Frequency Raises $32M in Series A to Develop Progenitor Cell Therapy for Hearing Loss – Genetic Engineering & Biotechnology News

Frequency Therapeutics raised $32 million in a Series A financing to support development of its Progenitor Cell Activation (PCA) platform. The technology uses small molecules to trigger the division and differentiation of tissue-specific Lgr5+ progenitor cells and is initially being developed to generate new sensory cells in the inner ear as a treatment for noise-induced hearing loss. The Massachusetts-based firm says other potential applications of the platform could span skin disorders, muscle regeneration, and gastrointestinal diseases.

Frequencys Series A fundraising round was led by Cobro Ventures. Other investors included Morningside Ventures, Emigrant Capital, Korean Investment Partnership, Alexandria Real Estate Equities, and additional U.S. and international investors.

The PCA platform was developed by Robert Langer, Sc.D., and Jeffrey Karp, Ph.D., at the Massachusetts Institute of Technology (MIT), and Harvard Medical School. Frequency was established in 2015 to develop the platform for therapeutic indications and has an exclusive global license to relevant IP from MIT and Partners Healthcare. Bob Langer's and Jeff Karps vision is to gain much of the same effect as gene therapy and CRISPR by using small molecules, which we believe are safer and allow for easier delivery, stated Marc Cohen, co-founder of Cobro Ventures and chairman of Frequencys board of directors, which was established earlier this year. Our data is very compelling and we are excited to be moving to the clinic in the next 12 to 18 months.

Frequencys co-founders published in vitro research demonstrating use of their small-molecule approach to trigger the differentiation of Lgr5-expressing progenitor cells into sensory hair cells, in the February 21 issue of Cell Reports in a paper titled "Clonal Expansion of Lgr5-Positive Cells from Mammalian Cochlea and High-Purity Generation of Sensory Hair Cells."

Commenting on the initial, hearing loss indicaiton for the PCA platform, David Lucchino, Frequencys co-founder, president and CEO, added, With no effective therapy available, this presents an enormous market opportunity and we believe there is even broader potential in indications beyond hearing loss with the further development of the PCA platform.

View original post here:
Frequency Raises $32M in Series A to Develop Progenitor Cell Therapy for Hearing Loss - Genetic Engineering & Biotechnology News

Mesoblast takes off as cell therapy for heart failure passes interim test – FierceBiotech

At the halfway point, a phase 3 trial of Mesoblast's off-the-shelf cell therapy for chronic heart failure is on track and should continue to completion, say advisers.

Shares in the Australian biotech hit a 12-month high on the update from the trial, which came after it enrolled 270 out of a planned total of 600 patients with moderate CHF andcruciallyrevealed no safety issues with Mesoblast's MPC-150-IM candidate.

The trial is still blinded so there is now way to tell if the therapy is having an effect, but it's a case of so far, so good, as the biotech looks ahead to completing the study next year.

The trial is seeing whether delivery of mesenchymal precursor cells (MPCs) via a catheter into the left ventricular heart muscle, to see if it can reduce non-fatal heart failure-related major adverse cardiac events (HF-MACE), such as death, needing a pacemaker implanted or undergoing a heart transplant.

Last year, Israeli drugmaker Teva returned rights to MPC-150-IM to Mesoblast, which it had inherited through its takeover of Cephalon. Mesoblast however decided to go it alone, a decision helped by a recent $40 million placement intended to help bring the CHF trial to fruition.

Analyst John Savin at Edison said in a recent note that the biotech may not have to wait for its own trial to complete before filing for U.S. approval. That could happen before the end of the year if a National Institutes of Health-sponsored trial of the therapy in end-stage heart failure patients hits the target.

The 159-patient NIH study is expected to complete enrolment in the first half andwith luckcould report top-line data before year-end, according to Savin. He reckons that could "lead to an application for accelerated approval" under the U.S. 21st Century Cures Act, which provides a speedy route to market for regenerative medicines.

"Passing this interim futility analysis for MPC-150-IM is an important milestone for Mesoblast and our cardiovascular disease program," commented CEO Silviu Itescu. "This validates our strategy and our prioritization of this valuable program." Analysts at Credit Suisse have previously suggested that that drug could be worth $4.1 billion in peak annual sales.

Mesoblast is not the only biotech looking at a stem cell approach to cardiovascular diseases. BioCardia is developing CardiAMP for CHF, reporting positive data from small phase 2 trial last year, while CardioCell presented mixed results on its candidate at the 2016 ESC Congress in Rome, and Celyad's C-Cure product failed to meet its objectives in its phase 3 CHART trial. Meanwhile, Belgium's TiGenix said recently its acute myocardial infarction therapy AlloCSC-01 hit its targets in a phase 1/2 trial.

The Australian biotech claimed an FDA green light to start trials of an MPC therapy given alongside corrective heart surgery for children with hypoplastic left heart syndrome (HLHS).

It's also in the build-up to a decision by option partner Mallinckrodt on its phase 3 MPC for chronic low back paindue in or before Septemberand a graft-versus-host disease (GVHD) candidate that should see pivotal results before year-end.

View original post here:
Mesoblast takes off as cell therapy for heart failure passes interim test - FierceBiotech

Spray-On Skin: ‘Miracle’ Stem Cell Treatment Heals Burns Without Scarring – Newsweek

Pennsylvania state trooper Matt Uram was talking with his wife at a July Fourth party in 2009 when a misjudged spray of gasoline burst through a nearby bonfire and set him alight. Flames covered the entire right side of his body, and after he fell to the ground to smother them, his wife beat his head with her bare hands to put out his burning hair. It was only on the way to the ER, as the shock and adrenaline began to wear off, that the pain set in. It was intense, he says. If you can imagine what pins and needles feel like, then replace those needles with matches.

