Category Archives: Treatments

Stem Cell Treatment for MS: Can’t We Move Faster?

(This first appeared as my column in www.multiplesclerosisnewstoday.com)

There is some good news about stem cell therapy and some that’s not so great.

A just-published study concludes that one form of human stem cell therapy is more effective at treating multiple sclerosis than the best of the MS medications being used currently.

The not-so-good news is that approval of this therapy in the U.S. still seems to be a long way off.

The treatment is known as high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT). The procedure, more widely known as HSCT, aims to suppress active disease and to prevent further disability by removing disease-causing cells and resetting the immune system. During the procedure doctors collect a patient’s blood-forming stem cells, give the patient high-dose chemotherapy to deplete the immune system, and then return the patient’s own stem cells to rebuild the immune system.

Stem cell replacement is better than MS drugs

The five-year study, that was published in the February issue of Neurology, shows that HDIT/HCT can result in sustained remission of relapsing-remitting MS. Five years after receiving HDIT/HCT, 69% of the trial participants had no progression of disability, relapse of MS symptoms or new brain lesions. And some of the patients had some of their symptoms improve. This occurred without taking any MS medications after the stem cells were replaced.

“These extended findings suggest that one-time treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications for people with a certain type of MS,” said Anthony S. Fauci, MD, the Director of the National Institute of Allergy and Infectious Diseases.

More study needed

But, here’s the not-so-good news. In a press release Fauci continued: “These encouraging results support the development of a large, randomized trial to directly compare HDIT/HCT to standard of care for this often-debilitating disease.” So, Dr. Fauci, how many more years will that take?  Granted, the study that was just completed involved only 24 volunteers, all of whom had aggressive, relapsing-remitting MS. We know the treatment carries some risks and that many participants in the study experienced some serious, but expected, side effects, such as infections. But, five years after receiving HDIT/HCT treatment, most trial participants remained in remission and their MS had stabilized. In addition, some participants showed improvements, such as recovery of mobility or other physical capabilities. It seems as if results such as that should shift research into high gear.

Is research moving fast enough?

Why, then, do investigators seem not to have greater urgency in making HDIT/HCT treatment available in the U.S.?  “If these findings are confirmed in larger studies, HDIT/HCT may become a potential therapeutic option for patients with active relapsing-remitting MS, particularly those who do not respond to existing therapies,” said Daniel Rotrosen, MD, director of NIAID’s Division of Allergy, Immunology and Transplantation.  

To me, that sounds like several more years of study before researchers will be ready to ask the U.S. Food and Drug Administration to approve this treatment. I’m not a doctor. I’m not a scientist. I’m just an MS patient who’s anxious to halt the progression of my disease and to walk better. Do I need to wait another five years or more while “a large, randomized trial” is conducted?

Just venting, I guess. But scientists have been studying stem cell treatments for years and it sure seems as if we’re still crawling when we should be cruising.

Pills, shots or infusions for your MS?

[This first appeared as my column on http://www.multiplesclerosisnewstoday.com]

Oral multiple sclerosis meds appear, more and more, to be the first choice of patients who are just beginning to receive an MS treatment. A recent report by the independent marketing research firm Spherix Global Insights shows that oral disease-modifying therapies captured a significantly higher share of the market at the end of 2016 compared to 2015.  Tecfidera was at the top of the pills list with Aubagio gaining ground. But, final FDA approval of the infusion drug Ocrevus (ocrelizumab) could be a game-changer in the pill-shot-infusion decision.

Ocrevus

Neurologists and many MS patients are very familiar with Ocrevus. There’s a lot of chatter about it in online MS gathering spots, and many doctors report they intend to use it as soon as it is approved. And, even though most of the “buzz” is about prescribing Ocrevus for PPMS patients, it’s expected that a lot of doctors also are looking at it for those with RRMS. It could push ahead of other infusions, such as Tysabri and Lemtrada. as well as oral meds such as Tecfidera and Aubagio. Spherix reports more than one third of the neurologists it surveyed said they would have been likely to have started patients on Ocrevus, rather than on Tysabri, had it been available.

Weighing the treatment options

What drives these drug decisions? According to a Spherix report RealWorld Dynamix™: DMT New Starts in Multiple Sclerosis, the drivers include clinical considerations such as safety and efficacy, as well as non-clinical concerns including patient requests and the policies of the insurance coverage of those patients. Interestingly, it appears that neurologists are becoming more comfortable using high-efficacy DMTs right away for patients with aggressive MS, rather than working up to them from drugs with lower efficacy, but lower risk or cost.

Be involved in the decision

As the patient you should take an active role in your drug selection. A Spherix report released last fall showed that newly diagnosed MS patients are doing their research about DMTs. One in five of them are asking their neurologists about specific MS drugs. Those doctors say that 27% of their patients have a high degree of involvement in choosing the DMT they will use, 43% have moderate involvement, and 30% only low involvement. Unfortunately, more than 50% of the neurologists surveyed say that they, alone, choose the DMT the patient will use. About a third say they present a few choices, and then let the patient decide.  Only 10% of the docs say they present all of the options and then let the patient decide.

How did you decide which DMT to use? Are you thinking about changing?

Lemtrada: Is this MS Drug HSCT “Lite?”

(This is an updated version of my column that appeared earlier this month on www.multiplesclerosisnewstoday.com)

My neurologist calls Lemtrada “HSCT lite.”  She says that not only is the drug able to reduce exacerbations and limit the overall progression of Multiple Sclerosis, it’s actually reversed some symptoms in some of her patients.   I guess I’m going to find out if she’s right about Lemtrada because I’m getting ready to start using it.

