Today was the doom day for may CTIC  investors.  Two decade old company had half a billion market capitalization a few days back.  

Advisers to the Food and Drug Administration, in a 9-0 vote, said the company’s single trial of pixantrone was inadequate to back marketing approval.

“I don’t see this as being a well-designed or well-executed study,” said Dr. Wyndham Wilson, a panel member and a lymphoma specialist at the National Cancer Institute.

Shares of Cell Therapeutics lost 44 cents, or 48 percent, to close at 47 cents Monday. Trading had been halted until the early afternoon. The stock has ranged between 11 cents and $2.23 over the last year. Shares had been halted ahead of the panel vote.

The panel decision makes it unlikely the FDA will approve pixantrone without additional data. The agency usually follows panel recommendations, and FDA scientists who spoke to the committee also were critical of the company’s study.

Cell Therapeutics wants to sell pixantrone under the name Pixuvri for treating non-Hodgkin’s lymphoma that has stopped responding to other treatments. The blood cancer affects about 66,000 Americans annually.

The company said pixantrone offered an effective therapy for patients whose cancer worsened after at least two prior chemotherapy regimens. Patients at that stage have no approved treatments and often live less than six months.

In the Cell Therapeutics study of 140 patients, 20 percent had a major decrease in their disease if they got pixantrone, compared to about 6 percent with a different medicine.

“It is clear to me pixantrone provides an important benefit in these very poor prognosis patients,” said Dr. John Leonard, a cancer specialist at Weill Cornell Cancer Center and a Cell Therapeutics consultant.

But FDA reviewers questioned the company’s conclusions. They said the study tested less than half the number of people originally planned and included just eight U.S. patients. The agency also said heart damage and decreased white blood cells were more common with pixantrone versus other cancer drugs.

Despite their concerns, several panel members said they saw signs the drug could be helpful and urged additional study.

“This drug has some activity. I don’t think anybody on this committee would debate that point. The population enrolled in this study, however, doesn’t reflect the U.S. population of lymphoma patients,” said Dr. Mikkael Sekeres, an oncologist at the Cleveland Clinic.

In a statement after the panel vote, Cell Therapeutics Chief Executive James Bianco said the company was “committed to working closely with the FDA to address the committee’s comments as quickly as we can.”

“We continue to believe that pixantrone should be considered as a treatment option for patients” with aggressive NHL, he said.

The FDA is scheduled to make a decision on the drug by April 23. Swiss drugmaker Novartis (NOVN.VX) has an option for a worldwide license to develop and sell the drug.

Pharma companies at the 2009 J.P. Morgan Healthcare Conference cited a willingness to do ‘more deals and bigger deals’.  This will hopefully give the required stimulus for the Biotech sector.  J.P. Morgan has released a list of Biotech stocks with “high strategic value” in 2010.

1. Acorda Therapeutics(ACOR)
2. AMAG Pharmaceuticals(AMAG)
3. OSI Pharmaceuticals(OSIP)
4. Medivation(MDVN)
5. United Therapeutics(UTHR)
6. Vertex Pharmaceuticals(VRTX)
7. Human Genome Sciences(HGSI)
8. Seattle Genetics(SGEN)
9. Savient Pharmaceuticals(SVNT)

Lehman Brothers also issued its 2010 biotech forecast “Opportunities for the U.S. biotechnology group are selective and we tend to prefer small-mid cap stocks over large-cap stocks overall”.  Their list includes:

1. Amylin Pharmaceuticals(AMLN)
2. Cephalon(CEPH)
3. Human Genome Sciences
4. Onyx Pharmaceuticals(ONXX)
5. Regeneron Pharmaceuticals(REGN)

In advance of the postponed advisory panel meeting, the FDA issued a critical review of pixantrone, highlighting numerous concerns about pixantrone’s safety and efficacy.

Shares of Cell Therapeutics were down 10% to 60 cents Monday after auditors attached a “going concern” letter to the company’s 10-K annual report filed with the Securities and Exchange Commission Friday.  For the investor, if the drug works and gets approval, auditors concern doesn’t matter.  But what if the drug fails to get an approval?