From the hospital, Uram was transferred to the Mercy Burn Center in Pittsburgh, where doctors removed all of the burned skin and dressed his wounds. It was on the border between a second- and third-degree burn, and he was told to prepare for months of pain and permanent disfigurement. Not long after this assessment, however, a doctor asked Uram if he would be willing to take part in an experimental trial of a new device.

The treatment, developed by German researcher Dr. Jrg Gerlach, was the worlds first to use a patients stem cells to directly heal the skin. If successful, the device would mend Urams wounds using his bodys ability to regenerate fully functioning skin. Uram agreed to the procedure without hesitation.

Five days after the accident, surgeons removed a small section of undamaged skin from Urams right thighabout the size of a postage stampand used it to create a liquid suspension of his stem cells that was sprayed in a fine mist onto the damaged skin. Three days later, when it was time to remove the bandages and re-dress the wounds, his doctor was amazed by what he saw. The burns were almost completely healed, and any risk of infection or scarring was gone.

Pennsylvania State Trooper Matt Uram's arm eventually healed without scarring. RenovaCare

A study subsequently published in the scientific journal Burns described how the spray was able to regrow the skin across the burn by spreading thousands of tiny regenerative islands, rather than forcing the wound to heal from its edge to the inside. The technique meant reducing the healing time and minimizing complications, with aesthetically and functionally satisfying outcomes, the paper stated.

Dozens more burn victims in Germany and the U.S. were successfully treated with the spray following Urams procedure, and in 2014 Gerlach sold the technology to RenovaCare. The medical technology startup has now transformed the proof-of-concept device from a complicated prototype into a user-friendly product called a SkinGun, which it hopes clinicians will be able to use outside of an experimental setting. For that to happen, RenovaCare is preparing clinical studies for later this year, with the aim of Food and Drug Administration approval for the SkinGun.

Once these obstacles are overcome, RenovaCare CEO Thomas Bold believes, the SkinGun can compete with, or even replace, todays standard of care.

Current treatment of severe burns involves transplanting healthy skin from one area of the body and stitching it to another in a process called skin grafting or mesh skin grafting. It is a painful procedure that creates an additional wound at the donor site and can cause restricted joint movement because the transplanted skin is unable to grow with the patient. It is able to cover an area only two to three times as large as the harvested patch. The current standard of care is just horrible, says Bold. We are part of regenerative medicineit is the medicine of the future and will be life-changing for patients.

RenovaCare's SkinGun sprays a liquid suspension of a patient's stem cells onto a burn or wound in order to regrow the skin without scars. RenovaCare

Beyond regulatory matters, there are also limitations to the technology that make it unsuitable for competing with treatments of third-degree burns, which involve damage to muscle and other tissue below the skin. Still, stem cell researcher Sarthak Sinha believes that while the SkinGun may not be that advanced yet, it shows the vast potential of this form of regenerative medicine. What I see as the future of burn treatment is not skin repair but rather functional regeneration of skin and its appendagessuch as hair follicles, glands and fat, says Sinha. This could be achieved by engaging deeper layers of skin and its resident stem cells to partake in tissue regeneration.

Research is already underway at RenovaCare to enable treatment of third-degree burns, which Bold describes as definitely within the range of possibility. Bold claims the adaptations to the SkinGun would allow it to treat other damaged organs using a patients stem cells, but for now the company is focusing solely on burns and wounds to skinthe largest organ of the human body.

Urams burns are now completely unnoticeable. There is no scar tissue or even pigment discoloration, and the regenerated skin even tans. If I show someone where I was burnt, I bet $100,000 they couldnt tell, he says. Theres no scars, no residual pain; its like the burn never happened. Its a miracle.

Uram is frustrated that the treatment is not available to other burn victims, particularly children. I want to see the FDA get off their butts and approve this, he says. A grown man like me to be scarred is OK, but think about the kids that have to live the rest of their lives with pain and scarring. Thats not OK.

Read the original post:
Spray-On Skin: 'Miracle' Stem Cell Treatment Heals Burns Without Scarring - Newsweek

Cell Therapy 2.0: Reprogramming the Brain’s Own Cells for Parkinson’s Treatment – Scientific American

For the past five decades pharmaceutical drugs like levodopa have been the gold standard for treating Parkinsons disease. These medications alleviate motor symptoms of the disease, but none of them can cure it. Patients with Parkinsons continue to lose dopamine neurons critical to the motor control centers of the brain. Eventually the drugs become ineffective and patients tremors get worse. They experience a loss of balance and a debilitating stiffness takes over their legs.

To replace the lost dopamine neurons, scientists have begun investigating stem cell therapy as a potential treatment or even a cure. But embryonic cells and adult stem cells have proved difficult to harness and transplant into the brain.

Now a study from the Karolinska Institute in Stockholm shows it is possible to coax the brains own astrocytescells that typically support and nurture neuronsinto producing a new generation of dopamine neurons. The reprogrammed cells display several of the properties and functions of native dopamine neurons and could alter the course of Parkinsons, according to the researchers. You can directly reprogram a cell that is already inside the brain and change the function in such a way that you can improve neurological symptoms, says senior author Ernest Arenas, a professor of medical biochemistry at Karolinska. Previously, scientists had to nudge specialized cells like neurons into becoming pluripotent cells before they could develop a different kind of specialized cell, he says. It was like having to erase all the written instructions for how a cell should develop and what job it should do and then rewriting them all over again. But Arenas and his team found a way to convert the instructions into a different set of commands without erasing them.

