1/29/11

Anal Pains

 Anal Pain
Pain coming from the bottom area is not something that we are used to feeling and, as a consequence, anal pain can cause a lot of worries.

What sort of pain is it?

 -A knife-like pain when you have your bowels open, and which may last for 10–15 minutes afterwards, is probably caused by an anal fissure. Some people describe it as like ‘passing glass’. In addition to the pain, you may notice some bright red blood on the toilet paper.
- A similar knife-like pain can be caused by herpes simplex virus.
- A nagging, aching discomfort made worse by defecation could be due to piles.
- A throbbing pain, worsening over a few days, and bad enough to disturb your sleep, is likely to be caused by an abscess.
- An occasional, severe, cramp-like pain deep in the anal canal, lasting about half an hour, is probably a condition called proctalgia fugax.
- A continuous aching pain in the anus needs to be sorted out by your doctor. It is most often caused by a back problem (when a part of the spine presses on a nerve).

Anal fissure

An anal fissure is a split in the anal skin, just inside the anus, usually towards the back. The pain of an anal fissure feels like being cut with glass when you have your bowels open and afterwards. It is worse when you have had a hard bowel motion. You may notice some bright red blood on the lavatory paper at the same time. You may be able to feel a small lump alongside the crack; this is a ‘skin tag’.
Anal fissures are most common in teenagers and young adults, and there has often been a period of constipation beforehand. Sometimes they follow childbirth. They usually heal on their own, but this often takes several weeks and the scar may split again.

What you can do. Do not feel anxious about the fissure. Although it is horribly painful, it is not a dangerous condition.

-Avoid constipation – this is very important. Keep your faeces soft by eating lots of bran cereals, fruit and vegetables.
- Smear some painkilling gel around the area just before you have your bowels open. You can buy a suitable painkilling gel from a pharmacy; they are sometimes labelled ‘for piles’ or ‘for haemorrhoids’. It will prevent the anus going into a spasm, which can make the problem worse. Do not use the gel at any other time, or for more than 1 week, because you can easily develop an allergy to its ingredients. If you notice soreness and itching, in addition to the sharp pain, it is quite likely that the gel is responsible.
- Warm baths twice daily can help, if you have the time. Put a handful of ordinary kitchen salt into the bath.


How your doctor can help. You should see your doctor if the problem is not improving after 3 weeks. If you feel anxious about this, look at the section on seeing your doctor about an anal problem. For some treatments, your doctor will need to send you to the ‘rectal clinic’ at your local hospital. Some of the treatments your doctor may use are listed below.
- A gel containing the drug diltiazem, applied twice daily for 6–8 weeks, seems to heal the fissue in 60–70% of cases.
- Glyceryl trinitrate ointment, applied several times a day, is as effective as diltiazem. However, about one in four people using this treatment get a headache as a side effect.

If ditiazem or glyceryl trinitrate do not work for you:

- Injections of botulinum toxin (Botox) into the muscle of the anus are a possibility. Botulinum toxin prevents spasm of the muscle, because it blocks transmission of nerve impulses to the muscle. Healing rates are about 69–90%.
- The most common treatment (before diltiazem gel and glyceryl trinitrate ointment came on the scene) used to be a small operation under a general anaesthetic. The operation is designed to overcome the spasm of the anal muscle. The pain relief is dramatic and instantaneous. In the past, this operation could leave you less able to control wind, but a new type of operation (tailored sphincterotomy) does not have this drawback (British Journal of Surgery 2005;92:403–8).

Unfortunately, the fissure comes back within 3–4 months in about one-third of people treated with glyceryl trinitrate or diltiazem, and in about 40% of people after treatment with botulinum toxin. Surgery seems to be the best long-term cure and is effective in about 90% of cases.
Herpesvirus infection
A herpesvirus infection can produce a pain similar to an anal fissure. Herpes can infect the anal area, either spread by the hands from a cold sore on the face, or transmitted as a sexual infection. At the anus, herpes often forms a crack rather than the small ulcers that tend to occur elsewhere. It can occur in individuals who have never had herpes elsewhere. The soreness occurs in episodes, each lasting for a few days. A genitourinary medicine clinic will be able to take a swab to check for the virus if you visit the clinic as soon as an episode starts; if you are worried about the thought of visiting a clinic, take a look at the section on visiting the clinic.