Other drug of Opaxio as a treatment for lung cancer is irrelevant as better alternatives are already in the market.  So  Pixantrone approval is very critical to the company. 

Cell Therapeutics’ total share count reached more than 615 million at the end of January, compared to 296 million shares at the end of January 2009, according to SEC filings.  Whereas, in 2009, the top five Cell Therapeutics executives earned $32 million in total compensation, most of which came in the form of company stock awards.  A classic case of Executives feasting on investors money.

But a few weeks after taking their first dose, nearly all of them began to recover.

Lee Reyes (30) of California, who had begun using a feeding tube because of a growth pressing against his throat, bit into a cinnamon roll. Randy Williams, 46, who drove 600 miles from his home in Arkansas to the M D Anderson Cancer Center in Houston for the experimental drug, rolled out of bed. “Something’s working,” he thought, “because nothing’s hurting.

It was a sweet moment, in autumn 2008, for Dr Keith Flaherty, the University of Pennsylvania oncologist leading the drug’s first clinical trial. A new kind of cancer therapy, it was tailored to a particular genetic mutation that was driving the disease, and after six years of disappointments, his faith in the promise of such a targeted approach finally seemed borne out. His collaborators at five other major cancer centres, melanoma clinicians who had tested dozens of potential therapies for their patients with no success, were equally elated.

In a kind of pinch-me exercise, the six doctors sent one another before and after CT scans of their patients.
The trial of PLX4032 offers a glimpse at how doctors, patients and drug developers navigate a medical frontier as more drugs tailored to the genetic profile of a cancer are being widely tested on humans for the first time.

Throughout the fall, the only two patients on the trial whose tumours continued to grow were the ones who did not have the particular gene mutation for which the drug had been designed. They were removed from the trial. By late December, tumours in the 11 patients who did have the mutation had shrunk. Those involved in the trial held their collective breath waiting to see how long the remissions would last.

One of them was Mark Bunting, 52, an airline pilot from Utah. His initial scan in early October showed the cancer in his bones, an incursion considered virtually impossible to reverse. After two months on the drug, it had all but disappeared. From New York, Paul B Chapman of Memorial Sloan-Kettering Cancer Center, perhaps the most determined sceptic of the group, acknowledged, “This looks impressive.”

It was a far cry from where they had been a year earlier, when a previous incarnation of the drug had no effect. Urged on by Flaherty and Chapman, the companies that owned it had spent months devising a new formulation that could be absorbed at higher doses.

But the new drug, still in the earliest phase of testing, had to pass several more hurdles before federal regulators would determine whether it was safe and effective enough for widespread use.

In December, as the doctors added more patients to the Phase 1 trial, looking for the highest dose they could give without intolerable side effects, they scrambled to prepare slides with graphs and statistics to convince the Food and Drug Administration that the drug should be tested in a larger Phase 2 trial. The agency required a summary of any and all side-effects there had been only a few and any deaths of patients on the study; thankfully, there had been none since the drug was reformulated. In a matter of days they needed to submit their findings for a prestigious meeting of clinical oncologists in June.

“We anticipate nice weather and look forward to meeting all our investors. But even if Mother Nature doesn’t cooperate, our Grand Opening will go on,” said Matthew Schissler, co-founder and CEO. The facility is at 1857 Helm Drive, Las Vegas 89119, near the city’s airport in the Spencer Airport Business Park.