By adding a cocktail of three genes and a small RNA moleculeNEUROD1, ASCL1, LMX1A and miR-218, respectivelythe researchers forced astrocytes to transform directly into dopamine neurons. In culture, once human astrocytes were successfully reprogrammed this way, they looked and acted just like normal midbrain dopamine neurons. The reprogrammed cells grew axons, the long fibers that make connections with other neurons, fired electrical signals and released dopamine.

In mice, researchers first destroyed dopamine neurons in one part of the brain to produce a model of Parkinsons. Then they injected the cocktail of genes into the brain and observed the mice as they walked on a tiny treadmill. Within five weeks the mice began to walk straighter, their movements were more coordinated and their posture was improved.

The results, which were published in Nature Biotechnology today, open the door to a novel therapeutic approach for Parkinsons. Directly converting astrocytes already present in patients brains could eliminate the need to search for donor cells as well as avoid the risk of immunosuppression of transplanted cells. The treatments would also produce proteins involved in normal cellular processes and may therefore be less likely to cause side effects compared with current drugs. This is like stem cell 2.0. Its the next-generation approach to stem cell treatments and regenerative medicine, says James Beck, vice president and chief scientific officer, for the nonprofit Parkinsons Disease Foundation who was not involved with the research.* An estimated one million Americans and more than 10 million people worldwide live with Parkinsons. Replacing their lost dopamine neurons with reprogrammed brain cells could make management of their motor symptoms much easier, he notes. Instead of having to take eight pills or more every day in the later stages of the disease, people could reduce the amount of medication they have to take, maybe even down to zero.

But there are some caveats: Unfortunately, this is not going to halt the course of Parkinsons, Beck warns. If patients lose more dopamine neurons as the disease progresses, scientists or physicians may have to repeat the process of reprogramming replacement cells. And the disease often affects more than just dopamine neurons. There is a whole host of nonmotor symptoms that accompany Parkinsons, including cognitive impairment, depression, gastrointestinal complications and autonomic dysfunction. Motor improvement is only half the battle, according to Beck.

Researchers need to ensure the cocktail of added genes is standardized and produces robust cells in further tests. They will also need to verify that the process does not change other cells in the brain before it is ready for human clinical trials. Only a few small fetal and stem cell graft trials have been conducted so farand with mixed results, Beck said. But although this direct-reprogramming technique is being developed in parallel to stem cellbased therapies, it could be instructive as well as improve from clinical trials, eventually replacing stem cell therapy in the next decade, he adds. This is an insight into what the future of Parkinsons treatment holds.

*Editor's Note (04/11/17): This sentence was edited after posting to update James Beck's title.

Read more here:
Cell Therapy 2.0: Reprogramming the Brain's Own Cells for Parkinson's Treatment - Scientific American

Mighty morphed brain cells cure Parkinson’s in mice, but human trials still far off – STAT

M

ice that walk straight and fluidly dont usually make scientists exult, but these did: The lab rodents all had a mouse version of Parkinsons disease and only weeks before had barely been able to lurch and shuffle around their cages.

Using a trick from stem-cell science, researchers managed to restore the kind of brain cells whose death causes Parkinsons. And the mice walked almost normally.The same technique turned human brain cells, growing in a lab dish, into the dopamine-producing neurons that are AWOL in Parkinsons, scientists at Swedens Karolinska Institute reportedon Monday in Nature Biotechnology.

Success in lab mice and human cells is many difficult steps away from success in patients. The study nevertheless injected new life into a promising approach to Parkinsons that has suffered setback after setback replacing the dopamine neurons that are lost in the disease, crippling movement and eventually impairing mental function.

advertisement

This is not going to happen in five years or possibly even 10, but Im excited about the potential of this kind of cell replacement therapy, said James Beck, chief scientific officer of the Parkinsons Foundation, which was not involved in the study. It could really give life back to someone with Parkinsons disease.

There is no cure for Parkinsons, a neurodegenerative disease that affects an estimated 10 million people worldwide, most prominently actor Michael J. Fox. Drugs that enable the brain to make dopamine help only somewhat, often causing movement abnormalities called dyskinesia as well as bizarre side effects such as a compulsion to gamble; they do nothing to stop the neurodegeneration.

As Parkinsons patients wait, Fox Foundation and scientist feud over drug trial

Rather than replacing the missing dopamine, scientists led by Karolinskas Ernest Arenas tried to replace dopamine neurons but not in the way that researchers have been trying since the late 1980s. In that approach, scientists obtained tissue containing dopamine neurons from first-trimester aborted fetuses and implanted it intopatients brains.Although a 2001clinical trialfound that the transplants partly alleviated the rigidity and tremors of Parkinsons, the procedure caused serious dyskinesia in about 20 percent of patients, Beck said. More problematic is that fetal issue raises ethical concerns and is in short supply.

It was clear that usable fragments of brain tissue were extremely difficult to recover, said Dr. Curt Freed, of the University of Colorado, who pioneered that work.

Instead, several labs have therefore used stem cells to produce dopamine neurons in dishes. Transplanted into the brains of lab rats with Parkinsons, the neurons reduced rigidity, tremor, and other symptoms. Human studies are expected to begin in the US and Japan this year or next, Beck said.