Abscess
An abscess close to the anus produces a throbbing pain that worsens over a few days, and is usually bad enough to disturb your sleep. You may be able to feel a tender swelling in the skin beside the anus, or the abscess may be hidden inside. This is unlikely to go away on its own; it needs to be lanced by a doctor.
Proctalgia fugax
Proctalgia fugax is a severe, cramp-like pain, deep in the anal canal. It usually lasts for a few seconds or minutes, but can sometimes last for up to half an hour. Between attacks there is no pain at all. Most sufferers have only 5 or 6 attacks a year. You may feel a need to defecate urgently, but nothing happens. It may even make you feel dizzy, or give you a headache. It occurs in both men and women. The pain often wakes sufferers at night, and men may have an erection at the same time. Some men experience it after sex. It is a mysterious condition; no one knows what causes it, but it is probably a spasm of the rectal or pelvic floor muscles and does not mean that you have anything seriously wrong. There are various methods of relieving the pain.
-Try putting pressure on the perineum (the area between the back passage and the vagina or base of the penis) by sitting on the edge of your bath or on a tennis ball.
- Sit in hot water or, alternatively, apply some ice.
- Two paracetamol tablets and a hot drink may give some relief.

The problem with medications for proctalgia fugax is that the episode is likely to be over before the drugs become active, but medication might be worth trying if your attacks last a long time. The usual treatment is the asthma drug salbutamol, inhaled from a puffer at the start of the attack . This is only available on prescription, for which you would need to see your doctor.

1/10/11

White Sands Missile Range: Cancer diagnosis spurs trip of a lifetime

WHITE SANDS MISSILE RANGE - From snowboarding with Shaun White to receiving a video message from Lance Armstrong, Ian Lockwood, 17, can say he has experienced many lasting memories.
These events would be considered life-changing to anyone else, but for Ian, it was when the high school football star learned he had brain cancer in July 2009 and again in September 2010 when after being in remission he learned he had a new growth of cancer and that surgery was no longer an option.
For most people the news would be devastating, but for Ian it was an opportunity to see and do what he had always dreamt of. On Dec. 15 Ian and his father traveled from Pensacola, Fla. to White Sands Missile Range for an aerial tour of the testing site and the White Sands National Monument.
"I've had doctors tell me how long I had to live...and how bad my cancer was, but I just kind of put it past me and live each day for what I have now," Ian said, in a previous TV interview.
Ian's father, Kenneth Lockwood, the director of engineering for the Long Range Systems Division at Eglin Air Force Base, Fla., requested to bring his son along on a mission at WSMR so that he could see the mission with his son.
The mission was a test of a continuing assessment of the Joint-Air-to-Surface Missile. Though the test had to be cancelled due to severe winds, Ian and his father were still able to get an aerial and ground tour of the testing site and the White Sands Monument, known for its pure white sand dunes.Before visiting the testing site, Ian received a brief tour of WSMR and its facilities. The tour included a visit to the new 2nd Engineer Battalion Headquarters and the Battalion's Motor Pool area, where Ian was able to test Army equipment, under supervision.
"He looked like he was having a really great time, it felt good to spend some time with him and give back to the community," said Staff Sgt. Michael Anderson, Operations Sergeant for the 573rd Clearance Co.
Under supervision, Ian searched for Improvised Explosive Devices in a Buffalo vehicle, using the vehicle's Interrogation Arm. Ian was also given a brief overview on the weapons used for daily combat and their functions.
"I'd have to say that was my favorite part of the day," Ian said. "It was pretty cool."
After receiving an extensive tour of the Navy facilities, and lunch at the Frontier Club, Ian received a tour of Cherokee, the Air Traffic Control facility located inside Cox Range Control Center.
Prior to his flight, Ian met with the Installation Commander, Brig. Gen. John S. Regan, and Test Center Director, William Ellis. Each awarded Ian a commemorative coin. Helicopter Pilot Jon D. Edwards and Co-Pilot, Randall Gyllespie, presented Ian with an official Army Air Division Pilot Suit, with his nametag stitched on. The flight, which only a handful of people get to take, was Ian's first helicopter ride.
"I just want to the thank everyone. I know how much it took to put all of this together. I really appreciate it," Kenneth said.
After getting a bird's eye view of the testing site, Ian, his father, and JASSM Test Director, Kenneth Bandy, toured the area on foot, looking for pieces of metal from previous tests. For Ian it was an opportunity to get a bigger piece of metal than his father had brought home for one of his siblings following a test. Both Kenneth and Bandy explained the logistics of the site and how tests are usually conducted, and what it takes to capture it on film.
"I had always wanted to come out here, but I never thought I'd get the opportunity to," Ian said.
Back home, Ian is class president and co-captain of his high school football team, the Raiders. Ian played his last game Oct. 8 after being informed he would need to undergo double treatments since surgery would not be enough. In his last game of the season Ian scored two touchdowns and his team won by twenty nine points. The Raiders have had their best season in school history, and according to the area media outlets, attribute their success to Ian's spirit in the face of adversity.
"We're all a real close group of guys. With my situation, I've made them realize what they had," Ian said. "They wanted to do good because they knew I might not be able to play next season."
Ian was invited to the Outback Bowl as the guest of honor. He said he would visit his family in New York after the Bowl and then go skiing in Colorado. Ian said his plans are to finish high school and continue to study either communication or nursing. Ian recently received an acceptance letter to Florida State University.

