators decision to place a so-called clinical hold on two early-stage trials of the medicine in blood cancers.

Novartis made history last week when it won approval for its $475,000 drug Kymriah, the first in a new class of treatments called CAR-T immunotherapies that use modified disease-fighting T cells to attack cancer.

While Novartis and rivals such as Juno and Kite use cells from the patients own body, Cellectiss gene edited cell therapy product offers an off-the-shelf, or allogeneic, option by deriving cells from healthy donors.

It is designed to help patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

However, the first patient treated in the BPDCN study, a 78-year-old man, died after experiencing cytokine release syndrome (CRS), a dangerous release of cell-signaling proteins.

The first patient treated in the AML trial, a 58-year-old woman, also experienced CRS and other symptoms but recovered.

Jefferies analysts said there was a chance that the adverse CRS events could be mitigated by lowering the dose and treating symptoms more aggressively, but more information was needed to assess prospects for UCART123.

The side effects could be caused in part by the fact that UCART123 cells come from a healthy donor, rather than the patients own body, they added.

Cellectis, which was founded in 1999, is also working on another off-the-shelf cell therapy called UCART19, which is being developed with Servier and Pfizer. That product is now being tested in trials for acute lymphoblastic leukemia.

UCART19 has already rescued two babies treated at Londons Great Ormond Street Hospital from previously incurable cancer.

Reporting by Ben Hirschler; editing by Jason Neely and Louise Heavens

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Cellectis shares slump as death puts cell therapy tests on hold - Reuters

France’s Cellectis told to pause cell therapy tests after death – Reuters

By Uncategorized

LONDON (Reuters) - French cell therapy specialist Cellectis, which is developing a gene-modified cancer treatment similar to Novartiss recently approved Kymriah, has been forced to suspend testing following a patient death.

Cellectis said on Tuesday it was working closely with the U.S. Food and Drug Administration in order to resume trials with a lower dose of the medicine UCART123.

Shares in the company fell 26 percent in morning trade following the U.S. regulators decision to place a so-called clinical hold on two early-stage trials of the medicine in blood cancers.

Novartis made history last week when it won approval for its $475,000 drug Kymriah, the first in a new class of treatments called CAR-T immunotherapies that use modified disease-fighting T cells to attack cancer.

While Novartis and rivals such as Juno and Kite use cells from the patients own body, Cellectiss gene edited cell therapy product offers an off-the-shelf, or allogeneic, option by deriving cells from healthy donors.

It is designed to help patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

However, the first patient treated in the BPDCN study, a 78-year-old man, died after experiencing cytokine release syndrome (CRS), a dangerous release of cell-signaling proteins.

The first patient treated in the AML trial, a 58-year-old woman, also experienced CRS and other symptoms but recovered.

Jefferies analysts said there was a chance that the adverse CRS events could be mitigated by lowering the dose and treating symptoms more aggressively, but more information was needed to assess prospects for UCART123.

The side effects could be caused in part by the fact that UCART123 cells come from a healthy donor, rather than the patients own body, they added.

Cellectis, which was founded in 1999, is also working on another off-the-shelf cell therapy called UCART19, which is being developed with Servier and Pfizer. That product is now being tested in trials for acute lymphoblastic leukemia.

UCART19 has already rescued two babies treated at Londons Great Ormond Street Hospital from previously incurable cancer.

Reporting by Ben Hirschler; editing by Jason Neely and Louise Heavens

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France's Cellectis told to pause cell therapy tests after death - Reuters

FDA Reaffirms Its Commitment to the Approval of Stem Cell Therapies Amidst Enforcement Actions Against … – Lexology (registration)

By Stem Cell Clinic

The U.S. Food and Drug Administration (FDA or Agency) has become increasingly active in stem cell and regenerative medicine product regulation within the past year previously announcing that it would release a modified framework for the regulation of these products in September. On August 28, FDA issued two news releases about recent enforcement actions taken against Florida and California stem cell companies and clinics, as well as a statement from FDA Commissioner Dr. Scott Gottlieb. Dr. Gottlieb noted the promise of adult stem cells as viable therapies with the appropriate testing and scientific evidence, and vowed to work with industry. He also, however, stated that the Agency would increase its enforcement activities against unscrupulous actors in this field.