In the Karolinska approach, there is no need to search for donor cells and no cell transplantation or [need for] immunosuppression to prevent rejection, Arenas told STAT. Instead, he and his team exploited one of the most startling recent discoveries in cell biology: that certain molecules can cause one kind of specialized cell, such as a skin cell, to pull a Benjamin Button, aging in reverse until they become like the embryonic cells called stem cells. Those can be induced to morph into any kind of cell heart, skin, muscle, and more in the body.

Muhammad Ali and Parkinsons disease: Was boxing to blame?

Arenas and his team filled harmless lentiviruses with a cocktail of four such molecules. Injected into the brains of mice with Parkinsons-like damage, the viruses infected plentifulbrain cells called astrocytes. (The brains support cells, astrocytes perform jobs like controlling blood flow.)The viruses also infected other kinds of cells, but their payload was designed to work only in astrocytes, and apparently caused no harm to the other cells.

The molecules, called transcription factors, reprogrammed some of the astrocytes to become dopamine neurons, which were first detected three weeks later in the mouse brains. The dopamine neurons were abundant 15 weeks later, an indication that after changing into dopamine neurons the astrocytes stayed changed.

Five weeks after receiving the injections, the mice, which used to have Parkinsons-like gait abnormalities, walked as well as healthy mice. That suggests that direct reprogramming [of brain cells] has the potential to become a novel therapeutic approach for Parkinsons, Arenas told STAT.

That could have value for preserving the brain circuitry destroyed by Parkinsons, said Colorados Freed.

A lot of hurdles need to be overcome before this becomes a Parkinsons treatment. The Trojan horse system for delivering the reprogramming molecules inside viruseswould need to turn more astrocytes into dopamine neurons and leave other kinds of cells alone: Although viruses getting into mouse brain cells apparently caused no harm, that might not be so in people. We will need to use virus with selective [attraction] for astrocytes, Arenas said.

The morphed cells would presumably be ravaged by whatever produced Parkinsons in the first place. But in other cell transplants, Arenas said, the disease catches up with transplanted cells in 15 to 20 years, buying patients a good period of time. He thinks it might be possible to give patients a single injection but hold off some of the reprogramming with a drug, turning it on when the brain again runs short of dopamine neurons.

The basic technology to develop such strategies currently exists, he said.

The Karolinska lab is working to make the techniquesafer and more effective, including by using viruses that would deliver reprogramming molecules only to astrocytes. We are open to collaborations aimed at human studies, Arenas said.

Would patients be willing to undergo brain injections? People with Parkinsons disease, Beck said, are willing to go through a lot for any hope of improvement.

Sharon Begley can be reached at sharon.begley@statnews.com Follow Sharon on Twitter @sxbegle

Read more:
Mighty morphed brain cells cure Parkinson's in mice, but human trials still far off - STAT

Stem Cell Firm Sued by One of Its Own Board Members – Courthouse News Service

StemedicaLAS VEGAS (CN) Founders of a Nevada stem cell research firm skimmed money from the $110 million they raised to develop treatments for Alzheimers disease, a member of the companys own board claims in court.

Tiara Holdings II LLC sued Stemedica Cell Technologies Inc. and its top three officers on April 6 in Clark County Court. The officers are CEO and Chairman of the Board Roger Howe, Vice Chairman and CEO Maynard Howe and President and Chief Medical Officer Nikolai Tankovich.

Dr. Anthony M. Marlon, a medical doctor and businessman, holds 430,000 shares of Stemedica through Tiara Holdings, where he is a member. He also is a member of the board of Stemedica, he says in the complaint.

He says Stemedicas founders have kept Tiara and independent auditors in the dark about its financial records and transactions.

Stemedicas founders have operated a nearly 10-year investment scheme, wherein they have raised over $110 million dollars from various individual investors for the purported purpose of funding and establishing a stem cell company, Tiara says in the lawsuit.

Tiara claims the Howes and Tankovich have used these investor funds, in whole or in part, to benefit themselves and their associates through excessive compensation and lavish personal expenses and related party transactions.

Stemedicas founds have concealed and perpetuated this fraud through purported operating subsidiaries, which permitted them to divert millions to benefit them without raising questions or concerns from Stemedicas investors and shareholders, Tiara says.

These subsidiaries apparently are the defendant Doe Individuals I-X and Roe Business Entities I-X.

Maynard Howe said in a telephone interview Monday that the allegations are completely false, and the matter is in an attorneys hands.

The most recent news statement on Stemedicas website, dated July 27, 2016, says the firm began its first U.S. clinical trials using adult allogeneic stem cells to treat Alzheimers disease, at nonparties Emory University and the University of California at Irvine.

Allogeneic cells are genetically different, though from individuals of the same species. Stemedicas statement says the clinical trials involve 40 subjects who were diagnosed with mild to moderate dementia caused by Alzheimers disease.

The National Institutes of Health says on its website: Stem cellshave the remarkable potential to develop into many different cell types in the body during early life and growth. In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive.

When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

In the lawsuit, Tiara says Stemedica sold securities that are not registered with any state or the federal government and raised about $15 million from July 2005 to June 2008. It says that money ultimately ended up in the bank account of entities and individuals controlled by Stemedicas founders.

Stemedica then raised more capital via securities sales, Tiara says. Marlon joined its board of directors in 2009. Tiara says Stemedica has not conducted a third-party audit of its finances and thwarted an audit attempt by refusing to pay invoices by the auditing firm, which stopped the process.

It claims that Stemedica officials stopped the audit to ensure it would not uncover any wrongdoing.