Brain cancer: a ray of hope

As we start this year, we would like to bring you up to speed on our recent progress in the discovery of new medications to treat brain tumours. Medical research is a long and trying process and each success must be celebrated. We think that being able to share this good news with you is the best present we could offer for the start of 2011.

The brain is without a doubt the most protected organ in the human body. On the outside, the skull can counter the majority of blows that could damage cerebral tissue, which could affect intelligence and basic physiological functions (heartbeat, breathing, etc.). In addition to this physical protection, the brain is also heavily protected internally with the help of an impenetrable barrier that controls access of blood products to the brain. Called the blood-brain barrier (BBB), this defense system plays an essential role in the maintenance of cerebral homeostasis by choosing which nutrients are needed to keep the brain healthy (glucose, amino acids and some hormones, among others), all the while preventing access to most unknown and potentially toxic substances.

Unfortunately, the BBB also prevents the entry of most medications and is therefore also a major obstacle in treating most diseases affecting the brain, particularly tumours.

CROSSING THE BARRIER

For the last 20 years, our lab has been working to solve the mysteries of the BBB in order to develop new therapeutic approaches that could cross the barrier, while also improving the effectiveness of brain cancer treatments. This research has allowed us to discover that certain peptides (derived from proteins) were able to interact with the receptors found on the surface of the barrier and, consequently, cross through it.

This is a major observation, as it allows us to see that these peptides could be used to transport cancer-fighting medications through the barrier, allowing them to directly act on brain tumours. Such an approach could revolutionize treatments for these cancers as, despite the major advancements in neurology in recent years, brain tumours remain extremely difficult to treat and survival over 11 months remains unlikely.

ENCOURAGING RESULTS

The therapeutic potential of this strategy is shown in the results obtained with a new medication that we have created, GRN1005, a derivative of the cancer-fighting agent taxol, developed by Angiochem. After demonstrating that this compound was active amongst animals, GRN1005 was the subject of two distinct clinical studies designed to determine the maximum tolerable dose, toxicity potential and in order to see preliminary signs of the drug's effectiveness.

Amongst patients who underwent intense treatments to fight solid tumours that had metastasized to the brain, as well as patients with brain tumours (glioblastoma), the studies showed that GRN1005 had significant therapeutic potential.

The medication is active against metastases coming from various types of tumours, including lung cancer, breast cancer and ovarian cancer. Through the first phase of the clinical study, the rate of response for the patients who had received the maximum tolerable dose of GRN1005 was 42%.

More interesting still, certain patients suffering from glioblastomas saw their tumours completely eradicated.

NEW MEDICATIONS?

A deal worth more than $35 million was recently signed with American company Geron to further develop this promising new treatment. Phase 2 of the clinical study will begin this year, using patients with metastases to the brain stemming from lung or breast cancer. If the results of these studies continue to be positive, the medication could be commercialized quickly for use with a larger number of patients suffering from brain tumours.

The ultimate goal of medical research remains the discovery of new ways to improve the population's health, particularly amongst the sick. As the Director of Neurosurgery at the University of Montreal's Hospital Centre and head of Prevention and Chair of Prevention and Treatment of Cancer at the Université du Québec à Montréal, I would like to express our delight in seeing a new family of medications joining the fight against brain cancer reach advanced clinical trial phases. It is our way of wishing you a Happy New Year!