On August 24, FDA issued a warning letter to U.S. Stem Cell Clinic, LLC in Sunrise, Florida. for violations of the Federal Food, Drug, and Cosmetic Act (FFDCA) namely, that the clinic (1) produced and administered unapproved stem cell treatments to patients; (2) violated current good manufacturing practices (cGMPs) and current good tissue practices (cGTPs); and (3) impeded FDAs inspection of the clinic. This same clinic was the subject of widespread stories in the popular press after publication of a New England Journal of Medicine article in March 2017 that reported varying degrees of vision loss in three patients who were treated with stem cells at the clinic. One day after FDAs news release about the warning letter, the clinic pushed back at the Agency by posting a public response and video.

On August 25, the U.S. Marshals Service seized non-commercial vials of live Vaccinia virus vaccine from StemImmune Inc. of San Diego, California. StemImmune allegedly used this vaccine, intended for very limited use against smallpox, to create an unapproved stem cell product by mixing the vaccine with stem cells and administering the product to cancer patients at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, California. The U.S. Department of Justice filed the seizure complaint and warrant on behalf of FDA in the U.S. District Court for the Central District of California (Southern Division).

These two recent events prompted Commissioner Gottlieb to issue a statement on August 28 about the future of regenerative medicine regulation at FDA. Dr. Gottlieb acknowledged the small number of unscrupulous actors that risk tarnishing the regenerative medicine field as a whole, as well as the sincere reports of the significant clinical potential of properly developed products. While promising additional details in the coming months, Dr. Gottlieb announced the following:

Although FDA has not yet released the full scope of its plans, companies, clinics and physicians currently developing or using stem cell products must recognize that the Agency is committed to taking enforcement actions when necessary to protect the public health. Therefore:

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FDA Reaffirms Its Commitment to the Approval of Stem Cell Therapies Amidst Enforcement Actions Against ... - Lexology (registration)

Zika kills brain cancer cells, may find use as therapy – The San Diego Union-Tribune

By Stem Cell Clinic

Zika, notorious for ravaging the brains of babies, could be useful in treating a particularly deadly brain cancer.

The virus kills glioblastoma stem cells, say researchers at UC San Diego and Washington University in St. Louis. They tested in human cell cultures and a mouse model of the disease

Moreover, Zika largely spared mature brain cells, the researchers say in a study published Tuesday in the Journal of Experimental Medicine.

People with glioblastoma rarely survive more than two years, although survival rates vary according to age and how aggressive the tumor is.

Study authors suggest a tamed version of the virus could be used along with other treatment to improve survival rates.

U.S. Senator John McCain was recently diagnosed with glioblastoma. The disease killed U.S. Senator Ted Kennedy, singer-actress Ethel Merman and retired baseball player Gary Carter.

Glioblastomas are treated with surgery, followed by radiation and chemotherapy. However, it is impossible to remove all the cancer without also removing healthy tissue and risking brain damage. So the cancer nearly always comes back.

Cancer stem cells cause the recurring tumors. These cells bear strong genetic resemblances to normal stem cells, and can proliferate greatly. Just one cell can regrow an entire tumor.

Glioblastoma stem cells also resist chemotherapy and radiation. But because they are stem cells, they are vulnerable to Zika. The virus causes an abnormally small head, or microcephaly, by destroying immature neural cells.

The study authors say a modified Zika virus could be applied after surgery, penetrating to the remaining cancer cells and killing them. One author, Jeremy Rich, M.D., is a renowned brain cancer specialist who recently joined UC San Diego from Cleveland Clinic. The first author, Zhe Zhu, also researches at UCSD.

Brain cancer specialists not involved with the study said by email it is scientifically sound, but there is a long way to go before it could be used in patients.

The science in this study is good considering the limitations of test tube and mice models, said Keith Black, M.D., chair of neurosurgery at Cedars-Sinai Medical Center in Los Angeles. What we don't know is how these results will translate to humans, given how different the complex human tumors are compared to simplistic mice models.