Disclosure would jeopardize their continued ability to defraud past and future investors out of millions, Tiara says.

Tiara asks the court to order an accounting of Stemedicas financial records, rescind securities subscription agreements and return Tiaras investment.

It also seeks damages and punitive damages for fraud, breach of fiduciary duty, unjust enrichment and bad faith.

Tiara is represented by James Pisanelli with Pisanelli Bice, who did not return a phone call seeking comment Monday.

Continue reading here:
Stem Cell Firm Sued by One of Its Own Board Members - Courthouse News Service

Pioneering work on stem-cell therapies at UW deserves state support – The Seattle Times

At the University of Washingtons Institute for Stem Cell and Regenerative Medicine, scientists and physicians are manipulating stem cells to heal and restore the function of hearts, eyes, kidneys and other tissues.

IF you have a heart attack, hopefully youll survive. But your body will be forever changed. The worlds best doctors cant undo the damage; instead, drugs and devices will help you live with a heart whose function too often dwindles.

The body cannot replace muscle cells that die in heart attacks maladies that help make heart failure the No. 1 global cause of death and our nations biggest health care expense. These patients face daily medication, decreased energy and, for the lucky 0.1 percent, the ability to qualify for an extraordinarily costly heart transplant and anti-rejection medication that also leaves them more vulnerable to other diseases.

Thanks to medical advances, heart failure has become a chronic condition that people are now managing for decades. The same is true for diabetes, kidney disease and arthritis. But with that longevity comes a tether to drug regimens whose costs rise seemingly at whim.

Dr. Charles Murry is interim director of UW Medicines Institute for Stem Cell and Regenerative Medicine.

These chronic diseases are a major reason that health-care costs hold center stage in Americans consciousness.

Amid our collective uncertainty, medical science offers one path of relief. Specifically, the engineering of human cells and tissues to restore vitality to poorly functioning organs.

The medical conditions named above share a common root not addressed by todays best care: The body is missing a population of cells that do critical work. If we could restore that population, we could cure many chronic diseases.

At the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM), scientists and physicians are manipulating stem cells to heal and restore the function of hearts, eyes, kidneys and other tissues.

This year, we also seek a first-time investment from our state Legislature.

Weve pioneered techniques to grow unlimited human heart muscle cells in the lab. We were the first to transplant these cells into injured hearts and repair the injury with new tissue growth. UW Medicine will begin first-in-human tests of these cells in Seattle in 2019.

If this one and done treatment prevents heart failure in even the sickest 10 percent of heart-attack patients, our nation could save a staggering $3.5 billion per year in health-care costs. More importantly, these patients will lead longer, healthier, more productive lives.

Other ISCRM scientists are pursuing a gene therapy for muscular dystrophy, a devastating illness that often strikes young boys. The therapy, tested in Labrador puppies that were paraplegic as a result of the same, naturally occurring muscle-wasting disease, had the dogs leaping and frolicking in just weeks. A clinical trial is planned for 2018.

We are similarly probing therapies for cancer, kidney failure, diabetes and Alzheimers. And were doing this with the Northwests entrepreneurial spirit: In the past decade, ISCRM has patented 250+ discoveries with commercial potential and started 20 companies.

Legislatures in at least 11 other states, including California, New York, Wisconsin, Minnesota and Maryland, have invested cumulative billions in regenerative medicine. Most of that funding has gone to university-based research centers like ours.

To this point there has been no state investment in ISCRM. Nevertheless we have built a world-class program with federal grants and private philanthropy. But those dollars come in boom-and-bust cycles, and what we need now is stable funding to maintain competitiveness.

For this reason, the UW seeks $6 million in operating funds from the Legislature, starting with the next biennium, to recruit and retain top scientists, fund promising results at early stages, and train young researchers and clinicians.

We are grateful, at this juncture, that the state Senate included us in its initial budget.

We ask all legislators to invest in the health of our residents and in the promise of what weve accomplished so far. With stem-cell biology, we are ready to rebuild solid tissues like the heart and potentially cure our nations greatest cause of death and health-care expense.

Clinical success will make Washington a destination for heart repair and other regenerative therapies. This race is ours to lose.

Continued here:
Pioneering work on stem-cell therapies at UW deserves state support - The Seattle Times

California’s stem cell agency invites public to San Diego event April 20 – The San Diego Union-Tribune

Stem cell cures are real, and more are on the way. Thats part of the message Californias stem cell agency will deliver in a special patient advocate event in La Jolla on Thursday, April 20.

To be held from noon to 1 p.m., the California Institute for Regenerative Medicine (CIRM) event will take place at the Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive, La Jolla, CA 92037. Its across the street from the Salk Institute for Biological Studies.

Stem cell experts will describe the work done and in progress with the numerous kinds of stem cells, embryonic and non-embryonic, and the public will be able to ask questions.

Those interested in attending can RSVP via the Web at j.mp/cirmsd1.

The event, Stem Cell Therapies and You, is sponsored by CIRM and UC San Diego, which hosts one of CIRMs alpha stem cell clinics.

Four speakers are to present their perspectives on stem cell research:

-- Catriona Jamieson, director of the CIRM UC San Diego Alpha Stem Cell Clinic and an expert on blood cancers

-- Jonathan Thomas, chairman of CIRMs governing board

-- Jennifer Briggs Braswell, executive director of the Sanford Stem Cell Clinical Center

-- David Higgins, patient advocate for Parkinsons on the CIRM board

Click on the video slide show below to hear an interview with Thomas about the event:

No stem cell treatments funded by CIRM have yet been approved for use. But dozens of clinical trials with these experimental therapies are under way, and some patients have already been cured.