Leukemias , Lymphomas and other Blood Cancers

• Acute Biphenotypic Leukemia†
• Acute Lymphocytic Leukemia (ALL)
• Acute Myelogenous Leukemia (AML)
• Acute Undifferentiated Leukemia†
• Adult T Cell Leukemia/Lymphoma
• Chronic Lymphocytic Leukemia (CLL)
• Chronic Myelogenous Leukemia (CML)
• Hodgkin’s Lymphoma
• Juvenile Chronic Myelogenous Leukemia (JCML)
• Juvenile Myelomonocytic Leukemia (JMML)
• Multiple Myeloma
• Myeloid/Natural Killer (NK) Cell Precursor Acute Leukemia
• Non-Hodgkin’s Lymphoma
• Prolymphocytic Leukemia
• Plasma Cell Leukemia
• Waldenstrom’s Macroglobulinemia

Other Cancers

1. Brain Tumors††
2. Ewing Sarcoma†
3. Neuroblastoma
4. Ovarian Cancer††
5. Renal Cell Carcinoma††
6. Rhabdomyosarcoma
7. Small-Cell Lung Cancer††
8. Testicular Cancer††
9. Thymoma (Thymic Carcinoma)

Bone Marrow Failure Disorders

1. Amegakaryocytosis
2. Aplastic Anemia (Severe)
3. Blackfan-Diamond Anemia
4. Congenital Cytopenia†
5. Congenital Dyserythropoietic Anemia
6. Dyskeratosis Congenita
7. Fanconi Anemia
8. Paroxysmal Nocturnal Hemoglobinuria (PNH)
9. Pure Red Cell Aplasia

Hemoglobinopathies

1. Beta Thalassemia Major
   Sickle Cell Disease

Histiocytic Disorders

1. Familial Erythrophagocytic Lymphohistiocytosis
2. Hemophagocytosis
3. Langerhans’ Cell Histiocytosis (Histiocytosis X)

Myelodysplastic/Myeloproliferative Disorders

1. Acute Myelofibrosis†
2. Agnogenic Myeloid Metaplasia (Myelofibrosis)†
3. Amyloidosis
4. Chronic Myelomonocytic Leukemia (CMML)
5. Essential Thrombocythemia†
6. Polycythemia Vera†
7. Refractory Anemias (RA) including:
   • Refractory Anemia with Excess Blasts (RAEB)
   • Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
   • Refractory Anemia with Ringed Sideroblasts (RARS)

Inherited Metabolic Disorders

1. Adrenoleukodystrophy
2. Fucosidosis
3. Gaucher Disease†
4. Hunter Syndrome (MPS-II)
5. Hurler Syndrome (MPS-IH)
6. Krabbe Disease
7. Lesch-Nyhan Syndrome
8. Mannosidosis†
9. Maroteaux-Lamy Syndrome (MPS-VI)
10. Metachromatic Leukodystrophy
11. Mucolipidosis II (I-cell Disease)†
12. Neuronal Ceroid Lipofuscinosis (Batten Disease)†
13. Niemann-Pick Disease†
14. Sandhoff Disease†
15. Sanfilippo Syndrome (MPS-III)
16. Scheie Syndrome (MPS-IS)
17. Sly Syndrome (MPS-VII)
18. Tay Sachs†
19. Wolman Disease

Inherited Immune System Disorders

1. Chronic Granulomatous Disease
2. Congenital Neutropenia
3. Leukocyte Adhesion Deficiency
4. Severe Combined Immunodeficiencies (SCID) including:
   • Adenosine Deaminase Deficiency†
   • Bare Lymphocyte Syndrome
   • Chediak-Higashi Syndrome†
   • Kostmann Syndrome
   • Omenn Syndrome
   • Purine Nucleoside Phosphorylase Deficiency
   • Reticular Dysgenesis
5. Wiskott-Aldrich Syndrome
6. X-Linked Lymphoproliferative Disorder

Other Inherited Disorders

1. Cartilage-Hair Hypoplasia
2. Congenital Erythropoietic Porphyria (Gunther Disease)
3. DiGeorge Syndrome
4. Osteopetrosis

Other

1. Chronic Active Epstein Barr
2. Evans Syndrome
3. Multiple Sclerosis††
4. Rheumatoid Arthritis††
5. Systemic Lupus Erythematosus††
6. Thymic Dysplasia

Some distance away to the south, Dr Hemangi Sane spent the 31st in her Dadar flat making a mental list of changes. “My shoulders are no longer droopy. I can speak clearly and for hours at a stretch. I can walk without swaying,’’ the 34-year-old listed. Until August, she spoke mostly in whispers and, while walking with assistance, could only register the designs on the floor. “Now, I can hold my neck up and watch all the beautiful colours,’’ she added.