Before a Zika-based therapy can be tested in people, toxicity studies need to be completed in animals, along with regulatory approvals from the U.S. Food and Drug Administration and the institutes where the trial is to be conducted, Black said.

The approach has precedent in viral therapy by San Diegos Tocagen to treat glioblastoma, said Faith Barnett, M.D., a neurosurgeon with Scripps Green Hospital in La Jolla. Tocagens therapy is already being tested on glioblastoma patients. It uses poliovirus and retroviruses, a class of virus that includes HIV.

Whether Zika virus is a better vector needs to be determined, Barnett said. Clearly, we need creative approaches to improve current cancer therapies.

Unlike Zika, Tocagens viruses dont directly attack the brain cancer cells. Instead, they deliver a gene to the cancer that primes it for destruction when exposed to a drug precursor or prodrug.

The prodrug is converted into a toxic drug inside the cancer cells through an enzyme the gene codes for. Normal cells dont get the gene, and so are unaffected by the prodrug.

Go online to http://tocagen.com/patients for more information on Tocagens clinical trials.

For further reading

McCain completes round of radiation, chemo for brain cancer

UC San Diego hires renowned brain cancer expert Jeremy Rich

Blocking a tumor suppressor gene actually slows down one kind of glioblastoma

Survival time increases for those with deadly brain cancer: Study

Cancer genes hide outside chromosomes

Mom delays cancer care to protect baby she says saved her

Gary Carter to treat brain tumor with chemotherapy

Glioblastoma Anti-Angiogenesis Resistance Mechanism Found by Salk Researchers

bradley.fikes@sduniontribune.com

(619) 293-1020

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Zika kills brain cancer cells, may find use as therapy - The San Diego Union-Tribune

Zika virus kills brain cancer stem cells; could potentially be used to treat deadly disease – Medical Xpress

By Stem Cell Treatment

September 5, 2017 Brain cancer stem cells (left) are killed by Zika virus infection (image at right shows cells after Zika treatment). A new study shows that the virus, known for killing cells in the brains of developing fetuses, could be redirected to destroy the kind of brain cancer cells that are most likely to be resistant to treatment. Credit: Zhe Zhu

While Zika virus causes devastating damage to the brains of developing fetuses, it one day may be an effective treatment for glioblastoma, a deadly form of brain cancer. New research from Washington University School of Medicine in St. Louis and the University of California San Diego School of Medicine shows that the virus kills brain cancer stem cells, the kind of cells most resistant to standard treatments.

The findings suggest that the lethal power of the virus - known for infecting and killing cells in the brains of fetuses, causing babies to be born with tiny, misshapen heads - could be directed at malignant cells in the brain. Doing so potentially could improve people's chances against a brain cancer - glioblastoma - that is most often fatal within a year of diagnosis.

"We showed that Zika virus can kill the kind of glioblastoma cells that tend to be resistant to current treatments and lead to death," said Michael S. Diamond, MD, PhD, the Herbert S. Gasser Professor of Medicine at Washington University School of Medicine and the study's co-senior author.

The findings are published Sept. 5 in The Journal of Experimental Medicine.

Each year in the United States, about 12,000 people are diagnosed with glioblastoma, the most common form of brain cancer. Among them is U.S. Sen. John McCain, who announced his diagnosis in July.

The standard treatment is aggressive - surgery, followed by chemotherapy and radiation - yet most tumors recur within six months. A small population of cells, known as glioblastoma stem cells, often survives the onslaught and continues to divide, producing new tumor cells to replace the ones killed by the cancer drugs.

In their neurological origins and near-limitless ability to create new cells, glioblastoma stem cells reminded postdoctoral researcher Zhe Zhu, PhD, of neuroprogenitor cells, which generate cells for the growing brain. Zika virus specifically targets and kills neuroprogenitor cells.