Most spectacularly, a number of children born with bubble baby disease, or SCID, have been cured of their immune deficiency by CIRM-funded research. Scientists extracted some of their blood-forming stem cells, repaired the genetic defect and then reinfused them into the children. The stem cells proceeded to build a functional immune system.

CIRM was given $3 billion by the states voters in a $6 billion bond issue in 2004 to develop new disease treatments with stem cells. (The remaining $3 billion represents bond interest). The agency has spent most of that money, and soon voters may be asked whether to appropriate more funding.

Do these results justify the $3 billion allocation? And do they justify more funding, whether by the state, biomedical companies or private philanthropy? Was it wise for CIRM to focus so heavily on research in its first years? (The agency was recently scrutinized by the biomedical publication Stat for funding just a trickle of clinical trials.)

And if CIRM runs out of cash, as is projected to occur by 2020, what happens to the work in progress?

These are some of the questions CIRM faces as its cash winds down over the next few years.

Thomas, the CIRM board chairman, said the event is one of a series in which CIRM presents its evidence not only to patient advocates, but to the taxpayers who fund CIRM.

This will be the first one, Thomas said. Well have one in Los Angeles, and have one in San Francisco, one in Sacramento and maybe the Central Valley.

Well hear the latest with projects that are in clinical trials. We have 30-plus now in clinical trials, Thomas said. A great many of those are being undertaken at our alpha stem cell clinics. A prominent one of course is at UC San Diego.

So well talk about what theyre doing but also about whats happening elsewhere in the network at the other alpha stem cell clinics.

bradley.fikes@sduniontribune.com

(619) 293-1020

View original post here:
California's stem cell agency invites public to San Diego event April 20 - The San Diego Union-Tribune

Cell (biology) – Wikipedia

The cell (from Latin cella, meaning "small room"[1]) is the basic structural, functional, and biological unit of all known living organisms. A cell is the smallest unit of life that can replicate independently, and cells are often called the "building blocks of life". The study of cells is called cell biology.

Cells consist of cytoplasm enclosed within a membrane, which contains many biomolecules such as proteins and nucleic acids.[2] Organisms can be classified as unicellular (consisting of a single cell; including bacteria) or multicellular (including plants and animals). While the number of cells in plants and animals varies from species to species, humans contain more than 10 trillion (1012) cells. Most plant and animal cells are visible only under a microscope, with dimensions between 1 and 100micrometres.[4]

The cell was discovered by Robert Hooke in 1665, who named the biological unit for its resemblance to cells inhabited by Christian monks in a monastery.[5][6]Cell theory, first developed in 1839 by Matthias Jakob Schleiden and Theodor Schwann, states that all organisms are composed of one or more cells, that cells are the fundamental unit of structure and function in all living organisms, that all cells come from preexisting cells, and that all cells contain the hereditary information necessary for regulating cell functions and for transmitting information to the next generation of cells.[7] Cells emerged on Earth at least 3.5 billion years ago.[8][9][10]

Cells are of two types, eukaryotic, which contain a nucleus, and prokaryotic, which do not. Prokaryotes are single-celled organisms, while eukaryotes can be either single-celled or multicellular.

Prokaryotic cells were the first form of life on Earth, characterised by having vital biological processes including cell signaling and being self-sustaining. They are simpler and smaller than eukaryotic cells, and lack membrane-bound organelles such as the nucleus. Prokaryotes include two of the domains of life, bacteria and archaea. The DNA of a prokaryotic cell consists of a single chromosome that is in direct contact with the cytoplasm. The nuclear region in the cytoplasm is called the nucleoid. Most prokaryotes are the smallest of all organisms ranging from 0.5 to 2.0m in diameter.[12]

A prokaryotic cell has three architectural regions:

Plants, animals, fungi, slime moulds, protozoa, and algae are all eukaryotic. These cells are about fifteen times wider than a typical prokaryote and can be as much as a thousand times greater in volume. The main distinguishing feature of eukaryotes as compared to prokaryotes is compartmentalization: the presence of membrane-bound organelles (compartments) in which specific metabolic activities take place. Most important among these is a cell nucleus, an organelle that houses the cell's DNA. This nucleus gives the eukaryote its name, which means "true kernel (nucleus)". Other differences include:

All cells, whether prokaryotic or eukaryotic, have a membrane that envelops the cell, regulates what moves in and out (selectively permeable), and maintains the electric potential of the cell. Inside the membrane, the cytoplasm takes up most of the cell's volume. All cells (except red blood cells which lack a cell nucleus and most organelles to accommodate maximum space for hemoglobin) possess DNA, the hereditary material of genes, and RNA, containing the information necessary to build various proteins such as enzymes, the cell's primary machinery. There are also other kinds of biomolecules in cells. This article lists these primary components of the cell, then briefly describes their function.

The cell membrane, or plasma membrane, is a biological membrane that surrounds the cytoplasm of a cell. In animals, the plasma membrane is the outer boundary of the cell, while in plants and prokaryotes it is usually covered by a cell wall. This membrane serves to separate and protect a cell from its surrounding environment and is made mostly from a double layer of phospholipids, which are amphiphilic (partly hydrophobic and partly hydrophilic). Hence, the layer is called a phospholipid bilayer, or sometimes a fluid mosaic membrane. Embedded within this membrane is a variety of protein molecules that act as channels and pumps that move different molecules into and out of the cell. The membrane is said to be 'semi-permeable', in that it can either let a substance (molecule or ion) pass through freely, pass through to a limited extent or not pass through at all. Cell surface membranes also contain receptor proteins that allow cells to detect external signaling molecules such as hormones.