1/8/11

Scientists pinpoint deadly brain tumour's origin

A team of researchers have discovered the type of cell that is at the origin of brain tumours known as oligodendrogliomas, which are a type of glioma - a category that defines the most common type of malignant brain tumour.
Investigators found that the tumor originates in and spreads through cells known as glial progenitor cells - cells that are often referred to as "daughter" cells of stem cells.
The work comes at a time when many researchers are actively investigating the role that stem cells, which have gone awry, play in causing cancer. For scientists trying to create new ways to treat brain tumours, knowing whether stem cells or progenitor cells are part of the process is crucial.
"In many ways progenitor cells are controlled by completely different signaling pathways than true stem cells," said Steven Goldman, a University of Rochester neurologist who was part of the study team.
"Knowing which type of cell is involved gives us a clear look at what drug approaches might be useful to try to stop these tumours. Comparing normal progenitor cells to progenitors that give rise to tumors gives us a roadmap to follow as we try to develop new treatments."
The study focuses on oligodendrogliomas, a type of tumor that presents with symptoms much like other brain tumours - headaches, seizures, and cognitive changes. The tumors are treated with a combination of surgery, radiation, and chemotherapy. Oligodendrogliomas at first are less deadly and invasive than most other gliomas. Unfortunately, treatments like surgery typically slow or stop the tumour initially, but it usually returns, often in a much more aggressive form than it was to begin with. The majority of patients with oligodendrogliomas ultimately die from the disease.
In order to identify better treatments for this tumour, researchers need to know what cell type in the brain gives rise to it. Despite abundant clinical experience with this type of cancer, no one had ever defined oligodendroglioma's cell of origin.
To answer this question, the team used a common brain tumour drug, temozolomide, to test several types of cells from both human and mouse tumours. They found that the drug was effective against oligodendroglioma cells and normal glial progenitor cells, and much less effective against either brain stem cells or other brain tumours called astrocytomas.
The work is the latest in a string of findings that progenitor cells are the origin for some brain tumours.

A Childhood Cancer is Different from Adult Cancers

Earlier in mid-December, a study was published reporting that a cancer in children is very different from cancers in adults. Specifically, the study suggests that there are far fewer mutations present in some childhood cancerous tumors than in tumors of adult cancers. This could have significant implications for not only how we approach and treat childhood cancers, but how we treat combat cancers in general.

The study, which was published in the journal Science, was a large collaborative effort involving 20 different medical institutes, cancer centers, and universities in the U.S. that looked at the genetics of a very aggressive brain cancer found primarily in children called medulloblastoma. Although medulloblastoma tumors originate in a certain area of the brain, the cancer can spread through the spinal fluid to other parts of the central nervous system (which includes the brain and spinal cord). Every year, about 1 in 200,000 children under the age of 15 are diagnosed with having medulloblastoma, and although therapies to treat them have improved, still many cannot be cured and often there are severe side-effects from the therapies. It’s a devastating cancer. Medulloblastoma is very rare in people over 40, which most likely has to do with how medulloblastoma comes to be.
Researchers have recently found that childhood medulloblastomas have many fewer mutations than different adult cancers.
Click to enlarge photo

Researchers have recently found that childhood medulloblastomas have many fewer mutations than different adult cancers.

Although its identity has been hotly debated in recent years, it’s now thought that medulloblastoma is created from cells that were primitive neural stem cells. Such neural stem cells are normally present during fetal development, but in patients with medulloblastoma, these stem cells have mutations in their DNA that led to the formation of a highly malignant tumor.

The study To test whether, and how, medulloblastoma in children is different from adult cancers, the researchers looked at the DNA from 22 different medulloblastoma samples from children as well as DNA from several different adult cancers (including pancreatic, glioblastoma, colorectal, and breast cancer tumors). The DNA sequences of all known genes were analyzed both in tumors and in normal, non-cancerous tissues taken from the same patients. In this way, the scientists could determine which genes were different, or mutated, in the tumors compared to healthy tissue. This is commonly done when studying cancer, and identifying which genes are mutated helps us better understand what causes cancer.

Far fewer mutations in a childhood cancer than adult cancers Interestingly, the scientists found that the medulloblastoma tumors in children had about five to ten times fewer mutated genes than several different adult tumors studied (which were not medulloblastomas). However, while the childhood tumors had many fewer mutations, over one third were mutations known to disrupt gene function, which is a much higher percentage than in the adult tumors analyzed. This may simply be because the adult cancers are in older tissues, and these tissues have had a longer opportunity to gain random mutations over time, but it’s difficult to tell. Either way, this shows that childhood cancer is very different from adult cancers.