In collaboration with co-senior authors Diamond and Milan G. Chheda, MD, of Washington University School of Medicine, and Jeremy N. Rich, MD, of UC San Diego, Zhu tested whether the virus could kill stem cells in glioblastomas removed from patients at diagnosis. They infected tumors with one of two strains of Zika virus. Both strains spread through the tumors, infecting and killing the cancer stem cells while largely avoiding other tumor cells.

The findings suggest that Zika infection and chemotherapy-radiation treatment have complementary effects. The standard treatment kills the bulk of the tumor cells but often leaves the stem cells intact to regenerate the tumor. Zika virus attacks the stem cells but bypasses the greater part of the tumor.

"We see Zika one day being used in combination with current therapies to eradicate the whole tumor," said Chheda, an assistant professor of medicine and of neurology.

To find out whether the virus could help treat cancer in a living animal, the researchers injected either Zika virus or saltwater (a placebo) directly into the brain tumors of 18 and 15 mice, respectively. Tumors were significantly smaller in the Zika-treated mice two weeks after injection, and those mice survived significantly longer than the ones given saltwater.

If Zika were used in people, it would have to be injected into the brain, most likely during surgery to remove the primary tumor. If introduced through another part of the body, the person's immune system would sweep it away before it could reach the brain.

The idea of injecting a virus notorious for causing brain damage into people's brains seems alarming, but Zika may be safer for use in adults because its primary targets - neuroprogenitor cells - are rare in the adult brain. The fetal brain, on the other hand, is loaded with such cells, which is part of the reason why Zika infection before birth produces widespread and severe brain damage, while natural infection in adulthood causes mild symptoms.

The researchers conducted additional studies of the virus using brain tissue from epilepsy patients and showed that the virus does not infect noncancerous brain cells.

As an additional safety feature, the researchers introduced two mutations that weakened the virus's ability to combat the cell's defenses against infection, reasoning that the mutated virus still would be able to grow in tumor cells - which have a poor antiviral defense system - but would be eliminated quickly in healthy cells with a robust antiviral response.

When they tested the mutant viral strain and the original parental strain in glioblastoma stem cells, they found that the original strain was more potent, but that the mutant strain also succeeded in killing the cancerous cells.

"We're going to introduce additional mutations to sensitize the virus even more to the innate immune response and prevent the infection from spreading," said Diamond, who also is a professor of molecular microbiology, and of pathology and immunology. "Once we add a few more changes, I think it's going to be impossible for the virus to overcome them and cause disease."

Explore further: Scientists to test Zika virus on brain tumors

More information: Zhu et al., 2017. J. Exp. Med. jem.rupress.org/cgi/doi/10.1084/jem.20171093

In a revolutionary first, Cancer Research UK-funded scientists will test whether the Zika virus can destroy brain tumour cells, potentially leading to new treatments for one of the hardest to treat cancers.

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Zika virus kills brain cancer stem cells; could potentially be used to treat deadly disease - Medical Xpress

A Napa family struggles to give their child a normal life – Napa Valley Register

By Stem Cell Doctors

Every mom anticipates her childs first day at kindergarten.

For Jessica Pequeno, that kind of milestone is something this mom is only now ready to imagine.

The last time the Napa Valley Register wrote about the Pequeno family, it was October 2015. Their then 22month-old toddler Xavier was about to begin the fight of his life against an immune deficiency disorder so rare it had no name.

Today, there is good news about Xavier and his family.

The now 4-year-old completed a grueling, yet successful stem cell transplant, just started his second year of preschool and is making progress with his health, said his mother. Come this time next year, shell be enrolling him in kindergarten.

Every day is different. We still deal with so many unknowns, she said. But, Hes doing so much better than anybody would have ever expected.

Just getting to this point was a long road.

Two years ago, the Pequenos told their story in hopes of finding a bone marrow transplant match for their son. Doctors originally told them there was no match within the family. Fortunately, after a second set of tests, the Pequenos middle son, Higinio Pequeno IV, was identified as a partial match.

That news was awesome, said Jessica Pequeno.

The family prepared for the transplant to take place in June 2015, but a stubborn infection put those plans on hold. By December, his health care team at the University of California at San Francisco wouldnt even give us odds as his percentage of survival because they didnt know, she said.