The cytoskeleton acts to organize and maintain the cell's shape; anchors organelles in place; helps during endocytosis, the uptake of external materials by a cell, and cytokinesis, the separation of daughter cells after cell division; and moves parts of the cell in processes of growth and mobility. The eukaryotic cytoskeleton is composed of microfilaments, intermediate filaments and microtubules. There are a great number of proteins associated with them, each controlling a cell's structure by directing, bundling, and aligning filaments. The prokaryotic cytoskeleton is less well-studied but is involved in the maintenance of cell shape, polarity and cytokinesis.[17] The subunit protein of microfilaments is a small, monomeric protein called actin. The subunit of microtubules is a dimeric molecule called tubulin. Intermediate filaments are heteropolymers whose subunits vary among the cell types in different tissues. But some of the subunit protein of intermediate filaments include vimentin, desmin, lamin (lamins A, B and C), keratin (multiple acidic and basic keratins), neurofilament proteins (NF - L, NF - M).

Two different kinds of genetic material exist: deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). Cells use DNA for their long-term information storage. The biological information contained in an organism is encoded in its DNA sequence. RNA is used for information transport (e.g., mRNA) and enzymatic functions (e.g., ribosomal RNA). Transfer RNA (tRNA) molecules are used to add amino acids during protein translation.

Prokaryotic genetic material is organized in a simple circular DNA molecule (the bacterial chromosome) in the nucleoid region of the cytoplasm. Eukaryotic genetic material is divided into different, linear molecules called chromosomes inside a discrete nucleus, usually with additional genetic material in some organelles like mitochondria and chloroplasts (see endosymbiotic theory).

A human cell has genetic material contained in the cell nucleus (the nuclear genome) and in the mitochondria (the mitochondrial genome). In humans the nuclear genome is divided into 46 linear DNA molecules called chromosomes, including 22 homologous chromosome pairs and a pair of sex chromosomes. The mitochondrial genome is a circular DNA molecule distinct from the nuclear DNA. Although the mitochondrial DNA is very small compared to nuclear chromosomes, it codes for 13 proteins involved in mitochondrial energy production and specific tRNAs.

Foreign genetic material (most commonly DNA) can also be artificially introduced into the cell by a process called transfection. This can be transient, if the DNA is not inserted into the cell's genome, or stable, if it is. Certain viruses also insert their genetic material into the genome.

Organelles are parts of the cell which are adapted and/or specialized for carrying out one or more vital functions, analogous to the organs of the human body (such as the heart, lung, and kidney, with each organ performing a different function). Both eukaryotic and prokaryotic cells have organelles, but prokaryotic organelles are generally simpler and are not membrane-bound.

There are several types of organelles in a cell. Some (such as the nucleus and golgi apparatus) are typically solitary, while others (such as mitochondria, chloroplasts, peroxisomes and lysosomes) can be numerous (hundreds to thousands). The cytosol is the gelatinous fluid that fills the cell and surrounds the organelles.

-4500

-4000

-3500

-3000

-2500

-2000

-1500

-1000

-500

0

Many cells also have structures which exist wholly or partially outside the cell membrane. These structures are notable because they are not protected from the external environment by the semipermeable cell membrane. In order to assemble these structures, their components must be carried across the cell membrane by export processes.

Many types of prokaryotic and eukaryotic cells have a cell wall. The cell wall acts to protect the cell mechanically and chemically from its environment, and is an additional layer of protection to the cell membrane. Different types of cell have cell walls made up of different materials; plant cell walls are primarily made up of cellulose, fungi cell walls are made up of chitin and bacteria cell walls are made up of peptidoglycan.

A gelatinous capsule is present in some bacteria outside the cell membrane and cell wall. The capsule may be polysaccharide as in pneumococci, meningococci or polypeptide as Bacillus anthracis or hyaluronic acid as in streptococci. Capsules are not marked by normal staining protocols and can be detected by India ink or methyl blue; which allows for higher contrast between the cells for observation.[19]:87

Flagella are organelles for cellular mobility. The bacterial flagellum stretches from cytoplasm through the cell membrane(s) and extrudes through the cell wall. They are long and thick thread-like appendages, protein in nature. A different type of flagellum is found in archaea and a different type is found in eukaryotes.

A fimbria also known as a pilus is a short, thin, hair-like filament found on the surface of bacteria. Fimbriae, or pili are formed of a protein called pilin (antigenic) and are responsible for attachment of bacteria to specific receptors of human cell (cell adhesion). There are special types of specific pili involved in bacterial conjugation.

Between successive cell divisions, cells grow through the functioning of cellular metabolism. Cell metabolism is the process by which individual cells process nutrient molecules. Metabolism has two distinct divisions: catabolism, in which the cell breaks down complex molecules to produce energy and reducing power, and anabolism, in which the cell uses energy and reducing power to construct complex molecules and perform other biological functions. Complex sugars consumed by the organism can be broken down into simpler sugar molecules called monosaccharides such as glucose. Once inside the cell, glucose is broken down to make adenosine triphosphate (ATP), a molecule that possesses readily available energy, through two different pathways.