What has brought about the subtle changes in Ankur and Hemangi is a dose of stem cell therapy at the civic-run Sion Hospital four months back. A fortnight back, the US gave the green signal for the first clinical trial of stem cell therapy for spinal cord patients, but in Mumbai the therapy has for the past two years been bringing smiles and enhancing the quality of life for seriously ill patients.

While Ankur suffers from muscular dystrophy, a disorder in which the muscles waste away, Hemangi has motor neuron disease, which is better known as the condition that crippled British scientist Stephen Hawking. Both are terminal conditions.

Stem cells are the master cells of the body, having the ability to take on the function of any organ cell. In the last five years, many centres across India have been offering stem cell therapy to patients but a complete cure is still a distant dream, admit doctors.

Sceptics abound. A senior doctor who doesn’t want to be named feels that stem cells are, at present, over-rated. “There is no known cure for progressive neurological disorders. While studies are being done across the world, none have shown any concrete results in academic sense,’’ said the doctor. Another doctor alleged that the talk of stem cell therapy only resulted in patients making a beeline for private clinics that offered the same therapy for lakhs of rupees. “Patients and their families are so desperate that they don’t really think long-term,’’ he added.

But the degree of changes in the patient’s life is there for all to see. Incidentally, two former city mayors played a role in getting both Ankur and Hemangi to the Sion Hospital’s stem cell therapy laboratory. While Dr Shubha Raul was instrumental in getting the hospital team led by neurosurgeon Dr Alok Sharma to evaluate Ankur, Vishaka Raut is Hemangi’s aunt.

“My aunt was confident that stem cells would help me, but I was sceptical, having tried out everything from ayurveda to cosmic therapy,’’ said Dr Hemangi, who was working as an internal medicine specialist in New York when the diagnosis was made.

The first patient whom Sion Hospital treated, Ravindra Ahire, who met with a bike accident in April 2007 and sustained severe spinal cord injuries, is, in fact, walking with a stick. “We have treated 76 patients with various diseases so far and most are doing much better than what they were doing before the therapy,’’ said Dr Sharma, who began the stem cell treatment after years of conducting laboratory studies.

“Muscular atrophy and motor neuron disease can bring about a complete change in the patient’s life. By offering them stem cell therapy, we have managed to brighten their lives to an extent. They are more independent than before,’’ said Dr Sandhya Kamat, dean of Sion Hospital.

In fact, Dr Sharma feels that Ankur’s recovery “after stem cell therapy is nothing short of miraculous’’. For one, Ankur was the first patient on a ventilator to be treated by the Sion team.

“While the results of stem cell therapy with spinal cord injury and multiple sclerosis patients are good, the results with motor neuron disease are not as encouraging. Yet, approximately half the patients do show a positive response. Fortunately, Dr Hemangi was one of them,’’ said Dr Sharma.

HOFFMAN ESTATES:  Life Spine announced today that the U.S. Food and Drug Administration (FDA) has given 510(k) marketing approval to the SENTINEL Occipito-Cervico-Thoracic (OCT) System.

The SENTINEL OCT System provides rigid stabilization and promotes fusion from the occiput to thoracic spine, and features streamlined implants and instruments to address complicated procedures. Polyaxial screws are available in multiple diameters and lengths, and offer a generous cone angulation, which simplifies alignment with the rod and minimizes rod contouring.

The addition of the SENTINEL OCT System to the current broad product offering is a watershed event providing the ability to offer surgeons a full range of solutions to address spinal pathologies from the occiput to the sacrum. The SENTINEL OCT System makes its mark as the twentieth 510(k) approval to date for Life Spine.