The likely suspects How can we use our knowledge of these differences between childhood and adult cancers to help us better combat cancer? To answer this question, it’s important to look at exactly what the researchers found, which comes down to some very specific genes that probably play central roles in causing cancer.

Some of the genes commonly mutated in the medulloblastoma tumors belong to two different groups of well-studied genes, the Hedgehog and Wnt gene groups. (Many genes have somewhat whimsical names, mostly due to early studies in fruit flies; the Hedgehog genes, for example, don’t have anything to do with the hedgehog animal.) These groups of genes are thought to primarily function during embryonic development and during some cancers. Specifically, the mutation of some Hedgehog and Wnt genes has previously been shown to correlate with the occurrence of different cancers, including medulloblastoma.

This recent study not only confirmed previous studies linking Hedgehog and Wnt genes to medulloblastoma, but the researchers also discovered that other genes, including ones not previously suspected, may also be involved in causing medulloblastoma. These newly suspected genes belong to the histone-lysine N-methyltransferase gene family, whose members actually control whether many other genes are active or not. Consequently, by having mutations in these identified genes (which are specifically the histone-lysine N-methyltransferase genes MLL2 and MLL3), the tumors have changed the function of many other genes, causing a kind of snowball effect.

The researchers found that MLL2 or MLL3 were mutated and consequently no longer active in 16 percent of the medulloblastoma tumors they looked at. This suggests that MLL2 and MLL3 normally play important roles in preventing the occurrence of medulloblastoma—that they are what are called “tumor suppressor genes”. Additionally, it’s known that these genes are also very important during normal brain development, which again emphasizes how this childhood cancer is different from adult cancers; the childhood cancer is almost certainly the result of something going wrong during development.

Contributing to how we view cancer While the most significant finding in this study was how many fewer mutations are present in a primarily childhood cancer, medulloblastoma, compared to adult cancers, understanding the specific genes revealed to be key to causing medulloblastoma will not only change how we view and treat childhood cancer, but will also help us better understand which genes are key players in causing cancer in general. It’s becoming clearer that childhood cancer, as shown here with medulloblastoma, is due to problems that manifest during fetal development.

Additionally, the family of genes that MLL2 and MLL3 belong to has already been suspected of being involved in the occurrence of many other types of cancer, and this most recent finding with medulloblastoma warrants further investigation of this gene family and its connections to cancer.

Lastly, because this childhood cancer has many fewer mutated genes than adult cancers, it gives researchers a few genes to really focus on; the genes mutated in medulloblastomas may be the few, most essential genes responsible for this kind of cancer and most likely other cancers. Although applying these findings to other cancers may be difficult; in this study, childhood medulloblastomas were not compared to adult medulloblastomas (which are very rare, but may reveal additional differences), and clearly this study has shown that not all cancers are alike.

Fighting cancer with gene therapy To date, over 80 cancers have had all of their known genes analyzed for apparent mutations. As we better understand which genes are most important for causing, and preventing, cancer, researchers can not only develop better methods for identifying the type of cancer a patient has, but can also develop better therapies to target these specific genes. For example, in medulloblastoma, thanks to this recent study, the development of drugs that target the genes MLL2 and MLL3 is a very appealing prospect, but a better understanding of these genes (and improvements in gene therapies in general) is needed before such therapies can become a reality.

For more on medulloblastoma and recent comparisons of this childhood cancer to adult cancers, see the original article published in the journal Science in December 2010, read coverage of this article by The Scientist, or see Wikipedia’s article “Medulloblastoma.”

How a little boy with cancer became a hero


At first, Mary Lamantia couldn't quite place the teen who reached out to her just before the holidays, but when he declared on a Facebook page that her son Ryan was his hero, she was determined to remember.

Walter Wetzel had met Ryan nearly eight years ago in a hospital waiting room. Both were very sick — Ryan with brain cancer, Walter with leukemia. Walter, then 9, began making silly faces at the little boy sitting across from him.

Soon after, Ryan, who was 3 at the time, made his way into Walter's chemo room and chatted about going home to change into his Teenage Mutant Ninja Turtles costume and ride his Big Wheel to his cousin Catlyn's house.