Lemtrada Targets MS-related Cells

Lemtrada, which carries the generic name alemtuzumab, was originally approved by the Federal Drug Administration, at a much higher dose, to treat B-cell chronic lymphocytic leukemia.  The drug is a humanized monoclonal antibody which seeks out and destroys specific immune cells that carry a protein called CD52.  These are believed to be the white blood cells that attack the central nervous system in people with MS.  After those cells are removed, the body manufactures new white blood cells that don’t carry the MS-related protein.  Thus, the similarity to stem cell replacement therapy.  That therapy uses chemotherapy to destroy most of the body’s immune system cells. Then, fresh stem cells are infused into the body which, over time, create new white blood cells.

Unlike other Disease Modifying Therapies (DMTs), which require injections every few days or monthly infusions, patients receiving Lemtrada are treated once a year, for two years.   The first year the drug is infused daily for five days in a row, on an out-patient basis. Each infusion takes about four hours and the patient is watched for another two hours afterwards.  A year later that process is repeated, but for only three days.  And that’s it. (In a few cases patients have undergone a third round of infusions).

lemtrada-graphic

In a study, conducted by researchers at Queen Mary University of London and published in the October, 2016 issue of Neurology, nearly half the patients treated with alemtuzumab (Lemtrada) showed improvements in all 7 functions on the EDSS functional scale two years after treatment began. (EDSS functions include things such as limb movement, numbness, speech and bladder and bowel functions).  Those results, the researchers write, “suggest that such disabilities may often be reversible (at least partially) in patients with active RRMS if they receive suitable therapy, irrespective of the type of baseline functional deficit.”

My own neurologist tells me that one of her wheelchair-bound patients has regained some walking ability.  There are stories about similar mobility improvements by patients who have commented on the Facebook page “Lemtrada for MS Treatment.”  And, there are also stories there from patients who saw no improvement.  It’s worth a look by anyone considering using this drug.

There are Risks

This treatment is not without serous risks.  They include autoimmune problems that could result in severe bleeding or kidney problems, serious infusion reactions and an increased chance of getting certain types of cancer.  Patients receiving Lemtrada are required to have their blood and urine tested monthly and they’re monitored very carefully for as long as four years.

Some patients are also reporting side effects during and after the infusion.  These can include fatigue, headaches, hives, a fever nausea and leg pain.  Many of these are being reported on the “Lemtrada for MS Treatment” Facebook page

Anyone considering Lemtrada will have to have a discussion with their neurologist and then determine whether the possible benefits of this treatment outweigh the risks.  For me, the balance falls on the side of the potential benefits.  My first series of infusions is scheduled for the first week in December and I’m sure that I’ll be writing at least one column that week with an IV stuck in my vein.  Stay tuned.

 

Whole body cryotherapy for multiple sclerosis?

Whole body cryotherapy is one of the latest “treatments” claiming to help multiple sclerosis patients.

Those who sell WBC machines and who operate WBC “spas” claim that it can also help a range of other ailments, from asthma to rheumatoid arthritis.  They say it can improve blood circulation, increase metabolism, improve recovery and soreness after workouts and relieve joint and body pain. That’s a lot of problems, and the Food and Drug Administration warns users: it has no evidence that whole body cryotherapy treats, or helps, anything!  And the FDA has recently sent out a specific reminder that this “treatment” isn’t FDA-approved.

“Based on purported health benefits seen in many promotions for cryotherapy spas, consumers may incorrectly believe that the FDA has cleared or approved WBC devices as safe and effective to treat medical conditions,” says Dr. Aron Yustein, of the FDA’s Center for Devices and Radiological Health. “That is not the case.”

Cryotherapy uses techniques that can be as simple as using an ice pack to cool a part of your body.  You might have used it to ease your aching back.  Whole body cryotherapy is like cryotherapy on steroids.  It applies super-cold vapors, as cold as minus 300 degrees, over your whole body.  A person might stand in a can-like enclosure, with his or her head outside the can, while being exposed to those super-cold temperatures for two to four minutes.  Or, a group might be exposed while in a room.  Sometimes liquid nitrogen is used to generate the cold vapors.  Sometimes it’s just cold air.

FDA says the risks outweigh any benefits

What actually happens physiologically to your body when it’s super-cooled; to things like your blood pressure, heart rate and metabolism?

“We simply don’t know,” says the FDA’s Dr. Anna Ghambaryan.  But, Dr. Ghambaryan warns, while the healing benefits are unconfirmed the potential risks are apparent.  “Potential hazards include asphyxiation, especially when liquid nitrogen is used for cooling,” says Dr. Ghambaryan. Nitrogen vapors in a closed room lower the amount of oxygen in the room.  That can result in hypoxia, or oxygen deficiency, and that can end up with someone become unconscious.  Other risks include frostbite, burns and eye injury from the extreme temperatures.

If you decide to try whole body cryotherapy to treat your multiple sclerosis the FDA wants you make sure you know that it hasn’t cleared or approved any of these WBC devices for treatment of any medical conditions.

The FDA is also concerned that patients who opt for WBC treatment—especially in place of treatment options with established safety and effectiveness—may experience a lack of improvement or a worsening of their medical conditions.

If you still want to try it, or if you’re already using it, the FDA suggests that you, at least, talk about it with your doctor.