Cell division involves a single cell (called a mother cell) dividing into two daughter cells. This leads to growth in multicellular organisms (the growth of tissue) and to procreation (vegetative reproduction) in unicellular organisms. Prokaryotic cells divide by binary fission, while eukaryotic cells usually undergo a process of nuclear division, called mitosis, followed by division of the cell, called cytokinesis. A diploid cell may also undergo meiosis to produce haploid cells, usually four. Haploid cells serve as gametes in multicellular organisms, fusing to form new diploid cells.

DNA replication, or the process of duplicating a cell's genome, always happens when a cell divides through mitosis or binary fission. This occurs during the S phase of the cell cycle.

In meiosis, the DNA is replicated only once, while the cell divides twice. DNA replication only occurs before meiosis I. DNA replication does not occur when the cells divide the second time, in meiosis II.[20] Replication, like all cellular activities, requires specialized proteins for carrying out the job.

Cells are capable of synthesizing new proteins, which are essential for the modulation and maintenance of cellular activities. This process involves the formation of new protein molecules from amino acid building blocks based on information encoded in DNA/RNA. Protein synthesis generally consists of two major steps: transcription and translation.

Transcription is the process where genetic information in DNA is used to produce a complementary RNA strand. This RNA strand is then processed to give messenger RNA (mRNA), which is free to migrate through the cell. mRNA molecules bind to protein-RNA complexes called ribosomes located in the cytosol, where they are translated into polypeptide sequences. The ribosome mediates the formation of a polypeptide sequence based on the mRNA sequence. The mRNA sequence directly relates to the polypeptide sequence by binding to transfer RNA (tRNA) adapter molecules in binding pockets within the ribosome. The new polypeptide then folds into a functional three-dimensional protein molecule.

Unicellular organisms can move in order to find food or escape predators. Common mechanisms of motion include flagella and cilia.

In multicellular organisms, cells can move during processes such as wound healing, the immune response and cancer metastasis. For example, in wound healing in animals, white blood cells move to the wound site to kill the microorganisms that cause infection. Cell motility involves many receptors, crosslinking, bundling, binding, adhesion, motor and other proteins.[21] The process is divided into three steps protrusion of the leading edge of the cell, adhesion of the leading edge and de-adhesion at the cell body and rear, and cytoskeletal contraction to pull the cell forward. Each step is driven by physical forces generated by unique segments of the cytoskeleton.[22][23]

Multicellular organisms are organisms that consist of more than one cell, in contrast to single-celled organisms.[24]

In complex multicellular organisms, cells specialize into different cell types that are adapted to particular functions. In mammals, major cell types include skin cells, muscle cells, neurons, blood cells, fibroblasts, stem cells, and others. Cell types differ both in appearance and function, yet are genetically identical. Cells are able to be of the same genotype but of different cell type due to the differential expression of the genes they contain.

Most distinct cell types arise from a single totipotent cell, called a zygote, that differentiates into hundreds of different cell types during the course of development. Differentiation of cells is driven by different environmental cues (such as cellcell interaction) and intrinsic differences (such as those caused by the uneven distribution of molecules during division).

Multicellularity has evolved independently at least 25 times,[25] including in some prokaryotes, like cyanobacteria, myxobacteria, actinomycetes, Magnetoglobus multicellularis or Methanosarcina. However, complex multicellular organisms evolved only in six eukaryotic groups: animals, fungi, brown algae, red algae, green algae, and plants.[26] It evolved repeatedly for plants (Chloroplastida), once or twice for animals, once for brown algae, and perhaps several times for fungi, slime molds, and red algae.[27] Multicellularity may have evolved from colonies of interdependent organisms, from cellularization, or from organisms in symbiotic relationships.

The first evidence of multicellularity is from cyanobacteria-like organisms that lived between 3 and 3.5 billion years ago.[25] Other early fossils of multicellular organisms include the contested Grypania spiralis and the fossils of the black shales of the Palaeoproterozoic Francevillian Group Fossil B Formation in Gabon.[28]

The evolution of multicellularity from unicellular ancestors has been replicated in the laboratory, in evolution experiments using predation as the selective pressure.[25]

The origin of cells has to do with the origin of life, which began the history of life on Earth.

There are several theories about the origin of small molecules that led to life on the early Earth. They may have been carried to Earth on meteorites (see Murchison meteorite), created at deep-sea vents, or synthesized by lightning in a reducing atmosphere (see MillerUrey experiment). There is little experimental data defining what the first self-replicating forms were. RNA is thought to be the earliest self-replicating molecule, as it is capable of both storing genetic information and catalyzing chemical reactions (see RNA world hypothesis), but some other entity with the potential to self-replicate could have preceded RNA, such as clay or peptide nucleic acid.[29]

Cells emerged at least 3.5 billion years ago.[8][9][10] The current belief is that these cells were heterotrophs. The early cell membranes were probably more simple and permeable than modern ones, with only a single fatty acid chain per lipid. Lipids are known to spontaneously form bilayered vesicles in water, and could have preceded RNA, but the first cell membranes could also have been produced by catalytic RNA, or even have required structural proteins before they could form.[30]

The eukaryotic cell seems to have evolved from a symbiotic community of prokaryotic cells. DNA-bearing organelles like the mitochondria and the chloroplasts are descended from ancient symbiotic oxygen-breathing proteobacteria and cyanobacteria, respectively, which were endosymbiosed by an ancestral archaean prokaryote.

There is still considerable debate about whether organelles like the hydrogenosome predated the origin of mitochondria, or vice versa: see the hydrogen hypothesis for the origin of eukaryotic cells.

Excerpt from:
Cell (biology) - Wikipedia