It was the start of a friendship that would blossom during other hospital encounters over the next few months, but they would lose touch after Ryan transferred hospitals. Lamantia's memory of Walter faded during the years that followed, a time of needles, MRIs, chemo and radiation.

Hampton U's cancer center marks a milestone

Wednesday was a little bit of both. The family from London, Ohio, just west of Columbus, celebrated the last day of Reagyn's treatment at the newly opened Hampton University Proton Therapy Institute. But saying goodbye to the staff they saw almost daily wasn't easy.

"I will miss my doctors and everybody here," Reagyn said.

"I'm going to start to crying," mother Carol Semler said. "It's bittersweet. I'm excited to go home, but we've been here so long. This is like our family now."

The last day of treatment included gifts donated by local businesses and a serenade by radiation therapist Mike Freeman, who dressed up like Alan Jackson and sang "Gone Country."

Reagyn was the first pediatric patient to be treated at the cancer treatment center, which opened in August. A 200-ton cyclotron spins protons, which are found inside the nucleus of atoms, at 60 percent of the speed of light and beams them into treatment rooms.

The center initially treated prostate cancer patients but is now branching out to treat other tumors. Radiation oncologist Dr. Allan Thornton expects they'll treat pediatric patients from all over the world.

Reagyn was diagnosed with a brain stem glioma, a type of central nervous system tumor, in 2005. Every year, about 3,800 central nervous system tumors are diagnosed in children, making them the second most common childhood tumor after leukemia. Brain tumors account for about 21 percent of all childhood cancer up to age 14, according to the American Cancer Society.

Her parents knew something was wrong when Reagyn kept exhibiting flu-like symptoms and didn't have any energy. Then she lost muscle control in her eyes. Her parents took her to eye specialist, who saw fluid buildup and called for an immediate MRI. The scan detected the tumor.

Reagyn was admitted to the hospital that night and underwent emergency surgery to put a shunt in to drain fluid from her brain. The tumor was inoperable because of its location — in the part of the brain that controls balance, heart rate, swallowing and breathing, Thornton said.

The shunt relieved the pressure and swelling and doctors closely monitored the tumor. In 2008, it started growing again, and the family met Thornton, who was then with the Midwest Proton Radiotherapy Institute in Indiana. But it stopped growing and they shelved treatment.

In September, it started growing again. Reagyn came to Hampton Roads Nov. 9 to undergo treatment five times a week.

Reagyn also went to Children's Hospital of The King's Daughters in Norfolk for chemotherapy. Chemo will continue for about a year soon after she returns to Ohio.

Because there are only eight proton therapy treatment centers operating in the U.S., few other children's hospitals have this kind of resource nearby, said Dr. Eric Lowe, a CHKD pediatric oncologist

Until now, CHKD had to refer patients to proton therapy centers elsewhere.

"It's absolutely huge," Lowe said. "The advantage is it hopefully gives you a more focused radiation rather than having the collateral damage. Where that matters is in small spaces. Who has small spaces? Kids."

Reagyn's mom, dad and little sister were around for her treatment. Her three other siblings stayed in Ohio with grandparents.

"She's doing great, other than being a little tired and her appetite decreasing. She hasn't had any of the other symptoms she could have had," Carol Semler said. "You can look at her and not even know she has it."

Reagyn's mother remembers what it was like to hear her daughter had an inoperable brain tumor.

"It felt like someone just stuck a knife in me. You go to the hospital and you see these kids with life-threatening illnesses and you never think it's going to be you," Carol Semler said.

"I was scared," Reagyn said of first learning she had a brain tumor. Now, "it's not as scary as it was when I first heard."

She's looking forward to hanging out with friends and seeing her cat, Angel, which her parents got her when she first got sick.

Reagyn likes gymnastics, cheerleading and swimming, but Thornton told her to take it easy the next few months. He'll work with her doctors in Ohio and monitor her progress.

"We would expect the tumor to shrink over the next six months until it is only scar tissue," Thornton said.

Though the tumor was inoperable, it was a grade that was treatable, Thornton said.

"She has a very, very good prognosis," he said.

Brain cancer: a ray of hope

As we start this year, we would like to bring you up to speed on our recent progress in the discovery of new medications to treat brain tumours. Medical research is a long and trying process and each success must be celebrated. We think that being able to share this good news with you is the best present we could offer for the start of 2011.