They finally had a name for his disease IKBa gain of function mutation with ectodermal dysplasia but there were too many unknowns.

Pequeno said she realized the stem cell transplant was a kind of a now-or-never situation.

We just kept saying, We just have to keep doing this. Giving up wasnt an option.

On Dec. 1, 2015, Xavier was admitted to the hospital for the transplant. The process began with eight days of chemotherapy followed by the stem cell transplant.

Putting a line in his femoral artery, blood was collected from Higinio, then 10. Then a machine separated the stem cells from the blood. Higinios stem cells were then given to Xavier. The stem cells were put into a vein, much like a blood transfusion. The stem cells are then meant to travel to the bone marrow, engraft, and hopefully begin making new, normal blood cells.

On the day of the transplant, the whole Pequeno family, including her husband Higinio, son Higinio and daughter Maya and Jessica Pequenos mom were there. Seeing those potentially life-saving cells go into her son was very emotional, said Jessica Pequeno.

We all cried, she said. It was really scary, but you cant stop. You have to keep going.

During the procedure, Xavier was awake, she said. But the side-effects of the chemotherapy were starting to set in. His hair was falling out, and he had stopped eating and drinking because his mouth sores were so bad and painful, she said. He was on morphine for the pain.

The waiting began. Would the stem cell transplant be a success?

The family was told that Xavier would likely spend many months in the hospital. We planned to be separated as a family for at least six months, said Pequeno. We just expected it to be really hard.

She spent her nights in the room with her son, sleeping on a blow-up twin mattress. The rest of her family went back to Napa. Because Jessica was unable to work and her husband couldnt work because he needed to have knee surgery, the family had moved in with Jessicas mother.

Meanwhile, doctors continued to check Xaviers blood to see if his body was responding to the stem cell transplant.

Every day Id ask, Where are we at? his mother said.

And then, one day in early January, the doctors came to see Xavier, and they said, We have good news.

The transplant was starting to work and the new cells were starting to grow, she said.

I cried, said Pequeno. It happened so much faster than what they had expected.

By the end of January, Xavier was well enough to go home to Napa.

It was scary to come home and super exciting, she said.

Back at home, a new routine was created. Xavier was still taking 25 different medications, some multiple times per day. He had a gastrostomy or G tube for feeding the nutritional liquid he eats and a central line a thin, flexible tube used to give medicines, fluids, nutrients, or blood products over a long period of time.

Honestly I dont remember a lot of it. It becomes a big blur, said Pequeno.

The family continued to visit UCSF at least once a week for blood counts and other checks. There were more ups and downs. Infections and illnesses caused him to be hospitalized for days at a time in February, May and June. His central line got infected. He got shingles.

His immune system was still really weak, said Pequeno.

But he kept bouncing back.

Just two weeks ago, doctors finally removed his central line.

It was a huge step, she said.

Challenges remain. Before the stem cell transplant, Xavier had about 5 to 10 percent of a normal immune system. Now he has about 60 to 70 percent, doctors said.

Were starting to learn hes really prone to sinus and respiratory infections, and viruses, said Pequeno. His body just doesnt fight like everyone elses.

Other habits are harder to change.

Before Xavier went to preschool, Pequeno and her family were able to carefully control his exposure to germs.

When he was able to go to preschool, I wasnt in control of those environments anymore. Its really hard. It gets easier, but it takes a while to learn how to kind of let go, she said.

Today, Xavier takes only six medications a day and can receive infusions of antibodies at home. Visits to UCSF have been cut back to once every four to five weeks.

Developmentally, Xavier is doing well, she said.

He has some hearing loss, which we continue to monitor. Its hard to say if its a side effect of chemo or other drugs. Right now it doesnt affect his speech. He also has skin, hair and teeth health issues to manage.

Xavier doesnt complain at lot, said Pequeno.

Hes always handled everything so well. When he suffered, He would get quiet. Even now when hes not feeling good, instead of crying like many small children would, Xavier is quiet.

Financially, its hard because Im still not able to work, she said.