Life Spine is dedicated to improving the quality of life for spinal patients by increasing procedural efficiency and efficacy through innovative design, uncompromising quality standards, and the most technologically advanced manufacturing platforms. Life Spine, which is privately held, is based in Hoffman Estates, Illinois. For more information, please visit http://www.lifespine.com.

Biotechnology is technology based on biology, agriculture, food science, and medicine. Modern use of the term usually refers to genetic engineering as well as cell- and tissue culture technologies.

Biotechnology has applications in four major industrial areas, including health care (medical), crop production and agriculture, non food (industrial) uses of crops and other products (e.g. biodegradable plastics, vegetable oil, biofuels), and environmental uses.

For example, one application of biotechnology is the directed use of organisms for the manufacture of organic products (examples include beer and milk products). Another example is using naturally present bacteria by the mining industry in bioleaching. Biotechnology is also used to recycle, treat waste, clean up sites contaminated by industrial activities (bioremediation), and also to produce biological weapons.

There are different brances of  Biotechnology as a science. Red biotechnology is applied to medical processes. Some examples are the designing of organisms to produce antibiotics, and the engineering of genetic cures through genomic manipulation.  In this article we will look into red biotechnology primarily.

Most traditional pharmaceutical drugs are relatively simple molecules that have been found primarily through trial and error to treat the symptoms of a disease or illness. Biopharmaceuticals are large biological molecules known as proteins and these usually target the underlying mechanisms and pathways of a malady (but not always, as is the case with using insulin to treat type 1 diabetes mellitus, as that treatment merely addresses the symptoms of the disease, not the underlying cause which is autoimmunity); it is a relatively young industry. They can deal with targets in humans that may not be accessible with traditional medicines. A patient typically is dosed with a small molecule via a tablet while a large molecule is typically injected.

Small molecules are manufactured by chemistry but larger molecules are created by living cells such as those found in the human body: for example, bacteria cells, yeast cells, animal or plant cells.

Modern biotechnology is often associated with the use of genetically altered microorganisms such as E. coli or yeast for the production of substances like synthetic insulin or antibiotics. It can also refer to transgenic animals or transgenic plants, such as Bt corn. Genetically altered mammalian cells, such as Chinese Hamster Ovary (CHO) cells, are also used to manufacture certain pharmaceuticals. Another promising new biotechnology application is the development of plant-made pharmaceuticals.

The truth is, it’s hard to find a successful large-cap biotechnology company because most get acquired before they reach large-cap status. Companies in biotech  industry are always in need of cash, whereas  big pharmaceutical firms are cash-rich and are  in desperate need of pipeline drugs.  These small-cap biotech start up firms are there easy catch.

Most biotechnology companies are started by a brilliant scientist with an idea, sadly a brilliant scientist is not always a brilliant businessman.  These companies dream of becoming the next Genentech, Amgen, or Genzyme. But building a successful biotechnology company takes a lot more than just a brilliant scientist with a great idea.

Most of the biotech companies trade at sub-$1 levels, because biotech drug development is both extremely expensive and very high-risk. As a result, most biotechnology companies either fail in the clinical program or run out of money while trying.  Most pharmaceutical names are sitting on billions of dollars in cash, while their late-stage research pipelines are drying up.

Most Biotech company management  are overconfident, under experienced, and fail to truly understand the competitive landscape for their drug. This results in biotech companies reluctant to partner with pharmaceutical companies too early in the development process, because they think they can either do it alone or they will command a far greater price the further along they develop the asset. 

In theory, this is true. The difficulties of commercializing a successful biotech drug, however, only increase as development pushes further along. And successfully completing a Phase III trial is not the finish line, it is just the start of a whole new marathon that now includes preparing an NDA/BLA, getting that application passed by the FDA, manufacturing the drug, and then, managing a sales force, as well as Wall Street expectations. As a result, an asset that was partnerable after encouraging Phase II data becomes unpartnerable after the Phase III trial fails or the FDA requests additional data prior to approval.