The brain is without a doubt the most protected organ in the human body. On the outside, the skull can counter the majority of blows that could damage cerebral tissue, which could affect intelligence and basic physiological functions (heartbeat, breathing, etc.). In addition to this physical protection, the brain is also heavily protected internally with the help of an impenetrable barrier that controls access of blood products to the brain. Called the blood-brain barrier (BBB), this defense system plays an essential role in the maintenance of cerebral homeostasis by choosing which nutrients are needed to keep the brain healthy (glucose, amino acids and some hormones, among others), all the while preventing access to most unknown and potentially toxic substances.

Unfortunately, the BBB also prevents the entry of most medications and is therefore also a major obstacle in treating most diseases affecting the brain, particularly tumours.

CROSSING THE BARRIER

For the last 20 years, our lab has been working to solve the mysteries of the BBB in order to develop new therapeutic approaches that could cross the barrier, while also improving the effectiveness of brain cancer treatments. This research has allowed us to discover that certain peptides (derived from proteins) were able to interact with the receptors found on the surface of the barrier and, consequently, cross through it.

This is a major observation, as it allows us to see that these peptides could be used to transport cancer- fighting medications through the barrier, allowing them to directly act on brain tumours. Such an approach could revolutionize treatments for these cancers as, despite the major advancements in neurology in recent years, brain tumours remain extremely difficult to treat and survival over 11 months remains unlikely.

ENCOURAGING RESULTS

The therapeutic potential of this strategy is shown in the results obtained with a new medication that we have created, GRN1005, a derivative of the cancer-fighting agent taxol, developed by Angiochem. After demonstrating that this compound was active amongst animals, GRN1005 was the subject of two distinct clinical studies designed to determine the maximum tolerable dose, toxicity potential and in order to see preliminary signs of the drug's effectiveness.

Amongst patients who underwent intense treat-m ents to fight solid tumours that had metastasized to the brain, as well as patients with brain tumours (glioblastoma), the studies showed that GRN1005 had significant therapeutic potential.

The medication is active against metastases coming from various types of tumours, including lung cancer, breast cancer and ovarian cancer. Through the first phase of the clinical study, the rate of response for the patients who had received the maximum tolerable dose of GRN1005 was 42%.

More interesting still, certain patients suffering from glioblastomas saw their tumours completely eradicated.


NEW MEDICATIONS?

A deal worth more than $35 million was recently signed with American company Geron to further develop this promising new treatment. Phase 2 of the clinical study will begin this year, using patients with metastases to the brain stemming from lung or breast cancer.

The ultimate goal of medical research remains the discovery of new ways to improve the population's health, particularly amongst the sick. As the Director of Neurosurgery at the University of Montreal's Hospital Centre and head of Prevention and Chair of Prevention and Treatment of Cancer at the Université du Québec à Montréal, I would like to express our delight in seeing a new family of medications joining the fight against brain cancer reach advanced clinical trial phases. It is our way of wishing you a Happy New Year!

Dr. Richard Beliveau holds the chair in cancer prevention and treatment at the University of Quebec in Montreal.

Washington Week: GOP Gets Started on Reform Repeal

WASHINGTON -- The week began with the 112th Congress being sworn in and its new GOP members vowing to begin the process to repeal the Democrats' healthcare reform law. By the end of the week, Republicans were one procedural step closer to that goal.

Eric Cantor (R-Va.), the Majority Leader of the new Republican-controlled House of Representatives, introduced the two-page "Repealing the Job-Killing Health Care Law Act" early in the week, which would repeal the Affordable Care Act (ACA) and all other laws changed by the reform law "as if such Act had never been enacted." It would also repeal the healthcare provisions in the companion bill, known as the reconciliation act, that fixed some parts of the main law.

The House Rules Committee, which determines rules to govern floor debate of bills, approved a rule to govern floor debate on Thursday, and on Friday, the House voted 236 to 181 -- with a handful of Democrats joining Republicans in opposition to the reform law -- to approve the rule.

The House is expected to vote on the repeal bill on Wednesday, where it is expected to pass easily. Senate passage, however, is unlikely given the Democrats' control of that chamber. Even if it did clear the Senate, President Obama has said he would veto it.

Sharfstein to Depart FDA; Emanuel to Leave White House

Joshua Sharfstein, MD, a pediatrician and the FDA's deputy commissioner, is leaving the agency to become the Secretary of Health and Mental Hygiene for the state of Maryland.

CQ HealthBeat reported the news early in the week, and Sharfstein and Maryland Gov. Martin O'Malley confirmed it on Wednesday.