Xaviers medical care is provided by Partnership HealthPlan/Medi-Cal and California Childrens Services. Her husband went back to work. Pequeno is taking classes at Napa Valley College while her son is in preschool.

I want to be a nurse but I want to go into pediatrics I want to teach parents how to advocate for their kids.

One of the most significant changes for Pequeno was becoming more confident in working with health care providers regarding her sons care.

Nobody could hand me a book when this started (that said) these are the things you need to know and questions to ask. No one told me I was the captain of his team. Her confidence grew. You have to get comfortable in that role.

The past several years have left a lasting imprint on the whole family, she said. Signs of post-traumatic stress have been seen in all family members. Learning coping skills and how to manage stress is important.

Especially for their son Higinio, said his mother. Its not easy for young boy to come to terms with what his brother went through and his own unique contribution.

I dont think any 10-year-old is capable of understanding the weight that carries, she said.

The struggles havent ended, said Pequeno.

Weve just learned to manage them and adjust and deal with the financial part. We juggle. You learn how to change your priorities.

Its easy to say her son looks healthy, said Pequeno, but thats also frustrating because it takes so much work to get him to continue to look like that.

It definitely takes a toll and lot of work and sacrifice to keep him where hes at, she said.

And Xaviers condition isnt going away, she noted. This is something we will manage for the rest of his life one way or the other.

People say, Oh youre so strong. But I think that as a mom, you just do it, said Pequeno. You pull the strength from somewhere. Because you dont give up on your kids.

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A Napa family struggles to give their child a normal life - Napa Valley Register

FDA Approves New Cancer Treatment – Alive For Football

By Gene Therapy Clinics

Analyst John Newman of Canaccord takes yesterday's FDA approval of Novartis' chimeric antigen receptor T-cell (CAR-T) therapy Kymriah as a meaningful victory for Kite Pharma Inc's (NASDAQ:KITE) fellow CAR-T therapy candidate Axi-cel.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb.

An FDA advisory committee had recommended the therapy for approval in July to treat the relapse of a blood cancer known as B-cell acute lymphoblastic leukemia, or ALL. While this was a groundbreaking approval, the therapy will cost $475,000 for 1 treatment, according to Kaiser Health News.

The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the USA market - and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumors, too.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. The FDA said hospitals and clinics must become certified to distribute the treatment, meaning they are prepared to recognize and treat CRS and other potentially fatal neurological events.

Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus. Once re-injected into the patient's body, the T cells modified to replicate quickly, and the remission is rapid.

The therapy is being licensed and brought to market by the pharmaceutical company Novartis.

"Novartis is collaborating with (Centers for Medicaid Services) to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month".

The price of the therapy is tagged at around $500,000. An immune overreaction called "cytokine release syndrome" can trigger high fevers, plummeting blood pressure and in severe cases organ damage, requiring special care to tamp down those symptoms without blocking the cancer attack. Because Kymriah can have life-threatening side effects, including unsafe drops in blood pressure, the FDA is requiring that hospitals and doctors be specially trained and certified to administer it, and that they stock a certain drug needed to quell severe reactions.

For now, it is only approved for use on ALL.

Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society, is excited by the drug's approval, but he has some reservations. This is elegant science. "There is still much more work to do", Lichtenfeld told Healthline. The safety and effectiveness of Kymriah was tested in one small clinical trial of 63 children and young adults with relapsed or refractory B-cell precursor ALL. Even then, it is prescribed only as a last resort when other forms of treatment have failed.

Still, "a far higher percentage of patients go into remission with this therapy than anything else we've seen to date with relapsed leukemia", said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the study sites.

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FDA Approves New Cancer Treatment - Alive For Football

Global Cancer Biological Therapy Analysis & Forecast 2016 to 2023 – Digital Journal

By Gene Therapy Clinics

Cancer Biological Therapy Analysis & Forecast: Global Industry Analysis, Capacity, Production, Price, Revenue, Size, Share, Growth, Trends, and Forecasts 2017-2022

This press release was orginally distributed by SBWire

Lawndale, CA -- (SBWIRE) -- 09/04/2017 -- Introduction Biological therapy treatment is done with the help of living organisms, parts of living organisms or laboratory manufactured version of such content. There are various types of biological therapies, which inhibit specific molecules involved in development and growth of cancer tumor. Such therapies known as; cancer targeted therapies.