During his time at the FDA, Sharfstein worked toward giving FDA regulatory control of tobacco products, which culminated in a 2009 bill that did just that.

Sharfstein, along with FDA Commissioner Margaret Hamburg, MD, has also played a role in turning the FDA into a more aggressive regulator. Under their dual leadership, the agency began issuing more warning letters to drug, device, and food companies and also began pressing criminal charges against drug companies.

Later in the week, Kaiser Health News reported that Ezekiel Emanuel, MD, a healthcare adviser in the White House Office of Managment and Budget, left his post to return to his old job at the National Institutes of Heath. Health policy experts told Kaiser Health News that his departure is no surprise and that he was always just "on detail" to the White House.

Emanuel's brother, Rahm, recently stepped down as President Obama's chief of staff to run for mayor of Chicago.

End-of-Life Provision Dropped

The Obama administration dropped a regulation specifying that Medicare will pay for end-of-life counseling, just days after the rule went into effect.

The rule, which went into effect on Jan. 1, said that as part of an annual wellness visit, Medicare will pay for elective discussions about end-of-life plans, which can, in turn, be used to prepare an advance directive stating what treatments a patient would want and treatments they would not want.

A similar provision was in the original healthcare reform law but was dropped after it ignited a firestorm of controversy centered on so-called "death panels."

The current end-of-life counseling provision was included in a final rule setting Medicare payment rates for annual wellness visits.

However, because the provision was not included in the proposed regulation, which was published in July, the Obama administration felt that "the opportunity for the public to comment on it was too limited," an administration official told MedPage Today. Hence, the decision to drop it, which was announced on Jan. 5.

Still, "nothing about this change should inhibit patients and physicians from having this kind of discussion," the official added.

Slowdown in Healthcare Spending Growth

Spending on healthcare grew at its slowest rate in 50 years, mostly because of recession-fueled cutbacks by businesses, governments, and households, according to a Centers for Medicare and Medicaid Services report.

The growth in healthcare spending has slowed every year since 2002, but 2009's was particularly pronounced -- growing at a rate of 4%, down from 4.7% in 2008, according to the authors of the agency's annual health spending report, published in Health Affairs.

Despite the slowdown in the growth rate, the U.S. still spends $2.5 trillion on healthcare, or $8,086 per person.

The newest figures do not take into account any changes made by the ACA, which was signed into law in 2010.

Food Safety Bill Signed into Law

President Obama signed a food safety bill into law Tuesday that is expected to bring the most sweeping changes to the nation's food system in at least the past 20 years.

The $1.4 billion bill aims to improve prevention of food contamination, allows the FDA to issue mandatory recalls if businesses do not voluntarily recall harmful foods, requires grocery stores and other food sellers to notify consumers if they have sold food that has been recalled, and improves disease surveillance so outbreaks can be discovered earlier.

While the law authorizes $1.4 billion in new spending over the next five years, the money will still have to be appropriated by Congress.

FDA Issues New Tobacco Regulation

New FDA guidance issued Wednesday requires tobacco companies to prove any product introduced or changed after Feb. 15, 2007 is "substantially equivalent" to products on the market before that date.

Equivalency applications are due by March 22 of this year. After that date, tobacco manufacturers will have to submit a new product application and get a marketing order before introducing a new product.

Wednesday's announcement represented the latest step in the FDA's implementation of the Tobacco Control Act of 2009.

During a telephone briefing, Lawrence Deyton, MD, director of the agency's Center for Tobacco Products, explained that "until now, tobacco products have been only mass-consumed products in which users don't know what they're consuming," because producers can alter the contents or ingredients "without anyone knowing."

FDA Approves ESC Trial for Macular Degeneration

A clinical trial of a human embryonic stem cell (ESC) therapy for age-related macular degeneration has been approved by the FDA, making it the first such study for a common disease.

Advanced Cell Technology, based in Marlborough, Mass., said it had received the agency's clearance to begin a phase I/II trial in 12 patients with age-related "dry" macular degeneration, which affects about 10 to 15 million people in the U.S.

Patients will receive implants with retinal pigment epithelial (RPE) cells derived from the company's ESC lines.

Other clinical trials of ESC-based therapies are under way or approved to begin, but they have been for comparatively rare conditions: certain severe spinal cord injuries and Stargardt's macular dystrophy, a childhood-onset form of blindness that resembles age-related macular degeneration.