The global cancer biological therapy market is expected to reach USD 82,276.8 million by 2023 at a CAGR of 4.7% during the forecasted period.

The global cancer biological therapy market is segmented on the basis of phases, types, end users and regions. On the basis of phases, the market is segmented into phase I, phase II and phase III. In stage I & II the real impact of these therapies is seen and giving a success rate of 35% in Phase 1 and 20% in Phase II. The success rate of phase I is 35%.

On the basis on types, the global cancer biological therapy market is segmented into monoclonal antibodies, cancer growth blockers, interferons, interleukins, gene therapy, targeted drug delivery, colony stimulating factor, cancer vaccines and others. Monoclonal antibodies accounted for the largest market share of the global cancer biological therapy market. Colony stimulating factor is the fastest growing market at a CAGR of 5.2% during the forecasted period.

On the basis on end users, hospitals & clinics dominates the global cancer biological therapy market. Registering USD 26,790.6 million in 2016 and expected to reach at USD 38,471.9 million by 2023 at the rate of 4.4 % from 2016-2023.

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On the basis of regions, the market is segmented into North America, Europe, Asia-Pacific and the Middle East & Africa. North America has the dominating market for cancer biological therapy. The cancer biological therapy market for North America is estimated at USD 19,481.2 million in 2016 and expected to reach by USD 29,516.9 million by 2023 at a fastest CAGR of 5.10%.

Key Players The leading market players in the global cancer biological therapy market include Merck Inc., F. Hoffmann-La Roche Ltd, Novartis AG, Amgen Inc., Bristol-Myers Squibb, Celgene, ELI Lilly and Company, EnGeneIC, and Pfizer Study objectives - To provide detailed analysis of the market structure along with forecast of the various segments and sub-segments of the global cancer biological therapy market - To provide insights about factors influencing and affecting the market growth - To provide historical and forecast revenue of the market segments and sub-segments with respect to countries - To provide historical and forecast revenue of the market segments based on channels, applications, and regions for the global cancer biological therapy market. - To provide strategic profiling of key players in the market, and comprehensively analysing their market share, core competencies, and drawing a competitive landscape for the market - To provide economical factors that influences the global cancer biological therapy market - To provide detailed analysis of the value chain and supply chain of the global cancer biological therapy market Target Audience - Pharmaceutical Companies - Pharmaceutical Suppliers - Cancer Research Organizations - Potential Investors - Key Executive (CEO and COO) and Strategy Growth Manager - Reaserch Companies Key Findings - North America accounted for the largest market share in the global cancer biological therapy market, USD 19,481.2 million in 2016 and expected to reach by USD 29,516.9 million by 2023 at a fastest CAGR of 5.10% - Colony stimulating factor is the fastest growing segment with a CAGR of and 5.2% in the global cancer biological therapy market, by types - Hospitals and clinics is contributing remarkable share in the market registering 47.8% in the global cancer biological therapy market, by end users in 2016

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The reports also covers regional analysis - North America o US o Canada - Europe o Germany o France o U.K. o Italy o Spain o Rest of Europe - Asia Pacific o Japan o China o India o Republic of Korea o Rest of Asia-Pacific - Middle East & Africa o Middle East o Africa

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About Provue Market Research Provue Market Research is a single destination for all the industry, company and country reports. We feature large repository of latest industry reports, leading and niche company profiles, and market statistics released by reputed private publishers and public organizations. We provide our clients not only with market statistics unveiled by avowed private publishers and public organizations but also with vogue and newest industry reports along with pre-eminent and niche company profiles.

For more information on this press release visit: http://www.sbwire.com/press-releases/cancer-biological-therapy-anal/release-857498.htm

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Global Cancer Biological Therapy Analysis & Forecast 2016 to 2023 - Digital Journal

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